Kadimastem Secures US Patent for Cell Therapy AstroRx
An upcoming trial will test if dosing every 3 months can delay disease progression
The patent, granted by the U.S. Patent and Trademark Office, supports an upcoming Phase 2a clinical trial that’s planned to start in the U.S. next year.
The patent joins several others Kadimastem has secured in the U.S., Israel, and Europe for ALS treatments, and for screening new therapies.
“The patent announced today strengthens our intellectual property portfolio in the field of ALS and other neurodegenerative diseases. Not only is it a good sign that the patent was granted in the United States in advance of the next trial, but the timing also gives AstroRx a clear advantage as it increases the strategic value of the product,” Asaf Shiloni, CEO of Kadimastem, said in a company press release.
AstroRx is an off-the-shelf, investigational cell therapy composed of astrocytes. In normal circumstances, these star-shaped cells support neurons and help maintain a healthy brain environment. They become abnormal in people with ALS and are thought to contribute to disease progression.
The astrocytes that make up AstroRx are derived from human embryonic stem cells and are expected to make up for the lack of functional astrocytes, preventing disease worsening when injected into patients’ spinal canal.
The safety and preliminary effectiveness of AstroRx was initially assessed in a Phase 1/2 trial (NCT03482050), which investigated increasing doses of the medication in 10 adults with early stage ALS at a single center in Israel.
The first five patients in the trial received a single infusion of AstroRx at a dose of 100 million cells and saw a slowing in disease progression over the first three to four months following dosing.
A second group of five patients then received a single infusion containing 250 million AstroRx cells. It also safely and significantly slowed disease progression in the first three months following treatment in this group of patients, but a decrease in effectiveness was observed after six months, suggesting repeated doses may be required to maintain the treatment’s effect.
The upcoming Phase 2a trial in the U.S. will evaluate if dosing with AstroRx every three months can continuously delay disease progression.
“The ALS program is progressing well. The company’s broad patent portfolio was strengthened today, and we are on track to begin the much anticipated clinical trial of AstroRx within months. I am excited to see our original vision come to fruition. The goal is to improve the treatment of ALS patients,” Michel Revel, MD, PhD, chief scientist at Kadimastem, said.
Kadimastem is planning to submit an investigational new drug application to the U.S. Food and Drug Administration requesting clearance to conduct ALS clinical trials of AstroRx. The application is set for submission later this year and, if cleared, the company plans to initiate the trial next year.