Exhibit 99.1
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Proteostasis Therapeutics to Initiate First Ever Personalized Medicine-Based Clinical Trial, CHOICES, in European CF Patients with Genotypes Ineligible for Approved CFTR Modulators
OngoingEx-Vivo Study Supports Initiation of Clinical Trial in 2020 with Potential to Serve as the Basis for an MAA in 2021
BOSTON, Mass. – December 11, 2019 – Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF), today announced positive, initialex-vivo results of PTI’s proprietary cystic fibrosis transmembrane conductance regulator (CFTR) modulators,PTI-801,PTI-808, andPTI-428, in individuals with CF who are ineligible for the current standard of care CFTR modulator therapies due to their genotype. The data are part of apan-European strategic initiative, known asHIT-CF (Human Individualized Therapy of CF), which seeks to accelerate the development of, and access to, personalized therapies for CF patients, beginning with those for whom no currently approved CFTR modulator therapy is indicated.
HIT-CF is sponsored by the European Commission Horizon 2020 program, in whichCF-Europe, a patient organization representing more than 48,000 individuals with CF, collaborates and with the European Cystic Fibrosis Society Clinical Trial Network(ECFS-CTN), which is recruiting adult CF patients into theex- vivostudy through its 43 clinical trial centers.HIT-CF collects tissue samples from CF patients 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.
As of today, 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 intoHIT-CF’s clinical trial known as “CHOICES” (Crossover trial based onHumanOrganoidIndividual response inCF -EfficacyStudy), which is designed to evaluate the translation of organoidex-vivo response to potential clinical benefit, such as changes in FEV1 and sweat chloride. CHOICES, which is expected to initiate inmid-2020, will be the first ever personalized medicine-based study in CF, with initial data expected by the end of 2020. Fully funded by theHIT-CF, this trial is a placebo controlled, double blind, crossover study with an8-week treatment period and 6 months of uninterrupted dosing. The results may serve as the basis for a potential Marketing Authorization Application with the European Medicines Agency (EMA) in 2021 through a novel regulatory pathway which is being pursued jointly by Proteostasis andHIT-CF. The CHOICES clinical study is part of PTI’s broader clinical development strategy for its CFTR modulator candidates that is already separately funded for the common genotypes.
Results from theHIT-CF project to date will be presented at the Keystone Symposia on Tissue Organoids titled “Tissue Organoids as Models of Host Physiology and Pathophysiology of Disease (J1)” taking place on January19-23, 2020 in Vancouver, BC, Canada.
“Proteostasis is honored to have been invited to participate in theHIT-CF project and is the only company in the group with a combination of novel CFTR modulators being testedex-vivo. We are very enthusiastic about the progress of the study,” said Geoffrey Gilmartin, M.D., M.M.Sc., Chief Medical Officer of Proteostasis Therapeutics. “In Europe alone, there are