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Tolero Pharmaceuticals Receives FDA Orphan Drug Designation for Alvocidib for the Treatment of Acute Myeloid Leukemia

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April 23, 2014

Tolero Pharmaceuticals, Inc., a clinical-stage company developing treatments for serious hematological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for Alvocidib for the treatment of patients with acute myeloid leukemia (AML). Alvocidib is being tested in patients with intermediate or high-risk AML, which is determined by a profile of clinical, cytogenetic and molecular features, and typically, individuals with this profile have a poor prognosis due to limited treatment options.

“The granting of Orphan Drug Designation by the FDA for Alvocidib in acute myeloid leukemia is a very important milestone in the development program for Alvocidib, and emphasizes our view that this is an important disease area of great unmet medical need,” said David J. Bearss, Ph.D., Tolero’s Chief Executive Officer. “Tolero Pharmaceuticals is pleased with the results that Alvocidib has demonstrated in patients with AML and we are actively pursuing the development of Alvocidib for intermediate and high-risk AML patients who desperately need new options for the treatment of their disease. We look forward to sharing detailed study results of Alvocidib that support further development of the compound at an upcoming medical meeting this year.”

Alvocidib has been evaluated in multiple Phase 2 clinical trials involving approximately 400 patients with both relapsed/refractory or frontline, previously untreated intermediate and high-risk AML. In these trials, Alvocidib has been evaluated as a single agent as well as in combination with approved agents including cytarabine and mitozantrone. Alvocidib is currently being investigated in a Phase 2 study for the treatment of intermediate and high-risk AML in combination with standard of care agents.

Orphan designation by the FDA is granted to promote the development of drugs that target conditions affecting 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments. Orphan designation qualifies a company for benefits that apply across all stages of drug development, including an accelerated approval process, seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.

About Alvocidib

Alvocidib is a potent cyclin-dependent kinase (CDK) small molecule inhibitor in development as a frontline combination therapy for acute myeloid leukemia and relapsed/refractory chronic lymphocytic leukemia and other hematologic disorders. CDKs are regulatory proteins and enzymes that are critical to a cell’s ability to replicate as well as critical regulators of gene expression. Because of the key role CDK deregulation plays in runaway cell division and growth, CDK inhibitors remain an attractive target for the treatment of various cancers.

About Acute Myeloid Leukemia

About 18,000 plus new cases of AML are expected to be diagnosed this year in the United States, according to estimates from the American Cancer Society. AML is a very rapidly growing cancer of the bone marrow and the myeloid lineage of blood cells. It is the most common acute leukemia in adults and accounts for approximately one-third of all adult leukemias in the United States and Europe. Typically, the prognosis of AML patients is categorized as low, intermediate or high-risk based on a profile of clinical, cytogenetic and molecular features which are used to assign risk of a poor outcome for treatment. Intermediate and high-risk AML constitute a significant percentage of all cases of adult AML. For these patients, treatment options are limited and their prognosis is usually poor. Alvocidib is currently being investigated in this specific patient population.

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