Kadimastem, NLS merge to advance ALS therapy AstroRx, other drugs
Phase 2a study of AstroRx is planned in US after January 2025
Kadimastem, the developer of the experimental amyotrophic lateral sclerosis (ALS) therapy AstroRx, is merging with NLS Pharmaceutics to combine efforts to advance both companies’ drug portfolios.
The combined company will focus on moving AstroRx and a diabetes drug being developed by Kadimastem through clinical trials. A Phase 2a study of AstroRx is planned in the U.S. after January 2025, after the agreement is finalized. The decision follows the U.S. Food and Drug Administration (FDA) clearance of an application submitted last year requesting the study.
“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,” Michel Revel, MD, PhD, Kadimastem’s chief scientific officer, said in a joint press release. “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 help advance NLS’s platform of dual orexin receptor agonists (DOXA) for a number of brain conditions. These are molecules that activate the orexin receptor 1, which is found in nerve cells, to reduce inflammation and nerve cell degeneration in ALS and other neurodegenerative conditions.
“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,” said NLS CEO Alex Zwyer.
ALS is caused by the progressive damage and loss of specialized nerve cells, called motor neurons, that control voluntary muscle movement. While many mechanisms are believed to contribute to disease progression, astrocytes, the star-shaped cells that normally support and protect nerve cells, but become reactive and toxic in ALS, have been established as important players in nerve cell loss.
Results of Phase 1/2 trial of AstroRx
AstroRx is a cell-based therapy designed to compensate for the malfunctioning of astrocytes in ALS, which should help prevent further damage and slow disease progression.
The cells within it are derived from human embryonic stem cells that are treated to mature into functional astrocytes, which are stored to create an off-the-shelf product that can be immediately used in patients. The therapy is administered directly into the spinal canal, that is, via intrathecal injection.
In a previous Phase 1/2a clinical trial (NCT03482050) in Israel, researchers tested the safety and efficacy of AstroRx in 10 ALS patients who’d been diagnosed within the previous two years. The participants received a single injection, at a dose of 100 million or 250 million cells, and were followed for a year.
The cell-based therapy significantly reduced disease progression, as assessed by the decline on ALS Functional Rating Scale Revised scores, by 53% over the first three months. The effect was greater in patients who received the higher dose and in those with rapidly progressing disease. Its efficacy was lost later, however, indicating repeat injections will be needed to maintain an effect.
AstroRx was safe and well tolerated in the trial, with the most common side effects being mild to moderate in severity.
The upcoming Phase 2a trial in the U.S. will test whether repeat dosing of AstroRx, every three months, can slow ALS progression in the long run. The trial is expected to take place at multiple sites.
Kadimastem recently partnered with Pluri to produce AstroRx for the upcoming trial at its 47,000-square-foot cell-therapy production facility.
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