Novartis AG Remains Hush-Hush About CAR-T Trial But the FDA is Impressed and Thinks It's a Breakthrough

Published: Apr 20, 2017

Novartis AG Remains Hush-Hush About CAR-T Trial But the FDA is Impressed and Thinks It's a Breakthrough April 18, 2017
By Mark Terry, Breaking News Staff

Although Switzerland-based Novartis is keeping the data mostly to themselves, they did allow the U.S. Food and Drug Administration (FDA) to have a peek at its data for CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapeutic. And the FDA apparently liked what it saw, granting the compound Breakthrough Therapy designation for adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more previous therapies.

“At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL,” said Vas Narasimhan, Global Head of Drug Development and chief medical officer of Novartis, in a statement. “We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible.”

Novartis inked a global collaboration deal with the University of Pennsylvania in 2012 for CTL019, which was developed by the university. Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications. The FDA accepted its Biologics License Application (BLA) for CTL019 for the treatment of r/r pediatric and young adult patients with B-cell ALL in March 2017.

“BDTs are increasingly common,” writes John Carroll, for Endpoints News, “but this one is a bit different than your run-of-the-mill open door promise at the agency. While Novartis and Kite have been running neck and neck to get the first pioneering approval for a CAR-T, Novartis is taking its first shot at children with acute lymphoblastic leukemia while Kite shoots for DLBCL. Kite has put its DLBCL data up for the world to see, at 3 and 6 months, and now CAR-T observes want to see how they match up in the coming rivalry.”

Carroll argues that the FDA granting a BTD to Novartis suggests that the company might have a lead over Kite, but it’s no guarantee. Kite’s CAR-T, KTE-C19, also received a BDT for DLBCL, transformed follicular lymphoma and primary mediastinal B-cell lymphoma. Actually making a decision on which is better—at least in a head-to-head comparison of the drugs in the same diseases—will require Novartis to actually publicly release data.

The Breakthrough Therapy designation was based on results from a multi-center Phase II JULIET trial. It’s the second global CAR-T trial by Novartis. The first was ELIANA looking at CTL019 in r/r B-cell ALL. Novartis claims they will present data from JULIET at an upcoming medical meeting.

“We are encouraged by the FDA’s recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review,” said Carl June, director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania, in a statement. “Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers.”

Another company in the race is Juno Therapeutics . The company is reorganizing its CAR-T research-and-development group and recently hired Sunil Agarwal to head it. Prior to joining Juno, Agarwal was the chief medical officer and executive vice president at Ultragenyx.

“Sunil is a strong strategic thinker and experienced drug developer who will be a great addition to our executive team,” said Hans Bishop, Juno’s president and chief executive officer, in a statement. “He has an established track record of bringing innovative therapies to market, demonstrated by the breadth and quality of the programs he has led. His drug development experience, instincts, and scientific judgment will be important for our next phase of growth, and we will benefit from his experience and insight in both our strategy and execution.”

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