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QR-010 for cystic fibrosis

ProQR is developing an investigational product, QR-010 for cystic fibrosis patients that suffer from the F508del mutation. F508del is the most common mutation in cystic fibrosis and affects more than 70% of all CF patients. Despite significant efforts in drug development, CF patients 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 stop the progression of cystic fibrosis.

Two global clinical trials for QR-010 in CF patients were started in 2015: a Phase 1b single-ascending dose (SAD) and multiple ascending dose (MAD) study in 64 patients (Study 001) and a proof of concept study in 16 CF patients (Study 002). Study 002 and the SAD portion of Study 001 were completed and data were presented during the North American CF Conference (NACFC) in October 2016.

Read the interview with Daniel de Boer and Noreen Henig on the QR-010 development program and the initial results of the first clinical trials.

A more detailed explanation of the initial trial results was published in a press release on October 27, 2016.


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

Testing of QR-010 in patients is underway in several countries around the world. The first trial has now been completed. A second trial is ongoing. This phase 1b clinical trial is necessary to evaluate the safety and tolerability of QR-010. We will also be looking for the first signs of patient benefit from QR-010. Phase 1 trials are necessary to progress to other types of trials designed to evaluate the safety and efficacy of an investigational drug over a longer duration.

Hospitals in Belgium, Canada, Czech Republic, Denmark, France, Germany, Italy, United Kingdom, Spain and the USA are participating in our ongoing Phase 1b clinical trial. To take part in this clinical trial, participants must be diagnosed with cystic fibrosis and have two copies (homozygous) of the F508del mutation.

About CF

What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic disease affecting more than 70,000 patients worldwide. CF patients 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 CF patients 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 common inflammation. This process destroys the lungs and leads to frequent hospitalizations and lung failure and death at an average age of 27 years.
What causes cystic fibrosis?
CF is caused by mutations (or defects) 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 CF patients. The lack of CFTR function results in reduced chloride transport that causes thick and sticky mucus in several vital organs in the body.

One in every 3600 babies is born with CF, mainly Caucasians in USA, EU and Australia. In several parts of the world CF is now part of newborn screening programs and patients get diagnosed early in life. There are many CF mutations known, the F508del mutation is the most common mutation which affects 70% of all CF patients.
Treatment
CF patients are treated with a number of palliative medicines that target symptoms of the disease, varying from pancreatic enzymes and vitamins to mucus thinners and antibiotics. Patients spend a significant amount of their day taking their medicines, which interfere with daily activities. For some patients with specific CF mutations, there are other therapies available that aim to treat the basic defect 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, founded in 2012 to develop a life changing RNA therapy for cystic fibrosis patients and patients suffering from other severe genetic disorders.
What is QR-010?
QR-010 is an experimental medicine that is designed to treat people with cystic fibrosis that have at least one copy of the F508del mutation. In seventy percent of people with cystic fibrosis, the disease is caused by the F508del mutation in the CFTR gene. QR-010 is a unique medicine. It is designed to target the underlying defect in patients, aiming to restore the function of the CFTR protein and stop progression of the disease. QR-010 has shown promising results in the lab and in a first clinical trial in CF patients.
When and where will QR-010 be tested?
One clinical trial of QR-010 in CF patients with the F508del mutation was completed, a second trial is currently ongoing. As with every trial that tests a new medicine in patients for the first time, the primary goal of the ongoing trial is to confirm that the drug is safe. Potential therapeutic effects could also be observed during this trial. Trials of QR-010 run in different parts of the world. Information about our studies can be found here.
Can I participate in the trial?
If you have cystic fibrosis with two copies of the F508del mutation, you may be eligible to participate in our clinical trial of QR-010 that is currently ongoing. Please speak to your doctor about whether participation in the trial is right for you. Detailed information about the trial can be found here.
How do I help by participating in the trial?
Without clinical trials, life-saving therapies could never be approved and available to patients around the world. We gain valuable information from our trials, which help 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 will not 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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