eFFECTOR Therapeutics to Collaborate with the Northwestern University Division of Hematology and Oncology on an Investigator-Initiated Phase 1 Dose Escalation Trial Evaluating Tomivosertib in Patients with Acute Myeloid Leukemia

Trial designed to capitalize on previously published results that showed preclinical activity of tomivosertib in AML models


SOLANA BEACH, Calif. and REDWOOD CITY, Calif., Oct. 24, 2023 (GLOBE NEWSWIRE) -- eFFECTOR Therapeutics, Inc. (NASDAQ: EFTR), a leader in the development of selective translation regulator inhibitors (STRIs) for the treatment of cancer, today announced the initiation of dosing in an investigator-initiated Phase 1 dose escalation trial evaluating tomivosertib in patients with relapsed/refractory Acute Myeloid Leukemia (AML). The trial will be conducted at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and chaired by Shira Dinner, M.D., Associate Professor of Medicine (Hematology and Oncology). Leonidas Platanias, M.D., Ph.D., Director, Robert H. Lurie Comprehensive Cancer Center and Professor of Medicine (Hematology and Oncology) and Biochemistry and Molecular Genetics, and Jessica Altman, M.D., Professor of Medicine (Hematology and Oncology) will serve as Co-Chairs of the trial. Tomivosertib is an inhibitor of mitogen-activator protein kinase interacting kinase (MNK) 1 and 2 and blocks phosphorylation of eukaryotic initiation factor 4E (eIF4E).

“AML is one of the most common types of acute leukemia, and unfortunately, development of resistance to existing therapies leads to relapse in too many patients,” said Dr. Platanias. “Additionally, for older and high-risk younger adult patients who cannot tolerate intensive chemotherapy, there are few treatment options. MNK phosphorylation of eIF4E is a critical step in AML and our lab has recently demonstrated that tomivosertib and venetoclax act synergistically to increase AML cell death. Once we’ve identified the appropriate dose for tomivosertib in AML, we hope to expand the trial to test a combination of tomivosertib with venetoclax and azacytidine.”

Doug Warner, M.D., chief medical officer of eFFECTOR Therapeutics added: “We are excited to collaborate with the Northwestern team to further explore tomivosertib in hematologic settings as eFFECTOR continues to evaluate tomivosertib’s potential for treatment of solid tumors in non-small cell lung cancer.”

More details on the investigator-initiated study can be found on ClinicalTrials.Gov (NCT05744739).

About eFFECTOR Therapeutics

eFFECTOR is a clinical-stage biopharmaceutical company pioneering the development of a new class of oncology drugs referred to as STRIs. eFFECTOR’s STRI product candidates target the eIF4F complex and its activating kinase, mitogen-activated protein kinase interacting kinase (MNK). The eIF4F complex is a central node where two of the most frequently mutated signaling pathways in cancer, the PI3K-AKT-mTOR and RAS-MEK-ERK pathways, converge to activate the translation of select mRNA into proteins that are frequent culprits in key disease-driving processes. Each of eFFECTOR’s product candidates is designed to act on a single protein that drives the expression of a network of functionally related proteins, including oncoproteins and immunosuppressive proteins in T cells, that together control tumor growth, survival and immune evasion. eFFECTOR’s lead product candidate, tomivosertib, is a MNK inhibitor currently being evaluated in KICKSTART, a randomized, double-blind, placebo-controlled Phase 2b trial of tomivosertib in combination with pembrolizumab in patients with metastatic non-small cell lung cancer (NSCLC). Zotatifin, eFFECTOR’s inhibitor of eIF4A, is currently being evaluated in Phase 2a expansion cohorts in certain biomarker-positive solid tumors, including ER+ breast cancer and KRAS-mutant NSCLC. eFFECTOR has a global collaboration with Pfizer to develop inhibitors of a third target, eIF4E.

Forward-Looking Statements

eFFECTOR cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to: the
clinical development of tomivosertib and potential benefits of the collaboration and the potential therapeutic benefits of tomivosertib. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; the success of our clinical trials and preclinical studies for our product candidates is uncertain; the inability to realize any benefits from the collaboration; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of our product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; and other risks described in our prior filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

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