Sarepta Therapeutics Inc (NASDAQ:SRPT) shares plunged more than 21% after it revealed a patient with Duchenne muscular dystrophy (DMD) has died following treatment with its gene therapy ELEVIDYS.
Although acute liver injury is a known potential side effect of ELEVIDYS and similar therapies, this level of severity had not been previously reported, the company said in a statement.
The patient was also found to have a recent cytomegalovirus (CMV) infection, which may have contributed to liver damage.
Sarepta is continuing its investigation, with plans to update the prescribing information and notify relevant health authorities and physicians.
Safety concerns loom: analysts
Analysts at Jefferies expect stock weakness as uncertainty about the safety of ELEVIDYS will loom unless Sarepta deems the death to not be drug-related.
“Management plans to update the ELEVIDYS prescribing information to ‘appropriately’ represent this event, so we presume the death could be ELEVIDYS-related,” analysts wrote in a note to clients.
“Although liver tox is a known side effect, death caused by acute liver failure would represent a new safety signal for ELEVIDYS, which has been dosed in more than 800 patients clinically and commercially.”
The analysts noted that liver injury is a known risk within the first 90 days of ELEVIDYS treatment, and careful monitoring could mitigate such events.
While acute liver injury is common across AAV gene therapies, this safety issue underscores the unpredictability of risks, complicating investments in gene therapies. As a result, the benefit-risk assessment for Elevidys has worsened due to the increased safety concerns, Jefferies believes.
The analysts repeated their ‘Buy’ rating on Sarepta, noting the bull case is that the unmet need in DMD remains high.
They have a $165 price target on the company’s shares, which traded hands at about $80 on Tuesday morning.