On February 26, 2016 Portola Pharmaceuticals (Nasdaq:PTLA) reported a corporate update and its financial results for the fourth quarter and year ended December 31, 2015 (Press release, Portola Pharmaceuticals, FEB 26, 2016, View Source;p=RssLanding&cat=news&id=2143706 [SID:1234509238]).
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"During the fourth quarter of 2015, we executed on several milestones that we expect will make 2016 a year of unprecedented progress. We plan to commercially launch andexanet alfa and report pivotal Phase 3 results from our APEX study that, if positive, will support the global approval of betrixaban," said Bill Lis, chief executive officer of Portola. "Both have the potential to benefit the patients and transform the field in the multibillion dollar thrombosis market. Additionally, our R&D pipeline continues to progress with our ongoing Phase 1/2 study with cerdulatinib, our oral, dual syk/JAK inhibitor in refractory/relapsed hematologic cancer patients."
Recent Achievements, Upcoming Events and Milestones
Betrixaban – an FDA-designated Fast Track oral Factor Xa inhibitor anticoagulant in development for the prevention of venous thromboembolism (VTE) in acute medically ill patients.
Plan to report topline results from the 7,514-patient pivotal Phase 3 APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) Study in early April.
Plan to submit a New Drug Application to the FDA in the second half of 2016, subject to positive APEX data.
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 and who are admitted to the hospital with uncontrolled bleeding or need urgent surgery.
The FDA accepted the Biologics License Application (BLA) for filing February 16, 2016. The BLA was granted a priority review under an Accelerated Approval pathway, and assigned a PDUFA date of August 17, 2016. We plan to launch andexanet alfa commercially in the U.S. in the second half of 2016, subject to FDA approval.
The full data set from the ANNEXA-R study with rivaroxaban was presented in a Late-Breaking Clinical Trial Session at the American Heart Association’s Scientific Sessions 2015 and simultaneously published in the New England Journal of Medicine.
Licensed development and commercial rights in Japan to Bristol-Myers Squibb Company and Pfizer Inc. Separately, entered into a clinical collaboration agreement with Bayer HealthCare to include its Factor Xa inhibitor rivaroxaban in the development program in Japan.
Continue to enroll patients in ANNEXA-4, a Phase 4 confirmatory study of patients receiving apixaban, rivaroxaban, edoxaban or enoxaparin who present to the hospital with an acute major bleed.
Plan to present data from the Phase 2 proof-of-concept andexanet study with betrixaban in healthy volunteers at a medical conference in 2016 The study was designed to evaluate safety and define the dose of andexanet required to reverse the anticoagulant effect of betrixaban.
Plan to complete FDA pre-approval inspection of the initial (Generation 1 2,500 liter scale at CMC Biologics). Generation 1 validation batch runs at the 6 x 2,000 liter scale are ongoing.
Completed two Generation 2 engineering batches manufactured by Lonza at the 10,000 liter scale, with target yields achieved. Expect commercial validation batches to begin in the second half of 2016.
Cerdulatinib – an oral, dual Syk/JAK kinase inhibitor in development to treat resistant or relapsed hematologic cancer patients.
Presented three abstracts at the 2015 ASH (Free ASH Whitepaper) Annual Meeting on cerdulatinib’s preclinical activity in chronic lymphocytic leukemia; clinical and pharmacodynamic results of a Phase 1/2 study with relapsed/refractory B cell malignancies; and pharmacokinetic modeling of cerdulatinib plasma concentrations in patients with relapsed/refractory B cell malignancies.
Continue to dose-escalate for clinical activity and tolerability in the ongoing Phase 1/2 study in patients with relapsed/refractory B-cell malignancies. Plan to initiate enrollment in expansion cohorts this year.
Corporate
Raised $162.7 million in net proceeds from an underwritten public offering of common stock in December 2015.
Fourth Quarter and Year-End Financial Results
Collaboration 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 $4.4 million for the fourth quarter of 2015 compared with $2.4 million for the fourth quarter of 2014. Collaboration revenue for the year ended December 31, 2015, was $12.1 million compared with $9.6 million for the year ended December 31, 2014.
Total operating expenses for the fourth quarter of 2015 were $70.7 million compared with $41.7 million for the same period in 2014. Total operating expenses for the fourth quarter of 2015 included $6.8 million in stock-based compensation expense compared with $2.5 million for the same period in 2014. Total operating expenses for the year ended December 31, 2015, were $239.2 million compared with $147.2 million for 2014. Total operating expenses for the full year ended December 31, 2015, included $22.9 million in stock-based compensation expense compared with $9.3 million for 2014. Research and development expenses were $200.4 million for the year ended December 31, 2015, compared with $123.6 million for 2014, as the Company continued to support its manufacturing scale-up of andexanet alfa in preparation for the BLA submission and commercial launch and work on its larger-scale Generation 2 manufacturing process at Lonza, its Phase 3 and Phase 4 ANNEXA studies of andexanet alfa, completing enrollment in the Phase 3 APEX Study of betrixaban, and its Phase 1/2a clinical study of cerdulatinib. Selling, general and administrative expenses for the fourth quarter of 2015 were $10.9 million compared with $7.0 million for the same period in 2014. Selling, general and administrative expenses for the year ended December 31, 2015, were $38.9 million compared with $23.6 million for 2014, 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 fourth quarter of 2015, Portola reported a net loss of $66.1 million, or $1.23 net loss per share, compared with a net loss of $39.3 million, or $0.82 net loss per share, for the same period in 2014. Net loss for the year ended December 31, 2015, was $226.5 million, or $4.36 net loss per share, compared with a net loss of $137.1 million, or $3.19 net loss per share, for the same period in 2014.
Cash, cash equivalents and investments at December 31, 2015, totaled $460.2 million compared with cash, cash equivalents and investments of $392.3 million as of December 31, 2014.