Insmed announced on July 16 positive 12-Month data from the ongoing open-label extension (OLE) study evaluating treprostinil palmitil inhalation powder (TPIP), administered once daily in patients with pulmonary arterial hypertension (PAH, World Health Organization Group 1). The OLE study is a non-placebo-controlled trial and was designed to evaluate the long-term safety, tolerability, and effectiveness of TPIP over 24 months in patients who completed the lead-in TPIP pulmonary arterial hypertension studies.
The open label extension data showed sustained benefit regardless of original treatment group (TPIP Continued, n=60 vs. Placebo Crossed, n=31):
By Month 12, both groups showed similar, sustained improvement:
- 6MWD: +55.7m (TPIP Continued) vs. +54.1m (Placebo Crossed)
- NT-proBNP: reduced ~60% in both groups (GMR 0.40 vs. 0.41)
- WHO Functional Class I/II: achieved by 78.3% (TPIP Continued) vs. 80.6% (Placebo Crossed)
- REVEAL Lite 2.0: improved 2.0 points (TPIP Continued) vs. 1.4 points (Placebo Crossed); ~65% of all patients across both groups reached “Refined Low Risk” status (<5% 3-year mortality risk)
- Safety (91 patients total): once-daily TPIP was generally well tolerated through Month 12, with no new safety signals. Most common side effects: headache, cough, nasopharyngitis. 4 deaths occurred, none considered treatment-related.
Bottom line: Patients who crossed over from placebo to TPIP essentially “caught up” to those who started on TPIP from the beginning — reinforcing the drug’s benefit and supporting the newly launched Phase 3 PALM-PAH trial.
Read more on the company press release at this link
See also our previous article here