Phase 3 Trial of NurOwn Cell Therapy Fully Enrolls 200 Patients, BrainStorm Announces

BrainStorm Cell Therapeutics announced that the Phase 3 clinical trial of its cell therapy candidate NurOwn in 200 amyotrophic lateral sclerosis (ALS) patients is fully enrolled and treatment is underway.

The randomized, double-blind and multicenter trial (NCT03280056) is assessing the safety and effectiveness of three administrations of NurOwn into the spinal canal. Patients will be given either NurOwn or placebo intrathecal injections every two months.

“The BrainStorm team is very excited to reach this important milestone in the development and potential commercialization of NurOwn in ALS. We have been fully dedicated to this journey since we began clinical trial enrollment in October 2017,” Chaim Lebovits, the president and CEO of BrainStorm, said in a press release.

NurOwn is a cell therapy that that takes mesenchymal stem cells (MSCs) from patients (autologous MSCs), which are able to generate various cell types. These MSCs are then converted into a cell-type — called MSC-NTFs — that produce neurotrophic factors (molecules which promote nerve tissue growth and survival). The converted cells are then returned through a transplant to the patients, either through an injection into the muscle or the spinal canal, to promote nerve tissue growth and survival.

The study’s primary goals of NurOwn’s safety and effectiveness will be measured using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) and compared to the placebo group. A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory factors, and cytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) following treatment with NurOwn.

“To date, a significant percentage of the 200 enrolled patients have received three treatments and we expect that all trial participants will complete planned study visits and assessments by October 2020,” Lebovits said.

This study is being carried out at the University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; the University of Massachusetts Medical School; and the Mayo Clinic.

If successful, trial results will be used to support an FDA request for approval of NurOwn as an ALS treatment, BrainStorm said.

The use of autologous MSC-NTF cells to potentially treat ALS was given orphan drug status by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

“We are very pleased to have fully enrolled our Phase 3 randomized placebo-controlled clinical trial in ALS and look forward to reporting top-line results before the end of next year. This clinical milestone is a key inflection point in the development of NurOwn,” said Ralph Kern, the chief operating officer and chief medical officer of BrainStorm.

This study is financially supported by a grant and other funding from the California Institute for Regenerative Medicine (CIRM), as well as other types of investment.

“Based on BrainStorm Cell Therapeutics’ scientific and early phase clinical trial data, CIRM awarded them $15.9 million to advance their investigational therapy NurOwn into a Phase 3 trial,” said Maria Millan, MD, president and CEO of the CIRM.

“Our support helped bring this program to three leading California medical centers. We congratulate Brainstorm Cell Therapeutics in completing enrollment, an important milestone toward developing a much-needed treatment for ALS,” Millan added.