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BCLI: Positive Interim Safety Analysis for Phase 3 Trial…

By David Bautz, PhD



Business Update

Positive Interim Safety Analysis for Phase 3 Trial

On August 23, 2018, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) announced a positive interim safety analysis by the Data Safety Monitoring Board (DSMB) for the Phase 3 trial of NurOwn® in amyotrophic lateral sclerosis (ALS). There were no significant safety issues and the DSMB recommended that the trial continue as planned. This is an important milestone for the company as it is the first trial to use repeated injections of NurOwn®, thus it is reassuring to see that there have been no safety issues thus far with repeated injections.

Thus far, a total of 82 subjects have been enrolled in the study and 61 of them are currently active. A large number of subjects have received two injections of NurOwn® with a smaller subset having already completed all three injections. At the current pace of enrollment, we believe a total of 110 subjects will be enrolled by January 2019 and that full enrollment of approximately 200 subjects in the study will be completed by mid-2019. With this timeframe, we estimate topline data would be reported in the first half of 2020.

The Phase 3 trial is taking place at six leading U.S. Medical centers. It is a randomized, double blind, placebo controlled, multi-dose trial that is expected to enroll approximately 200 patients randomized 1:1 to NurOwn® or placebo. Cells will be extracted from each patient one time prior to treatment, with all administrations of NurOwn® derived from the same extraction of cells thanks to a cryopreservation process developed for long-term storage of mesenchymal stem cells (MSC). As in previous studies, there will be a 3-month run-in period prior to the first treatment with two additional NurOwn® treatments occurring two and four months following the first treatment. The primary outcome of the study is the ALSFRS-R score responder analysis.

The company is focusing the trial on faster-progressing ALS patients since these patients demonstrated superior outcomes in the Phase 2 trial of NurOwn®. According to the company, approximately 50-60% of ALS patients could be considered “fast progressors”, defined as those who lose at least one point per month in the ALSFRS-R score, thus there are a large group of patients that could potentially be included in the trial.

The company has contracted with City of Hope’s Center for Biomedicine and Genetics to produce clinical supplies of NurOwn® for all of the centers participating in the Phase 3 trial. Recently, the company announced that the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute will serve as a second manufacturing facility. Dana Farber was previously used as a manufacturing facility of NurOwn® in the Phase 2 trial. In addition to supplying NurOwn® for the ongoing Phase 3 trial, we believe the Dana Farber facility will likely be used to manufacture NurOwn® for any additional clinical indications the company decides to pursue.


The positive interim safety analysis for the Phase 3 trial of NurOwn® in ALS is an important milestone for the company as it indicates that there will likely not be any issues with dosing patients’ multiple times. For a disease such as ALS, it was always thought that multiple treatments would be necessary, thus it is reassuring to see that repeat administrations of NurOwn® is not associated with any unforeseen safety issues. The trial appears to be enrolling at a good pace, and with enrollment projected to be completed by mid-2019 we estimate that topline data would be reported in the first half of 2020. As a reminder, the company has sufficient capital to complete the trial through the remaining portion of the grants from the California Institute for Regenerative Medicine (CIRM) and the Israel Innovation Authority along with the company’s current cash position. Our probability adjusted discounted cash flow analysis based only on the use of NurOwn® in ALS leads to a current valuation of $15.

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