Rocket Pharmaceuticals (RCKT) 7th Annual Evercore ISI HealthCONx Healthcare Conference summary

Pipeline and program updates

• Six programs in the pipeline, five in clinical stages, spanning lenti ex vivo and in vivo cardiac gene therapy portfolios.

• Two lenti programs submitted for BLA/MAA, with anticipated approval for LAD in 2025; PRVs expected for LAD, Fanconi, and Danon due to rare pediatric designation.

• Danon program fully enrolled as of September, with ongoing dosing and a broader update planned for the first half of 2025.

• PKP2 low-dose cohort fully enrolled; preliminary data expected in the first half of 2025; PKD program to start in 2025.

• BAG3 IND filing anticipated in the first half of 2025, expanding the cardiac gene therapy portfolio.

Danon program clinical and regulatory strategy

• Dosing completion lags behind enrollment due to additional FDA-mandated lab screenings and protocol requirements.

• Natural history study ongoing, enrolling both retrospective and prospective cohorts, supporting regulatory submissions.

• Path to full approval involves following the pivotal trial for up to five years, with secondary endpoints like hospitalizations and quality of life becoming primary for full approval.

• Female Danon trial planned to follow the current male trial, addressing a significant patient population due to longer female survival.

• Commercial and epidemiological updates, including patient-finding strategies, to be presented in the first half of 2025.

PKP2 and cardiac gene therapy development

• PKP2 program focuses on protein expression and clinical endpoints such as PVCs, NSVTs, EKG changes, and right ventricular function.

• FDA supportive of biomarker-based endpoints; preliminary data from low-dose cohort to inform next steps.

• Biopsies scheduled at multiple time points, including before six and 12 months, with updates planned for 2025.

• Early safety monitoring prioritized due to potential for arrhythmias, with efficacy expected to manifest between six and 12 months.