Capturing the value of Fidaxomicin - shareholderfiles.shareholder.com/downloads/OPTR/0x0x435614... · Capturing the value of Fidaxomicin January 12th 2011 Ticker: ... whether caused

0

Capturing the value of

FidaxomicinJanuary 12th 2011

Ticker: OPTR

www.optimerpharma.com

Confidential; Not for Reproduction1

This presentation contains forward-looking statements about Optimer Pharmaceuticals, Inc., including statements about the indications Optimer’s product candidates are designed to treat, regulatory filings related to Optimer’s product candidates. Optimer’s product development plans, potential commercialization and marketing strategies for Optimer’s product candidates, if approved, estimates of market adoption of Optimer’s product candidates, if approved, and potential efficacy and advantages of Optimer’s product candidates compared to existing therapies. These statements involve known and unknown risks that relate to the Company’s future events or future financial performance and the actual results could differ materially from those discussed in this presentation. Accordingly, the Company cautions investors not to place undue reliance on the forward-looking statements contained in, or made in connection with, this presentation.

Several factors may affect the ability of the Company to market any of its product candidates, the Company’s commercialization plans, the potential advantages of the Company’s product candidates and the Company’s ability to successfully execute it business and commercialization strategy. Among other things, the Company’s receipt of regulatory approval to market any of its product candidates is dependent on the Company’s ability to successfully complete clinical trials and submit applications for regulatory approval and on decisions of regulatory authorities which are outside of the Company’s control. In addition, success of any commercialization efforts are subject to risks relating to physicians’ willingness to prescribe the Company’s products, the incidence of the disease or condition the Company’s products are intended to treat, and the efficacy and cost of alternative treatments. Delays in clinical programs or submission of regulatory filings, whether caused by competitive developments, adverse events, patient enrolment rates, regulatory issues or other factors, could adversely affect the Company’s financial position and prospects. If the Company’s product candidates do not meet safety or efficacy endpoints in clinical evaluations, they will not receive regulatory approval and the Company will not be able to market them. If the Company is unable to raise additional capital when required or on acceptable terms, it may have to significantly delay, scale back or discontinue one or more of its research development programs or delay or scale back its commercialization efforts regarding any products for which it receives regulatory approval. The Company is at an early stage of development and may not ever have any products that generate significant revenue.

Additional risks and uncertainties relating to the Company and its business can be found in the "Risk Factors" section of Optimer's most recent Form 10-K filed with the SEC and any updates thereto contained in subsequent SEC filings. The forward-looking statements contained in this presentation represent the Company’s estimates and assumptions only as of the date of this presentation and the Company undertakes no duty or obligation to update or revise publicly any forward-looking statements contained in this presentation as a result of new information, future events or changes in the Company’s expectations.

THIS PRESENTATION CONTAINS STATEMENTS THAT HAVE NOT YET BEEN REVIEWED BY THE FDA AND IS SUBJECT TO CHANGE.

Forward looking statements


Strengthening our commercial business

Introducing Optimer

Headquarters San Diego,

California

Incorporated November 1998

Subsidiaries OBI (Optimer

Biotechnology,

Inc., Taiwan)

Employees 77

Exchange NasdaqGM

Ticker OPTR

Optimer at a glance

Greg Papaz, SVP Commercial Operations

Experience

▪ Rheumatology Sales Director, Roche

▪ Hospital Sales VP, Sanofi-Aventis

John Womelsdorf, VP Bus. Dev.

Experience

▪ VP Bus. Dev., Cyclacel

▪ Exec. Dir. Lic. and New Bus. Dev.; J&J

Kurt Hartman, General Counsel & Access

Experience

▪ Exec Dir Value & Access, Eisai

▪ US patient reimbursement lead, Roche

Hemal Shah, SVP Health Economics

Experience

▪ Exec Dir HEOR, Boehringer Ingelheim

Linda Amper, SVP Human Resources

Experience

▪ SVP of HR, OSI Pharmaceuticals

▪ VP of HR, New York Blood Center

Henry A. McKinnell, Board of Directors

Experience

▪ Chairman of the Board, Pfizer

▪ Chief Executive Officer, Pfizer


Topics for today’s discussion – Focus on Fidaxomicin

Market dynamics: Declining satisfaction and reduced use of Metro; significant unmet need due to recurrence

Value proposition: Clinical improvement and cost-savings from Fidaxomicin

Product positioning: Compelling 1st line therapy

Hospital engagement strategy: Our approach to reach physicians, hospitals, and payers


Market dynamics suggest the physicians are seeking better treatment

for C. Difficile

Increasing dissatisfaction with Metronidazole performance Zar et al. CID (2007) 45(3): 302-307 Bartlett. CID (2008) 46 (10): 1489-1492

Vancocin increasingly replaced in hospital by vancomycin “slurry” (generic)

Recurrence persists as an area of high unmet need unaddressed by current basket of “generic like” drugs


Despite IDSA recommendation for conservative use, hospital physicians

are choosing oral Vanco over Metro

1 Estimated from IDSA –Infectious Disease Society of America guidelines

2 Based on 131 physician hospital survey (ID, GI, Hospitalists/Internists)

25

39

75

58 3

0

Actual

prescribing behavior2 100

Anticipated share

by Tx guidelines1 100

MetroVanco Other

Vancomycin increasingly seems to be treating the infection

in a better manner than other available meds for C. Diff.

– Hospitalist

Expected vs. actual prescribing behavior

Percent


Our market projections already reflect a “generic like” market for Vanco

because of “slurry” use in the hospital

SOURCE: IMS; expert interviews; team analysis

Vancocin sales in Hospital1

$ Thousands

Flagyl and vancomycin slurry are very cheap

– Critical Care specialist

Since 2008, significant decline in hospital Vancocin

sales indicate prevalence of Vanco “slurry”

46.0

56.2

38.9

2008 2009

-17%

2010


31

33

22

38

30

5

7

5

33

50

14

10

0

0

0

100%

Safety profile 11 1

Efficacy in

mild-to-moderate CDI16 1

Efficacy in

severe complicated CDI74

0

Relapse/Recurrence rate

0

Efficacy in severe CDI 62

65 1

ID specialists & Internists

Level of need for new therapies

% of clinicians

Neutral

Somewhat low need

Very high need No need

Somewhat high need

SOURCE: Decision Resources

Physicians rate the need for therapies

that address recurrence as very high








Fidaxomicin has a strong value proposition in reducing recurrence in

key sub-populations

Recurrence

rate w/ Vanco

46%

Older age

(75+)

25%

Prior

episode

21% 20%

Immuno-

comp.

36%

Renally

impaired

30% 36% 27%30% 36%

Concom.

Abx

Fidaxomicin is clinically superior to Vancomycin for reducing

recurrence (by 47% from combined phase III trials)

Reduction of recurrence will drive significant improvement in quality

of life and cost effectiveness in patient populations at higher risk

% of CDI

cases

Optimer has

engaged

external

collaborators

to provide

peer-reviewed

analyses of

cost-

effectiveness

First line use based on narrow spectrum and

superior clinical profile for reducing recurrence

Physicians Payers Institutions


Fidaxomicin addresses CDI-associated costs directly, resulting in per-

patient savings of ~$4,000-8,000

Assumptions:

Cost of C. Diff episode is $13,000 or higher in US– O’Brien ICHE 2007;

Cost of VRE infection is $27,000-87,000 – Edlund et al, CID 1997; Salgado et al, ICHE 200

Cost of hospital isolation (VRE colonization) in acute setting $2,500: Kirkland & Weinstein Lancet 1999.

Increased rate of recurrence: Kelly, CP, LaMont, JT. Clostridium difficile – more difficult than ever. NEJM 2009;359(18):1932-40.

Prevent VRE infection (5% of 1/4 cases): 2 Vogel. CAMJ 2010

Economic

drivers Savings

Dollars

1,700-3,500

700-1,400Recurrence

1,000-2,500

500-1,100

VRE

colonization

Sub-pop.

complications

TBD

Total 3,800-8,500

Cost/case

Dollars

▪ Prevent first recurrence (1 in 6 cases)

▪ Prevent follow-on recurrence (1 in 15 cases)

▪ Prevent future isolation from VRE

colonization (1/4 cases)

▪ Prevent VRE infection (3-5% of 1/4 cases)

▪ Prevent treatment complications with co-

morbidity (e.g., chemotherapy for cancer)

Fidaxomicin value proposition

13,000-

90,000

27,000-

80,000

TBD

Health economics collaborations are validating

Fidaxomicin’s cost effectiveness message


What type of access would you expect

for Fidaxomicin on your formulary?

Hospitals will likely grant restricted access to Fidaxomicin,

physician responses suggest minimal impact

SOURCE: Two separate surveys of Physicians (N=131 & N=200)

66

34

1

Not approved

Access with

restrictions

Open

access

Percent

Physician’s

treatment

paradigm

% impact on

prescribing

behavior

Use Fidaxo for

severe C. diff

Not significant

Use Fidaxo

1st line

From 50% to 40%

Other Tx

paradigms

Not significant

▪ Behavior not impacted by generic availability, slurry usage, and payor restrictions

▪ Restrictions do not impact behavior in subpopulation-driven treatment paradigms

ID approval

most

common

restriction

mentioned


Payer access is not likely to be a barrier for Fidaxomicin

Payers likely to cover

Fidaxo at a price

premium

Payers unlikely to

switch drugs on

discharge

Impact of generic

Vancocin minimal in

short- to medium-term

Payers agree with our

positioning and benefit

All payers interviewed agree that Product X

could be positioned as the first line of treatment

for patients at-risk of recurrence

“We rank drugs as „clearly inferior, clinical parity,

or clearly superior.‟ This is clearly superior – it‟s

better on all relevant clinical aspects.”

“I‟d be a fool to switch a patient off of a drug

that‟s working when they come out of the

hospital”

“Vanco will probably get more favorable tier

status, but your product‟s status shouldn‟t

change.”

SOURCE: Payer executive interviews (N=8)








Physician intent to prescribe Fidaxomicin as 1st line treatment for C.

Diff. ranges from 35-50%

1 Similar matrix used, but not shown to calculate share

SOURCE: Survey of Physicians (N=200)

Prescribing

intent

(% of patients)

Physician’s

treatment

paradigm

Other Tx

paradigms

% of C.

Diff cases

28% 8%

% of

physicians

35%Use Fidaxo for

severe C. diff

35%36%

50%Use Fidaxo

1st line

37%27%

37%

Implied 1st line market share 32%

Implied 2nd line market share1 52%

Opportunity to increase market share by

▪ Move physicians to 1st line paradigm

▪ Increase use within each paradigm

X

X

X








We will communicate burden of C. Diff. pre-launch through medical

education of hospital stakeholders ...C

lin

ica

lC

os

t

Recurrence▪ Likelihood of recurrence

high in key sub-pops

VRE▪ Impact of colonization

and infection

Spores▪ Key factor in level of

infection in hospitals

Broad cost impact▪ Re-admission, isolation,

increased LOS

By sub-pop▪ E.g., delay to radiation for

cancer patients

Incidence▪ Incidence of CDI high in

key sub-populations

Concom. ABx▪ Outcomes for con-

comitant ABx patients

▪ Peer-reviewed publications

▪ Continuing Medical Education

▪ Targeted outreach to KOLs

– Clinical Ad. Boards, trial

leadership

▪ Collaborations with infection

control

Key messages Hospital engagement


... and communicate the strengths of Fidaxo after approval through

hospital engagementC

lin

ica

lO

the

r

VRE▪ Colonizes in gut 31% with

Vanco, 7% with Fidaxo

Resistance▪ Extremely low resistance

with in vitro testing

Broad cost impact▪ Reduced re-admission &

transmission, lower VRE

By sub-pop▪ e.g., delay to radiation for

cancer patients

Reportable disease▪ Standardize reporting for

hospitals/regions/ states

Recurrence▪ Reduces recurrence by

~50%

Spores▪ 2 log difference vs. Vanco

▪ Multi-disciplinary hospital

– Account representatives

– Health economists

– Medical education &

research liaisons

Guidelines▪ Influence guidelines to

include key sub-pops

Co

st

Key messages Hospital engagement


Peer-reviewed publications will support Fidaxomicin’s brand position

Category

Age (75+)▪ Highest risk sub-population; Fidaxo

improves outcomes and quality of life

Publication Topic

Concom.

Abx

▪ Fidaxo’s clinical benefits maintained

in sub-populations on Concom. Abx

Prior

episode

▪ As recurrence rates rise, Fidaxo

clinical benefits strengthen

Immuno▪ High cost of recurrence (i.e., relapse

in radiation, quality of life, LOS)

Renal

Impaired

▪ In development: Fidaxo beneficial

impact for Renal-insufficient patients

Journals will enable launch

Supplemental/Cascading

publications

▪ Targeted at stakeholders

▪ Will enhance circulation

Medical education

▪ Experts equipped with re-

prints

▪ Communications (e.g., Ad

Boards, Speaker programs

and Symposia)

Public relations impact

▪ Key milestone publicity

▪ Managed markets leverage

▪ KOL development

▪ Advocacy coalition outreach


Optimer will target ~70% of CDI incidence opportunity by going after

~20% of Hospitals (~1,100 hospitals)

CDI Cases

100%

70%

30%

Hospitals

100%

20%

80%

High concentration of CDI in hospitals Selection criteria

Strategy

▪ Top ~1,100 hospitals chosen due to

3 criteria

– CDI Incidence

▫ ~70% of CDI

– KOL recommendations

▪ AMCs: Prioritize by size & influence

(5-7 per HAM)

▪ Community hospitals: Prioritize by

size & Vancocin use (14-18 per HAM)

▪ Early adopters: Historically low

access barriers; 70% of Oral Vancocin

sales in 300 of 1,100 hospitals

▪ Other: Selected by proximity,

strategic access (e.g., VA/DOD)

Source: IMS Health


~68% of CDI is concentrated in 11 states

Same 11 states account for:

65% of hospitals

64% of beds

62% of Vancocin

Opportunity from under-reporting

Percent

By geography, CDI concentrated in 11 states; disease incidence tracks

with number of hospitals and beds

Arkansas

Arizona

California

Colorado

Iowa

Idaho

Kansa

s

Louisiana

Minnesota

Missouri

MontanaNorth

Dakota

Nebraska

New

Mexico

Nevada

Oklahoma

Oregon

South

Dakota

Texa

s

Utah

Washington

Wyomin

g

Alabama

Florida

Georgi

a

IllinoisIndiana

Kentucky

Missis-

sippi

North

Carolin

a

Ohio

Pennsy-

lvania

South

Carolina

Tennesse

e

West

Virginia

New Jersey

New Hampshire

New York

Vermon

tMassa-

chusetts

Connecticut

Rhode IslandMichigan

Wisconsin

Alaska

Hawaii

Delaware

Main

e

MarylandVirginia

Priority

Texas 8%

Pennsylvania 8%

Ohio 7%

New York 7%

New Jersey 4%

Missouri 5%

Michigan 5%

Massachusetts 4%

Illinois 8%

Florida 6%

California 6%

State % of US CDI

6%

5%

4%

7%

3%

2%

3%

2%

4%

8%

11%

% of 65+ pop.

Likely under-reporting

Source: IMS Health; 2009 US census


Our operating model will drive awareness and access with all relevant

stakeholdersNumber of employeesX

P&T Committee

▪ Infectious disease

▪ Pharmacy

▪ Management

Infection control

Procurement

Hospital

leadership

Physicians

Nurses

3-5 10-20 5-10 2-3

50-75

Quality

assurance

Health economists

Equip reps with

Hospital-focused

Budget Impact Model

Medical education

research liaisons

Communicate clinical

product differentiation;

Liaise with KOLs

Managed care

Achieve favorable

market access with

payers

Marketing

Communicate brand

value to influence key

stakeholders

Account representatives

▪ 5 major regions; 10-15 hospitals per rep

▪ Frequency and blend of rep/MSL driven by access,

control, system priority


Our aspirations for Fidaxomicin

Establish Fidaxomicin as 1st line treatment for C. Diff. in at-

risk of recurrence patients

Effectively communicate the clinical and cost-effectiveness

superiority of Fidaxomicin to the medical community

Create a nimble, best-in-class US commercial organization to

accelerate access for differentiated products in the hospital

Advance a lifecycle plan

Continue to build our IP protection


Product pipeline

Other ongoing business priorities – Pipeline

Market

Fidaxomicin

Fidaxomicin (oral

suspension)

▪ Fidaxomicin has been granted Fast-

Track status by the FDA and is an

investigational new drug in the Div.

of anti-Infective and Ophthalmology

products’ CMA pilot 2 program

Pruvel

(Prulifloxacin)

▪ Pruvel is currently marketed in

Japan and Europe by other

licensees of Nippon Shinyaku

OPT-822/OPT-821

(OBI, Taiwan

subsidiary)

▪ OPT-822 combined with an

adjuvant OPT-821, a carbo-hydrate-

based immunotherapy, is being

investigated by Optimer’s Taiwan

subsidiary, OBI for the treatment of

metastatic breast cancer

OPopS platform ▪ OPopS is a proprietary platform for

rapid carbohydrate synthesis which

has generated two out-licensed

candidates including OPT-822/821

Regulatory

reviewPhase 1 Phase 2

Preclinical

research Phase 3

CDI treatment vs. Vancocin

CDI – oral suspension

Infectious Diarrhea

Metastatic breast cancer

Ongoing

initiatives

Fidaxomicin (new

indications)Ongoing

initiatives

We aspire to be a leading hospital drug company in the US


Value creating events and milestones

2011

2010 Fidaxomicin Second Phase 3 trial successful top-line data

Fidaxomicin ECCMID & DDW Conferences

Fidaxomicin MAA Filed in EU

Fidaxomicin ICAAC & IDSA Conferences

Fidaxomicin NDA Filed in U.S.

New Hires General Counsel & Access and SVP, Comm. Ops

New Hires SVP, HEOR; SVP, HR; VP, Bus. Dev.

Fidaxomicin Validation of NDA Filing & Priority Review Decision

Fidaxomicin Peer-reviewed Publication (First Phase 3 trial)

OPT-822/821 Phase 2/3 trial initiation in Taiwan

Fidaxomicin Advisory Committee Meeting

Fidaxomicin PDUFA Decision Date

Fidaxomicin CHMP opinion / EMA Decision

Fidaxomicin U.S. Launch

Fidaxomicin EU Launch

Fidaxomicin EU and Pruvel strategic optionsOngoing

Documents

Capturing the value of Fidaxomicin - shareholderfiles.shareholder.com/downloads/OPTR/0x0x435614... · Capturing the value of Fidaxomicin January 12th 2011 Ticker: ... whether caused