Investing.com -- Shares of Rocket Pharmaceuticals (NASDAQ: RCKT ) plummeted 57% in premarket trading following the announcement of a patient death due to serious adverse events related to their clinical trial. The event has prompted a comprehensive investigation and discussions with the FDA, as the company seeks to understand the root cause of the complications.

The patient’s death occurred after complications linked to capillary leak syndrome, which emerged after the introduction of a new immune suppression agent in the pre-treatment regimen of the AAV9-Danon program. This agent was intended to mitigate complement activation seen in some participants. Due to this serious incident, Rocket has voluntarily paused further dosing in the study, with the FDA placing a clinical hold on the trial as of May 23 to allow for a detailed evaluation.

Rocket Pharmaceuticals reported having $318.2 million in cash, cash equivalents, and investments as of March 31. In light of the current situation, the company is refocusing its investments towards its AAV platform and is considering strategic options for the rest of its pipeline to optimize value.

This strategic shift is anticipated to result in a reduced cash expenditure, with the company now expecting its existing resources to fund operations into 2027. This forecast does not include any potential income from the sale of Priority Review Vouchers, which may be granted following the approval of therapies from its hematology portfolio.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.