Acute promyelocytic leukemia (APL) is a subtype of acute myeloid leukemia (AML).
The SPA is an agreement between Innovive and the FDA on the pivotal Phase II study of Tamibarotene, including the study design, the endpoints of the study, and the data analysis.
The SPA is intended to provide assurance that if pre-specified trial results are achieved, they may serve as the primary basis for an efficacy claim in support of a new drug application (NDA) by the company for Tamibarotene.
The study will be known as STAR-1 (a Phase II study of oral Tamibarotene in acute promyelocytic leukemia patients who have received prior therapy with ATRA and arsenic trioxide).
Under the SPA, STAR-1 will be an open-label, non-randomized, single-arm, multi-national study that will enroll 50 adult patients with relapsed or refractory APL following treatment with all-trans-retinoic acid (ATRA) and arsenic trioxide. Tamibarotene will be self-administered orally via tablets on an outpatient basis at a dose of 6 mg/m2 per day.
The primary objective of the study is to determine the rate of durable complete response for tamibarotene therapy when administered as a single agent to adult patients with relapsed or refractory APL. Secondary objectives are to determine the rates of morphologic leukemia-free state, partial response, cytogenetic complete response, and molecular complete response and to determine the safety profile, tolerability, and pharmacokinetic profile of tamibarotene in the indicated patient population.
“We will continue to work closely with the FDA throughout the initiation and duration of this pivotal trial in anticipation of filing an NDA for tamibarotene soon after completion of the trial,” said Steven Kelly, president and CEO of Innovive Pharmaceuticals.
Innovive acquired the North American rights to develop tamibarotene from TMRC, the company that markets the drug in Japan, where it is approved for the treatment of refractory APL.