Kadimastem Approaches the FDA for a Pre-IND Meeting
Sep. 2, 2015
The outline for the company’s phase I/IIa clinical trial will be determined in the talks that will follow with the FDA.
Ness Ziona, Israel, September 2, 2015, Israeli biotechnology company Kadimastem (TASE: KDST) announces today that it approached the FDA requesting a Pre-IND meeting, in order to discuss the development of the company’s cell-based therapy for the treatment of Amyotrophic Lateral Sclerosis (ALS).
The aim of the meeting is to present the complementary results of the proof-of-concept animal model trial to the FDA, to lay out the product manufacturing plan (chemistry, manufacturing and controls), to outline the future framework for safety trials and to present the clinical framework for phase I/IIa clinical trials. The result of the Pre-IND meeting will outline the agreed-upon path towards the application for clinical trials in humans, the IND, and the actual future clinical trials.
As preparation for the meeting, the company undertakes to submit to the FDA a comprehensive information package which includes the subjects to be discussed in the meeting.
The company recently reported success in a pre-clinical trial for the treatment of ALS in an additional animal model. The results of the trial demonstrate the efficacy of Kadimastem’s cell-based treatment for ALS. The trial tested the efficacy of injecting the brain supporting cells (astrocytes) produced through the company's unique technology from pluripotent stem cells. The astrocytes were injected into the spinal fluid of ALS rat model, and have shown an improvement in the rats’ life expectancy and motor functioning.
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 spinal cord, 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.
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.
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.