Key Takeaways
- Rocket Pharmaceuticals halted a study of a gene-therapy drug for Danon disease because a patient had a "Serious Adverse Event" and later died.
- The patient suffered from complications related to a blood vessel leak.
- The news sent shares of Rocket Pharmaceuticals plunging to an all-time low.
Rocket Pharmaceuticals (RCKT) shares plunged to an all-time low Tuesday when the drugmaker reported a patient taking its experimental treatment for Danon disease had a "Serious Adverse Event (SAE)" and later died, putting a halt to the research.
The company explained that in a 澳洲幸运5官方开奖结果体彩网:Phase 2 trial of its investigational gene therapy RP-A501, the patient experienced "clinical complications relate🌄d to a capillary leak syndrome," and subsequently "passed away after an acute systemic infection."
Rocket Pharmaceuticals said it is conducting a "comprehensive root cause analysis" that is currently focusing on "the recent introduction of a novel immune suppression agent to the pre-treatment regimen that had been implemented to mitigate complement activation observed in some patients."
After the incident, Rocket Pharmaceuticals paused the study, and the 澳洲幸运5官方开奖结果体彩网:Food and Drug Administration (FDA) placed a clinical hold on 🐻it. The company said it "is unable to provide guidance on the anticipated timing for completion of the Phase 2 trial."
Shares of Rocket Pharmaceuticals lost 60% their value in Tuesday morning trading, and are down about 80% year-to-date.
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