Proteostasis Therapeutics Files Investigational New Drug Application

Proteostasis Therapeutics, a developer of treatments for diseases caused by dysfunctional protein processing, said early Friday it filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for PTI-808, its cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Proteostasis plans to initiate a phase 1 study of PTI-808 in healthy volunteers in July, the company said in a statement.

By the end of June, the company expects to report preliminary data from multiple ascending dose (MAD) cohorts of two different CF patient populations: CF subjects receiving PTI-428 or placebo in addition to Orkambi as their background therapy for seven days and CF subjects receiving PTI-428 as their sole CFTR modulator therapy or placebo for seven days. After a washout period, patients on Orkambi background therapy who were enrolled in the 7-day dosing MAD cohort are eligible to enroll in a 28-day clinical study for PTI-428.

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