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Kadimastem Announces the Completion of Treatment for Cohort B of Its Phase 1/2a Clinical Trial in ALS Patients
NESS ZIONA, Israel, Dec. 23, 2019 /PRNewswire/ -- Kadimastem Ltd. (TASE: KDST), a clinical stage cell therapy company, today announced that treatment with a single dose of 250x10 AstroRx® cells to all 5 ALS patients in cohort B has been completed.
The primary objective of the trial is to evaluate the safety of injecting AstroRx®, an "off-the-shelf" clinical-grade astrocyte cell product, developed and manufactured by the Company, into the cerebro-spinal fluid (CSF) of ALS patients. A secondary objective of the trial includes preliminary efficacy.
The clinical trial is progressing as planned. Cohort B patients will be monitored for 6 months following treatment. In addition, the first patient of cohort C has already been enrolled. Cohort C is designed to assess long term safety and efficacy of a repeated dose administration of AstroRx® in 2 consecutive injections separated by an interval of 2-3 months. As previously reported in September 2019, positive interim results of cohort A demonstrated safety and statistically significant preliminary efficacy of a single dose of 100x10 AstroRx® cell administration. In light of these positive results, the company is taking the necessary steps to obtain approval to a proposed protocol amendment according to which the repeated administration will comprise of the same dose (100x10).
Results of cohort A and B are expected to be reported as planned and as previously announced, by the end of 2019 and during August 2020, accordingly. Results of cohort C are expected in the first half of 2021.
Rami Epstein, CEO of Kadimastem, commented: "Completing treatment for the additional 5 ALS patients in Cohort B, for a total of 10 patients treated with our product in our clinical trial, serves as an additional demonstration of our ability to develop and produce high quality clinical grade cells and takes us a significant step forward in our path to bringing innovative cure to ALS. The expected completion of cohort B 6-months follow-up period will allow us to assess the safety and preliminary efficacy of 250x10 cells, compared to that of the lower dose administered in cohort A."
Prof. Michel Revel, Founder and CSO of the Company, added: "The results of the next treatment group, Cohort C, in which each patient will be treated with 2 consecutive injections separated by an interval of 2-3 months, will allow us to assess the possible prolonged efficacy of the repeated dose, compared to the single dose treatment provided in cohorts A and B. The results that will be obtained from the different cohorts, will support us in the process of defining the dose and treatment regimen that will lead to most favorable results for patients over time."
AstroRx® is a clinical grade cell therapy product developed and manufactured by Kadimastem in its GMP-compliant facility, containing 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 spinal cord fluid of patients suffering from Amyotrophic Lateral Sclerosis (ALS) with the goal of supporting the malfunctioning cells in the brain and spinal cord, in order to slow the progression of the disease and improve patients' quality of life and life expectancy. AstroRx® has been shown to be safe and effective in preclinical studies. AstroRx® has been granted orphan drug designation by the FDA.
Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive fatal neurodegenerative disease causing disfunction 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 platform technology for the expansion and differentiation of Human Embryonic Stem Cells (hESCs) into clinical grade functional cells. AstroRx®, the Company's lead program, is a clinical-grade astrocyte cell therapy for the treatment of ALS, currently undergoing a Phase 1/2a clinical trial. In addition, preclinical trials are ongoing with the Company's IsletRx pancreatic functional islet cells for the treatment of insulin dependent diabetes. Kadimastem was founded by Prof. Michel Revel, CSO of the Company and Professor Emeritus of Molecular Genetics at the Weizmann Institute of Science. Prof. 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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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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