Rocket Q4 2024 Earnings Report
Key Takeaways
Rocket Pharmaceuticals reported a net loss of $60.3 million for Q4 2024. The company highlighted progress in its gene therapy pipeline, including ongoing clinical trials for RP-A501 and RP-A601, and regulatory reviews for KRESLADI and RP-L102. The company's cash, cash equivalents, and investments totaled $372.3 million, expected to fund operations into Q3 2026.
Dosing is ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease, with a program update expected in the first half of 2025.
Enrollment completed in the low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM with initial data expected in the first half of 2025.
Regulatory reviews are ongoing for KRESLADI (severe LAD-I) and RP-L102 (Fanconi Anemia).
Cash, cash equivalents and investments of approximately $372.3M, expected to fund operational runway into the third quarter of 2026.
Rocket
Rocket
Forward Guidance
Rocket Pharmaceuticals expects its current cash resources to be sufficient to fund operations into the third quarter of 2026, including AAV cGMP batch production and continued development of its clinical and preclinical programs.
Positive Outlook
- Continued development of six clinical and/or preclinical programs.
- Producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility.
- Advancing AAV cardiovascular programs
- Submission of complete BLA to resolve Complete Response Letter anticipated in 2025.
- Submission of the IND is anticipated in the first half of 2025.
Challenges Ahead
- Dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates
- The outcome of litigation
- Unexpected expenditures
- The integration of new executive team members and the effectiveness of the newly configured corporate leadership team
- Rocket’s ability to obtain and enforce patents to protect its product candidates