PTI-428, PTI-801 and PTI-808 Testing Underway in Organoids from CF Patients, with Potential for Clinical Data in 2020
HIT CF Project Funded by the European Union's Horizon 2020 Research and Innovation Programme
BOSTON, Feb. 27, 2019 /PRNewswire/ -- Proteostasis Therapeutics, Inc. (PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced it has been invited to join the HIT-CF Europe, a pan European strategic initiative which seeks to validate a personalized therapy approach for CF patients with extremely rare genetic mutations.
HIT-CF is a consortium of researchers, doctors, pharmaceutical companies and patient representatives, which aims to provide better treatment for people with CF. The project collects tissue samples from CF patients with diverse genetic profiles and develops patient-derived organoids, cell cultures that are genetically similar to the organ from which they are derived. The organoids are profiled in vitro via their response to drug candidates from the participating pharmaceutical companies. Based on the functional response, patients may be invited to participate in clinical trials with the investigational drugs to validate the ability of the in vitro response to predict clinical benefit. This project is fully funded by a €6.7M award from the Horizon 2020 EU funding program.
Unlike HBE (human bronchial epithelial) cells, which are currently derived from lungs that have been removed from patients, organoids can be more easily derived from a minimally invasive biopsy sample from any patient and used in experimental assays. Organoids can provide valuable insights on the donors including their likelihood of achieving improvements in pulmonary function and reductions in sweat chloride concentration with CFTR modulators based on the in vitro response to those drugs1. Additionally, HIT CF will explore novel regulatory pathways with the European Medicines Agency (EMA) to facilitate patient access to CFTR modulators based on organoid response.
"The ultimate goal of HIT-CF Europe is to develop a path for access to therapies for individual patients or patient groups who show a positive response to the therapy in an organoid test and pave the way for personalized medicine," said Prof. Dr. C.K. Van der Ent, Program Leader of the HIT-CF consortium. "We are thrilled to welcome Proteostasis to our consortium. With PTI's three novel CFTR modulators in advanced clinical trials and exciting early results in the organoid system, we are able to expand testing of the potential therapeutic options in the in vitro system and clinical trials. Through the European Cystic Fibrosis Society (ECFS) networks, which can access 34 clinical research centers across 12 countries, we have already collected biopsies from CF patients and are looking forward to expanding the biobank with samples from more than 500 patients across Europe."
"We are honored to be selected to participate in the HIT-CF Europe consortium and join forces with key thought leaders to explore the potential of a personalized medicine approach as a possible new frontier for CF therapy," said Meenu Chhabra, President and Chief Executive Officer of Proteostasis. "With over 2,000 mutations in the CFTR gene identified today, the current "one pill for all" approach is limited in its ability to reach all people with CF who might benefit from disease modifying CFTR modulators. The biology of CF tells us that the CFTR genotype is only one of the many factors that impact the disease phenotype and response to therapy. It is our responsibility to explore new drug development and access avenues that bridge the existing gap," said Ms. Chhabra.
Organoids are cell cultures that grow in a culture dish and look similar to the organ from which they are derived. Because organoids are made from stem cells, they contain the same mutations as the person from whom the biopsies are derived. Investigational drugs which target the basic defect of CF, will be used in the organoid system to evaluate rare mutations where the drugs may have a positive effect.
About HIT-CF Europe
HIT-CF Europe is a research project which aims to provide better treatment and better lives for people with cystic fibrosis (CF) and rare mutations. To achieve this, drug candidates of several companies are first tested in the laboratory on patient-derived organoids. Subsequently, based on the reaction in the organoids, a smaller group of patients will be assigned to a clinical trial with one or more investigational molecules from a participating pharmaceutical company.
All participating centers are part of the European Cystic Fibrosis Society – Clinical Trial Network (ECFS-CTN). The project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement No 755021. For more information, visit www.hitcf.org.
About Horizon 2020
Horizon 2020 is the biggest EU Research and Innovation programme ever with nearly €80 billion of funding available over 7 years (2014 to 2020) – in addition to the private investment that this money will attract. It promises more breakthroughs, discoveries and world-firsts by taking great ideas from the lab to the market.
About Proteostasis Therapeutics, Inc.
Proteostasis Therapeutics, Inc. is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. Headquartered in Boston, MA, the Proteostasis Therapeutics team focuses on identifying therapies that restore protein function. For more information, visit www.proteostasis.com.
To the extent that statements in this release are not historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "aim," "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements made in this release include, without limitation, statements regarding the potential of the consortium to collect, develop and test a sufficient number of patient-derived organoids, the potential of the consortium to validate a personalized therapy approach for clinical trials and therapy for CF patients with extremely rare and diverse genetic mutations, the testing of one or more of the drug candidates, including our CFTR modulators, may lead to a clinical trial in patients, the potential of the consortium to be able to maintain the provided funding, and that HIT CF will explore novel regulatory pathways with the EMA or will be able to facilitate patient access to investigational CFTR modulator therapies. Forward-looking statements made in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the possibility that the potential of our drug candidates in treating CF patients with extremely rare genetic mutations may not be realized, final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported by the Company or consortium, uncertainties inherent in the execution and completion of clinical trials, in the enrollment of CF patients in our or the consortium clinical trials in a competitive clinical environment, and those set forth in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2018 and our other SEC filings. We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
1 Berkers et al, Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis Cell Reports 26, 1701–1708, February 12, 2019