Kadimastem, the developer of AstroRx, an experimental therapy for amyotrophic lateral sclerosis (ALS), is merging with NLS Pharmaceutics to combine their efforts in advancing their respective drug portfolios. The combined company will focus on progressing AstroRx and a diabetes drug developed by Kadimastem through clinical trials.
A Phase 2a study of AstroRx is planned in the U.S. after January 2025, pending finalization of the merger agreement. This decision follows the U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug (IND) application for the study.
Michel Revel, MD, PhD, Kadimastem’s chief scientific officer, stated in a joint press release, “I’m excited about this merger because the combined company presents a significant opportunity to progress our portfolio and product candidates into and through clinical trials. Together with the assets from NLS, we believe that we will be well-positioned to promote and expand these promising treatments.”
The agreement will also facilitate the advancement of NLS’s platform of dual orexin receptor agonists (DOXA) for various brain conditions. These molecules activate the orexin receptor 1, found in nerve cells, to reduce inflammation and nerve cell degeneration in ALS and other neurodegenerative conditions.
NLS CEO Alex Zwyer commented, “I believe that the merger is an outstanding opportunity to progress our proprietary DOXA platform and help to enhance Kadimastem’s portfolio of neurodegenerative and diabetes candidates.”
AstroRx: Targeting Astrocytes in ALS
ALS is characterized by the progressive damage and loss of motor neurons, which control voluntary muscle movement. Astrocytes, star-shaped cells that support and protect nerve cells, become reactive and toxic in ALS, contributing to nerve cell loss. AstroRx is a cell-based therapy designed to compensate for the malfunctioning of astrocytes in ALS, aiming to prevent further damage and slow disease progression.
The therapy consists of functional astrocytes derived from human embryonic stem cells, creating an off-the-shelf product for immediate use in patients. AstroRx is administered directly into the spinal canal via intrathecal injection.
Phase 1/2a Trial Results
In a previous Phase 1/2a clinical trial (NCT03482050) conducted in Israel, researchers evaluated the safety and efficacy of AstroRx in 10 ALS patients diagnosed within the previous two years. Participants received a single injection of either 100 million or 250 million cells and were followed for one year.
The cell-based therapy significantly reduced disease progression, as measured by the decline in ALS Functional Rating Scale Revised scores, by 53% over the first three months. The effect was more pronounced in patients receiving the higher dose and those with rapidly progressing disease. However, the efficacy diminished over time, suggesting the need for repeat injections to maintain the therapeutic effect.
AstroRx demonstrated a favorable safety profile and was well-tolerated in the trial, with most side effects being mild to moderate in severity.
The upcoming Phase 2a trial in the U.S. will investigate whether repeat dosing of AstroRx every three months can sustain the slowing of ALS progression over the long term. The trial is anticipated to be conducted at multiple sites.
Kadimastem recently partnered with Pluri to manufacture AstroRx for the upcoming trial at its cell-therapy production facility.
