Portola Pharmaceuticals Inc.® (NASDAQ:PTLA), today provided a
corporate update and reported its financial results for the first
quarter ended March 31, 2016.
“During the first quarter of 2016, we continued to advance the
development of our three product candidates. We reported acceptance
of our ANDEXXA™ (andexanet alfa) BLA and Phase 3 topline data from
the APEX trial of betrixaban, completed a successful FDA
pre-approval inspection of our Generation 1 commercial
manufacturing process for ANDEXXA and completed the Phase 1
cerdulatinib study,” said Bill Lis, chief executive officer of
Portola. “We remain focused on successfully launching ANDEXXA, an
FDA-designated Breakthrough Therapy and filing an NDA for
betrixaban, an FDA-designated Fast Track Therapy, this year.”
Recent Achievements, Upcoming Events and
Milestones
ANDEXXA (andexanet alfa) – an FDA-designated Breakthrough
Therapy Factor Xa inhibitor antidote in development for reversal of
anticoagulation in patients treated with a Factor Xa inhibitor who
are admitted to the hospital with uncontrolled bleeding or who need
urgent surgery
- The FDA accepted Portola’s BLA submission for ANDEXXA for
filing in February 2016
- The FDA completed a successful pre-approval inspection of CMC
Biologics’ Generation 1 process; the inspection addressed the
Generation 1 2,500 liter scale and the 6x2,000 liter scale process
and confirmed alignment with the BLA
- Completed Generation 2 GMP batches at the 10,000 liter scale at
Lonza and achieved target yields
- Entered into collaboration agreements with all of the
manufacturers of oral Factor Xa inhibitors in Japan to develop
and/or commercialize andexanet alfa in that territory
- Enrollment remains on track for ANNEXATM-4, a Phase 3b/4
confirmatory study
- Plan to present data from the Phase 2 proof-of-concept study
with betrixaban in healthy volunteers at a medical conference this
year
- Preparing for commercial launch shortly after the PDUFA date of
August 17, 2016, if approved
- Plan to submit an MAA with the EMA in the third quarter of
2016
Betrixaban – an oral Factor Xa inhibitor anticoagulant in
development for the prevention of venous thromboembolism (VTE) in
acute medically ill patients
- Reported topline results from the pivotal Phase 3 APEX
Study
- Plan to present data from the APEX Study on Friday, May 27, at
the International Society on Thrombosis and Haemostasis (ISTH) 62nd
Annual SSC (Scientific and Standardization Committee) Meeting in
Montpellier, France; will hold an investor webcast directly
following the presentation to discuss the APEX Study data
- Plan to meet with the FDA in the second quarter and with the
European Medicines Agency (EMA) to discuss the APEX Study results
and a regulatory path forward
- Pending discussions, plan to submit a New Drug Application
(NDA) with the FDA and a Marketing Authorization Application (MAA)
with the EMA by the end of the year
Cerdulatinib – an oral, dual syk/JAK inhibitor in development to
treat resistant or relapsed hematologic cancer patients
- Achieved the maximum tolerated dose and completed the Phase 1
study; plan to present results in a poster presentation at the
American Society of Clinical Oncology 2016 Annual Meeting in
June
- Initiated a Phase 2 study and expect to enroll the first
patient during the second quarter
First Quarter 2016 Financial
ResultsCollaboration revenue earned under Portola's
collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer
Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Lee's
Pharmaceutical was $8.3 million for the first quarter of 2016
compared with $2.4 million for the first quarter of 2015. The
increase in revenue was primarily the result of achieving certain
milestones from Portola’s Daiichi Sankyo and Bayer and Janssen
clinical agreements with the filing of the ANDEXXA BLA.
Total operating expenses for the first quarter of 2016 were
$73.6 million compared with $48.9 million for the same period in
2015. Total operating expenses for the first quarter of 2016
included $7.1 million in stock-based compensation expense compared
with $5.2 million for the same period in 2015.
Research and development expenses were $58.8 million for the
first quarter of 2016 compared with $39.9 million for the first
quarter of 2015 as Portola continued to support its manufacturing
scale-up of ANDEXXA in preparation for commercial launch and work
on its larger-scale Generation 2 manufacturing process at Lonza,
the Phase 3b/4 ANNEXA-4 study of ANDEXXA, and the Phase 1/2a
clinical study of cerdulatinib.
Selling, general and administrative expenses for the first
quarter of 2016 were $14.8 million compared with $9.0 million for
the same period in 2015 as the Company increased headcount to
support its growth and increased pre commercial launch activities,
including hiring key regional sales directors and national account
managers and further developing medical affairs.
For the first quarter of 2016, Portola reported a net loss of
$65.0 million, or $1.15 net loss per share, compared with a net
loss of $46.9 million, or $0.95 net loss per share, for the same
period in 2015.
As of March 31, 2016, cash, cash equivalents and investments
totaled $421.0 million compared with cash, cash equivalents and
investments of $460.2 million as of December 31, 2015.
2016 Annual Financial GuidanceFor the fiscal
year 2016, Portola expects total pro-forma operating expenses to be
between $295 million and $320 million, excluding stock-based
compensation. These expenses will be primarily in support of
submissions of Generation 1 and Generation 2 manufacturing scale-up
of ANDEXXA in preparation for commercial launch, the ongoing
ANNEXA-4 study of ANDEXXA, the U.S. launch of ANDEXXA, the
submission of NDA and MAA for betrixaban, pending regulatory
discussions, and the Phase 2 clinical study of
cerdulatinib.
Non-GAAP Financial ProjectionThis press release
and the reconciliation table included herein include a non-GAAP
projection of 2016 operating expenses, excluding stock-based
compensation. A reconciliation to projected GAAP 2016 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, May 5, 2016, at 4:30 p.m. Eastern
Time via phone, please dial (844) 452-6828 from the
United States and Canada or +1 (765) 507-2588
internationally, and use the passcode 89833554. 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
three programs, including betrixaban, an oral, once-daily Factor Xa
inhibitor; ANDEXXA™ (andexanet alfa), a recombinant protein
designed to reverse the anticoagulant effect in patients treated
with an oral or injectable Factor Xa inhibitor; and cerdulatinib, a
Syk/JAK inhibitor in development to treat hematologic cancers.
Portola's partnered program is focused on developing selective Syk
inhibitors for inflammatory conditions. For more information, visit
www.portola.com and follow the Company on Twitter
@Portola_Pharma.
Portola Forward-looking StatementStatements
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 "Recent Achievements, Upcoming Events and Milestones,"
our goal of becoming a fully commercialized biopharmaceutical
company, the projected timing of our product launches, the
occurrence and timing of planned discussions and 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: failure to obtain FDA approval for one or more
of our product candidates, 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
quarterly report on Form 10-Q, which we expect to file today. 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.
|
Unaudited Condensed Consolidated Statements of
Operations |
(In thousands, except share and per share data) |
|
Three Months Ended March 31, |
|
2016 |
|
|
2015 |
Collaboration and
license revenue |
$ |
|
8,258 |
|
|
|
$ |
|
2,359 |
|
Operating
expenses: |
|
|
|
|
|
|
Research and development |
|
|
58,813 |
|
|
|
|
|
39,858 |
|
Selling, general and
administrative |
|
|
14,751 |
|
|
|
|
|
9,005 |
|
Total operating expenses |
|
|
73,564 |
|
|
|
|
|
48,863 |
|
Loss
from operations |
|
|
(65,306 |
) |
|
|
|
|
(46,504 |
) |
Interest
and other income (expense),net |
|
|
332 |
|
|
|
|
|
(409 |
) |
Net
loss |
$ |
|
(64,974 |
) |
|
|
$ |
|
(46,913 |
) |
Net loss
attributable to Noncontrolling interest (Development Partner) |
|
— |
|
|
|
— |
Net loss
attributable to Portola |
$ |
|
(64,974 |
) |
|
|
$ |
|
(46,913 |
) |
Shares
used to compute net loss per share attributable to Portola common
stockholders: |
|
|
|
|
|
|
Basic and diluted |
|
|
56,397,881 |
|
|
|
|
|
49,527,850 |
|
Net loss
per share attributable to Portola common stockholders: |
|
|
|
|
|
|
Basic and diluted |
$ |
|
(1.15 |
) |
|
|
$ |
|
(0.95 |
) |
|
|
|
|
|
|
|
Unaudited Condensed Consolidated Balance Sheet
Data |
(In thousands) |
|
March 31, 2016 |
|
December 31, 2015 |
|
(Unaudited) |
Cash,
cash equivalents and investments |
$ |
420,979 |
|
$ |
460,161 |
Receivables from
collaborators |
|
7,500 |
|
|
1,000 |
Prepaid
research and development |
|
10,854 |
|
|
16,976 |
Total current
assets |
|
442,890 |
|
|
465,577 |
Property
and equipment, net |
|
6,779 |
|
|
6,243 |
Intangible asset |
|
3,151 |
|
|
3,151 |
Total
assets |
|
463,997 |
|
|
502,924 |
Accounts payable |
|
13,281 |
|
|
10,279 |
Accrued
research and development |
|
17,574 |
|
|
24,195 |
Accrued compensation
and other liabilities |
|
5,434 |
|
|
8,285 |
Deferred
revenue (current portion and long-term) |
|
51,508 |
|
|
27,016 |
Total current
liabilities |
|
53,227 |
|
|
51,146 |
Total
liabilities |
|
90,457 |
|
|
72,601 |
Total Portola
stockholders’ equity |
|
370,613 |
|
|
427,396 |
Noncontrolling interest (Development Partner) |
|
2,927 |
|
|
2,927 |
Total liabilities and
stockholders equity |
|
463,997 |
|
|
502,924 |
|
|
|
|
|
|
Reconciliation
of GAAP to Non-GAAP Projected Operating
Expenses |
|
(in millions) |
|
|
|
|
|
Low |
|
High |
2016 Operating
Expenses—GAAP |
$ |
334 |
$ |
359 |
Stock-based compensation expense |
|
39 |
|
39 |
2016 Operating Expenses—Non-GAAP |
$ |
295 |
$ |
320 |
|
|
|
|
|
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
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