Grant Awarded to Advance ALS Drug Trial


Experimental drug showed strong efficacy in numerous cellular animal models of amyotrophic lateral sclerosis (ALS).

The ALS Association and ALS Finding a Cure are awarding a $2.96 million grant to support a phase 2 trial for Amylyx Pharmaceuticals’ investigational drug AMX0035, for the treatment of amyotrophic lateral sclerosis (ALS).

The grant is part of the ALS Accelerated Therapeutics Initiative (ALS ACT), which was undertaken by the 2 organizations in 2014, according to a press release by The ALS Association.

“To accelerate developing new treatments for ALS, the 2 foundations issued a global, competitive call for grant proposals for therapies that had a strong scientific rationale and where a biomarker of a drug effect could be measured,” said Merit Cudkowicz, MD, chief medical officer of ALS Finding a Cure, chairman of Neurology at Massachusetts General Hospital. “AMX0035 was chosen as one of the top new therapeutic candidates to bring forward for ALS.”

AMX0035 is a combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each of the compounds exhibited strong efficacy in numerous cellular animal models of ALS.

Additionally, PB and TUDCA have been tested in clinical trials of ALS individually, showing safety and tolerability, and preliminary signs of efficacy. Amylyx has demonstrated a synergistic effect between PB and TUDCA, which suggests that the combination may be more effective in ALS patients compared to either compound being used alone, according to the press release.

The trial is expected to launch later this year to test the tolerability and safety of AMX0035, as well as the functional outcomes. A major part of the trial will be an analysis of biomarkers of inflammation, cell function, and neuronal damage.

Furthermore, a new measure of muscle strength that has been tightly correlated with ALS disease progression will also be included.

“I am thrilled to evaluate AMX0035 in this upcoming study,” said principal study investigator Sabrina Paganoni, MD, PhD. “We also expect the biomarker and muscle strength analyses to advance our understanding of ALS and improve our ability to measure and target this disease.”

ALS is a neurodegenerative disease that affects the nerve cells in the brain and spinal cord, resulting in paralysis and death. At this time, there is no known cure, and Rilutek is the only FDA-approved drug that modestly extends survival.

Veterans are twice as likely to develop ALS as the general population, according to the press release.

“The ALS Association is pleased to support this important clinical trial designed to incorporate biomarkers to track changes in the disease process,” said Lucie Bruijn, PhD, MBA, chief scientist for The ALS Association. “Results from this trial will significantly contribute to biomarker development and inform us about the value of using this combined therapeutic approach.”

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