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Terns looks like a rival to Novartis with drug for chronic myeloid leukemia

December 8, 2025
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By Adam Feuerstein, Damian Garde, and Katherine MacPhail

It rains in Florida. A lot. This ASH meeting started sunny and wonderful, but quickly turned rainy and miserable. It's just another reason why ASH should camp itself permanently in San Diego each December. 

Study suggests Terns leukemia drug could be  successor to Novartis blockbuster

IMG_4066 (1)Adam Feuerstein/STAT

Terns Pharmaceuticals reported an update on its targeted leukemia drug that maintained and even boosted high molecular response rates in advanced-stage patients. 

The study results, while still early, are likely to draw even more positive attention from investors who already view the Terns drug as a potential successor to a commercial blockbuster from Novartis. 

At 24 weeks, four escalating doses of the Terns drug, called TERN-701, achieved a major molecular response of 64% in patients with chronic myeloid leukemia, a slow-growing cancer that starts in myeloid cells. The 28 patients evaluable had already experienced treatment with a median of three drugs.

TERN-701 has garnered considerable attention for its potential to be superior to Scemblix, Novartis' current and best-in-class CML drug that is now annualizing at $1.4 billion in sales. Novartis recently increased its peak sales forecast for Scemblix to $4 billion from $3 billion. 

In Novartis' Phase 1 study (not entirely comparable but similar enough), Scemblix showed a 24% rate of major molecular response. 

"TERNS-701 is not just another asciminib," Terns CEO Amy Burroughs told STAT, using the scientific name for Novartis' Scemblix. "The data shows that this drug is working differently. We're not only able to dose higher because we have a better safety profile, but there's also something different about the way this drug is working. We have better potency, we have better target coverage."

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The benefits of being a trillionaire

Eli Lilly has quite a lot of money. Demand for its blockbuster GLP-1 treatment is driving revenue to new heights, and the promise of its next-generation obesity drugs has its market value hovering around an industry-leading $1 trillion.

That weight-loss windfall is funding big swings in the company's research, allowing Lilly to run ambitious, costly trials at which its peers might blanch. 

"A lot of our work is focused on what is going to move the needle the most," Chief Medical Officer David Hyman said in an interview. "Those studies are universally large and hard and long and expensive. But that's why Lilly exists."

ASH provides a shining example: Lilly presented data Sunday from a rare head-to-head study, comparing its blood cancer drug Jaypirca against Imbruvica, the market-leading medicine from Johnson & Johnson and AbbVie. 

The trial met its goal of establishing non-inferiority, and the results consistently favored Lilly's drug, including on the secondary endpoint of progression-free survival. That suggests Jaypirca could be preferable for patients with chronic lymphocytic leukemia and small lymphocytic lymphoma regardless of whether they've tried other drugs, said Jennifer Woyach, an Ohio State University hematologist who led the study.

For Lilly, the results bolster Jaypirca's commercial profile in a competitive space that includes medicines from AstraZeneca and BeOne. The study also illustrates the benefits of conducting trials that will unearth the kind of data doctors actually want to see, said Jacob Van Naarden, president of Lilly Oncology.

"There's almost nothing more frustrating than spending a ton of money and time on one of these programs, generating 'positive' data, and then having the field say, 'Yeah, but I don't really know what this means,'" Van Naarden said. "I would just rather answer the real question, even if it takes on more risk."


Kura's frontline AML push

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With so-called menin inhibitors now approved for patients with certain types of advanced acute myeloid leukemia, research efforts are shifting to find uses for the genetically targeted drugs in newly diagnosed patients.

Kura Oncology presented preliminary results today from a study that combines Komzifti, its menin-blocker, with two standard AML treatments. In an analysis that encompassed 40 patients with newly diagnosed, menin-susceptible AML, the triplet regimen showed a complete remission rate of 86% with a manageable safety profile. 

Syndax Pharma reported results from its own menin-inhibitor combination study in newly diagnosed AML patients on Saturday at the ASH meeting. 



Few sickle cell patients are receiving genetic medicines

Genetix Biotherapeutics (you remember it as Bluebird Bio, before being acquired and taken private by its new owners) has treated 37 patients with Lyfgenia, its gene therapy for sickle cell disease, the company reported during a presentation Monday. 

That's…not a lot, and tracks similarly low numbers from Vertex Pharmaceuticals, which has administered Casgevy, its CRISPR-based treatment, to 39 patients with sickle cell and beta-thalassemia, according to its most recent earnings report.

Both treatments were approved in December 2023, but commercial rollouts have been slow due to the complicated, months-long process required to make and receive the treatment. 

Genetix's "infusion rate" for Lyfgenia is 23% — 39 patients treated out of 169 patients who have started the cell collection process. 

The company has done better with Zynteglo, its gene therapy for beta thalassemia that was approved 2022. Seventy-eight patients have been treated out of 122 patients starting the cell collection process. 


Wall Street's view

It's Monday, which means investors have had a chance to react to all the data presented over the last three days. 

Your ASH-stock winners are Fulcrum Therapeutics, Terns Pharma, Cullinan Therapeutics, Immix Biopharma and Arcellx. 


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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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