Pharmaceutical companies developing treatments for blood diseases will present data from clinical trials at the American Society of Hematology's annual meeting Dec. 7-10, showcasing cell therapies and a mechanism called Bruton's tyrosine kinase inhibition, as well as new uses for approved drugs.
Cancer treatments are set to dominate the American Society of Hematology, or ASH, conference as late-breaking abstracts include data from Amgen Inc., Sanofi, GlaxoSmithKline PLC and Celgene, recently acquired by Bristol-Myers Squibb Co. in a $95 billion bet on cell and cancer therapies.
Amgen will present two late-stage studies — one for its immune system-charging cancer medicine Blincyto in children with leukemia and another for its cancer therapy Kyprolis in patients with multiple myeloma.
Also in multiple myeloma, Celgene's phase 3 study dubbed Quazar AML-001 will demonstrate the effectiveness of the company's oral drug CC-486, and a study from GSK will show how the drug GSK2857916 performs in patients whose multiple myeloma has returned.
Sanofi, on the other hand, is taking a different route from its cancer-focused competitors at ASH with its plans to reveal results from a study of sutimlimab, a therapy for the rare anemic condition cold agglutinin disease. Cowen analyst Steve Scala said in an October note that the sutimlimab results would be a key near-term catalyst for the French company.
Cell therapy on the rise
Several companies have lined up cell therapies at 2019's ASH conference, presenting data for a new wave of treatments that are approaching regulatory approval.
Data for Celgene's cell therapy liso-cel will be important for the company's new owner, Bristol-Myers. With plans to file for U.S. Food and Drug Administration approval by the end of the year, the treatment is one of a few assets on which Bristol-Myers based its valuation of Celgene. The companies will present results from the study called Transcend in patients with large B cell lymphoma.
SVB Leerink analyst Geoffrey Porges said in an October note that the planned submission for liso-cel would place a potential U.S. approval in mid-2020. The unveiling of the full Transcend results at ASH will be important for its market future: In the study, four patients died of treatment-related issues, but the cell therapy was effective in 73% of patients. Liso-cel would be competitive with Yescarta from Gilead Sciences Inc. and Kymriah from Novartis AG, both of which are already on the market.
Johnson & Johnson will present results from two studies involving its own cell therapy treatment called JNJ-4528 in multiple myeloma. The therapy reduced cancer in 90% of patients, according to the Cartitude-1 study, and the company will present full results Dec. 9.
J&J and its Chinese partner Legend Biotech Corp. also ran another study comparing two types of manufacturing practices — the same therapy with different manufacturing, called LCAR-B38M in this case, showed only slightly less efficacy than in the Cartitude study, according to top-line results, reducing cancer in 88% of patients.
Battle of the BTK inhibitors
Next-generation Bruton's tyrosine kinase, or BTK, inhibitors — a type of treatment that blocks a signaling pathway on which certain blood cancers are highly dependent — have yielded divisive results leading up to the ASH conference, with clear winners and losers in topline data.
AbbVie Inc.'s Imbruvica is the leader in chronic lymphocytic leukemia, or CLL, but new data from AstraZeneca's Calquence — recently approved by the FDA — could help the British company take some of that market share down the road. Full data from a study called Elevate to be presented at ASH showed a survival rate of 90% for 30 months in patients taking a combination of Calquence and Roche Holding AG's Gazyva. Calquence has found success in CLL in previous studies, as well.
Also in CLL, Eli Lilly and Co. will showcase the first human data for its BTK inhibitor Loxo-305, which the company picked up in its acquisition of Loxo Oncology early in 2019.
AstraZeneca's and Lilly's BTK results stand out against data from potential competitor Sunesis Pharmaceuticals Inc. Leerink analyst Jonathan Chang said in a Dec. 5 note that Sunesis's vecabrutinib results disappointed investors and were positive for competitors, which also include ArQule Inc. ArQule is set to present its own CLL results at ASH.
Chang said the Sunesis data "effectively removes one competitive threat from the equation" while investors wait for ASH updates from the other three companies.