No treatment-related serious adverse events nor dose-limiting toxicities were reported during the 6- month post-treatment follow up
Clinically meaningful decline in disease progression was observed during the first 3-month of the 6-month follow up period, consistent with the results reported previously for Cohort A
NESS ZIONA, ISRAEL – December 15, 2020 – Kadimastem Ltd. (TASE: KDST), a clinical stage cell therapy company, announced today encouraging results of Cohort B of its Phase 1/2a clinical trial of AstroRx® for patients with Amyotrophic Lateral Sclerosis (ALS). The primary objective of the trial was to evaluate the safety of AstroRx®, an "off-the-shelf", clinical-grade, astrocyte cell product, developed and manufactured by the Company. A secondary objective of the trial included preliminary efficacy assessments.
Kadimastem scientists looking at astrocyte cells
comprising the AstroRx® product for the treatment of ALS.
All 5 patients included in cohort B were treated with a dose of 250x106 AstroRx® cells and completed the 6-month post-treatment follow up period.
No treatment-related serious adverse events (SAEs) nor dose-limiting toxicities were reported during the 6- month post-treatment follow up period of Cohort B, indicating a positive safety profile of the AstroRx® dose provided.
An efficacy analysis of the 6-month follow up period was performed to assess the duration of which the treatment maintains its therapeutic effect after a single AstroRx® administration. The efficacy assessment was based on the ALS Functional Rating Scale-Revised (ALSFRS-R), the gold standard outcome for assessment of ALS progression.
The rate of ALS disease progression in Cohort B patients, as assessed by the ALSFRS-R slope difference between pre-treatment and post-treatment periods, was significantly reduced from -1.43/month during the 3-4 month run-in period before treatment to -0.78/month during the 3-month follow-up after treatment, reflecting a clinically meaningful signal and statistically significant (p=0.0023) decline of 45% in the disease progression rate. At the end of the 6-month post-treatment period, the rate of ALSFRS-R progression was similar to the rate that was measured before treatment.
Encouraging results were also reported for Cohort A earlier this year, in which 5 patients were treated with a lower dose of 100x106 AstroRx® cells, presenting a similar product safety profile and clinical benefit.
"The results after 6 months of follow up are encouraging, as they suggest a clinically meaningful signal of effect for a period of 3 months by a single administration of AstroRx® and confirm the safety of AstroRx®. These results support a further, 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.
Professor Michel Revel, Founder and CSO of the Company, added, “The 6-month follow up data demonstrates the safety of AstroRx® as a treatment for ALS. The results also support the strategy of a repeated administration of the AstroRx® cells using the intrathecal route. As such, we are planning to continue with this strategy during the next phase of the clinical development program.”
AstroRx® is a cell therapy product developed and manufactured by Kadimastem at its GMP-compliant facility. AstroRx® contains functional, healthy astrocytes (nervous system support cells) derived from human Embryonic Stem Cells (hESC) that aim to protect diseased motor neurons through several mechanisms of action.
The Company's technology enables the injection of AstroRx® cells into the cerebrospinal cord fluid (CSF) of patients using a standard lumbar puncture procedure. The goal is to support the malfunctioning cells in the brain and spinal cord of patients suffering from Amyotrophic Lateral Sclerosis (ALS), in order to slow the progression of the disease, improve quality of life and extend life expectancy for these patients. AstroRx® has been granted orphan drug designation by the FDA for the treatment of ALS.
AstroRx® has shown to be safe and effective in preclinical trials and a Phase 1/2a open label clinical trial at Hadassah Medical Center, Ein-Kerem, Israel. The objective of the trial was to evaluate the safety, tolerability, and efficacy of escalating doses of AstroRx® cells in patients with ALS. Treatment was administered in addition to the appropriate standard-of-care.
Scientific literature indicates that astrocyte cells play a substantial role in supporting malfunctioning cells also in other neurodegenerative diseases, in addition to ALS. Accordingly, the Company is also evaluating other potential applications of the AstroRx® cell product.
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive fatal neurodegenerative disease causing dysfunction in the upper and lower motor nerves that control muscle function. ALS leads to muscle weakness, loss of motor function, paralysis, breathing problems, and eventually death. The average life expectancy of ALS patients is 2-5 years. According to the ALS Therapy Development Institute, it is estimated that there are approximately 450,000 ALS patients worldwide of which 30,000 reside in the US. According to the ALS Foundation for Life, the annual average healthcare costs of an ALS patient in the US are estimated at US$ 200,000. Thus, the annual healthcare costs of ALS patients in the US alone amount to US$ 6 Billion.
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 for the treatment of ALS, subject to a Phase 1/2a clinical trial. IsletRx is the Company's second product in development. IsletRx is comprised of functional pancreatic islet cells intended to treat patients with insulin dependent diabetes. IsletRx demonstrated safety and efficacy in a proof of concept preclinical study. 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).
Forward Looking Statement
This document may include forward-looking information as defined in the Securities Law, 5728 – 1968. Forward-looking information is uncertain and mostly is not under the Company's control and the realization or non-realization of forward-looking information will be affected, among other things, by the risk factors characterizing the Company's activity, as well as developments in the general environment and external factors affecting the Company's activity. The Company's results and achievements in the future may differ materially from any presented herein and the Company makes no undertaking to update or revise such projection or estimate and does not undertake to update this document. This document does not constitute a proposal to purchase the Company's securities or an invitation to receive such offers. Investment in securities in general and in the Company in particular bears risks. One should take into account that past performance does not necessarily indicate performance in the future.
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