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Portola Pharmaceuticals Reports First Quarter 2015 Financial Results and Provides Corporate Update
"This is a monumental year for the company," said
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
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's64th 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 Congressin 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.
$109 millionin 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
- 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
- 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
Lonzato support expected broad worldwide demand
Submit a BLA to the
FDAunder an Accelerated Approval pathway at the end of 2015
- Continue to enroll patients in the Phase 4 ANNEXA confirmatory study
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 Chicagoat 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
Total operating expenses for the first quarter of 2015 were
Portola reported a net loss of
Conference Call Details
To access the live conference call today,
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, an
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.
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
|PORTOLA PHARMACEUTICALS, INC.|
|Unaudited Condensed Statements of Operations Data|
|(In thousands, except share and per share data)|
|Three Months Ended|
|Collaboration and license revenue||$ 2,359||$ 2,372|
|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,|
|Cash, cash equivalents and investments||$ 456,373||$ 392,303|
|Total current assets||403,450||315,077|
|Property and equipment, net||3,211||2,776|
|Accrued and other liabilities||24,761||13,966|
|Total current liabilities||40,793||41,131|
|Total stockholders' equity||418,975||347,802|
Michele Mantynen Portola Pharmaceuticalsir@portola.com Media: Joey FleuryBrewLife email@example.com 415.946.1090