(Reuters) -Amylyx Pharmaceuticals said on Wednesday it will stop development of its experimental drug for a rare brain disorder after it failed to improve symptoms in patients during a mid-stage trial.
Shares of the company fell 1.7% in premarket trading after the drug, AMX0035, showed no benefit over a placebo at 24 weeks in patients with a rare and fatal brain disorder called progressive supranuclear palsy (PSP).
AMX0035 was approved by the Food and Drug Administration to treat amyotrophic lateral sclerosis in 2022 and was sold under the brand Relyvrio in the U.S. But the company voluntarily withdrew the drug from the U.S. and Canada last year after failing in a key late-stage trial.
“While a slight disappointment, we believe investors had very low expectations for the data,” TD Cowen analysts wrote in a client note.
The PSP trial failure has prompted the company to halt both the mid-stage study and its open-label extension, Amylyx said. The company will also not conduct a late-stage study of the drug for the disorder.
“We are disappointed in these results,” said Chief Medical Officer Camille Bedrosian, adding that the company had set a “high bar” for AMX0035 in PSP.
PSP affects about seven in 100,000 people worldwide, according to the company. The brain disorder, which impacts movement, balance, eye motion, swallowing, and speech, currently has no FDA-approved treatments.
Amylyx is also testing AMX0035 as a potential treatment for Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss, it said.
The company also has other experimental drugs, including avexitide for post-bariatric hypoglycemia, a condition where people who have had weight-loss surgery experience dangerously low blood sugar levels.
The company bought avexitide from Eiger BioPharmaceuticals last year.
(Reporting by Kamal Choudhury in Bengaluru; Editing by Leroy Leo)
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