Investor Reception ASH 2015 - MorphoSys AG · The MorphoSys Pipeline 25 Clinical Product Candidates, 104 Total Program Partner Target Disease Area Discovery Preclinic Phase 1 Phase

Investor Reception

ASH 2015

December 7, 2015

MorphoSys AG - Investor Event ASH - December 7, 2015 1

Agenda


8:00 Simon Moroney, D.Phil., CEO

Welcome & Introduction

8:05 Arndt Schottelius, MD, Ph.D., CDO

MOR208 – Results of phase 2 with MOR208 monotherapy in NHL

8:15 Jennifer Woyach, MD

Assistant Professor of Internal Medicine, Ohio State University, USA

MOR208 in CLL

8:35 Prof. Dr. Heinz Ludwig

Director of the Wilhelminen Cancer Research Institute, Vienna, Austria

MOR202 – Results of ongoing phase 1/2a study in MM

8:50 Q&A

Closing Remarks

Food, Drinks

Safe Harbor

MorphoSys AG - Investor Event ASH - December 7, 2015

This presentation includes forward-looking statements.

Actual results could differ materially from those included in the forward-looking statements due to

various risk factors and uncertainties including changes in business, economic competitive

conditions, regulatory reforms, foreign exchange rate fluctuations and the availability of financing.

These and other risks and uncertainties are detailed in the Company’s Annual Report.

3


The MorphoSys Pipeline

25 Clinical Product Candidates, 104 Total

Program Partner Target Disease Area Discovery Preclinic Phase 1 Phase 2 Phase 3

Bimagrumab (BYM338) Novartis ActRIIB sIBM (musculoskeletal)

Guselkumab (CNTO1959) Janssen IL23p19 Psoriasis

Gantenerumab Roche Amyloid-ß Alzheimer’s disease

MOR208 - CD19 ALL, CLL, NHL

MOR103/GSK3196165 GSK GM-CSF Inflammation

MOR202 - CD38 Multiple myeloma

BHQ880 Novartis DKK-1 Multiple myeloma

CNTO3157 Janssen - Inflammation

CNTO6785 Janssen - Inflammation

LFG316 Novartis C5 Eye diseases

LJM716 Novartis HER3 Cancer

BPS804 Mereo/Novartis Sclerostin Brittle bone syndrome

Tarextumab (OMP-59R5) OncoMed Notch 2 Solid tumors

VAY736 Novartis BAFF-R Inflammation

MOR209/ES414 Emergent PSMA/CD3 Prostate cancer

Anetumab Ravtansine (BAY94-9343) Bayer Mesothelin (ADC) Solid tumors

BAY1093884 Bayer TFPI Hemophilia

BI–836845 BI IGF-1 Solid tumors

NOV–7 Novartis - Eye diseases

NOV–8 Novartis - Inflammation

NOV-9 Novartis - Diabetic eye diseases

NOV-10 Novartis - Cancer

NOV-11 Novartis - Blood disorders

PF-05082566 Pfizer 4-1BB Solid tumors

Vantictumab (OMP-18R5) OncoMed Fzd 7 Solid tumors

MOR106 Galapagos - Inflammation

MOR107 (LP2) - AT2-R Fibrosis

Immuno-oncology program Merck Serono - Cancer

Immuno-oncology program Immatics - Cancer

6 MOR programs - - Various

90 Partnered Programs

13 MOR Programs

1 Outlicensed Program

Most advanced development stage

In addition, 26 partnered programs in pre-clinic, and 43 partnered programs in discovery

The MOR Portfolio


Program Indication Target Discovery Preclinic Phase 1 Phase 2 Phase 3

Unpartnered

MOR208 NHL

CD19CLL

ALL

MOR202 Multiple myeloma CD38

MOR107 Fibrosis AT2-R

Immuno-oncology program(Immatics)

Cancer Various

6 Programs Various Various

Co-development & co-promotion

MOR209/ES414 (Emergent)

Prostate cancer PSMA / CD3

MOR106(Galapagos)

Inflammation Undisclosed

Immuno-oncology program(Merck Serono)

Cancer Undisclosed

Outlicensed to GSK

MOR103/GSK3196165

RA/handosteoarthritis

GM-CSF

FTD, orphan status US & EU

Orphan status US & EU

Phase 2a study of single-agent MOR208 in

patients with relapsed or refractory B-cell

non-Hodgkin’s lymphoma

Dr. Arndt Schottelius


MOR208

Introduction


MOR208 is an Fc-engineered humanized monoclonal antibody that targets CD19

CD19, a B-lymphocyte lineage specific surface antigen, is the earliest and most broadly expressed

of the selective B-cell markers, and is highly expressed in most B-cell NHLs1,2

Consequently, a CD19 antibody may have clinical utility as a new therapeutic approach to treat B

cell malignancies

A phase I study has shown MOR208 to be generally safe and well-tolerated, with encouraging single-

agent activity in patients with CLL

Recommended intravenous dose, 12 mg/kg, weekly3

1 Wang K, et al. Exp Hematol Oncol 2012;1:362 Schuurman HJ, et al. Am J Pathol 1988;131:102-11

3 Woyach JA, et al. Blood 2014;124:3553-60CLL, chronic lymphocytic leukemia;

NHL, non-Hodgkin’s lymphoma

MOR208 - Phase 2 in R/R NHL

Objectives of the Study


Primary

Antitumor activity of single-agent MOR208 in adult patients with R-R NHL who had received at least

one prior therapy containing the CD20 antibody, rituximab

Primary endpoint: objective response rate

Secondary

Duration of response and PFS

Safety and tolerability

Potential immunogenicity

Pharmacokinetics and pharmacodynamics

PFS, progression-free survival; R-R, relapsed or refractory


Baseline Characteristics: Heavily Pre-treated Patients

9MorphoSys AG - Investor Event ASH - December 7, 2015

CharacteristicDLBCL*

n=35

FL/iNHL*

n=45

MCL

n=12

Total

n=92

Median age, years 71 66 64.5 66.5

Male, n (%) 24 (69) 21 (47) 11 (92) 56 (61)

Ann Arbor stage, n (%) I–II 4 (11) 5 (11) 1 (8) 10 (11)

III–IV 30 (86) 40 (89) 11 (92) 81 (88)

Missing 1 (3) 0 0 1 (1)

ECOG PS, n (%) 0-1 32 (91) 44 (98) 11 (92) 87 (95)

2 3 (9) 1 (2) 1 (8) 5 (5)

Prior lines of therapy, n (%) 1 15 (43) 17 (38) 3 (25) 35 (38)

2 8 (23) 7 (16) 1 (8) 16 (17)

≥3 12 (34) 21 (47) 8 (67) 41 (45)

Last rituximab dose <6 months, n (%) 14 (40) 6 (13) 1 (8) 21 (23)

Prior stem-cell transplant 2 (6) 7 (16) 1 (8) 10 (11)

Data cutoff November 11, 2015

* DLBCL and FL cohorts expanded leading to an overall enrollment of 92 patients; Other iNHL cohort not expanded as response was

deemed too heterogeneous between different lymphoma subtypes; data are presented for the combined FL/other iNHL cohort

Advanced Stage, Heavily Pre-treated Patients.

Jurczak W et al., #1528 ASH 2015


Promising Efficacy Results in DLBCL, FL, iNHL


Best overall response,* n (%)DLBCL

n=35FL/iNHL n=45

MCL

n=12

Total

n=92

Complete response 2 (6) 5 (11) 0 7 (8)

Partial response 7 (20) 7 (16) 0 14 (15)

Stable disease 5 (14) 21 (47) 6 (50) 32 (35)

Progressive disease 11 (31) 7 (16) 5 (42) 23 (25)

Not evaluable‡ 10 (29) 5 (11) 1 (8) 16 (17)

ORR (all patients) 9 (26%) 12 (27%) 0 21 (23%)

ORR (evaluable patients§) 9 (36%) 12 (30%) 0 21 (28%)

*Investigator assessed

Encouraging Single-agent Activity for MOR208 in NHL

‡Post-baseline response assessment not performed/data unavailable §n = 25, 40, 11 and 76, respectively

ORR (CR + PR), objective response rate



Long Duration of Responses in DLBCL, FL/iNHL


0.0 5.0 10.0 15.0 20.0 25.0

Months

Pati

en

ts w

ith

CR

or

PR

Duration of response

DLBCL, n=9

Indolent NHL,* n=12

Time to response, n=21

Ongoing response, n=9

The longest response duration observed so far exceeded 20 months in both DLBCL and FL

FL/iNHL, n=12



Adverse events


Grade 3/4,* n (%)DLBCL

n=35

FL/iNHL

n=45

MCL

n=12

Total

n=92

Hematological

Any 9 (26) 4 (9) 1 (8) 14 (15)

Neutropenia 6 (17) 2 (4) 0 8 (9)

Anemia 3 (9) 0 0 3 (3)

Thrombocytopenia 2 (6) 1 (2) 1 (8) 4 (4)

Non-hematological

Dyspnea 2 (6) 1 (2) 1 (8) 4 (4)

Fatigue 1 (3) 1 (2) 0 2 (2)

Hypokalemia 1 (3) 1 (2) 0 2 (2)

Pneumonia 2 (6) 0 0 2 (2)

MOR208 12mg/kg was well tolerated with favorable safety profile

Infusion-related reactions reported in only 9 (10%) of 92 patients

Treatment emergent adverse events reported in ≥2 patients


Conclusions


Encouraging single-agent activity for MOR208 in NHL

Promising objective response rates observed; 36% in the DLBCL cohort and 30% in the combined

FL/iNHL cohort (evaluable patients)

2 complete responses in the DLBCL cohort

5 complete responses in the combined iNHL cohort

Longest duration of response to date is 20.3 months for DLBCL and 20.3 months for iNHL (both

ongoing)

MOR208 was well tolerated — infusion-related reactions reported only in 10% of patients and were

typically grade 1/2

Favorable pharmacokinetic profile and very low immunogenicity

Protocols are being developed for trials which combine MOR208 with other anti-lymphoma therapies

(e.g. lenalidomide and bendamustine)

MOR208

Comprehensive Clinical Development Plan


Indication 2015 2016 2017 2018

NHL

DLBCL

CLL

ALL

Phase 2

Phase 2/3

IIT: Investigator-initiated trial

MOR208 (12mg/kg) + lenalidomide; 2nd line R/R; N=80

Safety evaluation leading into anticipated pivotal study

MOR208 (12 mg/kg) + bendamustine; 2nd line R/R; N~320

MOR208 (12mg/kg) + idelalisib; BTKi-failures; N=120

MOR208 (9mg/kg) + lenalidomide; R/R, naive & Richter’s Transformation; MOR208 + ibrutinib in ibrutinib

failures; N=80 (Ohio State Univ. IIT)

MOR208 (12mg/kg) + NK cells; pediatric ALL; N=13 (St Jude’s IIT)

MOR208 (12 mg/kg); N=92

A Phase II Study of the Fc Engineered CD19

Antibody MOR208 in Combination with

Lenalidomide for Patients with Chronic

Lymphocytic Leukemia (CLL)

Jennifer Woyach, MD

Assistant Professor of Internal Medicine, Ohio State University


Open-label, multi-dose, single-arm, dose escalation study

Enrolled 27 heavily pretreated high risk patients suffering from relapsed or refractory CLL

Primary objective: safety, tolerability, pharmacokinetic profile

Secondary objective: anti-tumor activity

Dosing in 6 cohorts:

Dosing Scheme:

MOR208 in Clinical Development

Phase 1 in R/R CLL – Study Design


A Phase 1 Study of MOR208 to Evaluate the Safety, Tolerability, and Pharmacokinetics in Patients With

Relapsed or Refractory Chronic Lymphocytic Leukemia (R/R CLL)

d1 d4 d8 d15 d1 d8 d15d22 d22

cycle 1

maintenance with 4 monthly infusions

offered to patients with at least SD in phase I

cycle 2

Dose (mg/kg)0.3

mg/kg

1

mg/kg

3

mg/kg

6

mg/kg

9

mg/kg

12

mg/kg

No of patients (total n = 27) 1 1 3 3 316

(including P2a expansion)

MOR208 - Phase 1 in R/R CLL

Safety Overview: MOR208 is Well Tolerated


Treatment-related adverse Events0.3 mg/kg

(N=1)

1 mg/kg

(N=1)

3 mg/kg

(N=3)

6 mg/kg

(N=3)

9 mg/kg

(N=3)

12 mg/kg

(N=16)Total (%)

Any Event 1 1 3 3 2 14 24 (88.9%)

Grade 3/4 Toxicities

Grade 4 Neutropenia lasting ≥ 7 days with febrile

neutropenia (DLT)1 1 (3.7%)

Neutropenia 1 1 2 (7.4%)

Thrombocytopenia 2 2 (7.4%)

Tumor Lysis Syndrome 1 1 (3.7%)

Increased AST 1 1 (3.7%)

Grade 1/2 Toxicities Occurring in >10% of patients

Infusion Reaction 1 1 1 2 2 11 18 (66.7%)

Increased ALT 2 1 2 5 (18.5%)

Increased AST 1 2 1 4 (14.8%)

Fever 1 1 1 1 4 (14.8%)

Thrombocytopenia 1 2 3 (11.1%)

Chills 1 1 1 3 (11.1%)

Peripheral Sensory Neuropathy 1 2 3 (11.1%)

Infusion reactions (IRR) of grade 1 or 2 were the most common related AEs.IRR occurred only during 1st infusion and no grade ≥3 IRR were reported.

The rate of infections was very low. MTD was not reached. Woyach et al, Blood 2014


High Single Agent Overall Response Rate


Best Response, n (%)

0.3 - 9 mg/kg

(N=11)

12 mg/kg

(N=16)

Total

(N=27)

Response by IWCLL 2008 criteria (CT scan)

Complete Response 0 0 0

Partial Response 2 (18%) 6 (38%) 8 (30%)

Stable Disease 7 (64%) 10 (62%) 17 (63%)

Progressive Disease 2 (18%) 0 2 (7%)

ORR in 12mg/kg (recommended phase 2 dose): 38% (IWCLL2008)

Responses by NCI96 criteria (physical exam)

Complete Response 0 0 0

Partial Response 6 (55%) 12 (75%) 18 (67%)

Stable Disease 5 (45%) 4 (25%) 9 (33%)

Progressive Disease 0 0 0

MOR208 shows encouraging single-agent efficacy

Woyach et al, Blood 2014


Encouraging ORR in Subgroup Analysis (IWCLL 2008)


Best Response

(IWCLL2008), n (%)

N

(all)

ORR

(all patients)

N

(12 mg/kg)

ORR

(12 mg/kg)

Overall 27 30% 16 38%

Del17p 10 30% 3 67%

Del11q 8 38% 5 40%

Largest LN > 5 cm 9 11% 5 20%

Largest LN ≤ 5 cm 18 39% 10 50%

MOR208 also shows promising efficacy in high risk patients


Phase 1 in R/R CLL

Best ALC Reduction and Tumor Shrinkage


Median absolute lymphocyte count reduction from baseline: 91%Pronounced nodal size reduction from baseline by CT scan

ALC: absolute lymphocyte count

-100

10

0

-10

-20

-30

-40

-50

-60

-70

-80

-90

1.0 mg/kg

9.0 mg/kg

3.0 mg/kg12.0 mg/kg

0.3 mg/kg

6.0 mg/kgDose

% C

hange f

rom

Base

line


40

20

0

-20

-40

-60

-80

-100

Best ALC Reduction from Baseline Tumor Shrinkage (Lymph Nodes)

Phase 1 in R/R CLL

Long Progression-Free Survival


Patients within the 12 mg/kg extended dosing (up to 6 cycles) show impressive PFS

Median, 420 days 14 months

12 mg/kg expansion cohort (N=8)

Max. MOR208 treatment period



MOR208 ranks highest among various α-CD19 & α-CD20 antibodies

38%24% 30%

23%13%

MOR20812m g /k g( n =16 )

MED I - 551ph a se I / I I 12m g /k g

( n =26 )

Ob in u t u zu mab ph a se I I( n =20 )

O f a t u m um abph a se I I I( n =196 )

R i t u x im a b( n =110 )

Single-agent Antibodies in R/R CLL*

MEDI-551 data source: Poster ASCO 2013, 12mg/kg dosing group

Obinutuzumab data source: GAUGUIN study, Cartron et al, Blood 2014

Ofatumumab data source: control arm in ibrutinib vs. O phase III trial (RESONATE, ASCO 2014)

Rituximab data source: Late breaking abstract #6, ASH 2013

Criteria: Hallek et al 2008 (including CT)

ORR

PD

SD

α-CD19 mabs α-CD20 mabs

*IWCLL2008 criteria

**14 months within expansion cohort;

NR: not reported

Non-evaluable/

info not available if SD or PD

PFS:(months)

NR** 10.7 5.5814**

Best

ORR:

Phase 1 in R/R CLL

MOR208 is Superior to Other CD19 & CD20 MAbs

MOR208 in CLL: Ongoing Activities


Investigator sponsored trial (IST) - PI Jennifer Woyach, OSU

Single center, open-label, single arm study in CLL patients < 80 years of age

4 cohorts, recruiting ~80 pts:

A. Combination with lenalidomide in previously untreated patients, ineligible for approved chemo-

and/or immunotherapy options

B. Combination with lenalidomide in R/R disease

C. Combination with lenalidomide in Richter’s disease

D. Combination with ibrutinib in pts with R/R disease under ibrutinib

Address preclinical synergy of MOR208 & Lenalidomide in CLL patients

Cycle 1

MOR208 1 mg/kg D1

MOR208 9 mg/kg D2,8,15,22

+

Lenalidomide 2.5 mg po D9-28

Cycle 2-12

MOR208 9 mg/kg D1

+

Lenalidomide 2.5 mg po D1-28

(dose escalation to 10mg)

Cycle 13+

Optional

lenalidomide

alone until

progression

Woyach et al, #2953 ASH2015

Phase 2 IST in R/R & Treatment Naïve CLL

MOR208 Combo with Lenalidomide


Characteristic Previously Untreated

n (%)

Relapsed/Refractory

n (%)

Total Patients 7 9

Median Age (range) 67 (44-75) 70 (62-74)

Gender

Male 6 (86) 8 (89)

Female 1 (14) 1 (11)

Median number of prior therapies

(range)

0 3 (1-8)

IgVH Unmutated 6 (86) 7 (78)

Complex Stimulated Karyotype 1 (14) 7 (78)

Interphase Cytogenetics

del(13q14.3) 4 (57) 6 (66)

Trisomy 12 3 (43) 0

del(11q22.3) 1 (14) 3 (33)

del(17p13.1) 1 (14) 1 (11)

Patient Characteristics





Relapsed/Refractory Cohort (B)

Toxicities Occurring in >2 Patients, n (%)

Previously Untreated Cohort (A)

Toxicities Occurring in >1 Patient, n (%)

Grade 3+ Grade 3+

Hematologic Toxicities

Neutropenia 6 (66.7) 1 (14.3)

Non-Hematologic Toxicities

Hyperglycemia 2 (22.2) 0

Infusion reaction 0 1 (14.3)

Upper respiratory infection 0 1 (14.3)

Hypertension 2 (22.2) 2 (28.6)

The combination of MOR208 and lenalidomide has been well tolerated.

Toxicities Grade 3/4 Without Regard to Attribution





Preliminary Efficacy Evaluation

T r e a tm e n t N a iv e C o h o r t

P a tie n t

Ch

an

ge

in

Ly

mp

h N

od

e D

iam

ete

r

1 2 3 4

-1 0 0

-8 0

-6 0

-4 0

-2 0

0

6 m o n th e va lu a tio n

1 2 m o n th e va lu a tio n

Patient Best

Response

08* PR

09* PR

10* PR

14* PR

11 NE+

12 NE+

17 NE++

So far 3/9 patients with R/R disease and 4/7 patients with treatment naïve disease responded to therapy. Responses deepened over time.

R e la p s e d /R e fr a c to r y C o h o r t

P a tie n t

Ch

an

ge

in

Ly

mp

h N

od

e D

iam

ete

r

1 2 3 4 5

-1 0 0

-5 0

0

5 0




P a tie n t

Ch

an

ge

in

Ly

mp

h N

od

e D

iam

ete

r

1 2 3 4 5

-1 0 0

-5 0

0

5 0



R/R = relapsed/refractory, PU = previously untreated; NE = Non-evaluable

Patient Best

Response

01 PR

02* PR

03* PR

06 SD

07 SD

04 PD

05 PD

18 NE**

20 NE**


P a tie n t

Ch

an

ge

in

Ly

mp

h N

od

e D

iam

ete

r

1 2 3 4 5

-1 0 0

-5 0

0

5 0



Previously Untreated Relapsed/Refractory

* Still on therapy

** Have not yet reached first response evaluation

+ Discontinued due to infusion reaction

++ Discontinued due to unrelated toxicity


Phase 2 IST in R/R & Naïve CLL

MOR208 Combo with Lenalidomide / Ibrutinib


The combination of MOR208 and lenalidomide has preliminary activity in relapsed/refractory and

treatment-naïve CLL

This combination has been well tolerated, although escalation of lenalidomide above 2.5 mg has been

difficult

Responses have generally deepened over time. 5 patients have completed 12 cycles of therapy, and

2 of these remain on lenalidomide

Correlative studies to evaluate T and NK cell number and function are underway

Cohorts are currently being accrued to evaluate this combination in Richter’s transformation as well as

adding MOR208 to ibrutinib in patients undergoing molecular relapse

Conclusions


Ibrutinib Treatment in CLL

Incidence of Discontinuation


Maddocks et al, JAMA Oncol. 2015 Apr;1(1):80-7

Ibrutinib Treatment Failures


Maddocks et al, JAMA Oncol. 2015 Apr;1(1):80-7

Overall Survival in PatientsDiscontinuation after AEs (days)

Overall Survival in Patients Discontinuation after Progression on

ibrutinib (months)

Planned Phase 2 Study in CLL

MOR208 in Combination With Idelalisib


A Phase II, Single Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of

MOR208 Combined with Idelalisib in Patients with Relapsed or Refractory CLL/SLL Previously

Treated with Bruton’s Tyrosine Kinase (BTK) Inhibitor

Primary endpoint: ORR

N = 120

Patients: R/R CLL/SLL not responding or not able to tolerate BTK-inhibitor Tx (e.g. ibrutinib)

MOR208 12mg/kg: 3 cycles qw -> q2w until progression

Idelalisib 150 mg BID: continuously up to max. 24 cycle

Planned Start in Q1 2016

MOR202

A Novel CD38 mAb in Multiple Myeloma

Univ. Prof. Dr. Heinz Ludwig

Director of the Wilhelminen Cancer Research Institute

Vienna, Austria


MOR202 Mechanisms of Action:

High ADCC and ADCP Activity

32

MOR202

Fully human

monoclonal IgG1

antibody directed

against CD38

MOR202 induces

potent immune

effector mechanisms:

ADCC and ADCP


ADCC: Antibody-Dependent Cell-Mediated Cytotoxicity

ADCP: Antibody-Dependent Cell-Mediated Phagocytosis

CDC: Complement-Dependent cytotoxicity

no CDCCDC is suspected to be a

major contributor to

infusion reactions*

*Tawara et al. J Immunol 2008

MOR202 shows similar ADCC activity

on MM cells, but spares NK cells


In vitro ADCC activity profile of MOR202, surrogates of Daratumumab and Isatuximab and a variant

of MOR202

% s

pecific

killin

g

MOR202

Daratu

mumab

Isatuximab

Afucosylated .

MOR202

Daratu

mumab

Isatuximab

Afucosylated

0

10

20

30

40

50

60 AMO-1 NCI-H929

% s

pe

cif

ic N

K c

ell k

illin

g

MOR202 Daratumumab Isatuximab Afucosylated0

5

10

15

20

25

30

35

40

45

50

55

60

65

70

variant of MOR202Surrogate Surrogate

CD38 high expressing MM cell lines CD38 low expressing NK cells

Boxhammer et al, #3015 ASH 2015

CD38 mAbs in MM

MOR202 - Best-in-class Infusion Tolerability


MOR202 Daratumumab Isatuximab

ADCC (max) +++ +++ +++

ADCP (max) +++ +++ ?

CDC (max) - +++ +

NK cell sparing +++ + +

Infusion volume 250 ml 500-1000 ml ?

Speed of infusion 125 ml / hStart at 50 ml /h**

If IRR: restart with 25 ml/h?

Infusion time 2h6,5 h (1st infusion)

3,5 h (3rd infusion)4-6 h

IRRs

(with Steroids)+ 6% 70 / 77% 52%

MOR202: Raab et al. IMW 2015Daratumumab: Lokhorst et al. NEJM 2015

Isatuximab: Martin et al. ASCO 2014** Moreau @ Janssen Symposium IMW 2015+ Even without Steroids, MOR202 shows IRRs of only 40%

MOR202

Phase 1/2a in Relapsed / Refractory MM


Study design

Phase 1/2a multicenter, open-label, dose escalation study

3+3 design plus expansion cohorts

11 sites in Germany and Austria

Patient population

Patients with relapsed/refractory myeloma

for MOR202 +/- dex and combo with pomalidomide/dex: failure of ≥ 2 prior therapies

MOR202 combo with lenalidomide/dex: failure of ≥ 1 prior therapy

Outcome measures

Determination of MTD, recommended dose, regimen

Safety profile

Immunogenicity

Pharmacokinetic

Preliminary efficacy

Raab et al, #3035 ASH 2015

MOR202 - Phase 1/2a General Study

Design

Part A Cohorts 1–8

0.01 0.04 0.15 0.5 1.5 4.0 8.0 16.0

mg/kg q2w

Dose-escalation cohorts

3 + 3 scheme, 2-hour IV infusion

Confirmatory cohorts

at RP2D

Part B

4.0 mg/kg weekly

Part C

4.0 8.0 16.0 mg/kg weekly + DEX*

MOR202 +/- DEX

at RP2D/schedule


MO

NO

Part D

8 16 mg/kg weekly/DEX* + POM

Part E

8 16 mg/kg weekly/DEX* + LEN

MOR202/DEX + POM

MOR202/DEX + LEN

Available Data as of November 2015

CO

MBO

* 20mg DEX for patients >65 years, 40mg DEX for patients <65 years

MOR202 – Phase 1/2a Patient Characteristics of Clinically Relevant Cohorts


Schedule

MOR202 dosing

Patient number

q1w + Dex

4–16 mg/kg

n=10

q1w + POM/Dex

8 mg/kg

n=3

q1w + LEN/Dex

8 mg/kg

n=4

Median age, years 68 64 59

Gender, %

Male

Female

60

40

67

33

75

25

Karnofsky PS, %

Median 90 100 95

Lines of prior therapy, n

Median 4 3 2

Prior ASCT, % 80 33 75

Prior therapies, %

Bortezomib

Lenalidomide

Cyclophosphamide

Melphalan

Doxorubicin

Thalidomide

Pomalidomide

Carfilzomib

Panobinostat

100

100

90

80

60

50

10

10

0

100

100

33

100

0

0

0

0

0

100

25

100

75

50

0

0

0

25

Raab et al., #3035 ASH 2015

MOR202 – Phase 1/2a

Safety of Clinically Relevant Cohorts


Most frequently reported treatment emergent adverse events

Schedule

Patient number

q1w + Dex

n=10

q1w + POM/Dex

n=3

q1w + LEN/Dex

n=4

TEAEs, n (%) Grade ≥3 Any ≥3 Any ≥3 Any

Haematological

Lymphocyte count decreased

Neutrophil count decreased

WBC count decreased

Thrombocytopenia

Anaemia

2 (20%)

2 (20%)

0

1 (10%)

1 (10%)

3 (30%)

3 (30%)

2 (20%)

3 (30%)

2 (20%)

1 (33%)

2 (67%)

0

0

1 (33%)

1 (33%)

2 (67%)

1 (33%)

1 (33%)

1 (33%)

2 (50%)

1 (25%)

1 (25%)

1 (25%)

1 (25%)

2 (50%)

1 (25%)

3 (75%)

1 (25%)

1 (25%)

Non-haematological

Fatigue

Back pain

Diarrhoea

Hyperuricaemia

Insomnia

Polyneuropathy

0

0

0

0

0

0

5 (50%)

3 (30%)

0

2 (20%)

3 (30%)

2 (20%)

0

0

1 (33%)

0

0

0

1 (33%)

1 (33%)

1 (33%)

0

0

1 (33%)

0

0

0

1 (25%)

0

0

0

0

2 (50%)

1 (25%)

0

0

Only 3/52 patients (6%) discontinued due to AEs with a suspected relationship to MOR202

No treatment-related deathsRaab et al, #3035 ASH 2015


Infusion-related reactions (IRRs)

39

Data from patients treated with the clinically relevant dose regimens who received ≥ 1 dose of

MOR202.

Dex, dexamethasone; LEN, lenalidomide; POM, pomalidomide; IRR, infusion-related reaction.

MOR202 q1w + Dex No IRR IRR Grade 1

4 & 8mg/kg + Dex

16 mg/kg + Dex

8 mg/kg + POM/Dex

8 mg/kg + LEN/Dex

7

2

3

4

0

1

0

0

Total; n (%) 16 (94 % ) 1 (6%)




Best maximum change in M-protein*

40

Data from patients treated with the clinically relevant dose regimens who received > 1 treatment

cycle

*Updated data from 23 November 2015. Serum M-protein data are shown.

In case serum M-protein is not available urine M-protein data are used (S, serum; U, urine)




Time on study and best response*

41

Data from patients treated with the clinically relevant dose regimens who received > 1 treatment

cycle.

* Updated data from 23 November 2015. **Not confirmed yet.

Dex, dexamethasone; LEN, Lenalidomide; MR, minor response; POM, Pomalidomide; PD, progressive disease; PR, partial

response; q1w, weekly; SD, stable disease; VGPR, very good partial response.




Summary of Preliminary Efficacy Data


Outcome of Single Agent MOR202 (weekly + Dex)

VGPR and PR: 3/9 evaluable patients

SD: 6/9 evaluable patients

ORR of 30%

Outcome of combo of MOR202 with ImiDs

VGPR and PR: 3/6 evaluable patients

MR: 1/6 evaluable patients

Responder Analysis (all patients)

Rapid decrease in M-Protein

Improvement in quality of remission with

longer treatment duration

Ongoing responses: 5/6 patients

Best stabilization: 52+ weeks (12+ months)

DoR 2+, 3, 3+, 3+, 4+, 8+ monthsData from patients treated with the clinically relevant dose

regimens who received > 1 treatment cycle.

* Updated data as of 23 Nov 2015.

** Not confirmed yet.


Conclusion


HuCAL IgG1 antibody binding unique epitope on CD38

One of only three CD38 antibodies in clinic

Main mode of action: ADCC and ADCP

Potent killing of MM cells

Low killing of healthy/effector cells

Active as single-agent therapy (ORR of 33%)

Active in combination with LEN/DEX or POM/DEX (clinical benefit rate of 67%)

Further cohorts will evaluate MOR202 16 mg/kg q1w in combination with LEN/DEX and POM/DEX

Excellent safety profile with only 7% IRR

MOR202 doses up to 16 mg/kg can be safely administered as a 2-hour IV infusion

Questions & Answers


Simon Moroney, D. Phil.

Arndt Schottelius, M.D., Ph.D.

Steffen Heeger, M.D., Ph.D.

Jennifer Woyach, MD

Assistant Professor of Internal Medicine

Ohio State University

Prof. Dr. Heinz Ludwig

Director of the Wilhelminen Cancer Research Institute,

Vienna, Austria

HuCAL®, HuCAL GOLD®, HuCAL PLATINUM®, CysDisplay®, RapMAT®, arYla® , Ylanthia® and 100 billion high potentials® are registered trademarks of MorphoSys AG.

Slonomics® is a registered trademark of Sloning BioTechnology GmbH, a subsidiary of MorphoSys AG.

Dr. Claudia Gutjahr-Löser

Head of Corporate Communications & IR

Phone +49 (0)89 / 899 27-122

Fax +49 (0)89 / 899 27-5122

Email [email protected]

Thank You

www.morphosys.com

Documents

Investor Reception ASH 2015 - MorphoSys AG · The MorphoSys Pipeline 25 Clinical Product Candidates, 104 Total Program Partner Target Disease Area Discovery Preclinic Phase 1 Phase