By Adriano Marchese
Rocket Pharmaceuticals shares fell sharply in premarket trading Tuesday after a trial for its investigational therapy for Danon disease was halted by regulators following the death of a patient that developed complications while on the drug.
Shares fell 62% ahead of the morning bell at $2.39.
The late-stage biotechnology said Tuesday that a patient participating in second phase of its RP-A501 trial experienced an unexpected serious adverse event from which the patient later died. In particular, the patient experienced symptoms of capillary leak syndrome, a condition where fluid leaks from blood vessels into surrounding tissues, and died after an acute systemic infection.
Rocket voluntarily paused all dosing in the study, and on May 23, the Food and Drug Administration placed a clinical hold on the trial to allow for further evaluation.
The company said it is working with a regulators, experts and investigators and is looking to restart the trial as quickly as possible. However, it said that it is unable provide guidance on when it expects to complete the phase 2 trial while the clinical hold is in place.
Danon disease is a rare genetic disorder that affects mainly the heart, muscles, eyes and brain, where cells struggle to break down molecules which leads to a buildup of harmful substances.
Write to Adriano Marchese at adriano.marchese@wsj.com
(END) Dow Jones Newswires
May 27, 2025 07:50 ET (11:50 GMT)
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