A month after filing for Canadian approval for its lead ALS candidate, Amylyx Pharmaceuticals has picked up $135 million to bankroll a potential commercial launch and support a pipeline of other candidates for neurodegenerative diseases, including the one causing a stir this summer: Alzheimer’s disease.
The Cambridge, Massachusetts-based biotech raked in the oversubscribed series C Tuesday to support the clinical development and launch plans, pending a Canadian green light for the amyotrophic lateral sclerosis (ALS) treatment. The novel combination of compounds called AMX0035 was found to slow the progression of ALS in a phase 2/3 study presented last September.
Viking Global Investors led Tuesday’s financing round, with participation from Bain Capital Life Sciences, Perceptive Advisors and about a dozen other backers.
AMX0035 combines two orphan drugs—taurursodiol and Buphenyl—into an oral medication that works to protect brain cells’ energy-producing mitochondria and bolster the endoplasmic reticulum, ensuring constructed proteins fold and operate correctly.
The therapy was put up for a Health Canada nod last month and Amylyx intends to submit for a marketing approval with the European Medicines Agency by the end of this year. FDA regulatory updates are to come, the company said.
Amylyx also presented a trial design for an international phase 3 study of the drug in May.
The biotech has beefed up its board and leadership in recent months to support a commercial roll-out, including the additions of Amicus Therapeutics CFO Daphne Quimi and Paul Fonteyne, the former president and CEO of Boehringer Ingelheim’s U.S. operations. Amylyx also hired Chris Aiello in April to lead operations in Canada as general manager, after leading the rare disease and rare blood disorder units at Sanofi Genzyme’s Canadian outfit.
Amylyx also landed a new CFO in January with a direct connection to the ALS movement, picking up former Alkermes CFO James Frates, who is the cousin of the driving force behind the Ice Bucket Challenge. Funds raised from the viral ALS awareness social media movement helped support Amylyx’s own trial.
The 2020 Fierce 15 winner raised $30 million last summer for AMX0035 and a midstage trial for a potential Alzheimer’s treatment. Last November, Amylyx said the last patient had completed the phase 2 trial, dubbed Pegasus, with top-line data due in the first half of this year. The company’s website now says the read out will happen sometime this year.
The company could benefit from the revived interest in Alzheimer’s following the FDA’s controversial approval of Biogen’s Aduhelm. The decision has been criticized by federal, congressional, industry and hospital system stakeholders alike, but competing companies have seen a rise in investment interest.