Progressing Towards Clinical Trials: Kadimastem Proves Efficacy Of Its Unique ALS Treatment In Pre-Clinical Trials

The company will approach the FDA to continue the approval process for its clinical trials

Yossi Ben-Yossef, Kadimastem’s CEO: “The excellent results of the trial pave the way for our clinical trials, and allow the company to launch the product’s manufacturing according to GMP standards”

NESS ZIONA, Israel--(BUSINESS WIRE)--Israeli biotechnology company Kadimastem (TASE: KDST) reports success in a pre-clinical trial for the treatment of ALS in another animal model. The results of the trial demonstrate the efficacy of Kadimastem’s cell-based treatment for ALS. In light of the successful conclusion of the proof of efficacy stage and the considerable advancement of the product’s development, the company intends to take another step in its process with the FDA, and file a Pre-IND in the upcoming weeks.

The trial tested the efficacy of injecting the brain supporting cells (astrocytes) produced through the company’s unique technology from pluripotent stem cells. Pluripotent stem cells are cells which have the ability to differentiate into any type of cells in the body. The astrocytes were injected into the spinal fluid of ALS rat model, and have shown a significant improvement in the rats’ life expectancy.

Kadimastem’s unique technology enables the production of the astrocytes which will serve as the treatment for the patients, as an off-the-shelf product for the treatment of large patient populations. The injection of cells into the spinal fluid is a standard procedure performed routinely in hospitals worldwide. The company found that such injections into the spinal fluid enable the cells to disperse throughout the central nervous system, and it thus established this method of cell penetration in the future treatment of patients. The injecting of healthy and functioning astrocytes into the nervous system of patients may provide systemic support for the damaged motor neurons, thereby inhibiting disease progression and improving the patients’ life quality and expectancy.

Professor Michel Revel, the company’s Chief Scientist, explained: “In addition to prolonging the rats’ lives, a significant improvement occurred in the motoric abilities of the treated rats compared to those who were not treated. The efficacy of the treatment was also demonstrated in parameters indicating the inhibition of disease onset and its progression. The success of the trial strengthens our intention to develop and manufacture a product, to be used in the future as an off-the-shelf product, without the need for drugs which prevent graft immuno-rejection. The company’s treatment is capable of affecting the course of the disease and improving the patients’ life quality and expectancy.”

Yossi Ben Yossef, the company’s CEO, stated: “After the proof of concept and efficacy of the ALS treatment, we are at the front of the global trend of cell-based therapies for incurable diseases. The excellent results of the trial pave the way for our clinical trials, and allow the company to launch the product’s manufacturing according to GMP standards. The results constitute a significant milestone, which will help us advance operative, strategic and business moves in this field.”

Professor Tamir Ben-Hur, Head of the Department of Neurology in Hadassah Medical Center in Jerusalem, and an advisor to the company, noted: “These pre-clinical results demonstrate the unique ability of the technology developed by Kadimastem for the treatment of ALS. The development of an off-the-shelf product will enable the company to disseminate the product and to provide treatment to a wide population of patients, which is a significant advantage. The progression of the company in the regulatory pathways in the upcoming months will enable testing the treatment’s safety and efficacy in humans in the near future, in phase I/IIa clinical trials, which the company is planning to conduct under FDA and Israeli Ministry of Health approvals.”

About ALS

ALS (Amyotrophic Lateral Sclerosis) is the most severe of a group of neurodegenerative diseases that damage motor neurons, and control most of the muscles in the body. The destruction of these neurons causes complete paralysis of the muscles they control. Currently, the disease is incurable and there is no significant drug treatment available. In recent years, there is growing evidence that in ALS patients, the ability of nervous system support cells (astrocytes) to maintain a supporting microenvironment for motor neurons is impaired.

About Kadimastem

Kadimastem is a biotechnology company, operating in the field of regenerative medicine – a groundbreaking field in which the malfunctioning of organs which leads to diseases is repaired by external cells, tissues or organs. The company specializes in the development of human stem cell-based medical solutions for the treatment of diabetes and neurodegenerative diseases, such as ALS and Multiple Sclerosis. The company was founded in August 2009 by Professor Michel Revel and Yossi Ben Yossef, and is traded on the Tel Aviv Stock Exchange (TASE: KDST). Kadimastem employs 32 people, of which 11 are PhDs, and its 1,700m² offices and labs are located in the Ness Ziona Science Park.

Kadimastem was founded based on patent protected technology that was developed at the Weizmann Institute of Science, in Prof. Michel Revel’s laboratory. Prof. Revel, who serves as the company’s Chief Scientist and director, developed Merck KGaA’s blockbuster drug, Rebif® for the treatment of MS (sales of around $2.4 billion sales in 2014).

Based on the company’s unique platform, Kadimastem is developing two types of medical applications: A. Regenerative medicine, which repairs and replaces organs and tissue by using functioning cells differentiated from stem cells. The company focuses on transplanting healthy brain cells to support the survivability of nerve cells as cell therapy for ALS, and transplanting insulin-secreting pancreatic cells for the treatment of insulin-dependent diabetes; B. Drug screening platforms, which use functional human cells and tissues to discover new medicinal drugs. The company has two collaboration agreements with leading global pharmaceutical companies.

The company is headed by Yossi Ben-Yossef, an entrepreneur with extensive experience in life sciences companies. The company’s chairman is Dr. Eli Opper, formerly the Chief Scientist of the Israeli Ministry of Industry, Labor and Trade, and its investors include Altshuler Shaham Investment House, foreign investors (Julien Ruggieri and Avi Meizler), and the company’s founders.

Kadimastem has an extensive scientific advisory board, featuring prominent scientists and pioneers: in the embryonic stem cells field, Professor Benjamin Reubinoff and Professor Joseph Itskovich, in the neurodegenerative disease field, Professor Tamir Ben-Hur, and in the diabetes field, Professor Shimon Efrat and Professor Eddy Karnieli.

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