On July 17th, our partner Northeast ALS Consortium (NEALS) hosted a webinar, “NurOwn® Clinical Development Program,” which comprehensively explained past phase II results, the upcoming phase III study and Israeli Hospital Exemption Program. To give a full picture of the company’s past progress and future endeavors, the webinar was led by leadership at Brainstorm Cell Therapeutics, including President and CEO Chaim Lebovits and Chief Operating and Medical Officer Dr. Ralph Kern. Clinicians involved in the upcoming phase III trial also spoke, including Drs. Robert Brown, Anthony Windebank and Merit Cudkowicz. The Association has partnered with NEALS for over eight years and we are happy to collaborate to disseminate the most accurate, up-to-date information regarding ALS clinical trials.

Phase II Trial Summary

To start the presentation, Drs. Ralph Kern and Robert Brown gave a brief overview of the phase II Brainstorm clinical trial that was reported out to the field last July. It was a randomized, double blind, placebo-controlled trial using a single dose of NurOwn® administrated intrathecally (via spinal cord). The main outcome tested was safety, while efficacy was tested secondarily. The phase III trial did not enroll enough patients to prove efficacy. The study met its primary endpoint, since NurOwn® was safe and well tolerated. For a brief overview of this study, read our blog post here. Information from Brainstorm is found here.

To add to this information, the phase II study also explored potential ALS biomarkers (i.e. a method to track whether NurOwn® stem cells reach its target and are working as designed) in participant cerebral spinal fluid (CSF). They reported an increase in neurotrophic factors (i.e. proteins that help maintain the viability of neurons) and a significant decrease in inflammatory biomarkers in patients’ CSF who received NurOwn® compared to patients on placebo. These results support NurOwn®’s designed mechanism of action.

Phase III Update

The planned phase III trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of repeated administration of NurOwn® in people with ALS. The trial will test 200 people with ALS with a 1:1 randomization, so 100 participants will receive NurOwn® and 100 participants will receive placebo. The study is designed to run 28 weeks following the first treatment. In contrast to the single dose phase II trial, the phase III study will test three repeat doses of NurOwn® over a 16 week period. For major trial inclusion and exclusion criteria, please refer to webinar slides 23-24.

The upcoming phase III trial is currently in planning and The ALS Association will update the community once a start enrollment date is announced.

The trial will run in the U.S. at six approved medical centers. Five out of the six sites have been announced. The known sites with lead investigators are the following:

• University of Massachusetts Medical School, Worcester, Mass., Dr. Robert Brown
• Massachusetts General Hospital, Boston, Mass., Dr. Merit Cudkowicz
• California Pacific Medical Center, San Francisco, Calif., Dr. Robert Miller
• University of California Irvine, Los Angeles, Calif., Dr. Namita Goyal
• Mayo Clinic, Rochester, Minn., Dr. Anthony Windebank

For more information about the upcoming phase III trial, register for news alerts on the Brainstorm website here. Both the NEALS clinical trial site and clinical trials.gov will also be updated as more information about the Brainstorm trial is announced.

Hospital Exemption Program in Israel

Brainstorm is working with the Tel Aviv Sourasky Medical Center in Israel to explore the possibility of making NurOwn® available to people with ALS through a Hospital Exemption Program. More information from the company is found here and here. To explore more information regarding patient access to NurOwn® click here.

The program is not yet approved, but leadership is hopeful for approval in the near future. They will announce an update once they receive a decision from the Israeli Ministry of Health.

Other important news:

On July 21st, Brainstorm announced that the California Institute for Regenerative Medicine (CIRM) has awarded Brainstorm a grant of $16 million to support their upcoming NurOwn® trial. This is another important step to move the trial forward as quickly as possible.

Watch the webinar here.