On August 4, 2015 Portola Pharmaceuticals (Nasdaq:PTLA) provided a corporate update and reported its financial results for the second quarter ended June 30, 2015 (Press release, Portola Pharmaceuticals, AUG 4, 2015, View Source;p=RssLanding&cat=news&id=2075608 [SID:1234507007]).
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"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 William Lis, chief executive officer of Portola. "We also continue to advance the development of our dual kinase inhibitor cerdulatinib, which has demonstrated initial clinical proof-of-concept and good tolerability in our ongoing Phase 1/2a study in patients with relapsed/refractory hematologic cancers who have failed multiple therapies. We plan to have final data from this study by the end of the year."
Recent Achievements
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 U.S. Food and Drug Administration (FDA) breakthrough therapy designation and orphan drug status on an Accelerated Approval pathway
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 Congress.
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) (Free ASCO Whitepaper) 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.
Corporate
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. Laurent as vice president of sales.
Planned Upcoming Events and Milestones
Betrixaban
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 FDA in 2016.
Andexanet Alfa
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 Lonza to supply expected worldwide demand.
Submit a BLA to the FDA under an Accelerated Approval pathway at the end of 2015.
Continue to enroll patients in the Phase 4 ANNEXA confirmatory study.
Cerdulatinib
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 Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Lee’s Pharmaceutical was $2.4 million for the second quarter of 2015 as it was for the second quarter of 2014.
Total operating expenses for the second quarter of 2015 were $61.2 million compared with $33.9 million for the same period in 2014. Total operating expenses for the second quarter of 2015 included $4.8 million in stock-based compensation expense compared with $2.4 million for the second quarter of 2014. Research and development expenses were $52.3 million for the second quarter of 2015 compared with $29.0 million for the second quarter of 2014 as the Company continued to support its manufacturing scale-up in preparation for commercial launch and its work on the second-generation manufacturing process of andexanet alfa, its Phase 3 and 4 studies of andexanet alfa, its Phase 3 APEX Study of betrixaban, and its Phase 1/2a clinical study of cerdulatinib. Selling, general and administrative expenses for the second quarter of 2015 were $8.9 million compared with $4.9 million for the same period in 2014 as the Company increased headcount to support its growth, resulting in higher headcount-related costs including stock-based compensation expense and commercial launch activities.
Portola reported a net loss of $58.3 million, or $(1.12) per share, for the second quarter of 2015 compared with a net loss of $31.4 million, or $(0.76) per share, for the second quarter of 2014. Shares used to compute net loss per share attributable to common stockholders were approximately 52.1 million for the second quarter of 2015 compared with approximately 41.2 million for the same period in 2014.
As of June 30, 2015, cash, cash equivalents and investments totaled $407.6 million compared with cash, cash equivalents and investments of $392.3 million as of December 31, 2014.