Clinical and Regulatory Catalyst Calendar

Company Ticker Price MktCp(M) Cat-Date Category Product Catalyst Summary Update

Aastrom

Biosciences ASTM $2.49 $96 11/14/11

Pending Pivotal

Phase 3

(REVIVE-CLI)

Clinical Trial

under SPA

Ixmyelocel-T

(bone

marrow,

patient-

derived cell

therapy

product)

In JUL 2011, announced SPA

agmt w/ FDA for design of

pending pivotal P3 trial for pts

w/ no-option critical limb

ischemia (CLI) that is

expected to begin during

4Q11, final 12-month results

from Phase 2b RESTORE-CLI

study will be presented at AHA

mtg on 11/14/11 8/16/11

Aastrom


IMPACT-DCM

Catheter Phase

2 Clinical Trial

Ixmyelocel-T

(bone

marrow,

patient-

derived cell

therapy

product)

Completed pt enrlmt (21) in

DEC 2010 for treatment of

severe heart failure (dilated

cardiomyopathy or DCM) via

direct catheter injection and

expects to report six-month

results during 3Q11,

ClinicalTrials.gov ID

NCT01020968 8/16/11

Aastrom


IMPACT-DCM

Surgical Phase

2 Clinical Trial

Ixmyelocel-T

(bone

marrow,

patient-

derived cell

therapy

product)

In JAN 2011, reported six-

month interim results for pts w/

dilated cardiomyopathy (DCM)

w/ no safety concerns and

positive trends in quality of life

and functional parameters in

treatment group, will present

final 12-month results at heart

failure society mtg on 9/19/11,


NCT00765518 8/16/11

ACADIA

Pharma ACAD $1.31 $69 9/30/12

Phase 3 Clinical

Trial

Pima-

vanserin

(once-daily

oral tablet)

(5-HT2A

receptor

inverse

agonist)

In OCT 2010, regained all

rights to drug w/ ongoing P3

trial that is expected to enroll

170 pts w/ Parkinson’s

disease psychosis,


NCT01174004 w/ results

expected during 3Q12 6/16/11

AcelRx

Pharma ACRX $3.20 $62 12/31/11

Pending Pivotal

Phase 3 Clinical

Trials

ARX-01

(Sufentanil

NanoTab

PCA

System)

On 6/30/11, obtained $20M

secured loan agmt to fund

three planned P3 trials for

ARX-01, expects to begin

enrolling pts in first P3 trial

(post-op pain after abdominal

surgery) during 2H11 and

begin enrolling pts in a second

P3 trial vs. standard of care (IV

PCA morphine) in early 2012

(1Q12 estimate) 7/5/11

Achillion

Pharma ACHN $5.46 $381 12/31/11

Phase I Clinical

Trial

ACH-2684

(pan-

genotypic

HCV

protease

inhibitor)

On 5/25/11, began dosing in

trial to assess safety and anti-

viral activity of drug being

developed for chronic hepatitis

C virus (HCV) infection,

expects to enroll up to 78

healthy volunteers and 40

HCV pts, expects report proof-

of-concept data by year-end

2011 5/25/11

Achillion

Pharma ACHN $5.46 $381 3/31/12

Phase I Clinical

Trial

ACH-2928

(NS5A

inhibitor)

On 7/12/11, began dosing

subjects in P1 trial for

compound being developed

for treatment of chronic

hepatitis C virus (HCV)

infection, expects to enroll 48

healthy volunteers and 20

HCV pts, estimated results

during 1Q12 7/12/11

Achillion

Pharma ACHN $5.46 $381 12/31/11

Phase 2a

Clinical Trial

ACH-1625,

a hepatitis C

virus (HCV)

protease

inhibitor

In MAR 2011, reported top-

line results for once-daily

dosing in combo w/ standard

of care treatment for chronic

hepatitis C (HCV) infection w/

75-81% of pts achieving rapid

virologic response, expects to

begin 12-week segment of trial

during 2Q11 and report viral

response (12-week EVR) data

by year-end 2011 5/25/11

Adeona

Pharma AEN $0.62 $17 12/31/13

Phase 2 Clinical

Trial

Trimesta

(estriol)

As of 8/1/11, 140 of 150 pts

enrolled in ongoing P2 trial in

combo w/ COPAXONE for

Relapsing Remitting Multiple

Sclerosis (RRMS), expects to

complete enrlmt by yr-end

2011, ClinicalTrials.gov ID


expected during 2H13 8/15/11

Advanced

Cell

Technology ACTC $0.16 $256 9/30/13

Phase I/II

Clinical Trial

Retinal

Pigment

Epithelial

(RPE) Cells

In JUL 2011, began dosing

pts in each of two P1/2 trials

for eye conditions (Stargardt's

Macular Dystrophy or SMD

and Dry Age-Related Macular

Degeneration or Dry AMD),

plans to enroll 12 pts in each

trial w/ 12-month primary

endpts, ClinicalTrials.gov IDs

NCT01345006 and

NCT01344993 w/ results for

both trials expected 3Q13 7/15/11

Advaxis ADXS $0.14 $33 3/31/12

Phase 2 Clinical

Trial

ADXS11-

001 (live,

attenuated

Listeria

monocytoge

nes or Lm-

based

immuno-

therapy)

On 7/25/11, announced

revised protocol in ongoing

cervical dysplasia (a pre-

cancerous condition aka CIN)

P2 trial expected to accelerate

enrlmt by up to 5 months in

mid-dose group, expects to

report low-dose trial results in

early 2012 (1Q12 estimate),


NCT01116245 7/25/11

Aegerion

Pharma AEGR $14.50 $305 12/31/11

FDA New Filing

(NDA), MAA /

EMA (Europe)

New Filing

Lomitapide

(AEGR-733)

(Microsomal

Triglyceride

Transfer

Protein or

MTP

Inhibitor)

Expects to file NDA and MAA /

EMA by yr-end 2011 based on

56-week data from pivotal P3

trial for treatment of genetic

disorder resulting in very high

cholesterol levels (HoFH),

expects to submit full 78-week

data as supplement to support

regulatory approval filings 8/17/11

Aeterna

Zentaris AEZS $1.90 $184 12/31/11

FDA New Filing

(NDA)

AEZS-130

(orally active

growth

hormone

stimulating

agent)


pivotal P3 trial under SPA for

oral diagnostic test for adult

growth hormone deficiency

met primary endpt, preparing

for pre-NDA mtg w/ FDA

followed by expected NDA

filing (late 2011-early 2012

estimate) 8/30/11

Aeterna

Zentaris AEZS $1.90 $184 12/31/11

Pivotal Phase 3

(X-PECT)

Clinical Trial

under SPA

Perifosine

(KRX-0401)

(PI3K/Akt

pathway

inhibitor anti-

cancer

agent)

On 8/31/11, North American

licensee KERX announced

indpt data safety monitrg bd

completed interim analysis

and recm’d trial continue as

planned for ongoing pivotal P3

trial in pts w/ metastatic

colorectal cancer, FDA Fast

Track status,



expected by late 2011-early

2012 8/31/11

Aeterna

Zentaris AEZS $1.90 $184 12/31/12

Pivotal Phase 3

Clinical Trial

under SPA

Perifosine

(KRX-0401)

(PI3K/Akt

pathway

inhibitor anti-

cancer

agent)

In DEC 2009, North American

licensee KERX began

relapsed / refractory multiple

myeloma (MM) pivotal trial,



expected during 4Q12, FDA

Orphan Drug and Fast Track

status 7/8/11

Affymax AFFY $4.28 $152 3/27/12

FDA Decision

(NDA)

Peginesatid

e (once-

monthly

anemia

drug)

On 8/11/11 announced

PDUFA decision goal date of

3/27/12 under standard 10-

month review, seeking

approval as once-monthly

anemia treatment for adult pts

w/ chronic kidney disease on

dialysis, received $10M pmt

from partner Takeda for NDA

accptce and due to receive

$50M if approved 8/11/11

Agenus

(formerly

Antigenics) AGEN $0.49 $56 12/31/11

Phase 2 Clinical

Trials

Prophage

(Patient-

Specific

Therapeutic

Cancer

Vaccine)

(Approved in

Russia as

Oncophage

for kidney

cancer)

Single-arm P2 trial for

recurrent brain cancer (GBM)

indicates median survival of

324 days and 11 pts still alive

(four beyond one year) w/


NCT00293423 and results

expected 4Q11,


NCT00905060 for trial in

combo w/ TEMODAR for

newly diagnosed glioma w/

results expected 1Q14 8/17/11

Alexion

Pharma ALXN $59.48 $10,976 10/7/11

FDA Decision

(sBLA) (Priority

Review)

SOLIRIS

(eculizumab

)

On 6/1/11, announced FDA

accepted sBLA filing w/ six-

month priority review for

expected decision by 10/7/11,

seeking expanded approval

for for treatment of pts w/

atypical Hemolytic Uremic

Syndrome (rare blood clotting

disorder) 6/1/11

Alexza

Pharma ALXA $1.22 $88 2/4/12

FDA Decision

(NDA)

ADASUVE

(Staccato

loxapine)

(formerly AZ-

004)

On 8/19/11 announced FDA

accepted NDA resubmission

(in response to CRL issued in

OCT 2010) for six-month

Class 2 review w/ PDUFA

decision goal date of 2/4/12,

seeking aprvl for rapid

(inhaled) treatment of agitation

in adults with schizophrenia or

bipolar disorder, expects

advisory panel mtg to be

scheduled 8/19/11

Alimera

Sciences ALIM $7.18 $225 11/12/11

FDA Decision

(NDA) (Class 2

Resubmission),

MAA / MHRA

(Europe)

Decision

ILUVIEN

(fluocin-

olone

intravitreal

insert)

(sustained

eye drug

delivery

system)

In MAY 2011, announced

NDA resubmission in

response to DEC 2010 CRL,

seeking approval for diabetic

macular edema w/ PDUFA

decision goal date of 11/12/11

under six-month Class 2

review and no advisory panel

mtg to be held, MAA filed for

EU approval in JUL 2010 w/

MHRA (UK) and expected

decision during 1Q12,

licensed from PSDV 8/31/11

Alkermes ALKS $16.43 $1,610 12/31/11

Phase 2 Clinical

Trial, Phase I / II

Clinical Trial

ALKS 33

(oral opioid

modulator)

On 7/7/11, announced P2 trial

for binge eating disorder failed

to meet endpt,


NCT01366001 for P1/2 trial

for cocaine abuse w/ results

expected mid-2012,


NCT01381107 for P1/2 trial in

combo w/ buprenorphine

(ALKS 5461) for depression

w/ results expected 2H11 7/7/11

Alkermes ALKS $16.43 $1,610 6/30/12

Phase 2 Clinical

Trial

ALKS 37

(orally active

peripheral

opioid

antagonist)



expected mid-2012 for P2 trial

in up to 150 adults with opioid-

induced constipation (OIC) 7/7/11

Alkermes ALKS $16.43 $1,610 12/31/11

Phase I / II

Clinical Trial

ALKS 5461

(oral combo

of ALKS 33

+

buprenorphi

ne)

On 6/15/11, began trial for

refractory depression not

controlled by std therapy,

expects to enroll 32 pts and

report top-line results 2H11 6/15/11

Alkermes ALKS $16.43 $1,610 12/31/11

Pending Pivotal

Phase 3 Clinical

Trial

ALKS 9070

(once-

monthly

injected pro-

drug

formulation

that converts

to

aripiprazole)

On 6/30/11 announced

positive results in P1B trail for

pts w/ schizophrenia and

expects to begin pivotal

clinical program by year-end

2011, is a novel once-monthly

formulation of approved drug

ABILIFY 6/30/11

Allon

Therapeutic

s

(NPCUF.PK

) NPC.TO $0.37 $29 12/31/12

Pivotal Phase

2b / 3 Clinical

Trial under SPA

Davunetide

(derived

from neuro-

protective

brain

protein,

ADNP)

As of 8/12/11, has enrolled

approx. 75% of pts in ongoing

pivotal trial for treatment of

progressive degenerative

brain disease (progressive

supranuclear palsy), expects

to complete enrlmt by yr-end

2011and report data about

one year later (4Q12 estimate)


NCT01110720 8/23/11

Alnylam ALNY $6.37 $272 11/20/11

Phase I Clinical

Trial

ALN-TTR01

(systemic

RNA

interference

or RNAi

therapeutic

for

amyloidosis)

In JUL 2010 began dosing in

P1 trial, will enroll up to 28 pts

w/ amyloidosis (condition that

damages peripheral nerves

and heart), expects to present

data at scientific mtg Nov. 20-

22 in Japan, developed using

TKMR RNAi delivery tech 8/2/11

Alnylam ALNY $6.37 $272 12/31/11

Phase I Clinical

Trial

ALN-PCS

(RNAi

therapeutic

for the

treatment of

severe

elevated

cholesterol

levels)

On 7/11/11, announced filing

of CTA for clearance to

conduct P1 trial in UK in 32

healthy volunteers and

expects to present initial safety

/ activity data by year-end

2011 7/11/11

Alnylam ALNY $6.37 $272 12/31/12

Phase 2b

Clinical Trial

ALN-RSV01

(inhaled

treatment,

given by

nebulizer)


NCT01065935 for ongoing

P2B trial in combo with

standard of care in

Respiratory Syncytial Virus

(RSV) infected adult lung

transplant patients, results

expected during 4Q12,

partnered w/ CBST 8/2/11

Amarin AMRN $10.71 $1,426 9/30/11

FDA New Filing

(NDA), Pending

Cardiovascular

Outcomes Study

(REDUCE-IT)

under SPA

AMR101

(icosapent

ethyl)

Prescription

Grade

Omega-3

Fatty Acid)

Expects to file NDA by end of

SEP 2011 seeking approval

for treatment of pts w/ very

high triglycerides (>=500

mg/dl) w/ addt’l data from P3

trials to be presented at AHA

mtg Nov 15-16, on 8/10/11

reported SPA agmt w/ FDA for

long-term (six-year) study in

8,000 pts expected to begin in

2011 w/ target of 50% enrolled

by yr-end 2012 8/10/11

Amgen AMGN $54.44 $50,308 4/26/12

FDA Decision

(sBLA)

XGEVA

(denosumab

)


PDUFA action goal date of

4/26/12 for expected FDA

decision seeking expanded

approval to treat men with

castrate-resistant prostate

cancer to reduce the risk of

developing bone metastases,

currently approved for

prevention of skeletal-related

events in pts w/ cancer that

has spread to bones 8/22/11

Amgen AMGN $54.44 $50,308 12/31/11

Pivotal Phase 3

Clinical Trial

OncoVEX

GM-CSF

(therapeutic

cancer

vaccine)

Ongoing pivotal P3 trial in pts

w/ malignant melanoma (skin

cancer) fully enrolled w/

results expected during 2H11,


NCT00769704 7/29/11

Amicus

Therapeutic

s FOLD $3.93 $136 12/31/11

Phase II and III

Clinical Trials

AMIGAL

(migalastat

HCl) for the

Treatment of

Fabry

Disease

Expects to complete pt enrlmt

in ongoing P3 (Study 011) trial

4Q11, treat first pt in P3

(Study 012) trial 3Q11, and

report prelim results in P2

(Study 013) trial in combo w/

enzyme replcmt therapy

4Q11, partnered w/ GSK 8/9/11

Amylin

Pharma AMLN $10.65 $1,554 10/22/11

FDA Decision

(sNDA)

BYETTA

(exenatide)


submission of sNDA, seeking

FDA approval for expanded

use as add-on therapy to

basal insulin for adult type 2

diabetics not achieving

adequate blood sugar control,

estimated FDA decision by

10/22/11 under a standard 10-

month review period,

partnered w/ LLY 12/22/10

Amylin

Pharma AMLN $10.65 $1,554 12/31/11

FDA New Filing

(BLA)

Metreleptin

(recom-

binant

methionyl

human

leptin)

In JUN 2011 reported results

of treatment IND study w/

improved diabetes and lipid

control in pts w/ lipodystrophy,

FDA Orphan Drug and Fast

Track status, expects to

submit final portion of rolling

BLA filing for chemistry,

manufacturing, and controls

(CMC) section by year-end

2011 6/20/11

Amylin

Pharma AMLN $10.65 $1,554 1/28/12

FDA Decision

(NDA)

BYDUREON

(exenatide

extended-

release

injectable

suspension)

On 8/10/11 reported PDUFA


under six-month Class 2 NDA

resubmission, seeking

approval as once-weekly

treatment for type 2 diabetes,

in JUN 2011 received

approval in Europe, partnered

w/ LLY and ALKS receives

drug delivery royalties 8/10/11

Anacor

Pharma ANAC $4.82 $136 12/31/11

Phase 2 Clinical

Trial

AN2728 and

AN2898

(boron-

based PDE-

4 inhibitors)

In MAY 2011, dosed first pt in

P2 trial to evaluate both

compounds compared to their

ointment vehicles (no drug

included) for treatment of mild-

to-moderate atopic dermatitis

w/ results expected during

2H11 8/12/11

Anacor

Pharma ANAC $4.82 $136 6/30/12

Phase 2b

Clinical Trials

GSK ‘052

(GSK22510

52, formerly

AN3365)

(systemic

antibiotic)

In JUN 2011, partner GSK

began two P2B trials for

treatment of complicated

urinary tract infections (cUTI)

and complicated intra-

abdominal infections (cIAI),

ClinicalTrial.gov IDs

NCT01381562 and



Anacor

Pharma ANAC $4.82 $136 12/31/12

Pivotal Phase 3

Clinical Trials

AN2690

(topical anti-

fungal

product)

Expects to complete enrlmt in

two ongoing P3 trials during

2H11 w/ results expected

during 2H12, being evaluated

for treatment of fungal

infections of the nail and nail

bed (onychomycosis),


NCT01270971 8/12/11

Anadys

Pharma ANDS $0.67 $38 12/31/11

Phase 2a

Clinical Trial

ANA773

(oral TLR7

interferon

inducer)

Expects to begin P2A trial in

Europe during 3Q11 and

complete dosing in first cohort

4Q11 in combo w/ / ribavirin

for treatment of hepatitis C

virus (HCV) infection 8/12/11

Anadys

Pharma ANDS $0.67 $38 10/31/11

Phase 2b

Clinical Trial

Setrobuvir

(ANA598)

(Non-

Nucleoside

HCV

Polymerase

Inhibitor)

In JUL 2011, completed pt

enrlmt in ongoing P2B trial in

combo w/ PEG-interferon and

ribavirin for chronic hepatitis C

virus (HCV) infection, expects

all pts to complete 12-wk visit

by end 3Q11 w/ prelim 12-wk

data shortly after (OCT 2011

estimate) w/ 24-wk anti-viral

data expected around yr-end

2011 8/12/11

Anika

Therapeutic

s ANIK $5.88 $80 12/31/11

FDA Decision

510(k)

Hyaloglide

(healing aid

gel for

tendon and

nerve

surgeries)

As of AUG 2011, reported

disagreement w/ FDA which is

leaning toward the PMA route

for review, estimate for 2H11

update on status 8/8/11

Anika

Therapeutic

s ANIK $5.88 $80 12/31/11

FDA Decision

510(k)

Hyalofast

(cartilage

generation

product)

As of AUG 2011, provided

guidance to receive FDA

510(k) marketing clearance by

yr-end 2011, submission

currently being reviewed by

FDA biologics division,

product includes patient-

derived bone marrow stem cell

component 8/8/11

Anika

Therapeutic

s ANIK $5.88 $80 12/31/11

FDA Decision

510(k)

Hyalonect

(bone wrap

mesh for

repair of

fractures)

As of AUG 2011, provided

guidance to receive FDA

510(k) marketing clearance

w/in next couple of months

(2H11 estimate) 8/8/11

Anika

Therapeutic

s ANIK $5.88 $80 12/31/11

FDA Decision

(PMA Medical

Device) or

Potential FDA

Advisory Panel

Meeting

Monovisc

(single-

injection, six-

month bio-

polymer

arthritis

product)

In AUG 2011, announced 2Q

results and still awaiting FDA

response on pending PMA

(2H11 estimate for either

decision or possible advisory

panel mtg being scheduled)

following late APR 2011 mtg

w/ FDA, no safety issues and

discussing efficacy results,

PMA originally filed in DEC

2009 8/8/11

Antares

Pharma AIS $2.24 $231 12/8/11

FDA Decision

(NDA)

ANTUROL

(transdermal

oxybutynin

ATD gel)


exclusive licensing agmt w/

WPI including regulatory

approval and sales-based

milestone pmts plus

escalating royalties on US and

Canada product sales w/



month review 7/11/11

Anthera

Pharma ANTH $4.61 $188 3/31/12

Pivotal Phase 3

(VISTA-16)

Clinical Trial

under SPA

A-002

(varespladib

methyl)

(once-daily,

oral inhibitor

of sPLA2

enzyme)

Ongoing pivotal P3 trial in

high-risk acute coronary

syndrome pts on track to

accrue 385 primary endpoint

events 1Q12, DSMB met in

early JUL 2011 (fourth time)

and recommended trial

continue as planned w/ DSMB

efficacy data statistical review

expected 2H11 8/1/11

Anthera

Pharma ANTH $4.61 $188 12/31/11

Phase 2b

(PEARL-SC)

Clinical Trial

A-623 (Sub-

cutaneous

Dual

Inhibitor of

BAFF or

BLyS)

As of 6/30/11, pt enrlmt

exceeded 50% w/ last pt on

track to be randomized during

4Q11, during 2Q11 DSMB

recommended trial continue

as planned w/ no safety

concerns, ClinicalTrials.gov ID

NCT01162681 for P2B trial in

lupus pts w/ results expected

during 1H12, decision

expected 2H11 on potential pt

enrlmt expansion 8/1/11

Apricus

Biosciences APRI $4.49 $89 3/31/12

MAA / EMA

(Europe),

Switzerland

Decisions

VITAROS

(alprostadil

topical

formulation)

In JUL 2011, announced

Swiss MAA filing and in JUN

2011 reported MAA accepted

for rvw in Europe (estimated

decisions during 1Q12),

approved by Health Canada

as topical treatment for erectile

dysfunction (ED) in NOV

2010, in AUG 2011 reported

plans to file for aprvl in Latin

America w/ first filing expected

w/in 8-10 wks 8/11/11

Ardea

Biosciences RDEA $15.30 $410 9/30/11

Pending Phase

3 Clinical Trials

Lesinurad

(formerly

RDEA594)

(a selective

URAT1

transporter

inhibitor for

treatment-

resistant

gout)

In JAN 2011, reported positive

top-line results in Phase 2b

study in combo w/ allopurinol

for treatment-resistant gout w/

addt’l open-label results

presented in MAY 2011,

expects to report results of

FDA end-of-P2 mtg during

3Q11 to support planned

pivotal P3 trials 8/17/11

Arena

Pharma ARNA $1.26 $184 12/31/11

FDA

Resubmission to

Complete

Response Letter

(CRL)

Lorcaserin

(a selective

serotonin 2C

receptor

agonist)

In AUG 2011, reported lower

brain concentration of drug in

humans vs. rats and re-

adjudication of female rat

mammary tumor diagnoses,

expects to submit MAA for EU

approval in 2012 and

response to OCT 2010 by yr-

end 2011 to address non-

clinical trial issues cited by

FDA such as brain tumors in

rats, partnered w/ Eisai 8/9/11

Ariad

Pharma ARIA $9.41 $1,247 10/17/11

FDA Reply to

Accept NDA and

Issue PDUFA

Date (mid-Oct),

MAA / EMA

(Europe)

Decision (1Q12)

Ridaforolimu

s (mTOR

inhibitor,

oral anti-

cancer

agent)

In AUG 2011 EMA completed

admin validation process for

MAA seeking EU approval for

metastatic soft-tissue or bone

sarcomas, submitted by

partner MRK in JUL 2011 w/

estimated decision 1Q12,

expected FDA reply w/in 60

days (mid-OCT 2011) to

accept NDA filing and issue

PDUFA decision goal date 8/18/11

Ariad

Pharma ARIA $9.41 $1,247 12/10/11

Pivotal Phase 2

(PACE) Clinical

Trial

Ponatinib

(AP24534)

(pan-BCR-

ABL inhibitor

anti-cancer

agent) (once-

daily 45 mg

tablet)

On 8/2/11, reported pt enrlmt

expected to close during 3Q11

(six months ahd of schedule)

in ongoing pivotal P2 trial in

pts w/ blood-based cancers

CML and Ph+ ALL, has

submitted to present interim

data at ASH mtg Dec 10-13,

expects to file NDA in mid-

2012 w/ six-month priority rvw

request 8/2/11

ArQule ARQL $4.13 $222 6/30/12

Phase 2 Clinical

Trials

Tivantinib

(ARQ 197)

(c-Met

inhibitor anti-

cancer

agent)

Ongoing P2 trials for primary

liver cancer (HCC)

(ClinicalTrials.gov ID


1H12), in combo w/ ERBITUX

+ irinotecan for colorectal

cancer (NCT01075048 w/

results 1Q13), in combo w/

TARCEVA for lung cancer

(NCT01395758 w/ mid-2012

results) 7/21/11

ArQule ARQL $4.13 $222 6/30/13

Pivotal Phase 3

Clinical Trial

under SPA

Tivantinib

(ARQ 197)

(c-Met

inhibitor anti-

cancer

agent)

In NOV 2010, began pivotal

P3 trial in combo w/

TARCEVA for non-small cell

lung cancer (NSCLC) that is

expected to enroll 1,000 pts,

partnered with Daiichi Sankyo,



expected mid-2013 7/21/11

Array

BioPharma ARRY $2.13 $121 12/31/11

Phase 2 Clinical

Trials

ARRY-520

(KSP

inhibitor anti-

cancer

agent)

Ongoing P2 single-agent trial

for multiple myeloma (MM)


NCT00821249) w/ results

expected by yr-end 2011, P1B

combo trial w/ VELCADE for

MM (ClinicalTrials.gov ID

NCT01248923) w/ results


Array

BioPharma ARRY $2.13 $121 3/31/12

Phase 2 Clinical

Trial

ARRY-797

(p38

inhibitor)

Being evaluated in P2 trial w/

150 pts for treatment of

chronic pain in pts w/

osteoarthritis, expects to report

top-line results during 1Q12,

being compared to OxyContin

and placebo for moderate to

severe pain while pts continue

taking NSAID drugs 8/9/11

Array

BioPharma ARRY $2.13 $121 6/30/13

Phase 2 Clinical

Trial

MEK162

(ARRY-162)

(MEK

inhibitor anti-

cancer

agent)

In APR 2011, achieved $10M

milestone pmt from partner

NVS for start of P2 trial in pts

w/ melanoma,




Array

BioPharma ARRY $2.13 $121 12/31/11

Phase I Clinical

Trial

ARRY-502

(CRTh2

antagonist

for allergic

inflammation

)

Expects to complete enrlmt

during 3Q11 and report top-

line results by yr-end 2011 in

healthy volunteers to support

planned P2A trial in pts w/

persistent asthma 8/9/11

Array

BioPharma ARRY $2.13 $121 12/31/11

Phase 1b

Clinical Trial

ARRY-614

(p38/Tie2

inhibitor anti-

cancer

agent)

Expects to report top-line

results by yr-end 2011 for

Myelodysplastic Syndrome

(MDS), ClinicalTrials.gov ID

NCT00916227 8/9/11

Array

BioPharma ARRY $2.13 $121 12/31/11

Phase 2 Clinical

Trials

Selumetinib

(AZD6244)

(MEK

inhibitor anti-

cancer

agent)

Ongoing P2 trials include

ClinicalTrials.gov IDs

NCT01116271 for colon

cancer w/ data expected

during 1Q12, NCT00890825

for non-small cell lung cancer

(NSCLC) w/ data expected

2H11, and NCT00936221 for

melanoma w/ results expected

2H11, partnered w/ AZN 7/7/11

AspenBio

Pharma APPY $3.49 $28 9/30/11

Pre-Pivotal

Clinical Trial

(Medical Device

/ Diagnostic

Test)

AppyScore

(blood-

based

diagnostic

screening

test for

appendicitis)


expected pre-pivotal trial

interim results during 3Q11,

also submitted pre-IDE

package to FDA and plans to

meet to discuss clinical /

regulatory pathway including

design of planned pivotal trial

based on the mtg and pre-

pivotal trial results 7/19/11

Astex

Pharma

(formerly

SuperGen) SUPG $2.07 $192 3/6/12

FDA Decision

(sNDA)

DACOGEN

(decitabine)

(currently

approved for

pts w/

myelodyspla

stic

syndromes

or MDS)

In JUL 2011, partner Eisai

announced FDA accepted

sNDA for review w/ expected

decision by 3/6/12 under

standard 10-month rvw,

seeking expanded approval

for acute myeloid leukemia

(AML) based on P3 (DACO-

016) trial w/ non-stat sig 2.7

month increase in survival, EU

partner JNJ expected to file for

approval in Europe by year-

end 2011 7/20/11

Astex

Pharma

(formerly

SuperGen) SUPG $2.07 $192 12/31/12

Phase 2 Clinical

Trial

Amuvatinib

(MP-470)

(oral

tyrosine

kinase

inhibitor or

multi-TKI

anti-cancer

agent)



expected during 4Q12 for

open-label P2 trial in combo

w/ platinum-etoposide

chemotherapy in pts w/ small

cell lung cancer (SCLC) who

have not responded or

relapsed to standard therapy,

Astex merger closed in JUL

2011 w/ new ticker ASTX to be

effective during 3Q11 7/20/11

Athersys ATHX $1.95 $46 12/31/12

Phase 2 Clinical

Trial

MultiStem

(Adult

Progenitor

or MAPC

Stem Cell

Technology)

On 3/14/11, began enrolling

pts for treatment of ulcerative

colitis, partnered with PFE for

this indication, expects to

enroll up to 126 pts w/ primary

safety and efficacy endpts

evaluated after 8 weeks w/ a

12-month follow-up period,




Athersys ATHX $1.95 $46 12/31/11

Phase I Clinical

Trial

MultiStem

(Adult

Progenitor

or MAPC

Stem Cell

Technology)


positive interim results in

ongoing P1 trial for prevention

of graft vs. host disease

(GvHD), ClinicalTrials.gov ID

NCT00677859 and final

results expected 2H11 8/17/11

Auxilium

Pharma AUXL $15.52 $744 6/30/12

Pivotal Phase 3

(IMPRESS)

Clinical Trials

XIAFLEX

(collag-

enase

clostridium

histolyticum)

During 3Q11 completed

dosing of pts in ongoing P3

trial for treatment of Peyronie's

disease w/ top-line data

expected during 2Q12 to

support potential sNDA filing

(current approved for

treatment of adult Dupuytren's

contracture), ClinicalTrials.gov

ID NCT01243411, BSTC

receives royalties on sale of

drug 9/2/11

AVEO

Pharma AVEO $15.55 $670 12/31/11

Pivotal Phase 3

(TIVO-1) Clinical

Trial

Tivozanib

(AV-951)

(oral, once-

daily

vascular

endothelial

growth factor

or VEGF

inhibitor)

In AUG 2010, completed pt

enrlmt in ongoing pivotal P3

trial compared to NEXAVAR

for pts w/ advanced kidney

(renal) cancer,



expected during 4Q11 at the

earliest, partnered w/ Astellas 7/29/11

AVEO

Pharma AVEO $15.55 $670 6/30/12

Phase 1b / 2

Clinical Trial

Ficlatuzuma

b (AV-299)

(anti-

hepatocyte

growth factor

or HGF

antibody)

Has completed pt enrlmt in P2

portion of ongoing P1B/2 trial

in combo w/ IRESSA as first-

line treatment for pts w/ non-

small cell lung cancer

(NSCLC), ClinicalTrials.gov ID



AVI

BioPharma AVII $1.07 $145 3/31/12

Phase I Clinical

Trial

AVI-7100

(broad

spectrum

influenza

drug

candidate)

On 6/22/11, began dosing

healthy subjects in P1 trial,

being developed for treatment

of influenza (flu infection),




AVI

BioPharma AVII $1.07 $145 6/30/12

Phase 2 Clinical

Trial

Eteplirsen

(AVI-4658)

(exon

skipping

therapy)

On 8/17/11, began dosing pts

in P2 trial for treatment of

Duchenne muscular

dystrophy w/ results expected

around end 2Q12 to support

planned pivotal P3 trial during

2H12, ClinicalTrials.gov ID

NCT01396239 8/17/11

BioCryst

Pharma BCRX $2.85 $128 12/31/11

Phase 2b

Clinical Trial

BCX4208

(oral purine

nucleoside

phos-

phorylase

inhibitor)

In DEC 2010, began enrolling

in 250-patient trial as an add-

on therapy in gout patients

who have not responded to

allopurinol therapy alone,

expects to complete the P2b

trial in late 2011 (4Q11

estimate) 6/16/11

BioCryst

Pharma BCRX $2.85 $128 6/30/13

Phase 3 Clinical

Trial

Peramivir

(IV) (anti-

viral drug for

flu)

Clinical-Trials.gov ID

NCT00958776 for P3 trial of

pts hospitalized w/ influenza

(flu virus), results expected

during 2Q13 after revising

primary endpt in ongoing P3

trial and reporting results from

first P3 trial in JAN 2011 w/ no

safety concerns and

reductions in flu virus levels 8/26/11

Biodel BIOD $1.16 $45 10/31/11

Phase I Clinical

Trial, Pending

Phase 2 Clinical

Trial

BIOD-105

and BIOD-

107 (rapid-

acting

insulin)

On 8/4/11, reported

completion of P1 testing w/ sig

more rapid absorption and

similar injection site tolerability

to HUMALOG, ongoing P1

insulin pump study w/ top-line

results expected in OCT 2011,

expects to select lead ultra

rapid-acting insulin for further

devlmt along w/ timelines

4Q11 8/22/11

BioDelivery

Sciences BDSI $3.18 $94 12/31/11

FDA Decision

(new "retail"

REMS program)

(Risk Evaluation

and Mitigation

Strategy)

ONSOLIS

(BEMA

Fentanyl)

In JUN 2011, reported less

restrictive modified REMS

program expected to be

finalized this summer (2H11

estimate for FDA reply), would

offer access to broader pt

population similar to

competing fast-acting fentanyl

pain drugs 7/18/11

BioDelivery

Sciences BDSI $3.18 $94 12/31/11

Pending Pivotal

Phase 3 PK

Clinical Trial

BEMA

Buprenorphi

ne &

Naloxone

Combo

On 9/6/11 reported P1 results

to support novel formulation of

drug combo used in

SUBOXONE for treatment of

opiate dependence, expects to

begin pivotal bioequivalence

trial in DEC 2011 and report

results during 1Q12 to support

planned 505(b)(2) NDA filing

2H12 9/6/11

BioDelivery

Sciences BDSI $3.18 $94 9/30/11

Pivotal Phase 3

Clinical Trial

BEMA

Bupren-

orphine

On 7/25/11, announced last pt

completed randomized portion

of pivotal P3 trial for treatment

of moderate to severe chronic

pain and expects to report top-

line results in mid-late SEP

2011 to support potential

505(b)(2) NDA filing during

1H12 7/25/11

Biogen Idec BIIB $91.77 $22,258 12/31/11

Pivotal Phase 3

(CONFIRM)

Clinical Trial

BG-12

(dimethyl

fumarate)

In APR 2011, reported positive

top-line data from first of two

pivotal trials as oral mono-

therapy for relapsing-remitting

multiple sclerosis, expects to

report P3 CONFIRM trial data

during 4Q11,


NCT00451451 8/9/11

Biogen Idec BIIB $91.77 $22,258 3/31/13

Pivotal Phase 3

(EMPOWER)

Clinical Trial

Dexpramipe

xole




ongoing pivotal P3 trial in pts

w/ Amyotrophic Lateral

Sclerosis (ALS) (Lou Gehrig’s

disease) 8/9/11

Biogen Idec BIIB $91.77 $22,258 12/31/13

Pivotal Phase 3

(DECIDE)

Clinical Trial

Daclizumab

high-yield

process

(DAC HYP)

(CD25-

binding

antibody)

On 8/9/11, reported positive

results in pivotal P2B trial for

pts w/ relapsing-remitting

multiple sclerosis (RRMS)

over one yr, ongoing second

pivotal P3 trial w/

Clinicalrials.gov ID



partnered w/ ABT 8/9/11

BioMarin

Pharma BMRN $28.06 $3,138 12/31/11

Phase 2 Clinical

Trial

PEG-PAL

(PEGylated

recombinant

phenyl-

alanine or

PHE

ammonia

lyase)

In AUG 2010, reported prelim

results with top-line results

expected during 3-4Q11 to

support planned pivotal P3

study during 1-2Q12, enzyme

substitution therapy for the

treatment of phenyl-ketonuria

(PKU) with potential to treat

patients whose blood PHE

levels are not adequately

controlled by KUVAN 8/17/11

BioMarin

Pharma BMRN $28.06 $3,138 6/30/13

FDA Decision

510(k) Medical

Device

Hand-Held

Blood Phe

(phenyl-

alanine)

Monitor

Expects to receive FDA 510(k)

marketing clearance by mid-

2013 (1-3Q13 guidance) for

handheld blood Phe monitor

for pts w/ phenylketonuria to

support medical management

of the condition which is

treated w/ BMRN’s marketed

drug KUVAN 8/17/11

BioMarin

Pharma BMRN $28.06 $3,138 6/30/12

Pivotal Phase 3

Clinical Trial

Amifampridi

ne (3,4-

diaminopyri

dine)

In JUN 2011, dosed first pt in

pivotal P3 trial for treatment of

Lambert-Eaton Myasthenic

Syndrome (LEMS) to support

potential NDA filing during

1H12, approved in Europe in

JAN 2010 8/17/11

BioMarin

Pharma BMRN $28.06 $3,138 12/31/12

Pivotal Phase 3

Clinical Trial

GALNS

(BMN 110 or

N-acetyl-

galactosami

ne 6-

sulfatase)

In FEB 2011, began pivotal P3

trial for treatment of lysosomal

storage disorder Mucopoly-

saccharidosis Type IVA (MPS

IVA), expects to enroll 160

subjects w/ top-line results

expected during 2H12 to

support regulatory approval

filings during 4Q12-1Q13 8/17/11

BioMimetic

Therapeutic

s BMTI $3.18 $89 9/30/11

FDA Decision

(PMA) (Medical

Device), CE

Mark (Europe)

Decision

Augment

Bone Graft

(synthetic,

off-the-shelf

bone growth

factor

product)

In MAY 2011, FDA Advisory

Panel voted 12-6 in support of

safety, 10-8 in support

efficacy, and 10-8 in support

of benefit to risk profile, in JUL

2011 met w/ FDA and expects

follow-up letter around SEP

2011 to outline post-panel

reqmts for PMA aprvl and will

provide update on status at

that time, expected CE Mark

decision in 2012 (1H12

estimate) 8/5/11

BioMimetic

Therapeutic

s BMTI $3.18 $89 6/30/12

Pivotal Clinical

Trial to Support

PMA (Medical

Device) FDA

Filing

Augment

Injectable

Bone Graft

In APR 2011, began enrolling

pts in pivotal trial for hindfoot

fusion indications, expects to

complete enrlmt phase of trial

in mid-2012, plans to submit

PMA based on final 12-month

safety and efficacy data 8/5/11

Bionovo BNVI $0.67 $37 10/31/11

Pending Pivotal

Phase 3 Clinical

Trial

MENERBA

(a selective

estrogen

receptor

modulator or

SERM)


successful completion of

tolerability trial and dosing

strategy for pending pivotal P3

trial expected to begin in fall

(OCT 2011 estimate) for

treatment of hot flashes,

expects 5 data safety

monitoring board rvws (first

4Q11) over course of trial,

expects to complete pt enrlmt

2Q12 and report top-line data

4Q12 8/30/11

BioSante

Pharma BPAX $2.51 $276 12/31/11

Pending Phase I

Clinical Trial

Melanoma

Cancer

Vaccine


exclusive license agmt w/

John P. Hussman Foundation

including $100,000 upfront

and $39M in potential

milestone pmts and royalties,

expects to begin P1 trial w/in

several months (2H11

estimate), Foundation will fund

P2 trial if P1 trial is successful 7/21/11

BioSante

Pharma BPAX $2.51 $276 11/14/11

FDA Decision

(NDA)

Bio-T-Gel

(testosteron

e gel)

Licensed to TEVA w/ PDUFA

action goal date of 11/14/11

for the treatment of low

testosterone levels in males

(hypogonadism), would

receive milestone payments

and royalties from TEVA if

approved 4/13/11

BioSante

Pharma BPAX $2.51 $276 12/31/11

Pivotal Phase 3

Clinical Trials

LibiGel

(testosteron

e gel)

On 5/31/11, completed enlrmt

in P3 safety trial which

continued as planned after

sixth DMC safety rvw w/

results expected during 3Q12

after 12-month follow-up on

last pt dosed to support

potential NDA filing by year-

end 2012, expects to report

data from two P3 efficacy trials

under SPA during 4Q11 5/31/11

Cardiome

Pharma CRME $3.53 $216 12/31/12

Phase 3 Clinical

Trial

Vernakalant

(IV

formulation)

(MK-6621)

In JUL 2011, announced

transfer of rts from Astellas to

MRK w/ ongoing P3 trial for

pts w/ abnormal heart rhythm

atrial fibrillation,



expected during 4Q12, in SEP

2010 approved in Europe,

also being developed in oral

formulation w/ MRK 7/27/11

Cardiovascu

lar Systems CSII $13.13 $228 12/31/12

Phase 3 (ORBIT

II) Clinical Trial

Diamondbac

k 360°

Orbital

Atherectomy

System

In MAY 2011, received FDA

clearance to complete enrlmt

of 429 pts for treatment of

calcified heart vessel

(coronary) lesions following

rvw of initial data from 50 pts,


NCT01092416 w/ expected

completion of trial during

4Q12 8/4/11

Cardium CXM $0.21 $18 12/31/11

FDA Decision

510(k) Medical

Device

Excellagen

(2.6%

collagen

topical gel

pre-filled

syringes)

During 2Q11 submitted

manufacturing info to FDA in

support of 510(k) submission,

seeking marketing clearance

for topical wound

management for a variety of

dermal wounds, believes it

has addressed all outstanding

issues w/ estimated FDA

decision by late 2011-early

2012 but no formal decision

deadline 8/2/11

Catalyst

Pharma

Partners CPRX $1.30 $28 12/31/12

Pivotal Phase

2b / 3 Clinical

Trial

Vigabatrin

Oral Tablets

(CPP-109)


(200) during 1H12 and report

top-line results during 4Q12

for ongoing study in

collaboration w/ Nat’l Inst.

Drug Abuse (NIDA) for

patients w/ cocaine addiction,


NCT01281202 8/16/11

CEL-SCI

Corp. CVM $0.39 $82 6/30/12

Pivotal Phase 3

(IT-MATTERS)

Clinical Trial

Multikine

(immuno-

therapy

mixture of

naturally

derived

cytokines)



expected mid-2012 for Phase

3 study of Multikine plus

standard of care (SOC) vs.

SOC treatment only previously

untreated pts w/ advanced

squamous cell carcinoma of

the oral cavity or soft palate 5/17/11

Cell

Therapeutic

s CTIC $1.13 $219 12/31/13

Phase 2 Clinical

Trial

OPAXIO

(paclitaxel

poliglumex,

CT-2103)

(formerly

XYOTAX)

In AUG 2011, opened enrlmt

for randomized P2 trial for pts

w/ newly diagnosed brain

cancer (GBM) in combo w/

radiation (RT) vs.

temozolomide (TMZ) plus RT,




Cell

Therapeutic

s CTIC $1.13 $219 12/31/11

FDA

Resubmission

(NDA) (4Q11),

MAA / EMA

(Europe)

Decision

(1Q12), Pivotal

Phase 3 (PIX-R)

Clinical Trial

(4Q12)

PIXUVRI

(pixantrone

dimaleate)

Expects to resubmit NDA in

response to CRL during

4Q11w/ anticipated Class 2

six-month review, expects to

receive EMA opinion on

European approval decision

for non-Hodgkin's lymphoma

(NHL) 1Q12 following 120-day

response on 8/23/11,



expected 4Q12 for P3 B-cell

lymphoma trial 8/23/11

Celldex

Therapeutic

s CLDX $2.64 $117 12/31/11

Pending Phase

3 (ACT IV) and

Phase 2 Clinical

Trials

Rindopepim

ut (CDX-

110)

(therapeutic

brain cancer

vaccine

targeting

EGFRvIII)

In AUG 2011, announced

finalized protocol for pending

P3 trial expected to enroll up

to 374 pts at 150 global sites

w/ newly-diagnosted brain

cancer (GBM) and begin

during 2H11 along w/ P2 trial

alone or in combo w/

AVASTIN for recurrent or

refractory GBM 8/3/11

Celldex

Therapeutic

s CLDX $2.64 $117 12/31/11

Phase 2b

Clinical Trial

CDX-011

(glem-

batumumab

vedotin)

(monoclonal

antibody-

drug

conjugate)

In SEP 2010, began P2B trial

in glycoprotein NMB

(GPNMB) expressing

refractory breast cancer

patients, expects to complete

enrollment of 120 patients by

year-end 2011,


NCT01156753, received FDA

Fast Track Status in May 2010 8/3/11

Celldex

Therapeutic

s CLDX $2.64 $117 12/31/11

Phase I / II

Clinical Trial

CDX-1401

(NY-ESO-1

tumor

antigen

targeting

human

monoclonal

antibody)




assess safety, immune

response and anti-tumor

activity in combo w/ immune

stimulating agents for pts w/

cancers that express tumor

antigen NY-ESO-1 8/3/11

Celsion CLSN $3.61 $95 12/31/13

Phase I / II Open-

Label (DIGNITY)

Clinical Trial

ThermoDox

(doxorubicin

encapsulate

d in a heat-

activated

liposome)

On 6/8/11, announced DSMB

recommended trial advance to

P2 portion after review of

safety data from P1 portion for

treatment of recurrent chest

wall (RCW) breast cancer,


NCT00826085 w/ final results

expected late 2013-early 2014 8/9/11

Celsion CLSN $3.61 $95 12/31/11

Pivotal Phase 3

(HEAT) Clinical

Trial under SPA

ThermoDox

(doxorubicin

encapsulate

d in a heat-

activated

liposome)

In AUG 2011, completed pt

enrlmt (600) in ongoing pivotal

P3 trial in combo w/ RFA for

primary liver cancer (HCC),

expects to reach trigger of 190

PFS events during 3Q11 for

planned interim efficacy

analysis during 4Q11 and final

data expected in 2012,


NCT00617981 8/10/11

Chelsea

Therapeutic

s CHTP $4.18 $259 9/30/11

FDA New Filing

(NDA), Phase 2

Clinical Trial,

Phase 3 Clinical

Trial

NORTHERA

(droxidopa)

(orally active

synthetic

precursor of

norepinephri

ne)

Expects to file NDA during

3Q11 seeking approval for

treatment of symptomatic

NOH (neurogenic orthostatic

hypotension), ongoing P3 trial

(Study 306) for potential falls

claim indication w/ results


expects to report top-line

results from ongoing P2 trial

by yr-end 2011 for treatment

of fibromyalgia 8/8/11

Chelsea

Therapeutic

s CHTP $4.18 $259 9/30/11

Phase 2 Clinical

Trial

CH-4051

(orally

available

anti-folate

agent) (L-

isomer of

CH-1504)


DSMB rvw of safety data and

trial continued as planned to

begin enrlmt of high-dose

groups for treatment of for

rheumatoid arthritis compared

to treatment with methotrexate,

expects to report un-blinded

interim efficacy data in SEP

2011 w/ full results expected

during 2Q12 7/26/11

Columbia

Labs CBRX $2.14 $187 2/26/12

FDA Decision

(NDA)

Prochieve

(8% Progest-

erone Gel)

On 6/27/11 FDA accepted

NDA w/ PDUFA decision goal

date of 2/26/12 under

standard 10-month rvw,

received $5M milestone pmt

from partner WPI for accepted

NDA (due to receive $30M

upon US commercialization),

seeking approval to reduce

risk preterm birth in women w/

short cervix in mid-trimester of

pregnancy 6/27/11

Corcept

Therapeutic

s CORT $2.75 $232 2/17/12

FDA Decision

(NDA)

CORLUX

(mifepriston

e)


accepted NDA w/ PDUFA


under standard 10-month

review, seeking approval for

treatment of the hormonal

disorder Cushing's Syndrome,

in AUG 2011 reported no

advisory panel mtg will be

scheduled to rvw NDA 8/22/11

CorMedix CRMD $1.04 $11 9/30/11

Phase 2 Clinical

Trial

Deferiprone

(CRMD001)

(a novel oral,

twice-daily

formulation

of iron-

binding

drug)

On 9/6/11, amended agmt w/

Shiva Biomedical to extend

start of pt dosing in pivotal trial

to 1Q12, expects to report

results from ongoing

biomarker proof of concept

trial by end 3Q11 to support

start of pivotal trial during

1H12 9/6/11

CorMedix CRMD $1.04 $11 12/31/11

Pending Pivotal

Phase 3

(Medical

Device) Clinical

Trial (2H11), CE

Mark (Europe)

Decision (1H12)

Neutrolin

(CRMD003)

(1.35%

taurolidine,

4% citrate

and

1000u/mL

heparin)

On 7/7/11 announced filing for

CE Mark (Europe) for

prevention of catheter-related

blood infections and to

maintain catheter patency in

dialysis catheters w/ expected

decision during 1H12, expects

to begin pivotal P3 trial (FDA)

during 2H11 following JUN

2011 request for designation

filing 7/7/11

Cubist

Pharma CBST $32.46 $1,983 3/31/13

Pivotal Phase 3

Clinical Trials

CXA-201

(novel

cephalospori

n antibiotic

in combo

with

tazobactam)

On 8/1/11, announced start of

pivotal P3 trials in pts w/

complicated urinary tract

infections (cUTI)

(ClinicalTrials.gov IDs

NCT01345929 and


expected 1Q13), expects to

begin P3 trial for complicated

intra-abdominal infections

(cIAI) by yr-end 2011 and P3

trials for hospital-acquired

pneumonia in 2012 8/1/11

Curis CRIS $3.00 $230 12/31/11

FDA New Filing

(NDA)

Vismodegib

(GDC-0449)

(RG3616)

(Hedgehog

pathway

inhibitor, anti-

cancer

agent)


partner Roche presented

pivotal P2 trial data in pts w/

advanced basal cell

carcinoma (BCC) and expects

to file NDA in 2011 based on

positive results which met

primary endpt, median

duration of response was 7.6

months and progression-free

survival (PFS) was 9.5 months 6/20/11

Cyclacel

Pharma CYCC $0.74 $40 9/30/11

Phase 2 Clinical

Trial, Pivotal

Phase 3

(SEAMLESS)

Clinical Trial

under SPA

Sapacitabin

e (CYC682)

(oral

capsules) (a

nucleoside

analogue

anti-cancer

agent)

In JAN 2011, began enrolling

pts in pivotal P3 trial as front-

line treatment of elderly

patients w/ newly diagnosed

acute myeloid leukemia

(AML), DSMB review expected

during 3Q11 to enable start of

randomized portion of trial

expected to complete enrlmt

2H12 w/ final results 2H13,

expects to present P2 lung

cancer data 2H11 8/12/11

Cytokinetics CYTK $1.10 $79 12/31/11

Phase 1 and 2a

Clinical Trials

CK-

2017357 (a

fast skeletal

muscle

troponin

activator)

Expects to report results from

ongoing P2A trial for

amyotrophic lateral sclerosis

(ALS) (Lou Gehrig’s disease)

by year-end 2011, expects to

report results from ongoing

P2A trial for myasthenia gravis

by year-end 2011, expects to

report P1 drug / drug

interaction trial results during

2H11 7/29/11

Cytokinetics CYTK $1.10 $79 6/30/13

Phase 2b

Clinical Trial

Omecamtiv

Mecarbil

(intravenous

formulation)

In APR 2011, began enrolling

pts w/ partner AMGN for

treatment of acute heart

failure, expects to enroll 600

pts in 3 ascending-dose

groups compared to placebo,



expected during 1H13,

discussing plans w/ AMGN to

begin clinical trials for oral

formulation of the drug 7/29/11

Cytomedix CMXI $0.38 $19 6/30/12

FDA Decision

510(k)

Angel Whole

Blood

Separation

System


submission of FDA 510(k)


for expanded use of device for

processing a mix of both blood

and bone marrow as a source

of platelet rich plasma (PRP)

containing stem cells,

estimated FDA reply by mid-


deadlines for med devices 9/1/11

Cytori

Therapeutic

s CYTX $3.01 $162 12/31/11

CE Mark

Clearance, EU

Indication-for-

Use Decision,

Pivotal

European

(ADVANCE)

Clinical Trial

Celution

One System

Began enrolling pts 1Q11 for

pivotal EU acute heart attack

(MI) trial and expects to

complete pt enrlmt during

1H13, expects to receive CE

Mark clearance for next-gen

Celution One by yr-end 2011,

expected indication-for-use

decision (EU) for no-option

chronic myocardial ischemia

pts by early 2012 (1Q12

estimate) 8/10/11

CytRx CYTR $0.36 $53 3/31/12

Phase 2 Clinical

Trials

Tamibaroten

e (oral

synthetic

retinoid anti-

cancer

agent)



expected 1Q12 for ongoing

STAR-1 study under SPA for

relapsed or refractory acute

promyelocytic leukemia (APL),



trial in combo w/ paclitaxel and

carboplatin for advanced non-

small-cell lung cancer

(NSCLC) w/ results expected

2H12 8/1/11

CytRx CYTR $0.36 $53 12/31/11

Phase 1b

Clinical Trial,

Pending Phase

2b Clinical Trial

INNO-206

(tumor-

targeting pro-

drug of

chemo drug

doxorubicin)

In APR 2011, began open

label P1B trial



expected 4Q11) in up to 24

pts w/ primarily soft tissue

sarcomas that failed standard

treatments and expects a P2B

trial for soft tissue sarcomas in

2011, FDA Orphan drug

status for pancreatic cancer

and sarcoma 8/9/11

CytRx CYTR $0.36 $53 12/31/11

Phase 2 Clinical

Trials

Bafetinib

(formerly

INNO-406)

(an orally

available,

dual Bcr-Abl

and Lyn

kinase

inhibitor)

On 6/13/11, reported prelim

P2 results for B-cell chronic

lymphocytic leukemia (B-CLL)

pts (16) w/ evidence of clinical

activity and low incidence of

adverse events and will enroll

more pts, ClinicalTrials.gov





expected 4Q11 for P2 prostate

cancer trial 6/13/11

DARA

BioSciences DARA $1.96 $10 9/30/11

Phase 1b

Clinical Trial

DB959 (dual

PPAR delta /

gamma

agonist)

In JUN 2011, reported positive

safety results in P1A trial and

expects to report results of

ongoing P1B trial during

3Q11, being developed as

oral drug for treatment of type

2 diabetes 6/28/11

DARA

BioSciences DARA $1.96 $10 12/31/11

Pending Phase

2 Clinical Trial

KRN5500

(novel

spicamycin

derivative, IV

formulation)

Expects to begin second P2

trial for during 2H11, in AUG

2011 received FDA Fast Track

status for treatment of chemo-

inducted neuropathic (nerve)

pain in pts w/ cancer 8/18/11

Delcath

Systems DCTH $3.69 $177 12/31/11

FDA New Filing

(NDA), Phase 2

Clinical Trial

Delcath

Percutaneou

s Hepatic

Perfusion

(PHP)

System

Expects to re-file 505(b)(2)

NDA by year-end 2011 in

response to FEB 2011 refusal

to file letter from FDA

requesting addt’l

manufacturing and safety

data, seeking approval for the

local delivery of melphalan in

patients w/ metastatic

melanoma in the liver, on

9/1/11 reported P2 colon

cancer spread to liver trial

failed had no sig responses 9/1/11

DepoMed DEPO $5.78 $320 10/31/11

Pivotal Phase 3

(BREEZE-3)

Clinical Trial

under SPA

SERADA

(extended

release

gabapentin)

In MAR 2011, completed

enrlmt in pivotal P3 trial for the

non-hormonal treatment of

menopausal hot flashes w/


(mid-late OCT 2011 estimate),

same drug approved by FDA

as GRALISE for post-herpetic

neuralgia (nerve pain that

persists after shingles

infection) in JAN 2011 8/1/11

Derma

Sciences DSCI $8.09 $85 6/30/12

Pending Pivotal

Phase 3 Clinical

Trial

DSC127 (an

Angiotensin

analog, a

naturally

occurring

peptide)

In MAY 2011, reported P2 trial

data for diabetic foot ulcer pts

w/ a 27% wound healing

improvement vs. placebo,

expects to conduct end of P2

mtg w/ FDA during 4Q11 and

begin pivotal P3 trial during

1H12 8/16/11

Discovery

Labs DSCO $2.08 $50 12/31/11 FDA New Filing

AFECTAIR

(disposable

delivery

device for

aerosolized

medicine)

On 7/29/11, announced plans

to file for FDA approval during

4Q11 and EU approval during

1H12 for disposable delivery

device for pts on ventilator

support to receive aerosolized

medications 7/29/11

Discovery

Labs DSCO $2.08 $50 9/16/11

FDA Reply to

Accept NDA

Resubmission

and Issue

PDUFA

Decision Date

SURFAXIN

(lucinactant)

(synthetic,

peptide-

containing

lung

surfactant)

On 9/2/11 submitted complete

response to CRL issued by

FDA in 2009, seeking

approval for the prevention of

respiratory distress syndrome

(RDS) in premature infants,

expected FDA reply in two

weeks (9/16/11) to accept

NDA and issue PDUFA

decision goal date w/ a six-

month Class 2 review period

expected 9/6/11

Durect Corp. DRRX $1.37 $120 12/31/11

Pivotal Phase 3

(BESST)

Clinical Trial

POSIDUR

(Saber

Bupiv-

acaine) Post-

Operative

Pain Relief

Depot

On 9/6/11, announced dosing

of last pt in ongoing pivotal P3

trial for novel long-acting (3-

day) formulation of approved

drug bupivacain for post-

surgical pain relief w/ top-line

data expected during 4Q11 to

support planned NDA filing


NCT01052012, US partner is

HSP, EU partner is Nycomed 9/6/11

Dyax DYAX $1.47 $145 12/31/12

Phase 2 Clinical

Trial

KALBITOR

(ecallantide)

(reversible

inhibitor of

plasma

kallikrein)

In AUG 2011, began treating

pts in ongoing P2 trial for

acute treatment of angiotensin

converting enzyme (ACE)

inhibitor-induced angioedema

(severe allergic reaction), FDA

approved as KALBITOR for

treatment of acute attacks of

hereditary angioedema,

expects to report results

during 2H12 8/5/11

Dynavax DVAX $2.24 $276 12/31/11

Phase I Clinical

Trial

DV1179

(TLR7&9

inhibitor)

On 4/20/11, began dosing in

Phase I trial expected to enroll

24 healthy subjects divided

into three dose groups,

partnered w/ GSK and will

receive $6M milestone pmt for

starting the trial, being

developed for treatment of

lupus, expects to report data

later this year (2H11 estimate) 4/20/11

Dynavax DVAX $2.24 $276 12/31/11

FDA New Filing

(BLA) (4Q11),

Phase 3 Clinical

Trial (1Q12)

HEPLISAV

(hepatitis B

vaccine)


agreed manufacturing

consistency demonstrated in

P3 trial that concluded in JUL

2011, plans to file BLA by year-

end 2011 w/ ongoing P3 trial

in chronic kidney disease pts

w/ results expected in early

2012 (1Q12 estimate) 7/28/11

Echo

Therapeutic

s ECTE $3.84 $132 9/30/11

FDA Decision

510(k) Medical

Device

Prelude

SkinPrep

and 4%

Lidocaine

Cream


expected 510(k) marketing

clearance during 3Q11 based

on FDA feedback to partner

Ferndale Pharma (private),

seeking approval as faster-

acting local anesthetic (skin

numbing agent prior to needle

sticks), will receive $750,000

milestone pmt upon FDA

approval plus double-digit

royalties 2/8/11

Elan ELN $10.00 $5,867 6/30/12

Phase 3 Clinical

Trial

Bapineuzum

ab (AAB-

001)

Experimental treatment for

mild to moderate Alzheimer's

disease (AD), PFE and JNJ

also have stakes in the

development of this

compound while Elan has

retained approx. 25% stake,


NCT00574132 with results

expected during 3Q12 and


expected 2Q12 8/17/11

Endo

Pharma ENDP $30.34 $3,537 12/13/11

FDA Decision

(NDA)

OPANA ER

(new

formulation

of extended-

release

oxymorphon

e)


accepted its complete

response to JAN 2011 CRL

seeking approval for new

formulation of pain drug

designed to deter abuse,


12/13/11 6/24/11

Endocyte ECYT $10.92 $388 6/30/13

Pivotal Phase 3

(PROCEED)

Clinical Trial

EC145

(folate

receptor

targeted anti-

cancer

agent) and

EC20

(companion

imaging

diagnostic)

Expects to file MAA during

1Q12 seeking EMA / EU

approval for treatment of pts

w/ folate-receptor positive

platinum-resistant ovarian

cancer, in MAY 2011 began

enrolling pts in pivotal P3 trial

w/ companion diagnostic

(EC20) to identify folate over-

expression, ClinicalTrials.gov

ID NCT01170650 w/ results


EnteroMedic

s ETRM $2.40 $67 3/31/13

Pivotal Phase 3

(ReCharge)

Clinical Trial

Maestro

RC2 System

(VBLOB

Vagal Nerve

Blocking

Device)


by yr-end 2011 for ongoing

pivotal P3 trial in obese pts,




Enzon

Pharma ENZN $8.21 $400 12/31/11

Phase 2 Clinical

Trials

PEG-SN38

(PEGylated

form of the

active

metabolite of

the cancer

pro-drug

Camptosar /

irinotecan)


decision to discontinue

program for metastatic colon

cancer after ongoing P2 trial

completed w/



expected 4Q11,


NCT01036113 for P2 trial in

pts w/ previously treated

metastatic breast cancer w/

results expected 2H11 5/19/11

EpiCept EPCT $0.38 $27 12/31/12

Phase I / II

Clinical Trial

Crolibulin

(EPC2407)

(vascular

disruption

anti-cancer

agent)

In DEC 2010, NCI / NIH

began P1/2 trial in combo w/

cisplatin for pts w/ solid

tumors (focusing on anaplastic

thyroid cancer),




EpiCept EPCT $0.38 $27 12/31/11

Pending Pivotal

Phase 3 Clinical

Trial w/ SPA

Request

AmiKet

(topical

analgesic

cream)

Expects to submit pivotal P3

trial protocol w/ SPA request to

FDA during 2H11 for

treatment of chemo-induced

neuropathic pain, seeking

partner to fund P3 trial 8/15/11

EpiCept EPCT $0.38 $27 12/31/11

Pending

Confirmatory

Phase 3 Clinical

Trial w/ SPA

Request, Post-

Marketing

(Europe)

Clinical Trial

CEPLENE

(histamine

dihydro-

chloride)

Expects to meet w/ FDA

during 3Q11 to discuss

proposed confirmatory P3 trial

w/ SPA request for treatment

of acute myeloid leukemia

(AML) in first remission,

ongoing post-approval study

in Europe w/ initial data from

75 pts expected in early 2012

(1Q12 estimate) 8/15/11

EXACT

Sciences EXAS $7.44 $392 9/30/12

Pivotal (DeeP-

C) Clinical Trial

(Medical Device

/ Diagnostic)

Cologuard

(stool DNA

based

molecular

diagnostic

test)

In JUL 2011, announced start

of pivotal trial for diagnostic

screening test for early

detection of colorectal cancer

and pre-cancers (CRC),

expects to enroll 10-12,000

subjects over approx. 12

months at cost of $20M,




support planned PMA filing in

2012 8/3/11

Exelixis EXEL $6.69 $863 9/30/11

Pivotal Phase 3

(EXAM) Clinical

Trial under SPA,

Pending Pivotal

Phase 3 Clinical

Trial w/ SPA

Request

Cabozantini

b (XL184)

(MET,

VEGFR2,

and RET

inhibitor anti-

cancer

agent)

Expects to report top-line data

in pivotal P3 trial for medullary

thyroid cancer around end of

3Q11 to support planned

rolling NDA filing during 4Q11

(1Q12 estimate to complete),

expects to begin pivotal P3

trial under SPA by yr-end

2011 for castration-resistant

prostate cancer (CRPC) 8/5/11

Forest Labs FRX $33.08 $9,082 12/31/11

Phase 3 Clinical

Trials

Levomilnaci

pran (1S,2R-

milnacipran)

(active

enantiomer

of

LEXAPRO)


stat sig P3 results for

treatment of depression in

adults, expects to report

results from two addt’l P3

results by year-end 2011 and

spring 2012 as the isolated

active compound found in

marketed depression drug

LEXAPRO 7/18/11

Forest Labs FRX $33.08 $9,082 4/30/12

FDA Decision

(NDA)

Aclidinium

Bromide

(long-acting

inhaled anti-

muscarinic

agent)

On 6/30/11 announced NDA

submission seeking approval

for treatment of lung condition

(COPD), expected FDA

decision by 4/30/12 under a

standard 10-month review

period 6/30/11

Furiex

Pharma FURX $13.64 $135 1/25/12

FDA Decisions

(Two NDA

Resubmissions)

Alogliptin

and

Alogliptin /

Pioglitazone

(ACTOS)

Fixed-Dose

Combo

On 7/25/11, partner Takeda

announced two NDA

resubmissions based on

interim cardiovascular safety

results seeking approval for

new type of drug for type 2

diabetes both alone and in

combo w/ marketed diabetes

drug ACTOS w/ expected six-

month FDA rvw and decision

by 1/25/12, FURX due to

receive $25M on approval and

royalties 7/26/11

Furiex

Pharma FURX $13.64 $135 9/30/12

Phase 2 Clinical

Trial

JNJ-

32729463

(novel,

broad-

spectrum

fluoro-

quinolone

antibiotic)

Partnered with JNJ,



expected mid-2012 (2-3Q12)

for treatment of patients

requiring hospitalization for

community-acquired bacterial

pneumonia 8/4/11

Furiex

Pharma FURX $13.64 $135 9/30/11

Phase 2 Clinical

Trial

JNJ-

27018966

(Mu Delta)

(mu opioid

receptor

agonist,

delta

antagonist)

Last pt completed P2 trial for

pts w/ diarrhea-predominant

irritable bowel syndrome in

JUL 2011, ClinicalTrials.gov



Fast Track status announced

in JAN 2011, partnered w/ JNJ 8/4/11

Generex

Biotech GNBT $0.10 $30 9/30/11

Phase 3 Clinical

Trial

Generex

Oral-lyn

(buccal

insulin spray

product)


NCT00668850 for 26-week

open-label randomized P3

(084) trial that will be closed in

AUG 2011 w/ results expected

during 3Q11, evaluating oral

insulin spray w/ regular

human insulin in type 1

diabetics, plans to conduct

addt’l P3 trials that are

expected to be completed

during 2013 8/2/11

Gen-Probe GPRO $57.53 $2,772 3/31/12

FDA Advisory

Panel Meeting,

FDA Decision

(PMA)

PROGENSA

PCA3 Assay

On 8/29/11, SEC 8-K filing

indicated FDA Advisory Panel

mtg delayed until 1Q12 to

provide more time to rvw addt’l

info submitted in support of

PMA originally submitted in

SEP 2010, developed as

molecular diagnostic test

(urine samples) to help

determine need for repeat

biopsies in men suspected of

having prostate cancer 8/29/11

Gen-Probe GPRO $57.53 $2,772 3/31/12

FDA Decision

(PMA)

APTIMA

HPV

(human

papilloma-

virus) Assay

In NOV 2010, submitted PMA


for molecular diagnostic test to

detect high-risk HPV

infections associated w/

cervical cancer and pre-

cancerous lesions in women,

estimated FDA reply in late

2011-early 2012 but no formal

decision deadlines for medical

devices / diagnostics 8/29/11

Gen-Probe GPRO $57.53 $2,772 3/31/12

FDA Decision

510(k)

PANTHER

System

In MAY 2010 announced FDA

510(k) submission seeking

marketing clearance for fully

automated molecular

diagnostic testing system to

run tests such as APTIMA

Combo 2 (chlamydia and

gonorrhea test), received CE

Mark (Europe) in DEC 2010,

estimated FDA reply early-mid


deadline for med devices 8/29/11

GenSpera GNSZ $1.83 $41 12/31/11

Phase I Clinical

Trial, Pending

Phase 2 Clinical

Trial

G-202

(thapsigargi

n pro-drug

anti-cancer

agent)

Expects to report ongoing P1

trial results in pts w/ advanced

solid tumors during 4Q11 to

support start of P2 trial for


cancer during 4Q11 7/21/11

Gentium GENT $6.36 $95 11/1/11

MAA / EMA

(Europe)

Decision Defibrotide

On 6/1/11, announced MAA

seeking approval for

prevention and treatment of

hepatic veno-occlusive

disease (VOD) of the liver

following stem cell transplant

was accepted for accelerated

review (150 days) for expected

EMA decision by 11/1/11, in

AUG 2011 withdrew NDA

citing issues related to data

and conduct of trials 8/18/11

GeoVax

Labs GOVX $0.85 $13 6/30/12

Phase 2a

(HVTN 205)

(HIV Vaccine

Trials Network

Sponsored)

Clinical Trial

HIV (Human

Immuno-

deficiency

Virus)

Preventative

Vaccine

On 12/9/10, reported early

results in ongoing blinded

study indicating no safety

concerns and favorable

immune (T cell) responses in

180 pts to date, study initiated

JAN 2009 w/ HVTN and NIH,


in 2011 w/ full study results

expected in 2012 (1H12

estimate) 6/21/11

GeoVax

Labs GOVX $0.85 $13 3/31/12

Phase I / II

Clinical Trial

HIV (Human

Immuno-

deficiency

Virus)

Therapeutic

Vaccine

In MAY 2010, the AIDS

Research Consortium of

Atlanta received approval to

begin enrollment of 10-12 pts,

expects P1/2 trial to begin

generating vaccine safety and

performance data late 2011-


added third clinical site for trial

in JUN 2011 6/23/11

Geron GERN $2.58 $333 6/30/12

Phase 2 Clinical

Trials

GRN163L

(imetelstat

sodium)

(telomerase

inhibitor anti-

cancer stem

cell agent)

Ongoing Phase 2 studies

include ClinicalTrials.gov ID

NCT01137968 (results

expected mid-2012 for lung

cancer), NCT01256762

(results expected 1Q13 for

breast cancer), NCT01243073

(results expected 1Q13 for

essential thrombocythemia),


expected 1Q13 for multiple

myeloma) 6/20/11

Geron GERN $2.58 $333 12/31/12

Phase I Clinical

Trial

GRNOPC1

(a human

embryonic

stem cell or

hESC

product

candidate)

In JUN 2011, presented data

on first two spinal cord injury

pts w/ no safety concerns,




received FDA clearance to

expand eligibility criteria and

shorten time lag to 10 days

between enrlmt of new pts 7/29/11

Gilead

Sciences GILD 38.57 $29,755 9/30/11

Pivotal Phase 3

Clinical Trial

QUAD

single-tablet

HIV regimen

of

elvitegravir,

cobicistat

and

TRUVADA

(emtricitabin

e and

tenofovir)

On 8/15/11, announced first of

two pivotal P3 trials (Study

102) met primary endpt of non-

inferiority to ATRIPLA for

treatment of HIV-1 infection,

second P3 trial (Study 103) w/


to support planned NDA filing

in early 2012

8/15/11

Given

Imaging GIVN $14.31 $434 6/30/12

Pivotal Clinical

Trial to Support

FDA 510(k)

Submission

PillCam

COLON 2

video

capsule

endoscopy

In JUN 2011, began enrolling

pts in ongoing pivotal study for

product , designed to visualize

the colon in average risk

population unwilling to

undergo colonoscopy as less

invasive alternative test,

received CE Mark (Europe) in

SEP 2009, ClinicalTrials.gov



GTx, Inc. GTXI $3.29 $206 12/31/11

Phase 2b

Clinical Trial

CAPESARIS

(GTx-758)

(oral

selective

estrogen

receptor

alpha

agonist)

In JUN 2011, began open-

label P2b trial compared to

Lupron Depot for first-line

treatment advanced prostate

cancer, expects to report

primary efficacy results by yr-

end 2011, expects to begin

two addt’l P2 trials during

2H11 8/26/11

GTx, Inc. GTXI $3.29 $206 3/31/13

Pivotal Phase 3

Clinical Trials

OSTARINE

(GTx-024)

(selective

androgen

receptor

modulator)

In JUL 2011 began two

ongoing pivotal P3 trials for

prevention and treatment of

muscle wasting in patients

with non-small cell lung

cancer (NSCLC),


NCT01355484 and



Guided

Therapeutic

s GTHP $0.90 $44 12/31/11 Feasibility Study

Biophotonic

Disease

Detection

Platform

On 8/10/11, announced start

of human testing for light-

based detection of Barrett’s

Esophagus (esophageal

cancer precursor condition),

being developed w/ Konica

Minolta, expects to enroll 40

subjects and complete testing

by yr-end 2011 8/10/11

Guided

Therapeutic

s GTHP $0.90 $44 3/31/12

FDA Advisory

Panel Meeting

(1Q12 est) to

Review PMA

Filing, CE Mark

(Europe)

Decision (2H11

estimate)

LuViva

Advanced

Cervical

Scan and

Guide

On 9/1/11, announced that it

provided requested info to

FDA in support of PMA

submission , FDA may

request more info or schedule

advisory panel mtg during

1Q12 9/1/11

Halozyme

Therapeutic

s HALO $6.28 $651 4/30/12

FDA Decision

(BLA)

HyQ

(subcutaneo

us immune

globulin / IG

formulation

with

rHuPH20 /

hyaluronidas

e enzyme)

In JUL 2011, licensee BAX

reported positive P3 results for

novel formulation of IG (dosed

once every 3-4 weeks) used in

pts w/ primary

immunodeficiency in support

of a BLA filing which has

expected FDA decision date of


month review period, HALO

due to receive milestone pmts

and royalties under 2007 agmt 7/11/11

Halozyme

Therapeutic

s HALO $6.28 $651 9/30/11

Phase 2 Clinical

Trials

Ultrafast

PH20-

Insulin

Analogs

In SEP 2010, began two P2

trials w/ one in Type 1

diabetics (Clinical Trials.gov

ID NCT01194245) and one in

Type 2 diabetics


NCT01194258) w/ pt enrlmt

completed in JAN 2011 and

results expected 3Q11 6/21/11

Hansen

Medical HNSN $3.82 $210 12/31/11

FDA Decision

510(k) Medical

Device

Magellan

Robotic

System

On 7/25/11, announced CE

Mark received for system to

assist physicians for

peripheral vascular disease

device placements (e.g.

stents), expects full market

launch in Europe during

1H12, announced FDA 510(k)

submission in APR 2011 w/

estimated FDA decision

during 4Q11 but no formal

decision deadline 7/25/11

HeartWare HTWR $59.86 $834 12/31/11

FDA Decision

(PMA) (Medical

Device)

HeartWare

Ventricular

Assist

System

On 3/16/11, FDA accepted

PMA submission for full

review, seeking marketing

clearance as a bridge to heart

transplantation for patients

with end-stage heart failure,

official filing date is 12/28/10

w/ estimated FDA decision

during 4Q11 but no formal

decision deadlines for medical

devices 3/17/11

Horizon

Pharma HZNP $8.16 $156 9/30/11

FDA New Filing

(NDA)

LODOTRA

(NP01)

(programme

d-release

low-dose

prednisone)

Expects to file NDA during

3Q11 seeking approval for

novel formulation of approved

steroid drug for reduction in

morning stiffness associated

with rheumatoid arthritis (RA),

approved in Europe, IPO on

7/28/11 for 5.5M shares

priced @$9 7/29/11

Idenix

Pharma IDIX $5.43 $522 12/31/11

Pending Phase

2b Clinical Trial

IDX184

(hepatitis C

virus, HCV

nucleotide

pro-drug)


removed full clinical hold (due

to elevated liver function tests)

and placed on partial clinical

hold, expects to begin a P2B

trial during 2H11 in combo w/

pegylated interferon and

ribavirin for hepatitis C virus

(HCV) infection 6/21/11

Idera

Pharma IDRA $1.65 $46 12/31/11

Pending Phase

2 Clinical Trial

IMO-2125

(TLR9

Agonist)

In APR 2011, announced

delay to planned start of 12-

week P2 trial in combo w/

ribavirin for chronic hepatitis C

virus (HCV) infection pending

chronic non-clinical toxicology

study data that is expected

2H11 8/5/11

Immuno-

Cellular

Therapeutic

s IMUC $1.55 $44 12/31/12

Phase 2b

Clinical Trial

ICT-107

autologous

(patient-

derived)

dendritic cell

(DC)

therapeutic

cancer

vaccine

In FEB 2011, first pt enrolled

in trial for treatment of brain

cancer (GBM), expects to

enroll up to 200 pts by mid-

2012 and complete P2B trial

by late 2013 w/ interim results



NCT01280552 7/22/11

Immuno-

medics IMMU $3.83 $289 3/31/14

Pivotal Phase 3

(EMBODY 1 &

2) Clinical Trials

Epratuzuma

b

(monoclonal

antibody

targeting

CD22

receptors on

B-

lymphocytes

)

Licensed to UCB for all auto-

immune indications, began

enrolling pts in two pivotal

trials in DEC 2010 for

moderate to severe lupus

(SLE) to enroll 780 pts each

and last max of 54 weeks,


NCT01262365 &



ImmunoGen IMGN $10.40 $794 6/30/12

Phase 2 Clinical

Trial, Pivotal

Phase 3

(EMILIA) Clinical

Trial

Trastuzuma

b DM1 (T-

DM1)

Partner Roche expects to

report P3 (EMILIA) trial data

mid-2012 to support FDA and

EMA approval filings soon

after for second-line treatment

of HER2+ metastatic breast

cancer (MBC), ongoing P3

(MARIANNE) study for first-

line treatment of MBC to

support planned approval

filings in 2014 8/17/11

Impax Labs IPXL $18.33 $1,205 12/31/11

FDA New Filing

(NDA)

IPX066

(extended-

release

carbidopa

plus

levodopa)

On 8/15/11, announced third

pivotal P3 trial for pts w/

advanced Parkinson’s disease

(PD) to improve motor

symptoms met primary endpt,

plans to file NDA during 4Q11

w/ partner GSK expected to

file MAA for EU approval in

2012 8/15/11

Incyte INCY $14.50 $1,830 3/31/12

Phase 2b

Clinical Trial

LY3009104

(an oral

JAK1/JAK2

inhibitor)

(formerly

INCB28050)

On 10/20/10, earned $19M

milestone payment from

partner LLY, which initiated

Phase 2b study for treatment

of rheumatoid arthritis (RA),


during 2H11 and complete

three-month double-blind,

placebo-controlled phase of

the trial in early 2012 (1Q12

estimate) 7/28/11

Incyte INCY $14.50 $1,830 6/30/13

Pivotal Phase 3

(RESPONSE)

Clinical Trial

under SPA

Ruxolitinib

(INCB18424

)

On 7/28/11, announced enrlmt

behind schedule and plans to

work w/ FDA to amend study

protocol to accelerate pt

recrmt, ClinicalTrials.gov ID


expected mid-2013, partnered

w/ NVS 7/28/11

Incyte INCY $14.50 $1,830 12/3/11

FDA Decision

(NDA), MAA /

EMA (Europe)

Decision

Ruxolitinib

(INCB18424

)


accepted NDA w/ PDUFA


under six-month priority rvw

seeking approval for treatment

of myelofibrosis, ex-US

partner NVS submitted MAA in

JUN 2011 w/ estimated EU

approval decision by late 2011-

early 2012 8/3/11

Infinity

Pharma INFI $7.00 $186 3/31/12

Phase 2 Clinical

Trials

IPI-926

(Hedgehog

Pathway

Inhibitor anti-

cancer

agent)

In FEB 2011 began P2 portion

of P1b/2 trial in combo w/

GEMZAR for pancreatic

cancer and expects to

complete pt enrlmt by year-

end 2011, ClinicalTrials.gov


expected 1Q12, P2 sarcoma

trial w/ ClinicalTrials.gov ID


expected 4Q12, expects to

begin P2 trial for myelofibrosis

during 3Q11 6/23/11

Inhibitex INHX $3.07 $240 3/31/12

Phase 2 Clinical

Trials

INX-189

(oral, once-

daily NS5b

nucleotide

inhibitor)

On 8/8/11, reported plans to

begin P2 trial next week for

treatment of chronic hepatitis

C virus (HCV) infection in

combo w/ pegylated interferon

and ribavirin in genotype (GT)

2 and 3 treatment naïve HCV

pts w/ interim data expected

1Q12, expects to begin P2

trial in GT1 treatment naïve

HCV pts during 4Q11 8/8/11

Inovio

Pharma INO $0.67 $85 12/31/11

Phase I Clinical

Trials

Pennvax

HIV DNA

Vaccines

Expects to final immune

response and safety data from

two P1 trials in HIV-positive

volunteers by yr-end 2011 8/8/11

Inovio

Pharma INO $0.67 $85 12/31/11

Phase I Clinical

Trials

SynCon

Universal

Flu Vaccine

(VGX-3510)

(H1N1

seasonal +

H5N1 avian

influenza)

On 7/14/11, reported

significant immune responses

generated in P1 trial of avian

flu vaccine, launched second

P1 trial to evaluate minimally

invasive skin delivery w/ data

expected 4Q11, subjects

being screened for a P1 trial of

universal flu vaccine w/ interim

data expected 1Q12 7/14/11

Inovio

Pharma INO $0.67 $85 12/31/13

Phase 2 Clinical

Trial

VGX-3100

(cervical

cancer DNA

vaccine

delivered via

Cellectra

electro-

poration

device)

In MAR 2011, began trial for

cervical dysplasia (CIN 2/3 or

CIN 3 pre-cancerous

condition) caused by HPV,

expects to enroll 148 pts at 25

global sites, ClinicalTrials.gov



InSite Vision INSV $0.53 $50 12/31/11

Phase 2 Clinical

Trial

AZASITE

(Azithro-

mycin

Ophthalmic

Solution)

In DEC 2010, partner Inspire

Pharma (acquired by MRK in

APR 2011) began trial in up to

300 pts w/ blepharitis (eyelid

inflammation) at 25 US sites,

expects to report data during

2H11, INSV receives

AZASITE royalties 4/6/11


Pending Pivotal

Phase 3 Clinical

Trial under SPA

DexaSite

(ISV-305)

(dexa-

methaosone

), AzaSite

Plus (ISV-

502) (dexa-

methasone

& azithro-

mycin)

On 5/25/11, announced SPA

agmt w/ FDA for P3 trial of

AzaSite Plus and DexaSite in

pts w/ blepharitis (chronic

eyelid inflammation), expects

to enroll 900 pts in four arms,

estimate to begin trial during

2H11 5/25/11


Pending Phase

3 Clinical Trial

ISV-303 (low-

dose

bromfenac

delivered

with

DuraSite

technology)

On 3/22/11, reported Phase 1

/ 2 trial top-line results met

primary endpt to reduce pain

and inflammation associated

with eye surgery, plans to

discuss design of pivotal

Phase 3 program w/ FDA after

final study analysis (2H11

estimate for Phase 3 trial) 3/22/11

Insmed INSM $4.10 $102 12/31/11

Pending Pivotal

Phase 3 Clinical

Trials -- FDA

Clinical Hold

ARIKACE

(liposomal

amikacin for

inhalation)


placed clinical hold on

planned P3 trials for treatment

of cystic fibrosis (CF) pts w/

Pseudomonas lung infections

and pts w/ non-tuberculous

mycobacterial lung disease

based on concerns over long-

term rat cancer study, expects

to reply w/in 30 days for 2H11

estimate for FDA reply 8/2/11

Insulet PODD $16.85 $797 12/31/11

FDA Decision

510(k) Medical

Device

OmniPod

Insulin

Managemen

t System

(next-

generation

insulin

pump)

In MAY 2011 submitted 510(k)

seeking FDA marketing

clearance for next-generation

insulin pump (33% smaller,

25% lighter) w/ estimated FDA

decision by yr-end 2011,

received CE Mark clearance

(Europe) in late JUL 2011 8/3/11

INSYS

Therapeutic

s (planned

IPO as

INRX) NEOL $9.05 $4 12/31/11

Phase 2 Clinical

Trial

LEP-ETU

(novel

formulation

of anti-

cancer drug

paclitaxel or

TAXOL)



expected during 4Q11 for an

open-label study being

conducted in India for pts w/

metastatic breast cancer,

novel formulation designed to

increase solubility, decrease

infusion-related side effects,

and allow for administration of

higher doses of the active

drug 6/28/11

INSYS

Therapeutic

s (planned

IPO as

INRX) NEOL $9.05 $4 1/4/12

FDA Decision

(NDA)

Fentanyl

Sublingual

Spray

(Fentanyl SL

Spray)

Expected PDUFA decision

goal date of 1/4/12 under

standard 10-month review,

seeking FDA approval for

treatment of breakthrough

cancer pain episodes as a

single-unit dose device that is

designed for rapid absorption

and pain relief 5/20/11

IntelGenx IGXT $0.69 $32 11/13/11

FDA Decision

505(b)(2) NDA

CPI-300

(450mg

bupropion)

(novel, high-

strength

formulation)

On 8/2/11, announced Pillar5

Pharma passed pre-approval

manufacturing inspection w/

no outstanding issues

reported by FDA (Form 483),


11/13/11 under six-month

Class 2 NDA resubmission in

response to FEB 2010 CRL

issued by FDA 8/2/11

InterMune ITMN $25.26 $1,535 3/31/13

Pivotal Phase 3

(ASCEND)

Clinical Trial

ESBRIET

(pirfenidone)

On 7/7/11, announced start of

pt enrlmt in pivotal P3 trial for

treatment of idiopathic

pulmonary fibrosis (IPF) (lung

disease), expects to complete

enrlmt during 1H12 and report

results mid-2013 to support

potential NDA resubmission to

CRL during 2H13,


NCT01366209, received EU

approval in MAR 2011 7/8/11

IRIS

International IRIS $7.78 $139 12/31/11

FDA Decision

510(k) Medical

Device

NADiA

ProsVue (a

prostate

cancer

prognostic

test)

During 2Q11 submitted all

requested info and software

validation data in support of

FDA 510(k) submission


to identify men at low risk of

prostate cancer recurrence

after surgical removal w/

estimate for FDA decision by

late 2011-early 2012 8/4/11

Ironwood

Pharma IRWD $11.39 $1,143 6/9/12

FDA Decision

(NDA)

Linaclotide

(orally

administere

d, once-daily

medication

that

increases

fluid in the

intestinal

tract)

On 8/9/11, announced NDA

filing w/ partner FRX seeking

approval for irritable bowel

syndrome with constipation

(IBS-C) and chronic

constipation (CC), expected

FDA decision by 6/9/12 under

standard 10-month review

period, European partner

Almirall expects to submit

MAA during 2H11 8/10/11

Isis Pharma ISIS $7.01 $699 6/30/12

FDA New Filing,

MAA / EMA

(Europe)

Decision

KYNAMRO

(mipomerse

n) (RNA-

based ApoB

synthesis

inhibitor)

On 7/28/11, announced MAA

filing seeking EMA approval

for treatment of two genetic

disorders (HoFH & HeFH)

resulting in very high

cholesterol levels w/ estimated

decision by mid-2012, expects

to file for FDA approval later

this year (2H11 estimate),

partnered w/ SNY 7/28/11

ISTA

Pharma ISTA $3.88 $130 12/31/11

Phase 3 Clinical

Trials

BROMDAY

Lower

Concentratio

n

In MAY 2011, began P3 trials

for low-dose BROMDAY

(0.09% once-daily dose

approved by FDA in OCT

2010 for post-op inflammation

and pain for cataract eye

surgery), expects to report

results during 4Q11 7/29/11

ISTA

Pharma ISTA $3.88 $130 12/31/11

Phase 3 Clinical

Trial under SPA

REMURA

(bromfenac

ophthalmic

solution for

dry eye)

On 7/28/11, announced first of

two P3 trials under SPA failed

to meet co-primary endpts for

treatment of dry eye, expects

to report top-line results from

second P3 trial during 4Q11 7/29/11

Keryx

Biopharma KERX $3.80 $265 6/30/12

Pivotal Phase 3

Clinical Trials

(US and EU)

under SPA (and

equivalent EMA

designation for

Europe)

ZERENEX

(ferric

citrate) (an

iron-based

phosphate

binder to

treat

elevated

phosphorus

in dialysis

patients)

In NOV 2010, reported short-

term efficacy study met all

primary and secondary

endpts, long-term safety study

(58-week study in approx. 300

patients) initiated SEP 2010

and expects to complete

during 2Q12 to support

potential NDA filing 3Q12,

Japan partner JT Torii began

Phase 3 program APR 2011 7/8/11

Keryx

Biopharma KERX $3.80 $265 12/31/11

Pivotal Phase 3

(X-PECT)

Clinical Trial

under SPA

Perifosine

(KRX-0401)

(PI3K/Akt

pathway

inhibitor anti-

cancer

agent)

On 8/31/11, announced indpt

data safety monitrg bd

completed interim analysis

and recm’d trial continue as

planned in pivotal P3 trial for

metastatic colorectal cancer,

North American rights to

perifosine in-licensed from

AEZS, FDA Fast Track status,




Keryx

Biopharma KERX $3.80 $265 12/31/12

Pivotal Phase 3

Clinical Trial

under SPA

Perifosine

(KRX-0401)

(PI3K/Akt

pathway

inhibitor anti-

cancer

agent)

In DEC 2009, began relapsed

/ refractory multiple myeloma

(MM) pivotal trial,




Orphan Drug / Fast Track

status, North American rts to

perifosine in-licensed from

AEZS 7/8/11

Lexicon

Pharma LXRX $1.18 $398 6/30/12

Phase 2b

Clinical Trial

LX4211

(once-daily

oral

selective

SGLT2

inhibitor)


pts w/ type 2 diabetes in P2B

trial expected to enroll 285

adults not adequately

controlled on metformin over

12 weeks to evaluate several

doses, ClinicalTrials.gov ID



Ligand

Pharma LGND $13.89 $273 12/31/11

Phase 3

(ENABLE-1 and

2) Clinical Trials

PROMACTA

(eltrombopa

g)

On 7/26/11, partner GSK

reported positive results in first

of two P3 trials w/ full results

expected during 2H11 to


during 2Q12 to expand

approval to include treatment

of pts w/ hepatitis C related

thrombocytopenia (low

platelets) 7/26/11

Ligand

Pharma LGND $13.89 $273 12/31/11

Phase 2 Clinical

Trial (4Q11),

Pending Pivotal

Phase 3 Clinical

Trial (1Q12)

Captisol-

enabled

melphalan

Expects to report full results of

ongoing P2 trial during 4Q11

w/ planned start of pivotal P3

trial in early 2012 (1Q12

estimate) that is designed to

enroll 60 pts and support

505(b)(2) NDA filing by mid-

2013 contingent upon trial

data, being developed as

improved formulation of

marketed drug for use in

multiple myeloma pts as pre-

transplant chemotherapy 6/22/11

Lpath LPTN $0.96 $58 6/30/12

Phase 1b

Clinical Trial

iSONEP

(S1P

inhibitor

antibody)

In DEC 2010, entered deal w/

PFE to develop treatment for

wet age-related macular

degeneration (wet AMD) and

other eye disorders w/ upfront

payment of $14M and cost-

sharing agmt,


NCT01334255 for P1b trial for

treatment of wet AMD

complication (PED) w/ results


MannKind MNKD $3.14 $411 9/30/12

Phase 2 Clinical

Trial

MKC1106-

MT

(therapeutic

cancer

vaccine,

immuno-

therapy)

In OCT 2010, first patient

dosed to evaluate safety /

tolerability and immune /

clinical response for advanced

melanoma (skin cancer),




MannKind MNKD $3.14 $411 12/31/12

Pivotal Phase 3

Clinical Trials

(Study 171 and

174) to Support

NDA

Resubmission

AFREZZA

(insulin

human

[rDNA

origin])

Inhalation

Powder

In AUG 2011 met w/ FDA to

confirm design of two pivotal

P3 trials to address JAN 2011

CRL, seeking approval for

inhaled insulin for treatment of

adults w/ type 1 & 2 diabetes,

estimate to complete trials by

FDA by late 2012-early 2013

to support planned NDA

resubmission 8/12/11

MAP

Pharma MAPP $12.70 $386 3/26/12

FDA Decision

(NDA)

LEVADEX

(dihydro-

ergotamine

or DHE oral

inhaler)


accepted for filing by FDA w/



month review, seeking

approval for orally inhaled

drug for treatment of acute

migraine headaches as a new

inhaled formulation of

approved drug DHE,

partnered w/ AGN 8/2/11

Medgenics MDGN $3.57 $36 12/31/11

Phase I / II

Clinical Trial

EPODURE

bio-pump

(six-month

erythropoieti

n / EPO

delivery)

Expects to report full results

during 4Q11 from ongoing

P1/2 dose-ranging trial for

treatment of anemia in pts w/

chronic kidney disease (CKD),


NCT00542568 8/23/11

Medicines

Company MDCO $13.81 $746 12/31/12

Pivotal Phase 3

Clinical Trials

(SOLO-1 and 2)

under SPA

Oritavancin

(glyco-

peptide intra-

venous

antibiotic)

In NOV 2010, announced SPA

agmt w/ FDA for pivotal trials

in pts w/ acute bacterial skin

and skin structure infection

(ABSSSI) to compare single

dose of oritavancin vs.

multiple doses of vancomycin,


NCT01252719 and



MediciNova MNOV $2.24 $34 12/31/11

Phase 2 (MN-

221-CL-007)

Clinical Trial

MN-221

(highly

selective ß2-

adrenergic

receptor

agonist)

In MAY 2011 reported 1Q11

results and provided guidance

to report results of ongoing P2

trial by year-end 2011,


NCT00838591 for intravenous

(IV) administration as add-on

to standard therapy in adults

w/ acute asthma

exacerbations 7/11/11

Medivation MDVN $17.48 $610 6/30/12

Phase 3 Clinical

Trial

Dimebon

(latrepirdine)

In APR 2011, reported P3 trial

for Huntington disease failed

to meet co-primary endpts,

ongoing P3 CONCERT trial

for mild-moderate Alzheimer’s

disease in combo w/ Aricept

top-line data expected during


NCT00829374, partnered w/

PFE 8/10/11

Medivation MDVN $17.48 $610 12/31/11

Phase 3

(AFFIRM and

PREVAIL)

Clinical Trials,

Phase 2 Clinical

Trials

MDV3100

(oral

androgen

receptor

antagonist)

Co-developed w/ Astellas,

expects to report interim

results for P3 AFFIRM trial w/


NCT00974311 by yr-end

2011 and top-line results in

2012, PREVAIL P3 study

began SEP 2010, began

Phase 2 TERRAIN study for

advanced prostate cancer

MAR 2011 and open-label trial

for first-line treatment in MAY

2011 8/10/11

MELA

Sciences MELA $2.26 $57 12/31/11

FDA Decision

(PMA) Medical

Device /

Diagnostic, CE

Mark (Europe)

Decision

MelaFind

(computer

vision

system for

early

melanoma

detection)

In FEB 2011 filed amended

PMA to limit proposed use to

dermatologists based on NOV

2010 advisory panel feedback,

on 5/12/11 submitted second

amendmt to PMA w/ training

program, has requested mtg

w/ FDA, 2H11 estimate for

FDA reply or update on PMA

status along w/ pending CE

Mark (Europe) submission 8/9/11

Micromet MITI $4.35 $400 3/31/13

Pivotal

European Phase

2 (BLAST)

Clinical Trial

(4Q12)

Blinat-

umomab

(MT103) (a

lymphoma-

directed,

recombinant

antibody)

In SEP 2010, began pivotal

EU trial in pts w/ minimal

residual disease B-precursor

acute lymphoblastic leukemia

(ALL), ClinicalTrials.gov ID


expected during 1Q13, in JUN

2011 reported P2 interim trial

data in adults w/ relapsed ALL

w/ 75% of pts achieving

complete remission (CR) 6/10/11

Myrexis

(formerly

Myriad

Pharma) MYRX $2.77 $72 6/30/13

Phase 2a and

Phase 2b

Clinical Trials

AZIXA (MPC-

6827)

(verubulin)

In JUN 2011, presented P2a

brain cancer (GBM) trial data

w/ median overall survival of

9.9 month, began P2b trial in

DEC 2010 for front-line

treatment of GBM in combo w/

standard of care to enroll up to

120 GBM pts,




Nabi

Biopharma NABI $1.84 $79 12/31/11

Pivotal Phase 3

Clinical Trial

under SPA

NicVAX

(Nicotine

Conjugate

Vaccine)

In JUL 2011, announced first

pivotal P3 trial did not meet

primary endpt w/ 11% quit

smoking rate that was similar

to placebo, was well tolerated

w/ no safety concerns, second

pivotal P3 trial ongoing,



expected late 2011-early

2012, partnered w/ GSK 8/4/11

Nektar

Therapeutic

s NKTR $5.28 $604 9/30/12

Pivotal Phase 3

Clinical Trials

NKTR-118

(oral

peripherally-

acting opioid

antagonist)

In MAR 2011, partner AZN

announced enrlmt of first pt in

trial for treatment of opioid-

induced constipation (OIC),




Nektar

Therapeutic

s NKTR $5.28 $604 3/31/12

Phase 2 Clinical

Trials, Pending

Phase 3 Clinical

Trial

NKTR-102

(topo-

isomerase I

inhibitor,

long-acting

polymer

conjugate )




open-label P2 trial in pts w/

metastatic platinum-resistant

ovarian cancer, plans to begin

P3 trial in 2011, also plans to

begin P3 trial for metastatic

breast cancer w/ P2 trial

results to be presented on 9-

Sept 8/5/11

Neoprobe NEOP $2.79 $248 6/30/12

Pending Pivotal

Phase 3 Clinical

Trial w/ SPA

Request

RIGScan

CR (tumor-

specific

targeting

agent)

On 3/7/11, completed pre-IND

mtg w/ FDA including SPA

request as part of amended

IND filing as radio-

pharmaceutical product for

detection of colorectal cancer

tumors, expects to finalize P3

trial protocol during 2H11 and

begin manufacturing to

support start of trial during

1H12 5/27/11

Neoprobe NEOP $2.79 $248 12/31/12

Phase 3 Clinical

Trial Lymphoseek




ongoing P3 trial in pts w/ head

and neck cancer, being

evaluated as a sentinel lymph

node tracing agent 7/28/11

Neoprobe NEOP $2.79 $248 10/10/11

FDA Reply to

Accept NDA

Filing and Issue

PDUFA

Decision Date Lymphoseek


filing seeking approval for use

in Intraoperative Lymphatic

Mapping for breast cancer and

melanoma, expected FDA

reply within 60 days

(10/10/11) to accept NDA for

review and issue PDUFA

decision goal date 8/11/11

NeoStem NBS $0.65 $64 12/31/11

Pending Phase

2 Clinical Trial

AMR-001

(autologous

or patient-

derived

bone

marrow

based

regenerative

cell therapy)

Expects to begin P2 trial

during 4Q11 for treatment of

heart attack (acute myocardial

infarction or AMI) and P1 trial

for heart failure in 2012,

expects to complete enrlmt in

P2 trial w/in 12 months and

report top-line data by mid-

2013 8/24/11

Nephros NEPH $2.07 $21 3/31/12

FDA Decision

510(k)

Hemodia-

filtration

(HDF)

System



marketing clearance for

dialysis filtration system

(OLpur MD220 diafilter and

OLpur H2H Hemodiafiltration

module) for pts w/ end-stage

renal (kidney) disease,

estimate for FDA reply during

1Q12 but no formal decision

deadlines 8/12/11

Neptune NEPT $3.31 $169 9/30/12

Phase 2 Clinical

Trial

CaPre

(Neptune

Krill Oil

derived

omega-3

fatty acid)

On 6/30/11, received Health

Canada clearance to conduct

P2 randomized, double-blind,

placebo-controlled trial in

approx. 430 pts w/ moderately

to very high triglycerides,

estimate to complete trial by

mid-2012 (2-3Q12 estimate) 7/29/11

Neuralstem CUR $1.25 $61 10/31/11

Phase 1a

Clinical Trial

NSI-189

(neuron

growth

stimulator)

In JUN 2011, was cleared to

dose final cohort of pts in

ongoing P1A trial


NCT01310881) to test safety

of experimental drug for major

depression, expects to

complete the trial in summer

to support planned start of

P1B trial by early fall (OCT

2011 estimate) 8/11/11

Neuralstem CUR $1.25 $61 6/30/12

Phase I Clinical

Trial

NSI-

566RSC

(human

spinal cord

derived

neural stem

cells)

On 6/14/11, updated status of

ongoing P1 trial for treatment

of ALS (amyotrophic lateral

sclerosis, or Lou Gehrig's

disease), safety monitoring

board reviewed data from first

12 pts and approved

continuation of trial, next three

pts due to receive injections

pending FDA approval (3Q11

estimate), ClinicalTrials.gov ID

NCT01348451 (mid-2012

results) 6/14/11

Neurocrine

Biosciences NBIX $5.76 $318 9/30/11

Pending Phase

2 Clinical Trial

NBI-98854

(VMAT2

Inhibitor)

Expects to begin P2 trial in

SEP 2011 for treating the

symptoms of tardive

dyskinesia (TD) in 32 pts over

two-week dosing period,

expects to report three-month

toxicology results during

4Q11, plans to conduct P2B

trial in early 2012 7/29/11

Neurocrine

Biosciences NBIX $5.76 $318 9/30/11

Pending Phase

3 Clinical Trials

ELAGOLIX

(orally active

non-peptide

GnRH

antagonist)

Expects to begin P3 trial for

uterine fibroids during 3Q11

and begin P3 trial for

endometriosis during 4Q11,

partnered w/ ABT 7/29/11

Nile

Therapeutic

s NLTX $0.65 $23 12/31/11

Phase I Clinical

Trial

Cenderitide

(natriuretic

peptide)

As of 8/15/11, all subjects

enrolled in low-dose cohort in

Part B of ongoing P1 trial for

heart failure pts (utilizing

partner Medtronic’s insulin

pump technology), expects to

complete enrlmt during 4Q11,




Northwest

Biotherapeut

ics NWBO $0.46 $43 6/30/12

Phase 2 Clinical

Trial

DCVax-

Brain

(patient-

derived,

dendritic cell

immune

therapy)

In MAY 2011, announced 4

US sites actively enrolling new

pts in ongoing P2 trial for

treatment of brain cancer

(GBM), ClinicalTrials.gov ID



NovaBay

Pharma NBY $0.85 $24 12/31/12

Phase 2b

Clinical Trial

NVC-422

(Aganocide

anti-

microbial

compound)

Partner Galderma will manage

P2B trial for treatment of for

impetigo (skin infection) w/

results expected during 2H12 8/17/11

NovaBay

Pharma NBY $0.85 $24 12/31/11

Phase 2 Clinical

Trial

NVC-422

(Aganocide

anti-

microbial

compound)

Expects to report prelim

results from ongoing P2 trial

for prevention of urinary

catheter blockage and

encrustation during 4Q11 to

support start of pivotal trial in

2012, being developed as

medical device (PMA) for

maintenance of catheter

patency 8/17/11

Novavax NVAX $1.73 $199 12/31/11

Pending Phase

1 and 2 Clinical

Trials

Seasonal

and

Pandemic

Influenza

VLP (Virus-

Like

Particle)

Vaccines

Awarded HHS BARDA

contract in FEB 2011

including $97M for 36-month

base period, expects to begin

seasonal flu vaccine P2 trials

during 4Q11 and pandemic flu

vaccine P1 trial in early 2012 8/15/11

Novavax NVAX $1.73 $199 9/30/11

Phase I Clinical

Trial

Respiratory

Syncytial

Virus Fusion

Protein

(RSV-F)

Vaccine

During 2Q11, completed

enrlmt of 150 subjects in

ongoing P1 trial to assess

safety and immune response

in adults for experimental RSV

vaccine w/ interim top-line

data expected during 3Q11,


NCT01290419 8/15/11

NPS

Pharma NPSP $6.85 $589 12/31/11

Pivotal Phase 3

(REPLACE)

Clinical Trial

NPSP558

(human

parathyroid

hormone or

PTH)

In MAR 2011, announced

randomization of last pt (135)

in pivotal trial for bio-

engineered formulation of

PTH for hormone replacement

therapy to treat hypo-

parathyroidism, expects to

report top-line data during

4Q11, will present data from

two investigator-initiated

studies in SEP 2011 at

medical mtg 8/3/11

NPS

Pharma NPSP $6.85 $589 12/31/11

FDA New Filing

(NDA), EMA /

MAA (Europe)

Decision

Gattex (US

brand

name),

Revestive

(EU brand

name)

(teduglutide)

On 8/18/11, submitted initial

CMC section of NDA seeking

approval as once-daily

subcutaneous treatment for

short bowel syndrome (SBS),

expects to complete NDA filing

4Q11, EU partner Nycomed

submitted MAA for EU

approval in MAR 2011

seeking approval w/ estimated

decision during 4Q11 8/18/11

Nymox

Pharma NYMX $7.67 $251 12/31/12

Pivotal Phase 3

Clinical Trials

NX-1207

(administere

d by

urologist at

office in a

few minutes

for benign

prostatic

hyperplasia

or BPH)


NCT00918983 &


expected from both pivotal P3

trials during 2H12, in APR

2011 reported new 39-45

month follow-up data w/ no

safety concerns and 11.5

point improvement in BPH

(enlarged prostate) symptoms 8/17/11

Omeros OMER $3.75 $83 6/30/12

Phase 3 Clinical

Trials

OMS103HP

(novel

formulation

of

ketoprofen,

amytriptyline

, and

oxymetazoli

ne)

Phase 3 clinical program will

evaluate safety and efficacy in

improving post-operative joint

function and reducing pain

following arthroscopic partial

meniscectomy (knee) surgery

w/ two P3 trials and first trial

(North America) data expected

during 1H12, plans to begin

second trial in Europe during

4Q11, a novel formulation of

approved drugs 6/13/11

Omeros OMER $3.75 $83 6/30/12

Phase 3 Clinical

Trials

OMS302

(novel

formulation

of anti-

inflammatory

and pupil

dilation /

mydriasis

drugs)

Phase 3 program will evaluate

the safety and efficacy in

patients undergoing intra-

ocular (eye) lens replacement

surgery, first trial (North

America) w/ data expected

during 1H12 and second trial

in Europe will begin following

discussions to validate pivotal

study design and endpoints 6/13/11

Omthera

Pharma

(PRIVATE) N/A N/A N/A 12/31/11

Pivotal Phase 3

(EVOLVE and

ESPRIT)

Clinical Trials

under SPA

EPANOVA

(novel

formulation

of EPA and

DHA omega-

3 fatty acids

to improve

bio-

availability)



expected 4Q11 (plans to file

NDA based on these results)

for P3 (EVOLVE) study for

treatment of very high

triglycerides (TGs) (over 500),



expected 3Q12 for P3

(ESPRIT) study as add-on to

statin drugs for TGs of 200-

500 to expand use 8/16/11

OncoGenex

Pharma OGXI $10.50 $102 12/31/13

Phase 3

(SATURN and

SYNERGY)

Clinical Trials

Custirsen

Sodium

(OGX-011 /

TV-1011)

Phase 3 SATURN trial for


cancer (CRPC) pts w/ disease

progression, ClinicalTrials.gov


expected during 4Q13, Phase

3 SYNERGY trial



expected during 4Q13 for first-

line treatment CRPC,

partnered w/ TEVA 8/5/11

Oncolytics

Biotech ONCY $3.58 $255 12/31/11

Pivotal Phase 3

Clinical Trial

under SPA (mid-

2012), Phase 2

Clinical Trials

(2H11)

REOLYSIN

(Reovirus)

In MAY 2010, began pivotal

P3 trial in combo w/ paclitaxel

and carboplatin for platinum-

refractory head and neck

cancer, ClinicalTrials.gov ID


expected mid-2012, multiple

ongoing P2 trials including

pancreatic cancer

(NCT00998322 – 4Q11),

head / neck cancer

(NCT00753038 – 2H11), lung

cancer (NCT00861627 –

2H11) 7/15/11

Oncothyreon ONTY $7.17 $301 6/30/12

Pivotal Phase 3

(START)

Clinical Trial

under SPA

STIMUVAX

(BLP25

liposome

therapeutic

cancer

vaccine)

In DEC 2010, indpt data

monitoring cmte (DMC) gave

ok to continue study in pts w/

non-small cell lung cancer

(NSCLC) w/ second interim

analysis expected 2H11 and

top-line data expected by mid-

2012 for pivotal P3 trial in

1476 pts, partnered w/ Merck

KGaA, ClinicalTrials.gov ID

NCT00409188 6/22/11

Oncothyreon ONTY $7.17 $301 3/31/13

Phase 2 Clinical

Trials

PX-866 (PI-

3-kinase /

PTEN / AKT

pathway

inhibitor anti-

cancer

agent)

Three ongoing P2 trials for

solid tumors, metastatic

colorectal or head / neck

cancer, and brain cancer

(GBM) w/ initial data expected

during 2H11 and final data


ClinicalTrials.gov IDs include

NCT01204099,

NCT01252628, and

NCT01259869 6/22/11

Onyx

Pharma ONXX $31.42 $1,997 12/31/11

FDA New Filing

(NDA) (3Q11),

Pivotal Phase 3

Combo

(ASPIRE)

Clinical Trial

(4Q11)

Carfilzomib

(selective

proteasome

inhibitor anti-

cancer

agent)

On 3/29/11, announced plans

to expand (from 84 to 300 pts)

pivotal European FOCUS trial

for relapsed / refractory

multiple myeloma (MM),

expects to complete NDA filing

during 3Q11 seeking

accelerated approval in US for

MM w/ FDA Fast Track status 7/1/11

Opko Health OPK $3.93 $1,132 12/31/11

Validation Study

(Molecular

Diagnostic Test)

Alzheimer’s

Disease

(AD)

Antibody-

Based

Diagnostic

Blood Test

Ongoing validation study in

580 pts w/ positive initial

results reported from 140

samples, final results

expected by late 2011 (4Q11

estimate), non-exclusive

collaboration agmt w/ BMY for

lead AD test w/ lung and

pancreatic markers

undergoing validation w/

expected completion by 4Q12 7/14/11

OraSure OSUR $6.87 $323 12/31/11

FDA New Filing

(PMA)

OraQuick

HIV OTC

Test

Expects to complete ongoing

clinical testing during 3Q11 for

over-the-counter version of

HIV diagnostic test via oral

fluids w/ enrlmt closed on

8/9/11, expects to file PMA in

three modules w/ first

expected in AUG 2011 and

expected completion of PMA

filing by yr-end 2011 8/9/11

Osiris

Therapeutic

s OSIR $5.04 $165 12/31/11

Phase 2 Clinical

Trial

Prochymal

(remestemc

el-L) (adult

bone

marrow

derived stem

cells for IV

infusion)

In MAR 2011, completed

enrlmt for patients

experiencing first heart attack

(acute myocardial infarction),



expected during late 2011-

early 2012 8/12/11

Osiris

Therapeutic

s OSIR $5.04 $165 12/31/12

Phase 3 Clinical

Trial

Prochymal

(remestemc

el-L) (adult

bone

marrow

derived stem

cells for IV

infusion)




ongoing open-label P3 trial for

treatment-refractory moderate

to severe Crohn’s disease,

resumed enrlmt of pts in this

trial MAY 2010 8/12/11

OXiGENE OXGN $1.11 $16 6/30/12

Phase I Clinical

Trial

OXi4503

(combretast

atin A1

diphosphate

or CA1P)

In MAY 2011, began P1 trial

for treatment of acute

myelogenous leukemia (AML)

or myelodysplastic syndrome

(MDS), expects to report data

during 1H12,


NCT01085656 8/26/11

Pacira

Pharma PCRX $7.71 $133 10/28/11

FDA Decision

(NDA)

EXPAREL

(bupiv-

acaine

extended-

release

liposome

injection)

(300mg /

20mL)

On 6/14/11, announced three-

month delay due to addt’l info

submitted at FDA request,

new PDUFA decision goal

date is 10/28/11, seeking

approval for post-surgical pain

relief by local administration

for a novel, long-acting

formulation of approved drug

bupivacaine 6/14/11

Palatin PTN $0.72 $25 12/31/11

Phase I Clinical

Trial

AZD2820

(melanocorti

n receptor

partial

agonist)




ongoing P1 trial, being

developed as single-agent

therapy for obesity that is

given via subcutaneous

administration, developed in

collaboration w/ AZN 8/5/11

Palatin PTN $0.72 $25 12/31/12

Phase 2b

Clinical Trial

Bremelanoti

de

(previously

PT-141)

(melanocorti

n agonist)


pts in P2B trial for treatment of

female sexual dysfunction

(FSD), expects to enroll 400

pts at 40 sites w/ 16 wks of

treatment, expects to report

results during 2H12,


NCT01382719 8/5/11

Peregrine

Pharma PPHM $1.23 $87 12/31/11

Pending Phase

3 Clinical Trial

COTARA

(targeted,

anti-cancer

monoclonal

antibody,

radio-

isotope

combo)

In JUN 2011 presented P2

open-label trial data for brain

cancer (recurrent GBM) w/

interim median overall survival

of 8.8 months in 41 pts, plans

to meet w/ FDA during 4Q11

to discuss pivotal

development pathway 7/14/11

Peregrine

Pharma PPHM $1.23 $87 12/31/11

Phase 2 Clinical

Trials

Bavituximab

(monoclonal

antibody that

targets cell

membrane

component

phosphatidyl-

serine or

PS)

Expects P2 front-line lung

cancer (NSCLC) interim data

by yr-end 2011, P2 second-

line NSCLC data 1H12, P2

pancreatic cancer trial


NCT01272791 results mid-

2012), P2 hepatitis C virus

trial (NCT01273948) results

1Q12, on 8/24/11 reported

20.7 median overall survival

(P2 single-arm breast cancer

trial) 8/24/11

Pharmacycli

cs PCYC $11.32 $682 12/31/11

Phase 1 and 2

Clinical Trials

PCI-32765

(Btk inhibitor

anti-cancer

agent)

Plans to begin P3 trials mid-

2012 for two types of

lymphoma (CLL/SLL and

MCL), expects to report

interim P2 data for MCL,

combo safety data for CLL and

pilot data for DLBCL during

2H11 8/17/11

Pharmacycli

cs PCYC $11.32 $682 12/31/12

Phase I / II

Clinical Trial

PCI-27483

(Factor 7a or

FVIIa

inhibitor,

small

molecule

anti-cancer

agent)

Being evaluated in ongoing

P1/2 trial in pts w/ locally

advanced or metastatic

pancreatic cancer with P1

portion completed and expects

to complete enrollment of P2

portion by yr-end 2011,




Pharmacycli

cs PCYC $11.32 $682 12/31/11

Phase I / II

Clinical Trial

PCI-24781

(HDAC

inhibitor anti-

cancer

agent)

Ongoing P2 portion of trial in

pts w/ follicular and mantle cell

lymphoma w/ data expected

by yr-end 2011 8/17/11

Pharmasset VRUS $70.11 $5,287 9/30/11

Pending Phase

2b (QUANTUM)

Clinical Trial

PSI-938

(nucleotide

analog HCV

polymerase

inhibitor)

On 8/24/11 received FDA Fast

Track status for treatment of

chronic hepatitis C virus

(HCV) infection, expects to

begin interferon-free combo

P2 (QUANTUM) trial w/ PSI-

7977 during 3Q11 8/24/11

Pharmasset VRUS $70.11 $5,287 12/31/11

Phase 2b

(ELECTRON)

Clinical Trial

PSI-7977

(nucleotide

analog HCV

polymerase

inhibitor)

On 9/6/11, reported sustained

virologic response (SVR)

results from P2B PROTON

study in previously untreated

pts w/ hepatitis C virus (HCV)

genotype 1 infection 2/ 98% of

pts achieving SVR12 (12

wks), expects to begin combo

trial 3Q11 w/ PSI-938 and

report results of P2B

ELECTRON trial 2H11 9/6/11

Pharmasset VRUS $70.11 $5,287 12/31/11

Phase 2b

Clinical Trials,

Pending Phase

3 Clinical Trial

Mericitabine

(RG7128,

MCB)

(nucleoside

analog HCV

polymerase

inhibitor)

Partnered with Roche, expects

to begin Phase 2 trial for

hepatitis C virus (HCV)

genotype 2 / 3 pts during

1H11 and begin Phase 3

program during 2011 to

support potential regulatory

approval filings in 2013,

PROPEL / JUMP-C trial final

data expected during 4Q11 6/13/11

PolyMedix PYMX $0.60 $64 3/31/12

Phase 2 Clinical

Trial

PMX-60056

(a synthetic,

small

molecule

blood

thinner /

heparin

antagonist)

In FEB 2011, began P2 trial

for reversal of heparin blood

thinning effects in pts

undergoing Percutaneous

Coronary Intervention (PCI)

procedures, will enroll up to 40

pts in the US,



expected late 2011-early 2012

(1Q12 estimate) 6/21/11

PolyMedix PYMX $0.60 $64 12/31/11

Phase I and II

Clinical Trials

PMX-30063

(novel once-

daily

intravenous

or IV

antibiotic)

In SEP 2010, began P2 trial at

multiple sites in Canada and

Europe for treatment of acute

bacterial skin and skin

structure infections caused by

Staph aureus,




PositiveID PSID $0.20 $9 3/31/12

FDA Decision

510(k) iglucose



marketing clearance for

wireless communication

system to facilitate diabetes

mgmt by creating electronic

journals of blood glucose

readings and aid in insurance

reimbsmt, estimated FDA

reply during 1Q12 but no

formal decision deadlines for

medical devices 7/11/11

Pozen POZN $2.71 $81 3/31/12

Pivotal Phase 3

Clinical Trials

under SPA

PA32540

(325mg

enteric

coated

aspirin plus

40mg

omeprazole)


NCT00960869 and

NCT00961350 for ongoing six-

month pivotal P3 efficacy trials

w/ enrlmt completed and

results expected 1Q12,


NCT00995410 for open-label

non-randomized 12-month

safety study which is

completed w/ data analysis

ongoing to provide long-term

data for planned NDA filing

3Q12 8/29/11

Progenics PGNX $5.48 $185 12/31/11

Phase 3 Clinical

Trial

RELISTOR

Oral Tablets

(methylnaltr

exone)

In JUL 2011 completed pt

enrlmt in ongoing P3 trial for

treatment of opioid-induced

constipation (OIC) in chronic

non-cancer pain pts w/ top-

line results expected late 2011-

early 2012 8/9/11

Progenics PGNX $5.48 $185 4/27/12

FDA Decision

(sNDA)

RELISTOR

Subcutaneo

us Injection

(methylnaltr

exone)

On 8/30/11 announced FDA

accepted sNDA, seeking

expanded FDA approval for

treatment of opioid-induced

constipation (OIC) in pts w/

chronic non-cancer pain,


4/27/12 under standard 10-

month rvw period, partnered

w/ SLXP 8/30/11

PROLOR

Biotech PBTH $4.46 $159 6/30/12

Pending Phase

3 Clinical Trial

hGH-CTP

(bio-better

formulation

of human

growth

hormone)


P2 trial results for long-acting

once-weekly human growth

hormone (hGH) formulation for

treatment of GH deficiency in

adults, expects to begin P3

trial in 2012 (1H12 estimate) 8/4/11

Protalix

BioTherape

utics PLX $4.51 $386 2/1/12

FDA Decision

(NDA)

UPLYSO

(taligluceras

e alfa) (a

plant-cell

expressed

form of the

enzyme,

glucocerebr

osidase)


accepted NDA for review as

six-month Class 2

resubmission in response to

FEB 2011 CRL, seeking

approval for enzyme-based

treatment of Gaucher disease

w/ PDUFA decision goal date

of 2/1/12, submitted MAA for

EU aprvl in NOV 2010 w/ late

2011-early 2012 estimate for

decision, partnered w/ PFE 8/17/11

pSivida PSDV $4.44 $92 11/12/11

FDA Decision

(NDA) (Class 2

Resubmission),

MAA / MHRA

Decision

(Europe)

ILUVIEN

(fluocin-

olone

intravitreal

insert)

(sustained

eye drug

delivery

system)

In MAY 2011, licensee ALIM

announced NDA resubmission

to DEC 2010 CRL, seeking

approval for diabetic macular

edema w/ PDUFA decision

goal date of 11/12/11 under a

six-month Class 2 review w/

no advisory panel mtg to be

held, MAA filed for EU

approval in JUL 2010 w/

MHRA and expected decision

during 1Q12, PSDV due to

receive $25M on FDA

approval 8/31/11

Qiagen QGEN $14.41 $3,371 6/30/12

FDA Decision

(PMA)

therascreen

KRAS RGQ

PCR Kit

On 8/5/11, announced second

PMA submission seeking FDA

marketing clearance as

companion diagnostic for

detection of KRAS mutations

for use w/ two drugs for

treatment of metastatic

colorectal cancer, estimated

FDA decision mid-2012 but no

formal decision deadlines 8/5/11

QLT Inc. QLTI $7.07 $353 12/31/11

Phase 1b

Clinical Trial

QLT091001

(oral

synthetic

retinoid

replacement

therapy for

11-cis-

retinal)

Has completed enrlmt in

Leber Congenital Amaurosis

14-pt cohort and expects to

complete enrlmt in 14-pt

Retinitis Pigmentosa cohort by

yr-end 2011,




QLT Inc. QLTI $7.07 $353 9/30/11

Phase 2 Clinical

Trial

L-PPDS

(latanoprost

punctal plug

eye drug

delivery

system)

Expects to report 4-week

treatment results for reducing

intraocular (eye) pressure in

pts w/ glaucoma during 3Q11

for novel delivery system of

approved drug XALATAN eye

drops, ClinicalTrials.gov ID

NCT01229982 8/11/11

QRxPharma

(QRX.AX) QRXPY $7.01 $202 10/25/11

FDA Reply to

Accept NDA

Filing and Issue

PDUFA

Decision Goal

Date

MoxDuo IR

(immediate

release 3:2

fixed-dose

ratio combo

of morphine

and

oxycodone)

On 8/25/11, completed NDA

filing w/ six-month priority

review request following initial

submission in JUL 2011,

seeking FDA approval for

treatment of moderate to

severe acute pain, expected

FDA reply w/in two months

(10/25/11) to accept NDA

filing and issue PDUFA

decision goal date 8/25/11

Raptor

Pharma RPTP $4.18 $145 12/31/11

Phase 3 Open-

Label Safety

Extension

Study, FDA New

Filing (NDA)

DR

Cysteamine

Gelatin

Capsules

(delayed-

release,

enteric-

coated

microbead

formulation)

On 7/25/11, reported pivotal

P3 trial met primary endpt w/

no safety concerns vs.

approved treatment

(Cystagon) for metabolic

disorder (cystinosis), expects

to report six-month open-label

extension trial data during

4Q11 to support planned early

2012 (1Q12 estimate) NDA

filing 7/25/11

Regeneron

Pharma REGN $68.37 $6,265 11/18/11

FDA Decision

(BLA)

(11/18/11), FDA

New Filing

(BLA) (2H11)

EYLEA

(aflibercept

ophthalmic

solution)

(VEGF Trap-

Eye)

On 8/16/11, reported 3-month

delay to 11/18/11 for BLA

decision seeking approval for

treatment of wet age-related

macular degeneration, in JUN

2011 FDA Advisory Panel

voted 10-0 in favor of

approval, co-developed w/

Bayer (ex-US), expects to file

BLA 2H11 for treatment of

macular edema due to central

retinal vein occlusion 8/16/11

Regeneron

Pharma REGN $68.37 $6,265 12/31/11

FDA New Filing

(BLA) (2H11),

Phase 2 & 3

Clinical Trials

(2H11 to 2012)

ZALTRAP

(aflibercept)

(VEGF Trap)

In APR 2011, reported P3

VELOUR trial met primary

survival endpt for metastatic

colon cancer, plans to file for

FDA & EMA approval during

2H11, expects to report P2

AFFIRM final results (first-line

colon cancer) 2H11 and P3

VENICE (prostate cancer) final

results in 2012 after JUL 2011

interim analysis, co-developed

with SNY 7/7/11

Regeneron

Pharma REGN $68.37 $6,265 9/30/13

Phase 2b / 3

(MOBILITY)

Clinical Trial

Sarilumab

(REGN88)

(SAR15319

1) (IL-6R

targeted

antibody)

In JUL 2011, reported positive

P2B trial results along w/

partner SNY for the treatment

of rheumatoid arthritis (RA) in

combo w/ methotrexate, will

select dose(s) to be evaluated

in P3 portion of the trial,




Regeneron

Pharma REGN $68.37 $6,265 9/30/11

FDA New Filing

(sBLA)

ARCALYST

(rilonacept)

Subcutaneo

us Injection

(fusion

protein that

blocks

cytokine

interleukin-1

or IL-1)


second Phase 3 trial for

prevention of gout flares in pts

receiving uric acid lowering

therapy met all primary and

secondary endpts, expects to

file a sBLA mid-2011 (2-

3Q11) to expand use of drug

which is currently approved for

inherited, auto-inflammatory

conditions known as CAPS 6/28/11

RegeneRx

Biopharma RGRX $0.26 $21 10/31/11

Phase 2 Clinical

Trial

RGN-259

(Tβ4

ophthalmic

solution)

On 8/24/11 completed enrlmt

of 72 pts in ongoing P2 trial for

dry eye syndrome, expects to

complete treatment of all pts

by 9/23/11 and report prelim

data in OCT 2011,


NCT01387347 8/24/11

Repligen RGEN $3.29 $101 3/31/12

Pilot Clinical

Trial

SecreFlo

(RG1068)

for

Pancreatic

Imaging

On 7/21/11, announced first pt

enrolled in pilot study to

evaluate ability of RG1068 to

improve pancreatic cancer

detection in combo w/ MRI

and CT scans, expects to

enroll 40 pts, ClinicalTrias.gov



Repligen RGEN $3.29 $101 12/31/11

FDA New Filing

(NDA)

SecreFlo

(RG1068)

for

Pancreatic

Imaging

In MAR 2011 reported P3 trial

met co-primary endpoints to

improve magnetic resonance

imaging (MRI) of the pancreas

and in JUN 2011 announced

conclusion of pre-NDA mtg w/

FDA, plans to submit NDA

along w/ regulatory approval

filings in Europe and Canada

later this year (2H11 estimate) 7/27/11

Repros

Therapeutic

s RPRX $4.76 $59 9/12/11

Phase 2 Clinical

Trial

Androxal

(enclom-

iphene)

In MAY 2011, provided update

w/ 66 men w/ Type 2 diabetes

randomized in trial, expects to

report interim results and be

fully enrolled (120-150 pts) in

SEP 2011 and report top-line

results for full study around

year-end 2011, will present

update on 9/12/11 9/1/11

Repros

Therapeutic

s RPRX $4.76 $59 9/12/11

Low-Dose

(Safety) Proellex

Clinical Trial

Proellex

(oral

selective

progest-

erone

receptor

modulator)


subjects in 9mg cohort

following safety rvw after 8

wks of dosing at 6mg w/ no

signals of liver toxicity

reported, enrolling 12 subjects

per dosing group w/ 12mg as

last in this study that is

expected to be completed

during 4Q11, will present

update on 9/12/11 at R&R

conf 9/1/11

Rexahn

Pharma RNN $0.95 $90 12/31/11

Phase 2b

Clinical Trial

SERDAXIN

(extended

release

clavulanic

acid

formulation)

In JUN 2010 completed pt

enrlmt for treatment of major

depressive disorder (MDD) w/

results expected during 4Q11 8/17/11

Rexahn

Pharma RNN $0.95 $90 12/31/11

Phase 2a Open-

Label Clinical

Trial

ARCHEXIN

(RX0201)

(protein

kinase Akt

inhibitor anti-

cancer

agent)


NCT01028495 w/ pt enrlmt

expected to continue thru

2011, being evaluated in

combo with GEMZAR

(gemcitabine) in patients w/

metastatic pancreatic cancer 8/17/11

Rigel

Pharma RIGL $7.07 $504 12/31/12

Pivotal Phase 3

(OSKIRA-1,2,3)

Clinical Trials,

Phase 2b

Clinical Trial

Fostamatinib

disodium

(oral agent

for

rheumatoid

arthritis)

In SEP 2010, partner AZN

began three pivotal P3 trials

for rheumatoid arthritis (RA)

pts taking methotrexate w/o

responding, expects to report

initial data during 2H12 to

support planned regulatory

aprvl filings in US and EU in

2013, during 1Q11 began

P2B mono-therapy trial in RA

pts w/ results expected 4Q12 8/2/11

Rockwell

Medical RMTI $8.25 $149 6/30/12

Phase 2

(PRIME) Clinical

Trial

Soluble

Ferric

Pyrophosph

ate (SFP)

(dialysate

iron)

(continuous

iron

replacement

therapy)

In FEB 2011, began enrolling

pts to evaluate potential

reduction in the need for

erythropoietin stimulating

agents (ESA) in dialysis

patients receiving SFP,

expects to complete enrlmt by

yr-end 2011 and report results

mid-2012, ClinicalTrials.gov

ID NCT01286012 7/27/11

Rockwell

Medical RMTI $8.25 $149 12/31/12

Pivotal Phase 3

(CRUISE-I & II)

Clinical Trials

Soluble

Ferric

Pyrophosph

ate (SFP)

(dialysate

iron)

(continuous

iron

replacement

therapy)

In MAR 2011, began enrolling

pts (300 each) in pivotal trialsl

to evaluate mean change in

hemoglobin levels between

SFP and placebo, expects to

complete enrlmt by yr-end

2011 w/ results expected late

2012 (4Q12 estimate) to

support planned NDA filing

late 2012-early 2013,


NCT01320202 and

NCT01322347 7/27/11

RXi Pharma RXII $1.00 $42 6/30/12

Pending Pivotal

Phase 3

(PRESENT)

Clinical Trial

under SPA

NeuVax

(peptide-

based

immuno-

therapy for

HER2+

breast

cancer)

In JUL 2011, acquired patent

rts for use in combo w/

Herceptin and in low-to-

intmdte HER2+ breast cancer

pts not eligible to receive

Herceptin, expects to begin

pivotal P3 trial (Prevention of

Recurrence in Early-Stage,

Node-Positive Breast Cancer

with Low to Intermediate

HER2 Expression) under SPA

during 1H12 7/13/11

Sangamo

BioSciences SGMO $5.06 $265 6/30/12

Phase I and

Phase I / II

Clinical Trials

SB-728-T

(Patient-

Derived T-

cells

Genetically

Modified at

CCR5 Gene

by Zinc

Finger

Nucleases

or ZFNs)



expected during 2H12 for

P1/2 trial in HIV infected

subjects not receiving HAART

treatment, reported positive

prelim data from Phase I trial

on 2/28/11 in HIV infected pts

receiving treatment w/

HAART, ClinicalTrials.gov ID



Sangamo

BioSciences SGMO $5.06 $265 12/31/11

Phase 2b (SB-

509-901)

Clinical Trial

SB-509 Zinc

Finger

Therapeutic

for Gene

Regulation

In JAN 2011, completed pt

enrlmt in P2B trial for

moderately severe diabetic

neuropathy and expects to

report results during 4Q11,


NCT01079325 7/28/11

Sanofi-

Aventis SNY $34.63 $92,959 12/31/11

Pivotal Phase 3

(CARE-MS II)

Clinical Trial

Alemtuzuma

b

(humanized

monoclonal

antibody

targeting

CD52 on T

& B cells)

On 7/11/11, reported first of

two pivotal P3 trials (CARE-

MS I) met one of two primary

endpts w/ second P3 trial


and regulatory approval filings

in US and EU expected during

early 2012for pts w/ multiple

sclerosis (MS) 7/11/11

Santarus SNTS $3.05 $184 12/31/11

FDA New Filing

(NDA)

Budesonide

MMX (local-

acting, oral

tablet

formulation)

Held pre-NDA mtg w/ FDA in

late MAY 2011 and plans to

submit NDA by yr-end 2011,

seeking approval for the

induction of remission of mild

or moderate active ulcerative

colitis 8/4/11

Santarus SNTS $3.05 $184 9/30/12

Pivotal Phase

3b Clinical Trial

to Support FDA

New Filing

(BLA) under

SPA

RHUCIN

(human C1

esterase

inhibitor)

In FEB 2011 received refusal

to file letter from FDA for BLA


of acute attacks of hereditary

angioedema, approved in

Europe OCT 2010, began

Phase 3b study FEB 2011

and expects to complete by

3Q12 to support potential new

BLA filing w/ SPA agmt

announced in AUG 2011 8/4/11

Santarus SNTS $3.05 $184 12/31/11

Phase 3 Clinical

Trials

Rifamycin

SV MMX

Being evaluated for treatment

of travelers’ diarrhea in two P3

trials, ClinicalTrials.gov ID


expected 2H11,




Sanuwave

Health SNWV $3.10 $65 6/30/12

FDA Decision

(PMA) dermaPACE

On 7/6/11, announced PMA

submission to FDA, seeking

marketing clearance for non-

invasive medical device for the

treatment of diabetic foot

ulcers based on 24-week

pivotal trial results in 206 pts,

estimated FDA decision mid-


deadlines for PMA filings 7/6/11

SciClone

Pharma SCLN $4.23 $247 6/30/12

Phase 2b

Clinical Trial

SCV-07

(gamma-D-

glutamyl-L-

tryptophan)

In JAN 2011, enrolled first pt

in P2b trial for prevention of

oral mucositis (OM) (a

common side effect of chemo

and radiation treatments for

head and neck cancer)




Sequenom SQNM $5.47 $543 12/31/11

Validation

Clinical Trial for

Trisomy 21

(T21) (Down

Syndrome)

Laboratory

DevelopedTest

(LDT)

MaterniT21

(non-

invasive

trisomy 21

lab-

developed

test)

Expected submission (3Q11)

and publication (4Q11) of LDT

clinical validation study results

to support expected launch

during 4Q11-1Q12 for non-

invasive blood test to detect

chromosome 21 (associated

w/ fetal Down syndrome), also

developing in-vitro diagnostic

(IVD) device w/ expected FDA

new filing (PMA) during 4Q12-

1Q13 8/5/11

Sinovac

Biotech SVA $2.53 $138 3/31/12

Phase 2 Clinical

Trial

EV71

(Enterovirus

71) Vaccine

(Hand, Foot

and Mouth

Disease or

HFMD)


of P2 trial for HFMD vaccine

candidate, expects to

complete study in six months

(1Q12 estimate for results) to

support start of P3 trial in

spring 2012 during HFMD

outbreak season 6/29/11

Soligenix SNGX $0.26 $57 3/31/12

Phase 1b

Clinical Trial

RiVax (ricin

toxin sub-

unit vaccine)

In NOV 2010, completed pt

enrlmt w/ prelim results

indicating no safety concerns,

will assess immune response

duration one year after

vaccination, ClinicalTrials.gov



received FDA Orphan Drug

Status in JAN 2011 6/20/11

Soligenix SNGX $0.26 $57 12/31/11

Pivotal

Confirmatory

Phase 3

(SUPPORTS)

Clinical Trial

under SPA

orBec (oral

beclomethas

one

dipropionate

or BDP) (a

steroid

formulated

for local

effect in the

GI tract)


expanded ptnshp w/ Sigma-

Tau into Europe w/ $5M

upfront and $11M potential

milestone pmts plus 40%

royalty / manufacturing supply

agmt, expects to complete

ongoing confirmatory pivotal

P3 trial during 2H11 for

treatment of acute

gastrointestinal (GI) graft vs.

host disease 7/28/11

Spectranetic

s SPNC $5.52 $185 3/31/13

Pivotal Phase II /

III (EXCITE-ISR)

Clinical Trial

Turbo Elite

Laser and

Turbo

Tandem

Laser Guide

Catheters

In JUN 2011, announced first

pt enrolled in trial to compare

excimer laser atherectomy in

conjunction with balloon

angioplasty vs. balloon

angioplasty alone in the

treatment of above-knee

peripheral artery in-stent

restenosis, ClinicalTrials.gov


expected 1Q13 to support

potential FDA 510(k)

submission 7/19/11

Spectrum

Pharma SPPI $8.13 $432 11/20/11

FDA Decision

(Prior Approval

Supplement)

ZEVALIN

(ibritumoma

b tiuxetan)

Injection


accepted prior approval

supplement w/ expected

decision by 11/20/11 to

remove “bio-scan”

requirement as part of

treatment regimen for non-

Hodgkin’s lymphoma (NHL) 3/22/11

Spectrum

Pharma SPPI $8.13 $432 12/31/11

Phase 2 Clinical

Trials

Belinostat

(PXD101) (a

novel

histone

deacetylase

or HDAC

inhibitor anti-

cancer

agent)

On 3/28/11, passed interim

safety and futility analysis (45

pts dosed) for pivotal P2 trial

for Peripheral T-Cell

Lymphoma (PTCL), expects to

complete enrlmt of 100 pts in

2011 and file NDA in 2012, in

DEC 2010 completed enrlmt

in Phase 2 trial for carcinoma

of unknown primary origin w/


Spectrum

Pharma SPPI $8.13 $432 6/30/12

Pivotal Phase 3

Clinical Trials

EOquin (apa-

ziquone)

For the treatment of non-

muscle invasive bladder

cancer, licensed to AGN,

Phase 3 top-line data

expected in 2012 (1H12

estimate) to support potential

NDA filing in 2012,


NCT00598806 &

NCT00461591 4/28/11

StemCells STEM $2.29 $32 12/31/11

Phase I / II

Clinical Trial

HuCNS-SC

(purified

human

neural stem

cells)

In MAR 2011, began trial in

Switzerland for pts w/ chronic

spinal cord injury, expects to

enroll and dose first group of

pts in 2011, ClinicalTrials.gov

ID NCT01321333 7/29/11

StemCells STEM $2.29 $32 6/30/12

Phase I Clinical

Trial

HuCNS-SC

(purified

human

neural stem

cells)

In FEB 2011, completed pt

enrlmt and dosed final (fourth)

pt for treatment of nerve

myelination disorder (PMD) in

children, ClinicalTrials.gov ID



Sucampo

Pharma SCMP $3.13 $131 6/30/12

Europe and

Japan New

Drug Approval

Decisions

AMITIZA

(lubiproston

e)

In SEP 2010 submitted NDA

for approval in Japan w/

estimated decision during

2H11, in AUG 2011 submitted

MAA to MHRA seeking UK

approval for treatment of

chronic idiopathic constipation

(CIC) w/ estimated decision

w/in one year (3Q12 estimate),

already FDA approved 8/5/11

Sucampo

Pharma SCMP $3.13 $131 12/31/11

Pivotal Phase 3

Clinical Trial

AMITIZA

(lubiproston

e)

Has completed enrlmt in

ongoing P3 trial for opioid

induced bowel dysfunction

(OBD) w/ top-line results

expected by yr-end 2011 to

support potential NDA filing

during 1H12 8/5/11

Sunesis

Pharma SNSS $1.36 $64 6/30/12

Pivotal Phase 3

(VALOR)

Clinical Trial

Vosaroxin

(formerly

voreloxin)

(anti-cancer

quinolone

derivative)

In DEC 2010, first pt dosed in

pivotal P3 trial in combo w/

cytarabine for pts w/ first

relapsed or refractory acute

myeloid leukemia (AML),

expects interim analysis by

data safety monitrg board in

mid-2012, ClinicalTrials.gov

ID NCT01191801 w/ final


Synta

Pharma SNTA $3.88 $192 6/30/12

Phase I and II

Clinical Trials,

Pending Phase

2b Clinical Trial

Elesclomol

(program-

med cell

death /

apoptosis

anti-cancer

agent)

In FEB 2011, first pt treated in

single-agent P1 trial for acute

myeloid leukemia (AML) w/


NCT01280786,


NCT00888615 for P2 ovarian

cancer trial in combo w/

paclitaxel w/ results expected

during 1H12 for both trials,

expects to begin P2B trial for

lung cancer in 2011 7/20/11

Synta

Pharma SNTA $3.88 $192 3/31/12

Phase 2b / 3

(GALAXY)

Clinical Trial

Ganetespib

(formerly

STA-9090)

(Hsp90

inhibitor anti-

cancer

agent)

On 7/20/11, treated first pt in

P2B/3 trial in combo w/

docetaxel for non-small cell

lung cancer (NSCLC) and


results in early 2012 (1Q12

estimate) 7/20/11

Talon

Therapeutic

s (formerly

Hana

Biosciences) TLON $0.97 $21 9/19/11

FDA Reply to

Accept NDA

Filing and Issue

PDUFA

Decision Date,

Pending

Confirmatory

Phase 3

(HALLMARQ)

Clinical Trial

MARQIBO

(Optisome

encapsulate

d

formulation

of approved

cancer drug

vincristine)

On 8/29/11, reached SPA

agmt w/ FDA for planned P3

trial by yr-end 2011 in pts w/

acute lymphoblastic leukemia

(ALL), on 7/18/11 announced

NDA filing seeking

accelerated FDA approval for

treatment of adults w/

Philadelphia Chromosome

Negative ALL w/ expected

FDA reply by 9/19/11 to

accept NDA filing and issue

decision date 8/29/11

Targacept TRGT $15.60 $521 12/31/11

Pivotal Phase 3

Clinical Trials

TC-5214 (a

nicotinic

channel

blocker)

Partnered with AZN for add-on

depression treatment for non-

responders to first-line

therapy, ClinicalTrials.gov IDs

NCT01180400,

NCT01153347,

NCT01157078, and


expected during 4Q11-1Q12,

began P2b trial as switch

mono-therapy for non-

responders, planned NDA

filing during 2H12 6/21/11

Tekmira

Pharma TKMR $2.01 $23 12/31/11

Phase I Clinical

Trial

TKM-PLK1

(cell cycle

protein anti-

cancer

agent)

In DEC 2010, began dosing

patients for lead anti-cancer

RNA interference (RNAi)

candidate, expects to enroll up

to 52 pts w/ advanced solid

tumors, ClinicalTrials.gov ID




results by yr-end 2011 8/9/11

Tekmira

Pharma TKMR $2.01 $23 11/20/11

Phase I Clinical

Trial

ALN-TTR01

(systemic

RNA

interference

or RNAi

therapeutic

for

amyloidosis)

In AUG 2011 partner ALNY

announced plans to present

data at scientific mtg Nov. 20-

22 in Japan from ongoing P1

trial in up to 28 pts w/

amyloidosis (condition that

damages peripheral nerves

and heart), TKMR developed

RNAi drug delivery tech for

this compound 8/2/11

Tengion TNGN $0.80 $19 12/31/11

Phase I Clinical

Trial

Neo-Urinary

Conduit

(patient-

derived cells

w/ bio-

absorbable

scaffold)

As of AUG 2011, has enrolled

and implanted 3 pts in

ongoing P1 trial designed to

regenerate bladder tissue in

pts w/ bladder cancer,

proposed surgical

modifications to FDA w/

interim update on trial status





Theravance THRX $17.43 $1,477 9/30/12

Pivotal Phase 3

Clinical Trials

GSK573719

/ Vilanterol

('719 / VI)

(Dual

Bronchodilat

or Inhaler)

Expects to enroll over 5,000

pts w/ the lung condition

chronic obstructive pulmonary

disease (COPD) in seven P3

trials w/ results expected mid-

2012 (2-3Q12 estimate),

partnered w/ GSK 7/29/11

Theravance THRX $17.43 $1,477 12/31/11

Pivotal Phase 3

Clinical Trials

RELOVAIR

(fluticasone

and

vilanterol)

(once-daily

inhaled

steroid +

long-acting

beta

agonist)

Ongoing pivotal P3 trials for

chronic obstructive pulmonary

disease (COPD) and asthma

fully enrolled (11,000 pts) w/

results expected by late 2011-

early 2012, partnered w/ GSK 7/29/11

Thermo-

Genesis KOOL $1.58 $26 12/31/11

SFDA (China)

Decisions

BioArchive

System &

AXP

Platform

Partner Fenwal continues to

advance the registration

process for these cord blood

processing & storage products

in China w/ expected

approvals in China (SFDA)

during 2H11 5/6/11

Threshold

Pharma THLD $1.53 $75 9/30/11

Pending Pivotal

Phase 3 Clinical

Trial under SPA

TH-302 (a

hypoxia-

activated pro-

drug or HAP

anti-cancer

agent)

In FEB 2011 announced SPA

agmt w/ FDA for design of


advanced / metastatic un-

removable soft tissue sarcoma

in combo w/ doxorubicin that

is expected to begin during

3Q11 7/25/11

Threshold

Pharma THLD $1.53 $75 12/31/11

Phase I and II

Clinical Trials

TH-302 (a

hypoxia-

activated pro-

drug or HAP

anti-cancer

agent)


of trial in combo w/ AVASTIN

for pts w/ recurrent brain

cancer (glioma), in JUN 2011

began P1/2 trial in combo w/

SUTENT for variety of cancers

w/ ClinicalTrials.gov ID


expected mid-2013), P1

leukemia (NCT01149915) and

P2 pancreatic cancer

(NCT01144455) trial results


Titan

Pharma TTNP $1.50 $89 12/31/11

Phase 3 Open

Label Safety

Clinical Trial,

FDA New Filing

505(b)(2) NDA

Probuphine

(subcutaneo

us,

implantable

delivery of

bupren-

orphine over

six months)

Pre-NDA mtg w/ FDA planned

for 10/25/11 to discuss

505(b)(2) NDA filing based on

confirmatory P3 trial that met

primary endpt for treatment of

pts w/ opiate addiction,

expects to report open-label

safety study results by yr-end

2011 8/31/11

Transcept

Pharma TSPT $2.75 $37 12/31/12

Phase 2 Clinical

Trial

TO-2061

(low-dose

ondansetron

formulation)

(ondansetro

n (0.5mg

and 0.75mg

doses)

On 3/14/11, began trial for

treatment of obsessive

compulsive disorder (OCD)

pts w/ inadequate response to

first-line treatments, expects to

enroll approx. 150 pts,



expected during 3-4Q12 7/13/11

Tranzyme

Pharma TZYM $3.13 $74 6/30/12

Pivotal Phase 3

(ULISES 007

and 008)

Clinical Trials

Ulimorelin

(intravenous

GI pro-

motility

agent)

In FEB & MAR 2011, began

two pivotal P3 trials for post-

operative ileus (a temporary

halt of normal bowel motility

after surgery) in hospital and

acute care settings, expects to

complete both studies during

1H12, ClinicalTrials.gov IDs

NCT01285570 &

NCT01296620 8/12/11

Trius

Therapeutic

s TSRX $6.11 $174 3/31/12

Pivotal Phase 3

Clinical Trials

under SPA

Torezolid

Phosphate

(TR-701)

On 8/5/11, announced SPA

agmt for second planned P3

trial (expects to begin 4Q11)

for IV and oral dosage forms

for treatment of acute bacterial

skin and skin structure

infections (ABSSSI),


NCT01170221 for ongoing P3

trial expected to complete

enrlmt yr-end 2011 w/ results

expected early 2012 (1Q12

estimate) 8/5/11

Unigene UGNE $0.90 $84 12/31/11

Phase 2 Clinical

Trial

Oral PTH

(para-thyroid

hormone)

In APR 2011, completed pt

enrlmt in ongoing P2 trial for

oral parathyroid hormone

(PTH) analog for the treatment

of osteoporosis in

postmenopausal women,

expects to report top-line

results by year-end 2011,


NCT01321723, in AUG 2011

announced up to $2.2M

milestone pmts from partner

GSK as part of supply agmt 8/8/11

Unigene UGNE $0.90 $84 12/31/11

FDA New Filing

(NDA) (4Q11),

Phase 2 Clinical

Trial (1Q12)

Oral

Calcitonin

Licensed to Tarsa

Therapeutics (private) for

novel oral formulation of

calcitonin (currently marketed

as nasal spray) for post-

menopausal osteoporosis,

reported positive results in

pivotal trial w/ SPA agmt in

MAR 2011 and plans to

submit NDA by late 2011, in

JUL 2011 completed enrlmt in

P2 trial w/ interim data

expected in early 2012 (1Q12

estimate) 7/12/11

United

Therapeutic

s UTHR $42.80 $2,496 6/30/12

FDA New Filing

(NDA)

Oral

Treprostinil

(injected

version

approved as

Remodulin)

On 8/24/11, reported second

of two pivotal P3 trials did not

meet endpt for treatment of

pulmonary arterial

hypertension (PAH) but plans

to file NDA during 1H12 based

on results in previous P3 trial

in treatment-naïve pts plus

drug is already FDA approved

for PAH by IV, inhaled and

subcutaneous routes 8/24/11

Vanda

Pharma VNDA $5.61 $158 12/31/11

Pending Phase

2b / 3 Clinical

Trial

Tasimelteon

(VEC-162)

(melatonin

agonist)

Expects to begin trial in pts w/

Major Depressive Disorder

(MDD) during 2H11 compared

to placebo w/ an 8-week

treatment and optional open-

label extension period 3/31/11

Vanda

Pharma VNDA $5.61 $158 6/30/12

Pivotal Phase 3

Clinical Trial

Tasimelteon

(VEC-162)

(melatonin

agonist)

In AUG 2010, began study for

treatment of circadian rhythm

sleep disorders, received

Orphan Drug status Jan. 2010

for Non-24-Hour Sleep/Wake

Disorder (N24SWD) in blind

individuals, ClinicalTrials.gov

ID NCT01163032 w/ top-line


Ventrus

Biosciences VTUS $9.40 $117 6/30/12

Pivotal Phase 3

Clinical Trial

Diltiazem

Topical

Cream (VEN

307)

Ongoing P3 trial in Europe by

licensing partner SLA Pharma

w/ results expected during

2Q12, expects to complete

formulation program by 2Q12

and conduct either one addt’l

P3 trial in US w/ existing

formulation being studied in

Europe or two P3 trials in US

w/ the new formulation to

support planned NDA filing in

2013 8/16/11

Ventrus

Biosciences VTUS $9.40 $117 3/31/12

Pending Pivotal

Phase 3 Clinical

Trial w/ SPA

Request

Iferanserin

Topical

Ointment

(VEN 309)

In AUG 2011, opened enrlmt

for first of two planned pivotal

P3 trials for treatment of

symptomatic hemorrhoids,

expects to report top-line data

during 1Q12,


NCT01355874 8/16/11

Vermillion VRML $2.40 $36 9/30/11

Intended-Use

Population

Clinical Trial

(Diagnostic Test

Evaluation)

VASCLIR

Peripheral

Artery

Disease

(PAD) Blood

Test

On 8/1/11, announced noticed

of allowance for patent

covering biomarkers for PAD,

expects to report top-line data

during 3Q11 to provide a

finalized biomarker algorithm

for blood diagnostic test to

stratify risk of developing PAD 8/1/11

Vertex

Pharma VRTX $45.52 $9,472 12/31/12

MAA / EMA

(Europe)

Decision, Pivotal

Phase 3b

(OPTIMIZE)

Clinical Trial to

Support sNDA

New Filing

INCIVEK

(Telaprevir)

(VX-950 or

TVR) (HCV

protease

inhibitor)

In MAY 2011, received FDA

approval for treatment of

genotype 1 chronic hepatitis C

virus (HCV) infection, has

completed enrlmt in ongoing

P3B trial to evaluate twice-

daily dosing w/ results

expected as early as 2H12 to


by year-end 2012, positive

opinion adopted by EMA w/

expected EU approval 3Q11 7/29/11

Vertex

Pharma VRTX $45.52 $9,472 6/30/12

Phase 2

(ZENITH)

Clinical Trial,

Pending Pivotal

Phase 3 Clinical

Trial

INCIVEK

(Telaprevir)

(VX-950 or

TVR) (HCV

protease

inhibitor)


interim results in combo w/ VX-

222 (experimental polymerase

inhibitor) for treatment of HCV

and expects to report data

from all-oral treatment arms

during 1H12, expects to begin

P3 trial during 2H11 in combo

w/ pegylated-interferon and

ribavirin in people co-infected

with HCV and HIV to support

sNDA filing 7/26/11

Vertex

Pharma VRTX $45.52 $9,472 10/31/11

FDA and MAA /

EMA New

Filings

VX-770

(Cystic

Fibrosis

Trans-

membrane

Conduct-

ance

Regulator)

In MAR 2011, reported

positive results for oral

treatment for cystic fibrosis

(CF) from 24-week analysis of

P3 ENVISION trial and in JUN

2011 positive P3 final STRIVE

trial results to support planned

regulatory approval filings in

US and Europe in OCT 2011 7/29/11

Vical VICL $3.41 $245 6/30/12

Pending Phase

3 Clinical Trial

TransVax

CMV

Therapeutic

Vaccine


exclusive license agmt w/

Astellas Pharma, includes

$130M upfront plus milestone

pmts (w/ $35M due near-term)

and double-digit royalties,

expects to begin pivotal P3

trial for immune compromised

stem cell transplant pts and

P2 trial for solid organ

transplant pts for

Cytomegalovirus (CMV)

infection during 1H12 7/15/11

Vical VICL $3.41 $245 12/31/11

Pending Phase

3 Clinical Trial

Collategene

(gene-based

blood vessel

growth

factor)

Partner AnGes expected to

begin P3 trial by yr-end 2011

for the treatment of advanced

peripheral artery disease

(PAD) 6/22/11

Vical VICL $3.41 $245 6/30/12

Pivotal Phase 3

Clinical Trial

(AIMM) under

SPA

Allovectin-7

(gene-based

therapeutic

cancer

vaccine)

Expects to complete treatment

and follow-up in ongoing


metastatic melanoma in FEB

2012 w/ top-line results



NCT00395070 8/3/11

Vivus VVUS $8.17 $670 12/31/11

Observational

(FORTRESS)

Study to

Support FDA

Resubmission

(NDA), MAA /

EMA Decision

QNEXA

(phentermin

e plus

topiramate)

(controlled-

release,

once-daily

capsules)

Expects to report retrospective

fetal outcomes study following

topiramate exposure to assess

birth defect risk during 4Q11

to support planned NDA

resubmission and response to

MAA / EMA (EU approval) 120-

day questions during 4Q11 8/2/11

Vivus VVUS $8.17 $670 4/29/12

FDA Decision

(NDA)

Avanafil (an

oral

phospho-

diesterase

type 5 or

PDE5

inhibitor)


accepted NDA for standard 10-

month review w/ PDUFA

decision goal date of 4/29/12,


of erectile dysfunction (ED) 9/1/11

XenoPort XNPT $6.68 $237 12/31/11

PMDA Decision

(Japan) (2H11),

FDA Decision

(sNDA) (6/9/12)

HORIZANT

(gabapentin

enacarbil)

Extended-

Release

Tablets

In APR 2011 received FDA

approval along w/ US partner

GSK for Restless Legs

Syndrome in adults, expected

PMDA decision during 2H11

w/ partner Astellas Pharma,

sNDA filed on 8/9/11 by GSK


of post-herpetic neuralgia w/

expected decision by 6/9/12

under standard 10-month

review period 8/10/11

XenoPort XNPT $6.68 $237 12/31/11

Phase 2b

Clinical Trial

XP21279

(novel

formulation

converted

into L-Dopa /

dopamine)



P2B trial in combo w/

levodopa in pts w/ Parkinson’s

disease, expects to report top-

line results by yr-end 2011 8/4/11

XenoPort XNPT $6.68 $237 9/30/12

Pivotal Phase 3

Clinical Trial

under SPA

Arbaclofen

Placarbil

(AP)

(XP19986)




pivotal P3 trial for treatment of

spasticity in multiple sclerosis

(MS) pts to support potential

NDA 505(b)(2) filing as a new

formulation of approved

muscle relaxant drug baclofen 8/4/11

22nd

Century XXII $1.20 $33 11/30/11

Phase 2b

Clinical Trial

X-22 (very

low nicotine

/ VLN

cigarettes

smoking

cessation

aid)

On 8/8/11, first pts enrolled in

P2B trial for smoking

cessation aid product

candidate, expects FDA

decision on Fast Track status

by SEP 2011, expects to

report results from trial in late

NOV 2011 (four wks of

continuous smoking cessation

after program) 8/9/11

YM

BioSciences YMI $1.87 $206 12/31/11

Phase 2 and 3

Clinical Trials

Nimotuzuma

b

(humanized

epidermal

growth

factor,

EGFR-

targeting

monoclonal

antibody)

Japan licensee Daiichi Sankyo

has submitted to present lung

cancer data during 2H11, P2

trial for treatment of brain

cancer in children (glioma)

has completed enrlmt w/

results expected 2H11, EU

partner Oncoscience enrolling

pts for P2b/3 pancreatic

cancer trial (ClinicalTrials.gov

ID NCT00561990) w/ results


YM


Phase I / II

Clinical Trial,

Pending Phase

2 Clinical Trial

CYT387 (a

JAK1 / JAK2

Kinase

Inhibitor)

On 6/3/11 reported interim

data w/ 46% spleen response

and 50% anemia response,

completed enrlmt in MAY

2011 w/ full data expected

during 4Q11 at ASH mtg Dec.

10-13 for treatment of

myelofibrosis, expects to

begin 60-pt P2 twice-daily

dosing (BID) trial during 3Q11

to support start of pivotal P3

trial in 1H12 8/1/11

YM


Phase Ib / II

Clinical Trial

CYT997 (a

small

molecule

vascular

disrupting

agent or

VDA)

Expects to report preliminary

data from ongoing trial in brain

cancer (glioma) pts in combo

w/ carboplatin during 2H11,


NCT00650949, pt enrlmt has

closed for this trial 5/12/11

Zalicus

(formerly

Combinato-

Rx) ZLCS $1.43 $142 12/31/12

Phase 2b

(SYNERGY)

Clinical Trial

SYNAVIVE

(Z-102)

(Dipyridamol

e and

prednisolon

e once-daily,

modified-

release

combo

capsule)


of P2B trial in pts w/

rheumatoid arthritis w/ top-line

results expected during 2H12,

novel formulation of two

marketed drugs w/ patent

protection thru 2028 being

compared to placebo (primary

objective) and the two drugs

given independently

(secondary objective),


NCT01369745 6/29/11

Ziopharm

Oncology ZIOP $5.06 $346 12/31/11

Phase I Clinical

Trial

ZINAPAR

(Darina-

parsin or

ZIO-101)

(organic

arsenic anti-

cancer

agent)

Expects to begin two-stage

potential pivotal trial in late

2011 (4Q11) for treatment of

relapsed peripheral T-cell

lymphoma (PTCL), oral

formulation being studied in

P1 trial for solid tumors,




Ziopharm

Oncology ZIOP $5.06 $346 12/31/11

Phase I / II

Clinical Trial

Indibulin

(ZybulinTM

or ZIO-301)

(oral tubulin

binding anti-

cancer

agent)

Being evaluated in patients w/

metastatic breast cancer w/




Ziopharm

Oncology ZIOP $5.06 $346 12/31/12

Pivotal Phase 3

(PICASSO III)

Clinical Trial

ZYMAFOS

(Palifos-

famide) (ZIO-

201)


initiation of pivotal study for

front-line metastatic soft tissue

sarcoma w/ ClinicalTrials.gov

ID NCT01168791 and results

expected during 2H12, began

Phase I study for small cell

lung cancer in DEC 2010 6/10/11

Zogenix ZGNX $3.28 $112 3/31/12

FDA New Filing

(NDA)

ZOHYDRO

(hydrocodon

e extended-

release

capsules)

(previously

ZX002)

On 8/17/11 reported pivotal

P3 trial for treatment of

moderate to severe pain met

primary and secondary enpts

to support planned NDA filing


expects to finish long-term

safety study during 3Q11 8/17/11

Documents

Clinical and Regulatory Catalyst Calendar