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, prioritizing Danon, PKP2, and BAG3 programs.

• Deprioritized Fanconi anemia and PKD, seeking partnerships for these assets to concentrate resources on high-impact areas.

• In-house AAV manufacturing supports scalability and cost control for current and future programs.

• Wave two programs remain active but are deprioritized to focus on near-term value drivers.

Financial position and capital strategy

• Reported $222.8 million in cash as of Q3 2025, sufficient to fund operations into Q2 2027, excluding potential PRV proceeds.

• 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 (RP-A501)

• Danon disease is a severe X-linked cardiomyopathy with high early mortality; prevalence estimated at 15,000–30,000 in US/EU.

• Phase I data show durable LAMP2 expression and robust efficacy out to five years, with patients thriving beyond typical survival.

• Clinical hold in 2025 due to a safety event led to protocol modifications and dose recalibration; hold lifted in under three months.

• Phase II pivotal trial to resume dosing in 2026 with a staggered safety run-in; pediatric data at recalibrated dose support efficacy and safety.

• Natural history study validates rapid disease progression and supports small pivotal trial design.