Proteostasis Therapeutics, Inc. Presents New Data Demonstrating Potential for Genotype-Agnostic and Combination Therapies for People with Cystic Fibrosis
“We are strongly encouraged by our early findings and the potential implications for improving the lives of people with cystic fibrosis,” said
In the company’s poster presentation, “Characterization of CFTR amplifiers, mutation-agnostic modulators that increase protein levels and complement other CF therapeutic modalities,” company researchers describe a new class of modulator – an amplifier – which increases the levels of immature CFTR protein available for other modulators, such as correctors and potentiators, to act upon.
The study found that amplifiers
- increase CFTR immature protein and stabilize CFTR mRNA;
- increase substrate for additional modulators;
- work across CFTR genotypes; and,
- demonstrate activity in non-lung tissues and in vivo.
The early findings suggest the potential for the novel class of amplifiers to enhance CFTR translation efficiency across CFTR mutations and be used in combinations to boost the activity of additional modulators in treating CF.
In a second poster presentation, company scientists have identified a novel corrector with properties that differentiate it from clinical-stage correctors such as lumacaftor or VX-661. The study, “A novel corrector for F508del-CFTR that complements existing CFTR modulators in vitro,” demonstrated that exposure to Proteostasis Therapeutics’ corrector significantly increases the proportion of mature CFTR protein. Moreover, in vitro CFTR function is increased accordingly when the company’s corrector is dosed with other modulators, including lumacaftor, potentiators and its amplifier.
"We believe we have the potential to dramatically improve therapeutic outcomes for patients with cystic fibrosis," said Po-Shun Lee, M.D., Executive Vice President and Chief Medical Officer of
Following is a schedule of the company’s poster presentations and conference call:
Title: Characterization of CFTR amplifiers, mutation-agnostic modulators that increase protein levels and complement other CF therapeutic modalities
Title: A novel corrector for F508del-CFTR that complements existing CFTR modulators in vitro
Conference Call and Webcast
Time: 4:00 p.m. ET
The conference call can be accessed by dialing 1-877-626-4740 (toll-free domestic) or 1-281-973-6278 (international) and referring to conference ID 78792593. A live webcast and accompanying slide presentation will be available on the Event Calendar page in the Investors & Media section of the company’s website, www.proteostasis.com. A replay of the webcast will be available on the company’s website shortly after the conclusion of the conference call.
About Cystic Fibrosis
Cystic fibrosis (CF) is a life threatening, progressive genetic disease affecting an estimated 70,000-100,000 people worldwide. The disease is caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which acts as a passageway to promote the transport of salt and water to tissues such as the lungs, skin and pancreas. The defect disrupts healthy ion flow and causes a buildup of thick mucus and bacteria in several organs, resulting in persistent lung infections and the inability for the body to break down food and absorb vital nutrients. While advancements in research and treatments have extended the life expectancy for those living with the disease, CF remains a serious, life-limiting condition with no known cure.
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