D-day: Novartis arrives at a crossroad in the blockbuster race to win the first FDA OK for CAR-T

Three years of intense competition and billions of dollars in investments into developing the world’s first CAR-T therapy has come down to the wire today with the first FDA panel review for Novartis’ leading CTL019 (tisagenlecleucel) therapy.

Observers crowded into the presentation room this morning, rubbing shoulders with CAR-T celebs like Penn’s Carl June with many, many more looking online as Novartis and regulators began the painstaking process of breaking down the manufacturing process that goes into the lengthy and complex task involved in making these drugs. While most of these panel reviews come down to assessing the data and how results stack up against the gold standard on safety and efficacy, the agency today wants their experts to carefully consider if these drugs can consistently be made to order in a safe fashion by manufacturers who are still fine tuning their delivery times. In addition, they’ll need to discuss the potential threat of a toxicity profile that has yet to be fully established or completely understood.

But they’ll be putting all that into context of a new therapy that offers a breakthrough approach in treating advanced cancers for patients with few, if any, options. And they’ll be doing it at a time that the FDA has committed itself to accelerating the development of major new therapies under FDA commissioner Scott Gottlieb.

Brad Loncar, an investor who runs the $CNCR immunotherapy ETF, captured the key issues in an early tweet from the meeting.

Victor Lu at the FDA was given the mission of explaining the process of extracting cells from patients, engineering them with chimeric antigen receptors and then whipping that into a therapeutic punch involving tens of millions of T cells. While Novartis and the close runner up in the field have been manufacturing CAR-T for relatively small numbers of trial subjects, Novartis is asking for an OK to shift into the commercial sphere.

Novartis has experienced failures in making therapeutic doses for all of its patients. And it’s been working hard to get the vein-to-vein time down to about 22 days, according to today’s testimony. If they get a positive vote today, and a subsequent formal OK, look for plenty of careful comparisons among the leaders in the field to see who can do the best job on manufacturing.

Arie Belldegrun, Kite CEO

Kite CEO Arie Belldegrun, whose team will be up for review in coming months, discounted the horse race language that I have used just about weekly as the leaders in CAR-T racked up progress and setbacks. In a blog post today, he said he’d be watching as a lead cheerleader for the rival team at Novartis.

The talk of a “race” makes for great prose, but my desk isn’t covered in betting sheets. It’s covered in stacks of scientific papers that shows the promise of CAR-T technology. Stacks that are growing. With every paper I read, it becomes more and more clear that CAR-T is not a biotech race to be won, but rather a revolutionary therapy to be explored and applied to other cancers. That’s a task larger than any single company.

Belldegrun worked hard to be the first up for an FDA panel review, delayed by demands for more mature results. But there is one advantage, from a horse race perspective, in its position as a runner up. A win for Novartis today will make Kite’s task of winning approval much more straightforward. Kite, though, will likely have to field even more pointed questions about cerebral edema, which recently killed one patient in a clinical trial and derailed the lead therapy at Juno.

There’s plenty of drama ahead — and not just in the vote to come up later in the afternoon.

I’ll keep you posted.

Image: today’s FDA panel Brad Loncar

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Sr. Manager, Regulatory Affairs, CMC
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