Rocket Pharmaceuticals (RCKT) Q4 2025 earnings summary

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.