Rocket Pharmaceuticals (RCKT) investor relations material

Strategic focus and pipeline overview

• Focused on gene therapies for rare genetic cardiomyopathies: Danon disease, PKP2, and BAG3, each representing a major subtype.

• Shifted resources in recent years to prioritize cardiomyopathies, while maintaining legacy ex vivo lenti programs, including KRESLADI for LAD-I, Fanconi anemia, and PKD.

• KRESLADI launch planned for Q4, with pricing expected to align with other gene therapies and below the lifetime cost of non-therapy.

• Pipeline includes six clinical programs and additional preclinical assets, with multiple catalysts anticipated in the next 9–12 months.

Clinical development and safety insights

• Danon pivotal trial ongoing, with three patients dosed at a recalibrated 3.8E13 vg/kg, showing good safety and tolerability.

• Safety concerns with AAV gene therapy addressed by dose adjustment; TMA risk minimized at lower doses, with robust screening for predisposing factors.

• Efficacy at this dose supported by reductions in LV mass index and positive biomarker staining in phase I/II patients.

• Ongoing FDA discussions may require additional patients for the pivotal trial, which is seen as beneficial for commercial and regulatory purposes.

Patient identification and community engagement

• Over 50 patients enrolled in a natural history study to support trial recruitment and data robustness.

• Mission: Genome initiative provides no-cost genetic testing to facilitate early and accurate diagnosis.

• ICD-10 code for Danon in place for 29 months, with active outreach to HCPs and geneticists to encourage use and diagnosis.

• Educational campaigns target not only pediatric cardiologists but also neurologists, pediatricians, and other specialists due to varied symptom presentation.