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EX - 99.1 2 brhc10020565_ex99-1.htm EXHIBIT 99.1 Insmed® Exhibit 99.1 Insmed Announces Topline Results from Phase 1 Study of Treprostinil Palmitil Inhalation Powder ( TPIP ) -TPIP Generally Safe and Well Tolerated in Healthy Volunteers- -TPIP Showed Substantially Lower Cmax and Longer Half - Life than Currently Available Inhaled Treprostinil Therapy- --Pharmacokinetic Profile Supports Continued Development with Once - Daily Dosing- -Conference Call to be Held at 8:30 a.m. ET Today to Discuss Results— BRIDGEWATER , N.J. , February 19 , 2021 / PRNewswire / - Insmed Incorporated ( Nasdaq : INSM ) , a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases , today announced topline results from the Phase 1 study of treprostinil palmitil inhalation powder ( TPIP ) in healthy volunteers . Data from the study demonstrated that TPIP was generally safe and well tolerated , with a pharmacokinetic profile that supports once - daily dosing . A conference call will be held today , February 19 , 2021 , at 8:30 am ET , with Insmed management to further discuss these results and provide an update on the planned development pathway for ТРІР . " We are very pleased to share these encouraging Phase 1 results , which we believe validate several critical aspects of the TPIP profile and continue to build on the momentum of our earlier preclinical work , " said Martina Flammer , M.D. , MBA , Chief Medical Officer of Insmed . " Importantly , these findings support the continued development of TPIP with once - daily dosing in a clinical trial program for patients with pulmonary arterial hypertension ( PAH ) . This is a serious , progressive , and rare disease in which the current standard of care is limited by tolerability issues and a cumbersome dosing regimen . " The Phase 1 study of 42 healthy volunteers was designed to assess the safety , tolerability , and pharmacokinetics of TPIP in the setting of single - dose and multiple - dose administration . The highest dose tested as a single dose was 675 µg , and the highest dose tested in repeated dosing was 225 μg . The study demonstrated that TPIP was generally safe and well tolerated . The most common adverse events ( AEs ) across all cohorts in the study were cough , dizziness , headache , and nausea . Most AEs were mild in severity and consistent in nature with those typically seen with other inhaled prostanoid therapies . There were few moderate AEs and no severe or serious AEs . Subjects in the multiple dose panel that incorporated an up - titration approach beginning at 112.5 µg once - daily and progressing to 225 µg once - daily reported fewer AEs compared to the panel dosed with 225 µg once - daily from the first dose . Overall pharmacokinetic results demonstrated that treprostinil exposure ( AUC and Cmax ) was dose - proportional , with low to moderate inter - subject variability . Treprostinil was detected in the plasma at 24 hours at all doses and throughout the 48 - hour sampling period for the two highest doses . Compared with currently available inhaled treprostinil therapy , TPIP showed substantially lower Cmax and longer half- life . Insmed plans to present full data from this study at an upcoming medical meeting . " The positive results from this Phase 1 study provide clear support for advancing TPIP to the next stage of clinical development in PAH as well as exploring its potential in other serious pulmonary disorders , " said Will Lewis , Chair and CEO of Insmed . “ These findings represent a significant step toward unlocking the full potential of prostanoid therapy . With continued development of TPIP , we look forward to evaluating whether this novel treatment candidate may offer the potential for improved tolerability , dosing convenience , and efficacy for patients with PAH . "