- Amylyx Pharmaceuticals, a small biotech developing a promising experimental medicine for ALS, has raised $135 million from a group of venture capital firms to fund late-stage clinical testing and preparations for a potential launch should regulators in Canada and Europe approve the drug.
- Founded by two Brown University graduates eight years ago, Amylyx has pushed its therapy through a Phase 2 clinical trial with minimal funding, leaning on the support of groups like the ALS Association and a research center at Massachusetts General Hospital that's focused on the neurodegenerative disease.
- The $135 million Series C, which came from Viking Global Investors, Bain Capital and several other firms, is more than five times what Amylyx raised in two prior venture funding rounds in 2016 and 2020. Previous investors like Polaris Founders Capital and Belinda Termeer, widow of biotech pioneer Henri Termeer, also contributed funds.
Amylyx is on the cusp of accomplishing a rare feat in drug development. The Cambridge, Massachusetts-based biotech recently submitted for approval of its ALS drug in Canada and plans to do the same in Europe by the end of the year.
Few drugs are available for ALS, a devastating disease that damages nerve cells in the brain and spinal cord. In the U.S., treatment is largely limited to Radicava, a newer, modestly effective drug from Japan's Mitsubishi Tanabe Pharma, and riluzole, an older and now generic medicine.
Approval by either Canadian or European regulators of Amylyx's drug, called AMX0035, would be a major accomplishment for a company that was born out of conversations founders Josh Cohen and Justin Klee had while attending Brown in 2013. They raised a $5 million Series A round three years later and added another $20 million in a July 2020 Series B.
AMX0035 is a combination of two drugs though to protect neurons in the brain, both of which have previously been tested on their own in patients with ALS. Combining the two, Cohen and Klee thought, could slow the disease's progression.
A Phase 2 study, called CENTAUR, got underway in 2017 and by late 2019 had indicated treatment might slow the rate of patients' functional decline. Subsequent results, published in The New England Journal of Medicine in September 2020, showed participants given AMX0035 scored on average two points higher on a 48-point scale used by neurologists to assess ALS symptoms compared to those who received placebo.
Further analysis was even more encouraging, suggesting the drug was also helping study volunteers live longer.
The study was small, however, enrolling just 137 patients. And supporting evidence was more mixed, particularly around a blood test used by scientists to look for proteins associated with nerve degeneration.
Still, Amylyx concluded it had enough to take the drug to regulators to ask for an approval. In the U.S., though, the Food and Drug Administration asked the biotech to run another trial to confirm their findings, a decision that sparked backlash from the ALS patient community.
Criticism has grown following the FDA's controversial decision to approve Biogen's Alzheimer's drug Aduhelm on thin evidence it actually improves patients' lives.
"In the case of AMX0035 for ALS, there is evidence of clinical benefit and strong safety data but no biomarker data," wrote Calaneet Balas, the president and CEO of the ALS Association in a recent op-ed published in STAT. "In the case of Aduhelm for Alzheimer's, there is little evidence of clinical benefit, some safety concerns, and good biomarker data. Why won't the FDA approve AMX0035 based on clinical benefit when it approved Aduhelm based on biomarker data?"
For its part, Amylyx says it will continue to update the ALS community on its regulatory interactions with the FDA, as well as its plans for the Phase 3 trial asked for by the agency.
"We are having ongoing discussions with the U.S. FDA to discuss potential paths to advance AMX0035 through the clinical development process as quickly as possible," Cohen and Klee said in an emailed statement.
Note: This story has been updated with comment from Amylyx.