We have the exclusive right to develop and market a modified release formulation
of beraprost (beraprost-MR) in the United States, Canada, Mexico, South America,
Europe, Egypt, India, South Africa and Australia for the treatment of cardiovascular
indications, pursuant to our license agreement with Toray Industries, Inc. Beraprost
is a chemically stable, orally bioavailable prostacyclin analogue. Like natural
prostacyclin and Remodulin®, beraprost is believed to dilate blood vessels, prevent
platelet aggregation and prevent proliferation of smooth muscle cells surrounding
blood vessels. In 2007, Toray announced that beraprost-MR received regulatory approval
in Japan for use in the treatment of PAH.
We have been studying various formulations of beraprost since 2000. We completed a phase I safety trial of a reformulated, single-isomer version of beraprost (314d) in July 2012, and the data suggested that dosing 314d four times a day was safe. We believe that 314d and treprostinil have differing prostacyclin receptor-binding profiles and thus could provide benefit to certain groups of patients with differing sets of safety and efficacy profiles. We also believe inhaled treprostinil and 314d have complimentary pharmacokinetic and pharmacodynamic profiles, which indicates they should provide greater efficacy in combination for treating PAH. As a result, we are enrolling a phase III study called BEAT (BEraprost 314d Add-on to Tyvaso) to evaluate the clinical benefit and safety of 314d in combination with patients using Tyvaso® (treprostinil) Inhalation Solution who show signs of deterioration on inhaled treprostinil or have a less than optimal response to inhaled treprostinil treatment. We intend to enroll 240 patients in the study, which will have a primary endpoint of time to clinical worsening.