Rocket Pharmaceuticals (RCKT) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Gene therapy platforms and regulatory environment

• Two platforms: ex-vivo lentiviral (LV) for bone marrow diseases and in-vivo AAV for solid organ diseases, with five clinical programs and a sixth entering the clinic soon.

• LV is suited for hematologic and some neurologic diseases, while AAV targets heart, liver, and CNS disorders.

• Manufacturing and regulatory learnings are shared between platforms, creating operational synergies.

• FDA has become more collaborative, supporting lean, single-arm trials with biomarker endpoints.

• Early programs benefited from iterative engagement with regulators, leading to efficient trial designs.

Danon disease program progress

• Danon disease gene therapy uses AAV to restore LAMP2, rapidly clearing cellular debris and improving heart function.

• Phase 1 showed transformative results: all six patients had improved protein expression, reduced cardiac injury markers, and better quality of life.

• Pivotal Phase II is a single-arm trial with 12 patients, using reduction in LV mass and LAMP2 expression as primary endpoints.

• FDA agreed to full approval path based on five-year follow-up of treated patients, without requiring another trial.

• Enrollment is ongoing in the US and Europe, with no specific completion date disclosed.

Market opportunity and diagnosis challenges

• Danon disease prevalence estimated at 15,000–30,000 in US and Europe, but only about 20% are diagnosed due to frequent misdiagnosis.

• Diagnosis is complicated by non-cardiac symptoms and lack of awareness; gene testing at birth is needed for full capture.

• Efforts underway to partner with newborn screening institutes and educate physicians to improve diagnosis rates.