Amylyx Pharmaceuticals showed in a clinical trial that its amyotrophic lateral sclerosis (ALS) drug slowed the muscle-wasting disease’s progression. The drug is now being readied for a larger, pivotal study aiming to replicate those results, and the company has raised $135 million in new funding to support the research.

Cambridge, Massachusetts-based Amylyx had initially hoped the positive Phase 2 data would be enough to seek accelerated FDA approval. That pathway can get a drug to patients more quickly based on less evidence than is required of a standard drug review, but still requires a company to confirm patient benefit with additional post-marketing testing. The Series C round of funding announced Tuesday comes three months after Cambridge, Massachusetts-based Amylyx said the FDA would require data from a larger, placebo-controlled study before it could consider an application from the company.

Amylyx’s drug, AMX0035, is designed to interfere with a cellular pathway that leads to the death of nerve cells. The drug is a proprietary combination of taurursodiol and sodium phenylbutyrate, made in a powder formulation that must be dissolved in water and taken orally, twice daily.

The placebo-controlled Phase 2 study enrolled 137 ALS patients. In preliminary results released in late 2019, treatment with the drug for 24 weeks led to a statistically significant slowing of ALS progression, which was the main study goal. Patients were assessed according to a scale that measures how ALS patients perform doing things such as walking, dressing, eating, speaking, and breathing.

According to full Phase 2 clinical trial results published last September in the New England Journal of Medicine, patients treated with the Amylyx drug scored, on average, 2.32 points higher on the assessment scale compared to those in the placebo group. On secondary clinical trial goals assessing muscle strength, breathing, and hospitalizations, patients treated who received AMX0035 showed numerical improvement compared to those given a placebo.

Based on the Phase 2 results, Amylyx filed for regulatory approval of its drug in Canada in June. The company said it plans to submit the drug to the European Medicines Agency by the end of this year. The planned Phase 3 study to support an FDA submission is slated to begin later this quarter. That clinical trial will be conducted in the U.S. and Europe.

Amylyx said that the new round of capital will be used to support clinical development and potential launch of its ALS drug. The cash will also support continued development of AMX0035 in other diseases. An Alzheimer’s disease program is in Phase 2 testing. A program evaluating the drug in the rare Wolfram syndrome is in preclinical development.

Viking Global Investors led Amylyx’s Series C financing. Other investors in the round include Bain Capital Life Sciences, Perceptive Advisors, Rock Springs Capital, Woodline Partners, Marshall Wace, Tybourne Capital Management, Verition Fund Management, aMoon Fund, and Falcon Edge. Earlier investors Morningside Ventures, 683 Capital Management, Belinda Termeer, and Polaris Founders Capital also participated in the financing.

“We have a clear mission at Amylyx: to develop new medications for ALS and other progressive neurodegenerative diseases,” James Frates, chief financial officer of Amylyx said in a prepared statement. “We are eager to work with Viking and our syndicate of experienced biotechnology investors as we execute our plan to hopefully bring AMX0035 to people living with ALS as soon as possible.”

Photo: Matthew Horwood, Getty Images