Iterion Therapeutics Announces Initiation of Phase 1 Clinical Trial to Study Tegavivint in Acute Myeloid Leukemia

HOUSTON, Oct. 19, 2021 /PRNewswire/ -- Iterion Therapeutics, Inc., a venture-backed, clinical-stage biotechnology company developing novel cancer therapeutics, today announced the initiation of a Phase 1 clinical trial to investigate tegavivint as a potential treatment for acute myeloid leukemia (AML). This investigator-initiated trial is being led by Tapan M. Kadia, M.D., from the Department of Leukemia at The University of TexasMD Anderson Cancer Center.

Tegavivint is a potent and selective first-in-class inhibitor of Transducin βeta-like Protein One (TBL1), a novel downstream target in the Wnt/beta-catenin signaling pathway. Binding of TBL1 to beta-catenin in the nucleus is necessary for activation of beta-catenin-dependent gene transcription. Tegavivint's targeting of TBL1 prevents the TBL1/beta-catenin complex from forming and specifically inhibits beta-catenin's oncogenic activity without disrupting key cell membrane functions that have been linked to toxicity common to other drugs in this pathway.

"We are very excited about this trial to research tegavivint as a potential new treatment for AML, the most common type of leukemia in adults," stated Rahul Aras, Ph.D., CEO of Iterion Therapeutics. "Having recently established the drug's safety and clinical activity in a proof-of-concept Phase 1 study in desmoid tumors, we are expanding our clinical footprint to address additional cancers characterized by beta-catenin overexpression such as AML, NSCLC and certain pediatric cancers. The initiation of the AML clinical trial represents an important advance in this growth strategy."

Aberrant up-regulation of beta-catenin has been documented as essential for self-renewal, growth and survival of AML stem and blast progenitor cells. In AML preclinical models, tegavivint has shown single agent and combination efficacy with chemotherapy and targeted agents, without affecting normal hematopoietic stem cells. The Phase 1 trial is designed as a two-part, open-label, dose-escalation study to determine the maximum tolerated dose (MTD) and dose limiting toxicities (DLT) of tegavivint, as a monotherapy and in combination with decitabine, in patients with relapsed and refractory AML. More information on the clinical trial is available at https://clinicaltrials.gov/ct2/show/NCT04874480?term=Tegavivint&draw=2&rank=2.

"We welcome the opportunity to initiate this clinical trial of tegavivint, which targets TBL1, a novel therapeutic target in the Wnt-signaling pathway that has been implicated by several groups in the progression of AML," said Casey Cunningham, M.D., Chief Medical Officer of Iterion Therapeutics.  "This trial builds upon prior published research from MD Anderson led by Dr. Kapil N. Bhalla that defined the importance of the TBL1/beta-catenin biology in AML and the therapeutic potential for tegavivint in this patient population."

About Iterion Therapeutics
Iterion Therapeutics is a venture-backed, clinical stage biotechnology company developing novel cancer therapeutics. The company's lead product, tegavivint, is a potent and selective small molecule that binds to TBL1 in the nucleus inhibiting nuclear beta-catenin signaling and oncogenic activity. Research demonstrating potent anti-tumor activity in a broad range of pre-clinical models indicate that tegavivint has the potential for clinical utility in multiple cancer types. Tegavivint is currently the subject of a Phase 1/2a clinical trial in patients with progressive desmoid tumors and a Phase 1 clinical trial in patients with relapsed or refractory acute myeloid leukemia (AML). Iterion is also pursuing clinical programs in additional cancers where nuclear beta-catenin signaling has been shown to play a role, such as NSCLC and pediatric cancers, including sarcomas, lymphoma and other solid tumors. Iterion is the recipient of an up to $15.9 million Product Development Award from the Cancer Prevention and Research Institute of Texas (CPRIT).  For more information on Iterion, please visit https://iteriontherapeutics.com.

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