ProQR Announces First Quarter 2020 Operating and Financial Results
7 May 2020 at 7:00 AM EDT
Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;
Updated data from the Phase 1/2 InSight extension study of sepofarsen, including data from contralateral eye treatment, on track to be reported in H2 2020;
Three clinical stage RNA therapies in development for inherited retinal diseases, with fourth ophthalmic program slated to enter mechanistic proof-of-concept studies;
ProQR anticipates its cash runway will fund operations into H2 2022
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today reported its financial results for the first quarter ended March 31, 2020 and provided a business update.
“At the end of the first quarter we shared positive findings from the interim analysis of the Phase 1/2 Stellar trial of QR-421a for Usher’s syndrome, which adds to the growing body of evidence further validating the potential of our platform,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Based on these findings, we are continuing the trial as designed, with dose expansion and escalation cohorts planned. We are working closely with our clinical trial sites to monitor the evolving COVID-19 situation and preparing to rapidly ramp up enrollment once it is deemed safe to do so. In the second half of 2020 we look forward to sharing updated data from the Phase 1/2 InSight extension study of sepofarsen for LCA10, including data from the contralateral eye treatment. We are confident in our fundamentals – we have a productive platform, a deep pipeline, and are well capitalized – as we continue our work to bring novel RNA therapies to patients.”
Business Operations and Program Updates
Ongoing clinical studies of sepofarsen for LCA10, QR-421a for Usher syndrome and nsRP, and QR-1123 for adRP are all currently active, but the effects of the COVID-19 pandemic are resulting in disruptions to patient enrollment across these programs. In consultation with clinical trial sites, ProQR is implementing mitigation procedures that support a rapid ramp up in enrollment as soon as the disruption allows, including ongoing patient identification activities, pre-screening, and documentation for additional site activations. Additionally, ProQR is continuing to monitor previously enrolled trial subjects. The impact of COVID-19 continues to be a dynamic and evolving situation.
Sepofarsen, lead clinical candidate for Leber congenital amaurosis 10 (LCA10):
Based on COVID-19-related disruptions at clinical trial sites, the enrollment timeline for the pivotal Phase 2/3 Illuminate trial is delayed. Additional site activation and patient pre-screening activities are ongoing, which are designed to enable rapid ramp up in enrollment once the trial sites are able to dose patients.
Updated data from the Phase 1/2 InSight extension study of sepofarsen for LCA10, including data from contralateral eye treatment, are on track to be reported in H2 2020.
QR-421a for Usher’s syndrome and non-syndromic retinitis pigmentosa:
In March, the Company reported interim analysis findings from the Phase 1/2 Stellar trial of QR-421a in patients with Usher syndrome and non-syndromic retinitis pigmentosa, or nsRP. The data demonstrated that thus far, QR-421a is generally well tolerated with no serious adverse events noted. There were early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% (2 of 8) of patients in the trial, which support continuing the trial as designed, with both cohort expansion and dose escalation planned.
QR-1123 for autosomal dominant retinitis pigmentosa (adRP):
The Phase 1/2 Aurora trial is ongoing with initial data on track for 2021.
QR-504a for Fuchs Endothelial Corneal Dystrophy (FECD):
QR-504a is expected to be the Company's next pipeline candidate to enter clinical development for patients with FECD type 3 who are scheduled for corneal transplant.
My Retina Tracker Program:
In February, ProQR announced its participation in the Foundation Fighting Blindness “My Retina Tracker Program”, a collaborative, open access program in the United States providing no-cost genetic testing and genetic counseling for individuals with a clinical diagnosis of an inherited retinal disease, such as LCA and Usher syndrome.