BrainStorm Cell Therapeutics is still navigating a three-year regulatory journey for the NurOwn therapy that was initiated in February 2021, marked by the FDA’s initial discouragement of filing a Biologics License Application (BLA). However, the recent acquisition of three new patents for NurOwn and NurOwn-Exosomes remains a safeguard for NurOwn’s cell therapy, fostering hope for a strong market position upon potential approval in amyotrophic lateral sclerosis (ALS), says GlobalData, a leading data and analytics company.

GlobalData’s report “Amyotrophic Lateral Sclerosis: Opportunity Analysis and Forecast to 2029’’, reveals that 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 etiology and complex pathophysiology, resulting in a persistent unmet need for effective treatments. NurOwn utilizes 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.

Despite failing to meet its primary endpoints in the Phase III trial, the results showed that treatment with NurOwn significantly elevated markers of neuroprotection and simultaneously decreased markers of neuroinflammation and neurodegeneration. Specifically, a 11% decrease in a biomarker known as neurofilament light (NfL) was observed.

Momna Ali, Healthcare Analyst at GlobalData, comments: “The impact of NurOwn on NfL is noteworthy, especially in light of the FDA’s recent approval of tofersen (Qalsody; Biogen). 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.”

In December 2023, the European Patent Office has granted patent for NurOwn titled ‘Method of Qualifying Cells,’ outlining a process to determine if a cell population is a suitable therapeutic for ALS. Similarly, the Australian and Israeli Patent Offices have granted patents both titled ‘Cell-Type Specific Exosomes and Use Thereof,’ covering an isolated Exosomes population derived from MSC-NTF cells.

Ali concludes: “According to GlobalData’s Sales and Forecast Database, NurOwn is anticipated to generate global sales of $401 million in 2029 for ALS, both positioning Brainstorm Cell Therapeutics favorably for monetization and adding to the overall ALS treatment portfolio.”