Amylyx Discontinues Investigative Therapy Program In Rare Neurological Disorder

Amylyx Pharmaceuticals Inc. (AMLX) on Wednesday said that it is discontinuing its AMX0035 program in adults living with progressive supranuclear palsy.

AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol. The decision to discontinue the program was made after it failed to show differences compared to the placebo on primary or secondary outcomes at week 24 in a mid-stage trial. The company will not initiate the late-stage portion of the program, it said.

Progressive Supranuclear Palsy (PSP) is a rare, progressive neurological disorder characterized by problems with balance, eye movements, speech, and swallowing, often leading to frequent and unpredictable falls. According to Amylyx, the disease affects about seven in 100,000 people worldwide.

On Stocktwits, retail sentiment around the AMLX stock jumped from ‘bullish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume remained at ‘high’ levels. Shares of the company are down by 2% in the pre-market session.

“While we are disappointed in these results, we believe these data will inform the PSP trial literature as well as deepen scientific understanding of this devastating disease,” said Camille L Bedrosian, Chief Medical Officer at Amylyx.

Co-CEOs of Amylyx, Joshua Cohen and Justin Klee, said that the company’s priority remains on the late-stage trial of Avexitide, with enrollment expected to be completed in 2025 and topline data anticipated in the first half of 2026.

Avexitide is an investigational, glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism.

The company is also continuing development of AMX0035 in Wolfram syndrome, a rare, inherited genetic disorder, they said. Amylyx continues to expect its cash runway to extend through the end of 2026.

AMLX stock is up by 142% this year and has jumped 330% over the past 12 months. 

Read also: Ford Issues Nearly 356,000 Vehicles Recall In U.S., But Retail Investors Spot Emerging Catalysts

For updates and corrections, email newsroom[at]stocktwits[dot]com.