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: Two platforms - AAV-based therapies for cardiovascular diseases and ex vivo Lenti-based therapies for hematology [2][3] Core Points and Arguments Clinical Programs - Clinical Programs: Three hematology programs (LAD-I, Fanconi anemia, pyruvate kinase deficiency) and three cardiovascular programs (Danon disease, PKP2, BAG3) [2] - AAV Portfolio Focus: Increased focus on AAV programs due to high unmet need and potential for value creation [2][3] Danon Disease Program - Trial Resumption: The hold on the Danon program was lifted rapidly; trial will restart in the first half of 2026 with a staggered dosing approach [5][6] - Patient Safety: Previous safety events led to the removal of complement inhibitors from the trial design [5][6] - Regulatory Alignment: Ongoing positive interactions with the FDA regarding trial design and patient count [12][13] PKP2 Program - Trial Design Discussions: Ongoing discussions with the FDA about trial design; emphasis on a robust design that demonstrates clear benefit-risk [19][20] - Endpoints Consideration: Potential for composite endpoints due to the heterogeneity of the disease [21][22] BAG3 Program - Clinical Entry: The BAG3 program is set to enter the clinic next year, focusing on dilated cardiomyopathy (DCM) with traditional endpoints [25][26] - Predictability: DCM is better characterized, allowing for more predictable outcomes [25] Lenti-based Therapies - Focus Shift: A strategic decision to focus on AAV programs while seeking partnerships for Lenti-based therapies like Fanconi anemia [30][31] - PRV Eligibility: All Lenti programs (LAD, Fanconi, PKD, Danon) are eligible for Priority Review Vouchers (PRV), providing potential non-dilutive cash sources [34] CMC and Regulatory Updates - CMC Findings: Previous CMC findings have been addressed, and a resubmission to the FDA has been made, with hopes for approval by the PDUFA date of March 28 [35] Other Important Insights - Community Engagement: Strong commitment to patient advocacy and community engagement, especially following safety events [16][17] - Market Opportunity: The company is preparing for a comprehensive update on trial progress and market opportunity once clarity is achieved [10][15] This summary encapsulates the key points discussed during the conference call, highlighting Rocket Pharmaceuticals' strategic focus, clinical program updates, regulatory interactions, and community engagement efforts.