- An oral presentation on the final results from the proof-of-concept (PoC) nasal potential difference (NPD) trial will be given by
Steve Rowe, MD at the European Cystic Fibrosis Society(ECFS) conference.
- Preliminary data from the Phase 1b study, PQ-010-001 single ascending dose (SAD) cohorts of the ongoing Phase 1b will be presented in a poster, demonstrating single dose safety and evidence of systemic exposure following administration via inhalation in CF patients.
- Cohort 7 is completed and enrollment of the final cohort in the Phase 1b study, PQ-010-001, is expected to be completed in
- Topline safety and exploratory efficacy data from the multiple dose cohorts in the Phase 1b trial are expected to be released in
Oral presentation on
Poster presentation on
The Company will also present a poster titled: “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation [Poster #40]” during the session “Cell Biology/Physiology/New Therapies” on Friday
“QR-010 is an innovative approach to restoring CFTR function in patients with CF due to the F508del mutation. Last year, we demonstrated that QR-010 restores CFTR function as measured by a very specific assay, the nasal potential difference. Now we have shown that QR-010 can be detected in the blood following a single dose inhalation. We believe these results support the potential that QR-010 can treat all manifestations of CF,” said
QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The F508del mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (
About Cystic Fibrosis
Cystic fibrosis (CF) is the most common fatal inherited disease in the Western world and affects an estimated 65,000 patients worldwide. In people with CF, a defective CFTR gene causes a thick, buildup of mucus in the lungs, pancreas and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage and eventually, respiratory failure. There is no cure for CF. Disease manifestations lead to a shortened life expectancy with a median age of death of 27 years. Although over 1,900 CF-causing gene mutations have been identified, approximately 70% of all CF patients are affected by the F508del mutation. Among all CF patients, approximately 50% are homozygous for the F508del mutation.
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-010, our ongoing and planned discovery and development of QR-010 and its therapeutic potential, timing of enrollment and results from our clinical trials, and statements regarding the coverage of our patent portfolio, owned and in-licensed, including the duration of patent coverage. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the
Smital ShahChief Financial Officer T: +1 415 231 6431 firstname.lastname@example.org