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Disc Medicine Announces Presentation of Initial Data from RALLY-MF Phase 2 Trial in Patients with Myelofibrosis (MF) and Anemia at the 67th American Society of Hematology (ASH) Annual Meeting

On November 3, 2025 Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, reported two poster presentations, including initial data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of MF, at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held in Orlando, FL on December 6-9, 2025.

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"The highlight of our ASH (Free ASH Whitepaper) presentations this year will be an initial look at data from RALLY-MF, our Phase 2 trial of DISC-0974 in anemia of myelofibrosis," said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. "Following the strong Phase 1b results we presented at ASH (Free ASH Whitepaper) last year, we are eager to share an update on this program, including results from more patients from across the range of MF anemia patients, as a monotherapy and in combination with JAK inhibitor backbone therapies, including ruxolitinib and momelotinib. We will also present a poster on the design of our ongoing Phase 2 trial of DISC-3405 in polycythemia vera. We expect to share an initial readout for this trial, as well as for the Phase 1b trial of DISC-3405 in sickle cell disease, in 2026."

Management will host a call during the ASH (Free ASH Whitepaper) meeting to review highlights of the presented data and plans for next steps in development on Sunday, December 7 at 7:30am EST. Please register for the event on the Events and Presentations page of Disc’s website (View Source).

Details of Poster Presentations:

The abstracts are now available through the ASH (Free ASH Whitepaper) conference website. Pursuant to Disc Medicine practice, the clinical study abstracts published today contain previously presented data; new data and analyses are reserved for presentation at the conference.

DISC-0974 Poster Presentation:

Publication Number: 2042
Title: RALLY MF: A phase 2 Study of DISC-0974, an anti-hemojuvelin antibody, in patients with myelofibrosis and anemia
Date / Time: Saturday, December 6, 5:30 pm – 7:30pm EST
Presenting Author: Naseema Gangat, M.B.B.S.

DISC-3405 Poster Presentation:

Publication Number: 2053
Title: A phase 2, open-label study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of DISC-3405 in participants with polycythemia vera (PV)
Date / Time: Saturday, December 6, 5:30 pm – 7:30pm EST
Presenting Author: Marcus Carden, M.D., M.Sc.

(Press release, Disc Medicine, NOV 3, 2025, View Sourcenews-releases/news-release-details/disc-medicine-announces-presentation-initial-data-rally-mf-phase [SID1234659286])

Terns Announces Abstract with Positive Clinical Data for TERN-701 in Relapsed/Refractory CML Selected for Oral Presentation at 67th ASH Annual Meeting

On November 3, 2025 Terns Pharmaceuticals, Inc. (Terns or the Company) (Nasdaq: TERN), a clinical stage oncology company, reported that data from the ongoing CARDINAL trial of TERN-701, a novel investigational allosteric BCR::ABL1 inhibitor, in participants with previously treated chronic myeloid leukemia (CML) has been selected for oral presentation on December 8, 2025 at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place in Orlando, FL. The company will host a conference call and webcast for investors at 4:30pm ET following the ASH (Free ASH Whitepaper) presentation.

The abstract is now available on the ASH (Free ASH Whitepaper) website and details are summarized below. A more expansive and updated dataset from the CARDINAL trial will be presented at the ASH (Free ASH Whitepaper) Annual Meeting in December.

"We are pleased that data from our CARDINAL trial have been selected for oral presentation at ASH (Free ASH Whitepaper). These data further validate the potential of TERN-701 to be a new, game-changing therapy for CML. The 24 weeks MMR achievement rate with TERN-701 is unprecedented, trending at least two times higher than the rates reported in other Phase 1 studies of CML therapies that are approved or in development," said Amy Burroughs, chief executive officer of Terns.

"Importantly, TERN-701 also achieved consistently high overall (cumulative) MMR rates in key, difficult to treat patient subgroups while maintaining an encouraging safety profile. These emerging data strongly reinforce our conviction that TERN-701 has the potential to be a best-in-disease therapy, with broad opportunity across all CML treatment lines. We look forward to sharing additional data in December," added Ms. Burroughs.

The ASH (Free ASH Whitepaper) abstract published today reports data from the ongoing dose escalation and dose expansion parts of the CARDINAL study of TERN-701 in patients with previously treated CML. As of the June 30th, 2025, cutoff date, 55 patients were enrolled. Highlights include:

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Of 32 efficacy-evaluable patients:

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Overall (cumulative) major molecular response (MMR) rate of 75% (24/32) by 24 weeks, with 64% (14/22) achieving MMR and 100% (10/10) maintaining MMR

•
Overall (cumulative) MMR by 24 weeks in difficult to treat patient subgroups:

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69% (11/16) in patients with lack of efficacy to last tyrosine kinase inhibitor (TKI)

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60% (6/10) in patients who had prior asciminib

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67% (8/12) in patients with prior asciminib / ponatinib / investigational TKI

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No patients had lost MMR at the time of data cutoff

•
Enrolled patients had heavily pretreated, refractory disease:

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Median of 3 prior TKIs

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35% had ≥4 prior TKIs

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56% and 44% had baseline BCR::ABL1 >1% and >10%, respectively

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64% discontinued their last TKI due to lack of efficacy

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36% had prior asciminib treatment, 25% had prior ponatinib and/or an investigational TKI (olverembatinib / ELVN-001)

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13% with BCR::ABL1 mutations (9% with T315I and 4% with F317L)

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Encouraging safety profile:

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87% (48/55) patients remained on treatment as of the data cut-off; with discontinuations due to disease progression (n=4), adverse events (n=1), and consent withdrawal/lost to follow up (n=2)

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No dose-limiting toxicities (DLTs) were observed in dose escalation and a maximum tolerated dose (MTD) was not reached

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The majority (74%) of treatment-emergent adverse events (TEAEs) were low grade with no apparent dose relationship

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Most common TEAEs were diarrhea (22%), headache (18%) and nausea (16%), all Grade 1 or 2

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Grade 3 or higher TEAEs were all less than 10%, most commonly neutropenia (7%) and thrombocytopenia (4%)

•
TERN-701 exposures were approximately dose proportional across the dose range

Details for the ASH (Free ASH Whitepaper) oral presentation are as follows:

Title: CARDINAL: A Phase 1 study of TERN-701, a novel investigational allosteric BCR::ABL1 inhibitor for patients with previously treated CML

Presenter: Elias Jabbour, MD, Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center

Session Name: 632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Therapeutic agents to enhance patient outcomes

Session Date: December 8, 2025

Session Time: 2:45 – 4:15pm ET

Presentation Time: 2:45 – 3:00pm ET

Following the full presentation at the ASH (Free ASH Whitepaper) Annual Meeting, the presentation materials will be made available on the Terns website.

Company Conference Call and Webcast Information

Terns will host a conference call and webcast for investors at 4:30pm ET on December 8, 2025 following the oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting. Members of the Terns management team will discuss the TERN-701 data from CARDINAL and next steps in the development of TERN-701.

Webcasts can be accessed in the investor relations section of the Company’s website. A replay of the event will be available for a limited time.

About TERN-701 and CARDINAL Clinical Trial

TERN-701 is currently being evaluated in the CARDINAL trial (NCT06163430), a global multi-center dose escalation and dose-expansion clinical trial to assess safety, tolerability and efficacy in patients with previously treated chronic phase (CP) CML. The dose escalation portion of the CARDINAL trial completed in January 2025 with no dose limiting toxicities (DLTs) observed up to the maximum dose of 500 mg QD. Terns initiated the dose expansion portion of the trial in April 2025 with patients randomized to one of two dose cohorts (320 mg or 500 mg QD) with up to 40 patients per arm.

(Press release, Terns Pharmaceuticals, NOV 3, 2025, View Source [SID1234659283])

Syndax Reports Third Quarter 2025 Financial Results and Provides Business Update

On November 3, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported its financial results for the third quarter ended September 30, 2025, and provided a business update.

"The third quarter was another remarkable period of commercial and pipeline execution for Syndax. Demand remained strong for Revuforj and Niktimvo with over $75 million in combined net sales for the quarter," said Michael A. Metzger, Chief Executive Officer. "We also furthered our leadership in menin inhibition with the addition of Revuforj to the NCCN Guidelines for R/R NPM1m AML in late September followed by FDA approval in late October. Our expansion into this second indication is underway and we are making great progress driving awareness and generating demand. Additionally, we continue to advance the development of both Revuforj and Niktimvo in the frontline setting, further unlocking their multi-billion-dollar potential."

Recent Business Highlights and Anticipated Milestones

Revuforj (revumenib)

Achieved $32.0 million in Revuforj net revenue in the third quarter of 2025, representing a 12% increase over the second quarter of 2025. Total Revuforj prescriptions in the third quarter of 2025 were approximately 850, a 25% increase over total prescriptions in the second quarter of 2025.
Received U.S. FDA approval for Revuforj on October 24, 2025, for the treatment of R/R acute myeloid leukemia (AML) with a susceptible NPM1 mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. Revuforj is now the first and only FDA-approved therapy for both R/R AML with an NPM1 mutation and R/R acute leukemia with a KMT2A translocation.
Announced the inclusion of revumenib in the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines) for AML as a category 2A recommended treatment option for R/R NPM1m AML on September 18, 2025. The guideline update was based on positive pivotal results from the AUGMENT-101 trial of revumenib which were published in the journal Blood in 2025.
Announced that data from 12 revumenib abstracts, including 3 oral presentations, will be highlighted at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The abstracts present compelling results with revumenib in multiple acute leukemia subtypes across the R/R, frontline, and post-stem cell transplant settings.
Multiple trials evaluating revumenib in NPM1m and KMT2Ar acute leukemia across the treatment landscape are ongoing. These trials include:

EVOLVE-2: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial evaluating revumenib in combination with venetoclax and azacitidine in newly diagnosed NPM1m AML patients who are unfit for intensive chemotherapy. The trial is being conducted in collaboration with the HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies.
SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with newly diagnosed and R/R AML or mixed-lineage acute leukemia (MPAL) harboring either NPM1m, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. Data from the first cohort of newly diagnosed patients will be highlighted at the ASH (Free ASH Whitepaper) 2025 Annual Meeting in an oral presentation.
Intensive chemotherapy: Two ongoing Phase 1 trials evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed NPM1m or KMT2Ar acute leukemia patients. Preliminary data from both trials will be presented at the ASH (Free ASH Whitepaper) 2025 Annual Meeting.
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed older adults (≥60 years) with NPM1m or KMT2Ar AML. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial.
Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break Through Cancer, a collaboration between leading U.S. cancer research centers.
INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial.
Start-up activities are underway for two trials, known as the REVEAL trials, that will evaluate revumenib in combination with standard of care regimens in newly diagnosed acute leukemia patients with NPM1m or KMT2A-rearranged AML who are fit to receive intensive chemotherapy, with trial initiation expected by the end of 2025.
The Company is evaluating revumenib in patients with R/R metastatic microsatellite stable (MSS) colorectal cancer (CRC). The Company expects to report data from the trial at a medical conference in the first quarter of 2026.
Niktimvo (axatilimab-csfr)

Achieved $45.8 million in Niktimvo net revenue in the third quarter of 2025, representing a 27% increase over the second quarter of 2025. Syndax and Incyte are co-commercializing Niktimvo. Syndax records 50% of the Niktimvo net commercial profit, defined as net product revenue minus the cost of sales and commercial expenses. For the third quarter of 2025, Syndax’s share of the Niktimvo product contribution, reported as collaboration revenue, was $13.9 million.
Announced that data from 11 axatilimab abstracts, including 3 oral presentations, will be showcased at the 2025 ASH (Free ASH Whitepaper) Annual Meeting. The abstracts highlight the potential for axatilimab to provide long-term benefit in recurrent or refractory chronic GVHD and the tolerability of axatilimab with ruxolitinib in newly diagnosed chronic GVHD.
Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including:

A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
A pivotal Phase 3, randomized, double-blind, placebo-controlled, multi-center trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
Enrollment is ongoing in MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF). The Company expects to complete enrollment in the trial by the end of 2025 with topline data anticipated in the second half of 2026.
Third Quarter 2025 Financial Results

As of September 30, 2025, Syndax had cash, cash equivalents, and short- and long-term investments of $456.1 million and 87.2 million common shares and prefunded warrants outstanding.

Total revenue for the third quarter of 2025 was $45.9 million, which consisted of $32.0 million in Revuforj net revenue and $13.9 million in Niktimvo collaboration revenue. The collaboration revenue is derived from the $45.8 million in Niktimvo net revenue that was previously reported by the Company’s partner Incyte. Syndax records 50% of the Niktimvo net commercial profit, defined as net revenue (recorded by Incyte) minus the cost of sales and commercial expenses.

Third quarter 2025 research and development expenses decreased to $56.3 million from $71.0 million for the comparable prior year period. The decrease was primarily the result of a $15 million milestone payment paid by the Company upon Niktimvo’s approval, incurred in the third quarter of 2024 and not incurred in the 2025 period. Also contributing to the decrease was lower revumenib-related costs due the completion of the registrational trial in R/R NPM1m AML that was ongoing in the prior period and a reduction in CMC expenses due to the capitalization of inventory for commercial use.

Third quarter 2025 selling, general and administrative expenses increased to $44.9 million from $31.1 million for the comparable prior year period. The increase was primarily due to higher commercial costs and personnel and stock-based compensation expenses related to the commercial launches of Revuforj and Niktimvo in the 2025 period.

For the three months ended September 30, 2025, Syndax reported a net loss attributable to common stockholders of $60.7 million, or $0.70 per share, compared to a net loss attributable to common stockholders of $84.1 million, or $0.98 per share, for the comparable prior year period.

Financial Guidance

For the full year of 2025, the Company expects total research and development plus selling, general and administrative expenses to be $380 to $385 million, compared to prior guidance of $370 to $390 million, both estimates excluding an estimated $45 million in non-cash stock compensation expense.

Syndax expects that its operating expense base will remain stable over the next few years. As a result, Syndax expects that its cash, cash equivalents and short- and long-term investments, combined with its anticipated product revenue and interest income, will enable the company to reach profitability.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, November 3, 2025.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

Conference ID: Syndax3Q25
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

About Revuforj (revumenib)

Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options.

Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.

Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

About Niktimvo (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD, including a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774). Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

(Press release, Syndax, NOV 3, 2025, View Source [SID1234659282])

Syndax Announces Compelling Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) Data Accepted for Presentation at ASH 2025

On November 3, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that 23 abstracts, including six oral presentations, showcasing compelling Revuforj (revumenib) and Niktimvo (axatilimab-csfr) data were accepted for presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in Orlando, Florida, December 6-9, 2025.

"The breadth of the upcoming data presentations reflects the tremendous promise that Revuforj and Niktimvo hold across the treatment continuum for acute leukemia and chronic GVHD, respectively," said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. "In particular, we are excited to present new frontline datasets showcasing the tolerability of Revuforj in combination with standard of care therapies along with high rates of complete remission and MRD negativity, as well as the first real-world evidence for a menin inhibitor, and a retrospective review of usage in the post-transplant setting. We also look forward to the presentation of new data that highlight the potential for Niktimvo to provide long-term benefits in chronic GVHD and the feasibility of combining with ruxolitinib in newly diagnosed chronic GVHD."

The Company will host an in-person investor event, along with a live webcast, at the ASH (Free ASH Whitepaper) Annual Meeting on Monday, December 8, 2025, at 7:00 a.m. ET to discuss key data presented at the meeting. The live webcast will be available on the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.

Key abstracts accepted for presentation at ASH (Free ASH Whitepaper) 2025:

Revumenib:

An oral presentation will highlight results from a cohort of newly diagnosed patients in the Phase 2 SAVE trial of revumenib in combination with venetoclax and decitabine/cedazuridine in NPM1 mutated (NPM1m), KMT2A-rearranged (KMT2Ar), or NUP98-rearranged (NUP98r) acute myeloid leukemia (AML).
An oral presentation will report efficacy and safety by leukemia type (AML, ALL, or MPAL) in patients with R/R KMT2Ar acute leukemia in the Phase 2 portion of the pivotal AUGMENT-101 trial.
A poster presentation will highlight the first real-world experience with revumenib outside of a clinical trial setting, including in patients with KMT2Ar, NPM1m, or NUP98r acute leukemia.
Two poster presentations will report preliminary results from Phase 1 trials of revumenib in combination with intensive chemotherapy in newly diagnosed NPM1m, KMT2Ar, or NUP98r AML.
A poster presentation will highlight results from a retrospective review of pediatric patients with KMT2Ar, NUP98r, or NPM1m acute leukemia who received revumenib as a maintenance therapy following hematopoietic stem cell transplantation (HSCT).

Axatilimab:

An oral presentation will describe the safety and feasibility observed among patients with recurrent or refractory chronic graft-versus-host disease (GVHD) who transitioned from 0.3 mg/kg every 2 weeks dosing of axatilimab (FDA-approved dose) to 0.6 mg/kg every 4 weeks in the pivotal Phase 2 AGAVE-201 trial.
A poster presentation will highlight the long-term duration of therapy and safety of axatilimab among patients with recurrent or refractory chronic GVHD in the pivotal Phase 2 AGAVE-201 trial.
A poster presentation will report an interim safety analysis from a Phase 2 trial of axatilimab in combination with ruxolitinib in patients with newly diagnosed chronic GVHD.

The accepted abstracts listed below are now available online at the ASH (Free ASH Whitepaper) conference website. Copies of the oral and poster presentations will be made available in the ‘Publications & Meetings Presentations’ section of the Syndax website after the relevant embargoes lift.

Full list of abstracts accepted for presentation at ASH (Free ASH Whitepaper) 2025 (all times in ET):

Revumenib

Abstract Titles Presentation Details
Phase II study of the all-oral combination of revumenib (SNDX-5613) with decitabine/cedazuridine (ASTX727) and venetoclax (SAVE) in newly diagnosed AML Oral presentation
Abstract #: 47
Saturday, December 6
Session: 9:30-11:00 am
Revumenib for patients with relapsed or refractory (R/R) KMT2Ar acute leukemia: Outcomes by leukemia type in the Phase 2 AUGMENT-101 study Oral presentation
Abstract #: 1001
Monday, December 8
Session: 4:30-6:00 pm
Early real-world experience with revumenib outside of a clinical trial setting: A single center retrospective review of efficacy and tolerability Poster presentation
Abstract #: 3448
Sunday, December 7
Session: 6:00-8:00 pm
Phase 1 study of revumenib in combination with intensive chemotherapy (IC) in patients (pts) with newly diagnosed (ND) acute myeloid leukemia (AML) harboring genetic alterations in KMT2A, NPM1, or NUP98: SNDX-5613-0708 Poster presentation
Abstract #: 3425
Sunday, December 7
Session: 6:00-8:00 pm
Revumenib in combination with intensive induction and consolidation for newly diagnosed patients with NPM1-mutated or KMT2A-rearranged acute myeloid leukemia: Preliminary results from the Phase 1b ETCTN 10596 study Poster presentation
Abstract #: 5206
Monday, December 8
Session: 6:00-8:00 pm
Revumenib for patients with relapsed or refractory (R/R) nucleophosmin 1–mutated (NPM1m) acute myeloid leukemia (AML): Outcomes by prior treatment in the Phase 2 AUGMENT-101 study Poster presentation
Abstract #: 3418
Sunday, December 7
Session: 6:00-8:00 pm
Post-transplant maintenance with revumenib in children with HOX pathway-mutated AML Poster presentation
Abstract #: 3461
Sunday, December 7
Session: 6:00-8:00 pm
Trial in progress: A multicenter Phase I trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance after allogeneic hematopoietic cell transplant in patients with KMT2A-rearranged or NPM1-mutated acute leukemia Poster presentation
Abstract #: 5207
Monday, December 8
Session: 6:00-8:00 pm
Preliminary results of a Phase 1 study of the safety and tolerability of the combination of revumenib (REV) with gilteritinib (GILT) in relapsed/ refractory (R/R) acute myeloid leukemia (AML) Poster presentation
Abstract #: 3427
Sunday, December 7
Session: 6:00-8:00pm
Real-world treatment patterns and outcomes among patients with newly diagnosed NPM1-mutated acute myeloid leukemia in the United States Poster presentation
Abstract #: 3385
Sunday, December 7
Session: 6:00-8:00 pm
Menin inhibition as a new therapeutic option for the myeloproliferative neoplasms Oral presentation
Abstract #: 67
Saturday, December 6
Session: 9:30-11:00 am
Co-targeting menin and RAS in KMT2A-r/NPM1c AML with activated RTK//RAS/MAPK signaling Poster presentation
Abstract #: 5060
Monday, December 8
Session: 6:00-8:00 pm

Axatilimab

Abstract Titles Presentation Details
Safety and feasibility of 0.6 mg/kg every 4 weeks dosing of axatilimab in patients treated in the AGAVE-201 study Oral presentation
Abstract #: 272
Saturday, December 6
Session: 2:00-3:30 pm
Long-term treatment duration and safety of axatilimab among patients with chronic graft-versus-host disease in AGAVE-201 Poster presentation
Abstract #: 6010
Monday, December 8
Session: 6:00-8:00 pm
Axatilimab in combination with ruxolitinib in patients with newly diagnosed chronic graft-versus-host disease: Interim safety analysis of a randomized, Phase 2 study Poster presentation
Abstract #: 6012
Monday, December 8
Session: 6:00-8:00 pm
CSF-1R+ macrophages orchestrate human cutaneous chronic graft-versus-host disease Oral presentation
Abstract #: 588
Sunday, December 7
Session: 12:00-1:30 pm
Safety analysis of axatilimab in patients with chronic graft-versus-host disease in an expanded access program Poster presentation
Abstract #: 6008
Monday, December 8
Session: 6:00-8:00 pm
Trial in progress: A Phase 3, randomized, double-blind, placebo-controlled study of axatilimab and corticosteroids as initial treatment for moderate to severe chronic graft-versus-host disease Poster presentation
Abstract #: 4256
Sunday, December 7
Session: 6:00-8:00 pm
Pharmacodynamic analysis of AGAVE-201 indicates changes in CSF-1R-expressing cells and associated biomarkers potentially contributing to chronic graft-versus-host disease resolution Poster presentation
Abstract#: 2458
Saturday, December 6
Session: 5:30-7:30pm
Clinical and disease characteristics of initial participants at time of enrollment in THRIVE, a prospective, observational cohort study of patients at risk for chronic graft versus host disease Poster presentation
Abstract#: 2446
Saturday, December 6
Session: 5:30-7:30pm
CSF-1R inhibition and lenalidomide synergize to promote myeloma control after autologous stem cell transplantation Oral presentation
Abstract #: 689
Sunday, December 7
Session: 4:30-6:00 pm
CSF1R-CSF1 axis blockade with axatilimab effectively targets leukemia stem cells and monocytes in AML resistant to BH3 mimetics Poster presentation
Abstract #: 3276
Sunday, December 7
Session: 6:00-8:00 pm
Phase 1b/2 study of axatilimab in combination with azacitidine in advanced phase MPN, MDS/MPN overlap and high-risk CMML Poster presentation
Abstract #: 5607
Monday, December 8
Session: 6:00-8:00 pm

About Revuforj (revumenib)

Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.

About Niktimvo (axatilimab-csfr)

Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

Niktimvo is a trademark of Incyte.
All other trademarks are the property of their respective owners.

Revuforj (revumenib)

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME, QTc PROLONGATION, and TORSADES DE POINTES

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

QTc prolongation and Torsades de Pointes have occurred in patients receiving Revuforj. Correct hypokalemia and hypomagnesemia prior to and during treatment. Do not initiate Revuforj in patients with QTcF > 450 msec. If QTc interval prolongation occurs, interrupt, reduce, or permanently discontinue Revuforj.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, rash, and/or hypotension.

In clinical trials, DS occurred in 60 (25%) of 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia. Among those with a KMT2A translocation, DS occurred in 33% of patients with acute myeloid leukemia (AML), 33% of patients with mixed-phenotype acute leukemia (MPAL), and 9% of patients with acute lymphoblastic leukemia (ALL); DS occurred in 18% of patients with NPM1m AML. DS was Grade 3 or 4 in 12% of patients and fatal in 2 patients. The median time to initial onset was 9 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours in adults or dexamethasone 0.25 mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc Interval Prolongation and Torsades de Pointes: Revuforj can cause QT (QTc) interval prolongation and Torsades de Pointes.

Of the 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia in clinical trials, QTc interval prolongation was reported as an adverse reaction in 86 (36%) patients. QTc interval prolongation was Grade 3 in 15% and Grade 4 in 2%. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 10%, and the increase from baseline QTcF was greater than 60 msec in 24%. Revuforj dose reduction was required for 7% due to QTc interval prolongation. QTc prolongation occurred in 21% of the 34 patients less than 17 years old, 35% of the 146 patients 17 years to less than 65 years old, and 46% of the 61 patients 65 years or older. One patient had a fatal outcome of cardiac arrest, and one patient had non-sustained Torsades de Pointes.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to and throughout treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia

Embryo-Fetal Toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 9 (4%) patients who received Revuforj, including 4 with sudden death, 2 with differentiation syndrome, 2 with hemorrhage, and 1 with cardiac arrest.

Serious adverse reactions were reported in 184 (76%) patients. The most frequent serious adverse reactions (≥10%) were infection (29%), febrile neutropenia (20%), bacterial infection (15%), differentiation syndrome (13%), and hemorrhage (11%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were phosphate increased (51%), hemorrhage (48%), nausea (48%), infection without identified pathogen (46%), aspartate aminotransferase increased (44%), alanine aminotransferase increased (40%), creatinine increased (38%), musculoskeletal pain (37%), febrile neutropenia (37%), electrocardiogram QT prolonged (36%), potassium decreased (34%), parathyroid hormone intact increased (34%), alkaline phosphatase increased (33%), diarrhea (29%), bacterial infection (27%), triglycerides increased (27%), phosphate decreased (25%), differentiation syndrome (25%), fatigue (24%), edema (24%), viral infection (23%), decreased appetite (20%), and constipation (20%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec

SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: no overall differences were observed in the effectiveness of Revuforj between patients who were 65 years and older, and younger patients. Compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNINGS.

Niktimvo (axatilimab-csfr)

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
Infusion-Related Reactions
Niktimvo (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received Niktimvo in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%.

Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.

Embryo-Fetal Toxicity
Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.

ADVERSE REACTIONS
Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in >2 patients included infection (pathogen unspecified) (14%), viral infection (14%) and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia.

The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.

Clinically relevant adverse reactions in <10% of patients who received Niktimvo included:

Eye disorders: periorbital edema
Skin and subcutaneous skin disorders: pruritus
Vascular disorders: hypertension

Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions
Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.

USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.

Females and Males of Reproductive Potential
Pregnancy Testing
Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.

Contraception
Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.

DOSAGE AND ADMINISTRATION
Dosage Modifications for Adverse Reactions
Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.

(Press release, Syndax, NOV 3, 2025, View Source [SID1234659281])

Star Therapeutics to Present Interim Data from Phase 1/2 Multidose Study of VGA039 in von Willebrand disease at ASH Annual Meeting

On November 3, 2025 Star Therapeutics, a clinical stage biotechnology company discovering and developing best-in-class antibodies for bleeding disorders and other diseases, reported that the company will present an oral presentation on interim data from its Phase 1/2 multidose study of VGA039 for von Willebrand disease (VWD), along with additional posters on the program, at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held December 6-9, 2025, in Orlando, Fla. VGA039 is a first-in-class monoclonal antibody therapy with a novel mechanism of action that targets Protein S, thereby restoring balance to the blood clotting process.

Presentation details include:

Oral Presentation

Title: Subcutaneous, Every-Four-Week Maintenance Dosing of a Novel Protein S Antibody is Well-Tolerated and Substantially Reduces Bleeding Rates: Results from a Phase 1/2 Multidose Study of VGA039 in Patients with Von Willebrand Disease
Publication Number: 308
Oral Session: 323. Disorders of Coagulation, Bleeding, or Fibrinolysis, Excluding Congenital Hemophilias: Clinical and Epidemiological: Novel Insights into Diagnostics and Therapeutics of Bleeding in Inherited, Acquired Coagulopathies and BDUC
Presentation Date and Time: Saturday, December 6, 2025, 4:15 – 4:30 p.m. ET
Presenter: Allison Wheeler, M.D., MSCI
Poster Presentations

Title: The First Characterization of Disease Burden and Healthcare Resource Utilization for the Recent Definition of Severe Von Willebrand Disease Using a Large United States Real-World Dataset
Publication Number: 2611
Poster Session: 901. Health Services and Quality Improvement: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster I
Session Date and Time: Saturday, December 6, 2025, 5:30 – 7:30 p.m. ET
Presenter: Angela Weyand, M.D.
Title: VGA039 as a Protein S-Targeted Hemostatic Promoting Monoclonal Antibody, Promotes in-vitro Thrombin Generation in Plasma Samples from Subjects Across a Broad Range of Bleeding Disorders, Including Von Willebrand Disease, Hemophilia A, Hemophilia B and Hemophilia C
Publication Number: 1277
Poster Session: 321. Coagulation and Fibrinolysis: Basic and Translational: Poster I
Session Date and Time: Saturday, December 6, 2025, 5:30 – 7:30 p.m. ET
Presenter: Alina He, B.S.
Title: A Protein S-Targeting Monoclonal Antibody, VGA039, Improves Both Primary and Secondary Hemostatic Activity of Von Willebrand Disease Patient Blood in an ex vivo Vascularized Hemostasis-on-a-Chip
Publication Number: 3051
Poster Session: 321. Coagulation and Fibrinolysis: Basic and Translational: Poster II
Session Date and Time: Sunday, December 7, 2025, 6:00 – 8:00 p.m. ET
Presenter: Yumiko Sakurai, M.S.
About VGA039
VGA039 is a monoclonal antibody therapy with a novel mechanism of action that targets Protein S, thereby restoring balance to the blood clotting process. VGA039 has potential to be a universal hemostatic therapy that can treat numerous bleeding disorders, starting with VWD. As a subcutaneously self-administered antibody therapy with a convenient once monthly dosing regimen, VGA039 has the potential to dramatically reduce treatment burden for patients. VGA039 has received Fast Track and orphan drug designations from the United States Food and Drug Administration (FDA).

Interim positive data from a Phase 1 single ascending dose study of VGA039 in patients with VWD were previously reported at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2024. A Phase 1/2 multidose study is ongoing (NCT05776069), with an interim readout planned for presentation at the 2025 ASH (Free ASH Whitepaper) Annual Meeting. VGA039 has advanced into a Phase 3 study (NCT07115004), a global single arm cross-over study designed to investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. For additional information on our VIVID trials of VGA039, including how to enroll, please visit the website here.

About von Willebrand disease
Von Willebrand disease (VWD) is the most common inherited bleeding disorder in which the blood does not clot properly, caused by absent or defective von Willebrand factor (VWF). VWD patients may experience excessive bleeding with variability in severity and frequency, negatively impacting their daily lives. Current therapies for VWD prophylaxis include factor replacement therapies requiring multiple intravenous (IV) infusions every week. More than 130,000 people in the U.S. are diagnosed with VWD.

(Press release, Star Therapeutics, NOV 3, 2025, View Source [SID1234659280])