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Portola Pharmaceuticals Reports Fourth Quarter and Year-End 2014 Financial Results and Provides Corporate Update
"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
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
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'sedoxaban 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.
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 Bregeand Dr. Dennis Fenton, to the Board of Directors.
Anticipated Upcoming Events and Milestones
- 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
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
Lonzato support broader worldwide demand.
Submit a BLA to the
FDAunder an Accelerated Approval pathway at the end of 2015.
- 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
Total operating expenses for the fourth quarter of 2014 were
For the fourth quarter of 2014, Portola reported a net loss of
Cash, cash equivalents and investments at
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
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,
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, 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
|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,|
|Collaboration and license revenue||$ 2,412||$ 2,057||$ 9,625||$ 10,531|
|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)|
|PORTOLA PHARMACEUTICALS, INC.|
|Unaudited Condensed Balance Sheet Data|
|Cash, cash equivalents and investments||$ 392,303||$ 319,036|
|Total current assets||315,077||272,707|
|Property and equipment, net||2,776||2,600|
|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 stockholders' equity||347,802||296,335|
|Reconciliation of GAAP to Non-GAAP Projected Operating Expenses|
|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|