BrainStorm Cell Therapeutics’s NurOwn is approaching the conclusion of a three-year regulatory journey that commenced in February 2021 when the US Food and Drug Administration (FDA) advised against filing a Biologics License Application (BLA).

Armed with corrected Phase III data, BrainStorm announced a BLA submission in August 2022, which was then rejected in November the same year.

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By using the FDA’s File Over Protest procedure, the company secured an advisory committee meeting in March 2023.

In September 2023, the FDA’s advisory committee voted 17-1 against NurOwn’s efficacy for mild to moderate amyotrophic lateral sclerosis (ALS), citing concerns regarding mechanisms of action, manufacturing, and failure to meet the primary endpoint.

However, an announcement has revealed that the FDA has granted BrainStorm Cell Therapeutics a meeting on 6 December this year to discuss a Special Protocol Assessment for the overall protocol design of a potential confirmatory Phase III trial.

As per GlobalData‘s (a leading data and analytics company) Amyotrophic Lateral Sclerosis: Opportunity Analysis and Forecast to 2029 report, developing disease-modifying treatments for this rare and aggressive disease has proven challenging.

The high failure rate in ALS clinical trials is often attributed to the disease’s unclear aetiology and complex pathophysiology, resulting in a persistent unmet need for effective treatments.

NurOwn utilises a process where a patient’s bone marrow-derived mesenchymal stem cells are treated in the lab to produce neurotrophic factors.

These factors are then delivered through intrathecal injection into ALS patients to stimulate neuron regeneration and nerve-muscle interaction.

However, key opinion leaders (KOLs) interviewed by GlobalData express uncertainty about the readiness of this approach for ALS patients due to the lack of efficacy data and the challenging route of administration.

Some KOLs also highlighted concerns that NurOwn’s social media advertising campaigns targeting ALS patients might foster false hope and misconceptions among patients and their families.

Despite failing to meet its primary endpoints in its Phase III (NCT03280056) trial, the results showed that treatment with NurOwn significantly elevated markers of neuroprotection and simultaneously decreased markers of neuroinflammation and neurodegeneration.

Specifically, an 11% decrease in a biomarker known as neurofilament light (NfL) was observed.

The impact of NurOwn on NfL is noteworthy, especially considering the FDA’s recent approval of Biogen’s Qalsody (tofersen) earlier this year.

Similar to NurOwn, tofersen fell short of its primary endpoint. Tofersen, the first therapy for patients with SOD1 mutation-mediated ALS, gained approval based on reductions in the same biomarker.

According to GlobalData’s Sales and Forecast Database, NurOwn is anticipated to generate global sales of $401m in 2029 for ALS, both positioning Brainstorm Cell Therapeutics favourably for monetisation and adding to the overall ALS treatment portfolio.