Rocket Pharmaceuticals (NasdaqGM:RCKT) FY Conference Transcript

Summary of Rocket Pharmaceuticals FY Conference Call Company Overview - Company: Rocket Pharmaceuticals (NasdaqGM:RCKT) - Industry: Gene Therapy - Focus: Development of gene therapies for rare diseases, particularly in cardiomyopathy and hematology Key Points Company and Product Pipeline - Rocket Pharmaceuticals operates as a dual platform company with both in vivo AAV-based cardiac programs and ex vivo lentiviral-based hematology programs in development [2][3] - The company has six disclosed programs, including three cardiac programs targeting Danon Disease, PKP2-arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, collectively affecting at least 100,000 patients in the U.S. and Europe [2][3] - The lentiviral portfolio includes Leukocyte Adhesion Deficiency-I, Fanconi Anemia, and Pyruvate Kinase Deficiency, with a decision to pause new spending on the latter two to focus resources on AAV-based cardiac programs [3][4] Financial and Operational Updates - A workforce reduction was implemented to prioritize certain programs and extend cash runway, with cash expected to last into Q2 2027 [4] - The company aims to be first, best, and only in class for its gene therapy products, targeting diseases with increasing market sizes [3][4] Clinical Trials and Regulatory Interactions - The pivotal phase two trial for Danon Disease was put on clinical hold due to increased risk of TMA (Thrombotic Microangiopathy) but was lifted in less than three months after modifications to the treatment regimen [9][10] - The trial will start in early 2026, with a three-month troponin run-in period before patient treatment begins [10] - The pivotal trial design includes a 12-patient trial with composite endpoints of protein expression and LV mass index reductions, agreed upon with the FDA [24][30] Market Opportunity - The prevalence of Danon Disease is estimated at 15,000 to 30,000 patients in the U.S. and Europe, with ongoing efforts to identify more patients through genetic testing [34] - The company is positioned to potentially file for commercialization in the next few years, contingent on trial outcomes [35][36] Future Catalysts - Upcoming catalysts include the PDUFA date for CRISLATI, clarity on PKP2 trial design, and updates on the Danon program and BAG3 phase one start [61][62] - The company plans to proceed cautiously with its programs, focusing on one at a time to ensure successful execution [62] Additional Insights - The company has learned valuable lessons from the Danon Disease program that will inform future trials, particularly regarding safety monitoring and regulatory interactions [38][39] - The choice of vectors for gene therapy is strategic, with AAV9 used for Danon Disease and RH74 for PKP2 and BAG3, based on regulatory experience and patient community feedback [55] Conclusion - Rocket Pharmaceuticals is actively advancing its gene therapy programs with a focus on rare diseases, navigating regulatory challenges, and preparing for future commercialization opportunities while ensuring patient safety and efficacy in its clinical trials [61][62]