Kadimastem - Stem Cell to Cure Diseases

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FDA Granted Kadimastem Orphan Drug Status for AstroRx® for the Treatment of ALS

Nov. 4, 2018

The FDA grants orphan drug status to incentivize the development of one of a kind drugs and other medical treatments. This status grants the company that manufactures the drug marketing exclusivity for 7 years from the date of receipt of marketing approval of the drug. The recognition of orphan drug status also enables accelerated examination and response paths from the FDA and other regulatory authorities.

The granting of orphan drug status for the AstroRx® product is an acknowledgment of the uniqueness and medical potential of the product and will give a significant boost to its entry into the market once it's approved for marketing.

Kadimastem is progressing in the Phase 1/2a clinical trial in the Hadassah Ein Karem Hospital. The company completed enrollment of 5 patients for Cohort A and began cell transplantation in the patients. Results for Cohort A of the trial are expected in mid-2019.

AstroRx® is a unique, first in human, cell therapy product for ALS. The product consists of nerve-supporting cells (astrocytes), which are produced at Kadimastem from pluripotent stem cells with unique technology developed by the company. Treatment is administered by a simple injection into the spinal cord fluid of ALS patients, the advantage of AstroRx® is to replace the poor functioning of the supporting cells in the patient, with healthy cells.

Mr. Yossi Ben-Yosef, CEO of the company, said: "I am proud that the medical product in which we are conducting a clinical trial today, AstroRx®, has received orphan drug status. The FDA has provided further confirmation of the uniqueness and great medical potential of the product. I believe that the approval will increase the possibility of reaching the market faster and will provide significant advantages in marketing the product."

Prof. Michel Revel, Kadimastem's Chief Scientist, commented: "The FDA's decision is a recognition of the uniqueness and excellence of the Kadimastem's flagship product, which is in a clinical trial for the treatment of the severe neurological disease ALS. This recognition is a badge of honor for the company and will help accelerate the delivery of AstroRx® cells for use in ALS patients worldwide."

 About Kadimastem

Kadimastem Ltd. (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.


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