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Ambit Biosciences Corporation Announces Second Quarter 2013 Operational Results

   Ambit Biosciences Corporation Announces Second Quarter 2013 Operational
                                   Results

- Company to Host Conference Call and Webcast at 5:00 p.m. EDT, Today, August
13, 2013

PR Newswire

SAN DIEGO, Aug. 13, 2013

SAN DIEGO, Aug. 13, 2013 /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 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

  oRaised net proceeds of approximately $83 million after completion of an
    initial public offering of common stock, including $25 million in a
    concurrent private placement with existing investors
  oPresented results from the Phase 2 ACE study of quizartinib in patients
    with relapsed or refractory AML at the annual meeting of the American
    Society of Clinical Oncology (ASCO) and at the 18th Congress of the
    European Hematology Association
  oCompleted top line analyses from the Phase 2b clinical trial of
    quizartinib
  oHeld a Type C meeting with the FDA in June. FDA requested additional
    information and analyses be presented at end of Phase 2 (EOP2) meeting 
  oSubmitted four abstracts to the American Society of Hematology (ASH) for
    the annual meeting in December 2013

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

The Company expects the following events to occur during the second half of
2013 and the first half of 2014:

  oNov. 2013: Participation in an EOP2 meeting with the FDA to determine the
    regulatory path of quizartinib for relapsed and refractory AML as
    described above
  oDec. 2013: Presentation at ASH of the full Phase 2b data set from the 76
    patients with relapsed or refractory FLT3-ITD (+) AML treated at doses of
    30mg and 60mg of quizartinib, subject to acceptance of the abstract for
    presentation
  oDec. 2013: Presentation at ASH of (i) the Phase 1 study with quizartinib
    in combination with chemotherapy in younger newly diagnosed AML patient,
    (ii) an investigator sponsored study (AML-18 pilot) which combined
    quizartinib with chemotherapy in older newly diagnosed patients with AML
    and (iii) the TACL pediatric trial, each subject to acceptance of
    abstracts for presentation
  oEarly 2014: Initiation of the Phase 3 study of quizartinib in FLT3-ITD (+)
    relapsed or refractory AML patients
  o2Q14: Submission of an IND for AC708, a CSF1R inhibitor with potential
    utility in oncology and inflammatory diseases

Financial Results

Three months ended June 30, 2013 compared to quarter ended June 30, 2012

Revenues were $11.5 million and $5.2 million for the quarters ended June 30,
2013 and 2012, respectively. The increase of $6.4 million was primarily due
to an increase in license fee amortization related to the Company's
collaboration with Astellas Pharma. In March 2013, the Company received a
notice of termination of the agreement from Astellas, which termination will
be effective September 2013. Research and development expenses were $6.7
million and $10.8 million for the quarters ended June 30, 2013 and 2012,
respectively. The decrease of $4.1 million was primarily due to lower
quizartinib research and development expenses, resulting from a reduction in
the number of patients being treated and followed in the Company's Phase 2
clinical trial as the trial reached its completion. General and
administrative expenses were $2.2 million and $1.6 million for the quarters
ended June 30, 2013 and 2012, respectively. The increase of $644,000 was
primarily due to increased costs related to patent prosecution and increased
stock-based compensation expense.

Other income (net) was $2.5 million for the three months ended June 30, 2013,
and other expense (net) was $672,000 for the three months ended June 30,
2012. The increase of $3.1 million was primarily due to the change in fair
value of the Company's stock warrant liabilities, which is based on the change
in fair value of the underlying preferred or common securities.

Six months ended June 30, 2013 compared to six months ended June 30, 2012

Revenues were $18.1 million and $10.4 million for the six months ended June
30, 2013 and 2012, respectively. The increase of $7.7 million was primarily
due to an increase in license fee amortization related to the Company's
collaboration with Astellas Pharma. In March 2013, the Company received a
notice of termination of the agreement from Astellas, which termination will
be effective September 2013. Research and development expenses were $15.7
million and $22.0 million for the six months ended June 30, 2013 and 2012,
respectively. The decrease of $6.3 million was primarily due to lower
quizartinib research and development expenses, resulting from a reduction in
the number of patients being treated and followed in the Company's Phase 2
clinical trial as the trial reached its completion. General and
administrative expenses were $4.0 million and $3.3 million for the six months
ended June 30, 2013 and 2012, respectively. The increase of $670,000 was
primarily due to increased costs related to patent prosecution and increased
stock-based compensation expense.

At June 30, 2013, the Company had cash and equivalents of $85.3 million,
compared to $17.5 million at December 31, 2012. The increase of $67.8 million
was primarily due to the completion of an initial public offering that raised
net proceeds of $58.1 million and to the completion of a concurrent private
offering that raised net proceeds of $25.1 million, less cash used to fund
operations.

Financial Guidance

The Company expects its research and development expense to increase as it
prepares for the initiation of the Phase 3 clinical trial of quizartinib in
relapsed or refractory AML patients, which is expected to begin early in
2014. Under the base case operating plan, which assumes that the FDA will
require a randomized, controlled Phase 3 clinical trial for an NDA submission,
the Company expects cash and cash equivalents to be sufficient to fund this
trial and current operations through the receipt of top line data from the
Company's Phase 3 trial in relapsed/refractory AML.

Conference Call and Webcast

A conference call hosted by the Ambit management team will be webcast live
today or may be accessed by dialing 866-436-9172 for domestic callers and
630-691-2760 for international callers. Please specify to the operator that
you would like to join the "Ambit Second Quarter 2013 Financial Results Call,
conference ID#:35424048." If you are unable to listen to the live webcast, a
teleconference replay will be available through Monday, August 19, 2013.
Interested parties can access the replay by dialing 888-843-7419 or
630-652-3042 internationally and entering the conference number 35424048.

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 Friday, September 13,
2013. Please visitAmbit's website several minutes prior to the start of the
broadcast to ensure adequate time for any software download that may be
necessary.

 Ambit Biosciences Corporation
Selected Financial Information



Condensed Consolidated Statements of Operations
(unaudited, in thousands)
                               ThreeMonths         SixMonths

                               EndedJune30,      EndedJune30,
                               2013      2012       2013       2012
Revenues                       $ 11,547  $ 5,174    $ 18,139   $ 10,407
Operating expenses:
Research and development       6,664     10,811     15,669     21,951
General and administrative     2,197     1,553      3,973      3,303
Gain on sale of kinase

profiling services         —         (565)      —          (1,120)

business
Total operating expenses       8,861     11,799     19,642     24,134
Income (loss) from operations  2,686     (6,625)    (1,503)    (13,727)
Other income (expense)         2,474     (672)      (1,639)    (1,576)
Consolidated net income
                               $ 5,160   $ (7,297)  $ (3,142)  $ (15,303)
(loss)



Condensed Consolidated Balance Sheets
(in thousands)
                                            June30,     December31,

                                            2013        2012
                                            (unaudited)
Cash, cash equivalents                      $   85,285   $   17,481
Other assets                                998          1,948
Property and equipment, net                 799          560
Total assets                                $   87,082   $   19,989
Accounts payable, accrued expenses and
                                            $   8,904    $   12,923
other liabilities
Deferred revenue                            6,947        20,671
Warrant liabilities                         6,965        10,540
Redeemable non-controlling interest         —            3,323
Convertible preferred stock                 —            170,778
Stockholders' equity (deficit)              64,266       (198,246)
Total liabilities and stockholders' equity  $   87,082   $   19,989

About Ambit
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 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 and Ambit's
other drug candidates, the progress and expected timing of clinical trials,
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 financial guidance.. 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," "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.

Contacts:
Marcy Graham (Investors)
Ambit Biosciences
(858) 334-2125
mgraham@ambitbio.com

Ian Stone or David Schull (Media)
Russo Partners
(619) 308-6541
(212) 845-4271
ian.stone@russopartnersllc.com
david.schull@russopartnersllc.com

SOURCE Ambit Biosciences Corporation

Website: http://www.ambitbio.com