ProQR soon to announce proof-of-concept results for novel cystic fibrosis treatment

‘We will present top-level results from clinical proof-of-concept study, PQ-010-002 later this year during the North American Cystic Fibrosis Conference (NACFC),’ said Daniel de Boer, ProQR’s chief executive officer, in a recent press release.

Preliminary data from the second clinical trial – PQ-010-001 – may also be available at NACFC.

Pre-clinical data was already presented at the European Cystic Fibrosis Conference (ECFS) in June, in which QR-010 provoked ‘an unprecedented restoration of CFTR [Cystic Fibrosis Transmembrane conductance Regulator] function in the NPD [Nasal Potential Difference] test’. The ∆F508 mutation is known to delete three of the CFTR gene’s coding base pairs, resulting in the production of a misfolded and improperly functioning CFTR protein.

For CF patients, this restoration of CFTR means a potential stop of CF progression. The treatment is rather light too, as QR-010 can simply be self-administered by means of a handheld aerosol device and directly into the patient’s lungs. The molecule has been granted orphan drug designation in the United States and the European Union. The EU also co-funds its development through the H2020 PRO-CF-MED project.

Two clinical trials

PQ-010-002 and PQ-010-001 are key to the future success of QR-010, and ProQR is currently recruiting CF patients to carry them on.

PQ-010-002, the proof-of-concept study, is evaluating QR-010’s effect on NPD by measuring CFTR protein activity by how efficiently chloride is transported through the nasal cell membranes. The study plans to enrol 16 homozygous and heterozygous CF patients, with the option of enrolling a second 26-patient cohort.

PQ-010-001, on the other hand, is a Phase 1b randomised, double-blind, placebo-controlled and dose-escalation 28-day study across North America and Europe. QR-010 will be administered to 64 homozygous patients by inhalation three times a week for four weeks, with a view to evaluating the safety, tolerability, and pharmacokinetics of single- and multiple-ascending doses of inhaled QR-010. Other exploratory efficacy endpoints include measurements of sweat chloride, weight gain, lung function and the CFQ-R Respiratory Symptom Score.

A bright future

QR-010’s potential had already been acknowledged by the FDA in July, when ProQR received Fast Track designation – a status granted to drugs that are under development for serious conditions and have the potential to fulfil an unmet medical need.

‘We are very pleased with the Fast Track designation,’ de Boar said at the time. ‘It highlights the unmet medical need in cystic fibrosis and the need for innovative and more efficacious medicines for CF. We look forward to working with the FDA to bring QR-010 to patients faster.’

In its latest financial report, the Dutch company revealed that its cash position was EUR 85.5 million as of March 31, sufficient to allow for continued operations until mid-2018.

For more information please see:
ProQR website