Replies to post #185836 on Biotech Values

[DewDiligence]

PTLA starts phase-4* outcomes trial of Andexanet alfa:

http://finance.yahoo.com/news/portola-pharmaceuticals-initiates-phase-4-130000239.html

The open-label, single-arm, Phase 4 study is being conducted in patients receiving apixaban, rivaroxaban or enoxaparin…who present with an acute major bleed. Acute major bleeding includes life-threatening bleeding, bleeding associated with very low blood counts, or bleeding that occurs in a critical area such as the brain or surrounding the heart. The trial excludes bleeding due to major trauma and large blood vessel rupture.

Patients will receive andexanet alfa as an intravenous (IV) bolus followed immediately by a continuous infusion. The study is evaluating andexanet alfa's ability to decrease anti-Factor Xa activity and restore hemostasis in patients. Safety endpoints include overall 45 day safety, including an evaluation of thrombotic activity and antibody development. The study will be conducted at over 50 sites in North America and Europe.

PTLA plans to submit a BLA in late 2015 seeking accelerated approval based not on clinical outcomes, but rather on restoring FXa levels that have been lowered by taking Xarelto, Eliquis, or Lovenox. The phase-4 (technically phase-3b) trial described above is needed to convert an accelerated approval into a full-fledged FDA approval with a label that includes the ability to treat major bleeding episodes caused by FXa or LMWH anticoagulants.

*Technically, this is a phase-3b trial.