Rocket Pharmaceuticals (NASDAQ:RCKT) saw its stock tumble 57% in premarket trading after reporting a patient death linked to serious adverse effects during a clinical trial. This tragic incident has triggered an in-depth investigation and ongoing discussions with the FDA as the company works to determine the cause of the complication.
The patient’s passing was associated with capillary leak syndrome, which developed following the introduction of a new immune suppression drug in the pre-treatment protocol for the AAV9-Danon program. This medication was intended to reduce complement activation observed in some trial participants. In response to the event, Rocket has voluntarily halted further dosing, and the FDA imposed a clinical hold on the study as of May 23 to conduct a thorough review.
As of March 31, Rocket Pharmaceuticals held $318.2 million in cash, cash equivalents, and investments. Given the current circumstances, the company is redirecting its focus toward its AAV platform and evaluating strategic alternatives for other assets in its pipeline to maximize shareholder value.
This refocused strategy is expected to lower cash burn, with Rocket now projecting that its current funds will sustain operations through 2027. This outlook excludes any proceeds that might arise from selling Priority Review Vouchers, which could be awarded after approvals of therapies in its hematology portfolio.