United Therapeutics (UTHR) announced on September 2 (U.S. time) that its nebulized treprostinil inhalation solution (Nebulized Tyvaso) met the primary endpoint in its global Phase 3 clinical trial for idiopathic pulmonary fibrosis (IPF), successfully expanding to a third indication and driving its stock price up 32% in celebration. PHARMOSA BIOPHARM’s L606, currently the only long-acting nebulized treprostinil inhalation solution, is expected to benefit from this indication expansion and will share related revenue through its licensing partnership with Liquidia Technologies (LQDA).

Currently, only two oral drugs—Ofev (nintedanib) and Esbriet (pirfenidone)—are available for IPF treatment, yet patients often face challenges with efficacy and tolerability during long-term use. Although combined global sales of Ofev and Esbriet exceeded US$4 billion in 2024, only about 30% of IPF patients have received treatment, underscoring the unmet demand for new therapeutic options.

With the positive Phase 3 results, UTHR’s nebulized treprostinil is now positioned to expand beyond its current indications—pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD)—and enter the IPF market, which has an estimated 100,000 patients in the U.S.

PHARMOSA BIOPHARM’s L606, a 505(b)(2) new drug candidate and the only long-acting nebulized treprostinil formulation in development, is expected to gain from this expanded indication as well. Featuring enhanced dosing convenience, L606 will allow PHARMOSA BIOPHARM to share in the revenue growth alongside its licensing partner LQDA.

Resource: UTHR霧化Tyvaso成功擴第三適應症，國邑*L606可望搭順風車同享收益