Investor Reception ASCO June 6, 2016

Transcription

1 Investor Reception ASCO 216 June 6, 216 MorphoSys - ASCO - June 6, 216 1

2 Safe Harbor 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 - ASCO - June 6, 216 2

3 Agenda 6:3 Welcome by Arndt Schottelius, M.D. PhD Chief Development Officer of MorphoSys AG MorphoSys s growing proprietary portfolio of biopharmaceuticals Introduction of speakers 6:35 MOR28 Grzegorz S. Nowakowski, M.D. Mayo Clinic, Rochester, MN (US) 6:5 MOR22 Manik Chatterjee, M.D. PhD University Hospital of Würzburg, Germany 7:5 Q&A Session Manik Chatterjee, M.D. PhD Grzegorz S. Nowakowski, M.D. Arndt Schottelius, M.D. PhD, CDO Steffen Heeger, M.D. PhD, Head of Clinical Development 7:25 Closing Remarks Food, Drinks MorphoSys - ASCO - June 6, 216 3

4 The MorphoSys Pipeline 26 Clinical Product Candidates, 14 Total Program Partner Target Disease Area Discovery Preclinic Phase 1 Phase 2 Phase 3 Bimagrumab (BYM338) Novartis ActRIIB Musculoskeletal diseases Guselkumab (CNTO1959) Janssen IL23p19 Psoriasis Gantenerumab Roche Amyloid-ß Alzheimer s disease MOR28 - CD19 ALL, CLL, NHL MOR22 - CD38 Multiple myeloma MOR13/GSK GSK GM-CSF Inflammation Anetumab Ravtansine (BAY ) Bayer Mesothelin (ADC) Solid tumors BHQ88 Novartis DKK-1 Multiple myeloma BPS84 Mereo/Novartis Sclerostin Brittle bone syndrome CNTO3157 Janssen - Inflammation CNTO6785 Janssen - Inflammation LFG316 Novartis C5 Eye diseases LJM716 Novartis HER3 Cancer Tarextumab (OMP-59R5) OncoMed Notch 2 Solid tumors VAY736 Novartis BAFF-R Inflammation MOR29/ES414 Emergent PSMA/CD3 Prostate cancer MOR16 Galapagos - Inflammation BAY Bayer TFPI Hemophilia BI BI IGF-1 Solid tumors NOV 7 Novartis - Eye diseases NOV 8 Novartis - Inflammation NOV-9 Novartis - Diabetic eye diseases NOV-1 Novartis - Cancer NOV-11 Novartis - Blood disorders Utomilumab (PF ) Pfizer 4-1BB Solid tumors Vantictumab (OMP-18R5) OncoMed Fzd 7 Solid tumors MOR17 (LP2) - AT2-R Fibrosis Immuno-oncology program Merck - Cancer Immuno-oncology program Immatics - Cancer 6 MOR programs - - Various In addition, 24 partnered programs in pre-clinic, and 45 partnered programs in discovery Most advanced development stage 9 Partnered Discovery Programs 13 MOR Programs 1 Outlicensed Program MorphoSys - ASCO - June 6, 216 4

5 The MOR Portfolio 5 Clinical Product Candidates, 14 Total Program Indication Target Discovery Preclinic Phase 1 Phase 2 Phase 3 Unpartnered MOR28 DLBCL CLL CD19 FTD, orphan status US & EU Orphan status US & EU MOR22 Multiple myeloma CD38 MOR17 Fibrosis AT2-R Immuno-oncology program Cancer 6 Programs Various Various Co-development & co-promotion MHC-associated peptides MOR29/ES414 (Emergent) MOR16 (Galapagos) Immuno-oncology program (Merck Serono) Outlicensed to GSK Prostate cancer Inflammation Cancer PSMA / CD3 Undisclosed Undisclosed MOR13/ GSK RA Osteoarthritis of the hand GM-CSF MorphoSys - ASCO - June 6, 216 5

6 Subgroup analyses of diffuse large B-cell lymphoma (DLBCL) and indolent lymphoma cohorts from a phase IIa study of single-agent MOR28 in patients with relapsed or refractory non-hodgkin s lymphoma (R-R NHL) Grzegorz S. Nowakowski, M.D. Mayo Clinic, Minnesota, USA MorphoSys - ASCO - June 6, 216 6

7 MOR28 Inroduction CD19, a B-lymphocyte lineage specific surface antigen the earliest and most broadly expressed selective B cell marker highly expressed in most NHL Recognized importance of CD19 signaling B cell survival via PI3K/AKT and c-myc attractive target antigen in B-cell malignancies, especially for an extended treatment MOR28 is an Fc-engineered humanized monoclonal CD19 antibody leading to NK cell-mediated ADCC, ADCP and direct cytotoxicity We previously reported MOR28 to be well tolerated with preliminary single-agent activity in patients with R-R CLL and R-R NHL We now present data from DLBCL and indolent NHL (inhl) cohorts and planned subgroup analyses of prognostic and predictive biomarkers in the phase II study in R-R NHL ADCC MorphoSys - ASCO - June 6, 216 7

8 MOR28 in R/R NHL Study Design and Aims of the Subgroup Analyses Study Design and Treatment Aims of the Subgroup Analyses To assess the response to MOR28 in DLBCL and inhl patient cohorts, focusing on objective response rate (ORR), disease control rate (DCR), duration of response (DoR) and tumor shrinkage To assess key secondary endpoints, progression-free survival (PFS) and safety in DLBCL and inhl cohorts Preliminary analysis of efficacy in patient subgroups defined by baseline characteristics and potentially prognostic and/or predictive biomarkers * Until disease progression MorphoSys - ASCO - June 6, 216 8

9 MOR28 in R/R NHL Results 92 patients were enrolled in the study including 35 (38%) with DLBCL and 45 (49%) with inhl Data 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. *Includes follicular lymphoma and other indolent NHLs MorphoSys - ASCO - June 6, 216 9

10 MOR28 in R/R NHL Objective Response ORRs in DLBCL and inhl cohorts were 26% and 29%, the DCRs 4% and 73%, respectively Initial partial responses could deepen over the course of the treatment Data are n (%); *Investigator assessed. Includes follicular lymphoma and other indolent NHLs Post-baseline response assessment not performed/data unavailable; n=25, 4, 11 and 76, respectively MorphoSys - ASCO - June 6, 216 1

11 MOR28 in R/R NHL Tumor Shrinkage in NHL Subtypes Change in indicator lesions for individual patients in DLBCL and inhl cohorts (central assessment) during the course of study Target lesion shrinkage was also observed in the majority of patients with stable disease (5/6 DLBCL and 12/16 inhl) Percentage change from baseline in sum of product diameters. Central read with 2 readers; results of only 1 reader are displayed and best response indicated (local assessment). Correlation between readers for relative change of target lesion at all time points demonstrated by Spearman s r=.89 *Includes follicular lymphoma and other indolent NHLs. Data cutoff August 215 MorphoSys - ASCO - June 6,

12 MOR28 in R/R NHL Disease Control Rate (DCR) DCR was considered to be a relevant efficacy endpoint as the majority of patients with stable disease had marked target lesion shrinkage but as per study design were not treated beyond cycle 3 Subgroup analyses of DCR in DLBCL and inhl patients showed significant differences according to: prior lines of therapy NK cell count and CD16 expression international prognostic index score and DoR to prior treatment but not to FcyR high and low affinity variants Observed DCRs were equally encouraging for patients with rituximab-refractory and non-refractory disease. In DLBCL, 5 out of 9 responders were rituximab-refractory, as were 5 out of 13 inhl responders DCR did not differ across FcyR polymorphism subgroups Time to response and DoR : Median DoR in DLBCL was 2 months (95% CI 11.3 NA) with 3 ongoing responses Median DoR was not reached in inhl patients (95% CI 8. NA), with 72% of responders without disease progression at 16 months and 6 responses ongoing (Kaplan-Meier estimates) MorphoSys - ASCO - June 6,

13 Disease Control Rate in Patients with DLBCL or inhl 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 Clopper-Pearson confidence intervals Includes follicular lymphoma and other indolent NHLs. *p<.5, **p<.1. MorphoSys - ASCO - June 6,

14 MOR28 in R/R NHL Long Duration of Responses in DLBCL and FL/iNHL Long-lasting responses, up to >26 months PFS rate: >4% at 12 months * Includes follicular lymphoma and other indolent NHLs. One patient with stable disease had a late response (PR) after 17 months in follow-up. This patient is not shown in the figure. CR, complete response; DLBCL, diffuse large B-cell lymphoma; NHL, non-hodgkin s lymphoma; PR, partial response MorphoSys AG, Company Update - June 216 Jurczak et al., Abstract #7545, ASCO

15 MOR28 in R/R NHL Progression-free survival in NHL subgroups The 12-months PFS rate as 4% in both DLBCL and inhl. The 2-years PFS rate was 22% in DLBCL and 35% in inhl. In the subgroup of evaluable patients with LBCL and inhl: PFS was significantly longer in patients with a baseline NK cell count >1 cells/μl vs 1 cells/μl PFS was similar in patients with rituximab non-refractory and refractory tumors Patients without post-baseline radiological tumor assessment were censored at baseline Jurczak et al., Abstract #7545, ASCO 216 MorphoSys AG, Company Update - June

16 MOR28 in R/R NHL Safety The incidence of grade 3 hematologic treatment-emergent adverse events was low: DLBCL: 9/35 (26%) inhl: 4/45 (9%) including neutropenia, anemia and thrombocytopenia in 14%, 9% and 6% of DLBCL and 4%, % and % of inhl patients Infusion-related reactions were seen in 3/35 (9%) and 4/45 (9%) patients with DLBCL and inhl respectively; mostly of grade 1 2 (with one grade 4 dyspnea) There were no treatment-related deaths and no trend towards late toxicity MorphoSys - ASCO - June 6,

17 MOR28 in R-R NHL Conclusions MOR28 12 mg/kg showed encouraging preliminary single-agent activity in patients with R-R DLBCL or R-R inhl Objective response rates were 26% in the DLBCL cohort and 29% in inhl cohort Target lesion shrinkage demonstrated a clinical benefit in the majority of DLBCL and inhl patients with stable disease Long-lasting responses were seen in DLBCL and inhl, up to >26 months, and a PFS rate of 4% at 12 months Subgroup analyses of DCR and PFS are hypothesis generating for prognostic or predictive biomarkers and suggest: MOR28 was equally efficacious in NHL patients with rituximab refractory and non-refractory disease Patients with a high peripheral NK cell count at baseline benefited more from MOR28 treatment. Its utility as a predictive biomarker will be further investigated. MOR28 was well tolerated with a favorable safety profile including patients on long-term maintenance. MorphoSys - ASCO - June 6,

18 MOR22 alone and in combination with pomalidomide or lenalidomide in relapsed or refractory multiple myeloma: Data from clinically relevant cohorts from a phase I/IIa study Manik Chatterjee, M.D. PhD University Hospital of Würzburg, Germany MorphoSys - ASCO - June 6,

19 MOR22 Introduction CD38 is a 45 kda type II transmembrane glycoprotein widely expressed in many hematological malignancies including multiple myeloma MOR22 is a HuCAL-derived human immunoglobulin G1 CD38 antibody that has demonstrated high in vitro and in vivo efficacy in preclinical models of MM The main mode of action for MOR22-induced lysis of MM cells is ADCC and ADCP MOR22 does not induce CDC; CDC is suspected to be a major contributor to infusion-related reactions (IRRs) We now present further data from this study of patient cohorts treated with MOR22 in combination with dexamethasone (Dex) or with an immunomodulatory drug (IMiD) + Dex MorphoSys AG, Company Update - June

20 MOR22: Objectives of the Phase 1/2a Study in R-R Multiple Myeloma Primary Assess the safety profile and establish the maximum tolerated dose (MTD) and/or recommended dose of MOR22 in patients with R-R MM As monotherapy In combination with Dex In combination with pomalidomide (POM)/Dex In combination with lenalidomide (LEN)/Dex Assess immunogenicity of MOR22 Secondary Assess preliminary efficacy, pharmacokinetics and pharmacodynamics of MOR22 monotherapy and in combination with Dex, POM/Dex and LEN/Dex in patients with R-R MM MorphoSys - ASCO - June 6, 216 2

21 MOR22: Phase I/IIa, Open-label, Multicenter, Dose-escalation Study in R-R MM Standard 3+3 dose escalation study starting with MOR22 monotherapy of.1 mg/kg every two weeks (q2w) up to 16 mg/kg q2w and 8 mg/kg every week (q1w), followed by the combination cohorts with Dex only, and in combination with POM/Dex or LEN/Dex On completion of the dose escalation phase, confirmatory cohorts are planned to validate the MTD and/or recommended dose/schedule of MOR22 + Dex, and in combination with POM/Dex or LEN/Dex Patients had to have received at least two prior lines of therapy for the MOR22 + Dex and + POM/Dex cohorts or at least one prior line for the MOR22 + LEN/Dex cohort Dose escalation of combination cohorts (3+3 design) MOR22 + Dex 2-hour IV infusion of MOR22* ( mg/kg) q1w with Dex MOR22 + POM/Dex 2-hour IV infusion of MOR22* (8 16) mg/kg, q1w with POM (4mg po, d1-21)/dex, MOR22 + LEN/Dex: 2-hour IV infusion of MOR22* (8 16 mg/kg) q1w with LEN (25 mg po, d1-21)/dex, Confirmatory cohorts ( 6 patients each) each cohort to be expanded with MTD or recommended dose * During cycle 1, patients in all cohorts received/will receive a MOR22 loading dose on day 4. Low dose Dex was orally administered: 4 mg ( 75 years old) or 2 mg (> 75 years old) q1w. An additional dose was administered in cycle 1 on day 4 concomitant with loading dose. MorphoSys - ASCO - June 6,

22 MOR22: Phase I/IIa Study in R-R MM Baseline characteristics MOR22 schedule Dosing, mg/kg Patient number q1w + Dex q1w + POM/Dex 8 n=5 q1w + LEN/Dex 8, 16 n= n=16 Characteristic Median age, years Gender, % Male Female Karnofsky PS, % Median Lines of prior therapy, n Median Prior ASCT, % Prior therapies, % Bortezomib Lenalidomide Cyclophosphamide Melphalan Doxorubicin Thalidomide Pomalidomide Carfilzomib Panobinostat Refractory to*, n (%) Last prior therapy Any prior therapy (75) 13 (84) (8) 5 (1) (43) 4 (57) The study is ongoing; this is a report of interim safety, and preliminary efficacy data in the MOR22 treatment combination cohorts As of April 6, 216, a total of 63 patients had been treated with MOR22 monotherapy including 28 patients treated in combination with Dex only or an IMiD + Dex *Refractory is defined as resistance to treatment due to lack of response or progression of disease during treatment within 6 months of last therapy MorphoSys, MOR22C11 ASCO 216 Poster Presentation

23 MOR22: Preliminary Phase 1/2a Data Time on Study and Best Response* 7 out of 9 responses are ongoing Median time to response was 4 weeks; most responses deepened over time Most responses are ongoing, with the longest duration of response currently 44 weeks (ongoing) To date 4 responses have been seen in the MOR22 + Dex cohorts and 5 responses in cohorts of MOR22 with an IMiD/Dex In the cohorts of MOR22 with an POM/Dex, 2/5 responders achieved a CR MorphoSys AG, Company Update - June

24 % Change in M-Protein MOR22: Preliminary Phase 1/2a Data Best Maximum Change in M-protein* *Updated data from 2 April 216 CR, complete response; 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. MorphoSys AG, Company Update - June

25 MOR22 Very Low Rate of Infusion-Related Reactions Infusion Tolerability and Immunogenicity A 2-hour IV infusion was feasible in all patients IRRs occurred in 4 (14%) patients and were mainly limited to the first infusion Only 1 out of 3 patients (from all cohorts) tested so far developed a transient anti-mor22 antibody response Infusion-related Reactions to MOR22 Dex, dexamethasone; G, grade; IRR, infusion-related reaction; LEN, lenalidomide; n, number of patients; POM, pomalidomide; q1w, weekly. MorphoSys AG, Company Update - June

26 CD38 molecules/cell (ABC) Current Data Suggest CD38 Preservation During MOR22 Therapy 1 Biomarkers 1 Evaluation of CD38 molecules per cell (ABC) on MM patient bone marrow plasma cells at baseline and during MOR22 therapy Current data suggest CD38 expression is preserved during MOR22 therapy Data derived from patients from different dose cohorts and combination partners with most of them showing a response to treatment 1 Baseline Cycle 2 Day 1 (4 weeks) ABC, antibody bound per cell; Dex, dexamethasone; LEN, lenalidomide; POM, pomalidomide; q1w, weekly. MorphoSys AG, Company Update - June

27 MOR22: Preliminary Phase 1/2a Data Most frequently reported AEs* MOR22 schedule Patient number q1w + Dex n=16 q1w + POM/Dex n=5 q1w + LEN/Dex n=7 AEs, n (%) Grade 3 Any 3 Any 3 Any Any 14 (88) 16 (1) 5 (1) 5 (1) 7 (1) 7 (1) Hematological Leukopenia Neutropenia Lymphopenia Thrombocytopenia Anemia 2 (13) 4 (25) 6 (38) 3 (19) 2 (13) 8 (5) 7 (44) 7 (44) 6 (38) 4 (25) 2 (4) 4 (8) 2 (4) 1 (2) 1 (2) 4 (8) 4 (8) 2 (4) 3 (6) 2 (4) 2 (29) 2 (29) 2 (29) 1 (14) 1 (14) 5 (71) 2 (29) 3 (43) 2 (29) 1 (14) Non-hematological Fatigue Back pain Muscle spasms Diarrhea Nasopharyngitis Cough Rash Constipation 5 (31) 4 (25) 2 (13) 1 (6) 4 (25) 2 (13) 1 (6) 1 (2) 2 (4) 3 (6) 2 (4) 2 (4) 1 (2) 2 (4) 3 (6) 2 (4) 3 (43) 3 (43) 1 (14) 2 (29) 2 (29) 3 (43) Only one patient in the combination cohorts discontinued due to AEs with suspected causal relationship to MOR22. This AE (upper respiratory tract infection) had also a suspected causal relationship to LEN and Dex. There have been no treatment-related deaths. * Most frequently reported AEs defined as 15% in all MOR22 combination treatment cohorts (n=28). Composites of preferred terms included in Medical Dictionary for Regulatory Activities System Organ Classes: Blood and lymphatic system disorders and Investigations. MorphoSys, MOR22C11 ASCO 216 Poster Presentation

28 MOR22 in R-R MM Conclusions The MTD of MOR22 alone and in combination has not yet been reached MOR22 in doses up to 16 mg/kg can be safely administered as a 2-hour IV infusion MOR22 was well tolerated with a low incidence of IRRs, mainly limited to the first infusion Current data suggest CD38 expression on MM patient bone marrow plasma cells is preserved during MOR22 therapy In this heavily pre-treated RRMM population, MOR22 alone or in combination with IMiDs lead to encouraging and long lasting responses A high percentage of complete responses (2/5, 4%) has been observed so far in patients that receiving MOR22 in combination with POM/Dex Further cohorts will evaluate MOR22 16 mg/kg q1w in combination with POM/Dex and LEN/Dex MorphoSys, MOR22C11 ASCO 216 Poster Presentation

29 Questions & Answers Arndt Schottelius, M.D., Ph.D. Steffen Heeger, M.D., Ph.D. Manik Chatterjee, M.D. PhD University Hospital of Würzburg, Germany Grzegorz S. Nowakowski, M.D. Mayo Clinic, Minnesota, USA MorphoSys - ASCO - June 6,

30 Update from ASCO 216 MOR28 MOR28 shows disease control rate of 4% in R/R DLBCL and 73% in inhl patients Duration of response (CR or PR) of up to 26 months (responses ongoing) MOR28 leads to reduction in target lesion size also in patients with stable disease MOR28 shows similar progression-free survival (PFS) in rituximab-refractory and non-refractory patients MOR22 MOR22 (8 mg/kg) plus Pom/Dex in R/R multiple myeloma patients show two complete responses (CR) and two minor responses (MR) out of five patients MOR22 (8 mg/kg) plus Len/Dex in RRMM show one very good partial response (VGPR) and two partial responses (PR) out of four patients in this combination cohort with a scheduled response assessment after one treatment cycle First biomarker data suggest that CD38 expression on MM patient bone marrow plasma cells was preserved during MOR22 therapy MOR22 administered in doses of up to 16 mg/kg as a 2-hour intravenous infusion with low incidence of infusion-related reactions MorphoSys AG, Company Update - June 216 3

31 Thank You Dr. Claudia Gutjahr-Löser Head of Corporate Communications & IR Phone +49 ()89 / Fax +49 ()89 / investors@morphosys.com HuCAL, HuCAL GOLD, HuCAL PLATINUM, CysDisplay, RapMAT, aryla, Ylanthia and 1 billion high potentials are registered trademarks of MorphoSys AG. Slonomics is a registered trademark of Sloning BioTechnology GmbH, a subsidiary of MorphoSys AG.