Proteostasis Therapeutics has just announced results from their ex-vivo study of PTI CFTR modulators using organoids from patients living with cystic fibrosis (CF). These organoids are genetically identical, and have identical micro-anatomy as the patient they were derived from. Specifically, this study examined individuals who aren’t eligible for standard CFTR treatments because of their genotype.

Ex-Vivo Study

This study is measuring the ex vivo responsiveness to the PTI CFTR modulators (DIR, POS, NES). Over 370 patients have helped to form organic cultures. Of these, 85% have been eligible for this specific study.

Researchers estimate they will have approximately 500 samples by the end of the first quarter of 2020.

The preliminary results were presented January 19-23 at the Keystone Symposia on Tissue Organoids in Canada.

Other Trials

The aim of the ex vivo trial, and subsequent investigations, is to find more precise and personalized treatments for CF patients who have more rare phenotypes.

From the ex vivo study, patients will be selected to participate in a trial called CHOICES which aims to further investigate therapeutic efficacy. This trial will be the very first CF study exploring personalized medicine. The goal is to initiate this trial by the middle of this year.

CHOICES is a placebo-controlled study. Patients will receive the treatment for eight weeks followed by six months of dosing.

Alongside this trial, the MORE trial is also showing promise. This is a Phase 3 placebo-controlled global trial for CF patients who have the more common F508del mutation. This investigation will hopefully confirm the efficacy and the tolerability of the Proteostasis CFTR modulator triple combination for this subset of patients.