Rocket Pharmaceuticals (RCKT) investor relations material

Strategic focus and portfolio updates

• Shifted emphasis toward AAV-based cardiovascular gene therapies to maximize value and extend cash runway.

• Three main cardiovascular programs: Danon disease, PKP2, and BAG3, with BAG3 just entering the clinic.

• Lenti-based hematology programs (LAD-I, Fanconi anemia, PKD) are now positioned for partnerships rather than internal focus.

• All major programs (LAD, Fanconi, PKD, Danon) are PRV eligible, offering potential non-dilutive funding.

• Ongoing commercial and epidemiology analyses to support future launches.

Danon disease program progress

• Clinical hold lifted rapidly after addressing safety concerns; trial to restart in first half of 2026.

• Initial three patients to be dosed in a staggered fashion, followed by FDA alignment and then broader enrollment.

• Comprehensive update planned after safety run-in and regulatory discussions, including trial, epidemiology, and commercial insights.

• Community and patient advocacy groups remain supportive despite prior safety events.

• Treated patients show positive biomarker and quality of life improvements compared to untreated cases.

PKP2 and BAG3 program developments

• PKP2 trial at final dose; ongoing FDA discussions focus on robust trial design and appropriate endpoints.

• Considering composite endpoints due to disease heterogeneity; quality of life measures seen as important.

• Early PKP2 data show substantive patient improvement in daily function and symptoms.

• BAG3 program to start in clinic next year, targeting dilated cardiomyopathy with traditional endpoints.

• Phase 1 for BAG3 will use a traditional dose-escalation design, aiming to leverage data for pivotal trials.