Over the past few months, large biopharmas Amgen and Merck Serono have joined industry pioneer Novartis and others in striking licensing deals to access chimeric antigen receptor (CAR)-based cellular immunotherapies. Merck Serono of Darmstadt, Germany, in late March paid $115 million upfront and paired up with Intrexon of Germantown, Maryland, to buttress its growing immunooncology efforts with the Intrexon's nextgeneration CAR T-cell platform. In January, Amgen struck a broad strategic alliance with Kite Pharma, which will see each company develop CAR T-cell therapies based on Kite's platform and Amgen's oncology target expertise. Although many see these therapies as a new order of medicine--Celgene in Summit, New Jersey, Pfizer in New York, and GlaxoSmithKline in London, have inked alliances to enter the field over the past couple of years--until recently, many large pharmas have been unwilling to commit, possibly holding out for next-generation therapies (Table 1).
CAR T- cell treatments are a kind of adoptive cell therapy that comprises the ex vivo modification of T cells to direct a response to a tumor antigen. Early-stage results in CD19-positive blood cancers were striking, leading to frothy valuations for the field's technological trailblazers, including Kite of Santa Monica, California, and Seattle-based Juno Therapeutics, as well as pharma giant Novartis, which was the first company to invest heavily in the space. (Under the terms of a recently settled patent infringement case, Novartis will pay Juno milestones and royalties on net sales of products incorporating a certain CAR component.)
Among large pharmas, Basel-based Novartis is still the exception. Since its August 2012 deal with CAR T-cell leaders at the University of Pennsylvania in Philadelphia, the large pharma has "doubled down in the area," in particular, investing in the complex manufacturing processes necessary to make cell therapies a reality, says Usman Azam, global head of Novartis's cell and gene therapies unit. The Swiss pharma purchased a Morris Plains, New Jersey, immunotherapy manufacturing facility from Seattle-based Dendreon in December 2012 for $43 million. That facility, where Dendreon had previously manufactured the first US Food and Drug Administration-approved autologous cell therapy Provenge (sipuleucel-T), will help Novartis scale up production.
It was seminal results from UPenn researchers...