EXHIBIT 99.1
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Proteostasis Therapeutics Announces Positive Preliminary Results from
Proprietary Doublet in Cystic Fibrosis Patients
PTI-801 andPTI-808 Dose Response Observed Across All Cohorts and
Statistical Significance Achieved on All Endpoints
Mean Change from Placebo of up to +8% in ppFEV1and -13mM in Sweat Chloride Observed at Third Dose
Level; Fourth and Highest Dosing Cohort Ongoing
Screening of First Triplet Combination Dose Cohort Complete;
First Triplet Data Readout Remains on Track for this Quarter
Company to Host Conference Call and Webcast Today at 5:00 p.m. ET
BOSTON, Mass. – October 17, 2018 – Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced positive preliminary results from three doublet cohorts of the Company’s ongoing Phase 1, randomized, double-blind, placebo-controlled studies of the Company’s proprietary combination therapy doublet,PTI-808 andPTI-801, and an enrollment update from the triplet,PTI-808,PTI-801, andPTI-428, in subjects with cystic fibrosis.
These studies were designed to assess the safety, tolerability, and pharmacokinetics (PK) ofPTI-801, a third-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector,PTI-808, a novel CFTR potentiator, andPTI-428, a novel CFTR amplifier, compared to placebo over a14-day dosing period in CF subjects across dose levels of each modulator. Changes in sweat chloride (SC) concentration and in percent predicted FEV1 (ppFEV1) were assessed and evaluated as endpoints.
At the time of data cut, 21 subjects were randomized and treated in the first three dose cohorts of the doublet combination ofPTI-801 (100 mg, 200 mg, 300 mg) andPTI-808 (50 mg, 100 mg, 300 mg) or received placebo. PK results were as expected and confirmed aonce-a-day dosing profile for bothPTI-801 andPTI-808. In terms of safety, the compounds were generally well tolerated. No pulmonary exacerbations were reported in the dosing or follow up period of the study.
A dose-dependent improvement in ppFEV1 and sweat chloride concentration was observed across cohorts. For ppFEV1, a maximum and statistically significant increase from baseline to day 7 of +6.3% and at day 14 of +5.9% was reported in thePTI-801 300mg/PTI-808 300 mg dose cohort. A statistically significant increase from placebo was also noted from baseline to day 7 of +8.3% and at day 14 of +6.6% in this cohort. A statistically significant decrease in sweat chloride concentration from baseline was also observed at day 7 and at day 14 of approximately 13 mmol/L in thePTI-801 300mg/PTI-808 300 mg dose cohort. The fourth and highest dose cohort, patients receivingPTI-801 (400 mg) andPTI-808 (300 mg), is ongoing, with data expected in the first quarter of 2019.