股价跌超60%！Rocket基因疗法致患者死亡，FDA紧急叫停

Core Viewpoint - The FDA has paused Rocket Pharma's critical Phase 2 clinical trial for its gene therapy RP-A501 targeting Danon disease due to a serious adverse event resulting in a patient's death [1][2][3]. Group 1: Clinical Trial Impact - A serious adverse event occurred during the Phase 2 trial, leading to the death of a patient from complications related to capillary leak syndrome [2]. - The FDA issued a clinical hold on May 23, requiring further investigation into the cause of the event [2]. - Rocket Pharma is collaborating with the FDA, independent data safety monitoring boards, and clinical researchers to ensure patient safety and aims to resume the trial as soon as possible [5]. Group 2: Financial and Market Reaction - Following the announcement, Rocket's stock price plummeted over 62% in a single day, marking an over 80% decline year-to-date [3]. - Analysts estimate Rocket's quarterly cash burn to be between $40 million to $50 million, with the clinical hold expected to pressure the company's financial situation [7]. - As of the end of Q1, Rocket reported cash and cash equivalents totaling $318.2 million, which is projected to sustain operations until 2027, excluding potential revenues from expedited review vouchers for certain therapies [7]. Group 3: Industry Context - Other companies in the industry have also faced patient deaths in clinical trials, raising concerns about the long-term impact on gene therapy investments [6][7]. - Jefferies analysts suggest that the recent death has damaged the risk-benefit profile of the Danon project, while BMO Capital Markets remains optimistic about the potential efficacy of RP-A501 given the poor prognosis of Danon disease patients [7].