Rocket Pharmaceuticals
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Rocket Pharmaceuticals (RCKT) investor relations material

Rocket Pharmaceuticals Q4 2025 earnings summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Q4 2025 earnings summary26 Feb, 2026

Executive summary

  • Advanced three cardiovascular gene therapy programs, including resumption of pivotal Phase 2 trial for Danon disease in 1H 2026 and initiation of Phase 1 trial for BAG3-DCM in mid-2026.

  • KRESLADI™ for severe LAD-I remains on track for March 28, 2026 PDUFA date, with potential for a Rare Pediatric Disease Priority Review Voucher.

  • Organizational realignment led to disciplined resource allocation and reduced operating expenses.

Financial highlights

  • Cash, cash equivalents, and investments totaled $188.9M as of December 31, 2025, providing operational runway into Q2 2027.

  • Research and development expenses decreased to $142.0M for 2025 from $171.2M in 2024, reflecting lower manufacturing, development, and compensation costs.

  • General and administrative expenses fell to $86.5M in 2025 from $102.0M in 2024, mainly due to reduced commercial preparation and payroll expenses.

  • Net loss narrowed to $223.1M ($2.01/share) for 2025 from $258.7M ($2.73/share) in 2024.

  • Restructuring and related charges totaled $3.2M in 2025.

Outlook and guidance

  • Cash resources expected to fund operations into Q2 2027, excluding potential PRV proceeds.

  • Anticipates dosing additional Danon disease patients in 1H 2026 and first BAG3-DCM patient in mid-2026.

  • Ongoing FDA engagement for pivotal trial design in PKP2-ACM.

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TD Cowen 46th Annual Health Care Conference2 Mar, 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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