Momenta Pharmaceuticals, Inc. 34 th Annual J.P. Morgan Healthcare Conference January 13, 2016

Transcription

1 Momenta Pharmaceuticals, Inc. 34 th Annual J.P. Morgan Healthcare Conference January 13, 2016

2 Forward Looking Statements Statements in this presentation regarding management's expectations, beliefs, intentions, goals, strategies, plans or prospects, are forward-looking statements, which include, but are not limited to statements about: our biosimilar and complex generic collaborations, the timing of milestone and other payments or product revenue under our biosimilar and complex generic collaborations, the timing of clinical trials and trial results for our biosimilar and novel drug product candidates; the market opportunity for Glatopa TM, our M356 (40 mg) product candidate and our other product candidates; patent litigation relating to Glatopa, M356 (40 mg) and biosimilar product candidates; the potential indications for our biosimilar and novel product candidates; and our biosimilar strategy and our ability to successfully implement that strategy. Forward-looking statements may contain such words as "anticipate," "believe," "could," "hope," "target," "project," "goals," "potential," "predict," "might," "estimate," "expect," "intend," "is planned," "may," "should," "will," "look forward, "would" or similar terms. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual results to differ materially from those expressed or implied in such statements. These risks, include, but are not limited to: receipt of approval under the Hart-Scott-Rodino Antitrust Improvements Act for our Mylan collaboration agreement, delays in launch of our M356 (40 mg) product, disputes or disagreements with Mylan, Baxalta or Sandoz under our respective collaborations; unexpected expenses or inaccurate financial assumptions; adverse decisions or delays by courts or regulatory agencies; unfavorable regulatory guidance developments; safety, efficacy or tolerability problems with our product candidates; inability to partner the development and commercialization of our product candidates; increased competition for targeted indications and markets; and those risks described in the section Risk Factors in the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2015 filed with the Securities and Exchange Commission, as well as other documents that may be filed by Momenta from time to time with the SEC. Information provided in this presentation speaks only as of the date of this presentation and we assume no obligation to update forward-looking statements, whether as a result of new information, future events or otherwise. 2

3 Momenta Pharmaceuticals Overview Corporate Info Founded in 2001; IPO 2004 Located in Cambridge, MA >250 employees An Advanced Analytic Platform Expertise in high-resolution analytics, biological characterization and process engineering Driving Potential in Three Areas Complex Generics Biosimilars Novel Drugs 3

4 Building a Sustainable Biopharmaceutical Company Based on Proven Technology Biosimilars Novel Drugs Necuparanib Technology for the characterization of complex biologic mixtures Complex Generics Generic LOVENOX (Enoxaparin Sodium Injection) M923 (HUMIRA ) M834 (ORENCIA ) Portfolio of development candidates Novel Autoimmune Drugs M281 (Anti-FcRn) M230 (SIF3) hs-ivig Biological Characterization Control of Manufacturing Physicochemical Analytics 4

5 Biosimilars and Potentially Interchangeable Biologics Novel Drugs Technology for the characterization of complex biologic mixtures Complex Generics Generic LOVENOX (Enoxaparin Sodium Injection) Biosimilars M923 (HUMIRA ) M834 (ORENCIA ) Portfolio of development candidates Necuparanib Novel Autoimmune Drugs M281 (Anti-FcRn) M230 (SIF3) hs-ivig Biological Characterization Control of Manufacturing Physicochemical Analytics 5

6 Momenta s Approach to Biosimilars 1 Build a broad and diverse portfolio Portfolio of complex biologics including mabs and fusion proteins Capture scale, technology and regulatory synergies Smooth revenue flow 6

7 Building a Broad and Diverse Biosimilars Pipeline Targeting brand products with total 2014 WW sales of >25B Biosimilars Process Development Preclinical Clinical BLA M923 Adalimumab (HUMIRA ) (Autoimmune/Inflammation) Pivotal Patient Study Initiated M834 Abatacept (ORENCIA ) (Autoimmune/Inflammation) M615 M706 M710 M730 M740 Momenta & Baxalta Momenta & Mylan 7

8 Momenta s Approach to Biosimilars 1 Build a broad and 2 diverse portfolio Portfolio of complex biologics including mabs and fusion proteins Gain competitive advantage through our scientific approach and regulatory strategy Capture scale, technology and regulatory synergies Smooth revenue flow Biocharacterization platform, analytic toolset and process control designed to capture added value through: Extrapolation of indications and reduced trial scope Interchangeability designation Launch among first biosimilar for each product 8

9 Momenta s Approach to Biosimilars 1 Build a broad and 2 Gain competitive 3 diverse portfolio advantage through our scientific approach and regulatory strategy Portfolio of complex biologics including mabs and fusion proteins Capture scale, technology and regulatory synergies Smooth revenue flow Biocharacterization platform, analytic toolset and process control designed to capture added value through: Extrapolation of indications and reduced trial scope Interchangeability designation Launch among first biosimilar for each product Ensure products are positioned to capture the global opportunity through collaborations Identify and collaborate with strategic partners with: Global commercial capabilities Low cost manufacturing strategies 9

10 Collaboration Leverages Mylan s Financial Strength, Generics & Biosimilars Experience and Extensive Global Reach Mylan Total Revenue Global and Scalable Supply Chain $ in billions $5.5 $6.1 $6.8 $6.9 $

11 Mylan Biosimilars Collaboration Financial Terms $45M up front payment to Momenta 50/50 cost and profit share Mylan will fund its collaboration expenses, in part, through up to $200M in six contingent milestone payments Contingent Milestone Payments Up to $200M to Momenta & Shared Expenses Momenta Applies to Funding of Costs 50/50 Cost & Profit Share 11

12 Mylan Biosimilars Collaboration Roles & Responsibilities Momenta & Mylan will be collaborating across all activities through a Joint Steering Committee Momenta Momenta (M834 & Phase 1) Momenta (US) Momenta (Option to Co-Commercialize in US) Product Development Clinical Development Regulatory Activities Commercialization Mylan (Pivotal Trials for all except M834) Mylan (Ex US) Mylan 12

13 M834, Biosimilar ORENCIA (abatacept) candidate expected to enter the clinic in 2016 $ (M) Complex fusion protein with limited biosimilar competition 3,000 2,500 2,000 1,500 1, Source: EvaluatePharma Ltd. WW Orencia Sales Initiate M834 clinical trials ex-us US Targeting launch at market formation Unique product profile Only CTLA-4Ig approved for use in rheumatoid arthritis (RA) and juvenile idiopathic arthritis Considered to have better safety profile than anti-tnfs Only RA biologic without black box warning label Potential for wider use as 1 st - line biologic 13

14 M923, Biosimilar HUMIRA (adalimumab) Candidate in Pivotal Clinical Trial $ (M) Monoclonal antibody Largest selling therapeutic in the world Approved in 13 indications globally, 9 in the US Six additional indications in development for market growth 20,000 15,000 10,000 5,000 0 ex-us WW Humira Sales US Source: EvaluatePharma Ltd. Patent challenges initiated in 2015 consistent with our development & regulatory timelines Targeting First Regulatory Submission Targeting First Commercial Launch 14

15 M923 Meets Primary Endpoint in Human Pharmacokinetic (PK) Study 324 healthy volunteers randomized 1:1:1 to receive single 40 mg injection of M923, US HUMIRA, or EU HUMIRA, then followed for 71 days PK bioequivalence demonstrated for all primary endpoints (C max, AUC 0-inf and AUC ) across each pairwise comparison (M923/US HUMIRA, M923/EU HUMIRA and EU HUMIRA/US HUMIRA) Geometric LS mean ratios all close to unity (100%) 90% CIs well within the confidence bounds of % (range %) Incidence of AEs and ADAs for M923 was similar to reference products 15

16 Complex Generics Novel Drugs Biosimilars Necuparanib Technology for the sequencing of complex biologic mixtures Complex Generics Generic LOVENOX (Enoxaparin Sodium Injection) M923 (HUMIRA ) M834 (ORENCIA ) Portfolio of development candidates Novel Autoimmune Drugs M281 (Anti-FcRn) M230 (SIF3) hs-ivig Biological Characterization Control of Manufacturing Physicochemical Analytics 16

17 Glatopa TM is the First FDA-approved, Substitutable Generic Daily COPAXONE 20 mg for MS Glatopa scripts represent approximately 30% of the 20 mg glatiramer acetate market in the U.S. 1 YTD profit share of $37M as of Q In Q % profit share of $9M earned In Q % profit share of $28M, less a $9M deduction for pre-launch legal expenses earned Sandoz market strategy is to optimize Glatopa value versus driving share Strong collaboration with key market partners Addressing COPAXONE 20 mg Dispense As Written (DAW) and franchise rebating 1 Source: Symphony Health Solutions prescription data plus internal estimates to include key Glatopa accounts not reported in Symphony. 17

18 Sandoz Focus on Growing Glatopa TM in 2016 Potential for: Step edits Further formulary adoption Increase payer focus on long-term benefits and savings for chronic use generic ~10,000 new RRMS patients diagnosed annually Potential for: Further leveraging Novartis MS structure New marketing channels to educate physicians, patients and payers Scientific publications Counter Dispense as Written Campaign 18

19 M356, Generic COPAXONE 40 mg Offers Potential Revenue in Q Worldwide COPAXONE ( mg) sales are estimated to be $2.5B in 2017* Regulatory Legal Competition M356 under FDA Review Target action date received 30 month stay expires in Feb 2017 Markman hearing in February 2016 MYL IPR PTAB decision in August 2016 District Court Trial in September mg formulation contains the same API as 20 mg formulation MNTA/Sandoz only approved 20 mg generic to date 19 *Source: Evaluatepharma

20 Novel Drugs: Necuparanib Novel Drugs Biosimilars Necuparanib Technology for the sequencing of complex biologic mixtures Complex Generics Generic LOVENOX (Enoxaparin Sodium Injection) M923 (HUMIRA ) M834 (ORENCIA ) Portfolio of development candidates Novel Autoimmune Drugs M281 (Anti-FcRn) M230 (SIF3) hs-ivig Biological Characterization Control of Manufacturing Physicochemical Analytics 20

21 Significant Opportunity for Necuparanib as a Heparinbased, Multi-targeted Therapeutic for Cancers History of Heparins in Cancer Use of heparins to treat venous thrombosis in cancer patients has generated numerous reports of antitumor activity; however, the dose of these products has been limited by anticoagulant activity. 21 * Adapted from Hanahan and Weinberg Cell, 2011

22 Necuparanib Phase 2 Study Progressing Randomized, placebo (PBO) controlled study of NECU + ABX + GEM compared with PBO + ABX + GEM Enrolling approximately 150 patients Primary endpoint to evaluate overall survival in patients treated with NECU + ABX + GEM compared with PBO + ABX + GEM Orphan Drug and Fast Track designations granted by FDA Study protocol amended in December 2015 to further reinforce guidelines for diagnosing and treating thrombocytopenia Goal to report top line data in 2H

23 Novel Drugs: Autoimmune Drug Candidates Novel Drugs Biosimilars Necuparanib Technology for the sequencing of complex biologic mixtures Complex Generics Generic LOVENOX (Enoxaparin Sodium Injection) M923 (HUMIRA ) M834 (ORENCIA ) Portfolio of development candidates Novel Autoimmune Drugs M281 (Anti-FcRn) M230 (SIF3) hs-ivig Biological Characterization Control of Manufacturing Physicochemical Analytics 23

24 Novel Autoimmune Drug Candidates Target High Unmet Medical Needs Multiple Orphan Disease Indications Hypersialylated IVIg Anti-FcRn mab Selective immunomodulator FcRs Fab binding alters target function Fc-mediated CDC, ADCC IC deposition localizes inflammation and fibrosis IC-mediated autoantibody production amplification Systemic lupus erythematosus ANCA-associated vasculitis Anti-GBM nephropathy Antiphospholipid antibody syndrome Autoimmune hemolytic anemia CIDP Dermatomyositis/polymyositis Guillain-Barre syndrome Idiopathic thrombocytopenia purpura Myasthenia gravis Neuromyelitis optica Pemphigus Sjogren s syndrome Vasculitis.. and potentially subsets of psoriasis, RA, psoriatic arthritis, etc. CDC=complement-dependent cytotoxicity ADCC=Antibody-dependent cellular cytotoxicity IC= Immune complex 24

25 M281 is a Fully Human Monoclonal Antibody (mab) Targeting the Neonatal Fc Receptor (FcRn) Mechanism of IgG Recycling and anti-fcrn Antibody Blockade M281 inhibits human IgG binding to FcRn resulting in rapid clearance of IgG Potential to use acutely or intermittently for autoantibody clearance across a broad range of IgG-driven autoimmune diseases Adapted from NEJM 1999;340:

26 M281 Rapidly Reduces Circulating IgG Antibodies in Non-human Preclinical Studies Selective Induction of IgG Clearance in Cynomolgus Monkey M281 exhibits high affinity to human and non-human FcRn in preclinical studies M281 shows selective induction of human and non-human IgG clearance IND enabling toxicology studies completed Targeting clinical trial in mid

27 Momenta s Technology Platform has Generated a Robust Pipeline of Products & Product Candidates Biosimilars Novel Drugs Programs Preclinical/Process Development Clinical 1 ANDA/BLA/NDA Filed Marketed Complex Generics 1 Enoxaparin Sodium Injection (Generic LOVENOX ) Glatopa TM (Generic 20 mg/ml COPAXONE ) M356 (Generic 40 mg/ml COPAXONE ) ANDA Accepted Launched June 2015 M923 Adalimumab (HUMIRA ) Pivotal Patient Study Initiated M834 Abatacept (ORENCIA ) 5 Biosimilar Programs Necuparanib Pancreatic Cancer Phase 2 M281 (Anti-FcRn Antibody) M230 (Selective Immuno-modulator of Fc Receptors) hsivig (Hyper-sialylated IVIg) Momenta & Sandoz Momenta & Baxalta Momenta & Mylan Momenta 1 Clinical safety/efficacy trials have not been required for these complex generic drug applications 27

28 Momenta Near and Long-Term Goals BIOSIMILARS Progress Mylan biosimilars collaboration M834, biosimilar ORENCIA candidate, to enter the clinic M923, biosimilar HUMIRA candidate, on the market Launch 1-2 biosimilar product candidates per year COMPLEX GENERICS Grow Glatopa TM FDA approval of ANDA for M356, generic COPAXONE 40 mg M356, generic COPAXONE 40 mg, on the market NOVEL DRUGS Complete necuparanib Phase 2 enrollment M281, anti-fcrn antibody, to enter the clinic Launch necuparanib Launch 1-2 novel autoimmune drugs 28

29 Momenta Pharmaceuticals, Inc.