Momenta Pharmaceuticals — Technical Presentation (June 2020)
20 slides from Momenta Pharmaceuticals's technical presentation, Jun 15, 2020
Slide 1: Vivacity-MG Phase 2 Interim Analysis Topline Results Investor and Analyst Conference Call
Vivacity - MG Phase 2 Interim Analysis Topline Results Investor and Analyst Conference Call June 15, 2020
Slide 2: Agenda
Introduction • Craig Wheeler, President and Chief Executive Officer Trial Design and Overview of Results • Santiago Arroyo, SVP and Chief Medical Officer Closing Remarks • Craig Wheeler, President and Chief Executive Officer Question & Answer Session Agenda 2
Slide 3: Forward Looking Statements
Statements in this presentation regarding management's future expectations, beliefs, intentions, goals, strategies, plans or p rospects, are forward - looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including but not lim ited to statements about our nov
Slide 4: Myasthenia Gravis, a Rare Autoimmune Neuromuscular Disease
Myasthenia Gravis, a Rare Autoimmune Neuromuscular Disease 4 • Caused by circulating autoantibodies, most commonly against acetylcholine receptors ( AChR ) or the muscle - specific receptor tyrosine kinase ( MuSK ) • Autoantibodies disrupt these receptors at post - synaptic neuromuscular junctions,
Slide 5: Nipocalimab (M281): Attributes of a potential best-in-class FcRn Antagonist
Nipocalimab (M281): Attributes of a potential best - in - class FcRn Antagonist 5 Dosing Dose - dependent IgG reduction Rapidly infused IV Weekly SC option Highest IgG reduction observed, >80% Ability to maintain 100% receptor occupancy drives IgG lowering and ability to maintain low IgG levels Effi
Slide 6: Nipocalimab was well tolerated, safe and efficacious in patients with gMG
Nipocalimab was well tolerated, safe and efficacious in patients with gMG 6 Dosing Efficacy with monthly dosing and seen as early as two weeks Supports continued clinical development in gMG and subcutaneous formulation dose selection Efficacy Nipocalimab demonstrated efficacy at all doses tested Sta
Slide 7: Topline Results Represent an Interim Analysis on the Study
• Data set includes all patients through Day 57 (week 8) • Analysis is for the primary efficacy outcome (MG - ADL change) and safety and laboratory data available up to day 113 (week 16) • The study was designed to detect a dose responsive efficacy in MG - ADL with at least an 80% power and experime
Slide 8: Vivacity-MG Phase 2 Study Design
Vivacity - MG Phase 2 Study Design 8 Placebo Q2W M281 5 mg/kg/Q4W M281 30 mg/kg/Q4W M281 60 mg/kg/Q2W M281 60 mg/kg single dose Open Label Extension Trial R a n d o m i z a t I o n Screening up to 4 weeks Week s Treatment Follow - Up 0 4 2 6 1 8 8 8 Primary Endpoint Assessment Dosing (M281 or placeb
Slide 9: Vivacity-MG: Subject Disposition
9 Vivacity - MG: Subject Disposition 5 mg/kg Q4W 14 30 mg/kg Q4W 13 60 mg/kg single dose 13 60 mg/kg Q2W 14 Placebo 14* 1 (7.1%) Total completed @ D57 68 Randomized Discontinued treatment due to pandemic Discontinued treatment for other reasons 0 13 (92.9%) 2 (15.4%) 1 (7.7%) 10 (76.9%) 3 (23.1%) 0
Slide 10: Baseline Characteristics
Nipocalimab (n=54) Placebo (n=13) Age mean years (SD) 54.3 (17.0) 58.7 (18.2) Female n (%) 29 (53.7) 7 (53.8) Time since diagnosis mean months (SD) 80.0 (83.3) 139.9 (114.7) MG - ADL score mean (SD) 8.0 (2.8) 7.0 (2.7) QMG score mean (SD) 16.5 (3.5) 17.7 (4.4) MGFA class n (%) II 20 (37.1) 5 (38.5)
Slide 11: Treatment Emergent Adverse Event Overview
Treatment Emergent Adverse Event Overview 11 There were no clinically relevant CK elevations Nipocalimab (n=54) Placebo (n=14) Patients with Adverse Event (AE) n (%) 44 (81.5) 11 (78.6) Patients with AE grade ≥3 n (%) 0 4 (28.6) Most frequent AEs n (%) Exacerbation of MG 0 2 (14.3) Headache 6 (11.1)
Slide 12: Average Albumin Concentrations Were Within Normal Limits
12 Average Albumin Concentrations Were Within Normal Limits 1 patient had an asymptomatic Grade 2 Hypoalbuminemia in the 60 mg/kg Q2W 30 35 40 45 50 Serum Albumin (g/L) Baseline (N=67) Day 8 (N=27) Day 15 (N=66) Day 29 (N=61) Day 43 (N=63) Day 57 (N=59) Day 85 (N=54) Day 113 (N=46) Lower Limit of No
Slide 13: Rapid and Dose Related Reduction of IgG as Predicted
• Dose dependent IgG decreases • Rapid onset significant lowering of IgG within 1 st week • Maximal decrease achieved at 60 mg/kg Q2W • Similar reductions were seen in: - IgG subclasses - Anti - AChR antibodies • No changes in IgA or IgM concentrations Rapid and Dose Related Reduction of IgG as Pred
Slide 14: Robust and Statistically Significant Relationship Between IgG Reduction and Clinical Benefit
Robust and Statistically Significant Relationship Between IgG Reduction and Clinical Benefit • MG - ADL improvement is highly correlated with serum IgG reduction (p<0.0001) • MG - ADL and Anti - AChR receptor binding antibodies are also highly correlated (p<0.0001) 14 ,J* 5DWLR;IURP %DVHOLQH &KDQJH
Slide 15: Durable Responses at All Doses
Difference vs Placebo: 27.5% 30.8% 38.5% 48.9% p - value: 0.1044 0.1008 0.0484 0.0092 Durable MG - ADL Responses at All Doses 15 0 10 20 30 40 50 60 70 15.4% N=2/13 42.9% N=6/14 46.2% N=6/13 53.9% N=7/13 64.3% N=9/14 % Patients Pooled nipocalimab arms showed a 51.9% durable MG - ADL response vs 15.4
Slide 16: Significant Durable Reductions Across All Doses
16 Significant Durable Reductions in MG - ADL Across All Doses 30 mg/kg Q4W 5 mg/kg Q4W Durable Response 6 5 4 3 2 14 7 0 7 14 7% 7% 14% 29% 15% 43% 15% Durable Response 6 5 4 3 2 14 7 0 7 14 8% 8% 15% 8% 15% 8% 23% 15% 46% 15% 8% 8% 8% Durable response is defined as improvement in MG - ADL >=2, 3
Slide 17: Rapid Onset of Action with Clinical Response within First Two Weeks
Early Onset Responders (response within the first 2 weeks) 17 Rapid Onset of Action with Clinical Response within First Two Weeks 15.4 % 42.9 % 38.5 % 46.2 % 42.9 % 0 5 10 15 20 25 30 35 40 45 50 Placebo 5 mg/kg Q4W 30 mg/kg Q4W 60 mg/kg single dose 60 mg/kg Q2W MG - ADL % Response
Slide 18: Robust and Dose Responsive Improvement from Baseline
Robust and Dose Responsive MG - ADL Improvement from Baseline 18 Day 57 Day 29 Placebo 5 mg/kg Q4W 30 mg/kg Q4W - 1.4 - 1.7 - 2.9 - 3.9 - 3.1 -4.5 -4 -3.5 -3 -2.5 -2 -1.5 -1 -0.5 0 - 1.8 - 2.5 - 3.9 - 1.5 - 3.9 -4.5 -4 -3.5 -3 -2.5 -2 -1.5 -1 -0.5 0 MG - ADL Change from Baseline MG - ADL Change from
Slide 19: Building a Winning FcRn Franchise Based on Efficacy, Safety and Dosing
Building a Winning FcRn Franchise Based on Efficacy, Safety and Dosing 19 Dermatology e .g., Pemphigus Rheumatology e .g., Systemic lupus erythematosus (SLE), Myositis Nephrology e .g., Lupus nephritis MG Neurology Fetal / Maternal HDFN Fetal neonatal alloimmune thrombocytopenia (FNAIT) Congenital h
Slide 20: Vivacity-MG Phase 2 Interim Analysis Topline Results Investor and Analyst Conference Call
Vivacity - MG Phase 2 Interim Analysis Topline Results Investor and Analyst Conference Call June 15, 2020
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