Novartis will have the option to fully acquire Gamida Cell for $165m (€124m) in cash, depending on development milestones connected to Gamida’s NiCord, which are expected to come in 2015.
NiCord is currently being studied in a Phase I/II study as an investigational therapeutic for haematological malignancies such as leukaemia 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.
Gamida CEO Yael Margolin told us that NiCord is manufactured at the company’s base in Israel. She added that the investment agreement “does not include a license for the NAM technology.”
The NAM platform technology is now being applied to expand functional cells in culture, while attenuating the continuous morphological and functional alterations of cells during ex vivo cultures. The results of multiple studies using several animal models demonstrate that expanded cell grafts created using NAM technology displayed improved functionality following in vivo infusion. NAM technology is also expected to increase the clinical efficacy of therapeutic cell products.
In addition to the potential acquisition, Gamida will be entitled to potential future payments which can reach a total of $435m, depending on certain development and regulatory milestones and on sales of Gamida Cell's products.
Margolin added, “The investment and option agreement announced today demonstrates Novartis’ belief in the potential of Gamida Cell’s platform technology, product pipeline and team.”
In a standard individual cord blood unit, the limited number of stem cells compromises successful engraftment in an adult patient. To circumvent this, 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. The current Phase I/II single cord study will 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.