BrainStorm Cell Therapeutics Announces Positive Interim Safety Analysis
On Thursday, BrainStorm Cell Therapeutics announced that its Phase 3 trial for NurOwn in patients with ALS will be able to continue with no changes.
ALS, a disease that caught attention thanks to the Ice Bucket Challenge, is now much closer to a finding treatment, which showed positive improvement in previous studies.
On Thursday (August 23), BrainStorm Cell Therapeutics (NASDAQ:BCLI) announced its Phase 3 trial for NurOwn in patients with ALS, will be able to continue with no changes.
This decision was made based off of the successful completion of the prespecified interim analysis, done by the Data Safety Monitoring Board, which concluded there was no significant safety concerns. NurOwn is a cell therapy platform developing mesenchymal stem cells (MSCs) to treat human diseases.
The Phase 2a study results were done in Israel for NurOwn and ALS resulted in meaningful improvements in the rate of disease progression for the six months following treatment. In the Phase ½ study, 82 percent of participants showed improvements after six months following the trial in regards to ALS Functional Rating Scale (ALSFRS).
BrainStorm Therapeutics is overseeing another Phase 3 trial dose of NurOwn for ALS patients at six US locations. The trial is backed by a California Institute for Regenerative Medicine grant, which is expected to enroll 200 ALS participants. Currently, 82 patients have been enrolled and 61 are active in the trial.
The primary endpoint for the trial measures the ALSFRS 28 weeks following when participants receive the first of three NurOwn intrathecal treatments.
Already in the trial, most active participants have received two of the treatments, whereas other have received all three. BrainStorm expects to have 110 participants signed up by January 2019 with the remainder finished by mid 2019.
This would put the company in a position to file a biologics license application (BLA) for the US Food and Drug Administration (FDA) approval following the completion of all clinical trial evaluations.
The MSCs are multipotent precursor cells most commonly obtained from bone marrow, adipocytes or placenta. BrainStorm grows the cells and converts them into neurotrophic factors, which support the survival of neurons and treat neurodegenerative diseases.
While the ALS trial for NurOwn is the only clinical trial, the company is also working on preclinical development with progressive multiple sclerosis, autism, huntington’s disease and others.
ALS is a neurodegenerative disease which progressively degenerates motor neurons, eventually resulting in death. About 20,000 people have the disease in the US. The average life expectancy of two-to-five years after being diagnosed, according to the ALS Association. While there are only three approved drugs to treat the disease, there is no cure.
These targets align with BrainStorm’s focus of developing stem cell therapies derived from autologous bone marrow cells to treat neurodegenerative diseases. The NurOwn technology was developed by Ramot while the worldwide licensing agreement to develop and commercialize is held by BrainStorm.
Following Thursday’s announcement, BrainStorm’s share price increased 1.24 percent to US$4.09.
TipRanks shows Maxim Group analyst Jason McCarthy reiterated a “Buy” rating for the company on Thursday with a US$9.00 price target.
Investors can look forward to hearing about the company meeting its predicted 110 participants in the trial in January 2019, and could hear about the BLA also within the year.
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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.