Kadimastem asks for FDA to clear new AstroRx clinical trial
Continuous dosing will be tested after earlier trial showed diminished efficacy
Kadimastem is planning a Phase 2a clinical trial to test if repeat dosing of AstroRx, its investigational therapy for amyotrophic lateral sclerosis (ALS), can continuously delay the disease’s progression.
The study will investigate every three-month dosing after findings from an earlier Phase 1/2 trial (NCT03482050) showed a single infusion led to a clinically meaningful decline in disease progression over the first three months, but lost effectiveness later on.
The company recently submitted an investigational new drug (IND) application asking the U.S. Food and Drug Administration (FDA) to clear the trial.
“The submission of an IND to the FDA is a major milestone. It is very exciting to have arrived at this juncture. We eagerly await the approval to start this trial and wanting to help ALS patients as soon as possible,” said Michel Revel, MD, PhD, founder and chief scientific officer at Kadimastem, in a company press release.
AstroRx is an off-the-shelf, investigational cell therapy composed of healthy astrocytes derived from human embryonic stem cells. Astrocytes are star-shaped cells that support nerve cells in the brain and spinal cord, but are believed to go awry in ALS and contribute to disease progression.
Injected directly into the spinal canal (intrathecal administration), it’s expected to make up for the faulty astrocytes, preventing further damage to nerve cells and slowing disease progression.
AstroRx’s safety and preliminary signs of efficacy were investigated in the Phase 1/2 study in Israel, which enrolled 10 patients who’d been diagnosed with ALS in the past two years.
After an initial monitoring period of three months, patients were equally divided to receive a single dose of 100 million AstroRx cells (low dose) or 250 million AstroRx cells (high dose) and were monitored for up to a year.
Results from the open-label trial showed both doses induced a significant decrease in the rate of disease progression over the first three months — a 53% reduction. However, no benefits were observed in either group after six and 12 months’ follow-up, suggesting repeat dosing is necessary to maintain its efficacy.
“For the last year, we’ve reported our plans to submit an IND to the FDA with AstroRx for the treatment of ALS. The submission of the IND marks a significant milestone for the company, and subject to the approval of the FDA we hope to commence this very important multi-site trial and to bring this potential life enhancing therapy to the market and to patients as soon as possible,” said Ronen Twito, Kadimastem’s board executive chairman.
The investigational cell therapy was granted a U.S. patent in August 2022 for treating ALS and other neurodegenerative diseases. Patents are also in place in Japan and Israel. The company also continues to advance its diabetes program, according to Kadimastem CEO Asaf Shiloni.