Bristol-Myers is making a bee-line to the FDA with positive liso-cel data — but is it too late in the CAR-T game?
Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.
Provided there are no snafus or even modest stumbles now, they should get an OK within the 2020 timeline lined out in their $9 CVR for Celgene — the first in a trifecta of approvals required for a payout.
Whether they can go on to make it into a viable commercial therapy, though, is a whole other thing.
At first glance, there isn’t anything about the safety and efficacy data that would force a CRL or delay. In a large trial of patients with relapsed/refractory large B-cell lymphomas investigators tracked an outstanding 73% response rate and 53% complete response rate in heavily pretreated patients with few options.
That’s in line with Yescarta from Gilead’s Kite, which snagged an approval more than 2 years ago, just after Novartis’ Kymriah came through.
Then there’s safety. To be sure, the drug has safety issues. There were 4 patients in the study who died after treatment. Several others died for unrelated issues. But…with only a 2% rate of cytokine release syndrome, Bristol-Myers has a shot at a differentiated safety profile.
“There is a potential these patients can be treated on an outpatient basis,” says Samit Hirawat, the chief medical officer at Bristol-Myers. He noted that 26% of patients were never admitted, while 76% were admitted 4 days or later after therapy. The key is to treat them at a hospital with the infrastructure to provide care 24/7, so a patient can be admitted at any time later if needed.
How that will fly with payers after rivals have been on the market for about 3 years, with new evidence that earlier use of steroids can dramatically reduce CRS and considerable durability of response, will have to be seen.
But time is not on Bristol-Myers’ side. Liso-cel is the long delayed followup to the disastrous JCAR015, which killed a number of patients. Their setback threw them years off schedule. And rivals are advancing off-the-shelf alternatives or other new approaches that could knock the pioneers completely out of the game. In the meantime, Bristol-Myers is gathering its own durability data and will gradually see if their mix of CD4 and CD8 cells can do better.
The main interest now is in the timeline around the approval. Hirawat says they’ll ask for priority review, and there’s every reason to believe that regulators will move swiftly — barring a nasty surprise.