Sarepta shares tumble on second patient death after gene therapy treatment
June 16 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O), opens new tab plunged 34% in premarket trading on Monday, a day after the company disclosed a second case of patient death due to acute liver failure after receiving its gene therapy for a rare form of muscular dystrophy.
This marks the second fatality in just three months, with both patients being non-ambulatory and experiencing similar complications after receiving the therapy called Elevidys.
In response, Sarepta said on Sunday it has temporarily suspended shipments of Elevidys for patients unable to walk, while it works with experts and regulators to evaluate an enhanced immunosuppressive regimen.
"With the second death, it seems near certain that non-ambulatory is high risk, and the company may need to provide safety data with an enhanced immunosuppression regimen before it's approved for commercial use," analysts at Piper Sandler said in a note.
The brokerage cuts its rating on the Sarepta stock to 'neutral' from 'overweight' and slashed its price target to $36 from $70, the lowest among 26 analysts covering the stock.
Elevidys is the only gene therapy approved by U.S. drug regulators for Duchenne muscular dystrophy in patients aged four and above.
"The second patient death may raise serious questions on Elevidys' sales outlook," analysts at Jefferies said.
In April, the company paused some Elevidys trials in Europe following the first patient death a month earlier, though an independent panel upheld the therapy's benefit-risk profile.
Liver damage is a known risk with Elevidys and other gene therapies that use adeno-associated viral vectors to infuse modified genes.
Elevidys is a one-time gene therapy that delivers a micro-dystrophin gene to muscle cells, aiming to treat the root cause of Duchenne, which causes progressive muscle deterioration.
Roche (ROG.S), opens new tab, Sarepta's commercialization partner for Elevidys outside the United States, also said on Sunday that it has paused dosing of the gene therapy in non-ambulatory patients following the casualty.
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