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Kadimastem Reached Agreements with the FDA Regarding the Framework of the Clinical Trials in Humans for the Treatment of ALS

Dec. 10, 2015

Yossi Ben-Yossef, Kadimastem’s CEO: “The successful Pre-IND meeting with the FDA paves the way for IND submission, towards implementation of clinical trials”

Ness Ziona, Israel, December 10, 2015, Israeli biotechnology company Kadimastem (TASE: KDST) announces today, that following a meeting it held in November with the FDA, there is agreement with the FDA regarding the framework of the company’s future plans. The meeting with the FDA concerned the company’s cell-based product for the treatment of ALS.

The response received on December 9, 2015, from the FDA represents a summary of the agreements reached for the product manufacturing plans (chemistry, manufacturing and controls), the safety trial and the framework for the trials in humans, phase I/IIa clinical trials, which the company presented in the meeting.

The company estimates that in the upcoming year it will complete the necessary preparations for IND submission for a safety and product efficacy phase I/IIa clinical trial in humans, which will be conducted according to a protocol approved by the FDA.

The company's product (AstroRx) is a cell-based treatment (astrocytes) that Kadimastem developed for the treatment of ALS. Currently, there is no effective treatment available for the disease. Kadimastem’s unique technology enables the production of the cells which will serve as the treatment for the patients, under GMP standards, for the treatment of large patient populations.

The injection into the spinal fluid is a standard procedure performed routinely in hospitals worldwide. The company found that injecting the cells into the spinal fluid enables them 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 astrocyte cells 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, noted: “We are happy about the FDA’s positive response to the Pre-IND package the company submitted. The meeting exemplified the unique capabilities of the technology developed by Kadimastem for the treatment of ALS, as demonstrated in the POC tests in the ALS animal model. We are now preparing for the production of the product (AstroRX) under GMP manufacturing conditions in the company’s facility, which will be used for the safety tests, as well as for the clinical trials”.

Yossi Ben-Yossef, Kadimastem’s CEO, added: “The face to face meeting with the FDA is a significant milestone for the company, and it rapidly advances Kadimastem towards human trials. We believe that within around one year Kadimastem will complete the product safety tests and all the material required for IND submission, for the safety and efficacy phase I/IIa human clinical trial, which we intend to conduct under the approval of the FDA. The Innovative technology, the completion of the Pre-IND stage as mentioned, and the rapid progress, help the company to advance the operative, strategic and business steps in this area”.

Professor Tamir Ben-Hur, Head of the Department of Neurology and Brain Division in Hadassah Medical Center in Jerusalem, and an advisor for the company, noted: "Kadimastem is progressing in a logical and correct manner towards clinical trials in ALS. This includes proof of concept of the efficacy of the transplantation of cells in pre-clinical trial models, definition of the preferred path of injection of the cells and establishing the safety of this treatment".

 

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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