03-25-2021, 12:39 PM
$PRQR ProQR Announces Positive Results from Clinical Trial of QR-421a in Usher Syndr
<!-- SC_OFF --><div class="md"><p>ProQR announced results from a planned analysis of its <em>Phase</em> 1/2 <em>Stellar</em> <em>trial</em> of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to <em>USH2A</em> exon 13 mutations. In the <em>trial</em>, QR-421a demonstrated benefit on multiple measures of vision that moved in concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a single dose. QR-421a was observed to be well tolerated with no serious adverse events reported. Based on these findings, the Company plans to advance QR-421a to two parallel pivotal <em>Phase</em> 2/3 <em>trial</em>s by year end 2021 – one in early-moderate patients, one in advanced patients.</p> <p>"We're pleased to have met all the objectives we set for the <em>Stellar</em> <em>trial</em>, including determining suitable registration endpoints, the dose, dosing interval, and patient population for the <em>Phase</em> 2/3 pivotal <em>trial</em>s," said Aniz Girach, MD, Chief Medical Officer of ProQR, "With just a single dose, QR-421a demonstrated <em>clinical</em> proof of concept with benefit observed in treated eyes compared to the untreated eyes in multiple concordant measures of vision. As expected, we saw benefits in both advanced and early-moderate patients in this slow progressing, debilitating eye disease, allowing us to advance this important investigational therapy for all patients with Usher syndrome and nsRP due to <em>USH2A</em> exon 13 mutations. Based on preliminary Regulatory guidance, we plan to submit protocols to advance QR-421a to pivotal testing. This is our second program targeting a severe inherited retinal disease that is moving into pivotal <em>trial</em>s, which we believe further validates our RNA therapy platform and our capabilities to design and efficiently take these programs through <em>clinical</em> <em>develop</em>ment."</p> <p>"The safety profile and efficacy findings for QR-421a are very encouraging," said Robert Koenekoop, MD, MSc, PhD, FRCS©, FARVO, a <em>clinical</em>-scientist from the Montreal Children's Hospital and Professor of the McGill University Faculty of Medicine and Department of Pediatric Surgery. "Usher syndrome and non-syndromic retinitis pigmentosa due to <em>USH2A</em> exon 13 mutations are devastating retinal diseases representing a high unmet medical need, as there are no approved therapies to treat the severe vision loss associated with these diseases. Patients' biggest hope for a therapy is to stop disease progression and prevent vision loss, and these findings suggest that QR-421a has the potential to stabilize vision. I look forward to this exciting program advancing into pivotal <em>trial</em> <em>develop</em>ment." </p> </div><!-- SC_ON --> submitted by <a href="https://www.reddit.com/user/freeusa"> /u/freeusa </a> <br/> <span><a href="https://www.reddit.com/r/investing/comments/mccu5i/prqr_proqr_announces_positive_results_from/">[link]</a></span> <span><a href="https://www.reddit.com/r/investing/comments/mccu5i/prqr_proqr_announces_positive_results_from/">[comments]</a></span>Kind Regards R
<!-- SC_OFF --><div class="md"><p>ProQR announced results from a planned analysis of its <em>Phase</em> 1/2 <em>Stellar</em> <em>trial</em> of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to <em>USH2A</em> exon 13 mutations. In the <em>trial</em>, QR-421a demonstrated benefit on multiple measures of vision that moved in concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a single dose. QR-421a was observed to be well tolerated with no serious adverse events reported. Based on these findings, the Company plans to advance QR-421a to two parallel pivotal <em>Phase</em> 2/3 <em>trial</em>s by year end 2021 – one in early-moderate patients, one in advanced patients.</p> <p>"We're pleased to have met all the objectives we set for the <em>Stellar</em> <em>trial</em>, including determining suitable registration endpoints, the dose, dosing interval, and patient population for the <em>Phase</em> 2/3 pivotal <em>trial</em>s," said Aniz Girach, MD, Chief Medical Officer of ProQR, "With just a single dose, QR-421a demonstrated <em>clinical</em> proof of concept with benefit observed in treated eyes compared to the untreated eyes in multiple concordant measures of vision. As expected, we saw benefits in both advanced and early-moderate patients in this slow progressing, debilitating eye disease, allowing us to advance this important investigational therapy for all patients with Usher syndrome and nsRP due to <em>USH2A</em> exon 13 mutations. Based on preliminary Regulatory guidance, we plan to submit protocols to advance QR-421a to pivotal testing. This is our second program targeting a severe inherited retinal disease that is moving into pivotal <em>trial</em>s, which we believe further validates our RNA therapy platform and our capabilities to design and efficiently take these programs through <em>clinical</em> <em>develop</em>ment."</p> <p>"The safety profile and efficacy findings for QR-421a are very encouraging," said Robert Koenekoop, MD, MSc, PhD, FRCS©, FARVO, a <em>clinical</em>-scientist from the Montreal Children's Hospital and Professor of the McGill University Faculty of Medicine and Department of Pediatric Surgery. "Usher syndrome and non-syndromic retinitis pigmentosa due to <em>USH2A</em> exon 13 mutations are devastating retinal diseases representing a high unmet medical need, as there are no approved therapies to treat the severe vision loss associated with these diseases. Patients' biggest hope for a therapy is to stop disease progression and prevent vision loss, and these findings suggest that QR-421a has the potential to stabilize vision. I look forward to this exciting program advancing into pivotal <em>trial</em> <em>develop</em>ment." </p> </div><!-- SC_ON --> submitted by <a href="https://www.reddit.com/user/freeusa"> /u/freeusa </a> <br/> <span><a href="https://www.reddit.com/r/investing/comments/mccu5i/prqr_proqr_announces_positive_results_from/">[link]</a></span> <span><a href="https://www.reddit.com/r/investing/comments/mccu5i/prqr_proqr_announces_positive_results_from/">[comments]</a></span>Kind Regards R