Portola Pharmaceuticals (Nasdaq:PTLA) today provided a corporate
update and reported its financial results for the fourth quarter
and year ended December 31, 2014.
"We are off to a terrific start in 2015 as we build on the
momentum we established in 2014. We achieved several important
clinical, regulatory and manufacturing milestones that put us on
track to achieve our goal of launching three potentially
groundbreaking products – our first expected in 2016, our second
planned for 2017 and potentially a third thereafter with
cerdulatinib. Each of these products target multi-billion dollar
hospital or specialty-based markets in thrombosis and hematologic
cancers," said William Lis, chief executive officer of Portola.
"With betrixaban, the pivotal Phase 3 APEX Study successfully
passed its futility analysis, keeping it on track to be the first
potential anticoagulant approved for extended venous
thromboembolism (VTE) prophylaxis in acute medically ill patients.
We also reported additional positive Phase 3 trial results to
support the 2015 Biologics License Application for andexanet alfa,
our universal Factor Xa inhibitor antidote. Finally, for our oral,
dual Syk-JAK kinase inhibitor, cerdulatinib, we established proof
of concept in our ongoing Phase 1 study in patients with
hematologic cancers, and we are advancing to expansion cohorts.
During the remainder of 2015, we will focus on
pre-commercialization efforts for andexanet alfa and betrixaban,
and executing on several value-creating clinical and regulatory
milestones."
Recent Achievements
Betrixaban – Potential first-to-market oral Factor Xa inhibitor
anticoagulant for in-hospital and post-discharge prevention of
venous thromboembolism (VTE) in acute medically ill patients
- The Independent Data Safety Monitoring Committee (IDMC)
completed a futility analysis of the pivotal Phase 3 APEX Study and
recommended that the Company proceed with the study as planned
without modification based on its analysis of preliminary efficacy
trends and safety reports. Pooled blinded aggregate VTE event rates
in the study remain on target.
- Continued to enroll patients in APEX at more than 450 global
sites, with the study now greater than 70 percent enrolled.
Andexanet Alfa – Factor Xa inhibitor antidote on Accelerated
Approval pathway and U.S. Food and Drug Administration
(FDA)-designated breakthrough therapy
- Presented data from the first part (andexanet bolus only) of
the Phase 3 ANNEXATM-Apixaban study in an oral session at the
American Heart Association (AHA) Annual Scientific Sessions.
Results demonstrated that the study achieved all of its primary and
secondary endpoints with high statistical significance. Andexanet
was well tolerated with no serious adverse events reported.
- Announced that the first part (andexanet bolus only) of the
Phase 3 ANNEXA-Rivaroxaban study achieved all of its primary and
secondary endpoints with high statistical significance. Andexanet
was well tolerated with no serious adverse events reported.
- Presented positive results from a Phase 2 study demonstrating
that andexanet alfa reversed the anticoagulant effect of Daiichi
Sankyo's edoxaban in healthy volunteers at the American Society of
Hematology (ASH) 2014 Annual Meeting.
- Initiated ANNEXA-4, an open-label, single-arm, confirmatory
Phase 4 study in patients receiving apixaban, rivaroxaban,
enoxaparin or edoxaban who present with an acute major bleed to
support an Accelerated Approval pathway for andexanet.
- Granted orphan drug designation by the FDA's Office of Orphan
Products Development.
Cerdulatinib – Oral, dual Syk/JAK kinase inhibitor for
hematologic cancers
- Presented interim Phase 1 data in patients with
relapsed/refractory chronic lymphocytic leukemia and B-cell
non-Hodgkin lymphoma at the ASH 2014 Annual Meeting. The Phase 1
part of this Phase 1/2a study is ongoing, and we continue to seek
the maximum tolerated dose.
Corporate
- Announced that Robert Califf, M.D., retired from the Board of
Directors to accept a position as the Deputy Commissioner for
Medical Products and Tobacco at the FDA.
- Appointed two biotechnology industry veterans, Laura Brege and
Dr. Dennis Fenton, to the Board of Directors.
Anticipated Upcoming Events and Milestones
Betrixaban
- Conduct an additional planned safety review of the APEX Study
by mid-year.
- Complete patient enrollment in APEX by the end of 2015.
- Report top-line APEX data in early 2016.
- Submit a New Drug Application (NDA) to the FDA in 2016.
Andexanet Alfa
- Present data from the first part (andexanet bolus only) of the
Phase 3 ANNEXA-Rivaroxaban study on Monday, March 16, 2015, in an
oral session at the American College of Cardiology's (ACC) 64th
Annual Scientific Session in San Diego.
- Announce top-line data from the second part (bolus plus
continuous infusion) of the Phase 3 ANNEXA-Apixaban and
ANNEXA-Rivaroxaban studies in the first half of 2015.
- Initiate a Phase 2 betrixaban reversal study and report data
this year.
- Continue to conduct additional validation with CMC Biologics to
support a Biologics License Application (BLA) and commercial
launch.
- Scale-up commercial manufacturing process at Lonza to support
broader worldwide demand.
- Submit a BLA to the FDA under an Accelerated Approval pathway
at the end of 2015.
Cerdulatinib
- Complete the Phase 1 part of the study and seek to identify the
maximum tolerated dose.
- Begin enrolling patients with chronic lymphocytic leukemia and
non-Hodgkin lymphoma in clinical expansion cohorts.
Fourth Quarter and Year-End Financial
Results
Collaboration revenue for the fourth 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 $2.1
million for the fourth quarter of 2013. Collaboration revenue for
the year ended December 31, 2014, was $9.6 million compared with
$10.5 million for the year ended December 31, 2013.
Total operating expenses for the fourth quarter of 2014 were
$41.7 million compared with $27.4 million for the same period in
2013. Total operating expenses for the year ended December 31,
2014, were $147.2 million compared with $94.7 million for 2013.
Total operating expenses for the full year ended December 31, 2014,
included $9.3 million in stock-based compensation expense compared
with $5.0 million for 2013. Research and development expenses were
$123.6 million for the year ended December 31, 2014, compared with
$79.3 million for 2013, as the Company continued to support its
Phase 3 APEX Study of betrixaban, its Phase 3 and studies of
andexanet alfa, its Phase 1/2a clinical study of cerdulatinib, and
its manufacturing scale-up and build-out in preparation for the
launch of andexanet alfa. General and administrative expenses for
the fourth quarter of 2014 were $6.9 million compared with $4.8
million for the same period in 2013. General and administrative
expenses for the year ended December 31, 2014, were $23.6 million
compared with $15.4 million for 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 including compliance with the Sarbanes-Oxley Act of
2002.
For the fourth quarter of 2014, Portola reported a net loss of
$39.3 million, or $0.82 net loss per share, compared with a net
loss of $25.1 million, or $0.63 net loss per share, for the same
period in 2013. Shares used to compute net loss per share
attributable to common stockholders were 48.2 million for the
fourth quarter of 2014 compared with 39.8 million for the same
period in 2013. Net loss for the year ended December 31, 2014, was
$137.1 million or $3.19 net loss per share, compared with a net
loss of $83.4 million, or $3.65 net loss per share, for the same
period in 2013. Shares used to compute net loss per share
attributable to common stockholders were 43.0 million for 2014
compared with 22.8 million for 2013.
Cash, cash equivalents and investments at December 31, 2014,
totaled $392.3 million compared with $319.0 million as of December
31, 2013.
2015 Annual Financial Guidance
Portola expects to independently advance its three wholly-owned
assets through key milestones during 2015, including submitting the
BLA for andexanet and completing enrollment of the APEX study. For
2015, Portola expects total pro-forma operating expenses to be
between $220 million and $235 million, excluding stock-based
compensation. These expenses will be primarily in support of its
ongoing Phase 3 registration studies for its two lead programs,
including the APEX Study of betrixaban and the Phase 3 ANNEXA
series with andexanet alfa, the ongoing Phase 4 ANNEXA-4 study of
andexanet alfa, manufacturing work to produce andexanet alfa at
commercial scale, the BLA filing for andexanet alfa, and the Phase
1/2a clinical study of cerdulatinib. Portola expects to end 2015
with approximately $165 million to $180 million in cash, cash
equivalents and investments.
Non-GAAP Financial Projection
This press release and the reconciliation table included herein
include a non-GAAP projection of 2015 operating expenses, excluding
stock-based compensation. A reconciliation to projected GAAP 2015
operating expenses is provided in the accompanying table entitled
"Reconciliation of GAAP to Non-GAAP Projected Operating Expenses."
Portola management believes this non-GAAP information is useful for
investors because it provides information about the Company's
ability to independently advance its assets.
Conference Call Details
To access the live conference call today, March 2, 2015, at 8:30
a.m. Eastern Time via phone, please dial (877) 280-4958 from the
United States and Canada or +1 (857) 244-7315 internationally. The
participant passcode is 71124473. 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 program is 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, 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 – Bristol-Myers Squibb and Pfizer
(Eliquis [apixaban]), Bayer HealthCare and Janssen Pharmaceuticals
(XARELTO® [rivaroxaban]), and Daiichi Sankyo (edoxaban) – 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, 2015 annual financial
guidance, the timing and occurrence of events described under the
section "Anticipated Upcoming Events and Milestones," our goal of
becoming a fully commercialized biopharmaceutical company, and our
intention to independently advance our product candidates. 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; our ability to successfully build a
hospital-based sales force and commercial infrastructure;
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
which we expect to file on or about March 2, 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.
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|
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|
|
PORTOLA
PHARMACEUTICALS, INC. |
|
|
|
|
|
Unaudited Condensed
Statements of Operations |
(In thousands, except share
and per share data) |
|
|
|
|
|
|
Three Months
Ended |
Twelve Months
Ended |
|
December
31, |
December
31, |
|
2014 |
2013 |
2014 |
2013 |
Collaboration and license revenue |
$ 2,412 |
$ 2,057 |
$ 9,625 |
$ 10,531 |
Operating expenses: |
|
|
|
|
Research and development |
34,722 |
22,644 |
123,639 |
79,286 |
General and administrative |
6,950 |
4,769 |
23,552 |
15,423 |
Total operating
expenses |
41,672 |
27,413 |
147,191 |
94,709 |
Loss from operations |
(39,261) |
(25,356) |
(137,566) |
(84,178) |
Interest and other (expense) income,
net |
5 |
294 |
441 |
826 |
Net loss attributable to common
stockholders |
$ (39,256) |
$ (25,062) |
$ (137,125) |
$ (83,352) |
Shares used to compute net loss per share
attributable to common stockholders: |
|
|
|
|
Basic and diluted |
48,153,468 |
39,781,245 |
42,977,463 |
22,842,443 |
Net loss per share attributable to common
stockholders: |
|
|
|
|
Basic and diluted |
$ (0.82) |
$ (0.63) |
$ (3.19) |
$ (3.65) |
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PORTOLA
PHARMACEUTICALS, INC. |
|
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|
Unaudited Condensed
Balance Sheet Data |
(In thousands) |
|
|
|
|
December 31,
2014 |
December 31,
2013 |
ASSETS |
(Unaudited) |
Cash, cash equivalents and investments |
$ 392,303 |
$ 319,036 |
Total current assets |
315,077 |
272,707 |
Property and equipment, net |
2,776 |
2,600 |
Total assets |
416,495 |
325,731 |
Accounts payable |
14,084 |
3,232 |
Accrued and other liabilities |
13,966 |
17,796 |
Deferred revenue (current portion and
long-term) |
36,585 |
5,211 |
Total current liabilities |
41,131 |
25,555 |
Total liabilities |
68,693 |
29,396 |
Total stockholders' equity |
347,802 |
296,335 |
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|
|
|
Reconciliation of GAAP to
Non-GAAP Projected Operating Expenses |
(in millions) |
|
|
Low |
High |
2015 Operating
Expenses—GAAP |
$ 233.0 |
$ 248.0 |
Stock-based compensation
expense |
13.0 |
13.0 |
2015 Operating
Expenses—Non-GAAP |
$ 220.0 |
$ 235.0 |
CONTACT: Investor Contact:
Alexandra Santos
Portola Pharmaceuticals
ir@portola.com
650.246.7239
Media Contact:
Joey Fleury
BrewLife
jfleury@brewlife.com
415.946.1090
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