Ambit Biosciences AMBI, a
biopharmaceutical company focused on discovery and development of drugs
targeting unmet needs in oncology, autoimmune and inflammatory disease,
today announced a regulatory update after receiving feedback from the U.S.
Food and Drug Administration (FDA) on the development of quizartinib
(AC220), the Company's lead drug candidate, for the treatment of
relapsed/refractory acute myeloid leukemia (AML).
Based on feedback from the FDA, the Company continues to move forward with
its baseline plan to conduct a randomized, controlled Phase 3 clinical trial
in relapsed/refractory FLT3-ITD positive AML patients with an overall
survival endpoint as the basis for the potential initial approval of
quizartinib. The Company is continuing to work with the FDA to refine the
appropriate starting dose for the Phase 3 trial.
The Company has been in dialogue with the FDA related to acceptance of a
novel surrogate endpoint that could support accelerated approval based upon
the results of the Company's Phase 2 and Phase 2b trials. The FDA informed
the Company it does not agree that complete remission with incomplete
hematologic recovery (CRi) represents a surrogate endpoint reasonably likely
to predict clinical benefit. Based on the FDA's current position, the
Company does not plan at this time to submit a NDA for accelerated approval
of quizartinib based on the Phase 2 and 2b clinical trial data.
"We remain committed to moving quizartinib through its late stage
development as expeditiously as possible, given the limited treatment
options for FLT3 ITD positive AML patients who have relapsed or who are
refractory to prior treatments. We will continue to work with the clinical
community and the FDA to achieve this goal," said Michael A. Martino,
President and CEO of Ambit. "We believe that our data demonstrates the
clinical value and benefit of quizartinib for patients in an area of clear
and high unmet medical need. We look forward to presenting our data at ASH
next week and anticipate a high level of clinical enthusiasm."
Data from quizartinib studies will be presented at the ASH 55(th) Annual
Meeting at the Ernest Morial Convention Center in New Orleans, LA on
December 9, 2013.
Oral Presentations:
Results of Phase 2 Randomized, Open-Label, Study of Lower Doses of
Quizartinib in Subjects with FLT3-ITD Positive Relapsed Acute Myeloid
Leukemia (AML): The purpose of this study is to assess the efficacy and
safety of lower doses of quizartinib in the treatment of patients 18-years
or older with relapsed/refractory FLT3 ITD positive AML to further improve
the benefit:risk assessment of quizartinib. The data will be presented by
Dr. Jorge Cortes at 3:00 pm CT on Monday, December 9, in the La Nouvelle
Ballroom C. Quizartinib Can Be Safely Combined With Conventional
Chemotherapy In Older Patients With Newly Diagnosed Acute Myeloid Leukemia:
Experience From The AML Pilot Trial: This is the first presentation of
quizartinib use in combination with chemotherapy in newly diagnosed older
AML patients, testing the feasibility and dose which could be given
sequentially following conventional chemotherapy in patients over the age of
60 years. The data will be presented by Dr. Alan Burnett at 5:15 pm CT on
Monday, December 9, in the La Nouvelle Ballroom C. Results of Phase 1 Study
of Quizartinib In Combination with Induction and Consolidation Chemotherapy
in Younger Patients with Newly Diagnosed Acute Myeloid Leukemia: This dose
escalation study is the first to report data on quizartinib in combination
with standard induction and consolidation chemotherapy in patients between
the ages of 18 to 60-years with newly diagnosed AML, regardless of FLT3
status. The data will be presented by Dr. Jessica Altman at 5:30 pm CT on
Monday, December 9, in the La Nouvelle Ballroom C. A Phase I Study of
Quizartinib in Combination with Cytarabine and Etoposide in
Relapsed/Refractory Childhood ALL and AML: A Therapeutic Advances in
Childhood Leukemia & Lymphoma Study: This was the first clinical trial using
quizartinib in children between the ages of 1 month and 21-years with
relapsed/refractory AML or MLL-rearranged ALL. The data will be presented by
Dr. Todd Cooper at 5:45 pm CT on Monday, December 9, in the La Nouvelle
Ballroom C.
Poster presentations from the investigators from The Sidney Kimmel
Comprehensive Cancer Center, Johns Hopkins Hospital:
Activities Of Commonly Used Tyrosine Kinase Inhibitors Against Wild Type
c-KIT And Potential Impact On Normal Hematopoiesis. Session Name: Molecular
Pharmacology, Drug Resistance: Poster III. Presentation on Monday, December
9, 2013 from 6:00 - 8:00 pm in Hall E. The Combination Of FLT3 Inhibition
And Hypomethylation Confers Synergistic Antileukemic Effects On FLT3-ITD
Positive AML Cell Lines And Primary Cells: Session Name: Acute Myeloid
Leukemia: Therapy, excluding Transplantation: Poster III. Presentation on
Monday, December 9, 2013 from 6:00 - 8:00 pm in Hall E.
The abstracts can be accessed on the ASH website at http://hematology.org.
Conference Call and Webcast
A conference call to discuss the FDA response will be hosted by the Ambit
management team webcast live tomorrow morning, Wednesday, December 4, 2013,
at 9:00 am ET or 6:00 am PT and can be accessed by dialing 877-280-4954.
Please specify that you would like to join the "Ambit Biosciences FDA Update
Conference Call" and reference conference passcode 75822572. If you are
unable to listen to the live webcast, a teleconference replay will be
available through Wednesday, December 11, 2013. Interested parties can
access the replay by dialing 888-286-8010 and entering the conference
passcode 13352784.
The conference call webcast is accessible through the "Investors & Media"
section of the Ambit website at http://www.ambitbio.com. An online replay
will be available following the initial broadcast until Thursday, December
19, 2013. Please visit Ambit's website several minutes prior to the start
of the broadcast to ensure adequate time for any software download that may
be necessary.
About Ambit Biosciences
Ambit is a biopharmaceutical company focused on the discovery, development
and commercialization of drugs to treat unmet medical needs in oncology,
autoimmune and inflammatory diseases by inhibiting kinases that are
important drivers for those diseases. Ambit's lead drug candidate,
quizartinib (AC220), is a once-daily, orally-administered potent and
selective, inhibitor of FMS-like tyrosine kinase-3 (FLT3) and is currently
under clinical development in patients with relapsed/refractory AML and in
newly diagnosed AML patients in combination with chemotherapy as well as
maintenance following a hematopoietic stem cell transplantation (HSCT). In
addition to quizartinib, Ambit's clinical pipeline includes AC410, an oral
JAK2 inhibitor, and CEP-32496, a BRAF inhibitor licensed to Teva
Pharmaceutical Industries Ltd. Ambit's preclinical portfolio includes a
proprietary CSF1R inhibitor program.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not
historical facts are "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995, including statements
associated with Ambit's expectations regarding future development and
therapeutic potential of quizartinib and Ambit's other drug candidates,
progress and timing of Ambit's drug development programs, including the
submission of regulatory filings and planned discussions with regulatory
bodies, plans regarding future clinical trials of quizartinib, including the
starting dose for the Phase 3 clinical trial, and Ambit's other drug
candidates, the progress and expected timing of clinical trials including
the timing of initiation of the Phase 3 clinical trial, the presentation of
data from clinical trials, the clinical benefits to be derived from
quizartinib and Ambit's other drug candidates and the regulatory approval
path for quizartinib. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those expressed or
implied by such forward-looking statements. Words such as "plans,"
"expects," "anticipates," "hopes", "may," "will," "goal," "potential" and
similar expressions are intended to identify forward-looking statements.
These forward-looking statements are based upon Ambit's current expectations
and involve assumptions that may never materialize or may prove to be
incorrect. Actual results and the timing of events could differ materially
from those anticipated in such forward-looking statements as a result of
various risks and uncertainties, which include, without limitation, risks
associated with the process of discovering, developing and commercializing
drugs that are safe and effective for use as human therapeutics, and in the
endeavor of building a business around such drugs. These and other risks
concerning Ambit's programs are described in additional detail in Ambit's
SEC filings. All forward-looking statements contained in this press release
speak only as of the date on which they were made. Ambit undertakes no
obligation to update such statements to reflect events that occur or
circumstances that exist after the date on which they were made.
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