Investors Beware: Thin CAR-T Data Could Make or Break Juno and Kite Pharma
September 22, 2016
By Mark Terry, BioSpace.com Breaking News Staff
CAR-T therapy is a hot property right now. Chimeric antigen receptor (CAR) T-cells are engineered molecules that are grafted onto a monoclonal antibody to attack specific tumor cells. At the moment, two biotech companies, Juno Therapeutics and Kite Pharma , are facing pivotal decisions by the U.S. Food and Drug Administration (FDA). Cheryl Swanson, writing for The Motley Fool, takes a look at some obstacles the two companies are facing.
Juno, headquartered in Seattle, was leading the way until it announced in July that it was placing it Phase II trial of JCARO15 on a clinical hold after three patient deaths. JCARO15 is a CAR-T therapy in patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL).
That was such bad news for the industry that most companies working with CAR-T saw their stocks temporarily tank as a result. The deaths were eventually linked to reactions to the chemotherapeutic agent fludarabine, rather than JCARO15. The trial was modified as a result, which pushed the company’s accelerated FDA approval expected in 2017 to the first half of 2018.
Swanson writes, “Based on very preliminary data, the drug, JCARO15, appears to be extremely effective. Its Phase I trial showed a complete remission rate of 89 percent in ALL. Juno is now recruiting for its Phase II ROCKET trial for the therapy.”
As a result of Juno’s modifications, Kite is probably going to be the first company to file with the FDA for a CAR-T therapy. The company’s application for KTE-C19 will be based on topline data on the first 51 patients to be treated. KTE-C19 is being evaluated in patients with an aggressive form of Non-Hodgkin’s lymphoma (NHL).
Kite has also been tossed around as a possible acquisition target for Gilead Sciences , although almost any big company wanting to bolster its immune-oncology offerings is probably keeping an eye on Kite and Juno.
Todd Hagopian, who has a biotech fund at Marketocracy, told Forbes recently, “I believe that the CAR-T market is going to be huge in the future, and Kite will be the first-mover in the segment. If Gilead is looking to grab a large piece of the CAR-T piece, they will look to acquire Kite by the end of the year. The stock will likely go for twice what it is worth at the moment.”
Of course, if for some reason the FDA rejects Kite’s application, perhaps due to trial size, the stock will plummet like a rock.
On the positive side, Juno is partnered with Celgene , who tossed $1 billion into their collaboration last year.
Kite, Swanson writes, “has various collaborations with well-regarded research centers, such as the National Cancer Institute, as well as Swiss pharma Roche Holdings working on a combo drug treatment with Kite’s lead product, KTE-C19.”
Both Kite and Juno have accelerated approval status.
Still, both companies are depending on somewhat limited data and some troublesome adverse effects. Swanson says, “With several fatalities already occurring, the FDA will need a thorough justification that the benefits of these treatments outweigh the known risks before approval.”