Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease

Excerpt from the Press Release:

CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease. The hold was lifted in under three months, underscoring the efficiency of the FDA’s review process and Rocket’s commitment to expeditiously optimize safety and resume the trial.

In its correspondence, the FDA confirmed that Rocket satisfactorily addressed issues outlined in the clinical hold. The FDA authorized the pivotal study to resume first with a recalibrated dose of 3.8 x 10¹³ GC/kg of RP-A501 in three patients, treated sequentially with a minimum four-week interval between each treatment. This adjusted dose aligns with the lower range of administered doses that were associated with efficacy across multiple biomarkers, echocardiographic and clinical endpoints in the Phase 1 study, and has been determined as most likely to confer the safety and efficacy identified in the low-dose Phase 1 cohorts. In addition, Rocket will collaborate with investigators to implement an immunomodulatory regimen more closely reflecting that administered in the Phase 1 pediatric cohort. The revised regimen discontinues prophylactic use of a C3 complement inhibitor, while maintaining sirolimus, rituximab, and steroids. Additionally, the protocol will specify a lower threshold for administering a C5 inhibitor (eculizumab) in response to impending complement activation.

To date, six patients with Danon disease have been treated in the Phase 2 study with RP-A501. Further updates about the Phase 2 study can be expected following review of data from the next three patients.

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