Tolero Pharmaceuticals Joins The Leukemia & Lymphoma Society’s Groundbreaking Beat AML Master Clinical Trial for Patients with Acute Myeloid Leukemia

SALT LAKE CITY, Utah, March 26, 2020 /PRNewswire/ -- Tolero Pharmaceuticals, Inc., a clinical-stage company focused on developing novel therapeutics for hematological and oncological diseases, today announced that it has joined The Leukemia & Lymphoma Society (LLS) in the groundbreaking, collaborative Beat AML Master Clinical Trial for newly diagnosed patients 60 years of age or older with acute myeloid leukemia (AML). Tolero’s investigational agent, dubermatinib (TP-0903), an AXL receptor tyrosine kinase (RTK) inhibitor has been selected for a new arm in the trial for patients with TP53 mutations and/or complex karyotype.

AML is one of the deadliest blood cancers and the second most diagnosed type of leukemia in the U.S.¹ Although there have been recent advances in the treatment of AML, for patients with certain types of mutations, prognosis and responsiveness to therapy remains poor.² The Beat AML Master Clinical Trial aims to leverage the expertise of functional genomic technologies and pharmaceutical collaborators, using a personalized medicine approach to accelerate research findings and ultimately improve outcomes for AML patients. Patients with TP53 mutations have few effective treatment options as their response rate to chemotherapy is poor and long-term survival after stem cell transplant is rare.³ The incidence of TP53 mutations in AML has been reported to be between 5 and 19 percent of patients.^4,5,6,7,8

“Tolero Pharmaceuticals is proud to have been selected to join The Leukemia & Lymphoma Society in its efforts to apply a precision medicine approach toward fighting acute myeloid leukemia,” said David J. Bearss, Ph.D., Chief Executive Officer of Tolero Pharmaceuticals, Inc. “The Beat AML Master Clinical Trial provides us a unique opportunity to contribute to the advancement of science in AML and evaluate the potential of dubermatinib in a patient group which has a poor prognosis and limited treatment options.”

In the Phase 1b/2 study, dubermatinib in combination with decitabine will be evaluated in patients 60 years or older with newly diagnosed AML who have TP53 mutations and/or complex karyotype. The primary objectives of the study are to determine the safety and maximum tolerated dose of dubermatinib in combination with decitabine and evaluate the composite complete response rate. Secondary objectives of the study include overall survival and proportion of patients transitioning to allogeneic stem cell transplantation.

“We believe the Beat AML Master Clinical Trial is changing the treatment paradigm for AML as well as for clinical trials more broadly across cancer types,” said Amy Burd, Ph.D., Vice President of Research Strategy, The Leukemia & Lymphoma Society. “Through this trial, we have taken a tailored approach to treatment based on patients’ unique genomic profile, with the ultimate goal of identifying safe and effective targeted therapies for patients who previously had limited options. LLS is pleased that Tolero Pharmaceuticals is joining this unprecedented collaboration of top cancer centers and scientists, several top pharmaceutical companies, the U.S. Food and Drug Administration and a leading-edge genomics company to bring novel targeted therapies to patients faster.”

The trial is being conducted at several leading cancer centers across the United States.

About dubermatinib (TP-0903)

Dubermatinib is an investigational oral AXL receptor tyrosine kinase (RTK) inhibitor under evaluation in a Phase 1/2 study in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (NCT03572634) and a Phase 1a/b study in patients with advanced solid tumors (NCT02729298). Tolero is exploring parallel clinical development paths for dubermatinib in both solid and hematologic malignancies.

About AXL Kinase

AXL belongs to the TAM (Tyro3, AXL and Mer) family of receptor tyrosine kinases and is overexpressed in many human cancers.⁹ It plays a key role in tumor cell proliferation, survival, metastasis, cellular adhesion and avoidance of the immune response. The overexpression of AXL is associated with a poor patient prognosis and drug resistance.¹⁰

About The Leukemia & Lymphoma Society

The Leukemia & Lymphoma Society^® (LLS) is a global leader in the fight against cancer. The LLS mission: cure leukemia, lymphoma, multiple myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.

Founded in 1949 and headquartered in Rye Brook, NY, LLS has chapters throughout the United States and Canada. To learn more, visit www.LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.

About Tolero Pharmaceuticals, Inc.

Tolero Pharmaceuticals is a clinical-stage biopharmaceutical company researching and developing treatments to improve and extend the lives of patients with hematological and oncological diseases. Tolero has a diverse pipeline that targets important biological drivers of blood disorders to treat leukemias, anemia, and solid tumors, as well as targets of drug resistance and transcriptional control.

Tolero Pharmaceuticals is based in the United States and is an indirect, wholly-owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., a pharmaceutical company based in Japan. Tolero works closely with its parent company, Sumitomo Dainippon Pharma, and Boston Biomedical, Inc., also a wholly-owned subsidiary, to advance a pipeline of innovative oncology treatments. The organizations apply their expertise and collaborate to achieve a common objective - expediting the discovery, development and commercialization of novel treatment options.

Additional information about the company and its product pipeline can be found at www.toleropharma.com.

Tolero Pharmaceuticals Forward-Looking Statements

This press release contains “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding the research, development and commercialization of pharmaceutical products. The forward-looking statements in this press release are based on management’s assumptions and beliefs in light of information presently available, and involve both known and unknown risks and uncertainties, which could cause actual outcomes to differ materially from current expectations. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Information concerning pharmaceuticals (including compounds under development) contained within this material is not intended as advertising or medical advice.

References

1. Leukemia – Acute Myeloid – AML: Statistics. Doctor-Approved Patient Information from ASCO website https://www.cancer.net/cancer-types/leukemia-acute-myeloid-aml/introduction. Updated January 2019. Accessed February 2, 2020.
2. Watts J and Nimer S. Recent advances in the understanding and treatment of acute myeloid leukemia. F1000Research. 2018;7:F1000 Faculty Rev-1196.
3. Hunter AM and Sallman DA. Current status and new treatment approaches in TP53 mutated AML. Best Pract Res Clin Haematol. June 2019;32(2):134-144.
4. Falk IJ, Willander K, Chaireti R, et al. TP53 mutations and MDM2(SNP309) identify subgroups of AML patients with impaired outcome. Eur J Haematol. Apr 2015;94(4):355-362.
5. Grossmann V, Schnittger S, Kohlmann A, et al. A novel hierarchical prognostic model of AML solely based on molecular mutations. Blood. Oct 2012;120(15):2963-2972.
6. Seifert H, Mor B, Thiede C, et al. The prognostic impact of 17p (p53) deletion in 2272 adults with acute myeloid leukemia. Leukemia. Apr 2009;23(4):656-663.
7. Kadia T, Jain P, Ravandi F, et al. TP53 mutations in newly diagnosed Acute Myeloid Leukemia –Clinico-molecular characteristics, response to therapy, and outcomes. Cancer. November 2016. 122(22): 3484–3491.
8. Burd A, Levine R, Ruppert A, et al. Precision Medicine Treatment in Older AML: Results of Beat AML Master Trial. Blood (2019) 134 (Supplement_1): 175.
9. Glaser SP, Lee EF, Trounson E, et al. Anti-apoptotic Mcl-1 is essential for the development and sustained growth of acute myeloid leukemia. Genes Dev. 2012;26(2):120-125.
10. Chen R, Keating MJ, Gandhi V, Plunkett W. Transcription inhibition by flavopiridol: mechanism of chronic lymphocytic leukemia cell death. Blood. 2005;106(7):2513-2519.

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SOURCE Tolero Pharmaceuticals, Inc.