- The Food and Drug Administration agreed Tuesday to review an approval application for ide-cel, an experimental cell therapy developed by Bristol Myers Squibb and partner Bluebird bio. The agency set a decision deadline just four days ahead of the expiration of Bristol Myers' offer to pay $6 billion extra to shareholders of the former Celgene, a key part of the pharma's deal to acquire the company in 2019.
- If approved, ide-cel could be the first engineered cell therapy that treats a blood cancer called multiple myeloma in patients previously treated with several other drugs. Its most direct competition initially could come from GlaxoSmithKline's antibody drug Blenrep, but other rival treatments are advancing as well.
- Celgene's pipeline was a key factor in Bristol-Myers' $74 billion acquisition of the big biotech in early 2019. However, both ide-cel and another engineered cell therapy that were critical to the extra payouts Bristol-Myers offered have had hiccups along the way, making it uncertain whether Celgene shareholders will receive the additional cash.
Celgene gained rights to ide-cel through a 2018 deal with Bluebird bio, the experimental therapy's original developer. When Bristol-Myers made its bid to buy Celgene, the two sides couldn't agree to the long-term value of ide-cel, the now-approved multiple sclerosis drug Zeposia and another cell therapy called liso-cel.
In order to finalize the deal, the two sides agreed that if Zeposia and liso-cel gained approval by Dec. 31, 2020, and ide-cel by March 31, 2021, Bristol-Myers would pay out another $9 a share through an offer called a "contingent value right," or CVR. Hitting those marks appeared likely until the FDA rejected Bristol-Myers' application for ide-cel before review, citing a need for documentation on manufacturing processes.
With the acceptance of the new application, the FDA has now set a March 27, 2021, decision deadline, leaving precious little maneuvering room ahead of the CVR's expiration. Liso-cel, meanwhile, has a Nov. 16 FDA decision deadline.
In a Sept. 22 note to clients, Mizuho analyst Salim Syed wrote that Bristol-Myers has said the FDA has not completed inspections of the liso-cel manufacturing facilities, nor has the agency even scheduled those inspections — a potential hurdle for an on-time approval.
Ide-cel and liso-cel are both a new type of treatment called a chimeric antigen receptor T-cell, or CAR-T, therapies. The treatment involves harvesting a patient's immune cells, genetically modifying them and then infusing them back into patients to attack tumors.
Ide-cel works by identifying a protein called B cell maturation agent, commonly found on multiple myeloma cells. GSK's antibody drug Blenrep also targets BCMA, but destroys tumor cells by delivering a chemotherapy agent. The difference between the two, for now, is that Blenrep can only be used after patients have progressed on four lines of treatment, while Bristol Myers aims for ide-cel to be used after only three lines.
The FDA's initial rejection of ide-cel, meanwhile, allowed another competitor to close the gap on Bristol-Myers. Johnson & Johnson has been advancing a candidate called JNJ-4528, which it licensed from China-based firm Legend Biotech. J&J could seek U.S. approval by the end of 2020.