Despite failed trial, Brainstorm asks FDA to approve ALS drug

Dive Brief:

• Brainstorm Cell Therapeutics, a New York-based drugmaker, plans to ask the Food and Drug Administration to approve an experimental treatment for amyotrophic lateral sclerosis that failed a key clinical trial in late 2020.
• In a statement Monday, Brainstorm’s CEO Chaim Lebovits said the decision was largely based on additional analyses of the trial, which uncovered an error in the statistical methods used to assess results for therapy, called NurOwn.
• Brainstorm’s move comes less than a month after the FDA agreed to review another ALS drug with a mixed track record. Developed by Biogen, the drug, known as tofersen, was found in a late-stage study to not be significantly better than a placebo at slowing the fatal neurodegenerative disease. Despite those results, Biogen requested approval based on the apparent effect its drug has on a protein that research suggests is a likely indicator of neurological damage.

Dive Insight:

Almost two years ago, Brainstorm revealed NurOwn, which is a type of engineered stem cell therapy, did not succeed in a placebo-controlled study meant to show its merit as an ALS treatment. While the NurOwn arm performed as Brainstorm had anticipated, the response rate seen in the placebo group was much higher than expected, ultimately causing the drug to miss the trial’s main goal.

But Brainstorm now claims that, upon further evaluation and feedback from “key opinion leaders,” it believes there’s enough evidence to support an approval.

For example, the company noted that a correction was made to published data from its late-stage study. The correction, Brainstorm claimed, meant NurOwn succeeded on a secondary goal — the average change on a rating scale that measures the severity of ALS symptoms — in a pre-specified subgroup of participants.

In asking for approval based on a failed study, Brainstorm may present yet another challenge to the FDA office that evaluates new therapies for brain and nervous system disorders.

That office, headed by agency veteran Billy Dunn, has been in the spotlight in recent years. Several times, it has reviewed high-profile applications that tested how flexible it could be toward drugs that haven’t been definitively proven effective, but target devastating diseases with few other treatment options. Its approvals of a Duchenne muscular dystrophy drug in 2016 and, more recently, Biogen’s medicine for Alzheimer’s disease have been especially controversial.

Patients and advocacy groups have pressed the agency to be as flexible with potential therapies for ALS, given the dire nature of the disease, which is often fatal within two to five years after diagnosis. There are currently two drugs approved by the FDA to treat ALS, but the benefits they offer on function and survival are considered modest.

More options may become available soon, however. The FDA is set to make an approval decision by Sept. 29 on an ALS drug called AMX0035, which was developed by Massachusetts-based Amylyx Pharmaceuticals and showed positive effects on function and survival in late-stage testing.

And by Jan. 25, the agency should have a verdict on tofersen. Biogen has requested an accelerated approval that would hinge on the drug’s effects on “neurofilament light chain,” a protein that is of increasing interest in ALS research. According to Biogen, patients treated with tofersen experienced substantial reductions in the amount of this protein in their blood plasma.

Brainstorm announced these updates alongside a second quarter earnings report that showed the company had a little over $12 million in cash, cash equivalents and short-term bank deposits as of June 30. Brainstorm expects that its existing cash, together with other funding activities and sales of its common stock, will be enough to fund operations for at least the next year.

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