- Alexion Pharmaceuticals, now a unit of British drugmaker AstraZeneca, on Friday said it will discontinue clinical testing of its rare disease drug Ultomiris as a treatment for ALS due to lack of efficacy.
- The decision followed the recommendation of an independent data monitoring committee overseeing the Phase 3 trial after they reviewed data from an interim study checkpoint. The committee didn’t identify any new safety findings with Ultomiris, AstraZeneca said.
- The discontinuation is another setback in the search for more treatments for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, which is diagnosed in about 5,000 people in the U.S. each year, according to the CDC. An executive at Alexion said in a statement the company is "disappointed by this outcome and what it means for patients with this devastating disease."
While several ALS drugs are on the market, there's still a need for better treatment options to slow or reverse the neurological disease's steady progression. ALS advocates have pressured the FDA to take a more flexible approach to speed development and availability of potential therapies, as patient groups have in other fields like cancer.
Those calls have grown more intense following the FDA's controversial approval of Aduhelm, a Biogen drug for Alzheimer's disease, based on a biological disease marker rather than definitive proof of efficacy.
In ALS, by contrast, critics argue the FDA is moving more conservatively.
Earlier this year, for instance, Amylyx Pharmaceuticals reported that the FDA asked for data from an additional placebo-controlled study of its closely watched ALS drug before the company could submit it for approval. Frustrated by the agency's insistence for more data, patient groups have called on the FDA to approve Amylyx’s drug as soon as possible. In a small study last year, patients taking the treatment appeared to do modestly better at functions such as speaking, walking and writing, all of which are affected by ALS.
A drug developed by Ionis Pharmaceuticals and Biogen, meanwhile, is in its final stages of human testing with results expected in the near future. Known as tofersen, the drug is specifically designed for patients with certain genetic abnormalities that can cause ALS. If the late-stage study of about 180 adults with inherited ALS shows positive data, the companies plan to ask for FDA approval.
Alexion began its Phase 3 trial for Ultomiris in January 2020, 11 months before the company was acquired by AstraZeneca. In Friday's release, the company said patients enrolled in the trial will stop taking the medication and complete any necessary follow-up evaluations.