Attaching the umbilical cord

Novartis invests $35M in Gamida Cell with eye on stem cell therapies

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JERUSALEM—Last year, the Swiss pharma giant Novartis had a deal in place to buy Israeli stem cell research company Gamida Cell for $600 million, but Novartis officials changed their minds. Now Novartis is back, apparently interested in Gamida’s novel NiCord stem cell therapies.
According to the terms of the present deal, Novartis will make an initial payment of $35 million and receive 15 percent equity and an option to fully acquire Gamida Cell, which has signed an investment and option agreement with Novartis. The option can be exercised for a limited period of time following achievement of certain milestones connected to the development of NiCord, expected to be achieved during 2015. Then, Novartis will have the right to buy the Jerusalem-based company for $165 million up front and $435 million in specified milestones. The buyout option expires in the first half of 2016.
According to Gamida Cell President and CEO Dr. Yael Margolin, the agreement “demonstrates Novartis’ belief in the potential of Gamida Cell’s platform technology, product pipeline and team.” She believes that Novartis’ resources and commitment to further expand and develop its pipeline of cell therapy products will “position Novartis perfectly for an alliance with Gamida Cell.”
While umbilical cord blood is a rich source of stem or progenitor cells, each cord blood unit contains very little material. Gamida has developed a platform called NAM that expands these cells.
Gamida Cell is involved in a Phase 1/2 study of NiCord as an investigational therapeutic treatment for hematological malignancies such as leukemia and lymphoma to prove the value of this platform. NiCord is being used as the sole stem cell source in the study. Derived from a single cord blood unit, NiCord is 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 grafting in an adult patient. To keep this from happening, physicians provide patients with two cord blood units to achieve therapeutically meaningful cell numbers. Clinical results from a Phase 1/2 clinical study using a double cord protocol showed that NiCord (the expanded cord blood unit), along with an unmanipulated unit, demonstrated early and durable grafting, with the unmanipulated unit disappearing in most of the patients.
NiCord, which is being studied as a single expanded unit without co-infusion of a second unmanipulated cord, may provide clinical results comparable to those seen in a double cord setting, thus drastically changing treatment practice. The current Phase 1/2 single cord study of NiCord, the first of its kind, will be followed by a Phase 3 study that will begin at the end of 2015.
Gamida Cell is also recruiting for a Phase 1/2 study of NiCord for pediatric sickle cell disease, which affects 90,000 to 100,000 people in the United States alone and can be fatal. Currently, the only known cure for the disease is stem cell transplantation from a family-related matched donor.
Gamida Cell is also known for another cancer therapy: StemEx, a package of cells extracted from cord blood, ameliorated with Gamida Cell's platform technology and intended for leukemia and lymphoma patients unable to arrange a matching bone marrow transplant from a family member. Last year, the company's plan to seek an early approval of StemEx based on a Phase 2/3 single-arm study was rejected by the U.S. Food and Drug Administration, which insisted on a full late-stage study. By that time, Teva, which is one of Gamida’s shareholders, had already decided to stop working on joint projects related to StemEx, and Gamida Cell sought a new Big Pharma collaborator to complete the work.
Novartis describes cell therapy as one of its core areas of research. If Gamida Cell succeeds, it could be a key part of it.

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