Insmed Incorporated (NASDAQ:INSM) has just released positive topline results from its phase 2b study evaluating the efficacy and safety of treprostinil palmitil inhalation powder (TPIP) as once-daily therapy in patients with pulmonary arterial hypertension (PAH). The study, which was a randomized, double-blind, placebo-controlled trial, demonstrated a statistically significant 35% reduction from baseline in pulmonary vascular resistance (PVR) for the primary endpoint, with a p-value of less than 0.001.
Furthermore, the study also showed a 35.5 meter improvement in six-minute walk distance (6MWD) and a 60% reduction from baseline in NT-proBNP concentrations, a biomarker for cardiac stress, for the secondary efficacy endpoints. These results were assessed approximately 24 hours after administration, indicating sustained benefit throughout the 24-hour dosing period.
In terms of patient tolerance, TPIP was well-tolerated in the study, with 75% of patients titrating to the highest dose. Specifically, 84% of patients titrated to at least 480 µg once daily, and 75% titrated to the maximum allowed dose of 640 µg once daily. The overall rate of treatment-emergent adverse events (TEAEs) was 88.4% for patients who received TPIP, compared to 75.8% for patients who received a placebo.
Insmed plans to immediately engage with the U.S. Food and Drug Administration (FDA) to inform the phase 3 trial design for PAH. The company aims to initiate a phase 3 trial in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) before the end of 2025 and a phase 3 trial in patients with PAH in early 2026.
Dr. Gene Sullivan, Chief Product Strategy Officer of Insmed, emphasized the potential breakthrough for patients and the future of prostanoid therapy, stating that the results demonstrate TPIP's potential to be a highly effective and well-tolerated once-daily therapy for the treatment of PAH across disease severities and background treatment regimens.
The study was conducted at 44 sites globally, with a total of 102 patients randomized 2:1 to receive either TPIP or placebo for 16 weeks. Demographics and baseline characteristics were similar in both study arms.
Insmed plans to present detailed results from the phase 2b study of TPIP in PAH and the open-label extension at future medical meetings. The company will also be hosting an investor call to discuss the phase 2b study results.
TPIP, a dry powder formulation of treprostinil palmitil, is an investigational drug product that has not been approved for any indication in any jurisdiction. Insmed, headquartered in Bridgewater, New Jersey, is a global biopharmaceutical company striving to deliver first* and best-in-class therapies to transform the lives of patients facing serious diseases.
For further details, Insmed will be hosting a conference call to discuss the TPIP phase 2b study results in PAH. Today the company's shares have moved 27.43% to a price of $90.07. If you want to know more, read the company's complete 8-K report here.
