Portola Pharma Top-Line Results Miss Par for the Course

Portola Pharmaceuticals Inc. (NASDAQ: PTLA) made waves early on Thursday morning with less than favorable top-line results. The company announced top-line data from the Phase 3 APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) Study.

This study evaluated the superiority of extended-duration anticoagulation with oral betrixaban compared with standard of care anticoagulation with injectable enoxaparin for the prevention of venous thromboembolism (VTE), or blood clots, in acute medically ill patients.

Note that these are patients who were hospitalized for serious common medical conditions, such as heart failure, stroke, infection and pulmonary disease.

Unfortunately, the results from the study were such that the oral treatment data did not beat out the standard injectable treatment. Also there was no real statistical difference in major bleeding between these two groups.

For some background, APEX is a groundbreaking study because it is the first thrombosis prevention trial to incorporate an enrichment strategy and statistical analysis plan derived from the U.S. Food and Drug Administration (FDA) guidance document on enrichment strategies for clinical trials.
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Roughly 20 million acute medically ill patients in the G7 countries are at risk of developing VTE, either while in the hospital or following discharge. Over a million VTEs and 150,000 VTE-related deaths occur in acute medically ill patients each year in the G7 countries, despite the use of injectable enoxaparin in the hospital.

Bill Lis, CEO of Portola, commented:

While we understand that the interpretation of these statistical and clinical results will be subject to discussions with regulatory agencies, we and the APEX Study academic leadership believe that the data in Cohort 1 were sufficiently strong to support a full assessment of Cohort 2 and the overall study population. We believe the overall robustness of the efficacy and safety results in this high-risk patient population, including the positive net clinical benefit observed, provide ample evidence to support the submission of an NDA later this year.

Shares of Portola closed Wednesday down 2.6% at $28.62, with a consensus analyst price target of $57.60 and a 52-week trading range of $26.10 to $57.96. Following the release of the results, the stock was down 28% at $20.60 in early trading indications Thursday.