Cambridge firm Amylyx pulling its ALS drug off the market
Biotech plans to cut 70% of staff after clinical trial failure
Four weeks after reporting its controversial and costly drug for amyotrophic lateral sclerosis failed to provide any benefit to patients in a large clinical trial, Cambridge biotech Amylyx Pharmaceuticals said Thursday it was pulling the medicine from the market in the US and Canada.
The firm also said it plans to lay off 70 percent of its workforce to preserve its cash into 2026. Amylyx had 384 fulltime employees as of December 31. The layoffs will reduce the workforce to a little more than 100, said a company spokesperson.
The drug, which is called Relyvrio in the United States and Albrioza in Canada, “will no longer be available for new patients as of today,” Amylyx said in a news release. Patients who are currently on the medicine and want to continue taking it will be able to get it for free.
“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases,” Joshua Cohen and Justin Klee, cofounders and cochief executives of Amylyx, said in a statement.
Relyvrio cost about $163,000 a year per patient. It generated nearly $381 million in revenue last year for the 11year-old company.
While the decision to withdraw it from the market was commended by experts, it is a major blow to ALS patients, their families, and doctors, given the dearth of effective treatments for the fatal disease. It also caps a years-long drama during which the prospects of Amylyx’s drug fluctuated wildly, until the company made a major announcement last month.
In a stunning reversal on March 8, Amylyx released the final results of a 48-week, late-stage confirmatory study involving 664 people with ALS at more than 70 trial sites in the United States and Europe. Amylyx’s drug failed to outperform a placebo on an ALS functional rating scale that measured patients’ ability to perform daily living tasks, breathe, walk, speak, and swal
low.
Cohen and Klee said then that they were “surprised and deeply disappointed” and would consider withdrawing the drug from the market.
The drug was approved in September 2022, even though an advisory panel to the Food and Drug Administration initially recommended the agency reject it, citing weak scientific evidence of its benefits.
That panel later reversed itself amid wrenching pleas from patients and families affected by the paralyzing and fatal neurological condition, and after Amylyx presented more clinical data that it said showed the medicine extended patients’ lives.
Cohen and Klee made an unusual pledge at the time to pull Relyvrio from the market if a longer, larger study showed that the drug wasn’t effective.
About 3,900 ALS patients were taking the medicine in the United States at the end of the third quarter of last year, according to the most recent numbers from Amylyx.
Amylyx’s chief medical officer, Dr. Camille L. Bedrosian, said the company was continuing to work on another experimental treatment for ALS as well as a drug to treat Wolfram syndrome, a rare genetic disorder.
ALS — also known as Lou Gehrig’s disease — is diagnosed in an estimated 6,000 people a year in the US, according to UMass Chan Medical School. The rare disease affects nerve cells in the brain and spinal cord, gradually robbing patients of the ability to speak, eat, and, finally, breathe. Despite the best efforts of doctors and researchers, most patients die within three to five years of diagnosis.
An estimated 31,000 Americans were living with ALS as of 2017, according to the Centers for Disease Control and Prevention.
Dr. Merit Cudkowicz, chief of neurology at Massachusetts General Hospital and co-principal investigator of the small intermediate-stage study of 137 patients that led to the approval, said Amylyx made the right decision by pulling the drug.
Given the negative outcome of the confirmatory trial, she said in an email, “Physicians aren’t prescribing it anymore anyway and [it’s] unlikely insurance would cover [it] so [it] doesn’t change things day to day for people with ALS or clinicians.”
The nonprofit ALS association also commended Amylyx for pulling Relyvrio from the market.
The patient advocacy group said the FDA acted appropriately by approving the drug based on a mid-stage clinical trial and letting patients take it while a larger confirmatory trial took place.
“We believe the example of Relyvrio shows how the system can work,” the association said in a statement. “[The] FDA approved Relyvrio based on solid safety data and positive efficacy data from a phase 2 trial on function and survival. Safe and potentially effective treatments can be made accessible rapidly until further research can confirm their efficacy.”
Since 1995, only two drugs have been approved to treat ALS, and neither works very well. Scores of clinical trials of potential treatments have failed over the past decade.
Even though ALS has stymied multiple drug companies, the association offered a glimmer of hope, saying there are more than 40 potential treatments in the pipeline.
Cudkowicz was also hopeful. “There is more science and exciting new targets and approaches,” she said. “The ALS field is resilient and thinking about what is next.”