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Portola Pharmaceuticals Reports Second Quarter 2015 Financial Results and Provides Corporate Update
"We continue to make solid progress toward near-term significant milestones that will allow us to launch two of our late-stage thrombosis products that target multi-billion dollar hospital-based markets – andexanet alfa in 2016 and betrixaban in 2017. In preparation, we are building our commercial capabilities to launch our products independently, including bringing on a new head of commercial and business development and a head of sales," said
Betrixaban – Potential first-to-market oral Factor Xa inhibitor anticoagulant for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients
- The Phase 3 APEX Study is on track for completion by the end of the year. Study metrics, including pooled blinded aggregate event rates, remain on target.
The APEX Data Safety Monitoring Committee (DSMC) held its final review on
May 4, 2015.
Andexanet Alfa – Factor Xa inhibitor antidote with
Announced complete results from the second part of our Phase 3 ANNEXA™-A study, which evaluated the safety and efficacy of andexanet alfa administered as an intravenous bolus followed by a continuous two-hour infusion in healthy volunteers given the Factor Xa inhibitor Eliquis® (apixaban). The study achieved all primary and pre-specified secondary endpoints with high statistical significance, demonstrating that andexanet alfa produced rapid reversal of the anticoagulant effect of apixaban and sustained it for the duration of the infusion. The data were presented in a Late-Breaking Clinical Trial oral session at the
International Society on Thrombosis and Haemostasis(ISTH) 2015 Congress.
Presented new preclinical data demonstrating that andexanet alfa significantly reduced bleeding and reversed the anticoagulant effects of the Factor Xa inhibitor Xarelto® (rivaroxaban), as measured by anti-Factor Xa activity, in an animal model of bleeding. In contrast, the four-factor prothrombin complex concentrate (PCC) Kcentra® did not impact bleeding or the anti-Factor Xa coagulation biomarker. Results were presented in an oral session at the ISTH 2015
- Phase 4 ANNEXA confirmatory study is on track to support the submission of a Biologics License Application (BLA).
Cerdulatinib – Oral, dual Syk/JAK kinase inhibitor for hematologic cancers
Presented updated safety and efficacy data from the Phase 1 part of the ongoing Phase 1/2a study demonstrating evidence of clinical activity in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies, including partial responses in patients with chronic lymphocytic leukemia (CLL), follicular lymphoma (FL) and transformed FL. Results, presented in a poster discussion session at the
American Society of Clinical Oncology( ASCO) 2015 Annual Meeting, showed that cerdulatinib was well tolerated with no dose-limiting toxicities identified.
- Continued to dose-escalate in the Phase 1 part of the study to determine the maximum tolerated dose (MTD).
- Opened patient enrollment in two clinical expansion cohorts in the Phase 2a part of the study – one in patients with CLL/small lymphocytic leukemia and a second in patients with FL.
Expanded our executive team with the appointment of Tao Fu as executive vice president, chief commercial and business officer, to lead Portola's commercial operations and business development, and the appointment of
Randy St. Laurentas vice president of sales.
Planned Upcoming Events and Milestones
- Complete patient enrollment in the APEX Study by the end of 2015.
- Report topline APEX Study data in early 2016.
Submit a New Drug Application (NDA) to the
- Present topline results from Part 2 of the Phase 3 ANNEXA-R study with rivaroxaban in Q3 2015.
- Initiate a Phase 2 study with betrixaban in 2015.
- Complete commercial manufacturing process validation for BLA and commercial launch.
Scale-up the second-generation commercial manufacturing process at
Lonzato supply expected 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.
- Continue to dose-escalate in the Phase 1 part of the Phase 1/2a study to establish the MTD.
- Begin enrolling patients in two clinical expansion cohorts in the Phase 2a part of the study.
Second Quarter Financial Results
Collaboration revenue earned under Portola's collaborations with
Total operating expenses for the second 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 projected timing of our product launches, 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||Six Months Ended|
|June 30,||June 30,|
|Collaboration and license revenue||$ 2,385||$ 2,415||$ 4,744||$ 4,786|
|Research and development||52,300||28,983||92,158||57,138|
|Selling, general and administrative||8,912||4,937||17,917||10,177|
|Total operating expenses||61,211||33,920||110,075||67,315|
|Loss from operations||(58,827)||(31,505)||(105,331)||(62,529)|
|Interest and other income , net||498||155||89||453|
|Net loss attributable to common stockholders||$ (58,329)||$ (31,350)||$ (105,242)||$ (62,076)|
|Shares used to compute net income (loss) per share attributable to common stockholders:|
|Basic and diluted||52,147,146||41,228,885||50,844,697||41,119,310|
|Net loss per share attributable to common stockholders:|
|Basic and diluted||$ (1.12)||$ (0.76)||$ (2.07)||$ (1.51)|
|PORTOLA PHARMACEUTICALS, INC.|
|Unaudited Condensed Balance Sheet Data|
|June 30,||December 31,|
|Cash, cash equivalents and investments||$407,598||$392,303|
|Total current assets||417,474||315,077|
|Property and equipment, net||4,584||2,776|
|Accrued and other liabilities||24,492||13,966|
|Deferred revenue (current portion and long-term)||31,841||36,585|
|Total current liabilities||49,127||41,131|
|Total stockholders' equity||368,033||347,802|
CONTACT: Investor Contact:
Michele Mantynen Portola Pharmaceuticalsir@portola.com Media Contact: Julie Normart W2O Groupjnormart@w2ogroup.com