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A promising sickle cell pill and a new way to treat myelofibrosis

December 7, 2025

It's Day 2 of the ASH meeting. Let's get straight into the news. 

Fulcrum study suggests improved efficacy for higher dose sickle cell pill

Fulcrum Therapeutics said Sunday that a higher dose of its experimental pill for sickle cell disease was more effective at inducing an alternative form of the oxygen-carrying molecule hemoglobin — boosting hopes it could one day provide a simple and effective treatment for the disease.  

In the early stage study, 12 participants who received a 20mg daily dose of the Fulcrum drug, called pociredir, saw their levels of fetal hemoglobin rise from 7.1% of the total hemoglobin in their blood to 16.9%. 

Very high levels of fetal hemoglobin can virtually eliminate sickle cell symptoms, as evidenced by studies of patients who naturally have high levels of fetal hemoglobin and by the clinical trials of Vertex and CRISPR Therapeutics' gene editing treatment for sickle cell, Casgevy.

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Incyte showcases a new way to treat myelofibrosis

An experimental drug from Incyte achieved meaningful spleen response rates and improvements in disease symptoms in patients with advanced myelofibrosis, according to study results reported Sunday. 

The data, while preliminary, should bolster the company's efforts to develop a new type of drug for the bone-marrow cancer, by targeting a mutation in a protein called calreticulin. 

Incyte needs new medicines because Jakafi, its current myelofibrosis blockbuster with expected sales of $3.5 billion this year, is losing patent protection in 2028.

"Our goal, by the end of this decade or early 2030s at the latest, is to have a targeted drug for every patient with myelofibrosis," Incyte R&D chief Pablo Cagnoni told STAT.

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Puppies! 

IMG_4656Damian Garde/STAT

Hematology/oncology is serious work, but there's always time for puppy cuddles. 



New data from Gilead, Arcellx multiple myeloma CAR-T suggest benefit over rival treatment

With sales of its existing blood cancer CAR-T therapies weakening, Gilead Sciences needs anito-cel, its next CAR-T therapy for multiple myeloma, to succeed more than ever. 

On Saturday, the company and its partner Arcellx reported deepening and durable responses with no concerning safety issues in the latest analysis of a pivotal-stage clinical trial.

Leerink Partners analyst Daina Graybosch, in a research note to clients, said the anito-cel data supported a "best in class profile" relative to Carvykti, its entrenched multiple myeloma CAR-T competitor, that would be "sufficient to drive physician enthusiasm and uptake in the U.S."

Gilead reiterated its plan to secure anito-cel's approval and launch the drug in 2026. The company has not disclosed more specific timing, although analysts expect an imminent submission to the Food and Drug Administration and an approval in the middle or second half of 2026.

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Almost 1 in 3 families facing pediatric ALL experience new catastrophic financial challenges

Cancer treatment is expensive. It's not just the therapy itself, but also transportation costs, quitting or taking time off work, and potentially childcare, said Daniel Zheng, a cancer researcher and pediatric oncologist at Children's Hospital of Philadelphia. That can all add up to major financial hardships, he said

In a survey of 422 participants, Zheng found that nearly a third of families experience new financial hardships within two years of therapy for pediatric acute lymphoblastic leukemia, the most common childhood cancer. 

Zheng surveyed participants for these hardships by asking questions about being able to afford things like food and housing, and about how their income changed.

Housing insecurity was the largest financial toxicity, Zheng said, with 22.2% of participants noting it as a new development. 15.9% of participants reported difficulty or worries about paying for food, and 8.6% of participants for utilities. During the press briefing, Zheng recalled a patient whose family was struggling to balance medical expenses for their child and one of the parents, and afford childcare for other kids. 

"My friend, Katie Oranges, who was the nurse practitioner working with me, looked up and said, 'What's so sad is that for some of our families, their child's cancer diagnosis is not the hardest thing they're facing.' I think about this often, because it's devastating, heart breaking, deeply unfair," Zheng said. 

The work shows that health systems need to screen for financial insecurity throughout the treatment period, not just at the beginning, Zheng said, and that interventions to alleviate financial toxicity need to be studied. "The intervention I am most excited about is directed cash transfer," he said. "That's the simplest proof of concept we can use to address financial toxicity and lends itself to flexibility."

—Angus Chen


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Thanks for reading! Until tomorrow - Adam, Damian, and Katherine

Adam Feuerstein is a senior writer and biotech columnist for STAT, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a cohost of STAT's weekly "The Readout LOUD" podcast and the author of the weekly newsletter Adam's Biotech Scorecard.

Damian Garde is a reporter at large, live and feature journalism, covering the global drug industry and contributing to STAT's industry-leading events.

Katherine MacPhail is an editorial events programmer at STAT. 


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