Treadwell Announces Two Presentations at the 2020 ASH Annual Meeting Featuring Clinical Trial Updates on Lead Product Candidate, CFI-400945

NEW YORK–(BUSINESS WIRE)–Treadwell Therapeutics, a clinical-stage biotechnology company developing novel, small molecule therapeutics for highly aggressive cancers, today announced two presentations for the Company’s CFI-400945 program, an oral, first-in-class inhibitor of Polo-like Kinase 4 (PLK4) a critical regulator of mitotic progression, at the 62^nd American Society of Hematology (ASH) Annual Meeting and Exposition being held virtually from December 5-8, 2020. The first presentation described the efficacy results from an investigator-initiated Phase 1 dose escalation study in AML/MDS. The second was a “Trials in Progress” presentation on the upcoming Treadwell sponsored study in relapsed or refractory AML, MDS or CMML with study initiation planned for the first quarter of 2021.

“We are very encouraged by the promising preliminary Phase 1 study results which included single agent, durable remissions. This compelling early data supports the continued investigation of CFI-400945 for the potential treatment of patients with acute myeloid leukemia (AML),” said Principal Investigator, Karen W.L. Yee, Leukemia Program, Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University Health Network, Toronto, Ontario, Canada.

“Abnormal genetics, complex karyotypes, genomic instability characterize poor prognosis in AML. CFI-400945 inhibits PLK4, a highly conserved master regulator of centriole duplication, and is critical for maintenance of genomic integrity, making it an ideal candidate for the potential treatment of AML,” said Dr. Mark R. Bray, Chief Scientific Officer at Treadwell Therapeutics. “We are excited by the compelling Phase 1 trial results in AML and look forward to continuing to evaluate the promise of our lead candidate in company sponsored Phase 2 studies.”

2020 ASH Poster Presentations and Details:

• Preliminary Results from a Phase 1 Study of CFI-400945, a PLK4 Inhibitor, in Patients with Acute Myeloid Leukemia and High Risk MDS
  
  Publication Number: 1050
  
  Session: 616; Poster I
  
  Date and Time: December 5, 2020, 7:00 AM-3:30 PM

• A Phase 2 Open-Label, Multicenter, Dose Optimization Clinical Study of the Safety, Tolerability, Pharmacokinetic (PK) and Pharmacodynamic (PD) Profiles of CFI-400945 as a Single Agent or in Combination with Azacitidine or Decitabine in Patients with Acute Myeloid Leukemia, Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
  
  Publication Number: 1974
  
  Session: 616; Poster II
  
  Date and Time: Sunday, December 6 ^th, 7:00 AM-3:30 PM

In the presentation titled, “Preliminary Results from a Phase 1 Study of CFI-400945, a PLK4 Inhibitor, in Patients with Acute Myeloid Leukemia and High Risk MDS,” CFI-400945 demonstrated promising activity as a monotherapy in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) and a tolerable safety profile in an open-label, dose escalation, safety and pharmacokinetic study. Data showed that of six patients evaluable for response, two (33%) achieved complete remission (CR) as best response with 3 patients achieving stable disease (SD). (Read more…) Onset of response tended to be within 1 or 2 cycles. One CR occurred in a complex karyotype patient with a p53 mutation who previously failed 7+3 induction and had a durability of ~90 days. The second CR was in a previously untreated AML patient with a 5q deletion who is still in response after >200 days. One subject achieving SD as best response with duration of > 200 days was a previously untreated AML patient with a p53 mutation and had blast reduction from 38% to 9%, but complete blood counts remained below PR criteria. The most common non-hematological drug related toxicities of any grade, which occurred in over 20%, were diarrhea (44%), headache (44%), colitis (33%), vomiting (33%), bilirubin increase (22%), dizziness (22%), fatigue (22%), and nausea (22%).

A second presentation titled, “A Phase 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 or Decitabine in Patients with Acute Myeloid Leukemia,” details the study design for a Treadwell sponsored trial examining CFI-400945 in AML, MDS and chronic myelomonocytic leukemia (CMML). The study will use a standard 3 + 3 design and have four parts: Part 1A (1A), a single agent dose optimization lead-in, Part 1B (1B), a food effect portion once the MTD of 1A is determined, and Part 2, combinations with azacitidine (2A), and decitabine (2B). The efficacy endpoints for AML, MDS, and CMML include the overall response rate, and the CR rate per standard criteria.

About CFI-400945

CFI-400945 is a potent, selective, orally administered, first-in-class inhibitor of the serine/threonine kinase, polo-like kinase 4 (PLK4). PLK4 is a highly conserved master regulator of centriole duplication and is critical for 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 breast 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, with a company sponsored trial in relapsed or refractory leukemia to commence in the first quarter of 2021.

About Treadwell Therapeutics

Treadwell Therapeutics is a clinical-stage oncology company exploiting cancer cells’ vulnerabilities to develop first-in-class and best-in-class small molecules to address unmet needs in patients with cancer.

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

Contacts

Investors:
Christina Tartaglia

Stern Investor Relations, Inc.

212.362.1200

[email protected]