Gamida Cell has signed an investment and option agreement with Novartis. The option is exercisable for a limited period of time following achievement of certain milestones connected to the development of NiCord®, anticipated to be met during 2015. Completion of the transaction is subject to customary closing conditions.
Upon exercising the Option, Novartis would pay the other shareholders in Gamida Cell (the Sellers) cash payments of approximately $165 million, in accordance with the terms of the Agreement. In addition, the Sellers will be entitled to potential future payments which can reach a total of $435 million, depending on certain development and regulatory milestones and on sales of Gamida Cell's products.
Gamida Cell president and CEO Dr. Yael Margolin said, “The investment and option agreement announced today demonstrates Novartis’ belief in the potential of Gamida Cell’s platform technology, product pipeline and team. Novartis is a leading pharmaceutical company with a declared commitment to further expand and develop its pipeline of cell therapy products. This, combined with the breadth of its resources and experience, positions Novartis perfectly for an alliance with Gamida Cell.”
Gamida Cell chairman of the board and Clal Biotechnology Industries’ CEO Mr. Ruben Krupik said, “Every company experiences successes and challenges during the course of its evolution. It is the ones with particularly brilliant scientists and astute management teams that are able to successfully persevere to bring good products to the market. We believe that Gamida Cell is in this category. The investment and option agreement with Novartis could bring the company a significant step closer to this important goal.”
Gamida Cell is currently engaged in a Phase I/II study of NiCord® as an investigational therapeutic treatment for hematological malignancies such as leukemia and lymphoma. In this study NiCord® is being used as the sole stem cell source. NiCord® is derived from a single cord blood unit and expanded and enriched with stem cells using Gamida Cell’s proprietary NAM technology.
In a standard individual cord blood unit, the limited number of stem cells compromises successful engraftment in an adult patient. To circumvent this obstacle, physicians currently provide patients with two cord blood units to achieve therapeutically meaningful cell numbers. The positive clinical results of a Phase I/II clinical study using a double cord protocol, NiCord® (the expanded cord blood unit) along with an un-manipulated unit, showed early and durable engraftment with the un-manipulated unit disappearing in most of the patients.
These results were the basis for studying NiCord® as a ‘single’ expanded unit without co-infusion of a second un-manipulated cord. NiCord® may provide a single unit of expanded cord blood with clinical results comparable to those seen in a double cord setting, thus introducing a paradigm shift in treatment practice. The current Phase I/II single cord study of NiCord®, the first of its kind, is soon to be followed by a Phase III study planned to begin at the end of 2015.
Additionally, recruitment continues for Gamida Cell’s Phase I/II study of NiCord for pediatric sickle cell disease. SCD affects 90,000 to 100,000 in the US alone. Symptoms range in type and severity. However, SCD can be fatal. To date, the only known cure for SCD is stem cell transplantation from a family related matched donor.