Just in time to assure a decision before CVR deadline, Bristol Myers Squibb files NDA for Celgene/bluebird CAR-T
A new CAR-T therapy may be coming. And maybe $9 per share for Celgene investors, too.
Bristol Myers Squibb announced they submitted an NDA for the multiple myeloma “ide-cel” CAR-T therapy Celgene developed in partnership with bluebird. The therapy is one of the three that has to be approved to unlock the BMS-Celgene contingent value agreement that would give shareholders of the absorbed NJ company $9 per share. The first, ozanimod, was approved last week. The second, a CAR-T treatment for non-Hodgkin’s lymphoma called liso-cel, was submitted to the FDA in December.
As Mizuho’s Salim Syed pointed out in a note to investors, the timing is not random. The March 31 submission means that even under standard review, the FDA will issue a decision by March 31, 2021, the deadline for approval under the contingent value agreement.
Strong numbers bolster BMS’s submission. At ASH in December, they released Phase II data showing that in 140 patients with relapsed and refractory multiple myeloma, around three quarters responded and around a third showed a complete response. In the most effective dosing arm, 81.5% responded and 35.2% went into complete remission. The median duration was 11.3 months.
BMS isn’t alone in the hunt, although they are for now the furthest along. At the same conference, J&J unveiled a batch of earlier stage data. It was smaller — 29 patients — but every patient responded by month 6. Both therapies target BCMA, or B-cell maturation agent, a protein expressed almost exclusively on malignant cells.
The therapy, though, comes with the same potent side effects as previous CAR-T treatments. Cytokine release syndrome, the potentially deadly side effect in which activated white blood cells send the immune system into inflammatory overdrive, occurred in 83% of patients in the ide-cel trial. However, only 5.5% were classified as “severe.”
Liso-cel and ide-cel would be the third and fourth CAR-T treatments approved since Novartis’s Kymriah and Gilead/Kite Pharma’s Yescarta and were approved in 2017, although a second CAR–T therapy from Kite Pharma also currently sits before the FDA.
After the ASH data, analysts expect both BMS CAR-Ts to clear the finish line, although Syed raised questions about whether, amid Covid-19, the company’s cell therapy manufacturing facility in Washington state had been and would be able to be FDA-inspected. But noting that liso-cel already was submitted in December and given priority review in February, he gave a 90% chance of ide-cel approval and wrote that “we would argue there is a decent chance here the plant has already been inspected and the concerns may be overblown.”
How long the therapy remains a top option for multiple myeloma patients is a different question. At ASH, BMS also showed data for CC-93269, a BCMA-targeting bispecific antibody that put up “crazy numbers,” as Baird analyst Brian Skorney put it: 89% ORR, 33% CR. Regeneron also showed off its BCMA bispecific. Safer and dramatically simpler to administer, these antibodies may ultimately win out, Skorney said.
”We think the true commercial opportunity is realized by the most safely combinable asset with generic Revlimid/Velcade,” he wrote. “As such, although further behind than BCMA-CART, we see these drugs leaping ahead in the treatment paradigm, barring major safety issues.”