Replies to post #165360 on Biotech Values

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PTLA hopes they can get FDA approval for PRT-4445 based a single 100-200-patient PD study and that efficacy studies in bleeding patients can be done on a post-marketing basis. I think they’re dreaming.

Where did you see or hear that? To me it looks like they're planning a Phase III. Am I misinterpreting this?...

We've now completed a series of discussions with the FDA including just yesterday and end the Phase 2 meeting regarding the clinical and manufacturing path forward for andexanet alfa. We conducted these meetings yesterday with our academic leaders for the program and with our collaborators from J&J, Bayer and BMS Pfizer.

Based on these meetings we continue to plan for an expedited approval pathway for andexanet alfa that will allow us to bring this important product to the market as soon as possible.

The final agreement with the FDA is pending submission of the Phase 3 protocol which we plan to submit to the FDA later this year in anticipation of investigating these studies in the first half of 2014.

For andexanet alfa we expect the core data from our ongoing Phase 2 study with the Factor Xa inhibitor antidote this year XARELTO and to report data from additional Phase 2 study of andexanet alfa and other Factor Xa inhibitors including edoxaban, betrixaban, and low molecular heparin, enoxaparin. After our recent discussions with the FDA, we are moving forward to initiate our Phase 3 clinical study in andexanet alfa planned for the first half of 2014.

http://seekingalpha.com/article/1641092-portola-pharmaceuticals-ceo-discusses-2q-2013-results-earnings-call-transcript?source=email_rt_article_readmore

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PTLA: Portola Pharmaceuticals Announces Presentation of Preclinical Data on Andexanet Alfa (PRT44545), Factor Xa Inhibitor Antidote, at 2013 European Society of Cardiology Congress
Business Wire August 29, 2013 8:03 AM

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--
Portola Pharmaceuticals, Inc. (NASDAQ: PTLA [FREE Stock Trend Analysis]) today announced that it will present preclinical data on its investigational Factor Xa inhibitor antidote andexanet alfa (PRT4445*) in a moderated poster session at the upcoming 2013 European Society of Cardiology (ESC) Congress, which is taking place in Amsterdam from August 31-September 4.

“These data from a preclinical animal model are noteworthy because they demonstrate that the reversal of Factor Xa anticoagulant activity by andexanet alfa has the potential to reduce blood loss in anticoagulated patients experiencing an active bleed, an important point of differentiation with this compound,” said John T. Curnutte, M.D., Ph. D., executive vice president of research and development for Portola. “We have already established in a Phase 2 clinical study that andexanet alfa can reverse the anticoagulant activity of the Factor Xa inhibitor Eliquis® (apixaban), and, as part of an accelerated development plan, we expect to initiate a pivotal study in 2014 to further assess the effects of andexanet alfa on bleeding.”

Details of the moderated poster presentation follow:

Abstract title: PRT064445 (Andexanet Alfa) Reverses Rivaroxaban Induced Anticoagulation in a Rabbit Liver Laceration “Treatment” Model

Session: Antithrombotic Agents

Presentation date/time: August 31, 2013, 16:22 p.m. CEST

Location: Moderated Posters -- Village 9

Moderator: Stanley Hollenbach, J.D., vice president, pharmacology, Portola Pharmaceuticals

The andexanet alfa abstract is now publically available at http://spo.escardio.org/.

About Andexanet Alfa

Andexanet alfa (proposed INN) is a novel recombinant, modified Factor Xa molecule designed to reverse the anticoagulant activity in patients treated with a Factor Xa inhibitor who suffer an uncontrolled bleeding episode or need to undergo emergency surgery. Andexanet alfa is similar to native Factor Xa, but has been modified to restrict its biological activity to the reversal of the anticoagulant effects of Factor Xa inhibitors. Andexanet alfa acts as a Factor Xa decoy that binds and sequestors direct Factor Xa inhibitors in the blood. Once bound to andexanet alfa, the Factor Xa inhibitors are unable to bind to and inhibit native Factor Xa. The native Factor Xa is then available to participate in the coagulation process and restore hemostasis.

Results from a Phase 1 single ascending dose safety and tolerability study of andexanet alfa conducted by Portola in 32 healthy volunteers showed no apparent safety signals, including no thrombotic adverse events and no antibodies against andexanet alfa, endogenous Factor Xa or Factor X. Results from a Phase 2 proof-of-concept study of andexanet alfa were presented at the 2013 Congress of the International Society on Thrombosis and Haemostasis, demonstrating reversal of the anticoagulant activity of Bristol-Myers Squibb's and Pfizer's Factor Xa inhibitor Eliquis® (apixaban).

Read more: http://www.benzinga.com/pressreleases/13/08/b3874221/portola-pharmaceuticals-announces-presentation-of-preclinical-data-on-a#ixzz2dNIpY3lW