Ambit Biosciences Announces Fourth Quarter And Full-Year 2013 Operating Results

   Ambit Biosciences Announces Fourth Quarter And Full-Year 2013 Operating
                                   Results

Quizartinib Phase 3 Trial Expected to Begin in Second Quarter 2014

PR Newswire

SAN DIEGO, March 20, 2014

SAN DIEGO, March 20, 2014 /PRNewswire/ --Ambit Biosciences (NASDAQ: AMBI), a
biopharmaceutical company focused on discovery and development of drugs
targeting unmet needs in oncology, autoimmune and inflammatory disease, today
announced fourth quarter and full-year operating results for 2013. The
company will host a conference call today to discuss financial results for the
fourth quarter and full-year 2013, and to provide an outlook on plans for
2014.

"I am very pleased with the progress we made in 2013 across a broad number of
fronts. We completed enrollment in the Phase 2b clinical trial of
quizartinib, and presented these results at ASH. We finalized the Phase 3
protocol and are prepared to initiate enrollment in the second quarter. We
broadened the clinical development of quizartinib through Company and
investigator sponsored studies designed to generate data on the utility of
quizartinib beyond relapsed/refractory FLT3-ITD+ AML," said Michael Martino,
Ambit's President and Chief Executive Officer. "We strengthened the team with
outstanding additions to our board of directors and our operating team
including clinical development, marketing, financial and communications
expertise. Finally, we completed our IPO and provided necessary capital that
positioned the company to move forward with our development plans and our
expanded pipeline."

Operational Update
Phase 3 trial in relapsed/refractory FLT3-ITD+ AML: The Company plans to
initiate a registrational Phase 3 clinical trial in relapsed/refractory acute
myeloid leukemia (AML) patients with the FLT3-ITD mutation in the second
quarter of 2014. The interim analysis is anticipated in the second half of
2015 and top line data in the first quarter of 2016, both of which are event
driven outcomes.

"A Phase 3 trial is a huge undertaking and I am proud of our team and the
commitment they have shown throughout the process of preparing to initiate
this study," continued Martino. "We look forward to the trial's initiation in
the second quarter and their continued focus and dedication will be key
drivers of our success."

This Phase 3 clinical trial will compare quizartinib as monotherapy to one of
three chemotherapy regimens with a 2:1 randomization of quizartinib to
chemotherapy. The trial will be conducted in FLT3-ITD positive patients over
the age of 18 who have relapsed from, or are refractory to, frontline
chemotherapy, including those patients relapsing following a hematopoietic
stem cell transplant (HSCT).

Those patients who proceed to HSCT after quizartinib treatment will have the
option to reinitiate treatment with quizartinib following the transplant.
Quizartinib dosed as a continuous maintenance therapy following a HSCT has the
potential to increase duration of remission and overall survival.The trial
is expected to enroll approximately 326 patients in the United States, Western
Europe, Canada and Australia. The primary endpoint for the Phase 3 clinical
trial will be overall survival.

An interim analysis will be conducted and will include an adaptive design
component that will allow the Data Safety Monitoring Board (DSMB), to
recommend an increase in the number of patients if warranted. Enrollment is
expected to be completed in the second half of 2015 assuming there is no
increase in the number of patients following the interim analysis.

Ongoing Phase 1 post-transplant maintenance trial: A Company sponsored
Phase1 dose-escalating clinical trial is ongoing to evaluate quizartinib as a
maintenance therapy for patients with AML, irrespective of FLT3-ITD status,
who have received a HSCT and are currently in remission. The goals of this
clinical trial are to evaluate the safety and tolerability of quizartinib as a
maintenance therapy and to increase the duration of remission and prolong
overall survival. We expect data from this trial to be presented at a future
medical conference.

Investigator sponsored trials: An investigator-sponsored Phase 1/2 clinical
trial, referred to as the AML-18 study, is ongoing to evaluate quizartinib in
combination with conventional chemotherapy in newly diagnosed AML patients
over the age of 60, regardless of FLT3-ITD status. Preliminary data from this
study was presented at the 2013 Annual Meeting of ASH. A total of 55 patients
were enrolled. Quizartinib was given two days after each of two courses of
ADE (Ara-C/ Daunorubicin/ Etoposide) followed by one course of Daunorubicin
/Ara-C (DA). The study determined a maximum tolerated dose that can be given
sequentially after high-dose chemotherapy in older patients with newly
diagnosed AML. A follow-on investigator-sponsored Phase 3 clinical trial is
planned.

The LI-1 study, an on-going investigator-sponsored Phase 2/3 clinical trial,
is evaluating quizartinib in combination with low dose cytarabine in
previously untreated AML or myelodysplastic syndrome (MDS) patients over the
age of 60 who were not suited for standard induction chemotherapy. A total of
50 patients are planned to be enrolled prior to an analysis that will
determine whether or not the clinical trial will continue with additional
patients.

An investigator-sponsored Phase 1/2 dose escalation clinical trial is
currently underway to evaluate quizartinib in combination with either
5-azacitidine or low dose cytarabine for patients 18 years or older who had
relapsed AML or MDS. Pending completion of Part 1 (dose escalation phase),
the clinical trial is expected to proceed to Part 2, where
previously-untreated FLT3-ITD positive AML patients 60 years or older or
FLT3-ITD positive AML patients 18 years or older in first relapse will be
enrolled. Approximately 50 patients are planned to be treated in this
clinical trial.

In addition to the clinical development program for the treatment of adult
patients, an investigator-sponsored Phase 1 clinical trial has been completed
which evaluated quizartinib in 22 pediatric patients with either relapsed
acute lymphoblastic leukemia (ALL) or relapsed AML. This clinical trial was
sponsored by the Therapeutic Advances in Childhood Leukemia& Lymphoma
Cooperative Group and data was presented at the 2013 Annual Meeting of ASH. A
follow-on Phase 2 clinical trial in pediatric patients is planned.

Pipeline Development: The Company plans to move forward in the development of
its pipeline of proprietary kinase inhibitors in 2014, including AC410 and
AC708. AC410 is a potent, selective, orally-administered, small molecule
inhibitor of JAK2, which has potential utility for the treatment of autoimmune
and inflammatory diseases or as immunotherapy in oncology. AC708 is a potent
and selective small molecule inhibitor of colony stimulating factor 1 receptor
(CSF1R). CSF1R is thought to play a key role in the proliferation of
macrophages, which are involved in oncology, autoimmune and inflammatory
diseases.

Strategic Development: The Company continues to explore strategic
partnerships as a means to accelerate development and maximize the potential
of quizartinib and its other pipeline assets. The goal for quizartinib
remains to retain commercial rights in North America and enter into
collaborative arrangements for commercialization in other markets such as
Europe and Asia.

Fourth Quarter Results
Revenues were $1.3 million and $2.9 million for the quarters ended December
31, 2013 and 2012, respectively. The decrease of $1.7 million was primarily
due to the termination of the Company's collaboration with Astellas Pharma,
which occurred in September 2013. The decrease was partially offset by a $1.0
million clinical milestone from Teva Pharmaceuticals.

Research and development expenses were $6.1 million and $6.6 million for the
quarters ended December 31, 2013 and 2012, respectively. The decrease of
$480,000 was primarily due to lower quizartinib research and development
expenses. This resulted from a reduction in the number of patients being
treated and followed in our Phase 2 clinical trial, in which enrollment was
completed in late 2011. The decrease in expenses related to our Phase 2
clinical trial was partially offset by increased expenses related to our
upcoming Phase 3 clinical trial.

General and administrative expenses were $3.3 million and $2.0 million for the
quarters ended December 31, 2013 and 2012, respectively. The increase of $1.3
million was primarily due to increased costs related to stock-based
compensation expense, personnel and public company-related expenses.

Other income (net) was $6.1 million for the quarter ended December 31, 2013,
while other expenses (net) were ($274,000) for the quarter ended December 31,
2012. The improvement of $6.4 million was primarily due to the change in fair
value of the Company's stock warrant liabilities, which is driven by the
change in fair value of the underlying securities.

Full Year Results
Revenues were $27.1 million and $17.6 million for the year ended December 31,
2013 and 2012, respectively. The increase of $9.5 million was primarily due
to acceleration in the recognition of license fee revenue related to the
Company's collaboration with Astellas Pharma, which terminated effective
September 2013.

Research and development expenses were $26.3 million and $36.7 million for the
year ended December 31, 2013 and 2012, respectively. The decrease of $10.4
million was primarily due to lower quizartinib research and development
expenses. This resulted from a reduction in the number of patients being
treated and followed in our Phase 2 clinical trial, for which enrollment of
patients was completed in late 2011.

The Company expects its research and development expense to increase as it
conducts the Phase 3 clinical trial of quizartinib in relapsed or refractory
AML FLT3-ITD+ patients and in support of continued development of its
pipeline assets.

General and administrative expenses were $10.3 million and $6.6 million for
the full-year ended December 31, 2013 and 2012, respectively. The increase of
$3.8 million was primarily due to increases in stock-based compensation,
personnel-related costs, legal and accounting expenses, insurance costs, and
investor relations costs, the majority of which were due to our becoming a
public company in 2013.

Other expenses (net) were $4.2 million and $3.9 million for the year ended
December 31, 2013 and 2012, respectively.

At December 31, 2013, the Company had cash and equivalents of $71.2 million,
compared to $17.5 million at December 31, 2012. The increase of $53.7 million
was primarily due to the completion of our IPO and concurrent private
placement that raised $83.2 million in net proceeds, less cash used to fund
operations.

Conference Call and Webcast
A conference call hosted by the Ambit management team will be webcast live
today at 5:00 pm EDT (2:00 pm PDT) and will be webcast live on the Ambit
Biosciences website. To access the live teleconference call, dial
866-436-9172 in the U.S. and 630-691-2760 for international callers. Please
specify to the operator that you would like to join the "Ambit Fourth Quarter
and Full-Year 2013 Earnings Conference Call," referencing conference ID number
36742362. If you are unable to listen to the live webcast, a teleconference
replay will be available through Thursday, March 27, 2014. Interested parties
can access the replay by dialing 888-843-7419 and entering the passcode
36742362.

The conference call webcast is accessible through the "Investors & Media"
section of the Ambit Biosciences website at http://www.ambitbio.com. An
online replay will be available following the initial broadcast until
Thursday, April 24, 2014.

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 acute myeloid leukemia (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 expected timing of Ambit's drug development programs and clinical
trials, plans regarding future clinical trials of quizartinib and Ambit's
other drug candidates, the presentation of data from clinical trials, the
clinical benefits to be derived from quizartinib and Ambit's other drug
candidates, the regulatory approval path for quizartinib, the strength of
Ambit's balance sheet and adequacy of cash on hand, and Ambit's intentions
with respect to potential strategic partnerships . 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.

Ambit Contacts:
Marcy Graham                                        Andrew McDonald, Ph.D.
Executive Director, Investor Relations & Corp       LifeSci Advisors, LLC
Comm
Ambit Biosciences Corporation                       Founding Partner
858-334-2125                                        646-597-6987
mgraham@ambitbio.com                                andrew@lifesciadvisors.com



Ambit Biosciences Corporation

Selected Financial Information
Condensed Consolidated Statements of Operations

(in thousands)
                                  Three Months          Year

                                  Ended December 31,    Ended December 31,
                                  2013       2012       2013        2012
                                  (unaudited)
Revenues                          $ 1,276    $ 2,944    $ 27,093    $ 17,633
Operating expenses:
Research and development          6,131      6,611      26,284      36,731
General and administrative        3,293      2,017      10,342      6,550
Gain on sale of kinase profiling  —          (897)      (2,500)     (2,497)
services business
Total operating expenses          9,424      7,731      34,126      40,784
Loss from operations              (8,148)    (4,787)    (7,033)     (23,151)
Other income (expenses), net      6,103      (274)      (4,223)     (3,878)
Consolidated net loss             $ (2,045)  $ (5,061)  $ (11,256)  $ (27,029)



Ambit Biosciences Corporation

Selected Financial Information
Condensed Consolidated Balance Sheets

(in thousands)
                                                    December 31,  December 31,

                                                    2013          2012
Cash and cash equivalents                           $   71,189    $   17,481
Other assets                                        1,974         1,948
Property and equipment, net                         785           560
Total assets                                        $   73,948    $   19,989
Accounts payable, accrued expenses and other        $   6,708     $   12,923
liabilities
Deferred revenue                                    —             20,671
Warrant liabilities                                 9,650         10,540
Redeemable non-controlling interest                 —             3,323
Convertible preferred stock                         —             170,778
Stockholders' equity (deficit)                      57,590        (198,246)
Total liabilities and stockholders' equity          $   73,948    $   19,989

SOURCE Ambit Biosciences

Website: http://www.ambitbio.com
 
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