Treadwell Therapeutics, Inc.

With a Focus on Potential Pivotal Studies in 2025, Treadwell has Strengthened its Leadership Team by Appointing Brenda Marczi, PharmD, MBA as SVP and Head of Regulatory Affairs TORONTO & SAN FRANCISCO--(BUSINESS WIRE)-- Treadwell Therapeutics, a privately held clinical-stage biotechnology company pioneering and advancing novel first-in-class medicines for unmet needs in cancer, today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ocifisertib (CFI-400945), a first-in-class, investigational PLK4 inhibitor for the treatment of acute myeloid leukemia (AML). Ocifisertib is currently being evaluated in a Phase 1b/2 study in adults with relapsed/refractory AML following standard of care therapy. Orphan drug designation is granted by the FDA to drugs intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation. Under the FDA’s Orphan Drug Act, orphan drug status provides incentives, grants, tax credits and waiver of certain administrative fees as well as seven years of market exclusivity following marketing authorization. Treadwell also announced today the appointment of Brenda Marczi, as SVP and Head of Regulatory Affairs. Brenda comes to Treadwell with over 30 years of regulatory experience in the pharmaceutical and biotechnology industries. She most recently served as Senior Vice President, Regulatory Affairs at Tracon Pharmaceuticals, Inc., where she oversaw their regulatory function and led all of their strategies and filing activities in the US and UK. Brenda holds a Master of Science in Pharmaceutical Sciences from Rutgers University, a PharmD from the University of Maryland, and an MBA from Wharton, University of Pennsylvania. “The FDA’s decision to grant orphan drug designation, along with the previous FDA Fast Track designation for ocifisertib underscores Treadwell’s dedication to addressing this patient population with few treatment options. Patients with relapsed and/or refractory AML, in particular TP53 mutated disease suffer poor overall survival and represents a high unmet clinical need,” said Roger Sidhu, M.D., Acting CEO of Treadwell. “We look forward to advancing ocifisertib in partnership with investigators, regulators, patients and their families for those with limited treatment options in tough to treat AML. In addition, we are thrilled to have Brenda join us to lead Regulatory Affairs at Treadwell. She is a seasoned strategic leader with a broad experience in leading regulatory strategy from early development through approval. Her leadership will be invaluable to Treadwell as we advance ocifisertib into potentially pivotal studies in 2025.” About Treadwell Therapeutics Treadwell Therapeutics is a clinical-stage oncology company developing novel medicines to address unmet needs in patients with cancer. The Company's robust, internally developed clinical pipeline includes ocifisertib (PLK4 inhibitor), CFI-402257 (TTK/Mps1 inhibitor) and CFI-402411 (HPK1 inhibitor). Treadwell also has a broad pre-clinical pipeline with multiple biologic and next generation TCR based autologous cell therapy programs. For more information, please visit .

TORONTO and SAN MATEO, Calif., Dec. 15, 2023 (GLOBE NEWSWIRE) -- Treadwell Therapeutics, a clinical-stage biotechnology company developing novel first-in-class medicines for unmet needs in cancer, today announced a presentation for the Company's lead program, CFI-400945, a first-in-class inhibitor of Polo-like Kinase 4 (PLK4) being advanced in relapsed/refractory AML, at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition being held from December 9-12, 2023. Treadwell also conducted a clinical advisory board meeting on December 10th at ASH with experienced experts in the treatment of AML and in clinical trials of novel agents, which included current investigators and thought leaders new to the program. "CFI-400945 is demonstrating complete remissions as a single agent and in combination with azacitadine in relapsed, adverse risk AML, including those with TP53 mutant disease. As we optimize dose and schedule, we are encouraged by the data with this oral dosing regimen," said Principal Investigator and Advisory Board Co-Chair Dr. Gautam Borthakur, MD, Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. “The advisory board confirmed the enthusiasm about the data to date in the relapsed AML setting, in particular with TP53 mutant disease and look forward to working with Treadwell to advance and shape this program.” "CFI-400945, our lead program and first-in-class PLK4 inhibitor, represents an opportunity to address a substantial unmet in relapsed/refractory AML, including TP53 mutant AML," said Roger Sidhu, MD, Acting CEO at Treadwell Therapeutics. “We look forward to advancing the program into key dose expansion studies in monotherapy and in combination in 2024 with a view to initiating pivotal studies in 2025.” 2023 ASH Poster Presentations and Details: Title: Preliminary Results from a Phase 1b/2 Open-Label, Multicenter, Dose Optimization Clinical Study of the Safety, Tolerability, and Pharmacokinetic (PK) and Pharmacodynamic (PD) Profiles of CFI-400945 As a Single Agent or in Combination with Azacitidine in Patients (Pts) with Acute Myeloid Leukemia, Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (TWT-202) Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster III Publication Number: 4294 Session Date: Monday, December 11, 2023 Presentation Time: 6:00 PM - 8:00 PM Location: San Diego Convention Center, Halls G-H CFI-400945 has been generally well tolerated in this difficult to treat patient population, including patients whose disease has progressed on or following venetoclax based therapies​. Three of 6 evaluable patients with AML achieved a response (MLFS=2, CRi=1 with MRD+) at the 96mg dose​. After this data cut, one additional MLFS response (end of cycle 1) and two additional CRs (end of cycle 2) were seen in two patients treated with 80 mg + azacitidine combination cohort and became transplant eligible (PI communication)​. PK characteristics support daily dosing of CFI-400945 and PD studies are ongoing​. Dose expansions are planned and enrollment continues​. About Treadwell Therapeutics Treadwell Therapeutics is a clinical-stage oncology company developing novel medicines to address unmet needs in patients with cancer. The Company's robust, internally developed clinical pipeline includes CFI-400945 (PLK4 inhibitor), CFI-402257 (TTK/Mps1 inhibitor) and CFI-402411 (HPK1 inhibitor). Treadwell also has a broad pre-clinical pipeline with multiple biologic and next generation TCR based autologous cell therapy programs. For more information, please visit Contact ir@treadwelltx.com

– Treadwell to focus on advancing first-in-class PLK4 inhibitor CFI-400945 in AML towards potential pivotal studies in 2025 – Approximately 30% reduction in workforce to extend cash runway – Roger Sidhu, M.D. appointed as Acting CEO replacing founding Co-CEOs, Shane Burgess and Michael Tusche, Ph.D., Shane Burgess to transition to Executive Chair TORONTO and SAN FRANCISCO, Dec. 06, 2023 (GLOBE NEWSWIRE) -- Treadwell Therapeutics, a clinical-stage biotechnology company developing novel first-in-class medicines for unmet needs in cancer, today announced that it is realigning its pipeline, workforce and management structure to support its highest value program and extend its cash runway to prioritize the execution of key near term value drivers and clinical milestones. Treadwell has elected to focus on its first-in-class PLK4 inhibitor, CFI-400945, in relapsed/refractory AML by expanding the ongoing company sponsored TWT-202 study, building on exciting signals of efficacy seen at earlier phases, and advancing towards a potential pivotal study in 2025. The company will seek to capitalize on very promising proof-of-concept from sponsored clinical studies of CFI-402257 (TTK/Mps1 inhibitor) and CFI-402411 (HPK1 inhibitor) through further, innovative collaborative studies and work with potential partners to accelerate timelines. Shane Burgess, co-CEO has transitioned to the role of Executive Chair, while Michael Tusche, Ph.D., co-CEO has left the company to pursue other endeavours. Roger Sidhu, M.D., Chief Medical Officer, has been appointed Acting CEO. Dr. Sidhu brings to Treadwell an extensive track record of success over the last nearly 20 years in hematologic and solid tumor oncology research and development. He is a seasoned leader with deep translational, clinical and regulatory experience across multiple therapeutic modalities and platforms including small molecules, biologics and cell and gene therapies. Most recently, Dr. Sidhu was the Chief Medical Officer at Eterna Therapeutics, a gene editing and cell therapy company where he advanced multiple novel programs. Previously, Dr. Sidhu served as Executive Vice President and Chief Medical officer at Roivant Sciences. Roger was also the Chief Medical Officer at Cell Design Labs, up until its acquisition by the Gilead subsidiary Kite, where he subsequently served as VP, Clinical Development. Dr. Sidhu also held various roles of increasing responsibility at Amgen over a nearly decade that included managing FDA and EU approval of panitumumab in metastatic colorectal cancer. Dr. Sidhu is a Fellow of the Royal College of Physicians and Surgeons of Canada in both internal medicine and medical oncology. He earned his medical degree from Queen's University in Kingston, Ontario Canada. “Roger brings a unique and broad set of skills and strategic thinking to Treadwell over a long and successful career. We are confident that under his leadership, Treadwell will aggressively advance into an exciting new chapter in our mission to address unmet needs in difficult to treat cancers with first-in-class medicines,” said Shane Burgess, Executive Chair of Treadwell Therapeutics. “In order to increase operational efficiency and to focus on key, value driving priorities, we have made the difficult business decision to reduce our workforce by approximately 30% and to redirect clinical efforts for several Treadwell-sponsored trials to other avenues. We would like to extend our deep gratitude to the Treadwell team, investigators and patients involved in our clinical programs. These actions allow us to focus on investing in key near term milestones for CFI-400945.” “Treadwell has developed a broad clinical and research-stage pipeline of first-in-class small molecules, biologics and TCR-based cell therapies. We have reached an inflection point which allows us to focus on the most near-term path to value with CFI-400945 in relapsed/refractory AML with a view to advancing to pivotal stage studies,” said Roger Sidhu, M.D., Acting CEO of Treadwell Therapeutics. “Given the very promising clinical data we have generated, we also look forward to advancing CFI-402257 (TTK inhibitor) and CFI-402411 (HPK1 inhibitor) with external collaborators and investigators moving forward in breast cancer and immunotherapy-based treatment of cancer, respectively, in addition to actively seeking partners for our pre-clinical programs.” About Treadwell Therapeutics Treadwell Therapeutics is a clinical-stage oncology company developing novel medicines to address unmet needs in patients with cancer. The Company's robust, internally developed clinical pipeline includes CFI-400945 (PLK4 inhibitor), CFI-402257 (TTK/Mps1 inhibitor) and CFI-402411 (HPK1 inhibitor). Treadwell also has a broad pre-clinical pipeline with multiple biologic and next generation TCR based autologous cell therapy programs. For more information, please visit . Contact ir@treadwelltx.com