Portola Pharmaceuticals Reports Third Quarter Financial Results and Provides Corporate Update

Portola Pharmaceuticals Reports Third Quarter Financial Results and Provides Corporate Update

SOUTH SAN FRANCISCO, Calif., Nov. 10, 2014 (GLOBE NEWSWIRE) -- Portola
Pharmaceuticals (Nasdaq:PTLA) today provides a corporate update and
reports its financial results for the quarter ended September 30, 2014.

"The third quarter marked another successful period of execution for
Portola as we achieved a number of milestones across our three
wholly-owned clinical programs. We announced positive Phase 3 study
results with our universal Factor Xa inhibitor antidote, andexanet
alfa, in which all primary and secondary endpoints were achieved with
high statistical significance. We increased enrollment and remain on
track to complete the betrixaban pivotal Phase 3 APEX Study by the end
of 2015. In addition, we have made the decision to advance our oral,
dual Syk-JAK kinase inhibitor, cerdulatinib, to the next stage of
development based on the tolerability and clinical activity seen thus
far in the ongoing Phase 1 study in patients with hematologic cancers.
We are striving to advance the field of thrombosis and blood cancers by
targeting significant areas of unmet medical need and by using
biomarker and genetic development approaches to increase the
probability of success of our programs," said William Lis, chief
executive officer of Portola. "Going forward, we expect a number of
catalysts this year and next that could drive additional stockholder

Recent Achievements

Betrixaban - Potential first-to-market oral Factor Xa inhibitor
anticoagulant for in-hospital and post-discharge blood clot prevention
in acute medically ill patients

  -- Increased patient enrollment to 58 percent in the pivotal Phase 3 APEX
     Study, which now has more than 450 global sites.
  -- Pooled blinded aggregate venous thromboembolism (VTE) event rates in the
     APEX Study remain on target.
  -- The American Heart Journal published a description of the modification to
     the APEX Study primary analysis plan. The study will formally analyze a
     pre-specified biomarker-defined subpopulation that is likely to achieve a
     larger than average response to treatment with betrixaban, while
     protecting the statistical power to achieve significant efficacy in the
     broader study population.
  -- The Independent Data Monitoring Committee (IDMC) held its fourth planned
     safety review and recommended that Portola proceed with the APEX Study as


Andexanet Alfa - Factor Xa inhibitor antidote and FDA-designated
breakthrough therapy

  -- Reported positive topline data from the first part of the Phase 3
     ANNEXA(TM)-Apixaban study, which met its primary and secondary endpoints
     with high statistical significance. Andexanet alfa immediately and
     significantly reversed the anticoagulation activity of apixaban and was
     well tolerated with no serious adverse events reported. The full data
     will be presented in an oral session at the American Heart Association
     (AHA) Annual Scientific Sessions on November 17, 2014.
  -- Reached agreement with the U.S. Food and Drug Administration (FDA) and
     European Medicines Agency that the Phase 4 confirmatory study will be a
     single-arm trial using a historical control.
  -- Executed a supply agreement with Lonza for the commercial manufacturing
     of andexanet alfa to support anticipated global demand.
  -- Positive Phase 2 data on the reversal of edoxaban (Daiichi Sankyo)
     accepted for presentation at the American Society of Hematology (ASH)
     Annual Meeting on December 8, 2014.

Cerdulatinib* - Oral, dual Syk/JAK kinase inhibitor for hematologic

  -- Established that cerdulatinib is an active drug and is well tolerated in
     the Phase 1 dose escalation study in patients with B-cell lymphomas and
     leukemia. The trial is ongoing and the maximum tolerated dose in the
     Phase 1 study has not yet been reached.
  -- Based on the interim data, Portola is advancing cerdulatinib to the next
     stage of clinical development - clinical expansion cohorts that will
     include patients who have failed current therapies or have relapsed due
     to mutations.



  -- Raised approximately $175 million in net proceeds from a public offering
     of shares of common stock.

Anticipated Upcoming Events and Milestones


  -- Complete a futility analysis of the APEX Study in the first quarter of
  -- Conduct an additional planned safety review of the APEX Study by the IDMC
  -- Complete patient enrollment in APEX by the end of 2015 and report data in
     early 2016

Andexanet Alfa

Phase 3 and Regulatory Milestones:

  -- Report full data from the first part of the Phase 3 ANNEXA-Apixaban study
     in an oral presentation at the AHA Annual Scientific Sessions on November
     17, 2014
  -- Report topline data from the first part of the ongoing Phase 3
     ANNEXA-Rivaroxaban study in late 2014
  -- Report data from the second part of the Phase 3 ANNEXA-Apixaban and
     ANNEXA-Rivaroxaban studies in the first half of 2015
  -- Initiate a Phase 4 confirmatory study in late 2014 or early 2015
  -- File a Biologics License Application (BLA) with the FDA for conditional
     approval under an Accelerated Approval pathway at the end of 2015

Phase 2 Milestones:

  -- Present Phase 2 edoxaban data (abstract #4269) at ASH on December 8, 2014
  -- Initiate a Phase 2 proof-of-concept study with betrixaban in 2015



  -- Continue enrolling patients in the Phase 1 portion of the study to reach
     the maximum tolerated dose
  -- Present Phase 1 results in patients with relapsed/refractory B-cell
     malignancies (abstract #3103) at the ASH Annual Meeting on December 7,
  -- Begin enrolling patients with chronic lymphocytic leukemia and
     non-Hodgkin lymphoma in clinical expansion cohorts

Third Quarter Financial Results

Collaboration revenue for the third quarter of 2014 earned under
Portola's collaborations with Bristol-Myers Squibb Company and Pfizer,
Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Lee's
Pharmaceutical was $2.4 million compared with collaboration revenue of
$2.8 million for the third quarter of 2013.

Total operating expenses for the third quarter of 2014 were $38.2
million compared with $22.0 million for the third quarter of 2013.
Total operating expenses for the third quarter of 2014 included
stock-based compensation expense of $2.2 million compared with $1.5
million for the third quarter of 2013. Research and development
expenses were $31.8 million for the third quarter of 2014 compared with
$18.1 million for the third quarter of 2013, as the Company continued
to support its Phase 3 APEX Study of betrixaban, multiple Phase 3
ANNEXA studies and Phase 2 proof-of-concept studies of andexanet alfa,
and its Phase 1/2a clinical study of cerdulatinib. General and
administrative expenses for the third quarter of 2014 were $6.4 million
compared with $3.9 million for the third quarter of 2013, as the
Company increased headcount to support its growth, resulting in higher
headcount-related costs including stock-based compensation expense,
higher legal and professional fees to support general corporate
activities, and ongoing costs associated with being a public company.

Portola reported a net loss of $35.8 million, or $0.86 net loss per
share, for the third quarter of 2014 compared with a net loss of $18.6
million, or $0.53 net loss per share, for the third quarter of 2013.
Shares used to compute net loss per share attributable to common
stockholders were approximately 41.4 million for the third quarter of
2014 compared with approximately 35.2 million for the same period in

As of September 30, 2014, cash, cash equivalents and investments
totaled $262.0 million, which includes payments of $17.8 million
received from collaboration partners during the third quarter, compared
with cash, cash equivalents and investments of $319.0 million as of
December 31, 2013. In October 2014, the company raised approximately
$175 million in net proceeds through a follow-on offering of common
stock that is not reflected in the third quarter cash balance.

Conference Call Details

To access the live conference call today, November 10, 2014, at 4:30
p.m. Eastern Time via phone, please dial (877) 415-3183 from the United
States and Canada or +1 (857) 244-7326 internationally. The participant
passcode is 50361848. Please dial in 10 minutes prior to the start of
the call. To access the live and subsequently archived webcast of the
conference call, go to the Investor Relations section of the Company's
website at: http://investors.portola.com. Please connect to the website
at least 15 minutes prior to the call to allow for any software
download that may be necessary. A replay of the webcast will be
available on the Company's website for 30 days following the live

About Portola Pharmaceuticals, Inc.

Portola Pharmaceuticals is a biopharmaceutical company developing
product candidates that could significantly advance the fields of
thrombosis and other hematologic diseases. The Company is advancing its
three wholly-owned programs using novel biomarker and genetic
approaches that may increase the likelihood of clinical, regulatory and
commercial success of its potentially life-saving therapies. Portola's
partnered program is focused on developing selective Syk inhibitors for
inflammatory conditions.


Portola's wholly-owned, oral, once-daily Factor Xa inhibitor betrixaban
is being evaluated in the only biomarker-based Phase 3 study for
hospital-to-home prophylaxis of venous thromboembolism (VTE) in acute
medically ill patients. Betrixaban's distinct properties may have the
potential to allow the agent to demonstrate efficacy without the
significant increase in the rate of major bleeding that was seen in
this patient population with other Factor Xa inhibitors. If approved,
betrixaban could be the first anticoagulant for both hospital and
post-discharge VTE prophylaxis and the standard of care in this large
market of more than 20 million patients in the G7 countries alone.

Andexanet Alfa

Andexanet alfa, a recombinant modified human Factor Xa molecule, has
the potential to be a first-in-class antidote to reverse the effects of
Factor Xa inhibitors in patients who suffer a major bleeding episode or
who require emergency surgery. Andexanet alfa has been designated as a
breakthrough therapy by the U.S. Food and Drug Administration. Portola
has entered into Phase 3 clinical collaboration agreements with all of
the manufacturers of direct Factor Xa inhibitors - Bristol-Myers Squibb
and Pfizer (Eliquis [apixaban]), Bayer HealthCare and Janssen
Pharmaceuticals (XARELTO(R) [rivaroxaban]), and Daiichi Sankyo
(edoxaban) - while retaining all commercial rights to andexanet alfa.
The Company is currently evaluating andexanet alfa in the Phase 3
ANNEXA(TM) (Andexanet Alfa a Novel Antidote to the Anticoagulant
Effects of fXA Inhibitors) registration studies.

Cerdulatinib* (PRT2070)

Portola's product candidate in the area of hematologic cancer,
cerdulatinib, is an orally available molecule that uniquely inhibits
two validated tumor proliferation pathways - spleen tyrosine kinase
(Syk) and janus kinase (JAK). It is currently being evaluated in a
Phase 1/2a proof-of-concept study in patients with B cell leukemias or
lymphomas with a focus on genetically-defined subtypes, as well as in
patients who have failed therapy due to relapse or acquired mutations.

For more information, visit www.portola.com and follow the Company on
Twitter @Portola_Pharma.

Forward-looking Statement

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.
Because such statements are subject to risks and uncertainties, actual
results may differ materially from those expressed or implied by such
forward-looking statements. Such statements include, but are not
limited to, anticipated enrollment in our APEX Study, our intention to
advance cerdulatinib to the next stage of development, expected
benefits from biomarker or genetic approaches to clinical development,
potential for upcoming events to build stockholder value, and the
timing and occurrence of events described under the section
"Anticipated Upcoming Events and Milestones." Risks that contribute to
the uncertain nature of the forward-looking statements include: we
expect to incur losses for the foreseeable future and will need
additional funds to finance our operations; our operating results
fluctuate significantly; our estimates regarding our ability to
initiate and/or complete our clinical trials and the timing and expense
of these trials may not be accurate; enrollment in our clinical trials
may be delayed; our clinical trials may not demonstrate the efficacy
and safety of our product candidates; we may not be able to manufacture
our product candidates on a commercial scale in a timely or
cost-efficient manner; our estimates regarding expenses and capital
requirements may not be accurate; regulatory developments in the United
States and foreign countries may adversely affect our operations or
prospects; we must obtain and maintain intellectual property protection
for our product candidates; the loss of key scientific or management
personnel could adversely affect our operations; our stock price may be
volatile; and investors in our common stock could incur substantial
losses and securities or industry analyst reports could cause our stock
price to decline. These and other risks and uncertainties are described
more fully in our most recent filings with the Securities and Exchange
Commission, including our Annual Report on Form 10-K and our Quarterly
Report on Form 10-Q for the third quarter of 2014, which we expect to
file on or about November 10, 2014. All forward-looking statements
contained in this press release speak only as of the date on which they
were made. We undertake no obligation to update such statements to
reflect events that occur or circumstances that exist after the date on
which they were made.

*Cerdulatinib is a proposed International Nonproprietary Name (pINN).

CONTACT: Investor Contact:
         Alexandra Santos
         Portola Pharmaceuticals
         [email protected]

         Media Contact:
         Joey Fleury
         [email protected]