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ProQR Shares Gain 62% on Stellar Results in Phase 1/2 Usher Syndrome Trial

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ProQR Therapeutics shares reached a new 52-week high after the company reported highly positive results from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.

Clinical stage biopharmaceutical company ProQR Therapeutics N.V. (PRQR:NASDAQ), which is focused on the development of transformative RNA based therapies for inherited retinal diseases (IRDs), yesterday announced "results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations."

ProQR Therapeutics stated that in the Phase 1/2 Stellar study, after a single dose of QR-421, patients showed improvement across multiple recognized measures for assessing vision including overall visual acuity, visual fields and optical coherence tomography (OCT) retinal imaging. The company reported that QR-421a was well tolerated in the study and noted that no serious adverse events occurred.

The firm advised that based upon the promising results observed in the Phase 1/2 study, it is proceeding with plans to advance QR-421a to two parallel pivotal Phase 2/3 trials, one for early-moderate patients and the other for advanced patients, by the end of this calendar year.

ProQR Therapeutics' Chief Medical Officer Aniz Girach, M.D., commented, "We're pleased to have met all the objectives we set for the Stellar trial, including determining suitable registration endpoints, the dose, dosing interval, and patient population for the Phase 2/3 pivotal trials...With just a single dose, QR-421a demonstrated clinical 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 USH2A exon 13 mutations."

Robert Koenekoop, M.D., MSc, Ph.D., FRCS(C), FARVO, a clinical-scientist from the Montreal Children's Hospital and Professor of the McGill University Faculty of Medicine and Department of Pediatric Surgery, remarked, "The safety profile and efficacy findings for QR-421a are very encouraging...Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A 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."

The company plans to submit protocols to commence two pivotal Phase 2/3 clinical trials before year-end 2021. The first anticipated Phase 2/3 study is to be named as the "Sirius" trial which will be designed to focus on advanced patients with baseline best corrected visual activity (BCVA) ≤20/40. The study will be structured as a double-blind, placebo-controlled, multiple-dose study over a 24-month period and will enroll about 100 adults with Usher syndrome and nsRP due to v exon 13 mutations, including both homozygous and heterozygous patients. The pre-determined primary endpoint in the study will be BCVA at 18 months and will include two different dosage arms and a placebo control group.

The firm stated that it also plans to conduct a similar Phase 2/3 "Celeste" trial in parallel and simultaneously in early-moderate patients. The Celeste study calls for enrollment of 100 patients to be studied over 24 month with the primary pre-established endpoint of evaluating static perimetry at 18 months. The company explained that "static perimetry assesses visual fields and retinal sensitivity in the peripheral retina."

Benjamin R. Yerxa, Ph.D., CEO at the Foundation Fighting Blindness, stated, "There are currently no available treatments for the more than 16,000 patients with Usher syndrome 2A and nsRP due to USH2A exon 13 mutations and we are excited about the potential for QR-421a to address this significant unmet need...We are pleased to see QR-421a advancing to pivotal testing and proud to support the work of ProQR as they advance their pipeline of RNA therapies to potentially help children, adults, and families who are affected by blindness caused by USH2A mutations and other rare inherited retinal diseases."

The firm stated that the Phase 1/2 Stellar clinical trial of QR-421a included a total of 20 patients, of which 14 received a single dose of QR-421a and six were given a single sham procedure for masking. The 14 patients who were actually treated with QR-421a varied in their stage of disease with six being classified as advanced and eight categorized as early-moderate patients.

The company listed that Usher syndrome is the leading cause of combined deafness and blindness and that added that those afflicted with Usher syndrome type 2a are typically born with hearing loss and experience progressive vision loss in adulthood. The firm said that "vision loss can also occur without hearing loss in a related disease called non-syndromic retinitis pigmentosa." The company pointed out that these conditions can frequently be caused by mutations in the USH2A gene and advised that currently there are no approved pharmaceutical treatments for vision loss arising from USH2A mutations.

The company stated that QR-421a is an investigational RNA therapy that is designed to restore functional usherin protein and is intended to be administered through intravitreal injections in the eye.

ProQR Therapeutics is a clinical stage biopharmaceutical company headquartered in the city of Leiden in the Netherlands. The company concentrates its development efforts on creating transformative RNA therapies for use in treating severe genetic rare diseases including Leber congenital amaurosis (LCA) and inherited retinal diseases such as Usher syndrome and retinitis pigmentosa (RP).

ProQR Therapeutics NV began the day with a market capitalization of around $284.8 million with approximately 50.14 million shares outstanding and a short interest of about 2.3%. PRQR shares opened 2% higher today at $5.81 (+$0.13, +2.29%) over yesterday's $5.68 closing price and reached a new 52-week high this morning of $9.4394. The stock has traded today between $5.80 to $9.4394 per share and closed at $9.22 (+$3.54, +62.32%).


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