Ness Ziona, Israel, October 9, 2018, Israeli biotechnology company Kadimastem (TASE: KDST) announces progress in the Phase 1 / 2a Clinical Trial - The company completed enrollment of 5 patients for Cohort A during September and performed the first ever astrocyte (AstroRx®) cell transplantation in the first ALS patient of the clinical trial. Results for Cohort A are expected in mid-2019.
With the progress in the clinical trial, Kadimastem joins a league of leading biotech companies in the field of regenerative medicine. The expected market is estimated at billions of dollars.
Kadimastem was founded in 2009 based on science developed at the laboratory of Prof. Michel Revel at the Weizmann Institute of Science in Israel, as part of a license agreement signed with Yeda Research and Development Company Ltd., the Weizmann Institute's technology transfer arm. The company has invested more than $40 million in development of the company's technologies.
The innovative technology developed in Kadimastem by Prof. Michel Revel, the company's Chief Scientist, and Dr. Michal Izrael, the VP R&D for Neurodegenerative Diseases, is a unique approach to cell transplantation in which healthy functioning cells are produced to replace the cells whose functioning is damaged in ALS, thus forming a type of spare part for the nervous system.
The clinical trial examines the safety and efficacy of the cell therapy following the transplantation in 21 ALS patients. The trial is being conducted at the Department of Neurology at Hadassah Ein Karem Medical Center in Jerusalem and is progressing as planned. The results of the trial for Cohort A are expected mid-2019.
Prof. Tamir Ben Hur, Director of the Department of Neurology and Head of the Department of Neurosurgery at the Hadassah Medical Center in Jerusalem, said: "This is the first ALS patient in the world to be treated with a treatment based on human embryonic stem cells, which have been developed in Israel from start to finish. The translation of the years of investment and development into the patient's treatment is very exciting. We have an optimal combination of academia, industry and medicine.”
The company's product is intended to be an off-the-shelf product that will be able to address the entire ALS population globally. Astrocytes (AstroRx®), which are the brain supporting cells, are produced in the company's clean rooms using the company's unique technology. The embryonic stem cells used are licensed from Hadasit and were originally produced in the laboratory of Prof. Benjamin Reubinoff, Head of the Obstetrics and Gynecology Department at Hadassah Medical Center, Jerusalem, and Head of the Hadassah Human Embryonic Stem Cell Research Center.
The scientific team at Kadimastem developed a unique process to expand the embryonic stem cells (the raw material) on an industrial scale and cause the cells to undergo full differentiation into the brain' supporting cells, the astrocytes.
Prof. Michel Revel, Kadimastem's Chief Scientist, commented: "Kadimastem has now completed years of research and development for its cell therapy product designed to treat ALS. This, following years of basic research that succeeded in using human embryonic stem cells to produce astrocytes, the specialized cells that support survival of neurons in the human brain, and following proof of efficacy in ALS disease models in animals. At the end, an industrial pharmaceutical production process has been developed at Kadimastem and has received all the regulatory approvals for use in humans. The successful injection of the AstroRx cells to the first patient of the clinical trial is an important milestone in the clinical development of this cell therapy product for the treatment of the ALS disease."
Mr. Yossi Ben-Yosef, CEO of the company, said: "After years of scientific work, we have achieved a scientific breakthrough in the medical world. The injection into the first patient marks a significant milestone in our clinical trial progress with business implications for Kadimastem. I would like to thank the Israel Innovation Authority and the public of investors who express confidence in the company and help us in our efforts to bring a cure to the patients suffering from this as yet incurable disease."