Ambit's Oncology Drug in Phase III - Analyst Blog

Ambit Biosciences Corporation AMBI announced that it has commenced a phase III study (QUANTUM-R) on its lead oncology candidate, quizartinib. Quizartinib is a once-daily, orally administered FMS-like tyrosine kinase-3 (FLT3) inhibitor.

The study (n=326) will compare quizartinib as a monotherapy to one of three chemotherapy regimens (2:1 randomization of quizartinib to chemotherapy) in patients (> 18 years) suffering from relapsed or refractory FLT3-ITD positive acute myeloid leukemia (AML), including those whose disease relapsed after hematopoietic stem cell transplantation (HSCT). The primary endpoint of the study is overall survival. Ambit Biosciences expects to complete patient enrolment by the second half of 2015.

Quizartinib has orphan drug status in both the U.S. and the EU for the AML indication.

We remind investors that the U.S. Food and Drug Administration (FDA) denied accelerated approval status to Ambit Biosciences' quizartinib based on results from phase II and phase IIb studies in December last year. The FDA disagreed with the fact that complete remission with incomplete hematologic recovery (CRi) represents a surrogate endpoint that is reasonably likely to predict clinical benefit of quizartinib to AML patients.

Meanwhile, Ambit Biosciences is exploring various strategic partnership options for the development and commercialization of quizartinib in ex-U.S. markets and for the advancement of other pipeline candidates. Quizartinib is also in three investigator-sponsored studies for AML.

One of the currently approved drugs for AML is Celgene Corp's CELG Vidaza. Meanwhile, Sunesis Pharmaceuticals, Inc. SNSS is developing vosaroxin in combination with Dacogen for previously untreated AML and high-risk myelodysplastic syndrome (MDS).

Ambit Biosciences holds a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Alkermes plc ALKS carrying a Zacks Rank #1 (Strong Buy).