Rocket Pharmaceuticals Advances Gene Therapy Pipeline for Rare Disorders
核心洞察
Rocket Pharmaceuticals completed enrollment in the Phase 2 pivotal study of RP-A501 for Danon disease (搜索), with updated Phase 1 data expected at AHA 2024.
Enrollment finished in the low dose cohort of the Phase 1 study of RP-A601 for PKP2-ACM (搜索), with preliminary data anticipated in the first half of 2025.
The company initiated a rolling BLA submission for RP-L102 for Fanconi Anemia (搜索) and anticipates FDA approval of KRESLADI for severe LAD-I in 2025.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced significant progress in its gene therapy pipeline for rare disorders, according to its third-quarter financial results for 2024. The company is advancing multiple clinical programs targeting diseases with high unmet needs, including Danon disease (搜索), PKP2-arrhythmogenic cardiomyopathy (ACM), Fanconi Anemia (搜索) (FA), and Leukocyte Adhesion Deficiency-I (搜索) (LAD-I).
RP-A501 for Danon Disease
Rocket Pharmaceuticals completed enrollment in the Phase 2 pivotal study of RP-A501 for Danon disease (搜索) in September. Danon disease is a devastating heart failure condition. Dosing is ongoing in the Phase 2 study (NCT06092034). Updated Phase 1 data will be presented at the American Heart Association’s 2024 Late-Breaking Science sessions on November 18.
RP-A601 for PKP2-ACM
Enrollment has been completed in the low dose cohort of the Phase 1 study of RP-A601 for PKP2-ACM (搜索) (NCT05885412). Preliminary data from this study is expected in the first half of 2025. PKP2-ACM affects approximately 50,000 people in the U.S. and Europe.
RP-L102 for Fanconi Anemia
The company has initiated a rolling Biologics License Application (BLA) for RP-L102, an investigational gene therapy for Fanconi Anemia (搜索) (FA). Regulatory review by the European Medicines Agency is also underway. An ICD-10 code has been secured from the Centers for Medicare and Medicaid Services to document patients with FA.
KRESLADI for Severe LAD-I
The U.S. Food and Drug Administration (FDA) is reviewing additional Chemistry Manufacturing and Controls (CMC) information for KRESLADI™ (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (搜索) (LAD-I). Approval is anticipated in 2025.
Financial Position and Leadership
As of September 30, 2024, Rocket Pharmaceuticals reported cash, cash equivalents, and investments of $235.7 million, which are expected to fund operations into 2026. Mikael Dolsten, M.D., Ph.D., has been appointed to the Board of Directors. Dr. Dolsten is currently the Chief Scientific Officer, President, Research & Development at Pfizer Inc.
"Rocket made meaningful progress during the third quarter, notably with the completion of enrollment in the RP-A501 program for Danon disease (搜索), low dose cohort enrollment completion in the RP-A601 program for PKP2-ACM (搜索), and appointment of seasoned pharmaceutical executive, Mikael Dolsten to our Board of Directors," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. "As we continue to pursue our mission of seeking gene therapy cures for patients with rare and devastating diseases, we remain focused on expediently advancing our deep pipeline of cardiovascular and hematology programs."