- Brainstorm Cell Therapeutics now has a tougher decision to make regarding its experimental treatment for ALS, announcing Monday that the Food and Drug Administration does not believe there is enough evidence to support asking for approval.
- Brainstorm, a New-York based company trying to treat neurodegenerative illnesses through the use of adult stem cells, reported in November that a late-stage study testing its treatment in patients with rapidly progressing ALS, or amyotrophic lateral sclerosis, had failed. Brainstorm blamed the failure on better-than-expected results in the study's placebo group, and argued there were still enough positive findings to warrant the treatment moving forward.
- In spite of the FDA's feedback, Brainstorm has not yet decided whether it will submit an approval application anyway. CEO Chaim Lebovits said in a statement that his company will first consult with regulatory advisors, statisticians, principal investigators, as well as ALS experts and advocacy groups to evaluate the pros and cons of a submission. According to Brainstorm, the FDA noted that its conclusion does not prevent the company from filing an application.
ALS, better known to some as Lou Gehrig's disease, is a debilitating illness characterized by weakness, fatigue, loss of muscle control and, eventually, paralysis and difficulty breathing. Estimates hold that of the roughly 15,000 to 30,000 ALS patients living in the U.S. at a given time, just 20% will live longer than five years following a diagnosis.
Like many neurodegenerative diseases, ALS has proven exceptionally challenging to address. Treatment is currently limited to two medicines, both of which have their limitations. Companies like Cytokinetics and Biogen have tried bringing new drugs to market, but suffered major setbacks in late-stage testing.
Brainstorm's treatment, which is made using a cell technology platform called NurOwn, looked as though it had joined that list when the company reported disappointing data in November. While the late-stage study found a 35% response rate among NurOwn-treated patients, that wasn't significantly better than the 28% of placebo-treated patients who also experienced a response. Per the study design, a response was defined as a 1.25-point improvement each month on a widely-used scale for measuring function in ALS patients.
The negative readout didn't deter Brainstorm, however. Though the placebo group did better than anticipated, Brainstorm's Chief Medical Officer Ralph Kern said that could have been the result of the trial enrolling an unusually large number of patients with severe ALS — a group that is often less responsive to treatment and harder to measure responses for.
Moreover, the response rate in the NurOwn-treated group lined up perfectly with the 35% Brainstorm expected to see when designing the trial. Response rates were also more favorable for Brainstorm's treatment when looking at a subset of patients with early ALS, the company said.
"We do feel, all of us in Brainstorm reviewing the data ... that this clearly has a pathway — probably more than one pathway — for an approval without doing another trial," Lebovits said on a call with investors following the release of the data.
Yet, the FDA's feedback suggests Brainstorm might not have many options other than to generate more data.
On their end, investors don't appear confident that there's a path forward for Brainstorm's treatment. Company shares were down 32% Monday morning to trade at around $2.20.
Brainstorm's NurOwn takes stem cells from a patient's bone marrow, engineers them to secrete molecules that help protect and grow neurons, and then injects them back into the patient in the hope that they can slow or stop neurodegeneration.
Lebovits noted in a statement Monday that his company remains "well funded," with a current position of over $40 million in cash. He said Brainstorm "will soon announce our next flagship product and program for other diseases with unmet needs."