Portola Pharmaceuticals (NASDAQ:PTLA) gave back some of Friday's 33% gain as it came back from the holiday, dropping 9.1%.
The stock had moved up as the company's NDA for betrixaban was accepted for priority review by the FDA.
For one, Morgan Stanley thinks doctors will go for the anticoagualant. “We continue to believe physicians are likely to support betrixaban given the unmet need in VTE prophylaxis, however, the statistical review is likely to be the greatest area of pushback,” writes analyst Matthew Harrison.
But an advisory committee hearing will be key to shaping investor's views on the drug, intended for acutely ill patients with risk factors for venous thromboembolism.
Harrison has a rating of Overweight and a price target of $29, implying 27% upside from today's lower price.
Also, the European Medicines Agency has validated its MAA for betrixaban for extended-duration prophylaxis of VTE in adults with acute medical illness and risk factors for VTE. The EMA's CHMP is reviewing the application under a standard 210-day review period.
Privately held Dermavant Sciences secures a global exclusive license to Portola Pharmaceuticals' (PTLA -0.8%) cerdulatinib for topical non-cancer applications.
Cerdulatinib is a dual inhibitor of enzymes called spleen tyrosine kinase (Syk) and janus kinase (JAK).
Dermavant intends to develop it for the topical treatment of a range of dermatologic conditions. Portola is developing the candidate for the treatment of blood cancers. It retains the rights to all non-topical formulations.
Portola Pharmaceuticals (PTLA +11%) heads north on slightly higher volume in response to its announcement that is has inked an unsecured $50M loan agreement with Pfizer (PFE +0.2%) and Bristol-Myers Squibb (BMY +1.6%) that will fund the continued development of Factor Xa antidote AndexXa (andexanet alfa).
Under the terms of the agreement, the companies will each loan Portola $25M with principal and interest to be repaid through royalties on AndexXa commercial sales.
Portola intends to resubmit its U.S. marketing application in 2017. It received a Complete Response Letter (CRL) from the FDA in August on its first try. Its marketing application in Europe is currently under review.
Portola Pharmaceuticals (NASDAQ:PTLA) is up 6% premarket, albeit on only 1,355 shares, in response to it announcement of positive interim results from a Phase 3b/4 study, ANNEXA-4, assessing the Factor Xa inhibitor antidote AndexXa (andexanet alfa) in reversing acute major bleeding associated with the anticoagulant. The data were presented today at the European Society of Cardiology Congress in Rome.
A preliminary analysis on interim results from 67 patients (47 evaluated for efficacy) showed a 30-minute bolus of AndexXa rapidly and substantially reversed anti-Factor Xa activity and sustained the reversal when followed by a 120-minute infusion. The majority of patients (79%) achieved good or excellent hemostasis (bleeding stopped) over a 12-hour period after the infusion.
Stuart Connolly, M.D., study Executive Committee chairman and professor in the Department of Medicine of the Faculty of Health Sciences at McMaster University in Hamilton, Ontario says, "In this preliminary analysis, AndexXa was effective in rapidly reversing anti-Factor Xa inhibitor activity and restoring normal blood clotting in real-world patients with Factor Xa-related bleeding. Based on these interim results, we believe that ANNEXA-4 is on track to achieve its co-primary endpoints. The hemostatic efficacy results are especially important because no FDA- or EMA-approved antidote is available for these patients and no existing therapies, including plasma-derived products for warfarin reversal, have demonstrated reversal of Factor Xa inhibitor activity or clinical efficacy and safety."
The interim data showed no AndexXa-associated infusion reactions and no patients developed antibodies to Factor Xa or Factor X or neutralizing antibodies to AndexXa. During the 30-day follow-up period, thrombotic events occurred in 12 patients (18%) while 10 patients died (15%), both within the expected range for this population.
On August 17, the company received a Complete Response Letter (CRL) from the FDA regarding its Biologic License Application (BLA) for AndexXa. It plans to meet with the agency as soon as possible to resolve the issues and clarify a path forward. Its Marketing Authorization Application (MAA) is currently under review in Europe.
Management will host a webcast today at 11:00 am ET to discuss the results.
Portola Pharmaceuticals (NASDAQ:PTLA) plummets 18% premarket on increased volume in response to its announcement that it received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) seeking approval of anticoagulant reversal agent AndexXa (andexanet alfa).
The company says most of the requested information relates to manufacturing although the agency also wants more data to support inclusion of edoxaban [Daiichi Sankyo's (OTCPK:DSKYF)(OTCPK:DSNKY) Savaysa] and enoxaparin [Sanofi's (NYSE:SNY) LOVENOX] in the label. It also stated that it needs more time to complete its review of the clinical amendments to Portola's post-marketing commitments that were submitted recently.
Andexanet alfa is a modified human Factor Xa molecule that acts as a decoy to target and sequester, with high specificity, Factor Xa inhibitors in the blood. Once bound, the Factor Xa inhibitors are unable to inhibit Factor Xa and normal hemostatic processes are restored.
Management will host a conference call this morning at 8:30 am ET to discuss the situation.
Flashing an enormous red flag to investors, Portola Pharmaceuticals (PTLA -10.4%) is showing unsettling bearish action before the expected FDA announcement today on the company's marketing application for anticoagulant reversal agent AndexXa (andexanet alfa). Today's action is wide and loose on 4x higher volume, not what you want to see before a binary event. Longs better hedge their positions immediately.
Portola Pharmaceuticals (NASDAQ:PTLA) is ahead 3% premarket on light volume. Today is the FDA's PDUFA date for the company's marketing application seeking approval of anticoagulant reversal agent AndexXa (andexanet alfa). Commercial launch preparations are underway.