Novartis' leading CAR-T cancer therapy gains breakthrough status at the FDA

The FDA has provided its special "breakthrough" status for Novartis' ($NVS) closely-watched development program for a personalized CAR-T cancer therapy, promising to help speed development for engineered T cells.

Novartis CEO Joe Jimenez vowed to Forbes recently that he had essentially provided a blank check to the team at the University of Pennsylvania which is developing CTL019. The program relies on adapting T cells with chimeric antigen receptors. Patients' T cells are removed and then genetically modified with new DNA to program them to attack a specific target on cancer cells. The engineered T cells are then infused back into the patient, where they multiply in the body and proceed to hunt for cancer cells, helping to guard against recurrence.

What's the breakthrough designation mean for Novartis? There are no guarantees, but regulators in the cancer division under Richard Pazdur essentially are vowing to keep something of an open-door in place for the program. Pazdur's group has taken a leading role in providing accelerated approvals for cutting-edge treatments, often lopping out mid-stage trials and allowing developers to cut straight to pivotal work.

It's also a chance for Novartis to claim boasting rights for a full 5 BTDs, a rare feat in the industry, where breakthrough designations are used as evidence of R&D success and an effective focus on unmet medical needs. Novartis' top executive team is going all out on the cancer front. It essentially swapped out its struggling vaccines division to get ahold of GlaxoSmithKline's ($GSK) cancer drug portfolio. And it sees oncology as a major driver of future drug sales, with this kind of cutting-edge technology at the forefront.

David Porter

In a recent Q&A with FierceBiotech Editor John Carroll, Penn's David Porter called this program one of the most exciting he'd seen in his 20-year career.

"We've now treated 80-plus patients in our trials with CAR-T cells, and are seeing remissions stretching toward four years for first patients," Porter noted in early June. "This therapy, CTL019, could change the paradigm for treating patients with refractory (treatment-resistant) leukemia."

"This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population," said Penn's pioneering Carl H. June, who's taken the lead on the program. "We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in Phase II trials."  

The field is not without controversy. A Seattle-based start-up, Juno Therapeutics, is pursuing claims that June and Penn purloined some of its scientific founders' secrets when they got started. Novartis has promised to fight this one out every step of the way.

CAR-T research has been exploding, though. Just days ago Kite Pharma ($KITE) rode the CAR-T wave to a $128 million IPO. Last month, Pfizer ($PFE) signed a sweeping CAR-T development deal with Cellectis. Bluebird bio ($BLUE), which has been building its reputation in next-gen gene therapies, struck a deal with Celgene ($CELG) back in 2013 agreeing to explore Baylor's work in CAR-Ts. And two weeks ago bluebird acquired Pregenen, another Seattle company which had been launched by some of the scientific pioneers in the field.

- here's the release

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