Rocket Pharmaceuticals Reports $61.3 Million Net Loss in Q1 2025, Focuses on Gene Therapy Development

Rocket Pharmaceuticals, Inc. reported its financial results for the first quarter of 2025, revealing a net loss of $61.3 million, a slight improvement from the $62.1 million loss recorded in the same period of 2024. The company continues to operate without generating any revenue, maintaining its focus on the development of gene therapies for rare diseases. Total operating expenses for the quarter were $64.4 million, down from $67.4 million a year earlier, primarily due to a reduction in research and development (R&D) expenses, which decreased to $35.9 million from $45.2 million. This decline was attributed to lower manufacturing and development costs, as well as a decrease in R&D headcount.

In terms of strategic developments, Rocket Pharmaceuticals has made significant progress in its clinical programs. The company has two clinical-stage and one pre-clinical stage in vivo adeno-associated viral (AAV) programs, including ongoing trials for Danon disease (RP-A501) and Plakophilin-2 Arrhythmogenic Cardiomyopathy (RP-A601). The company announced that it reached alignment with the FDA on the pivotal study design for RP-A501, with enrollment completion reported in September 2024. Additionally, the FDA has granted priority review for the Biologics License Application (BLA) for RP-L201, targeting severe Leukocyte Adhesion Deficiency-I.

Operationally, Rocket Pharmaceuticals has seen changes in its employee headcount, which has contributed to the reduction in R&D expenses. The company reported a total of 106.8 million shares outstanding as of March 31, 2025, an increase from 106.5 million shares at the end of 2024. The company’s cash and cash equivalents decreased significantly to $49.8 million from $163.6 million at the end of the previous fiscal year, reflecting ongoing investments in R&D and operational activities. The total assets of the company stood at $471.1 million, down from $527.7 million at the end of 2024.

Looking ahead, Rocket Pharmaceuticals anticipates that its existing cash, cash equivalents, and investments will be sufficient to fund operations into the fourth quarter of 2026. The company continues to face risks associated with drug development, including the need for additional financing to support its clinical trials and operational needs. The management remains focused on advancing its gene therapy programs and is optimistic about the potential for future revenue generation upon successful regulatory approvals.