Kadimastem 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.
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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.