Rocket Pharmaceuticals (RCKT) investor relations material

Strategic focus and pipeline updates

• Six disclosed programs span in vivo AAV-based cardiac and ex vivo lentiviral-based hematology therapies, with a shift in resources toward AAV cardiac assets for higher near- and medium-term value.

• Workforce reduction implemented to prioritize key programs and extend cash runway into Q2 2027, with further extension possible via a Priority Review Voucher.

• Fanconi Anemia and Pyruvate Kinase Deficiency programs paused, with resources redirected to cardiac portfolio; partnership opportunities are being explored for these assets.

Danon Disease program developments

• Phase one results showed robust protein expression and significant biomarker and functional improvements in all patients, leading to a pivotal phase two trial.

• Phase two encountered safety issues (TMA and capillary leak syndrome), resulting in a clinical hold after a patient death; hold was lifted after reverting to a phase one-like immunomodulatory regimen and recalibrating the dose down by 40%.

• Patient enrollment for the restarted trial is expected in early 2026, with a 12-patient design agreed upon with FDA; further patient numbers may be required depending on safety data.

• Manufacturing is in-house, providing cost and timeline advantages for future commercialization.

PKP2 and BAG3 cardiac programs

• PKP2-arrhythmogenic cardiomyopathy program demonstrated robust protein expression and clinical improvements in three patients at a single dose, with pivotal trial design discussions ongoing.

• At least 50,000 patients in the US and Europe are affected by PKP2, with potential for multiple therapies in the market.

• BAG3-associated dilated cardiomyopathy program cleared IND, with phase one anticipated to start in 2026; endpoints likely to focus on protein expression and LV function.