QR-010 for cystic fibrosis

ProQR is developing an investigational product, QR-010 for people with cystic fibrosis (CF) that have the F508del mutation. F508del is the most common mutation in CF and affects more than 85% of all CF patients. Despite significant efforts in drug development, people with CF still have a limited life expectancy.

QR-010 is being developed as a regularly inhaled therapy. Unlike any other CF drug currently on the market, it is designed to bind specifically to the defective CFTR RNA and to restore the function of the CFTR protein. With this we aim to treat the underlying cause of the disease and stop the progression of cystic fibrosis.

Two global clinical trials for QR-010 in people with CF have been completed.Study 001, a Phase 1b safety and tolerability clinical trial in 70 people with CF due to two copies of the F508del mutation (homozygotes) and Study 002, a proof of concept clinical trial in 18 people with CF due to one or two copies of the F508del mutation (compound heterozygotes and homozygotes).

In both clinical trials QR-010 was observed to be safe and well-tolerated and both trials showed encouraging signals that QR-010 has the potential to be a meaningful therapy for people with CF that have two copies of the F508del mutation (homozygotes).

Read the interview with Daniel de Boer and Noreen Henig on the QR-010 development program and our patient centric approach.

A more detailed explanation of the preliminary top-line results were published in press releases on September 25, 2017 (results for Study 001) and October 27, 2016 (results for Study 002).


QR-010 has received orphan drug designation from the U.S. Food and Drug Administration and European Medicines Agency and was granted fast-track status by the U.S. Food and Drug Administration. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 633545.

Clinical trials & participation

The first clinical trials of QR-010 were completed with encouraging results and currently there are no trials ongoing for QR-010. The first trials were necessary to move to the next phase of trials designed to evaluate the safety and efficacy of an investigational drug over a longer duration. Please check this website as we will post updates on our plans for further clinical trials here.

About CF

What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic disease affecting over 75,000 people worldwide. People with CF have viscous mucus accumulating in their vital organs. The mucus clogs tubes and organs thereby disrupting several processes in the body.

Pancreatic enzymes, necessary to digest food, are blocked from entering the intestines, so people with CF are not able to digest food by themselves. The thick layer of mucus in the lungs is a great environment for destructive bacteria. The thick mucus makes it hard to clear the lungs from these bacteria by coughing and results in regular infections and inflammation. This process injures the lungs and leads to frequent hospitalizations and lung failure and death at an average age of 30 years.
What causes cystic fibrosis?
CF is caused by mutations (or mistakes) in a gene that is present in the DNA. This gene is responsible for expression of a protein called CFTR (CF transmembrane conductance regulator). As a result of the mutation, CFTR protein has no or limited function in people with CF. The lack of CFTR function results in reduced chloride transport that causes thick and sticky mucus in several vital organs in the body.

CF mainly affects Caucasians in North America, Europe and Australia. In several regions of the world CF is now part of newborn screening programs and people are diagnosed early in life. There are many CF mutations known, the F508del mutation is the most common mutation which affects 85% of all CF patients.
Treatment
CF is treated with a number of palliative medicines that target symptoms of the disease, varying from pancreatic enzymes and vitamins to mucus thinners and antibiotics. People with CF spend a significant amount of their day taking their medicines, which interfere with daily activities. For some people with specific CF mutations, there are other therapies available that treat the underlying cause of the disease. For many patients however, more efficacious medicines are needed because a substantial unmet medical need remains.

For more information see the website of the Cystic Fibrosis Foundation.

FAQ

Who is ProQR?
ProQR Therapeutics is a European company headquartered in Leiden, the Netherlands, founded in 2012 to develop a life changing RNA therapy for people with CF and other severe genetic disorders.
What is QR-010?
QR-010 is an experimental medicine that is designed to treat people with CF that have at least one copy of the F508del mutation. In 85% percent of CF patients, the disease is caused by the F508del mutation in the CFTR gene. QR-010 is designed to target the underlying cause of the disease, aiming to restore the function of the CFTR protein and stop progression of the disease. QR-010 has shown promising results in the laboratory and in the first clinical trials in CF patients.
When and where will QR-010 be tested?
The first clinical trials of QR-010 were completed and there are currently no trials ongoing. If you are interested in participating in potential future QR-010 clinical trials, please check this website as we will post updates on our plans here.
How do I help by participating in clinical trials?
Without clinical trials, life-saving therapies could never be approved and available to people around the world. We gain valuable information from our trials, which helps us make the best and safest medicines possible.
Can I stay on treatment after I participate in a trial?
The benefit of QR-010 therapy needs to be proven in clinical trials before anyone can take it as a treatment.
Can I get QR-010 in a compassionate use program?
QR-010 is currently not available for compassionate use.
I have another CF mutation than F508del, will QR-010 work for me?
QR-010 is specifically designed to target the F508del mutation and is not expected to work in other mutations. ProQR is passionate about CF and is in discovery phase looking at several other genetic mutations that we can possibly develop treatments for in the future similar to QR-010.

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