QR-010 for cystic fibrosis

ProQR is developing an investigational product, QR-010 for cystic fibrosis (CF) patients that suffer from the F508del mutation. F508del is the most common mutation in CF and affects 85% 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 the disease.

Two global clinical trials of 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 part of Study 001 were completed and data were presented during the North American CF Conference (NACFC) in October 2016. The MAD part of Study 001 has completed enrollment and top-line data will be announced in September 2017. Full data from the study are expected to be presented during the North American CF Conference (NACFC) in November 2017.

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.


QR-010 has received orphan drug designation from the U.S. Food and Drug Administration and European Medicines Agency. 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 CF patients is underway in several countries around the world. Study 002 and the SAD part of Study 001 have been completed. Study 001 is ongoing but all patients have been included in the trial. Study 001 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.

About CF

What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic disease affecting over 75,000 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 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 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.

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 patients 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 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 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, founded in 2012 to develop a life changing RNA therapy for CF patients and patients living with 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 people with CF, 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 cause of the disease 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 laboratory and in a first clinical trial in CF patients.
When and where will QR-010 be tested?
Our clinical trials of QR-010 in CF patients with the F508del mutation are fully enrolled, and one trial (Study 002) was completed in 2016. 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 signals for therapeutic effects could also be observed during this trial. Information about our studies can be found here.
Can I participate in the trial?
Ongoing QR-010 clinical trials are fully enrolled. 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 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 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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