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Rocket Pharmaceuticals (RCKT) investor relations material
Rocket Pharmaceuticals Q3 2025 earnings summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Q3 2025 earnings summary6 Nov, 2025
Executive summary
Focused on gene therapies for rare cardiovascular and hematologic diseases, with a strategic shift in July 2025 to prioritize AAV-based cardiovascular programs and de-prioritize certain hematology programs.
Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026 after FDA lifted clinical hold; three additional patients to be dosed at recalibrated levels.
KRESLADI™ for severe LAD-I has a PDUFA date set for March 28, 2026, with eligibility for a Rare Pediatric Disease Priority Review Voucher.
Advancements in AAV cardiovascular gene therapy portfolio, including RP-A601 for PKP2-ACM and RP-A701 for BAG3-DCM, with ongoing FDA engagement and trial preparations.
Leadership strengthened with appointments of new Chief Medical Officer, Chief Operating Officer, Chief Commercial & Medical Affairs Officer, and Chief Science and Gene Therapy Officer.
Financial highlights
Net loss for Q3 2025 was $50.3 million, an improvement from $66.7 million in Q3 2024; net loss for the nine months ended September 30, 2025 was $180.6 million, compared to $198.4 million in the prior year period.
R&D expenses decreased to $34.1 million in Q3 2025 from $42.3 million in Q3 2024; nine-month R&D expenses were $112.7 million, down from $133.9 million year-over-year.
General and administrative expenses fell to $18.4 million in Q3 2025 from $27.1 million in Q3 2024; nine-month G&A expenses were $71.8 million, down from $76.6 million.
Cash, cash equivalents, and investments totaled $222.8 million as of September 30, 2025, expected to fund operations into Q2 2027.
108,208,643 shares of common stock were outstanding as of September 30, 2025.
Outlook and guidance
Current resources are expected to fund operations into Q2 2027, following the July 2025 reorganization.
Anticipated dosing of additional patients in RP-A501 Phase 2 study in 1H 2026; ongoing regulatory and clinical milestones for other pipeline assets.
Focused on advancing AAV cardiovascular programs and supporting regulatory submissions for KRESLADI™.
De-prioritization of FA and PKD programs; seeking external partnerships for these assets.
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Next Rocket Pharmaceuticals earnings date
Q4 202526 Feb, 2026
Next Rocket Pharmaceuticals earnings date
Q4 202526 Feb, 2026
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Frequently asked questions
Rocket Pharmaceuticals Inc is a clinical-stage biotechnology company focused on developing gene therapies for rare and life-threatening genetic diseases. The company’s pipeline includes gene therapy programs targeting both hematologic and cardiovascular disorders. Rocket Pharmaceuticals uses lentiviral and adeno-associated virus (AAV) vectors to deliver corrective genes to patients' cells, aiming to provide long-term, potentially curative treatments. Its therapies are designed to address the underlying genetic causes of diseases, with a focus on disorders with limited treatment options. The company is headquartered in Cranbury, New Jersey, and its shares are listed on the NASDAQ.
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