Treadwell Announces Ocifisertib, a First-in-Class PLK4 Inhibitor, has Received Orphan Designation from U.S. FDA for the Treatment of Acute Myeloid Leukemia

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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 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 www.treadwelltx.com.

Contact

ir@treadwelltx.com

Treadwell Announces the Formation and Members of Scientific Advisory Board

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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 formation of a Scientific Advisory Board comprised of prominent oncology leaders and drug developers. The SAB will work closely with Treadwell to advance the company’s pipeline of small molecules, biologics and cell therapies.

“We are delighted and honored to be working with this prestigious group of innovators and thought leaders as our Scientific Advisory Board. We are very much looking forward to collaborating with this group to inform and shape our research and development efforts in advancing our pipeline,” said Roger Sidhu, M.D., Acting CEO of Treadwell. “This SAB brings tremendous experience in advancing novel therapeutics in oncology from early to late stage development including drug approvals. We are confident that this group will be invaluable in supporting the development of our pipeline with a focus on our lead program CFI-400945, a PLK4 inhibitor currently being advanced in AML.”

The founding members of the Treadwell Scientific Advisory Board are:

Antoni Ribas, M.D., Ph.D., is currently Professor of Medicine, Surgery, and Molecular and Medical Pharmacology at the University of California Los Angeles (UCLA), directory of the Tumor Immunology Program at the Jonsson Comprehensive Cancer Center and director of the Parker Institute for Cancer Immunotherapy at UCLA. Dr. Ribas has played a pivotal role in the development and approval pembrolizumab in advanced melanoma and multiple additional drugs in this disease. Dr. Ribas was formerly President of the American Association for Cancer Research and has been elected to the National Academy of Medicine.

Carl June, M.D., is the Richard W. Vague Professor in Immunotherapy in the Department of Pathology and Laboratory Medicine in the Perelman School of Medicine at the University of Pennsylvania and director of Center for Cellular Immunotherapies at the University of Pennsylvania. Dr. June is internationally recognized for his role in pioneering CAR-T cell therapy which led to the first FDA-approved cellular therapy for children and young adults with acute lymphoblastic leukemia. Dr. June has published more than 500 manuscripts and is the recipient of multiple prestigious scientific achievement awards and is a member of the National Academy of Medicine and the National Academy of Sciences.

Pasi A. Jänne, M.D., Ph.D., is a world renowned translational medical oncologist and the Senior Vice President of Translational Medicine at Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School. He is the Director of the Belfer Center for Applied Cancer Science. He was a co-discoverer of epidermal growth factor receptor (EGFR) mutations that has led to the development of multiple therapeutic strategies for patients with EGFR mutant lung cancer. Dr. Jänne has received the 2024 Medal of Honor from the American Cancer Society and multiple awards from ASCO, AACR and ESMO.

S. Gail Eckhardt is Associate Dean of Experimental Therapeutics at Baylor College of Medicine and Associate Director of Translational Research at the Dan L Duncan Comprehensive Cancer Center. Dr. Eckhardt has served on numerous committees and study sections, including the ASCO Molecular Oncology Task Force, the ASCO Board of Directors, the FDA Oncology Drugs Advisory Committee, and the National Cancer Institute (NCI) Cancer Centers Study Section. She serves on 10 external advisory boards of NCI-designated cancer centers, is a past member of the Board of Directors of the Association of American Cancer Institutes (AACI) and previous Chair of the Cancer Prevention and Research Institute of Texas’ (CPRIT) Clinical Trials Advisory Committee. She is a current member of the National Academies of Science, Engineering, and Medicine’s Cancer Policy Forum. Dr. Eckhardt serves on the Board of Directors of Syros Pharmaceuticals and Exelixis and the Scientific Advisory Board of Amgen.

Lillian L Siu, M.D., FRCPC, is an internationally recognized expert in the development of novel cancer therapeutics. Dr. Siu is a senior medical oncologist at the Princess Margaret Cancer Centre and a Professor of Medicine at the University of Toronto. She is the Director of the Phase I Program and Co-Director of the Bras and Family Drug Development Program at the Princess Margaret Cancer Centre, and holds the BMO Chair in Precision Genomics. Dr. Siu served on the Board of Directors for the American Society of Clinical Oncology (ASCO) and the American Association for Cancer Research (AACR). Internationally, Dr. Siu was the recipient of the US NCI Michaele C. Christian Award in Oncology Drug Development in 2010. She was awarded the TAT 2020 Honorary Award for contributions in the development of anticancer drugs. In 2023, Dr. Siu is currently the co-Editor-in-Chief for AACR’s newest journal Cancer Research Communications and is on the editorial board for Cell and Cancer Cell.

Patricia LoRusso, has been a practicing academic medical oncologist performing clinical/translational research in early phase clinical trials for over 30 years, and is currently the Associate Director of Experimental Therapeutics at the Yale Cancer Center and director of their Early Phase Clinical Trials Program. She has had continuous NIH/NCI peer review funding for over 28 years, as well as numerous other team science grants, including SPORE funding and other funding mechanisms such as Stand Up to Cancer (Co-Leader: Melanoma Dream Team), the Department of Defense (DOD) and the Komen Foundation (Co-leader, KG111063:Targeting Stem Cells in Triple-Negative Breast Cancer (TNBC) in Different Racial Populations). In addition to serving in NCI extramural positions, Dr. LoRusso is currently serving a 3-year term as the chair of their New Agents Committee (NAC), committees for the Cancer Research United Kingdom (CRUK). Dr. LoRusso has also served in leadership positions of several other organizations, including the Board of Directors and current President-elect of the AACR, and education and scientific committees of the ASCO. Internationally, she has taught several clinical trials educational workshops, educating many physicians and scientists across the globe. Working closely over the past 3 decades with patients suffering from advanced malignancies, Dr. LoRusso has become an advocate, not only for cancer researchers and clinicians, but more importantly for the patients and their caregivers.

Tak Wah Mak, Ph.D., co-founder of Treadwell Therapeutics, is one of the world’s most cited and accomplished scientists. Dr. Mak is currently a Senior Scientist at Princess Margaret Cancer Centre, Director of the Campbell Family Institute for Breast Cancer Research and Professor, Department of Medical Biophysics at the University of Toronto. Dr. Mak is credited with the discovery of the T-cell receptor and pioneering work in the genetics of immunology, including publishing a landmark paper on the discovery and function of the immune checkpoint protein CTLA-4. Dr. Mak is also the founder of Agios Pharmaceuticals, whose lead compound, IDHIFA®, was approved by the FDA for acute myelogenous leukemia in 2017. Dr. Mak has served as author on over 1000 peer-reviewed articles. Dr. Mak is a Fellow of the AACR Academy, has received honorary degrees from many universities around the world and has received various international awards such as the Paul Ehrlich Prize, the Ludwig Darmstaeder Prize and most recently the 2023 International Award for Extraordinary Achievement in Cancer Research from the Pezcoller Foundation and the American Association for Cancer Research (AACR).

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 www.treadwelltx.com.

Contact

ir@treadwelltx.com