New Israeli Production Site for NurOwn Certified for Quality

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by Marta Figueiredo PhD |

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BrainStorm Cell Therapeutics announced that a new cleanroom facility for manufacturing NurOwn, its experimental cell-based therapy for amyotrophic lateral sclerosis (ALS), has received good manufacturing practice (GMP) certification from the Israel Ministry of Health.

GMPs are standards set to ensure that batches of a medicine are produced with consistent high quality.

This means that the facility — comprising three state-of-the-art cleanrooms leased at the Tel Aviv Sourasky Medical Center’s Institute for Advanced Cellular Therapies — is compliant with Israeli GMPs, which are aligned with those of the European Union.

“The GMP certification of these additional cleanrooms means that we now have more than doubled the capacity to supply NurOwn to ALS patients across Israel and Europe,” Chaim Lebovits, BrainStorm’s CEO, said in a press release.

“This is critical, as ALS is a truly debilitating disease and patients are in urgent need of new therapies,” Lebovits added. “We remain committed to addressing this unmet need and continue to discuss our clinical data with leading experts as we assess potentially regulatory pathways for NurOwn in various jurisdictions.”

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In October 2020, Catalent Biologics agreed to manufacture NurOwn under current GMPs at its new cell therapy manufacturing facility in Houston, supporting the therapy’s commercial supply in case of regulatory approval.

NurOwn involves a first step in which mesenchymal stem cells (MSCs) — stem cells that can generate a variety of other cells — are collected from a patient’s own bone marrow.

MSCs are then grown and matured, under patented conditions, into cells that produce high levels of neurotrophic factors (NTF), molecules that promote nerve cell growth and survival.

Finally, the mature cells, called MSC-NTF cells, are injected into the patient’s spinal canal to reach the brain and spinal cord and exert their neuroprotective effects. Using a patient’s own cells minimizes the risk of an immune reaction, as might occur with cells from a donor.

As such, NurOwn is designed to promote and support nerve cell repair, potentially slowing or preventing the progression of ALS and other neurodegenerative diseases.

NurOwn was given orphan drug designation in both the U.S. and European Union, and fast track designation in the U.S. for the treatment of ALS; all are meant to speed its clinical development and regulatory review.

Promising Phase 2 results in people with fast-progressing ALS prompted the launch of a Phase 3 trial (NCT03280056) to confirm NurOwn’s benefits in a larger patient population.

The trial evaluated the therapy’s safety and effectiveness in 189 people with rapidly progressing ALS, who were randomly assigned to a total of three spinal canal injections, given every other month, of either NurOwn or a placebo.

While NurOwn failed to significantly slow disease progression relative to placebo in the overall patient population, the experimental therapy was found to promote clinically meaningful responses in a pre-specified group of participants with less advanced disease.

While BrainStorm and regulatory agencies discuss possible next steps in NurOwn’s development, the company opened an early access program making NurOwn available to ALS patients who completed the Phase 3 trial and met certain eligibility criteria.

Based on the trial’s findings, those at earlier stages of the disease will be the first to receive the treatment.

“We are proud to be the first company to have been granted GMP certificate at the Sourasky Hospital’s Institute for Advanced Cellular Therapies, that hosts a newly built state-of-the-art cleanroom suites to advance our proprietary cellular technology platform in ALS and other neurodegenerative diseases,” Lebovits said.

NurOwn is in clinical trials as a potential treatment for multiple sclerosis, and in preclinical studies for two other neurodegenerative disorders, Parkinson’s disease and Huntington’s disease. Research is also being conducted in autism spectrum disorder.