Zacks_Analysts' Blog : UTHR Presents Data from FREEDOM-M - Analyst Blog

United Therapeutics Corporation (UTHR) recently presented much-awaited results from a phase III study (FREEDOM-M) conducted with oral treprostinil in patients with pulmonary arterial hypertension (PAH).

Preliminary data showed that oral treprostinil met its primary endpoint of change in six-minute walk distance (6MWD) at 12 weeks. Patients in the oral treprostinil arm improved their median 6MWD by about 23 meters compared to patients on placebo. While the median change from baseline in the oral treprostinil group was 25 meters, the change was -5 meters for placebo at week 12. Despite the statistically significant results, United Therapeutics shares were down 5.9% as the improvement in 6MWD was below expectations.

A significant improvement was also observed in the combined 6MWD and Borg Dyspnea Score rating (shortness of breath test).

However, United Therapeutics said that no significant difference between oral treprostinil and placebo was observed in several secondary efficacy measures like change in Borg Dyspnea Score rating, trough walk at Week 11, change in Dyspnea Fatigue Index, change in WHO functional class, time to clinical worsening and PAH signs and symptoms.

The randomized, double-blind, placebo-controlled FREEDOM-M study enrolled 349 patients who were not receiving any approved PAH medication. The primary analysis was conducted on the 228 patients who received the 0.25 mg tablet at randomization. Meanwhile, an analysis of all the participants in the study showed that patients in the oral treprostinil arm witnessed a 25.5 meter improvement in their median 6MWD compared to patients in the placebo arm.

Treprostinil is already available in an injectable form under the trade name Remodulin and an inhaled version under the trade name Tyvaso. The successful development of oral treprostinil would allow United Therapeutics to have a varied range of therapies in the PAH market. Currently available therapies in the PAH market include products like Gilead’s (GILD) Letairis and Pfizer’s (PFE) Revatio among others.

Neutral on United Therapeutics

We currently have a Neutral recommendation on United Therapeutics. Now that results from FREEDOM-M are out, we expect investor focus to shift to the FREEDOM-C2 study which will report preliminary data in September 2011. The FREEDOM-C2 study is being conducted with 313 patients who are receiving an endothelin receptor antagonist and/or a PDE-5 inhibitor.