On November 7, 2016 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported a corporate update and reported its financial results for the third quarter ended September 30, 2016 (Press release, Portola Pharmaceuticals, NOV 7, 2016, View Source;p=RssLanding&cat=news&id=2220288 [SID1234516691]).
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"We continue to focus on obtaining regulatory approval for our two lead programs in the United States and EU. Both programs have the potential to become the standard of care in two growth areas of thrombosis that have a high unmet medical need and limited treatment options. If approved, betrixaban, our oral Factor Xa inhibitor, would be the first anticoagulant indicated for extended VTE prophylaxis in the over 24 million medically ill patients admitted to the hospital annually in the G7. AndexXa, our much anticipated Factor Xa inhibitor antidote, would be the first agent approved to treat the growing number of patients admitted to the hospital with life-threatening anticoagulant-related bleeding. We have made important progress toward bringing both betrixaban and AndexXa to market. We submitted an NDA for betrixaban in the United States and plan to submit an MAA in the EU," said Bill Lis, chief executive officer of Portola. "Also, we continue to engage in positive, productive dialogue with the FDA to resolve outstanding questions regarding the Complete Response Letter for AndexXa, most of which are focused on manufacturing."
Recent Achievements, Upcoming Events and Milestones
Betrixaban – an oral Factor Xa inhibitor anticoagulant in development for the prevention of venous thromboembolism (VTE) in acute medically ill patients; designated Fast Track status by the U.S. Food and Drug Administration (FDA)
Submitted a New Drug Application (NDA) to the FDA seeking approval to market betrixaban for extended-duration prophylaxis of VTE in acutely ill medical patients with risk factors for VTE
Plan to submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) by year-end
Three abstracts on results of sub-analyses of the Phase 3 APEX Study conducted by the PERFUSE Study Group were accepted for oral and poster presentations at the upcoming American Heart Association Scientific Sessions 2016
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening bleeding or when urgent surgery is required; designated a Breakthrough Therapy and an Orphan Drug by the FDA
Submitted an MAA, which completed the validation period and was accepted for review by the EMA
Continued productive dialogue with the FDA regarding the Complete Response Letter; Portola currently plans to resubmit the BLA in the second quarter of 2017
Presented a preliminary analysis of interim data from the ongoing Phase 3b/4 ANNEXA-4 Study in patients with acute major bleeding in a Late-Breaking Science Hot Line session at the European Society of Cardiology 2016 Congress with simultaneous publication online by The New England Journal of Medicine
Plan to present data from the ongoing Phase 2 study of andexanet alfa reversal of betrixaban in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2016
Cerdulatinib – an oral, dual Syk/JAK inhibitor in development to treat resistant or relapsed hematologic cancer patients
Continued to enroll patients in a Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies
Two abstracts were accepted for presentation at the upcoming ASH (Free ASH Whitepaper) Annual Meeting 2016
Third Quarter 2016 Financial Results
Collaboration revenue earned under Portola’s collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, and Daiichi Sankyo was $9.3 million for the third quarter of 2016 compared with $2.9 million for the third quarter of 2015. The increase in revenue was primarily due to $2.5 million received upon achievement of a milestone from the Bayer and Janssen Phase 3 agreement, $2.5 million received upon achievement of a milestone from the Daiichi Sankyo Phase 3 agreement, and incremental revenue of $2.0 million from three collaboration and license agreements executed in the first quarter of 2016 to develop and commercialize andexanet alfa in Japan.
Total operating expenses for the third quarter of 2016 were $100.8 million compared with $58.5 million for the same period in 2015. Total operating expenses for the third quarter of 2016 included $7.8 million in stock-based compensation expense compared with $6.1 million for the same period in 2015.
Research and development expenses were $87.2 million for the third quarter of 2016 compared with $48.4 million for the third quarter of 2015. The increase in R&D expenses was primarily due to a $27.3 million impairment charge for AndexXa manufacturing expenses that Portola prepaid to CMC Biologics. These expenses were intended to be credited against future batch manufacturing costs for the Line C, or 6×2,000 liter, manufacturing process. This impairment charge was triggered by the Company’s decision to focus on Line A/B manufacturing at CMC Biologics and Gen 2 manufacturing at Lonza, and suspend Line C manufacturing at CMC Biologics.
Selling, general and administrative expenses for the third quarter of 2016 were $13.6 million compared with $10.1 million for the same period in 2015 as the Company increased headcount to support its growth and increased commercial launch preparation activities for AndexXa in advance of the PDUFA date in August 2016.
For the third quarter of 2016, we reported a net loss of $92.9 million dollars, or a net loss per share of $1.64, compared with a net loss of $55.2 million dollars, or a net loss per share of $1.05, for the same period in 2015. Net loss for the third quarter of 2016 included the $27.3 million impairment charge. Shares used to compute net loss per share attributable to common stockholders were approximately 56.5 million for the third quarter of 2016 compared with approximately 52.6 million for the same period in 2015.
As of September 30, 2016, cash, cash equivalents and investments totaled $274.6 million compared with cash, cash equivalents and investments of $460.2 million as of December 31, 2015.