On November 4, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended September 30, 2025.

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"We are a global commercial company with multiple products generating meaningful growth that is expected to accelerate from anticipated launches from our late-stage clinical pipeline," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We announced today that we bolstered our balance sheet with a royalty financing ahead of pivotal milestones expected over the next year to support multiple late-stage data readouts, multiple regulatory submissions, and multiple launches. This includes the highly anticipated phase 3 study readouts for UX143 in osteogenesis imperfecta around the end of the year."

Third Quarter 2025 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 47,003 $ 35,604 $ 136,810 $ 112,294
Royalty revenue – U.S. and Canada 57,186 55,985 177,122 163,432
Royalty revenue – Europe 7,754 6,258 21,282 18,376
Total Crysvita Revenue 111,943 97,847 335,214 294,102
Dojolvi 24,275 21,374 64,491 57,091
Evkeeza 16,717 10,657 42,321 21,788
Mepsevii 6,998 9,616 23,695 22,372
Total revenues $ 159,933 $ 139,494 $ 465,721 $ 395,353

Total Revenues
Ultragenyx reported $160 million in total revenue for the third quarter of 2025, which represents 15% growth compared to the same period in 2024. Crysvita revenue in the third quarter 2025 was $112 million, which includes product sales of $47 million from Latin America and Türkiye. Dojolvi revenue in the third quarter 2025 was $24 million. Evkeeza revenue in the third quarter 2025 was $17 million as we continue to launch in the Ultragenyx territories outside of the United States.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
Total revenues $ 159,933 $ 139,494 $ 465,721 $ 395,353
Operating expenses:
Cost of sales 27,991 21,021 79,655 59,834
Research and development 216,212 170,109 546,720 510,099
Selling, general and administrative 86,620 80,351 261,063 239,115
Total operating expenses 330,823 271,481 887,438 809,048
Net loss $ (180,413 ) $ (133,516 ) $ (446,444 ) $ (435,798 )
Net loss per share, basic and diluted $ (1.81 ) $ (1.40 ) $ (4.55 ) $ (4.91 )

Operating Expenses

Total operating expenses for the third quarter of 2025 were $331 million, including non-cash stock-based compensation of $37 million.

Net Loss
For the third quarter of 2025, Ultragenyx reported net loss of $180 million, or $1.81 per share basic and diluted, compared with a net loss for the third quarter of 2024 of $134 million, or $1.40 per share basic and diluted.

Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $447 million as of September 30, 2025.

The company announced today that it received $400 million through the sale of an additional 25% of its royalty interest on the future sales of Crysvita in the United States and Canada, to OMERS. Payments to OMERS will begin in January of 2028. OMERS will also continue to receive 30% of Crysvita net sales in the U.S. and Canada following the achievement of the 2022 royalty purchase agreement transaction’s cap of 1.45 times the purchase price. Total payments to OMERS pursuant to the new agreement are capped at 1.55 times the 2025 purchase price.

For the three months ended September 30, 2025, net cash used in operations was $91 million and for the nine months ended September 30, 2025 was $366 million.

2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14-20% compared to 2024.

Reaffirm for the full year 2025:

Total revenue to be in the range of $640 million to $670 million
Crysvita revenue to be in the range of $460 million to $480 million
Dojolvi revenue to be in the range of $90 million to $100 million
Ultragenyx also reaffirmed its net cash used in operations guidance for 2025, which is expected to modestly increase compared to 2024 and its path to full year GAAP profitability in 2027.

Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Final analysis for Phase 3 Orbit and Cosmic studies around the end of 2025

The Phase 3 Orbit and Cosmic studies, which evaluate setrusumab in pediatric and young adult patients with OI, are progressing towards final analyses at which time patients will have been on therapy for at least 18 months. Data from these studies are expected around the end of 2025.

GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 Aspire study fully enrolled; Phase 3 data expected in the second half of 2026

In July 2025, enrollment of the global Phase 3 Aspire study was completed with 129 patients screened and randomized across 28 global sites. Participants are randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. Data from this study are expected in the second half of 2026.

Enrollment has begun in the supportive Phase 2/3 Aurora study, which is evaluating GTX-102 in other Angelman syndrome genotypes and ages.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): expect to resubmit Biologics License Application (BLA) early in 2026

Following receipt of a Complete Response Letter (CRL), the company has had constructive formal and informal discussions with the FDA. The additional clinical data requested by the agency, and that will be included in the BLA, continues to show a durable treatment effect across multiple biomarkers and further clinical separation from natural history, while maintaining an acceptable safety profile.

The company plans to resubmit the BLA early in 2026 and will be followed by an up to 6-month review per FDA regulations.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA rolling submission underway, expect to complete in the fourth quarter of 2025

Rolling submission of a BLA for DTX401 for the treatment of GSDIa began in August 2025. The BLA will include data from the randomized, placebo-controlled Phase 3 study that demonstrated statistically significant and clinically meaningful reductions in daily cornstarch intake compared with placebo at Week 48. It will also include longer-term data that was announced in September 2025 that demonstrated patients showed an even greater reduction in mean daily cornstarch intake in the 48-week crossover period. Both the originally treated DTX401 group (n=20) and the crossover group (n=19) who received DTX401 at Week 48 had a mean reduction in daily cornstarch intake of 61% at Week 96. Quality of life improved following treatment with DTX401 for patients in both groups as measured by the Patient Global Impression of Change (PGIC). At Week 96, improvements in disease management were reported by 83% (10/12) of patients in the DTX401 group and 95% (18/19) of patients in the crossover group.

Rolling submission of the BLA is expected to complete in the fourth quarter of 2025.

UX701 AAV gene therapy for Wilson Disease: Cohort 4 enrollment complete, data expected in the first half of 2026

In September 2025, the company completed enrollment of the fourth cohort evaluating a 4.0e13 GC/kg dose in the ongoing, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. A total of five patients were enrolled in Cohort 4. These patients received immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Data from this study are expected in the first half of 2026.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, November 4, 2025, at 2 p.m. PT/5 p.m. ET to discuss the third quarter 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for three months.

(Press release, Ultragenyx Pharmaceutical, NOV 4, 2025, View Source [SID1234659374])

On November 4, 2025 Actinium Pharmaceuticals, Inc. (NYSE American: ATNM) ("Actinium" or the "Company"), a leader in the development of differentiated targeted radiotherapies, reported that new preclinical data for its lead antibody radioconjugate program, ATNM‑400, will be presented at the 2025 San Antonio Breast Cancer Symposium (SABCS) taking place December 10‑14, 2025 in San Antonio, Texas.

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Title: Anti‑tumor activity of ATNM‑400, a first‑in‑class Actinium‑225 antibody radioconjugate, in tamoxifen and trastuzumab resistant breast cancer models

Abstract Number: 2069
Presentation Number: PS4‑04‑26
Date/Time: Thursday, December 11, 2025, 5:00 PM–6:30 PM CT
Session: Poster Session 4

The ATNM-400 data presentation at SABCS follows prostate cancer data presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting and the Society of Nuclear Medicine and Molecular Imaging (SNMMI) annual meeting, NSCLC data was presented at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), from which key findings are summarized below.

ATNM ‑ 400: Program and Multi-Indication Opportunity Overview

ATNM-400 is a first-in-class antibody-radioconjugate powered by Actinium-225 (Ac-225), designed to deliver potent alpha-particle radiation directly to tumor cells. The therapy’s high linear energy transfer (LET) enables precise tumor cell killing with minimal off-target exposure, while its target – a disease-driving protein linked to resistance and poor prognosis – is overexpressed across multiple solid tumors, including prostate, lung, and breast cancers.

Preclinical studies show ATNM-400 delivers best-in-class efficacy, overcomes resistance to, and synergizes with, standard-of-care therapies, supporting broad development as a monotherapy, combination, or treatment alternative across multiple solid tumors.

Prostate Cancer Data Highlights

Superior efficacy and durability vs 177Lu-PSMA-617 (active agent in Pluvicto), 225Ac-PSMA-617, and ARPI enzalutamide (active agent in Xtandi), with durable tumor control beyond 100 days.
Improved overall survival compared to 177Lu-PSMA-617 and enzalutamide.
Overcomes resistance to these standard-of-care agents, showing sustained tumor control and survival benefit in 177Lu-PSMA-617 and enzalutamide resistant prostate cancer models.
Synergy with ARPI therapy as the ATNM-400 target is upregulated after enzalutamide; combination achieved complete tumor regression in 40% of animals.
PSMA-independent activity enables treatment of patient populations not eligible for or progressing on 177Lu-PSMA-617.
NSCLC Data Highlights

3–5x greater tumor growth inhibition vs. front-line osimertinib (Tagrisso, AstraZeneca) and EGFR tyrosine kinase inhibitor (TKI), second-line Dato-DXd (Datroway, AstraZeneca/Daiichi Sankyo) a Trop-2 ADC, and third-line amivantamab (Rybrevant, Johnson & Johnson) an EGFR-cMET bispecific. Combined 2024 sales of these agents exceeded $7B.
Target upregulation following EGFR inhibition; ATNM-400 + osimertinib achieved complete tumor regression in 100% of tumor-bearing animals demonstrating synergy of the combination.
Clinical rationale for combination supported by a study that showed EBRT or external-beam radiotherapy + osimertinib improved PFS to 32.2 months vs. 20 months with osimertinib alone (Sampath et al.1, Lancet eClinicalMedicine, 2025). ATNM-400 has the potential to deliver precision targeted, powerful alpha radiation via Ac-225 which on a per-cell basis is ~4–8x more biologically lethal than diffuse, low-energy EBRT beams. Clinically, this may translate to higher response rates, lower toxicity, and entry into previously untreatable market segments when osimertinib is combined with EBRT.
ATNM-400 Data Afford Development Opportunities in High-Value, Unmet Need Indications

The data package for ATNM‑400 across breast cancer, prostate cancer and NSCLC underscores Actinium’s intention to demonstrate the potential for this radiotherapy candidate to address unmet needs in high‑value cancer segments. Data from preclinical studies thus far show ATNM-400 delivers best-in-class efficacy, overcomes resistance to, and synergizes with, standard-of-care therapies, supporting broad development as a monotherapy, combination, or treatment alternative across solid tumors. Key development opportunities based on the data include:

Breast Cancer: Potential in hormone‑resistant (tamoxifen) and HER2‑resistant (trastuzumab) patients, which represents a significant therapeutic opportunity with the HER2-targeted therapy Herceptin (Roche and biosimilars) generating approximately $4.0 billion in sales in 2024.

Prostate Cancer: Broad use potential as a monotherapy, combination, or follow-on to ARPIs and PSMA radioligands; opportunity in the $10 billion ARPI market and Pluvicto non-responders or relapses who on balance might be expected to outnumber the patients treated with Pluvicto which generated $1.7 billion in revenue over the last twelve months.

Non-Small Cell lung Cancer: Superior anti-tumor activity compared to the leading first, second and third-Line approved EGFR mutant therapies that generated sales of over $7.0 billion in 2024 and mechanistic synergy with first-line therapy osimertinib, which accounted for approximately $6.6 billion in sales in 2024.

Sandesh Seth, Chairman and CEO of Actinium Pharmaceuticals, commented, "We’re excited to present ATNM-400’s breast cancer data at SABCS which expands our demonstration of its potential across multiple solid tumors. The strong single-agent efficacy of ATNM-400 and its ability to overcome resistance when coupled with enzalutamide (Xtandi) in prostate cancer and osimertinib (TAGRISSO) in lung cancer showcases Actinium’s capability for innovation by exploiting the power of a radiotherapeutic directed to a target linked to resistance and poor prognosis. We believe that this program has the potential to meaningfully improve outcomes for patients with difficult-to-treat cancers and look forward to the data at SABCS".

About ATNM-400

ATNM-400 is a highly innovative, first-in-class, and multi-indication Actinium-225 (Ac-225) targeted radiotherapy candidate in development for prostate cancer, non-small cell lung cancer (NSCLC) and breast cancer. ATNM-400 is highly differentiated in prostate cancer as it targets a distinct non-PSMA protein strongly implicated in prostate cancer disease biology including progression and treatment resistance. Unlike 177Lu-PSMA-617, the active agent in Pluvicto and the majority of radiotherapies under development, which rely on PSMA targeting, ATNM-400 is designed to maintain efficacy in low-PSMA or high-PSMA resistant disease, a major unmet clinical need as up to 30% of patients do not respond to PSMA radioligand therapies and up to 60% of patients have at least one PSMA-negative tumor lesion. Ac-225 delivers high-linear-energy-transfer alpha particles that induce irreparable double-strand DNA breaks, offering superior potency over beta emitters like Lutetium-177 (177Lu), and has a shorter tissue path length that may reduce off-target toxicity. The receptor specifically targeted by ATNM-400 continues to be expressed at a high level even after androgen receptor inhibitor (ARPI) and ATNM-400 has shown to overcome resistance to the ARPI therapy enzalutamide and work synergistically in combination with enhanced tumor control including complete tumor regression. In NSCLC, ATNM-400 has shown superior efficacy compared to approved first, second- and third-line EGFR therapies including small molecules, antibody drug conjugates and bispecific antibodies that is synergistic with osimertinib, an EGFR tyrosine kinase inhibitor (TKI) that is a standard of care therapy approved for treatment of patients in the frontline setting and is also able to overcome osimertinib resistance.

Prostate cancer is the most commonly diagnosed cancer in men, with ~1.5 million new cases globally and over 313,000 expected in the U.S. in 2025. While early-stage disease is typically managed with surgery, radiation, and ARPI therapy, up to 20% of cases progress to mCRPC – a lethal stage with limited treatment options. Targeted radiotherapy is a growing field in prostate cancer, dominated by PSMA-targeting agents like Pluvicto, which had sales of over $1.3 billion in 2024, yet up to 30% of patients either lack or have no PSMA expression and virtually all patients develop resistance to Pluvicto within 1-year. In the U.S., 40,000–60,000 mCRPC patients annually progress after ARPI therapy with approximately 35% of patients progressing within 1-year. As a class, ARPI therapies had sales of over $10.0 billion in 2024 including enzalutamide (Xtandi) that led the class with sales of over $5.9 billion in 2024, highlighting a significant unmet need. Lung cancer is the leading cause of cancer deaths and there are there are over 200,000 new cases expected in the U.S. in 2025 and over 2 million cases globally. NSCLC accounts for approximately 85% of all lung cancer cases. EGFR targeting therapies including front-line osimertinib (Tagrisso, AstraZeneca) an EGFR tyrosine kinase inhibitor (TKI), second-line Dato-DXd (Datroway, AstraZeneca/Daiichi Sankyo) a Trop-2 ADC, and third-line amivantamab (Rybrevant, Johnson & Johnson) an EGFR-cMET bispecific had sales of approximately $7 billion in 2024 with the EGFR TKI Osimertinib (TAGRISSO, AstraZeneca) generating sales of $6.6 billion in 2024. Breast cancer is the most diagnosed cancer among woman in the United States with approximately 316,950 women expected to be diagnosed with the disease in 2025 according to the National Cancer institute. It is estimated that approximately 200,000 women are living with metastatic breast cancer in 2025, which is expected to grow to 250,000 in 2030. Of those diagnosed, hormone receptor-positive, HER2-negative (HR+/HER2-) breast cancer accounts for 70-75% of breast cancer, representing the largest subtype. In this setting, tamoxifen and trastuzumab (Herceptin, Roche and biosimilars) generated sales of approximately $4.0 billion in 2024. Across prostate cancer, NSCLC and breast cancer, ATNM-400 has demonstrated treatment paradigm changing potential in these indications, which have over 800,000 new cases in the U.S. alone.

(Press release, Actinium Pharmaceuticals, NOV 4, 2025, View Source [SID1234659392])

On November 3, 2025 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported three presentations featuring bezuclastinib, including two oral presentations in NonAdvanced Systemic Mastocytosis (NonAdvSM), at the 67th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) being held December 6-9, 2025 in Orlando, FL. Cogent also announced today plans to describe its novel JAK2 V617F mutant-selective inhibitor as part of a poster presentation at the 2025 ASH (Free ASH Whitepaper) annual meeting.

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"On behalf of our clinical trial investigators, Cogent is honored to announce multiple presentations from the SUMMIT trial at this year’s annual ASH (Free ASH Whitepaper) meeting, including two oral presentations which will highlight the exciting results that bezuclastinib demonstrated in NonAdvSM patients," said Andrew Robbins, the company’s President and Chief Executive Officer. "These data support our conviction that based on bezuclastinib’s potential to fundamentally modify the disease in NonAdvSM patients, it has the potential to become the preferred standard of care in this patient population. In addition, at ASH (Free ASH Whitepaper) we look forward to sharing preclinical data from our newest research program, a novel JAK2 V617F mutant-selective inhibitor which we believe has best-in-class potential based on its potency and selectivity."

Bezuclastinib Oral Presentations

Efficacy and safety results from the primary analysis of the pivotal Summit trial: Bezuclastinib in adults with non-advanced systemic mastocytosis

Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Expanding the Therapeutic and Prognostic Landscape in Myeloproliferative Neoplasms, Mastocytosis and Hypereosinophilic Syndrome
Presenter: Lindsay Rein, MD, Associate Professor of Medicine in the Division of Hematologic Malignancies and Cellular Therapy at Duke University
Session Date and Time: December 6, 2025, 9:30 AM – 11:00 AM ET
Presentation Time: 9:45 AM – 10:00 AM ET
Location: – Room – W414CD

The effect of bezuclastinib on the pathobiology of mastocytosis: Changes in BM mast cells, tryptase, and KIT p.D816V variant allele frequency from the pivotal Summit trial

Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Drivers and Mast Cells and Blasts, Oh My! – Insights and Treatments for MPNs and Mastocytosis.
Presenter: Dr. Tracy George, MD, President and Chief Scientific Officer at ARUP Laboratories, Professor of Pathology at the University of Utah School of Medicine
Session Date and Time: December 8, 2025, 4:30 PM – 6:00 PM ET
Presentation Time: 5:00 PM – 5:15 PM ET
Location: – West Hall D2

Bezuclastinib Poster Presentation
Relationship between KIT inhibition by bezuclastinib and effects on disease burden in mouse models of systemic mastocytosis

Session Name: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster III
Session Date and Time: December 8, 2025, 6:00 PM – 8:00 PM ET
Location: – West Halls B3-B4

JAK2 Poster Presentation
Preclinical characterization of a novel, wild-type-sparing, JAK2 V617F mutant-selective inhibitor

Session Name: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster II
Session Date and Time: December 7, 2025, 6:00 PM – 8:00 PM ET
Location: West Halls B3-B4

Full abstracts will be available for online viewing via the ASH (Free ASH Whitepaper) Annual Meeting website: View Source

(Press release, Cogent Biosciences, NOV 3, 2025, View Source [SID1234659249])

On November 3, 2025 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported receipt of a $30 million milestone payment under its collaboration agreement with Kyowa Kirin in connection with the dosing of the first patient in the second of two KOMET-017 Phase 3 registrational trials of ziftomenib, a once-daily, investigational oral menin inhibitor. Kura previously announced receipt of the first $30 million milestone triggered by patient dosing in the KOMET-017 trials in October 2025. Kura has received a total of $105 million in milestone payments to date and expects to receive up to $315 million in additional, near-term milestones under the collaboration.

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KOMET-017 (NCT07007312) comprises two independent, global, randomized double-blind, placebo-controlled Phase 3 trials to evaluate ziftomenib in combination with both intensive and non-intensive chemotherapy regimens in patients with newly diagnosed NPM1-mutated (NPM1-m) or KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). Kura believes KOMET-017 is the only menin inhibitor program actively pursuing registrational trials across both intensive and non-intensive chemotherapy settings.

(Press release, Kura Oncology, NOV 3, 2025, View Source [SID1234659265])

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.

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"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)

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 – 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])