Kadimastem announced 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.
Kadimastem chief scientist Michel Revel said: "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."
Kadimastem CEO Yossi Ben-Yossef said: "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."