SAN DIEGO – Early data on an antibody-drug conjugate (ADC) under development at Immunogen Inc. drew attention during the American Society of Hematology (ASH) Annual Meeting and Exposition for its potential meaning to Jazz Pharmaceuticals plc, which has an option on the candidate and two other Immunogen ADC programs.

SAN DIEGO – Incyte Corp.'s Jakafi (ruxolitinib), already approved for treating myelofibrosis (MF), polycythemia vera and thrombocythemia MF, could soon add steroid-refractory acute graft-vs.-host disease (GVHD) to its list of FDA-approved indications. If an upcoming FDA decision goes its way, it would be the first drug approved for acute GVHD in the U.S. The review, based on the phase II Reach1 study, is expected to yield a decision by Feb. 24.

SAN DIEGO – The dawn of regulator-approved chimeric antigen receptor (CAR) T-cell therapies such as Kymriah (tisagenlecleucel, Novartis AG) have provided doctors with important new options for treating people with several types of aggressive blood cancers. But how long the new therapies can help and whether their costs can be made sustainable remain open questions. New research presented at the American Society of Hematology (ASH) Annual Meeting and Exposition is shedding light on the duration question with what City of Hope's Joe Alvarnas called "practice-changing" results.

SAN DIEGO – Initial data from an ongoing phase I/II study of a CD19-directed CAR T-cell candidate under development at Celgene Corp. found that 81 percent of the 16 patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) treated so far responded to the medicine, with 43 percent of them demonstrating a complete response. The majority of patients evaluable for minimal residual disease (MRD), a measure that correlates with improved outcomes in the indication, achieved that state early on in their treatment.

Viacyte Inc., a company developing allogeneic cell replacement therapies for people with diabetes, has raised an $80 million series D financing to support further testing of its regenerative medicine approach. Bain Capital Life Sciences led the round, joined by TPG and RA Capital Management, as well as existing investor Sanderling Ventures and individual investors.

Marking its first regulatory win in the U.S. blood cancer space, Tokyo's Astellas Pharma Inc. has secured approval from the FDA for its oral acute myeloid leukemia (AML) therapy, Xospata (gilteritinib). Already approved in Japan, the FMS-like tyrosine kinase 3 (FLT3) inhibitor is now approved in the U.S. for the treatment of relapsed or refractory AML patients who test positive for the mutation, which is often associated with increased risk of relapse and poor survival.

A phase III trial testing a combination of Bristol-Myers Squibb Co.'s Opdivo (nivolumab) and Yervoy (ipilimumab) as maintenance therapy in some patients with extensive-stage small-cell lung cancer (SCLC) found it failed to improve overall survival vs. placebo. Results of the study, called Checkmate-451, together with Opdivo's recent failure to beat chemotherapy's OS benefit in relapsed SCLC during the confirmatory Checkmate-331 trial could put its accelerated approval in SCLC in jeopardy, Evercore ISI analyst Umer Raffat said.

Cardiovascular safety concerns about Zafgen Inc.'s midstage type 2 diabetes (T2D) candidate, ZGN-1061, moved the FDA to push pause on the company's planned U.S.-based trial of the drug with a clinical hold. The setback triggered memories of a death tied to an earlier and now-discontinued candidate Zafgen had advanced from the same class, MetAP2 inhibitors, sending company shares (NASDAQ:ZFGN) down 40.6 percent to $5.41 on Monday.

For anyone not hip-deep in the hot field of artificial intelligence (AI), its role in drug discovery — or in any endeavor, for that matter — can feel vague, especially relative to the concrete problems drugmakers face daily. The very definition of AI can seem fuzzy, touted at times as capable of doing everything from organizing your photos to driving your car. Yet, in the biopharma space, as dollars flow in and candidates rise up, clarity is surfacing, too, program by program, bringing with it a more down-to-earth explanation of how the technology is reshaping the discovery enterprise.