Treadwell Announces Initiation of Patient Dosing in TWT-202, a Phase 1b/2 study of PLK4 Inhibitor, CFI-400945, in Patients with Leukemia

Initial data to be presented in late 2021

NEW YORK--(BUSINESS WIRE)--Treadwell Therapeutics, a clinical-stage biotechnology company developing novel, cross-modality medicines for unmet needs in cancer, today announced the initiation of patient dosing in TWT-202, its Phase 1b/2 study to evaluate its CFI-400945, an oral, first-in-class inhibitor of Polo-like kinase 4 (PLK4) in patients with Leukemia as a monotherapy or in combination with hypomethylating agents. Dosing of the first patient in the trial commenced April 16th at the University of Texas MD Anderson Cancer Center, Houston, TX.

“Oral PLK4 inhibition opens a completely new approach to target therapeutic vulnerability in high risk MDS and AML and is very amenable to combination with other effective therapeutic agents,” said Principal Investigator, Dr. Gautam Borthukar, MD, Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center.

“We are excited about the initiation of TWT-202 with our highly potent PLK4 inhibitor,” said Dr. Michael Tusche, Treadwell co-CEO. “Previous clinical studies have shown that CFI-400945 can lead to single agent complete remissions in high-risk refractory AML patients. We look forward to continuing the development of this agent with the goal of delivering first in class medicines to patients in need.”

The Phase 1b/2 clinical trial of CFI-400945, is an open-label, multi-center, dose optimization study designed to assess the safety, tolerability, pharmacokinetic and pharmacodynamic profiles of CFI-400945 as a single agent or in combination with azacytidine or decitabine in patients with acute myeloid leukemia, myelodysplastic syndrome or chronic myelomonocytic leukemia. The trial will enrol approximately 72 patients at up to 20 sites in North America and Asia. It will involve 3 arms including monotherapy and combination dose optimization and expansion, as well as a food effect study.

About CFI-400945

CFI-400945 is a highly potent and selective inhibitor of the serine/threonine kinase PLK4, a cell cycle kinase known to be the master upstream regulatory of centriole duplication and is critical for the maintenance of genomic integrity. PLK4 is overexpressed in a variety of solid tumors and elevated expression is associated with poor clinical outcomes. Depletion of PLK4 expression in cancer cells by RNA interference leads to mitotic defects and cell death. PLK4 was identified as a drug target based on functional screening to identify vulnerabilities of genomically unstable cancers. Anti-tumor activity of CFI-400945 has been shown in mice bearing human cancer xenografts, including robust tumor growth inhibition and durable tumor regression in primary tumor xenografts from breast cancer. CFI-400945 is a potent, selective, orally administered, first-in-class inhibitor of the serine/threonine kinase, polo-like kinase 4 (PLK4). CFI-400945 is currently in multiple investigator-initiated studies in solid and liquid malignancies,

About Treadwell Therapeutics

Treadwell Therapeutics is a science driven, clinical-stage multi-modality oncology company developing first-in-class and best-in-class medicines to address unmet needs in patients with cancer.

The Company’s robust, internally developed pipeline includes a first-in-class PLK4 kinase inhibitor, CFI-400945 and a best-in-class TTK inhibitor, CFI-402257, and CFI-402411, an oral immunomodulatory kinase inhibitor with activity toward HPK1 in Phase 1/2 studies. Treadwell also has a robust pre-clinical pipeline with multiple Biologic and cell therapy programs. For more information, please visit www.treadwelltx.com.