A long-expected deal between Novartis and Gamida Cell was finally revealed yesterday, with the Swiss pharma giant paying $35m for an equity stake and an option to buy the Israeli stem cell researcher in the next couple of years. While the terms of transaction undoubtedly reflect this high-risk research area, it is clear that Novartis is committed to novel cell therapies.
Gamida believes that it has developed a technology that can overcome some of the major problems faced by stem cell transplants – notably, the need for follow-up infusions. With data due next year from two small studies of its lead clinical asset, NiCord, success would provide Novartis’s growing cell therapy unit another very novel area of science to pursue.
Expanding and grafting
Umbilical cord blood is a rich source of stem or progenitor cells, but each cord blood unit contains very little material. Numerous techniques have been developed to expand these cells – Gamida has developed a platform called NAM that does this.
It also hopes to show that the expanded cells that the NAM platform generates are better at forming lasting grafts. Although expanded stem cells have been shown to decrease the time to neutrophil engraftment, they disappear shortly after, and long-term engraftment must be provided by a second unit of un-manipulated cord blood.
The clinical and commercial potential of a technology that could remove the need for a second unit of unadulterated cord blood is clear. A trial is already under way to show this, with NiCord, Gamida’s lead project generated from the NAM platform.
The trial is run by Duke University Medical Center, and seeks to recruit around 10 patients with various haematological malignancies. The company announced last September that the first person in the study had been successfully transplanted. The main objectives are to measure NiCord-derived neutrophil engraftment 42 days after transplantation and to assess the incidence of secondary graft failure at 180 days.
A second study is ongoing in sickle cell anaemia, but patients are being co-transplanted with a secondary un-manipulated cord blood unit as well as NiCord. The single-arm study is also taking place at Duke University; primary endpoints are safety and the assessment of neutrophil engraftment at day 42, while secondary survival endpoints are being measured at 100 and 180 days.
Novartis’s option to buy the company can be exercised for a limited period of time after achievement of certain unspecified milestones connected to the development of NiCord. Although these were not specified they are expected to be met during 2015, so are likely to comprise data emerging from these studies, in particular the first trial only using the NiCord expanded cells.
Adding to the pipeline
Exercising the option would cost Novartis another $165m, while a further $435m could be paid out in future if development, regulatory and sales milestones are hit.
The Gamida technology clearly has much to prove before Novartis gets out its cheque book again. However, of all the big pharma players, Novartis undoubtedly knows this space.
At an investor day a couple of months ago the company described cell therapy as one of its core areas of research, and it recently created a cell and gene therapy unit. Most visibly it is working on various CART programmes, or personalised T-cell therapies, a hot area of immuno-oncology that could have applications in both haematological and solid tumours (ASH – Novartis pulls the CART in autologous T-cell therapy, December 9, 2013).
It also forged a research collaboration with Regenerex last September over its haematopoietic stem-cell based platform, FCRx. Work is initially focusing on kidney transplant; the companies believe that it has the potential to promote graft survival without the need for lifelong immunosuppression.
An internal project that appears to be attempting the same thing as NiCord is already in a phase II study. Novartis describes HSC835 as a novel cell therapy approach that enables an expanded, single umbilical cord blood-derived hematopoietic stem cell transplant. It is also being tested in patients with high-risk hematological malignancies, with data due in 2016.
The Gamida investment is clearly part of a broader investment from Novartis. And one that is likely to be followed by others.