Momenta Pharmaceuticals — Investor Presentation (January 2019)
26 slides from Momenta Pharmaceuticals's investor presentation, Jan 7, 2019
Slide 1: Forward-Looking Statements
Forward-Looking Statements 2 This presentation contains forward-looking statements about our financial outlook, business plans and objectives and other future events and developments. These forward-looking statements include, but are not limited to statements about our pipeline of novel drug candida
Slide 2: 2018: Transforming the Business vs. 2019: Advancing the Business
Completed Strategic Review Restructured organization to focus on novel drug development programs targeting rare immune-mediated disorders and late-stage biosimilars Streamlined Operations Completed a workforce reduction of 50% Changed infrastructure to support focus on novel candidates Exited five b
Slide 3: Promising Pipeline of Novel Drug Candidates
Promising Pipeline of Novel Drug Candidates 4 Phase 1 Phase 2 Phase 3 M281 (Anti-FcRn Antibody) M230 (Recombinant Fc multimer) M254 (Hyper-sialylated IgG) Sialylation Technology Fc Multimer Candidates* *Includes research collaboration with CSL Pre-Clinical
Slide 4: M281 (anti-FcRn): Purposefully Engineered for Best-in-Class
Once monthly or twice monthly dosing Potential for best-in-class dosing and adverse event profile Subcutaneous formulation in development Potential for self-administered dosing M281 (anti-FcRn): Purposefully Engineered for Best-in-Class 5 Highest IgG reduction Potential for efficacy improvement Targ
Slide 5: M281 has Broad Commercial Potential in Multiple Areas
M281 has Broad Commercial Potential in Multiple Areas 6 Autoimmune Diseases Maternal Fetal Disorders Product Immunogenicity M281 ~45 rare autoimmune disorders driven by autoantibodies 1-2 million US patients, high reliance on steroids, significant morbidity, few approvals Diseases caused by antibody
Slide 6: Phase 1 Multiple Dose Trial Supports Best-in-Class Potential
Phase 1 Multiple Dose Trial Supports Best-in-Class Potential 7 *M281 receptor occupancy as measured by percent unoccupied FcRn compared with baseline in monocytes. M281-treated cohort (n = 3). Placebo controls from each multiple-dose cohort were combined (n = 4). M281 exhibited dose-dependent IgG re
Slide 7: Evidence Points to Better Efficacy with Higher IgG Reduction in Myasthenia Gravis
8 Higher IgG reduction may result in better efficacy Efficacy includes quantitative MG (QMG) and MG composite (MGC) data (QMG for all but the lower plasmapheresis point). The arrow indicates a direct, linear relationship. Data from: Liu 2010; Guptil 2016. Evidence Points to Better Efficacy with High
Slide 8: M281 Phase 1 Study: Albumin Findings Related To Potency
M281 Phase 1 Study: Albumin Findings Related To Potency 9 30 mg/kg Single Dose 30 mg/kg Weekly Dose (4 weeks) Greatest reductions in albumin were observed in high dose cohorts 60 mg/kg single ascending dose group 15 mg/kg and 30 mg/kg multiple weekly dose cohorts Albumin reductions were asymptomatic
Slide 9: M281 Currently in Two Clinical Trials
M281 Currently in Two Clinical Trials Myasthenia Gravis (MG) Neuromuscular Autoimmune Disease Autoantibodies against acetylcholine receptors (AChR) or the muscle-specific receptor tyrosine kinase (MuSK) Effectively blocks the excitatory action of acetylcholine Bimodal age distribution: younger women
Slide 10: Generalized Myasthenia Gravis (gMG) Phase 2 Study Key Goals
Generalized Myasthenia Gravis (gMG) Phase 2 Study 11 Key Goals Primary Trial Objectives To evaluate the safety and tolerability of treatment with M281 To evaluate the efficacy as measured by the change in MG – Activities of Daily Living score Explore high doses and potential highest efficacy in clas
Slide 11: FcRn Inhibition May Impact Many Autoimmune Disorders
FcRn Inhibition May Impact Many Autoimmune Disorders 12
Slide 12: Hemolytic Disease of the Fetus and Newborn (HDFN) Phase 2 Clinical Study
Hemolytic Disease of the Fetus and Newborn (HDFN) Phase 2 Clinical Study 13 Proof of Concept in this indication should provide strong evidence for efficacy in additional Fetal Maternal indications Key Goals Primary Trial Objectives First-in-class in HDFN Broadening the label to alloimmune rare disor
Slide 13: M281 Near-term Activities
M281 Near-term Activities 14 Enroll MG Phase 2 clinical study for 2020 read-out with potential as best-in-class agent Progress HDFN study, building awareness of maternal fetal disorders Third indication (second in autoimmune disease) to initiate in 2019 Explore research collaboration opportunities i
Slide 14: M254 (Hyper-sialylated IgG): Designed to Increase Anti-Inflammatory Effect of IgG
M254 (Hyper-sialylated IgG): Designed to Increase Anti-Inflammatory Effect of IgG 15 Glycan Sialic acid Asn297 Heavy chain Light chain IVIg hsIgG Improved Treatment Paradigm Shorter infusion times and potential for subcutaneous administration Potency Potential for higher doses could enable greater i
Slide 15: IgG Revenue Growth Estimates
IgG Revenue Growth Estimates IgG used to Treat Immune-Mediated Indications is a High Value, Supply Constrained Opportunity IgG revenue >$8 billion in 2018 IgG market growing at 6 – 8% per year ~50% of IgG used in autoimmune indications IgG FDA approved for six indications and payers reimburse for us
Slide 16: Immune Thrombocytopenic Purpura (ITP): Autoimmune Disease
Immune Thrombocytopenic Purpura (ITP): Autoimmune Disease that Leads to Excessive Bruising and Bleeding 17 Abnormal autoantibodies bind to circulating platelet membranes leading to platelet destruction and subsequent reduction of the number of circulating platelets (thrombocytopenia) ITP affects chi
Slide 17: M254 Development Strategy: Fast to Proof-of-Concept
M254 Development Strategy: Fast to Proof-of-Concept 18 Strong Dataset at POC Clinical Differentiation Estimated 4-10 times more potent than IVIg Potential for improved tolerability Potential for dramatically improved treatment paradigm Potential for lower infusion costs Goal: Develop M254 as a super
Slide 18: M254: Phase 1/2 Study Design in ITP
M254: Phase 1/2 Study Design in ITP CTA Approval Received 19 The Phase 1/2 study is intended to provide safety and tolerability data along with proof-of-concept for potency in ITP patients
Slide 19: M230 (CSL730) - Recombinant Fc Multimer in Collaboration with CSL
M230 (CSL730) - Recombinant Fc Multimer in Collaboration with CSL 20 Potential first-in-class recombinant Fc multimer designed with enhanced avidity for Fc receptors M230 shows higher potency and efficacy at a lower dose than IVIg in multiple preclinical models Phase 1 trial in healthy volunteers is
Slide 20: New Drug Candidates Derived from Our Technology Platforms
Antibody Design & Engineering New Drug Candidates Derived from Our Technology Platforms 21 Disease Analytics Platform Fc Multimerization M230 SIFbodies M281 Sialylation Platform M254 Recombinant replacement therapies Development Discovery Biologics Process Development Expertise
Slide 21: Hyper-Sialylation and SIFbody Research Platforms
Hyper-Sialylation and SIFbody Research Platforms 22 Hyper-sialylation of N- and O-linked glycans on proteins Prevents clearance in liver via asialoglycoprotein receptor (ASGPR) Potential to optimize half-life of recombinant rare disease drugs and other therapeutic candidates 0 Time Serum-derived Rec
Slide 22: Complex Generics and Biosimilars: Fuel for Our Pipeline
Complex Generics and Biosimilars: Fuel for Our Pipeline 23 Marketed Phase 1 Phase 2 Phase 3 Enoxaparin Sodium Injection (Generic LOVENOX®) Glatopa® 20 mg (Generic COPAXONE® 20 mg) Glatopa® 40 mg (Generic COPAXONE® 40 mg) M710 Proposed Biosimilar to Aflibercept (EYLEA®) M923 Proposed Biosimilar to Ad
Slide 23: Biosimilars and Complex Generics
Biosimilars and Complex Generics 24 Enrollment on-going for pivotal Phase 3 trial initiated in patients with DME in 2018 Expect U.S. market formation in 2023, subject to positive Phase 3 results, marketing approval and patent considerations In collaboration with Mylan Abbvie settlement enables poten
Slide 24: 2019: A Year Focused on Operational Execution
2019: A Year Focused on Operational Execution Novel Drugs Biosimilars M281 (anti-FcRn) M254 (hsIgG) M230 (rFc multimer) Research Initiation of a third clinical study in 2019 Advance Phase 1/2 proof of concept study in ITP Study completion in 2019 (by partner CSL) Advance CSL research collaboration M
Slide 25: Momenta Pharmaceuticals, Inc.
Momenta Pharmaceuticals, Inc.
Slide 26: Momenta Pharmaceuticals, Inc.
Momenta Pharmaceuticals, Inc.
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