Portola Pharmaceuticals Reports First Quarter 2015 Financial Results and Provides Corporate Update
06 Mai 2015 - 10:01PM
Portola Pharmaceuticals (Nasdaq:PTLA) today provided a corporate
update and reported its financial results for the first quarter
ended March 31, 2015.
"This is a monumental year for the company," said William Lis,
chief executive officer of Portola. "We are now approximately one
year from the potential launch of our first commercial product,
andexanet alfa, in 2016 followed by a second product launch with
betrixaban in 2017. Additionally, we are advancing a third
clinical-stage program in hematologic cancers with our dual-acting
agent, cerdulatinib. Each of these products is wholly-owned,
addresses diseases where limited or no treatments exist, and
targets multi-billion dollar hospital or specialty-based
markets."
Recent Achievements
Betrixaban – Potential first-to-market oral Factor Xa inhibitor
anticoagulant for extended venous thromboembolism (VTE) prophylaxis
in acute medically ill patients
- The pivotal Phase 3 APEX Study successfully passed a futility
analysis, and the Independent Data Monitoring Committee (IDMC)
recommended that the study proceed as planned without modification
based on preliminary efficacy trends and safety reports.
- The IDMC held its fifth and final planned safety review and
recommended that the APEX Study proceed as planned.
- APEX Study enrollment is over 80 percent to date.
- Pooled blinded aggregate VTE event rates in the APEX Study
remain on target.
Andexanet Alfa – Factor Xa inhibitor antidote and U.S. Food and
Drug Administration (FDA)-designated breakthrough therapy and
orphan drug on an Accelerated Approval pathway
- Met all primary and secondary endpoints with high statistical
significance in the bolus only part of the Phase 3
ANNEXA™-Rivaroxaban study. Results were presented in an oral
session at the American College of Cardiology's 64th Annual
Scientific Session.
- Achieved all primary and secondary endpoints with high
statistical significance in both the bolus only and the bolus plus
infusion parts of the Phase 3 ANNEXA-Apixaban study. Full results
from the recently completed bolus plus infusion part of the study
will be presented in an oral session at the International Society
on Thrombosis and Haemostasis (ISTH) 2015 Congress in June.
Cerdulatinib – Oral, dual Syk/JAK kinase inhibitor for
hematologic cancers
- Continued to dose-escalate in the Phase 1 part of the Phase
1/2a study.
- Initiated two clinical expansion cohorts in the Phase 2a part
of the ongoing Phase 1/2a study -- one with chronic lymphocytic
leukemia/small lymphocytic leukemia patients and a second with
follicular lymphoma patients.
Corporate
- Raised approximately $109 million in net proceeds in a public
offering of common stock.
- Established a collaboration with Ora, Inc. for the development
of Portola's highly selective Syk inhibitor, PRT2761, in ophthalmic
diseases.
Planned Upcoming Events and Milestones
Betrixaban
- Complete patient enrollment in the APEX Study by the end of
2015
- Report top-line APEX Study data in early 2016
- Submit an New Drug Application (NDA) to the FDA in 2016
Andexanet Alfa
- Present final data from the Phase 3 ANNEXA studies in 2015
- Present new pre-clinical data comparing andexanet alfa and four
factor prothrombin complex concentrates (PCCs) at ISTH
- Initiate a Phase 2 betrixaban study in 2015
- Complete commercial manufacturing process validation with CMC
Biologics to support a Biologics License Application (BLA)
submission and commercial launch
- Scale-up the second-generation commercial manufacturing process
at Lonza to support expected broad worldwide
demand
- Submit a BLA to the FDA under an Accelerated Approval pathway
at the end of 2015
- Continue to enroll patients in the Phase 4 ANNEXA confirmatory
study
Cerdulatinib
- Present updated safety and efficacy data from the Phase 1 part
of the ongoing Phase 1/2a study at the 2015 American Society of
Clinical Oncology's (ASCO) Annual Meeting in Chicago at the end of
May
- Continue to dose-escalate in the Phase 1 part of the Phase
1/2a
- Begin enrolling patients in clinical expansion cohorts
First Quarter Financial Results
Collaboration revenue for the first quarter of 2015 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 for each of the first
quarters of 2015 and 2014.
Total operating expenses for the first quarter of 2015 were
$48.9 million compared with $33.4 million for the same period in
2014. Total operating expenses for the first quarter of 2015
included $5.2 million in stock-based compensation expense compared
with $2.3 million for the first quarter of 2014. Research and
development expenses were $39.9 million for the first quarter of
2015 compared with $28.2 million for the first quarter of 2014 as
the Company continued to support its Phase 3 APEX Study of
betrixaban, its Phase 3 and 4 studies of andexanet alfa, its Phase
1/2a clinical study of cerdulatinib, and its manufacturing scale-up
and build-out in preparation for the commercial launch of andexanet
alfa. Selling, general and administrative expenses for the first
quarter of 2015 were $9.0 million compared with $5.2 million for
the same period in 2014 as the Company increased headcount to
support its growth, resulting in higher headcount-related costs
including stock-based compensation expense and commercial launch
activities.
Portola reported a net loss of $46.9 million, or $0.95 net loss
per share, for the first quarter of 2015 compared with a net loss
of $30.7 million, or $0.75 net loss per share, for the first
quarter of 2014. Shares used to compute net loss per share
attributable to common stockholders were approximately 49.5 million
for the first quarter of 2015 compared with approximately 41.0
million for the same period in 2014.
As of March 31, 2015, cash, cash equivalents and investments
totaled $456.4 million compared with cash, cash equivalents and
investments of $392.3 million as of December 31, 2014.
Conference Call Details
To access the live conference call today, May 6, 2015, at 4:30
p.m. Eastern Time via phone, please dial (877) 415-3182 from the
United States and Canada or +1 (857) 244-7325 internationally, and
use the passcode 41011622. 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 event.
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 programs are focused on developing
selective Syk inhibitors for inflammatory conditions.
Betrixaban
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, an FDA-designated breakthrough therapy and
orphan drug, is a recombinant protein designed to reverse the
anticoagulant effect in patients treated with an oral or injectable
Factor Xa inhibitor. Andexanet alfa has the potential to be a
first-in-class antidote for anticoagulated patients who suffer a
major bleeding episode or require emergency surgery. Portola has
entered into Phase 3 clinical collaboration agreements with all of
the manufacturers of direct Factor Xa inhibitors while retaining
all commercial rights to andexanet alfa. The Company is currently
evaluating andexanet alfa in the Phase 3 and Phase 4 ANNEXA™
(Andexanet Alfa a Novel Antidote to the Anticoagulant Effects of
FXa Inhibitors) registration studies.
Cerdulatinib
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, statements regarding:
the timing and ability to achieve the milestones and events
described under the section "Planned Upcoming Events and
Milestones," our goal of becoming a fully commercialized
biopharmaceutical company, the potential of our three products to
save lives, the occurrence and timing of planned filings with the
FDA and the timing of our reporting of clinical data. Risks that
contribute to the uncertain nature of the forward-looking
statements include: our expectation that we will incur losses for
the foreseeable future and will need additional funds to finance
our operations; the accuracy of our estimates regarding our ability
to initiate and/or complete our clinical trials and the timing and
expense of these trials; the pace of enrollment in our clinical
trials; the results of our clinical trials related to the efficacy
and safety of our product candidates; our potential inability to
manufacture our product candidates on a commercial scale in a
timely or cost-efficient manner; the accuracy of our estimates
regarding expenses and capital requirements; our ability to
successfully build a hospital-based sales force and commercial
infrastructure; regulatory developments in the United States and
foreign countries; our ability to obtain and maintain intellectual
property protection for our product candidates; and our ability to
retain key scientific or management personnel. 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 From 10-Q
for the first quarter of 2015, which we expect to file on or about
May 6, 2015. 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.
|
|
|
PORTOLA
PHARMACEUTICALS, INC. |
|
|
|
Unaudited Condensed
Statements of Operations Data |
(In thousands, except share and
per share data) |
|
|
|
|
Three Months
Ended |
|
March
31, |
|
2015 |
2014 |
Collaboration and license revenue |
$ 2,359 |
$ 2,372 |
Operating expenses: |
|
|
Research and development |
39,858 |
28,155 |
Selling, general and administrative |
9,005 |
5,241 |
Total operating expenses |
48,863 |
33,396 |
Loss from operations |
(46,504) |
(31,024) |
Interest and other income (expense), net |
(409) |
298 |
Net loss |
$ (46,913) |
$ (30,726) |
Net loss per share attributable to common
stockholders: |
|
|
Basic and diluted |
$ (0.95) |
$ (0.75) |
|
|
|
|
|
|
Shares used to compute net loss per share
attributable to common stockholders: |
|
|
Basic and diluted |
49,527,850 |
41,001,623 |
|
|
|
PORTOLA
PHARMACEUTICALS, INC. |
|
|
|
Unaudited Condensed Balance Sheet
Data |
|
|
(In thousands) |
March 31, |
December 31, |
|
2015 |
2014 |
ASSETS |
(Unaudited) |
Cash, cash equivalents and investments |
$ 456,373 |
$ 392,303 |
Total current assets |
403,450 |
315,077 |
Property and equipment, net |
3,211 |
2,776 |
Total assets |
486,299 |
416,495 |
Accounts payable |
5,019 |
14,084 |
Accrued and other liabilities |
24,761 |
13,966 |
Deferred revenue |
34,227 |
36,585 |
Total current liabilities |
40,793 |
41,131 |
Total liabilities |
67,324 |
68,693 |
Total stockholders' equity |
418,975 |
347,802 |
CONTACT: Investor:
Michele Mantynen
Portola Pharmaceuticals
ir@portola.com
Media:
Joey Fleury
BrewLife
jfleury@brewlife.com
415.946.1090
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