This morning, clinical-stage biopharmaceutical company Proteostasis Therapeutics Inc. (PTI:NASDAQ), which is focused on the discovery and development of therapies to treat cystic fibrosis (CF), announced "positive, initial ex-vivo results of PTI's proprietary cystic fibrosis transmembrane conductance regulator (CFTR) modulators, PTI-801, PTI-808 and PTI-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 a pan-European strategic initiative, known as HIT-CF (Human Individualized Therapy of CF)."
The HIT-CF Europe research project's goal is to provide better treatment and improve the lives of people with CF and rare mutations. In the project, drug candidates are first tested on patient-derived organoids in qualified laboratories across Europe that are part of the European Cystic Fibrosis Society Clinical Trial Network. Based on the test results, a smaller group of patients will be invited to participate in a clinical trial with one or more investigational molecules from a participating pharmaceutical company.
The company explains in the report that organoids are cell cultures made from stem cells that grow in a culture dish and look similar to the organ from which they are derived. The firm advised that investigational drugs that target the basic defect of CF may have a positive effect and can be used in an organoid system to evaluate rare mutations.
According to the report, "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, which is designed to evaluate the translation of organoid ex-vivo response to potential clinical benefit." The CHOICES study is expected to commence in mid-2020 with initial data findings expected by year-end 2020. The firm noted that the trial will be the first ever personalized medicine-based study in CF. The firm is optimistic that the results may serve as the basis for a potential Marketing Authorization Application with the European Medicines Agency in 2021.
The company's Chief Medical Officer Geoffrey Gilmartin, M.D., M.M.Sc., commented, "Proteostasis is honored to have been invited to participate in the HIT-CF project and is the only company in the group with a combination of novel CFTR modulators being tested ex-vivo...In Europe alone, there are more than 2,300 adult patients whose genotypes render them ineligible for approved CFTR modulators and exclude them from participating in clinical trials with this drug class. This project's proposed personalized medicine approach is paving a potential new way to develop and provide access to novel CFTR modulators for patients with the most dire need for treatment options that target the cause of the disease. Additionally, based on an individual patient's disease phenotype and not just the genetic designation, this approach could also create a new path towards more effective treatment for all people with CF."
Christiane De Boeck, work package leader at HIT-CF and former president of ECFS, added, "The inequality in access to CFTR modulators is an acute problem across Europe where 1 in 5 individuals do not have a F508del mutation...At HIT-CF Europe, we believe that novel strategies such as personalized medicine and development of new treatment options are central to addressing the inequality of access across the continent. We are thrilled with these initial results and look forward to providing additional updates."
Proteostasis Therapeutics, headquartered in Boston, Mass., is a clinical stage biopharmaceutical company developing novel small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. The firm focuses more specifically on identifying therapies that restore protein function.
Proteostasis has a market cap of about $203.0 million with approximately 51.13 million shares outstanding and a short interest around 5.7%. The stock has a 52-week price range of $0.61-5.80. PTI shares opened today at $4.30 (+$0.33, +8.31%) over yesterday's $3.97 closing price. The stock has traded today between $4.105 and $4.72 per share and at present is trading at $4.295 (+$0.325, +8.19%).
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