RARE Daily

Amylyx Raises $135 Million to Support Development and Potential Launch of ALS Treatment

July 21, 2021

Amylyx Pharmaceuticals closed an oversubscribed $135 million series C financing to support the clinical development and potential launch plans for lead product candidate AMX0035, for the treatment of amyotrophic lateral sclerosis, and advance research and development in other neurodegenerative diseases.

Photo: James Frates, chief financial officer of Amylyx Pharmaceuticals

Viking Global Investors led the financing with participation from 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 Morningside Ventures, 683 Capital Management, Belinda Termeer, and Polaris Founders Capital also participated in the financing.

Amyotrophic lateral sclerosis (ALS), also referred to as “Lou Gehrig’s disease,” is a devastating progressive, and fatal motor neuron disease and affects approximately 1 out of 100,000 people. The average life expectancy of a person with ALS is approximately two to five years, and there is currently no cure for the disease.

AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicoltaurine), which were combined in a co-formulation to reduce neuronal death and dysfunction. AMX0035 was specifically co-formulated and manufactured by Amylyx to ensure proper absorption, exposure, and quality. AMX0035 targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

“We have a clear mission at Amylyx: to develop new medications for ALS and other progressive neurodegenerative diseases,” said James Frates, chief financial officer of Amylyx Pharmaceuticals. “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.”

As previously reported, Amylyx filed a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in June. Amylyx intends to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by the end of 2021. Amylyx will continue to update the global ALS community on global regulatory plans including with the U.S. Food and Drug Administration and plans to initiate a phase 3 clinical trial. Data on AMX0035 in people with ALS has been published in the New England Journal of Medicine and Muscle and Nerve.

Author: Rare Daily Staff

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