Syndax Highlights Recent Accomplishments and Anticipated 2025 Milestones at the 43rd Annual J.P. Morgan Healthcare Conference
Syndax Pharmaceuticals (SNDX) highlighted its achievements and 2025 milestones, featuring two major FDA approvals in 2024: Revuforj® for R/R acute leukemia with KMT2A translocation and Niktimvo™ for chronic GVHD treatment. Both drugs were added to NCCN Clinical Practice Guidelines.
Key developments include positive results from the AUGMENT-101 trial for Revuforj, with plans to submit an sNDA for R/R mNPM1 AML in 1H25. The company secured a $350 million royalty funding agreement with Royalty Pharma for Niktimvo's U.S. sales.
2025 milestones include launching Niktimvo in Q1, maximizing Revuforj adoption, and initiating multiple frontline trials. The company expects its current financial position to support operations through profitability.
Syndax Pharmaceuticals (SNDX) ha messo in evidenza i suoi successi e gli obiettivi per il 2025, con due importanti approvazioni della FDA nel 2024: Revuforj® per la leucemia acuta recidivante con traslocazione KMT2A e Niktimvo™ per il trattamento della GVHD cronica. Entrambi i farmaci sono stati aggiunti alle linee guida di pratica clinica NCCN.
Tra i principali sviluppi ci sono risultati positivi dal trial AUGMENT-101 per Revuforj, con piani per presentare una sNDA per R/R mNPM1 AML nel primo semestre del 2025. L'azienda ha assicurato un accordo di finanziamento in royalties di 350 milioni di dollari con Royalty Pharma per le vendite di Niktimvo negli Stati Uniti.
Le tappe fondamentali del 2025 includono il lancio di Niktimvo nel primo trimestre, la massimizzazione dell'adozione di Revuforj e l'avvio di più studi di prima linea. L'azienda prevede che la sua attuale posizione finanziaria sosterrà le operazioni fino a raggiungere la redditività.
Syndax Pharmaceuticals (SNDX) destacó sus logros y metas para 2025, con dos importantes aprobaciones de la FDA en 2024: Revuforj® para leucemia aguda R/R con translocación KMT2A y Niktimvo™ para el tratamiento de GVHD crónica. Ambos medicamentos fueron añadidos a las Guías de Práctica Clínica de NCCN.
Entre los desarrollos clave se encuentran los resultados positivos del ensayo AUGMENT-101 para Revuforj, con planes de presentar una sNDA para R/R mNPM1 AML en la primera mitad de 2025. La compañía aseguró un acuerdo de financiamiento por regalías de 350 millones de dólares con Royalty Pharma por las ventas de Niktimvo en EE.UU.
Los hitos de 2025 incluyen el lanzamiento de Niktimvo en el primer trimestre, maximizar la adopción de Revuforj y comenzar múltiples ensayos de primera línea. La empresa espera que su posición financiera actual respalde las operaciones hasta alcanzar la rentabilidad.
신닥스 제약(SNDX)은 2025년 목표와 성과를 강조했으며, 2024년에 두 가지 주요 FDA 승인이 있었습니다: Revuforj®는 KMT2A 전위가 있는 R/R 급성 백혈병에 대한 것이고, Niktimvo™는 만성 GVHD 치료를 위한 것입니다. 두 약물 모두 NCCN 임상 진료 지침에 추가되었습니다.
주요 발전 사항으로는 Revuforj에 대한 AUGMENT-101 임상시험에서 긍정적인 결과가 나왔으며, 2025년 상반기에 R/R mNPM1 AML에 대한 sNDA를 제출할 계획입니다. 이 회사는 Niktimvo의 미국 판매를 위해 Royalty Pharma와 3억 5천만 달러의 로열티 자금 조달 계약을 체결했습니다.
2025년 목표에는 Niktimvo의 1분기 출시, Revuforj 채택 극대화, 여러 1차 시험 시작이 포함됩니다. 이 회사는 현재의 재정 상태가 수익성을 달성할 때까지 운영을 지원할 것으로 기대하고 있습니다.
Syndax Pharmaceuticals (SNDX) a mis en avant ses réalisations et ses objectifs pour 2025, avec deux principales approbations de la FDA en 2024 : Revuforj® pour la leucémie aiguë R/R avec translocation KMT2A et Niktimvo™ pour le traitement de la GVHD chronique. Les deux médicaments ont été ajoutés aux lignes directrices de pratique clinique de la NCCN.
Parmi les développements clés, on note des résultats positifs de l'essai AUGMENT-101 pour Revuforj, avec des plans de soumettre une sNDA pour R/R mNPM1 AML au premier semestre 2025. L'entreprise a sécurisé un accord de financement par redevance de 350 millions de dollars avec Royalty Pharma pour les ventes de Niktimvo aux États-Unis.
Les jalons de 2025 incluent le lancement de Niktimvo au premier trimestre, la maximisation de l'adoption de Revuforj et le lancement de plusieurs essais en première ligne. L'entreprise s'attend à ce que sa position financière actuelle soutienne les opérations jusqu'à atteindre la rentabilité.
Syndax Pharmaceuticals (SNDX) hob ihre Erfolge und Ziele für 2025 hervor, einschließlich zwei wesentlicher FDA-Zulassungen im Jahr 2024: Revuforj® für R/R akute Leukämie mit KMT2A-Translokation und Niktimvo™ zur Behandlung der chronischen GVHD. Beide Medikamente wurden in die NCCN-Leitlinien aufgenommen.
Zu den wichtigsten Entwicklungen gehören positive Ergebnisse aus der AUGMENT-101-Studie für Revuforj, mit dem Plan, im ersten Halbjahr 2025 eine sNDA für R/R mNPM1 AML einzureichen. Das Unternehmen sicherte sich eine $350 Millionen große Lizenzgebühren-Finanzierungsvereinbarung mit Royalty Pharma für den Verkauf von Niktimvo in den USA.
Die Meilensteine für 2025 umfassen den Launch von Niktimvo im ersten Quartal, die Maximierung der Revuforj-Adoption und den Beginn mehrerer Frontline-Studien. Das Unternehmen erwartet, dass seine derzeitige finanzielle Lage die Abläufe bis zur Rentabilität unterstützen wird.
- FDA approval of two first-in-class drugs: Revuforj and Niktimvo
- Both drugs added to NCCN Clinical Practice Guidelines
- Secured $350 million royalty funding agreement with Royalty Pharma
- 100% overall response rate in BEAT AML trial for Revumenib combination therapy
- Strong cash position expected to fund operations through profitability
- None.
Insights
The dual FDA approvals and launches of Revuforj and Niktimvo represent major commercial milestones for Syndax. The inclusion of both drugs in NCCN guidelines significantly enhances their adoption potential and reimbursement prospects. The 100% response rate in the BEAT AML trial and 82% response rate in the SAVE trial demonstrate exceptional efficacy that could drive substantial market penetration.
The planned sNDA filing for revumenib in R/R mNPM1 AML could expand the addressable patient population significantly. The combination studies with standard-of-care agents like venetoclax suggest a strategic push toward earlier treatment lines, which typically generate higher revenues due to longer treatment durations.
The
The co-commercialization agreement with Incyte for Niktimvo helps share commercial costs while maintaining significant revenue potential. The expansion into multiple indications and treatment settings creates multiple shots on goal for revenue growth. The company's market position as the first approved menin inhibitor provides a significant first-mover advantage in a highly valuable therapeutic space.
The rapid inclusion in NCCN guidelines for both products indicates strong clinical acceptance and positions them well for market adoption. The comprehensive clinical development strategy across multiple treatment settings suggests potential for significant market expansion. Particularly noteworthy is the 95% composite complete remission rate in the BEAT AML trial, which could drive strong uptake in first-line settings.
The expansion into IPF with axatilimab represents a strategic move into a large additional market opportunity. The multiple ongoing combination trials with standard-of-care treatments indicate a well-planned market penetration strategy that could establish these drugs as backbone therapies in their respective indications.
– Launched Revuforj® (revumenib) for treatment of R/R acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older –
– Revumenib added to NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®)
for AML and ALL –
– Niktimvo™ (axatilimab-csfr) approved by U.S. FDA for treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg –
– sNDA filing for revumenib in R/R mNPM1 AML expected in 1H25 based on positive pivotal data from AUGMENT-101 trial –
"Building on a transformative 2024 with the FDA approvals of Revuforj® and Niktimvo™, we are focused on executing two outstanding
2024 Key Accomplishments
Revumenib:
- Launched Revuforj (revumenib), the first and only
U.S. Food and Drug Administration (FDA) approved menin inhibitor, in late November 2024. Revuforj was approved by the FDA under the Agency's Real-Time Oncology Review (RTOR) program for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older. - Revumenib was added to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) as a category 2A recommendation for R/R acute leukemia with a KMT2A rearrangement.1
- Announced that the primary endpoint was met in the protocol-defined efficacy population of 64 adults with R/R mNPM1 AML in the Phase 2 cohort of the AUGMENT-101 trial of revumenib.
- Reported additional positive results from a post-hoc efficacy analysis of all 77 R/R mNPM1 AML patients who met the efficacy evaluable criteria in the Phase 2 cohort of AUGMENT-101.
- Published data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in adult and pediatric patients with R/R KMT2A-rearranged (KMT2Ar) acute leukemia in the Journal of Clinical Oncology.
- Presented a larger data set with longer follow-up from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in R/R KMT2Ar acute leukemia at the 66th American Society of Hematology (ASH) Annual Meeting.
- Presented data from multiple ongoing trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape. These trials include:
- BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. Updated data from the trial showed an overall response rate (ORR)2 of
100% (37/37) and a composite complete remission (CRc) rate of95% (35/37). - SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or mixed-lineage acute leukemia (MPAL) harboring either mNPM1, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. Updated data that showed an ORR of
82% (27/33) and a CR/CRh rate of48% (16/33) were presented at the 66th ASH Annual Meeting.
- BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. Updated data from the trial showed an overall response rate (ORR)2 of
Axatilimab:
- Received
U.S. Food and Drug Administration (FDA) approval for Niktimvo (axatilimab-csfr) 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). - Announced axatilimab-csfr was added to the latest NCCN Guidelines as a category 2A recommendation for the treatment of GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.
- Published results from the pivotal Phase 2 AGAVE-201 trial of axatilimab in adult and pediatric patients with recurrent/refractory active chronic GVHD in the New England Journal of Medicine.
- Presented a secondary analysis of overall and organ-specific responses from the pivotal Phase 2 AGAVE-201 trial of axatilimab in adult and pediatric patients with recurrent/refractory active chronic GVHD who had received at least two prior lines of systemic therapy at the 66th ASH Annual Meeting.
- Initiated enrollment in the MAXPIRe trial, a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF).
- The Company's partner, Incyte, initiated 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.
- The Company's partner, Incyte, initiated a Phase 3, randomized, double-blind, placebo-controlled, multi-center trial that will investigate the use of axatilimab in combination with corticosteroids as initial treatment for chronic GVHD.
Corporate:
- Announced a
$350 million U.S. net sales of Niktimvo. Syndax expects that its cash, cash equivalents and marketable securities, together with the$350 million
Expected 2025 Key Milestones
Revumenib:
- Maximize
U.S. adoption of Revuforj as the preferred menin inhibitor, leveraging our first mover advantage and robust clinical data. - Submit a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025, followed by a potential FDA approval around year-end 2025.
- Publish pivotal data from AUGMENT-101 trial in R/R mNPM1 AML in the first half of 2025.
- Initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy in the first quarter of 2025.
- Report Phase 1 data from a trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias in the second half of 2025.
- Initiate multiple frontline trials evaluating revumenib in combination with intensive chemotherapy, starting in 2025.
- Present additional data at medical congresses from ongoing trials of revumenib in combination with standard-of-care agents.
Axatilimab:
- Launch Niktimvo in the
U.S. in early first quarter of 2025. In theU.S. , Niktimvo will be co-commercialized by Syndax and Incyte. - Complete enrollment in MAXPIRe Phase 2 IPF trial in 2025 with topline data expected in 2026.
Presentation at the 43rd Annual J.P. Morgan Healthcare Conference
- Syndax will webcast its presentation from the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025 at 10:30 a.m. PT (1:30 p.m. ET). A live webcast of the fireside chat can be accessed from 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.
About Syndax
Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit www.syndax.com/ or follow the Company on X and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Revuforj's commercial availability, changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
References
1. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia (Version 1.2025 – December 20, 2024); NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia (Version 3.2024 – December 20, 2024); NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Pediatric Acute Lymphoblastic Leukemia (Version 2.2025 – December 16, 2024). NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
2. Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS, and PR; Composite complete remission (CRc) includes CR, CRh, CRp, and CRi.
CR = Complete remission
CRh = Complete remission with partial hematologic recovery
CRp = Complete remission with incomplete platelet recovery
CRi = Complete remission with incomplete count recovery
MLFS = Morphologic leukemia-free state
PR = Partial response
Syndax Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
Tel 781.684.9827
SNDX-G
View original content:https://www.prnewswire.com/news-releases/syndax-highlights-recent-accomplishments-and-anticipated-2025-milestones-at-the-43rd-annual-jp-morgan-healthcare-conference-302348949.html
SOURCE Syndax Pharmaceuticals, Inc.
FAQ
What are the FDA-approved indications for Syndax's (SNDX) Revuforj?
When will Syndax (SNDX) submit the sNDA for Revumenib in R/R mNPM1 AML?
What were the clinical results of SNDX's BEAT AML trial for Revumenib?
When will Syndax (SNDX) launch Niktimvo in the United States?
What is the value of SNDX's royalty funding agreement with Royalty Pharma?
