The publication presents the company’s successful phase I/IIa clinical trial in Israel to evaluate the safety, tolerability, and therapeutic effects of intrathecal injection of AstroRx® in patients with ALS
Ness Ziona, Israel, February 15th 2023, Kadimastem (TASE: KDST), a well-known biotech company in the field of cellular therapy, which is in the clinical development stages of innovative products for the treatment of ALS and diabetes, announced today the publication of its Phase I/IIa clinical trial results in the peer-reviewed prestigious journal of Translational Medicine. The article describes the safety and efficacy results of AstroRx®; Kadimastem's cell therapy product in its first in-human phase I/IIa clinical trial in 10 ALS patients conducted in Israel from 2018 to 2020 (ClinicalTrial.gov: NCT03482050). Link to the article.
The article details the important milestone achieved by the company in concluding the study and achieving its targeted results of safety and clinical meaningful decline in disease progression rate. The study was a phase I/IIa, open-label, dose-escalating clinical trial to evaluate the safety, tolerability, and therapeutic effects of intrathecal injection of AstroRx® in patients with ALS.
Dr. Michal Izrael, Kadimastem's VP of R&D
Five patients were injected intrathecally with a single dose of 100x106 AstroRx® cells and five more patients with 250x106 cells (low and high dose, respectively). Safety and efficacy assessments were recorded for 3 months pre-treatment (run-in period) and 3, 6, and 12 months post-treatment (follow-up period). The study demonstrated that a single intrathecal administration of AstroRx® at both doses was safe and well tolerated. No adverse events (AEs) related to AstroRx® itself were reported. Transient AEs related to the intrathecal (IT) procedure were all mild to moderate.
The study demonstrated a clinically meaningful effect that was maintained over the first 3 months period after treatment, as measured by the pre vs post slope change of a clinical score known as ALS Functional Rating Score (ALSFRS-R). The effect was even more profound in a rapid progressor subgroup of 5 patients, after 3 months the clinical effect stopped. The clinical trial findings suggest that a single IT administration of AstroRx® to ALS patients at a dose of 100x106 or 250x106 cells is safe.
A signal of beneficial clinical effect was observed for the first 3 months following cell injection. These results support further investigation needed of repeated intrathecal administrations of AstroRx®, e.g., every 3 months, which is planned for a phase IIa clinical trial to be conducted in multiple US clinical sites following an FDA approval of the company’s IND submission if approved.
Kadimastem's AstroRx® cell product is an allogeneic cell-based product, composed of healthy and functional human astrocytes derived from embryonic stem cells. AstroRx® was shown to remove toxic excessive glutamate, reduce oxidative stress, secrete various neuroprotective factors, and act as an immunomodulator.
“These results support a further need for a randomized-controlled, clinical trial with repeated doses of AstroRx® in patients with ALS, in order to prolong the clinical effect observed by a single dose," stated Dr. Marc Gotkine, Head of the ALS Clinic at the Department of Neurology at Hadassah Medical Center, Jerusalem, and the Principal Investigator of the trial.
Dr. Michal Izrael, Kadimastem's VP R&D says: “We would like to thank the Hadassah Hospital clinical team, patients, and their families for their contribution. These are the fruits of Kadimastem's dedicated team. We are very excited to move toward our next Phase IIa clinical study with new off-the-shelf (“thaw and inject”) AstroRx® cell product which will be tested using repeated intrathecal injections of AstroRx®.”
Kadimastem is a clinical stage cell therapy company, developing and manufacturing "off-the-shelf", allogeneic, proprietary cell products based on its technology platform for the expansion and differentiation of Human Embryonic Stem Cells (hESCs) into functional cells. AstroRx®, the company's lead product, is an astrocyte cell therapy in clinical development for the treatment for ALS and in pre-clinical studies for other neurodegenerative indications.
IsletRx is the company's treatment for diabetes. IsletRx is comprised of functional, insulin and glucagon producing and releasing pancreatic islet cells, intended to treat and potentially cure patients with insulin-dependent diabetes.
Kadimastem was founded by Professor Michel Revel, CSO of the company and Professor Emeritus of Molecular Genetics at the Weizmann Institute of Science. Professor Revel received the Israel Prize for the invention and development of Rebif®, a multiple sclerosis blockbuster drug sold worldwide. Kadimastem is traded on the Tel Aviv Stock Exchange (TASE: KDST).
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