Medical Breakthrough on a Global Scale and Significant Achievement for Kadimastem

NESS ZIONA, Israel, October 9, 2018 /PRNewswire/ --

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."

Company product AstroRx® 

AstroRx® is an innovative cell therapy for ALS. The treatment consists of nerve-supporting cells (astrocytes), which are impaired in ALS patients. AstroRx® is produced in Kadimastem from human embryonic stem cells with unique technology developed by the company. The advantage of AstroRx® is to replace the poor functioning of the supporting cells in the patient, with healthy cells. AstroRx® operates in several mechanisms that simultaneously protect damaged nerve cells, as opposed to the currently approved single-agent drugs. AstroRx®'s unique properties may significantly slow the progression of the disease, as the company has shown. Currently, there are only two drugs approved by the US Food and Drug Administration (FDA) for the treatment of ALS, but they are only slightly slowing down the progression of the disease.  The results of Kadimastem's preclinical demonstrated that there has been a significant improvement in the musculoskeletal function of ALS model animals that were treated with AstroRx in comparison to untreated mice, and the efficacy of the treatment is also reflected in other indicators indicating a delay during the onset of the disease, and has been shown to be safe in comprehensive safety studies. , Injection of the cells into the spinal cord fluid enables their dispersion along the central nervous system providing effective defense for the motor nerve cells (motor nerves responsible for movement) in the brain and spinal cord. 

With the start of the clinical trial Kadimastem joins a team of leading biotech companies in the field of regenerative medicine, the expected market volume for the product is estimated at billions of dollars. 

About Kadimastem 

Kadimastem Ltd. (http://www.Kadimastem.com ) is a clinical stage biotechnology company developing industrial regenerative therapies based on human embryonic stem cells (hESC) for the treatment of neurodegenerative diseases such as ALS, as well as diabetes. The company was founded in 2009 by Prof. Michel Revel and Yossi Ben Yosef and is listed on the Tel Aviv Stock Exchange (TASE: KDST). The company's chairman is Dr. Eli Opper, formerly the Chief Scientist of Israel's Ministry of Industry, Trade, and Labor, and the company's Scientific Advisory Board consists of leading scientists and pioneers in their fields. The company's investors, in addition to its founders, include Altshuler Shaham Investment House, foreign investors (Julien Ruggieri and Avi Meizler) and additional institutional investors. 

Kadimastem was founded on the basis of patent-protected technologies developed in Prof. Michel Revel's laboratory at the Weizmann Institute of Science. Based on the company's unique technological platform, Kadimastem develops regenerative therapies to repair and replace tissues and organs by using functioning cells differentiated from stem cells. The company focuses on transplanting healthy brain cells to support the survivability of damaged nerve cells as cell therapy for ALS and transplanting insulin-producing pancreatic cell for the treatment of insulin-dependent diabetes. 

Kadimastem has an extensive scientific advisory board, featuring prominent scientists and pioneers: In the embryonic stem cell field - Professor Joseph Itskovitz-Eldor, a world renowned expert and pioneer in pluripotent stem cell research and former head of Obstetrics and Gynecology at Rambam Medical Center, serves both as a director and a senior strategic consultant in the company, and Prof. Benjamin Reubinoff, Director of the Hadassah Human Embryonic Stem Cell Research Center and Senior Physician at the Department of Obstetrics and Gynecology at the Hadassah University Medical Center; in the neurodegenerative disease field - Prof. Tamir Ben Hur, Head of the Department of Neurology and Division of Medical Neurosciences at Hadassah Medical Center in Jerusalem; and in the diabetes field, Prof. Shimon Efrat, professor of Human Molecular Genetics and Juvenile Diabetes at Tel Aviv University and a world renowned expert in cell replacement therapy for diabetes, and Prof. Eddy Karnieli, former Director of the Institute for Endocrinology, Diabetes and Metabolism at the Rambam Medical Center and a world renowned expert in these fields. 

Contact: 
Yehuda Feinberg
Tel: +972-73-7971604
Email: [email protected]

SOURCE Kadimastem