Catalyst Pharmaceuticals (CPRX) BofA Securities CNS Therapeutics Virtual Conference 2024 summary

Business development and strategic focus

• Focus remains on CNS and adjacent orphan disease areas, leveraging internal expertise and infrastructure to compete broadly in the orphan space.

• Actively engaged in multiple asset acquisition and partnership discussions, with a disciplined approach to fit and value.

• No current debt, with nearly $400 million available for deals and capacity for transactions up to $800 million.

• Growth is prioritized over synergy, aiming to build a robust pipeline and future valuation similar to the Horizon model.

• Confident in ability to integrate new assets quickly and effectively, as demonstrated with FIRDAPSE, FYCOMPA, and AGAMREE.

AGAMREE launch and market dynamics

• AGAMREE launch is tracking well, with patient accruals split evenly between Emflaza and prednisone, expanding the addressable market.

• 80% of centers of excellence and 140 of 250 target physicians have prescribed AGAMREE, with early adopters influencing broader uptake.

• Insurance coverage is strong, with 89% of patients approved in under 30 days; payer step edits are not a significant barrier.

• Product differentiation includes improved behavior, bone growth, bone density, and cardiovascular benefits, supported by ongoing long-term SUMMIT study.

• Market share and revenue per patient are expected to evolve as younger, lighter patients are treated for longer durations.

Market trends and future outlook

• The DMD steroid market is stable, with about 70% of patients currently treated and opportunity to increase initiation and duration of therapy.

• AGAMREE is positioned to address gaps in treatment by enabling earlier starts, optimal dosing, and longer therapy duration.

• Gross-to-net dynamics are expected to remain stable due to a balanced Medicaid/commercial payer mix.

• AGAMREE benefits from at least seven years of orphan exclusivity, with patents potentially extending protection to 2040.

• The company is optimistic about AGAMREE's potential to expand the market and achieve strong uptake, using Emflaza as a launch benchmark.