Israeli biotech firm's ALS treatment shows positive results in trials

"We are now focused on advancing strategic FDA regulatory processes that will necessitate the conduct of global multi-center clinical trials."

AstroRx cells (photo credit: Courtesy)
AstroRx cells
(photo credit: Courtesy)
Ness Ziona-based biotech firm Kadimastem announced Monday that its lead product AstroRx is showing positive results in the Phase1/2a clinical trials for treating ALS.
"The current study results are encouraging, as they suggest a clinically meaningful signal of effect. A further randomized, parallel-controlled, clinical trial is needed to confirm these positive effects of AstroRx in the treatment of ALS," Dr. Marc Gotkine, of the Department of Neurology at Hadassah Medical Center, Jerusalem and the principal investigator of the Phase 1/2a clinical trial of AstroRx, said in a statement.
“We are excited by the positive and consistent clinical results, demonstrating both the good safety profile and therapeutic effect of AstroRx," Kadimastem CEO Rami Epstein explained.
"The comparison between the ALSFRS-R change before and after treatment convey a clinically meaningful decline of more than 50% in disease progression rate for a period of three months for both cohorts. The effect was even more profound in patients who deteriorate rapidly. Repeated administrations of the treatment, planned to be assessed in future clinical trials, will hopefully extend the duration of the beneficial effect. These results strengthen our commitment to bringing an innovative therapeutic treatment to ALS, a devastating disease currently having no effective treatment.”
The trials were limited due to delays caused by the coronavirus pandemic, and due to the situation in Israel, the company decided not to use additional cohorts beyond Cohort A and B.
“The positive interim results of Cohort A and B strongly support our next global clinical development stages," Epstein added.
"We are now focused on advancing strategic FDA regulatory processes that will necessitate the conduct of global multi-center clinical trials. In support, the company is also dedicating resources to production process scale-up as well as to the development of a second generation of AstroRx as a frozen product that will accelerate our clinical development plan to support market authorization.”
“The strategic plan will benefit the patients and support the efforts of the medical community striving to bring a solution to this devastating disease," Kadimastem founder and CSO Prof. Michel Revel explained.
"We value the contribution of the patients who volunteered to participate in our trial, as well as the physicians and clinical team at Hadassah Medical Center and look forward to continuing our collaboration during the next stages of global clinical trials.”
Also known as Lou Gehrig's Disease, ALS is a rapidly progressive, fatal neurodegenerative disease-causing dysfunction in the upper and lower motor nerves that control muscle function. A release shared by the company described the disease’s symptoms as including muscle weakness, loss of motor function, paralysis, breathing problems and eventually death.
According to Epstein, most people diagnosed with ALS live only up to five years from the time of diagnosis. According to the ALS Therapy Development Institute, there are approximately 450,000 ALS patients worldwide.
Maayan Hoffman contributed to this report.