Proteostasis Therapeutics (NASDAQ:PTI) announces positive, initial ex-vivo results of its proprietary cystic fibrosis transmembrane conductance regulator (CFTR) modulators, PTI-801, PTI-808, and PTI-428, in individuals with cystic fibrosis (CF) who are ineligible for the current standard of care due to their genotype.
The data are part of a pan-European strategic initiative, known as HIT-CF (Human Individualized Therapy of CF), which seeks to accelerate the development of personalized therapies for CF patients, for whom no currently approved CFTR modulator therapy is indicated.
HIT-CF collects tissue samples and develops organoids, or miniaturized organs, that are genetically identical to the patient donor, and share the same micro-anatomy as the organ from which they were derived.
Thus far, rectal organoids from over 300 subjects have been collected for functional profiling and of those, 65 have been tested for response to PTI’s investigational drugs.
Early results support the initiation of enrollment of responding subjects into HIT-CF’s clinical trial known as “CHOICES” (Crossover trial based on Human Organoid Individual response in CF – Efficacy Study), which is designed to evaluate the translation of organoid ex-vivo response to potential clinical benefit, such as changes in FEV1 and sweat chloride.
CHOICES should launch in mid-2020 with initial data expected by year-end.
Results from the HIT-CF project to date will be presented at the Keystone Symposia on Tissue Organoids taking place on January 19-23, 2020 in Vancouver, BC, Canada.
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