Ambit Biosciences Corporation Announces Second Quarter 2013 Operational Results

SAN DIEGO, Aug. 13, 2013 /PRNewswire/ --Ambit Biosciences (AMBI), a biopharmaceutical company focused on discovery and development of drugs targeting unmet needs in oncology, autoimmune and inflammatory disease, today announced results for the second quarter and six months ended June 30, 2013. The company will host a conference call today to provide an operational update and discuss financial results, its first as a public company.

"The second quarter was transformative for Ambit. We successfully completed our initial public offering, which put us on solid financial footing to continue the clinical development of quizartinib, our lead drug candidate," said Michael Martino, president and Chief Executive Officer. "Our primary goal is to make this potentially important new therapy available to patients with acute myeloid leukemia (AML), who are in need of better treatment options."

Second Quarter Highlights and Recent Events

Quizartinib Program Update

The Company has completed preliminary top line analyses of the Phase 2b clinical trial of quizartinib. This trial studied 30mg and 60mg doses administered daily to FLT3 ITD positive patients 18 years and older who were relapsed or refractory to second line treatment or a hematopoietic stem cell transplant (HSCT). Data from the top line efficacy analysis at both 30mg and 60mg are consistent with data from the comparable patient cohort in the Company's Phase 2 trial (n=136) in which there was a 46% CRc rate with 35% of patients bridged to a HSCT. In addition, the top line analysis from the 30mg and 60mg doses in the Phase 2b trial suggests that Grade 3 QTc rate has been reduced with these lower doses as compared to the previously reported Phase 2 results and in line with the preliminary analysis of the first 39 patients in the Phase 2b trial. Other safety findings are consistent with the previously reported Phase 2 results and the preliminary analysis in the Phase 2b trial.

The Company held a Type C meeting with the FDA in June. In this meeting, the Company discussed the potential acceptability of composite complete response (CRc), and specifically complete remission with incomplete hematologic recovery (CRi), as a surrogate endpoint that could support an accelerated approval based on the two Phase 2 clinical trials. Historically the FDA has considered durable complete response (CR) as an acceptable endpoint for accelerated approval. However, the FDA noted at the June Type C meeting that the overall clinical benefit of quizartinib will be a review issue and asked the Company to provide additional data, including further analyses from its Phase 2 and Phase 2b clinical trials, and historical data to support CRi as an endpoint that is reasonably likely to predict clinical benefit.

"We are encouraged by the top line analysis of the data from the Phase 2b study and the recent Type C meeting discussions with the FDA," said Martino. "Our goal for the upcoming EOP2 meeting is to provide the FDA with the requested additional historical data to support CRi as an endpoint that is reasonably likely to predict clinical benefit, the full Phase 2b dataset and additional data analyses of both Phase 2 clinical trials supporting the clinical benefit of CRi. We are working diligently to provide the data requested; however with the additional work we now expect that the EOP2 meeting will take place in November. "

Until the outcome of the EOP2 meeting with the FDA is known, the Company's guidance remains that a randomized, controlled Phase 3 clinical trial of quizartinib with an overall survival endpoint will need to be completed prior to seeking regulatory approval. The Company remains on track to initiate the Phase 3 clinical trial in relapsed and refractory FLT3-ITD (+) AML patients in early 2014.

Fourabstractshave been submitted tothe American Society of Hematology(ASH)for the annual meetingin December 2013,including (1) the complete patient dataset from the Phase 2b study; (2) thepreliminary analyses fromthe Phase 1 study of quizartinib in combination with chemotherapy in younger newly diagnosed AML patients; (3) the complete patient dataset from the Phase 1b/2 investigator sponsoredAML 18 pilot study, which combined quizartinib with chemotherapy in older newly diagnosed AML patients, and (4) final data from the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) Phase 1 pediatric trial evaluating quizartinib in combination with chemotherapy in patients that are 21 or younger with relapsed ALL or AML.

Upcoming Events

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Ambit Biosciences Corporation Announces Second Quarter 2013 Operational Results