Myasthenia Gravis Market Assessment: Epidemiology, Treatment Landscape, Unmet Needs, Emerging Therapies, and Value & Access

Table of Contents

• A rare, chronic, autoimmune disease marked by muscle weakness and fatigue
• Figure 1.1. Select clinical manifestations of MG
• FcRn inhibition, B-cell directed therapies, and the complement pathway are common drug targets
• Figure 1.2 MG drugs targets in AChR+ patients

• Disease definition
• Figure 2.1. Diagnosed prevalent cases of MG by region
• Table 2.1 Diagnosed prevalent and drug-treated patients in the US and EU5

• Overview
• Figure 3.1. Diagnostic pathway for MG patients
• Diagnostic testing for auto-antibodies is standard and disease severity criteria for MG are clearly defined
• Table 3.1. MGFA Clinical Classification
• Figure 3.2. Neurologist-reported percentage of MG patients misdiagnosed/not diagnosed in a timely manner
• Figure 3.3. Neurologist-reported percentage of their patients in each clinical category
• Improvement of symptoms and restoring patient’s baseline function and quality of life are the ultimate goals
• Table 3.2. Treatment goals for MG
• Figure 3.4. Neurologist-reported proportion of MG patients currently treated with drug therapy
• Treatment flow for MG
• There is no standard approach to treatment; biologics are emerging as key players in recent years
• Figure 3.5. Neurologist-reported current treatment share for MG patients: 1st-line setting
• Figure 3.6. Neurologist-reported % of patients not well-managed by first-line therapy
• Figure 3.7. Neurologist-reported current treatment share for MG patients: 2nd-line and later settings
• Upsides and downsides of current non-biologic MG treatments
• Head-to-head comparison of FDA-approved biologics
• Table 3.3. Comparison of approved biologics
• Physician perspectives on current biologics
• Table 3.4 . KOL insights on approved biologics
• Cost, dosing convenience, and safety profile are the key factors that drive biologic choice
• Table 3.5. KOL feedback on biologics based on key differentiators that drive treatment choice
• With Argenx taking the lead, the race is on to develop more conveniently dosed biologics
• Table 3.6. Summary of the ADAPT-SC trial results
• Key treatment dynamics that will shape disease management and drug use in MG
• Figure 3.7. Important dynamics of MG market evolution

• Overview
• Figure 4.1. Top unmet needs in MG
• Figure 4.2. Neurologist-reported unmet needs in MG
• High unmet needs persist despite three approved biologics for MG

• Overview
• Figure 5.1. Percentage of neurologists (n=26) rating target as “promising” for MG (score of 6 or 7)
• The late-stage pipeline for MG is very active
• Table 5.1. Key MG therapies in Phase 3
• The late-stage pipeline for MG is very active
• Table 5.1. Emerging MG therapies in Phase 3 (cont.)
• Rozanolixizumab and zilucoplan are poised to be the latest entrants to the MG market
• Figure 5.2. Phase 3 results for UCB’s Rozanolixizumab in MycarinG trial
• Figure 5.3. Phase 3 results for UCB’s Zilucoplan in RAISE trial
• The early-stage pipeline is also active and focused on largely similar mechanisms
• Table 5.2. Notable MG therapies in Phase 1 / 2 Development
• Neurologists’ opinions on the MG pipeline

• Overview
• Table 6.1. Current MG therapy pricing, U.S.
• Table 6.2. Typical U.S. commercial payer coverage ofrituximab for MG patients (Aetna, BCBS, Cigna, United)
• Prior authorization criteria for biologics
• Table 6.3. Typical U.S. commercial payer coverage of biologics for MG patients
• Alexion and Argenx utilize patient support programs to expand access
• Figure 6.1. Growth in lives covered through VBAs since launch of Vyvgart

• Primary market research approach
• Epidemiology methodology

Samples

Companies Mentioned

• UCB
• Immunovant
• Janssen
• Alexion/Astrazeneca
• Regeneron
• Sanofi
• Hoffmann-La Roche
• RemeGen
• NMD Pharma
• Cartesian Therapeutics
• Cabaletta Bio
• IASO Bio