Amylyx Secures Funding for Therapy Candidate AMX0035

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by Vanda Pinto, PhD |

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Amylyx Pharmaceuticals has raised $135 million to advance the clinical development and the potential launch of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS).

The Series C financing round, led by Viking Global Investors, also will be used to support further research and development of AMX0035 in other neurodegenerative diseases.

“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 press release.

“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,” he added.

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AMX0035 is an oral, fixed-dose combination of two small molecules, tauroursodeoxycholic acid and sodium phenylbutyrate, that have been used in the clinic and are known to be safe and well-tolerated.

The treatment is expected to prevent nerve cell death by blocking stress signals in two cellular structures: mitochondria and the endoplasmic reticulum. Mitochondria are organelles responsible for energy production and the endoplasmic reticulum is involved in protein production, modification, and transport.

Amylyx recently requested approval of AMX0035 as a treatment for ALS in Canada, and is planning to file a similar application with the European Medicines Agency by the end of the year.

The submission was grounded on positive results from the CENTAUR Phase 2/3 trial (NCT03127514), which investigated the safety and efficacy of AMX0035 in 137 adults recently diagnosed with sporadic or familial ALS and with rapidly progressing disease.

Participants were assigned randomly to receive either AMX0035 or a placebo  twice daily for 24 weeks (about six months), after which about 92% opted into an open-label extension part in which all are receiving the therapy for up to 30 months (about two-and-a-half years).

Findings from the trial showed that AMX0035 significantly slowed patients’ functional decline and reduced their risk of death — as well as the combined risk of death, tracheostomy, permanent assisted ventilation, or first hospitalization — by 44%. The therapy also was considered generally safe and well-tolerated.

Despite the promising results, and a petition asking for U.S. approval as quickly as possible, the U.S. Food and Drug Administration (FDA) requested data from an additional controlled clinical trial before considering AMX0035 for approval.

To that end, Amylyx plans to launch a global Phase 3 trial in the coming months to test AMX0035 against a placebo in up to 600 ALS patients whose symptoms began in the past two years.

Each participant will be assigned randomly to either AMX0035 or to a placebo for about 11 months, after which patients may continue or start using the therapy. Results from the PHOENIX trial, to be conducted at 55 sites across the U.S. and Europe, are expected to support a regulatory application with the FDA.

Also participating in the Series C financing round are: Bain Capital Life Sciences; Perceptive Advisors; Rock Springs Capital; Woodline Partners; Marshall Wace; Tybourne Capital Management; Verition Fund Management; aMoon Fund; and Falcon Edge.

Existing investors in the financing were Morningside Ventures, 683 Capital Management, Belinda Termeer and Polaris Founders Capital.