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Portola Pharmaceuticals Reports First Quarter Financial Results and Provides Corporate Update
"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
- 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 2014issue of the American Heart Journal.
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'soral, 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 Groupto 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.
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
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
- 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.
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) Congressin Barcelonaon 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.
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 Chicagoon June 1, 2014.
Report additional data from the Phase 1/2 trial in patients with refractory
NHLand CLL in 2014.
First Quarter Financial Results
Collaboration revenue for the first quarter of 2014 was
Total operating expenses for the first quarter of 2014 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 30 million patients worldwide.
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]),
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.
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
*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|
|Collaboration and license revenue||$ 2,372||$ 3,108|
|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|
|March 31,||December 31,|
|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|
|Accrued and other liabilities||14,990||17,796|
|Total current liabilities||32,798||25,555|
|Total stockholders' equity||269,192||296,335|