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Kadimastem Intends to Submit an Amendment of its ALS Phase 1/2a Clinical Trial Protocol Following Recent Positive Interim Results
NESS ZIONA, Israel, Nov. 18, 2019 /PRNewswire/ -- Kadimastem (TASE: KDST), a clinical stage cell therapy company, today announced its intention to submit an amendment of its ongoing Phase 1/2a clinical trial for the treatment of ALS patients to the Ethics Committee and the Israeli Ministry of Health.
AstroRx®, an "off-the-shelf" clinical grade cell product, developed and manufactured by the Company, is comprised of human astrocyte cells. The positive interim results of cohort A, reported in September 2019, demonstrated safety and statistically significant preliminary efficacy (p = 0.0023) of a single dose administration, indicating that the progression of the ALS disease was reduced in the first 3-months post-treatment period, compared to the pre-treatment period. The purpose of the amendment is to assess the safety and efficacy of a repeated low dose (100x106) administration of AstroRx® in cohort C in 2 consecutive injections separated by an interval of 2-3 months, rather than the repeated administration of the medium dose (250x106), as originally planned. As part of the proposed protocol amendment, cohort D will include a repeated dose of (250x106) AstroRx® cells (as originally planned in cohort C). The performance of cohort D will be discretionary, based on the results of previous cohorts.
The Phase 1/2a clinical trial is progressing, with cohort B patients currently treated with a single dose of (250x106) AstroRx® cells. Results of cohort A and B are expected to be reported as planned and previously announced, by the end of 2019 and in Q3 2020, accordingly. Results of cohort C are expected in the first half of 2021.
As the Phase 1/2a clinical trial progresses, the Company intends to continue discussions with the FDA during 2020, in order to submit an IND application by mid-2021 for the purpose of advancing to the next multi-center clinical trial in ALS. Said trial is intended to assess either the current version of the AstroRx® product or a frozen version of the product which is currently being developed by the Company. The frozen version of the AstroRx® product is expected to enable manufacturing cost reductions as well as improvement of logistic processes. First proof-of-concept of the frozen product is expected by mid-2020.
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).
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.
Yossi Nizhar, CFO
Investor Relations and Financial Media
Meirav Gomeh-Bauer, Development
Global Media & Collaborations
Dasy Mandel, Director of Business
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