Novartis Pharma AG

Phase 1/2 study initiated for ATX-559, a first-in-class inhibitor of DHX9, focused on patients with BRCA1/2-deficient breast cancer and MSI-H and/or dMMR solid tumors Second program targeting KIF18A anticipated to enter the clinic in 1H 2025, treating patients with chromosomally instable tumors Seasoned research leader Serena Silver, Ph.D., promoted to CSO; Robert A. Copeland, Ph.D., Co-Founder, President, CSO, to retire effective December 31, 2024 LEXINGTON, MA, USA I December 11, 2024 I Accent Therapeutics, a clinical-stage biopharmaceutical company pioneering novel, targeted, small molecule cancer therapeutics, today announced that the first patient has been dosed in the Phase 1/2 clinical trial evaluating the safety and tolerability of ATX-559, a first-in-class oral DHX9 inhibitor. The company also announced the retirement of Robert A. Copeland, Ph.D., Co-Founder, President, and Chief Scientific Officer (CSO), and the promotion of Serena Silver, Ph.D., to CSO. ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, which is reported to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need. The ATX-559 Phase 1/2 study ( NCT06625515 ) is designed to evaluate the molecule’s safety profile at multiple dose levels, assessing tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy. The trial is enrolling solid tumor patients, with a particular focus on patients with BRCA1- or BRCA2-deficient breast cancer and patients with MSI-H and/or dMMR solid tumors (including certain patients with colorectal, endometrial, gastric, and other cancers). The advancement of ATX-559 into the clinic, followed closely by the KIF18A program, which is expected to enter the clinic in 1H 2025, marks a critical milestone for the company to advance potentially transformative therapies for cancer patients. “We are thrilled to begin evaluating ATX-559 in cancer patients. While PARP inhibitors are useful standard treatments for BRCA1- and BRCA2-deficient breast cancer, the majority of patients with metastatic disease will likely need a different treatment within a year or two. Likewise, about half of patients with MSI-H/dMMR colorectal cancer patients will need additional treatments following PD-(L)1 inhibitors,” said Jason Sager, M.D., Chief Medical Officer of Accent Therapeutics. “With ATX-559 entering clinical trials and Accent’s KIF18A inhibitor poised to begin Phase 1 studies, we are well-positioned to translate our rigorous scientific research into the development of multiple new medicines for treating cancer. These programs represent years of the Accent Therapeutics team’s pioneering research and scientific rigor as we seek to identify meaningful new treatment options for patients who face these challenging malignancies.” Effective January 1, 2025, Serena Silver, Ph.D., will be promoted to the position of Chief Scientific Officer. Dr. Silver joined Accent in September 2022 as Vice President of Biology, bringing a breadth of experience across target discovery, drug discovery, and translational research. Prior to joining Accent, Dr. Silver was Vice President of Discovery Biology and Technologies at Fulcrum Therapeutics, where she led scientific teams to develop and deploy new assay modalities and complex cellular models of disease to identify targets and discover therapeutics for rare diseases. Previously, Dr. Silver led the Molecular Pharmacology group at Novartis Oncology, the Target ID and Validation team at Sanofi Oncology, and worked at the forefront of functional genomics screening technology at the Broad Institute. Dr. Silver holds a Ph.D. in Biology from the Massachusetts Institute of Technology and completed a postdoctoral fellowship at Harvard Medical School. “We are delighted to promote Dr. Silver to the position of Chief Scientific Officer. Her exceptional expertise in cancer biology and strategic vision make her the ideal scientific leader as we continue to advance the science underlying our novel DHX9 and KIF18A programs in our mission to bring innovative treatments to patients,” said Shakti Narayan, Ph.D., J.D., Chief Executive Officer of Accent Therapeutics. “Since co-founding Accent in 2017 and serving as President and CSO, Dr. Copeland’s visionary leadership has been instrumental in shaping our scientific direction. We want to thank Bob for his invaluable contributions to Accent and wish him the best for the future.” Dr. Copeland has served as Co-Founder, President, and Chief Scientific Officer at Accent since the company’s founding in 2017. He was formerly President of Research and CSO of Epizyme, Inc. and before that, Vice President of Cancer Biology in the Oncology Center of Excellence in Drug Discovery at GlaxoSmithKline. He has contributed to drug discovery and development efforts leading to 20 investigational new drugs entering human clinical trials. He is a member of the editorial boards of Molecular Cancer Therapeutics (AACR) and ACS Medicinal Chemistry Letters. He also serves on multiple biotechnology scientific advisory boards. Dr. Copeland received his doctorate in chemistry from Princeton University and was the Chaim Weizmann Fellow at the California Institute of Technology. He was elected a Fellow of the American Association for the Advancement of Science (AAAS) and of the Royal Society of Chemistry. “I am honored to step into the role of CSO at Accent,” said Dr. Silver. “Our team has made significant strides towards advancing transformative medicines for cancer patients, and I am excited to lead our scientific efforts as we enter the clinic. I look forward to leveraging our exceptional talent and rigorous scientific foundation to continue translating our discoveries into impactful therapies for patients.” In addition to ATX-559, Accent anticipates initiating a Phase 1 trial in 1H 2025 for its second program targeting KIF18A in chromosomally instable tumors. The transition to a clinical-stage company marks a meaningful inflection point as Accent continues to advance its pipeline of innovative cancer therapies. About ATX-559 ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, which is reported to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need. DHX9 has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability. In addition to exploiting key tumor vulnerabilities in DNA repair deficient backgrounds (e.g., BRCA) and hyper-mutated states (e.g., MSI-H/dMMR), Accent is exploring the sensitivity of other tumor types to DHX9 inhibition, and the potential to combine DHX9 inhibitors with other cancer treatments to maximize its full potential for helping patients. Therefore, this enzyme represents a compelling novel oncology target. Accent retains full worldwide rights to ATX-559, currently being evaluated in a Phase 1/2 clinical trial ( NCT06625515 ), and the DHX9 program. About KIF18A Accent’s second lead program is a potential best-in-class inhibitor for KIF18A which may address a large patient population across several cancer indications, including ovarian and triple negative breast cancer (TNBC). KIF18A is a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, but not in healthy cells. 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. Accent retains full worldwide rights to the KIF18A program, which is anticipated to enter the clinic in 1H 2025. About Accent Therapeutics Accent Therapeutics is pioneering a new class of small molecule precision cancer therapies targeting critical intracellular dependencies that span multiple types of cancer. Building upon industry-leading expertise in RNA-modifying proteins (RMPs) and the systematic mapping of both the RMP space and adjacent high-value areas for drug discovery, the company employs a flexible model that allows for a diversity of approaches to developing potentially transformative biomarker-driven cancer medicines. Accent’s therapies are designed for both novel and known, but suboptimally addressed, high-impact oncology targets with the potential to benefit large patient populations with significant unmet need. For more information on Accent’s mission to translate extraordinary science into life-changing therapeutics for patients living with cancer, visit www.accenttx.com or follow us on LinkedIn . SOURCE: Accent Therapeutics

Phase 1/2 study initiated for ATX-559, a first-in-class inhibitor of DHX9, focused on patients with BRCA1/2-deficient breast cancer and MSI-H and/or dMMR solid tumors Second program targeting KIF18A anticipated to enter the clinic in 1H 2025, treating patients with chromosomally instable tumors Seasoned research leader Serena Silver, Ph.D., promoted to CSO; Robert A. Copeland, Ph.D., Co-Founder, President, CSO, to retire effective December 31, 2024 LEXINGTON, Mass., Dec. 11, 2024 /PRNewswire/ -- Accent Therapeutics, a clinical-stage biopharmaceutical company pioneering novel, targeted, small molecule cancer therapeutics, today announced that the first patient has been dosed in the Phase 1/2 clinical trial evaluating the safety and tolerability of ATX-559, a first-in-class oral DHX9 inhibitor. The company also announced the retirement of Robert A. Copeland, Ph.D., Co-Founder, President, and Chief Scientific Officer (CSO), and the promotion of Serena Silver, Ph.D., to CSO. ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, which is reported to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need. The ATX-559 Phase 1/2 study (NCT06625515) is designed to evaluate the molecule's safety profile at multiple dose levels, assessing tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy. The trial is enrolling solid tumor patients, with a particular focus on patients with BRCA1- or BRCA2-deficient breast cancer and patients with MSI-H and/or dMMR solid tumors (including certain patients with colorectal, endometrial, gastric, and other cancers). The advancement of ATX-559 into the clinic, followed closely by the KIF18A program, which is expected to enter the clinic in 1H 2025, marks a critical milestone for the company to advance potentially transformative therapies for cancer patients. "We are thrilled to begin evaluating ATX-559 in cancer patients. While PARP inhibitors are useful standard treatments for BRCA1- and BRCA2-deficient breast cancer, the majority of patients with metastatic disease will likely need a different treatment within a year or two. Likewise, about half of patients with MSI-H/dMMR colorectal cancer patients will need additional treatments following PD-(L)1 inhibitors," said Jason Sager, M.D., Chief Medical Officer of Accent Therapeutics. "With ATX-559 entering clinical trials and Accent's KIF18A inhibitor poised to begin Phase 1 studies, we are well-positioned to translate our rigorous scientific research into the development of multiple new medicines for treating cancer. These programs represent years of the Accent Therapeutics team's pioneering research and scientific rigor as we seek to identify meaningful new treatment options for patients who face these challenging malignancies." Effective January 1, 2025, Serena Silver, Ph.D., will be promoted to the position of Chief Scientific Officer. Dr. Silver joined Accent in September 2022 as Vice President of Biology, bringing a breadth of experience across target discovery, drug discovery, and translational research. Prior to joining Accent, Dr. Silver was Vice President of Discovery Biology and Technologies at Fulcrum Therapeutics, where she led scientific teams to develop and deploy new assay modalities and complex cellular models of disease to identify targets and discover therapeutics for rare diseases. Previously, Dr. Silver led the Molecular Pharmacology group at Novartis Oncology, the Target ID and Validation team at Sanofi Oncology, and worked at the forefront of functional genomics screening technology at the Broad Institute. Dr. Silver holds a Ph.D. in Biology from the Massachusetts Institute of Technology and completed a postdoctoral fellowship at Harvard Medical School. "We are delighted to promote Dr. Silver to the position of Chief Scientific Officer. Her exceptional expertise in cancer biology and strategic vision make her the ideal scientific leader as we continue to advance the science underlying our novel DHX9 and KIF18A programs in our mission to bring innovative treatments to patients," said Shakti Narayan, Ph.D., J.D., Chief Executive Officer of Accent Therapeutics. "Since co-founding Accent in 2017 and serving as President and CSO, Dr. Copeland's visionary leadership has been instrumental in shaping our scientific direction. We want to thank Bob for his invaluable contributions to Accent and wish him the best for the future." Dr. Copeland has served as Co-Founder, President, and Chief Scientific Officer at Accent since the company's founding in 2017. He was formerly President of Research and CSO of Epizyme, Inc. and before that, Vice President of Cancer Biology in the Oncology Center of Excellence in Drug Discovery at GlaxoSmithKline. He has contributed to drug discovery and development efforts leading to 20 investigational new drugs entering human clinical trials. He is a member of the editorial boards of Molecular Cancer Therapeutics (AACR) and ACS Medicinal Chemistry Letters. He also serves on multiple biotechnology scientific advisory boards. Dr. Copeland received his doctorate in chemistry from Princeton University and was the Chaim Weizmann Fellow at the California Institute of Technology. He was elected a Fellow of the American Association for the Advancement of Science (AAAS) and of the Royal Society of Chemistry. "I am honored to step into the role of CSO at Accent," said Dr. Silver. "Our team has made significant strides towards advancing transformative medicines for cancer patients, and I am excited to lead our scientific efforts as we enter the clinic. I look forward to leveraging our exceptional talent and rigorous scientific foundation to continue translating our discoveries into impactful therapies for patients." In addition to ATX-559, Accent anticipates initiating a Phase 1 trial in 1H 2025 for its second program targeting KIF18A in chromosomally instable tumors. The transition to a clinical-stage company marks a meaningful inflection point as Accent continues to advance its pipeline of innovative cancer therapies. About ATX-559 ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, which is reported to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need. DHX9 has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability. In addition to exploiting key tumor vulnerabilities in DNA repair deficient backgrounds (e.g., BRCA) and hyper-mutated states (e.g., MSI-H/dMMR), Accent is exploring the sensitivity of other tumor types to DHX9 inhibition, and the potential to combine DHX9 inhibitors with other cancer treatments to maximize its full potential for helping patients. Therefore, this enzyme represents a compelling novel oncology target. Accent retains full worldwide rights to ATX-559, currently being evaluated in a Phase 1/2 clinical trial (NCT06625515), and the DHX9 program. About KIF18A Accent's second lead program is a potential best-in-class inhibitor for KIF18A which may address a large patient population across several cancer indications, including ovarian and triple negative breast cancer (TNBC). KIF18A is a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, but not in healthy cells. 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. Accent retains full worldwide rights to the KIF18A program, which is anticipated to enter the clinic in 1H 2025. About Accent Therapeutics Accent Therapeutics is pioneering a new class of small molecule precision cancer therapies targeting critical intracellular dependencies that span multiple types of cancer. Building upon industry-leading expertise in RNA-modifying proteins (RMPs) and the systematic mapping of both the RMP space and adjacent high-value areas for drug discovery, the company employs a flexible model that allows for a diversity of approaches to developing potentially transformative biomarker-driven cancer medicines. Accent's therapies are designed for both novel and known, but suboptimally addressed, high-impact oncology targets with the potential to benefit large patient populations with significant unmet need. For more information on Accent's mission to translate extraordinary science into life-changing therapeutics for patients living with cancer, visit or follow us on LinkedIn. Media Contact Amanda Sellers, Deerfield Group [email protected] SOURCE Accent Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

A case study on how a leading pharma company in hematology turned to DelveInsight for long-term competitive intelligence support. DelveInsight provided an in-depth analysis of competitive activity in key hematological malignancies, helping the company stay ahead of the competition. LAS VEGAS, Dec. 4, 2024 /PRNewswire/ -- DelveInsight, a leader in healthcare competitive intelligence and consulting, released a case study on Competitive Intelligence in the Hematology Domain. A Europe-based client approached DelveInsight to evaluate a long-term partnership for insights supporting their clinical, commercial, and launch strategies for a novel oncology product targeting acute myeloid leukemia (AML), multiple myeloma, and acute lymphoblastic leukemia (ALL). The client sought detailed intelligence on competitive activity, class-wise market trends, launch estimates in countries of interest, and regulatory landscapes to inform their development and commercialization plans. An additional request for the segregation of mutation/protein expression-specific therapies was also taken into consideration. DelveInsight provided timely, in-depth analysis of the competitive landscape, emerging therapies, and regulatory situations across multiple countries. This enabled the client to make informed decisions on clinical development, market positioning, and commercialization, helping them stay ahead of the competition in the hematology space. With hundreds of thousands of new cases diagnosed annually, hematologic cancers, such as acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), and multiple myeloma, among others present a serious global health challenge while also opening doors for breakthroughs in targeted and personalized treatments. These cancers affect the blood, bone marrow, and lymphatic system, and often require aggressive therapies due to their rapid progression and complex nature. AML and ALL are particularly aggressive, with AML being one of the most common forms of leukemia in adults and ALL affecting both children and adults. Innovations in precision medicine and immunotherapy are transforming hematology, with CAR-T therapies showing promise for leukemias and CELMODs, like Iberdomide, emerging as potential successors to REVLIMID for multiple myeloma. Bristol Myers Squibb's collaboration with Karyopharm on mezigdomide highlights the growing focus on CELMODs. Additionally, FDA-approved therapies like CARVYKTI and TECVAYLI, along with ongoing advancements in bispecific antibodies and proteasome inhibitors, are reshaping the multiple myeloma treatment landscape, despite safety, cost, and manufacturing challenges. The market is rapidly evolving, with new therapies continually entering the pipeline. Several companies, including Bristol Myers Squibb, Celgene, Arcellx, Novartis, Regeneron Pharmaceuticals, Cartesian Therapeutics, Heidelberg Pharma, BeiGene, CARsgen Therapeutics, C4 Therapeutics, AbbVie (TeneoOne), Roche (Genentech), Pfizer, Agios Pharmaceuticals, Jazz Pharmaceuticals, Astellas Pharma, Novartis Oncology, Daiichi Sankyo, AstraZeneca/Astex Pharmaceuticals, Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Autolus Therapeutics, Cellectis, Wugen, Servier, Allogene, and Precision BioSciences, are currently advancing the therapeutic landscape for hematological disorders. As research and innovation progress, there is renewed hope for improving survival rates and enhancing the quality of life for those affected by these challenging diagnoses. DelveInsight's Methodology for Competitive Intelligence in the Hematology Domain helped the client by: Comparative Analyses: Evaluating competitor products to identify strengths, weaknesses, and market positioning, enabling the client to understand where their product stands relative to competitors along with identifying the patient segment with high unmet need KOL Insights: Gathering opinions from Key Opinion Leaders (KOLs) on clinical development strategies, client's product acceptability scenarios, data presentation, probable uptake of their asset,, providing valuable perspectives to refine the client's approach. Current Treatment Paradigms: Analyzing existing treatment approaches to identify market trends, gaps, and unmet needs, helping the client position their product effectively in the market. In-depth Competitive Benchmarking: Assessing competitors' commercial launch preparedness, patient awareness campaigns, and pricing strategies to inform the client's product's market entry and competitive strategy. Market Intelligence: Providing insights into competitors' marketing, sales, and patient engagement approaches, guiding the client's commercialization and go-to-market strategies. Clinical Development Trends: Evaluating clinical trial timelines and strategies, tracking phase shifts in terms of probability of their entry into the higher phase, and analyzing trial data to anticipate future market shifts and inform strategic decision-making. Pricing and Access Strategies: Analyzing competitor pricing models and access strategies, allowing the client to make informed decisions on pricing and reimbursement for their product. Request a proposal to see how DelveInsight's Competitive Intelligence services can support your long-term strategic insights. How DelveInsight's In-Depth Competitive Intelligence in Hematology Helped Clients Stay Ahead in the Market Periodic Competitive Inputs: Provided regular updates on competitor activities, keeping the client informed of market shifts. Enhanced Market Awareness: Delivered ongoing insights into competitor strategies, enabling quick adaptation to market changes. Short-Term Strategies: Developed actionable strategies for immediate response to competitor moves. Counter-Messaging Design: Assisted in crafting counter-messaging strategies to address competitor claims and strengthen brand positioning. Positioning Refinement: Helped refine product positioning to gain a competitive advantage. Promotional Strategy Support: Offered recommendations on promotional tactics to boost market presence and attract key audiences. Why Choose DelveInsight? DelveInsight offers tailored, comprehensive competitive intelligence to keep your hematology product developmental and commercial strategy a step ahead. With expertise in AML, Multiple Myeloma, ALL, and other hematology domains, we support your clinical, commercial, and launch strategies with insights that matter. What Sets Us Apart: Timely Intelligence: Consistent, real-time updates on competitor activities, clinical shifts, and regulatory changes, ensuring you're never caught off-guard. Expert Analysis: From comparative assessments to pricing benchmarks, we provide actionable data that sharpens your positioning. Strategic Edge: We don't just offer data—we deliver strategic recommendations for counter-messaging, promotional tactics, and market adaptation. Choosing DelveInsight means partnering with a team dedicated to turning competitive intelligence into growth and success for your business. Transform your business with our case study. Schedule a call with our DelveInsight Business Development expert today! Conference Coverage Services : DelveInsight's Conference Coverage Services offer a thorough analysis of outcomes from major events like ASH, EHA, AACR, ESMO, ASCO, and others. This detailed examination provides businesses with essential insights for competitive intelligence and market trend forecasting, supporting the formulation of future strategies. Competitive Intelligence Services: DelveInsight's competitive intelligence services provide real-time, accurate insights across the pharmaceutical, life sciences, and medical diagnostics sectors, helping clients stay ahead in a dynamic market. With over 100 years of combined experience, our experts deliver actionable strategic insights, backed by robust research methodologies, to support critical business decisions and create a competitive advantage for our clients. Hematology Pipeline Assessment: Our hematology pipeline evaluation offers a comprehensive overview of cancer treatment options, clinical trials, and key areas within the hematological disorders field, all summarized in an accessible report format. The hematology-focused reports, available in our Report Store, provide in-depth insights into treatment strategies, mechanisms of action (MoAs), drug components, and launch plans for therapies in the late stages of development. This assessment enhances understanding of their launch timelines and strategic initiatives. R&D Analysis: Our skilled research and development analyst specializes in the complexities of hematology, offering essential insights and advice to clients aiming to advance in hematological research and development. They utilize a methodical assessment approach, aligned with industry norms, to track the progress of each hematology drug in both pre-clinical and clinical stages. DelveInsight's comprehensive hematological reports are crafted to reduce uncertainties and highlight overlooked areas, providing organizations with timely and strategic intelligence. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED