investor event ash 2016* - morphosys · single-agent mor208 in relapsed or refractory (r-r)...

$: Investor Event ASH 2016* - MorphoSys · Single-Agent MOR208 in Relapsed or Refractory (R-R) Non-Hodgkin’s Lymphoma (NHL): Results from Diffuse Large B-Cell Lymphoma (DLBCL) and$
Investor Event ASH 2016*

DECEMBER 5, 2016

* FOR INVESTORS ONLY. DO NOT DISSEMINATE.

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.

© MorphoSys AG, Investor Event, December 5, 2016 2

Agenda


8:00 Simon Moroney, D.Phil., Chief Executive Officer, MorphoSysMorphoSys‘s growing proprietary portfolio of biopharmaceuticals

8:10 Arndt Schottelius, M.D. Ph.D., Chief Development Officer, MorphoSysSingle-Agent MOR208 in Relapsed or Refractory (R-R) Non-Hodgkin’s Lymphoma (NHL):Results from Diffuse Large B-Cell Lymphoma (DLBCL) and Indolent NHL Subgroups of a Phase IIa Study

8:20 Kerry A Rogers, M.D., Division of Hematology Department of Internal Medicine, The OhioState UniversityUpdated Results from a Phase II Study of the Fc Engineered CD19 Antibody MOR208 in Combination with Lenalidomide for Patients with Chronic Lymphocytic Leukemia (CLL) and Richter's Transformation or Ibrutinib for Patients with Ibrutinib-Resistant Clones

8:35 Marc-Steffen Raab, M.D., Ph.D., University Hospital of Heidelberg, GermanyA Phase I/IIa Study of the CD38 Antibody MOR202 Alone and in Combination with Pomalidomide or Lenalidomide in Patients with Relapsed or Refractory Multiple Myeloma

8:50 Q&A Session

Recent Newsflow


November 2016:

Guselkumab in Moderate to Severe Plaque Psoriasis: Biologics License

Application Submitted by Janssen.

November 2016:

Strategic Partnership with LEO Pharma to Develop Antibodies in Dermatology

October 2016:

Start of New Partnered Clinical Program From Novartis

October 2016:

Updated response rates reported in RR multiple myeloma patients in higher

doses of MOR202 in combo with IMiDs in ongoing phase 1/2a trial

FIRST PRODUCT APPLICATION

SUBMITTED BY PARTNER

NEW STRATEGIC PARTNERSHIP

SUCCESSFUL CAPITAL INCREASE

GROWING CLINICAL PIPELINE

CLINICAL DATA UPDATES

November 2016:

Capital Raise of EUR 115m Intended to Support Further Pipeline

Development

PROGRAM PARTNER TARGET DISEASE AREA DISCOVERY PRECLINIC PHASE 1 PHASE 2 PHASE 3

Guselkumab (CNTO1959) Janssen IL23p19 Psoriasis

Gantenerumab Roche Amyloid-ß Alzheimer’s disease

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

BHQ880 Novartis DKK-1 Multiple myeloma

BI–836845 BI IGF-1 Solid tumors

Bimagrumab (BYM338) Novartis ActRIIB Musculoskeletal diseases

BPS804 Mereo/Novartis Sclerostin Brittle bone syndrome

CNTO3157 Janssen - Inflammation

CNTO6785 Janssen - Inflammation

MOR103/GSK3196165 GSK GM-CSF Inflammation

MOR202 - CD38 Multiple myeloma

MOR208 - CD19 ALL, CLL, NHL

Elgemtumab (LJM716) Novartis HER3 Cancer

Tarextumab (OMP-59R5) OncoMed Notch 2 Cancer

Tesidolumab (LFG316) Novartis C5 Eye diseases

Utomilumab (PF-05082566) Pfizer 4-1BB Solid tumors

VAY736 Novartis BAFF-R Inflammation

BAY1093884 Bayer TFPI Hemophilia

MOR209/ES414 Aptevo PSMA/CD3 Prostate cancer

MOR106 Galapagos - Inflammation

NOV–7 Novartis - Eye diseases

NOV–8 Novartis - Inflammation

NOV-9 Novartis - Diabetic eye diseases

NOV-10 Novartis - Cancer

NOV-11 Novartis - Blood disorders

NOV-12 Novartis - Prevention of thrombosis

NOV-13 Novartis - Cancer

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

MOR107 (LP2) - AT2-R Fibrosis

Immuno-oncology program Merck - Cancer

Immuno-oncology program Immatics - Cancer

6 MOR programs - - Various

The MorphoSys Pipeline28 Clinical Product Candidates, 110 Total*


In addition, 23 partnered programs in preclinic, and 50 partnered programs in discovery

2

15

11

* All product candidates are investigational and have not yet been established as safe and effective by regulatory authorities.

The MOR Portfolio5 Clinical Product Candidates*/**


** including MOR103, which is out-licensed to GSK

PROGRAM INDICATION TARGET DISCOVERY PRECLINIC PHASE 1 PHASE 2 PHASE 3

UNPARTNERED

MOR208 DLBCL (B-MIND)

CD19DLBCL (L-MIND)

CLL (planned)

CLL (IIT)

MOR202 Multiple Myeloma CD38

MOR107 Fibrosis AT2-R

Immuno-oncology program CancerMHC-associated

peptides

6 programs Various Various

CO-DEVELOPMENT AND CO-PROMOTION

MOR209/ES414 (Aptevo) Prostate cancer PSMA/CD3

MOR106 (Galapagos) Inflammation IL-17C

Immuno-oncology program

(Merck)Cancer Undisclosed

FTD, orphan status US & EU

Orphan status US & EU

* All product candidates are investigational and have not yet been established as safe and effective by regulatory authorities.

TODAY

A Vision for the Future


THE FUTURE

First product application submitted

for partnered antibody

Investing in proprietary pipeline

Strong technology platform

delivering differentiated

drug candidates

Fully-integrated biotech

Commercial footprint in EU

Sustainable income from partner

royalties

Fuels pipeline and R&D engine

Agenda






8:50 Q&A Session

AN INVESTIGATIONAL CD38 ANTIBODY IN CLINICAL STUDIES IN MULTIPLE MYELOMA (MM)

MOR202 - OverviewA Differentiated Antibody Option for Multiple Myeloma

Targeting CD38, a highly expressed and validated target in multiple myeloma

Preclinical models suggest that MOR202 may induce effector mechanisms ADCC and ADCP

KEY FEATURES & POSITIONING

Currently under clinical investigation as a potential option for RRMM

Pre-clinical data suggest preservation of NK cells and maintenance of CD38 expression

Biomarker data suggest expression of CD38 was preserved on bone marrow plasma cells during MOR202 treatment

Current clinical data suggest improved responses associated with longer treatment durations in patients in the higher

dosing cohorts, e.g.

− ORR of currently 100% in 16mg/kg MOR202+Len cohort;

− Responses currently ongoing for 14+ months versus 10+ months reported at ASCO 2016 (June 2016)

7% of patients showing IRRs of grade 1 or 2 in current phase 1/2a study

Analysts forecast central role and growing substantial market for CD38 mAbs in MM treatment in all lines

STATUS / NEXT DATA

Phase 1/2a dose escalation study ongoing, increasing number of patients to be studied in highest dosing cohorts

Updated clinical data at ASH 2016 and ASCO 2017


AN INVESTIGATIONAL FC-MODIFIED ANTIBODY TARGETING CD19

MOR208 - OverviewFc-modified Antibody to Treat B Cell Lymphoma


CD19 is preserved after treatment with B cell targeted therapies, as results from clinical/preclinical testing suggest

Preclinical models suggest that Fc-modification may enhance B cell depletion by ADCC, ADCP and direct cytotoxicity

KEY FEATURES & POSITIONING

Responses of >26 months in RR NHL patients reported in phase 2a monotherapy trial

No unexpected treatment-related safety signals reported to date in phase 2a monotherapy trial

Investigating the potential in phase 2 combination program in B-cell malignancies

− Further clinical investigation in RR DLBCL patients unsuitable for more aggressive therapies

− High unmet medical need in ibrutinib-refractory or ibrutinib-resistant CLL patients

Straightforward manufacturing

STATUS / NEXT DATA

R/R DLBCL: Two phase 2 combination trials ongoing (L-MIND & B-MIND), B-MIND planned to be transitioned into ph 3

Ibrutinib-refractory CLL: Phase 2 about to start (COSMOS)

CLL: IIT with Ohio State University ongoing, ASH update

Single-Agent MOR208 in Relapsed or Refractory (R-R) Non-Hodgkin’s Lymphoma (NHL): Results from Diffuse

Large B-Cell Lymphoma (DLBCL) and Indolent NHL Subgroups of a Phase IIa Study

Wojciech Jurczak,* Pier Luigi Zinzani, Gianluca Gaidano, Andre Goy, Mariano Provencio, Zsolt Nagy, Tadeusz Robak, Kami Maddocks, Christian Buske, Sumeet Ambarkhane, Mark Winderlich, Maren

Dirnberger-Hertweck, Jan Endell, Kristie A. Blum

*Jagiellonian University, Kraków, Poland


Study Design and Treatment

Non-randomized phase IIa multicenter study with 2-stage design (NCT01685008)

iNHL, indolent non-Hodgkin’s lymphoma; IV, intravenous; PR, partial response; SD, stable disease


Phase IIa Study of MOR208 in R-R NHL

• Primary objective

ORR in R-R NHL patients who had received at least one prior therapy containing rituximab

• Secondary objective

Duration of response and PFS

Safety and tolerability

Pharmacokinetics and pharmacodynamics

ORR, objective response rate; PFS, progression-free survival


Baseline Characteristics

Characteristic, n (%)DLBCLn=35

iNHL*n=45

MCLn=12

Totaln=92

Age, years Median 71 66 64.5 66.5

Sex Male 24 (69) 21 (47) 11 (92) 56 (61)

Ann Arbor stage 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 0-1 34 (97) 43 (96) 11 (92) 88 (96)

2 1 (3) 2 (4) 1 (8) 4 (4)

Prior lines of therapy 1 12 (34) 16 (36) 3 (25) 31 (34)

2 8 (23) 6 (13) 1 (8) 15 (16)

≥3 15 (43) 23 (51) 8 (67) 46 (50)

Rituximab refractory Yes 24 (69) 22 (49) 6 (50) 52 (57)

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

Prior stem cell transplantation Yes 4 (11) 8 (18) 1 (8) 13 (14)

*Includes follicular lymphoma and other indolent NHLsData are n (%) unless otherwise stated. Rituximab refractory was defined as patients who demonstrated less than a partial response or response lasting less than 6 months to a prior rituximab-containing regimen


Safety Profile

Grade ≥3 TEAEs,* n (%)DLBCLn=35

iNHL†

n=45MCLn=12

Totaln=92

Any‡ 19 (54) 14 (31) 4 (33) 37 (40)

Hematological¥

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

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

Anemia 3 (9) 0 0 3 (3)

Non-Hematological¥

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

Pneumonia 3 (9) 0 0 3 (3)

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

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

Infections and Infestations# 5 (14) 1 (2) 0 6 (7)

Infusion-related reactions,* n (%)DLBCLn=35

iNHL†

n=45MCLn=12

Totaln=92

Any 4 (11) 5 (11) 2 (17) 11 (12)

Grade 1/2 4 (11) 4 (9) 2 (17) 10 (11)

Grade 4 0 1 (2) 0 1 (1)

• There were no treatment-related deaths

Data are number of patients (%); *Treatment emergent adverse events (TEAEs) according to MedDRA preferred term (PT); ‡TEAEs including PT disease progression; ¥TEAEs of grade ≥3 reported in two or more patients overall; #TEAEs according to MedDRA system organ class; †includes follicular lymphoma and other iNHLs


Best Overall Response Rate


Best Tumor Shrinkage


Tumor Shrinkage: DLBCL

CR, complete response; PD, progressive disease


Tumor Shrinkage: iNHL Subtypes


Timing and Duration of Response

• 3 DLBCL patients still in remission, longest DoR >26 months, ongoing

• 6 iNHL patients still in remission, longest DoR >26 months, ongoing

• Median DoR 20.1 months in DLBCL and not reached in iNHL

R

R, rituximab refractory

R

R

RR

R

R

RR

DoR, duration of response

#

#

#

#

#

#

*

#

#

#

#


PFS in DLBCL and iNHL Subtypes


Subgroup Analysis of Disease Control Rate in DLBCL and iNHL Patients


PFS in Rituximab Refractory/Non-Refractory DLBCL and iNHL


Subgroup Analysis of Disease Control Rate in DLBCL and iNHL Patients


PFS in DLBCL and iNHL Patients with High and Low PeripheralNK Cell Count at Baseline


Summary and Conclusions

• ORR: 26% in DLBCL and 29% in iNHL

Target lesion shrinkage also observed in patients with stable disease (5/6 DLBCL and 14/17 iNHL)

• Long-lasting responses in DLBCL and iNHL

12 month PFS rate: 39% in DLBCL and iNHL

Longest responses: >26 months in DLBCL and iNHL

• MOR208 efficacious in patients with rituximab-refractory disease

• MOR208 well tolerated, also in long-term treatment

MOR208 showed encouraging single-agent activity in R-R DLBCL and R-R iNHL

MOR208 is currently being studied for the treatment of R-R DLBCL in:• A phase II trial in combination with lenalidomide (L-MIND)• A phase II/III trial in combination with bendamustine (B-MIND)


Agenda






8:50 Q&A Session


Woyach et al, ASH2016

Updated Results from a Phase II Study

of the Fc Engineered CD19 Antibody

MOR208 in Combination with

Lenalidomide for Patients with

Chronic Lymphocytic Leukemia (CLL)

and Richter’s Transformation or

Ibrutinib for Patients with Ibrutinib-

Resistant Clones

Kerry A Rogers, MDAssistant Professor

Division of Hematology Department of Internal Medicine,

The Ohio State University

Investigator Sponsored Trial (IST) at the Ohio State

University (OSU)


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 75 patients:

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

and/or immunotherapy options (planned 20 pts)

2. Combination with lenalidomide in R/R disease (planned 20 pts)

3. Combination with lenalidomide in Richter’s disease (planned 10 pts)

4. Combination with ibrutinib in pts with R/R disease under ibrutinib (planned 25 pts)




Characteristic Previously

Untreated

Number (%)

Relapsed/

Refractory

Number (%)

Richter’s

Number (%)

Ibrutinib

Progressing

Number (%)

Total Patients 11 11 5 7

Median Age (range) 62 (44-75) 70 (62-75) 60 (55-77) 62 (45-77)

Sex

Male 7 (64) 10 (91) 3 (60) 7 (100)

Female 4 (36) 1 (9) 2 (40) 0

Rai Stage

Low 1 (9) 0 NA 1(14)

Intermediate 4 (36) 4 (36) NA 3 (43)

High 6 (55) 7 (64) NA 3 (43)

Complex Stimulated

Karyotype3 (27) 9 (82) 4 (80) 6(86)

Interphase Cytogenetics

del(13q14.3) 4 (36) 7 (64) 3 (60) 1 (14)

Trisomy 12 5 (45) 1 (9) 1 (20) 2 (29)

del(11q22.3) 1 (9) 2 (18) 2 (40) 1 (14)

del(17p13.1) 1 (14) 2 (18) 4 (80) 5 (71)

Demographics - Baseline Characteristics

Study Design - Cohort 1



CYCLE 1

MOR00208 1 mg/kg IV on Day 1

MOR00208 9 mg/kg IV on Days 2, 8, 15 & 22

+

Lenalidomide 2.5 mg orally on Days 9-28

CYCLE 13+

Optional lenalidomide alone until progression

CYCLES 2-12


+

Lenalidomide 2.5 mg* orally on Days 1-28

* Dose escalation up to 10 mg daily

COHORT 1:

No previous treatment

Refuse or are ineligible for

standard therapy

Safety Profile - Cohort 1Combination of MOR208 and lenalidomide has been well tolerated



Cohort 1 Toxicities Occurring in >2 Patients Number (%)

Toxicity Grade

All 1/2 3 4

Hematologic Toxicities

Neutropenia 4 (36.4) 3 (27.3) 0 1 (9.1)

Lymphopenia 4 (36.4) 4 (36.4) 0 0

Thrombocytopenia 3 (27.3) 3 (27.3) 0 0

Non-Hematologic Toxicities

Hyperglycemia 8 (72.7) 7 (63.6) 1 (9.1) 0

Upper Respiratory Infection 6 (54.5) 6 (54.5) 0 0

Dyspnea 5 (45.5) 4 (36.4) 1 (9.1) 0

Infusion Reaction 5 (45.5) 3 (27.3) 2 (18.2) 0

Constipation 4 (36.4) 4 (36.4) 0 0

Cough 4 (36.4) 4 (36.4) 0 0

Diarrhea 4 (36.4) 4 (36.4) 0 0

Hyperhidrosis 4 (36.4) 4 (36.4) 0 0

Nausea 4 (36.4) 4 (36.4) 0 0

Increased ALT 3 (27.3) 3 (27.3) 0 0

Increased AST 3 (27.3) 3 (27.3) 0 0

Dry Mouth 3 (27.3) 3 (27.3) 0 0

Pain 3 (27.3) 3 (27.3) 0 0

Tinnitus 3 (27.3) 3 (27.3) 0 0




CYCLE 1



+


CYCLE 13+

Optional lenalidomide alone until progression

CYCLES 2-12


+



COHORT 2:

Relapsed or refractory disease




Cohort 1 Toxicities Occurring in >2 Patients Number (%)

Toxicity Grade

All 1/2 3 4


Neutropenia 8 (72.7) 2 (18.2) 5 (45.5) 1 (9.1)

Thrombocytopenia 9 (81.8) 8 (72.7) 0 1 (9.1)

Anemia 5 (45.5) 4 (36.4) 1 (9.1) 0


Hyperglycemia 10 (90.9) 8 (72.7) 2 (18.2) 0

Fatigue 8 (72.7) 7 (63.6) 1 (9.1) 0

Increased Creatinine 7 (63.6) 7 (72.7) 0 0

Bruising 6 (54.5) 6 (54.5) 0 0

Diarrhea 6 (54.5) 6 (54.5) 0 0

Dyspnea 6 (54.5) 6 (54.5) 0 0

Hypertension 6 (54.5) 4 (36.4) 2 (18.2) 0

Skin Disorder 6 (54.5) 6 (54.5) 0 0

Cough 5 (45.5) 5 (45.5) 0 0

Dizziness 4 (36.4) 4 (36.4) 0 0

Edema 4 (36.4) 4 (36.4) 0 0

Hypocalcemia 4 (36.4) 4 (36.4) 0 0

Hypomagnesemia 4 (36.4) 4 (36.4) 0 0

Infusion Reaction 4 (36.4) 4 (36.4) 0 0

Nausea 4 (36.4) 4 (36.4) 0 0

Pustular Rash 4 (36.4) 4 (36.4) 0 0

Abdominal Pain 3 (27.3) 3 (27.3) 0 0

Chills 3 (27.3) 3 (27.3) 0 0

Constipation 3 (27.3) 3 (27.3) 0 0

Flu Like Symptoms 3 (27.3) 3 (27.3) 0 0

Other GI Disorders 3 (27.3) 2 (18.2) 1 (9.1) 0

Hyperhidrosis 3 (27.3) 3 (27.3) 0 0

Hyperkalemia 3 (27.3) 3 (27.3) 0 0

Hypokalemia 3 (27.3) 3 (27.3) 0 0

Pain 3 (27.3) 3 (27.3) 0 0

Maculopapular Rash 3 (27.3) 3 (27.3) 0 0

Upper Respiratory Infection 3 (27.3) 2 (18.2) 1 (9.1) 0




CYCLE 1



+


CYCLE 4-12

MOR208 12 mg/kg IV on Days 1,15

+



CYCLES 2-3

MOR00208 12 mg/kg IV on Day 1, 8, 15 & 22

+


* Dose escalation up to 10 mg during C2

COHORT 3:

Richter’s Transformation




Cohort 3 Toxicities Occurring in >1 Patient Number (%)

Toxicity Grade

All 1/2 3 4


Decreased WBC 2 (40) 2 (40) 0 0

Thrombocytopenia 2 (40) 1 (20) 1 (20) 0


Hyperglycemia 5 (100) 2 (40) 3 (60) 0

Hyponatremia 3 (60) 2 (40) 1 (20) 0

Bruising 2 (40) 2 (40) 0 0

Cough 2 (40) 2 (40) 0 0

Dry Mouth 2 (40) 2 (40) 0 0

Fatigue 2 (40) 2 (40) 0 0

Hypoalbuminemia 2 (40) 2 (40) 0 0

Hypomagnesemia 2 (40) 2 (40) 0 0




CYCLE 1



+

Ibrutinib 420 mg daily

CYCLES 4-12

MOR00208 12 mg/kg IV on Days 1,15

+


CYCLES 2-3

MOR00208 12 mg/kg IV on Days 1,8,15,22

+


COHORT 4:

Ibrutinib resistance mutation in

>5% of cells while on ibrutinib

Safety Profile - Cohort 4Combination of MOR208 and ibrutinib has been well tolerated



Cohort 4 Toxicities Occurring in >1 Patient Number (%)

Toxicity Grade

All 1/2 3 4


Thrombocytopenia 2 (28.6) 2 (28.6) 0 0


Hyperglycemia 6 (85.7) 5 (71.4) 1 (14.3) 0

Hypertension 6 (85.7) 4 (57.1) 2 (28.6) 0

Back Pain 4 (57.1) 4 (57.1) 0 0

Fatigue 4 (57.1) 4 (57.1) 0 0

Pain 4 (57.1) 4 (57.1) 0 0

Increased Creatinine 3 (42.9) 3 (42.9) 0 0

Gastroesophageal Reflux Disease 3 (42.9) 3 (42.9) 0 0

Hyperuricemia 3 (42.9) 2 (28.6) 1 (14.3) 0

Musculoskeletal Disorder 3 (42.9) 3 (42.9) 0 0

Peripheral Sensory Neuropathy 3 (42.9) 3 (42.9) 0 0

Anorexia 2 (28.6) 2 (28.6) 0 0

Constipation 2 (28.6) 2 (28.6) 0 0

Diarrhea 2 (28.6) 2 (28.6) 0 0

Dyspnea 2 (28.6) 2 (28.6) 0 0

Hyponatremia 2 (28.6) 2 (28.6) 0 0

Insomnia 2 (28.6) 2 (28.6) 0 0

Mucositis 2 (28.6) 2 (28.6) 0 0

Nausea 2 (28.6) 2 (28.6) 0 0

Maculopapular Rash 2 (28.6) 2 (28.6) 0 0

Renal and Urinary Disorders 2 (28.6) 2 (28.6) 0 0



Variant allele frequency of BTK C481S in Cohort 4Preliminary evidence of activity against CLL cells with BTK C481S

Secondary objective:

To determine whether MOR00208

can eliminate cellular clones

that are resistant to ibrutinib

Conclusion



The combinations of MOR208 and lenalidomide and MOR208

and ibrutinib have been well tolerated in all patient cohorts

Preliminary efficacy has been seen in all cohorts, but

particularly interesting is activity in ibrutinib-resistant CLL

Agenda

© MorphoSys AG, Investor Reception, 2016-12-05 41





8:50 Q&A Session

A Phase I/IIa Study of the CD38 Antibody MOR202 Alone and in Combination with

Pomalidomide or Lenalidomide in Patients with Relapsed or Refractory Multiple Myeloma

Marc S. Raab,* Manik Chatterjee, Hartmut Goldschmidt,Hermine Agis, Igor W. Blau, Hermann Einsele,

Monika Engelhardt, Barbara Ferstl, Martin Gramatzki,Christoph Röllig, Katja Weisel, Tiantom Jarutat,

Dominika Weinelt, Jan Endell, Rainer Boxhammer, Christian Peschel

*Universitätsklinikum Heidelberg, Germany


MOR202 Mechanisms of Action

MOR202

• Fully human monoclonal IgG1 antibody directed against CD38

• MOR202 induces potent immune effector mechanisms: ADCC and ADCP

ADCC, antigen-dependent cell-mediated cytotoxicity; ADCP, antigen-dependent cell-mediated phagocytosis


Objectives

Primary• Assess the safety profile and establish the maximum tolerated dose (MTD) and/or

recommended dose of MOR202 in patients with RRMM: As monotherapy In combination with Dex In combination with pomalidomide (POM)/Dex In combination with lenalidomide (LEN)/Dex

• Assess immunogenicity of MOR202

Secondary• Assess preliminary efficacy, pharmacokinetics and pharmacodynamics of MOR202

monotherapy and in combination with Dex, POM/Dex and LEN/Dex in patients with RRMM

MTD, maximum tolerated dose; RRMM, relapsed or refractory multiple myeloma


MOR202 combinations: cohorts and treatment

As of November 14, 2016, 41 patients were treated in the combination cohorts with Dex only or an IMiD + Dex

• Patients were/will be treated until progressive disease or a maximum of 2 years• 1 treatment cycle is 28 days• During cycle 1, patients in all cohorts received/will receive a MOR202 loading dose on day 4• Low dose Dex was orally administered: 40 mg (≤ 75 years old) or 20 mg (> 75 years old) q1w. An additional dose

was administered in cycle 1 on day 4 concomitant with loading dose

d, day; IMiD, immunomodulatory drug; IV, intravenous; po, orally; q1w, weeklyPreliminary data 14 November 2016


ScheduleMOR202 dosePatient number

MOR202+Dex4–16 mg/kg q1w

n=18

MOR202+PomDex8,16 mg/kg q1w

n=9

MOR202+LenDex8,16 mg/kg q1w

n=14

Media age, years 67 64 66Lines of prior therapy, n (Median) 3 3 2Prior ASCT, % 78 56 79Prior therapies, %

Immunomodulatory drugsLenalidomide 94 100 43Thalidomide 39 11 14

Pomalidomide 11 11 0Proteosome Inhibitors

Bortezomib 100 100 86

Carfilzomib 6 11 0

Alkylating agents

Melphalan 100 100 93

Cyclophosphamide 94 67 79

Other agents

Doxorubicin 61 33 50

Panobinostat 0 11 7

Refractory to*, n (%)

Last prior therapy 10 (56) 9 (100) 7 (50)

Any prior therapy 11 (61) 9 (100) 9 (64)

Patient Characteristics of Clinically Relevant Cohorts

* Refractory is defined as resistance to treatment due to PD during treatment or within 2 months of last therapy; ASCT, autologous stem cell transplant


Safety: Adverse Events CTC Grade ≥3defined as ≥10% in at least one MOR202 combination treatment cohort

• Only two patients(G4 thrombocytopenia and serious G3 bacterial infection) discontinued in these cohorts due to AEs with a suspected causal relationship to MOR202

• No treatment-related deaths

AEs, n (%)MOR202+Dex

n=18

MOR202+PomDex

n=9

MOR202+LenDex

n=14

Any 15 (83) 8 (89) 12 (86)HematologicalLeukopenia 2 (11) 5 (56) 4 (29)Lymphopenia 7 (39) 2 (22) 7 (50)Neutropenia 4 (22) 6 (67) 5 (36)Thrombocytopenia 3 (17) 3 (33) 1 (7)Anemia 3 (17) 1 (11) 2 (14)CD4 lymphocyte decrease 0 1 (11) 2 (14)CRP increase 0 1 (11) 0Febrile neutropenia 0 1 (11) 0

Non-hematologicalPneumonia 1 (6) 3 (33) 1 (7)Hypokalemia 0 2 (22) 0Hypertension 2 (11) 2 (22) 1 (7)Hyperglycemia 1 (6) 0 2 (14)Diarrhea 0 1 (11) 0Hypophosphatemia 0 1 (11) 0Atrial flutter 0 1 (11) 0Skin infection 0 1 (11) 1 (7)Pneumonia pneumococcal 0 1 (11) 0

AE, adverse event; CRP, C-reactive protein;


Efficacy: Best Maximum Change in M-Protein

Data from 35 response-evaluable patients treated with clinically relevant dose regimens who received > 1 treatment cycleS, serum; U, urine


Data from response-evaluable patients treated with clinically relevant dose regimens who received > 1 treatment cycle

Efficacy: Time on Study by Best Response

CR, complete response; MR, marginal response; ORR, objective response rate; PD, progressive disease; PR, partial response; SD, stable disease; VGPR, very good partial response

MOR202/Dex


Efficacy: Time on Study by Best Response

Data from response-evaluable patients treated with clinically relevant dose regimens who received > 1 treatment cycle

MOR202 + IMiD/Dex


Efficacy: Progression Free Survival

PFS, progression-free survival


Conclusions

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

• MOR202 was well tolerated with a low incidence of IRRs, mainly limited to the first infusion

• MOR202 and Dex led to substantial disease control and long-lasting responses in this heavily pre-treated RRMM population

• MOR202 in combination with IMiDs and Dex showed encouraging signs of synergistic activity including complete remissions and emerging data on prolonged response


We deeply thank the patients, families, clinical

researchers, hospitals, and clinics that participate in

clinical trials testing the MOR202 drug candidate.

This study was sponsored by MorphoSys AG.


Q & A Session


Simon Moroney, D.Phil., MorphoSys AG

Arndt Schottelius, M.D. Ph.D., MorphoSys AG

Kerry A Rogers, M.D., The Ohio State University

Marc-Steffen Raab, M.D., Ph.D., University Hospital of Heidelberg


Appendix

PipelineSet to Deliver a Lot of Clinical Data*


PHASE 2016 2017

3

Guselkumab – Psoriasis

(VOYAGE 1)

Guselkumab - Pustular / Erythrodermic Psoriasis


(VOYAGE 2)


(NAVIGATE)

2

Guselkumab – Psoriatic Arthritis Anetumab Ravtansine – Mesothelioma (MPM) MOR103/GSK3196165 – RA

LJM716 – ESCC (+ BYL716) BI-836845 – Metastatic breast cancer MOR103/GSK3196165 – RA

MOR202 – Multiple Myeloma BI-836845 – CRPC (+enzalutamide) MOR103/GSK3196165 – Osteoarthritis

MOR208 – CLL (IIT) Bimagrumab – Hip fracture surgery MOR202 – Multiple Myeloma

MOR208 – NHL Bimagrumab – Sarcopenia (dose ranging) MOR208 – DLBCL (+ lenalidomide)

VAY736 – Pemphigus Vulgaris LFG316 – Panuveitis Tarextumab – Small cell lung canc

LFG316 – GA (+ CLG561) VAY736 – Primary Sjögren‘s Syndrome (PD)

1

Anetumab Ravtansine – Cancer Anetumab Ravtansine – Cancer

(+ pemetrexed/cisplatin)

LJM716 – Breast cancer

(+ BYL716/trastuzumab)

Gantenerumab – Alzheimer’s Anetumab Ravtansine – Ovarian cancer

(+ doxorubicin)

MOR106 – Inflammation

LJM716 – Breast/gastric cancer Anetumab Ravtansine – Hepatic/renal impairment MOR209 – Prostate cancer

BAY-1093884 – Bleeding disorders PF-05082566 – NHL/solid tumors

(+ rituximab)

BI-836845 – EGFR mutant NSCLC PF-05082566 – Solid tumors (+ MK-3475)

LFG316 – Kidney Transplantation Vantictumab – NSCLC & pancreatic cancer

* Anticipated primary completion dates, according to clinicaltrials.gov

Covering Analysts


INSTITUTION CONTACT

Baader Helvea Bruno Bulic

Berenberg Klara Fernandes

Bryan Garnier Mickael Chane-Du

Commerzbank Daniel Wendorff

Deutsche Bank Gunnar Romer

Edison Maxim Jacobs

Goldman Sachs Tim Woodward

HSBC Julie Mead

Independent Research GmbH Bernhard Weininger

J.P. Morgan Cazenove James Gordon

Kempen & Co. Anastasia Karpova

Landesbank Baden-Württemberg Timo Kürschner

Oddo Seydler Igor Kim

www.morphosys.com

Thank You

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.

Anke Linnartz

Head of Corporate Communications & IR

Phone +49 (0)89 / 899 27-404

Fax +49 (0)89 / 899 27-5404

Email [email protected]


investor event ash 2016* - morphosys · single-agent mor208 in relapsed or refractory (r-r)...

Documents