While several targeted therapies are approved for acute myeloid leukemia, a 2023 U.K. study found that median survival following diagnosis was only about seven months, highlighting the need not only for new therapies, but for a faster regulatory strategy. At the American Society of Hematology’s 67th annual meeting in Orlando Dec. 6, researcher Jesse Tettero presented data supporting the use of a measurable residual disease (MRD) surrogate endpoint in acute myeloid leukemia (AML) research. Through the Harmony Alliance study of 1,858 pooled patients using the MRD endpoint, “we could identify effective therapies earlier than overall survival, so that could be a benefit of four to five years. It, therefore, enables potential accelerated approval” if regulators like the FDA and EMA support using MRD as a surrogate endpoint. “When supported, it could lead to faster and smarter trials for AML patients,” said Tettero, a postdoctoral associate at Virginia Tech’s Fralin Biomedical Research Institute in D.C.

ASH 2025: Casgevy for kids? Expanding, improving SCD gene therapies

Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster. “It is not that long ago that there was public speculation about whether gene therapies for these diseases would ever be available for patients,” said Joanne Lager, chief medical officer of Genetix Biotherapeutics Inc., formerly Bluebird Bio Inc. Lager participated in a briefing Dec. 3 previewing oral presentations scheduled for the American Society of Hematology’s (ASH) 67th annual meeting in Orlando, Fla., Dec. 6-9. Her talk on Dec. 8 focused on what her company learned from creating a commercial gene therapy operation. Another gene therapy presentation on Dec. 6 rolled out results of exa-cel, the first and only approved CRISPR/Cas9 gene editing therapy, in pediatric patients with both transfusion-dependent beta-thalassemia or SCD with recurrent severe vaso-occlusive crises.

ASH 2025: New blood cancer therapies trump chemo, older drugs

Targeted therapies and immunotherapies continue to show better results than chemotherapy in investigator-initiated and company-sponsored cancer trials, and newer options demonstrate improvements over older ones, supporting potential shifts in how patients are treated. Lorenzo Falchi, of Memorial Sloan Kettering Cancer Center, reported at the American Society of Hematology’s meeting on a phase III study, published Dec. 7 in The Lancet, showing that a chemo-free fixed-duration regimen of CD3-CD20 bispecific antibody epcoritamab (Epkinly, Genmab A/S and Abbvie Inc.), monoclonal antibody rituximab (Rituxan, Roche’s Genentech) and immunomodulator lenalidomide (Revlimid, Bristol Myers Squibb Co.), vs. rituximab and lenalidomide (R²), led to a robust and lasting response in relapsed or refractory (r/r) follicular lymphoma patients. Another study, Bruin CLL-314, a randomized phase III published Dec. 7 in the Journal of Clinical Oncology, suggested that noncovalent BTK inhibitor pirtobrutinib (Jaypirca, Eli Lilly and Co.) offered equivalent or better outcomes compared to the older BTK inhibitor ibrutinib (Imbruvica, Johnson & Johnson and Abbvie) in treatment-naïve and r/r chronic lymphocytic leukemia and small lymphocytic lymphoma.

Positive phase Ib SCD data perk Fulcrum stock

Shares of Fulcrum Therapeutics Inc. (NASDAQ:FULC) were trading at $13.99, up $5.09, or 57%, on positive initial results from the ongoing 20-mg dose cohort of the phase Ib Pioneer trial with oral, once-daily fetal hemoglobin (HbF) inducer pociredir in sickle cell disease (SCD). Mean absolute HbF increased by 9.9% at six weeks of treatment with pociredir vs. 5.6% at the sixth week and 8.6% at week 12 in the 12 mg cohort, increasing from a baseline of 7.1% to 16.9%. As of the Nov. 11, 2025, data cutoff, seven of 12 patients (58%) achieved absolute HbF levels ≥20% at the sixth week, and all patients turned up a robust HbF increase. HbF levels of 20% are associated with about 90% of patients experiencing zero vaso-occlusive crises per year, noted Fulcrum.

Wave Life, Structure and Ascletis post wins in obesity management

Obesity management drugs continue to move forward in producing weight loss and in moving the market. Wave Life Sciences Ltd. saw its stock (NASDAQ:WVE) more than double at midday, with shares at $16.87 each, a jump of 125%, in the wake of positive phase I interim data showing WVE-007 produced fat loss much like a GLP-1 at three months but without muscle loss. Structure Therapeutics Inc.’s stock (NASDAQ:GPCR) had doubled at midday, with shares up 100% at $69.47 each, behind positive phase IIb top-line results from its oral GLP-1 receptor agonist aleniglipron. Clinically meaningful weight loss also was found in the placebo-controlled phase II study of Ascletis Pharma Inc.’s ASC-30, an oral small molecule GLP-1 receptor agonist. The three dosage levels hit their primary endpoints in the clinical trial.

Ireland plots to safeguard US investment in its biomanufacturing sector

Ireland is taking stock of the implications Trump administration tariffs and drug pricing policies hold for its world-leading biomanufacturing sector, which has been largely built on the back of investment by U.S. pharma companies – and is hugely reliant on exports to the U.S. The country of just under 5.5 million people is the world’s third largest exporter of pharmaceuticals, at more than €305 billion (US$355 billion) per annum, of which 43% by volume goes to the U.S.

14 psoriasis therapies projected to launch in China by 2027

Fourteen therapies to treat moderate to severe psoriasis are expected to enter the Chinese market in the next two years, according to Clarivate and BioWorld reports. Eleven of them are being developed by domestic biopharmaceutical firms. Novartis AG’s Cosentyx (secukinumab) has led the market in China since its 2019 launch. Competition, however, is expected to intensify over the next decade, potentially shifting sales rankings. “Emerging therapies will garner nearly $430 million (about 34% of the total market) in 2034,” according to Clarivate’s October DRG analysis. “Among these, we anticipate that Johnson & Johnson Innovative Medicine’s [oral drug] icotrokinra (JNJ-2113) will be the market share leader, contributing about 8% of total sales.”

Also in the news

Amneal, Amylyx, Ascentage, Biogen, BMS, Cantargia, Capricor, Cervomed, CG, Galimedix, Guard, Immatics, Impact, Jubilant, K36, Kala, Mural, Oric, OTR, Praxis, Protara, Rapport, Stoke, Takeda, Transcenta, Vanda, Xoma, Zhittya