The ProQR family is growing fast and we spoke to the Chief Medical Officer Aniz Girach who joined ProQR in 2019. We asked him about his impression of the company after he joined, as well as the new developments he’s been working on.
“I am an ophthalmologist by training, and also have experience with surgery. But for the last twenty-four years I have worked at various pharmaceutical companies. Big pharma, like Lilly and Merck, but also small biotech startups. The last company I worked at was Nightstar Therapeutics, which was exploring gene therapy for inherited retinal diseases. And now since 2019 I have been Chief Medical Officer at ProQR. I have felt part of the furniture very quickly!”
“First of all, the company stood out with some really interesting data and an excellent pipeline, and when I met some of the people it just clicked. There was immediate chemistry with the folks I met at ProQR. Part of that feeling comes from my background. As a doctor, my priority has always been with the patient, and I recognized that same priority here. Most pharmaceutical companies will “talk the talk”, but they don’t “walk the walk”. ProQR does “talk the talk”, but it also “walks the walk”. The employees that work here have that drive to stop people from going blind – it’s what makes them get out of bed in the morning. I’m convinced that this patient-focused approach is essential to achieve success, and I think it’s the result of the Dutch egalitarian culture as well as (our CEO) Daniel’s influence in the company. In any case: it feels like home.”
As a doctor, my priority has always been with the patient
“If you look at IRDs, there is really only one therapy that is approved by the regulators, and it’s for a very small segment of IRDs. But most IRDs are terrible and debilitating diseases that cause blindness, so there is a huge opportunity there. Most patients go blind at a young age too, so a treatment would really help the whole family, including any caregivers. Of course, it’s a business opportunity too, but I think RNA therapies have a big role to play here.”
“Well, coming from the gene therapy world I especially see the benefits of RNA therapy over gene therapy. For starters, with RNA we can target a disease at a much earlier stage than with gene therapy, which is a big win if you look at how the disease progresses. Then there is the surgery involved. RNA therapy is a simple, easily-administered injection in the back of the eye, without any general anesthesia. But with gene therapy, you have to do this very complicated injection underneath the retina at the back of the eye. This does require the use of general anesthesia, and it actually only targets the central area of the macula. What that means is that you will only save the central part of the vision and you basically still wait for the more peripheral vision to get worse as the disease progresses. In contrast, with the administration of RNA therapy in the vitreous liquid of the eye, potentially the entire retina is targeted.”
“We have just finished the Phase 1/2 trial of sepofarsen and we’re happy that the first study delivered truly positive results on all three of the important endpoints. The three endpoints we looked at were visual acuity, full field stimulus threshold and a mobility course. We published the preliminary data after three months in Nature Medicine, and these data were very predictive of the rest of the 12-month trial. The final results are encouraging, as we have not only successfully tested safety and dose of the therapy, but we also found efficacy for all three of the endpoints. We are now recruiting for the Phase 2/3 trial, which should replicate what we already found in the first trial.”
“This is really the most striking thing. Most of these patients are either blind or at some stage towards being blind. We included patients between 8 and 44 years old, and the average improvement in visual acuity, which is the standard test with the lines of letters in different sizes on an eye chart, was 5.5 lines. One patient even managed to improve approximately nine lines in this test! He came in at the beginning, only able to distinguish light from dark, and he called his doctor at some point saying that he was navigating an airport by himself because he could read the signs. This is of course doing wonders for the patient’s autonomy and self-confidence. It is the difference between being able to live a relatively independent life or not, between being able to read or not. And between being able to see your grandkids or not. Of course, we still need to confirm these results in the Phase 2/3 trial, but it has been really encouraging. Most patients would already be happy if we could halt progression of the disease, not even improvement. Which is in fact more normal in medicine; most therapies don’t cure but just slow down progression.”
[These results are] the difference between being able to... see your grandkids or not.
Find out more about sepofarsen and the ongoing Phase 2/3 clinical trial for Leber congenital amaurosis.
