Novartis invests in novel stem cell technology

Novartis is to invest $35 million in Israeli company Gamida Cell, to develop novel stem cell treatment NiCord for blood cancers such as leukaemia and lymphoma.

In return, Novartis will receive 15 per cent equity in the company and an option to acquire Gamida Cell, subject to certain milestones being reached in relation to NiCord.

The deal follows the Swiss pharma giant’s aborted attempt to buy Gamida Cell earlier this year for around $600 million. The new agreement means that if Novartis exercises its option to purchase the company, it would make cash payments of approximately $165 million to the other shareholders, plus potential future payments totalling $435 million, subject to certain development and regulatory milestones and sales.

NiCord is currently undergoing phase I/II trials into haematological disorders, in which it 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. If successful, it could represent a paradigm shift in treatment practice, moving away from use of two cord blood units to the NiCord double cord protocol.

In a standard individual cord blood unit, the limited number of stem cells compromises successful engraftment in an adult patient. To circumvent this obstacle, doctors currently provide patients with two cord blood units to achieve therapeutically meaningful cell numbers. Positive clinical results in phase I/II, using NiCord 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. A phase III study is scheduled to start at the end of 2015.

NiCord is also undergoing phase I/II trials for paediatric sickle cell disease (SCD), for which recruitment is ongoing. To date, the only known cure for SCD is stem cell transplantation from a family related matched donor.

Gamida Cell’s shareholders include Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva and Amgen. Its pipeline of stem cell therapy products is in development to treat conditions including blood cancers, solid tumours, non-malignant haematological diseases such as sickle cell disease and thalassemia, neutropenia and acute radiation syndrome, autoimmune diseases and genetic metabolic diseases, as well as conditions that can be helped by regenerative medicine.

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