News that six Celgene Corp. trials for blood cancer have been put on either full or partial hold was seen as a minor setback for the immuno-oncology juggernaut.
Celgene's Fusion program was paused by the U.S. Food and Drug Administration after risks came to light two months ago in a trial pairing Merck & Co. Inc.'s Keytruda with two Celgene drugs, Revlimid and Pomalyst, to fight multiple myeloma. A Bristol-Myers Squibb Co. trial combining its Opdivo with the two Celgene drugs for the same disease was also halted on the FDA's order.
The Fusion trials partnered Celgene therapies with Imfinzi, an AstraZeneca PLC drug that strengthens a body's immune capacity to target and fight cancer. Trials placed on partial clinical hold include three for multiple myeloma, one for high-risk diffuse large B cell lymphoma, and one for lymphoma and leukemia, all in the phase 1 or 2 stage.
A phase 1b trial using Imfinzi and Celgene's Revlimid against multiple myeloma in newly diagnosed patients was put on full hold.
Revlimid has led the multiple myeloma space on its own and is expected to be the top cancer treatment in 2022 with more than $14 billion in estimated worldwide sales, according to a report from market intelligence group Evaluate. The trials with Merck, Bristol-Myers and AstraZeneca paired the drug and other Celgene products with a relatively new class of cancer treatments that target PD-1 or PD-L1 proteins in the body to block cancer's spread.
The Fusion program pause suggests that the FDA is concerned about potential risks for all PD-1/PD-L1 inhibitors in combination with either Pomalyst or Revlimid, Credit Suisse analyst Vamil Divan said in a note.
The day before the FDA decision to pause the studies, Celgene CFO Peter Kellogg expressed excitement about the Fusion trials results coming out in the fall during a presentation at the Baird Global Healthcare Conference.
Asked about Merck's trial hold for multiple myeloma, Kellogg said Celgene is "taking time to take a look at what we saw there and talk to regulatory agencies."
The holds are meant to allow a deep dive into safety data to devise better safety mitigation and management, Canaccord Genuity analyst John Newman said in a Sept. 7 note. While the FDA actions could unsettle investors, as the holds are not specifically targeting the Fusion program, this is a minor negative for Celgene, he added.
Canaccord Genuity maintained its "buy" rating on Celgene.
It was always unlikely that the PD-1/PD-L1 class would be successful in every indication studied, Divan said. He added that for Bristol-Myers, Credit Suisse had not included multiple myeloma in its Opdivo sales forecasts so estimates are still on track.
Besides its Imfinzi partnership, Celgene is also co-developing BCG-A317, an anti-PD-1 cancer therapy like Merck's Keytruda, with BeiGene. However, the drug is targeting solid tumors at this time, leaving it out of what seems to be FDA concern based on blood cancer rather than the immunotherapies themselves.
"I think it's becoming clear to everybody that for the PD-1 and PD-L1 assets, it's not really going to be that much of a monotherapy game; it's going to be a combo therapy game," Kellogg said when discussing the BeiGene deal licensing its PD-1 asset. "To me, that transaction was a real win-win, and it gives us access to an exciting PD-1 program."