Rocket Pharmaceuticals (RCKT) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic focus and pipeline prioritization

• Refocused on cardiovascular gene therapies with clear biological rationale and regulatory paths, deprioritizing Fanconi anemia and PKD to concentrate resources on high-impact programs.

• Three main cardiac programs: Danon disease, PKP2 arrhythmogenic cardiomyopathy (ACM), and BAG3 dilated cardiomyopathy (DCM), each targeting major genetic cardiomyopathy phenotypes.

• In-house AAV manufacturing supports all disclosed programs, enabling cost control, process improvement, and scalability for future launches.

• Wave two pipeline remains active but slowed to focus on highest value drivers; additional programs are ready to advance as resources allow.

• Seeking partnerships for deprioritized programs to ensure patient access.

Financial position and capital strategy

• Reported $222.8 million in cash as of September 30, 2025, sufficient to fund operations into Q2 2027, excluding potential PRV sale.

• Potential Kresladi approval in March 2026 could yield a PRV, providing strategic non-dilutive capital and extending cash runway.

• Commercial launch of Kresladi expected to build infrastructure for future AAV program launches.

Danon disease program updates

• Danon disease gene therapy (RP-A501) shows durable LAMP2 expression and robust efficacy in Phase I, with patients thriving beyond typical survival ages.

• Clinical hold in 2025 due to a safety event was resolved in under three months after protocol modifications and dose recalibration.

• Phase II pivotal trial to resume with a staggered safety run-in at a lower dose, aiming to replicate Phase I efficacy and safety.

• Natural history study validates rapid disease progression, supporting a small pivotal trial design.

• Strong community interest persists despite prior safety events, with most males expected to be eligible for therapy.