Portola Pharmaceuticals (Nasdaq:PTLA) today provided a corporate
update and reported its financial results for the quarter ended
March 31, 2014.
"We have had a very productive quarter, and we remain confident
we can achieve our goal of bringing to market our three
wholly-owned treatments for patients with blood clots and blood
cancers," said William Lis, chief executive officer of Portola.
"Our strategy has been to take compounds with validated mechanisms
of action, advance them in underserved indications, and increase
their probability of success by using biomarkers and genetics to
guide clinical development. We believe this strategy differentiates
us as a company and can drive both short- and long-term value from
our three potentially groundbreaking products."
Recent Achievements
Betrixaban
- Increased the number of patients enrolled in the pivotal Phase
3 APEX Study (Acute Medically Ill VTE Prevention with Extended
Duration Betrixaban) of betrixaban, Portola's wholly-owned, oral,
once-daily Factor Xa anticoagulant, to approximately 40 percent at
more than 430 active global sites. The APEX Study is designed to
demonstrate superiority of extended-duration betrixaban compared to
in-hospital standard-of-care, injectable Lovenox® (enoxaparin), for
prevention of venous thromboembolism (VTE) in acute medically ill
patients. APEX, which is utilizing a novel biomarker approach to
identify and enroll patients most likely to benefit from therapy,
is the only pivotal thrombosis trial evaluating a single
anticoagulant for VTE prophylaxis in both the in-hospital and
post-discharge settings -- the period of highest VTE risk.
- The independent Data Safety Monitoring Committee (DSMC) held
its third planned review in March and recommended that Portola
proceed with the APEX Study as planned.
- The design and rationale of the APEX Study was published in the
March 2014 issue of the American Heart Journal.
Andexanet Alfa
- Entered into two new Phase 3 clinical collaboration agreements
with Bristol-Myers Squibb/Pfizer and Bayer/Janssen to study
Eliquis® (apixaban) and XARELTO® (rivaroxaban) with andexanet alfa.
We continue to retain 100 percent worldwide rights to this asset.
Andexanet alfa, an FDA-designated breakthrough therapy, is a
potential first-in-class antidote that is being developed to
reverse the anticoagulation activity of Factor Xa inhibitor-treated
patients who are suffering a major bleeding episode or who require
emergency surgery.
- Initiated a Phase 2 proof-of-concept study to evaluate
andexanet alfa as a reversal agent for edoxaban, Daiichi Sankyo's
oral, once-daily, direct Factor Xa inhibitor.
- Completed a Phase 2 proof-of-concept study evaluating andexanet
alfa as a reversal agent for enoxaparin, a low molecular weight
heparin.
- Continued to advance toward the goal of filing a Biologics
License Application (BLA) for andexanet alfa at the end of 2015 by
pursuing changes to the manufacturing strategy. The Company plans
to continue its ongoing work and expand its capacity at CMC
Biologics to include commercial supply for the potential BLA filing
and U.S. launch. The original plan with Lonza Group to increase
capacity and support broader worldwide supply with optimized cost
of goods following the potential BLA filing remains the same.
- Initiated two Phase 3 studies under the U.S. Food and Drug
Administration's (FDA) Accelerated Approval program to evaluate the
safety and efficacy of andexanet alfa with Eliquis and
XARELTO.
Cerdulatinib*
- Advanced to the third patient cohort in the dose-escalation
phase of the Phase 1/2 proof-of-concept clinical study of
cerdulatinib in patients with hematologic cancers, such as
non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia (CLL).
Cerdulatinib, Portola's wholly-owned oral, dual Syk-JAK inhibitor,
is being studied in patients with 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.
- Completed pharmacokinetic and pharmacodynamic assessments from
the dose-escalation phase of the Phase 1/2 study in patients with
NHL and CLL.
Corporate
- Appointed John H. Johnson, chairman, president and chief
executive officer of Dendreon Corporation, to Portola's board of
directors effective March 26, 2014.
Planned Upcoming Events and Milestones
Betrixaban
- Conduct additional planned DSMC reviews of the APEX Study.
- Complete the APEX futility analysis in early 2015.
- Complete patient enrollment in APEX by the end of 2015.
Andexanet Alfa
Series of Additional Phase 2 Milestones:
- Present during both oral and poster sessions an abstract titled
"Reversal of Enoxaparin-Induced Anticoagulation in Healthy Subjects
by Andexanet Alfa (PRT064445), an Antidote for Direct and Indirect
FXA Inhibitors -- A Phase 2 Randomized, Double-Blind,
Placebo-Controlled Trial" (abstract # COA01) at the 60th Scientific
and Standardization Committee meeting of the International Society
on Thrombosis and Haemostasis (ISTH) in Milwaukee, Wis. on June 25,
2014. The abstract received distinction as the top-rated abstract
in its category.
- Present a poster titled "Sustained Reversal of Apixaban
Anticoagulation with Andexanet Alfa Using a Bolus Plus Infusion
Regimen in a Phase 2 Placebo-Controlled Trial" (abstract #85838) at
the European Society of Cardiology (ESC) Congress in Barcelona on
August 30, 2014.
- Report Phase 2 proof-of-concept data with edoxaban in
2014.
- Initiate a Phase 2 proof-of-concept study with betrixaban in
2015.
Series of Phase 3/Regulatory Milestones:
- Report data from the first part of the Phase 3 study in the
fourth quarter of 2014.
- Report data from the second part of the Phase 3 study in the
first half of 2015.
- Initiate a Phase 3b/4 confirmatory study in late 2014 or early
2015.
- File a BLA for conditional approval under an Accelerated
Approval pathway at the end of 2015.
Cerdulatinib
- Present a poster titled "Pharmacokinetics and Pharmacodynamics
of the Dual Syk/JAK Inhibitor PRT062070 (cerdulatinib) in Patients
with Advanced B-cell Malignancies" (abstract #2619; poster board
#82) at the American Society of Clinical Oncology (ASCO) Annual
Meeting in Chicago on June 1, 2014.
- Report additional data from the Phase 1/2 trial in patients
with refractory NHL and CLL in 2014.
First Quarter Financial Results
Collaboration revenue for the first quarter of 2014 was $2.4
million earned under Portola's collaborations with Bristol-Myers
Squibb Company and Pfizer, Bayer Pharma and Janssen
Pharmaceuticals, Daiichi Sankyo and Lee's Pharmaceutical.
Collaboration revenue for the first quarter of 2013 was $3.1
million.
Total operating expenses for the first quarter of 2014 were
$33.4 million compared with $20.8 million for the first quarter of
2013. Total operating expenses for the first quarter of 2014
included $2.3 million in stock-based compensation expense compared
with $0.7 million for the first quarter of 2013. Research and
development expenses were $28.2 million for the first quarter of
2014, compared with $17.7 million for the first quarter of 2013, as
the Company continued to support its Phase 3 APEX Study of
betrixaban, multiple Phase 3 and Phase 2 proof-of-concept studies
of andexanet alfa, and its Phase 1/2 clinical study of
cerdulatinib. General and administrative expenses for the first
quarter of 2014 were $5.2 million compared with $3.0 million for
the first quarter of 2013, as the Company increased headcount to
support its growth, resulting in higher headcount-related costs,
including stock-based compensation expense and increased costs
associated with being a public company.
Portola reported a net loss of $30.7 million, or $0.75 net loss
per share, for the first quarter of 2014, compared with a net loss
of $18.1 million, or $12.94 net loss per share, for the first
quarter of 2013. Shares used to compute net loss per share
attributable to common stockholders were approximately 41.0 million
for the first quarter of 2014, compared with approximately 1.4
million for the same period in 2013.
As of March 31, 2014, cash, cash equivalents and investments
totaled $305.7 million, which includes payments of $16.4 million
received from collaboration partners during the first quarter,
compared with cash, cash equivalents and investment of $319.0
million as of December 31, 2013.
Conference Call Details
To access the live conference call today, May 12, 2014, at 4:30
p.m. Eastern Time via phone, please dial (866) 515-2912 from the
United States and Canada or +1 (617) 399-5126 internationally. The
participant passcode is 75374493. 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.
About Portola Pharmaceuticals, Inc.
Portola Pharmaceuticals is a biopharmaceutical company
developing product candidates that have the potential to represent
significant advances in 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 first-in-class 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 30
million patients worldwide.
Andexanet Alfa
Portola's second product candidate in the area of thrombosis,
andexanet alfa, has the potential to be a first-in-class reversal
agent to reverse the effects of Factor Xa inhibitors in patients
who suffer a major bleeding episode or who require emergency
surgery. Portola has entered into 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. Andexanet alfa has been
designated as a Breakthrough Therapy by the U.S. Food and Drug
Administration.
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 study in patients with 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, future financial
results, including operating expenses and cash position, pursuit of
strategic options, statements regarding: our plans for future
clinical studies, regulatory filings and pursuit of an Accelerated
Approval process for andexanet alfa, our manufacturing process and
timeline for andexanet alfa, expected benefits from biomarker or
genetic approaches to clinical development, and the timing and
occurrence of events described under the section "Planned 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. 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
first quarter of 2014, which we expect to file on or about May 12,
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).
PORTOLA
PHARMACEUTICALS, INC. |
|
|
|
Unaudited Condensed
Statements of Operations Data |
(In thousands, except share and
per share data) |
|
|
|
|
Three Months
Ended |
|
March
31, |
|
2014 |
2013 |
Collaboration and license revenue |
$ 2,372 |
$ 3,108 |
Operating expenses: |
|
|
Research and development |
28,155 |
17,722 |
General and administrative |
5,241 |
3,039 |
Total operating expenses |
33,396 |
20,761 |
Loss from operations |
(31,024) |
(17,653) |
Interest and other income (expense), net |
298 |
(489) |
Net loss |
$ (30,726) |
$ (18,142) |
Net loss attributable to common
stockholders: |
|
|
Basic and diluted |
$ (30,726) |
$ (18,142) |
|
|
|
Shares used to compute net loss per share
attributable to common stockholders: |
|
|
Basic and diluted |
41,001,623 |
1,401,677 |
Net loss per share attributable to common
stockholders: |
|
|
Basic and diluted |
$ (0.75) |
$ (12.94) |
|
|
|
PORTOLA
PHARMACEUTICALS, INC. |
|
|
|
Unaudited Condensed
Balance Sheet Data |
(In thousands) |
|
March 31, |
December 31, |
|
2014 |
2013 |
ASSETS |
(Unaudited) |
Cash, cash equivalents and investments |
$ 305,703 |
$ 319,036 |
Receivables from collaborations |
6,664 |
309 |
Total current assets |
273,266 |
272,707 |
Property and equipment, net |
2,670 |
2,600 |
Total assets |
320,239 |
325,731 |
Accounts payable |
7,380 |
3,232 |
Accrued and other liabilities |
14,990 |
17,796 |
Deferred revenue |
25,839 |
5,211 |
Total current liabilities |
32,798 |
25,555 |
Total liabilities |
51,047 |
29,396 |
Total stockholders' equity |
269,192 |
296,335 |
|
|
|
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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