Rocket Pharmaceuticals (RCKT) UBS Global Healthcare Conference 2024 summary

Platform overview and development strategy

• Two gene therapy platforms: Ex Vivo Lenti for hematology and In Vivo AAV for cardiac indications, with potential for future expansion.

• Focus on rare, severe, and often fatal diseases, especially those affecting children and adolescents.

• Indication selection targets direct gene/protein impact in the relevant cell, rapid clinical/regulatory paths, and increasing market size.

• Six programs in total: three hematology and three cardiac, each with growing addressable markets.

Regulatory progress and commercialization plans

• Addressing FDA's CMC-related CRL for LAD, with anticipated approval in the coming year.

• LAD market is small but offers a PRV opportunity and infrastructure-building experience.

• Fanconi anemia BLA rolling submission underway, aiming for launch in 2026.

• Both LAD and Fanconi expected to be commercial products by 2025/2026.

• Fanconi anemia has a larger TAM (~6,000 targetable cases), with pricing analogs to other Ex Vivo Lenti therapies.

Danon disease (RP-A501) clinical development

• Phase 2 pivotal trial fully enrolled (12 patients), focusing on LAMP2 protein expression and LV mass reduction as co-primary endpoints.

• Trial design informed by extensive FDA dialogue; 12-month endpoint, with top-line data expected after all patients reach this mark.

• Long-term phase 1 data update to be presented at AHA, with up to five years of follow-up.

• Age-agnostic benefit observed; both pediatric and adult patients show response.

• Accelerated and full approval paths discussed with FDA, with full approval requiring longer follow-up and more clinical endpoints.