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Lexington, MA 02421
Tel: (339) 970-7400

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APPROACH & PIPELINE

Small molecule therapies with expansive potential

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Our Approach

Our deep understanding of the key biological underpinnings of cancer and ability to identify, interrogate, and successfully drug intracellular enzyme targets enables us to advance unique small molecule therapeutics with expansive potential.

Leveraging our industry-leading expertise in genomic instability, we have built a flexible model that allows for a diversity of approaches with the goal of developing transformative biomarker-driven cancer medicines. Our therapies are designed for high-impact oncogenic targets with the potential to benefit large patient populations.

Our lead program, ATX-295, is a potential best-in-class inhibitor for KIF18A, a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, or CIN. KIF18A inhibitor treatment results in rapid cell death for cancers with an abnormal number of chromosomes (aneuploid) in vitro and in vivo, while cells with normal numbers of chromosomes (euploid) are unaffected. ATX-295 may address a large patient population across several cancer indications, including ovarian and squamous non-small cell lung cancer.

KIF18A is a selectively-essential target for tumors

with high levels of chromosomal instability (CIN)

 Illustration showing how KIF18A inhibition prevents cell division in cancer cells with chromosomal instability
Illustration showing how KIF18A inhibition prevents cell division in cancer cells with chromosomal instability
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Pipeline

Accent is relentlessly pursuing targets with the potential to improve outcomes for broad biomarker-driven patient populations. Accent retains full worldwide rights to the KIF18A program, currently being evaluated in an ongoing Phase 1/2 clinical study (NCT06799065) enrolling patients with locally advanced or metastatic solid tumors, including high-grade serous ovarian cancer and squamous non-small cell lung cancer.

For inquiries related to clinical studies, please contact us at clinicaltrials@accenttx.com

INDICATION
PRECLINICAL
IND-ENABLING
Phase 1/2
Registrational
High Grade Serous Ovarian Cancer (HGSOC)
PHASE 1/2

Platinum-Resistant Ovarian Cancer (PROC) - Redefining the Horizon for Ovarian Cancer

While initial platinum-based treatment for ovarian cancer often yields strong response, 60-70%1 of patients will eventually develop resistance. For patients facing platinum-resistant ovarian cancer (PROC), the treatment landscape has historically been limited to single-agent chemotherapies with low response rates and cumulative toxicities. Further, while next-generation antibody-drug conjugates (ADCs) may target tumor cells more selectively, most patients will still rapidly develop resistance, leaving significant unmet medical need for non-chemotherapy and non-antibody-based therapeutic options. At Accent, we recognize that these patients cannot wait for incremental progress. We are driven by the urgent need to advance ATX-295, our potentially best-in-class KIF18A inhibitor, for PROC patients and to provide a clinically meaningful and oral treatment option.

Squamous Non-Small Cell Lung Cancer (sqNSCLC)
PHASE 1/2

Squamous Non-Small Cell Lung Cancer (sqNSCLC) - Closing the Gap in Precision Oncology

Squamous cell carcinoma represents a significant portion - 20% to 30%2 - of all lung cancer diagnoses, yet it has remained largely in the shadow of the genomically-driven cancer precision medicine revolution we have witnessed in non-squamous NSCLC. Unlike other lung cancer subtypes, sqNSCLC lacks common actionable mutations, leaving patients heavily reliant on traditional chemotherapy and immunotherapies. When these standard options are exhausted, the therapeutic void is profound - current SOC is docetaxel with a response rate <10% and PFS <3 mo. We are committed to changing this narrative by targeting the unique high-CIN biology of sqNSCLC with ATX-295, aiming to provide meaningful benefits and improved quality of life for a patient population that has been historically underserved.

Additional Oncology Indications and Combinations
IND-ENABLING

Following the Biology to Expand Patient Impact

Because KIF18A inhibition targets a fundamental, shared biological vulnerability across multiple cancer types (e.g., breast, endometrial, head-and-neck), its potential reaches far beyond our initial clinical focus. We are actively researching and evaluating how we can help patients with high unmet medical need whose cancers share these same underlying biological signatures.

We are also rigorously exploring how ATX-295 can combine with both established standard of care and novel therapeutic agents. By combining with other mechanistically-aligned treatments, ATX-295 has the potential to deepen patient response and durability of treatment even further than either therapeutic alone.

PRECLINICAL
  1. Caruso et al., Ovarian Cancer: A Review; JAMA 2025
  2. Paik, Paul K. et al. Squamous Non-Small Cell Lung Cancer: Current and Emerging Treatment Options; Clinical Lung Cancer, October 2025.

For information on clinical trials, please visit https://clinicaltrials.gov.

Our Expanded Access Policy

Expanded access, or compassionate use, is the use of an investigational medicine prior to regulatory approval and outside of a clinical trial.

We understand that for patients with serious diseases such as cancer, waiting for an approved treatment can be difficult. However, Accent Therapeutics does not currently offer expanded access to our investigational products.

​We may reevaluate the status of our Expanded Access Policy as development of our investigational products progresses. Accent Therapeutics reserves the right to revise this Expanded Access Policy at any time.

We encourage you to speak with your physician about options that may be right for you. For inquiries related to clinical studies, please contact us at clinicaltrials@accenttx.com.

Presentations & Publications

Key scientific research published by the Accent team and collaborators.

4.21.26 | AACR 2026

Potent and Durable Anti-Tumor Activity of the Novel KIF18A Inhibitor, ATX-295, in Preclinical Models of Chromosomally Instable Tumors

View Presentation
10.22.25 | AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics 2025

Activity of the Novel KIF18A Inhibitor, ATX-295, is Enriched in Whole Genome Doubled Ovarian and TNBC Preclinical Models

View Presentation
10.22.25 | AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics 2025

ATX-559, a First-in-Class, Clinical-Stage DHX9 Inhibitor, as a Targeted Therapeutic for Molecularly Defined Tumors with Genomic Instability and Replicative Stress

View Presentation
6.2.25 | ASCO 2025

Trial in Progress: First-in-Human Study of ATX-559, an Oral Inhibitor of DHX9, in Patients with Advanced or Metastatic Solid Tumors, and Molecularly Defined Cancers

View Presentation
4.28.25 | AACR 2025

ATX-559, a First in Class DHX9 Inhibitor, and Targeted Therapeutic for Molecularly Defined Tumors with Genomic Instability and Replicative Stress

View Presentation
4.28.25 | AACR 2025

Activity of the Novel KIF18A Inhibitor, ATX-295, is Enriched in Whole Genome Doubled Ovarian Cancer Pre-Clinical Models

View Presentation
10.25.25 | ACS Medicinal Chemistry Letters

Discovery of Kinesin KIF18A Inhibitor ATX020: Tactical Application of Silicon Atom Replacement

View Publication
4.29.25 | Journal of Medicinal Chemistry

Discovery of ATX968: An Orally Available Allosteric Inhibitor of DHX9

View Publication
4.14.25 | Nature

Nature Magazine Communications Biology Characterization of exoribonuclease XRN1 as a cancer target and identification of adenosine-3’,5’-bisphosphate as a potent enzyme inhibitor

View Publication
4.9.25 | Nature

Characterization of exoribonuclease XRN1 as a cancer target and identification of adenosine-3’,5’-bisphosphate as a potent enzyme inhibitor

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11.26.24 | Cancer Research

A Potent, Selective, Small-Molecule Inhibitor of DHX9 Abrogates Proliferation of Microsatellite Instable Cancers with Deficient Mismatch Repair

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9.30.24 | Epigenetic Drug Discovery Book Chapter

RNA Modifying Proteins: Emerging Targets for Drug Discovery

View Publication

Partnerships

Our collaborations with strategic partners with deep oncology expertise enable us to further advance and achieve the rich therapeutic potential of our clinical programs. For more information about collaborations please contact us at collaboration@ACCENTTX.COM.

AstraZeneca Logo

Accent and AstraZeneca have a transformational partnership to discover, develop and commercialize certain novel therapeutics targeting RNA-modifying proteins (RMPs) for the treatment of cancer. The collaboration seeks to rapidly advance and achieve the rich therapeutic potential of the exciting programs under partnership by combining AstraZeneca's proven track record in bringing forward novel oncology medicines with Accent's industry-leading expertise in the biology, target identification and drug discovery of RMP-targeting therapies.

Boehringer Ingelheim Logo

Boehringer Ingelheim has entered into an agreement to acquire from Accent a novel preclinical small molecule program with an approach for tumors characterized by high interferon-stimulated gene (ISG) expression. Boehringer Ingelheim has assumed global responsibility for the program’s research, development, manufacturing, and commercialization, advancing it toward clinical evaluation.

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