|Over a week ago|
ProQR Therapeutics expects cash runwat to fund operations into 2H22 08:3805/0705/07/20
ProQR Therapeutics announces program updates » 08:3705/0705/07/20
Ongoing clinical studies…
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: 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% 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: The Phase 1/2 Aurora trial is ongoing with initial data on track for 2021. QR-504a for Fuchs Endothelial Corneal Dystrophy: 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.
ProQR Therapeutics reports Q1 EPS (EUR0.32) vs. (EUR0.36) last year » 08:3605/0705/07/20
"At the end of the…
"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."
|Over a month ago|
H.C. Wainwright cautiously optimistic after ProQR' STELLAR trial interim data » 10:4803/3103/31/20
H.C. Wainwright analyst…
H.C. Wainwright analyst Andrew Fein notes that ProQR Therapeutics released interim data from the Phase 1/2 STELLAR trial assessing the efficacy and safety of QR-421a in Usher Syndrome and non-syndromic Retinitis pigmentosa. The analyst remains cautiously optimistic due to the limited patient numbers and reported efficacy data. Given that the limited efficacy seen was on the lower end of the dose scale, he remains hopeful for improved efficacy at the higher doses. Safety data also bodes well for future read-outs, he adds. Fein has a Buy rating and a $20 price target on the shares.
ProQR Therapeutics to host conference call » 08:0203/3103/31/20
Management discusses positive findings from an interim analysis in the Phase 1/2 Trial of QR-421a in Usher Syndrome and non-syndromic Retinitis Pigmentosa (nsRP), as well as an update on business operations in relation to the COVID-19 pandemic, on a conference call to be held on March 31 at 8:15 am. Webcast Link
ProQR Therapeutics to host conference call » 07:5503/3103/31/20
Conference call to…
ProQR Therapeutics announces budget review process » 06:0903/3103/31/20
The COVID-19 pandemic is…
The COVID-19 pandemic is rapidly evolving and has prompted global health concerns, the duration, severity and exact impact of which are currently unknown and cannot be predicted with confidence. In consultation with the trial sites, due to the COVID-19 pandemic the Company also expects a delay in all of its ongoing and scheduled trials, including the pivotal trial of sepofarsen for Leber congenital amaurosis 10. For the trials of QR-421a and QR-1123, patients have already been identified for the next dose cohorts and the Company expects to begin dosing as soon as practical after clinical sites are ready and able to do so. This will be the same for the start of the clinical trial for QR-504a. The Company currently does not believe that its supply chain will be affected. Due to the COVID-19-related delays, the Company has undertaken a budget review process and now anticipates its cash runway will fund operations into the second half of 2022.
ProQR Therapeutics announces findings from Phase 1/2 Stellar trial » 06:0703/3103/31/20
ProQR Therapeutics announces positive findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A exon 13 mutations. "The goal of the interim analysis of this 24 month Stellar trial of QR-421a was to assess safety and early signs of efficacy for the purpose of informing next steps in development and future trial strategy," said the company. "We are pleased with the current safety profile and are very encouraged by early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% of QR-421a-treated patients thus far in this trial. The findings support continuing the trial as planned, with both cohort expansion and dose escalation in order to identify a potential development path to registration. Importantly, these data represent the second program from our ophthalmology pipeline that is supported by preclinical predictions from human retinal organoids, providing further validation of our translational approach and platform technology." The interim analysis (IA) is based on nine and three month data from the first and second dose cohorts, respectively, of the Stellar Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy. Across both cohorts thus far, QR-421a was observed to be generally well tolerated with no serious adverse events noted. In the six sham treated subjects, outcome measures demonstrated no consistent pattern of response above the "noise" level. In contrast, two of eight QR-421a-treated patients demonstrated benefit across multiple concordant outcome measures.
ProQR Therapeutics to host conference call » 04:5503/3103/31/20
Conference call to…
ProQR Therapeutics to host conference call » 14:0903/2603/26/20
Conference call to…