Rocket Pharmaceuticals (RCKT) investor relations material

Strategic focus and pipeline reprioritization

• Shifted focus to cardiovascular genetic medicines, targeting rare diseases with high unmet need and limited alternatives.

• Reprioritized pipeline to concentrate resources on cardiac gene therapy programs, deprioritizing legacy ex vivo lentiviral programs.

• Cardiomyopathy programs (Danon, PKP2, BAG3) identified as highest value opportunities, each with billion-dollar-plus potential.

• Actively exploring out-licensing or partnerships for Fanconi Anemia and PKD programs, with Fanconi nearing BLA readiness.

• Considering potential licensing of Kresladi to maintain focus on core cardiovascular assets.

Clinical progress and regulatory updates

• Danon pivotal study resumed after FDA lifted clinical hold; recalibrated dosing and revised immunomodulatory regimen implemented.

• Updates on Danon trial safety cohort, regulatory path, and commercial opportunity expected in the second half of the year.

• PKP2 program advancing to pivotal trial after promising phase I results; pivotal study design update anticipated later this year.

• BAG3 phase I trial starting soon, targeting dilated cardiomyopathy, the largest genetic cardiomyopathy segment.

• European and UK centers active for Danon, with US BLA prioritized before European filings.

Financial position and capital allocation

• Cash runway extended into Q2 2028 following $180 million PRV sale.

• Additional resources will be needed for full commercialization, especially for Danon.

• Capital deployment tied to milestone achievements and program de-risking.