ProQR Therapeutics N.V., a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, announced results for the third quarter of 2019.
“During the third quarter we made great progress towards our mission to bring novel RNA therapies to patients and we recently strengthened our capital position,” said Daniel A. de Boer, chief executive officer of ProQR. “In October we announced that LCA10 patients experienced a significant improvement in vision after treatment with sepofarsen in a Phase 1/2 trial. These results strengthen our confidence in the design of the ongoing pivotal Illuminate Phase 2/3 trial and our broader inherited retinal diseases pipeline. The Phase 1/2 trial of QR-421a in Usher syndrome is on track to deliver first clinical data in Q1 2020 and we are about to dose a first patient in a Phase 1/2 trial of QR-1123 for adRP. We strengthened our financial position with a public offering in October that will allow us to fund operations into 2022, well beyond the expected top-line readout of Illuminate.”
Corporate Highlights and Business Update
Sepofarsen (QR-110) for Leber’s congenital amaurosis 10 (LCA10)
- Presented positive top-line results from the Phase 1/2 clinical trial. In the trial, sepofarsen was observed to be well tolerated with rapid, significant and durable improvements in vision observed at month twelve. The target registration dose for the ongoing Phase 2/3 Illuminate trial showed a favorable benefit/risk profile in the Phase 1/2 trial.
- The top-line results from the Phase 1/2 trial support confidence in the design of the ongoing Phase 2/3 Illuminate trial that could be the sole registration trial for the program. Top-line data from Illuminate are expected during the first half of 2021.
- Received Rare Pediatric Disease designation from the Food and Drug Administration (FDA) for the treatment of LCA10.
QR-421a for Usher syndrome type 2
- The Phase 1/2 Stellar trial of QR-421a in patients with Usher syndrome type 2 is ongoing and on track to deliver interim data during the first quarter of 2020.
QR-1123 for autosomal dominant retinitis pigmentosa (adRP)
- The FDA cleared the Investigational New Drug (IND) application to start a first-in-human clinical trial in patients with adRP. ProQR plans to start enrolling patients in the Phase 1/2 Aurora trial in 2019.
Received Fast Track designation from the FDA.
Closed an underwritten public offering of 9,090,909 ordinary shares on October 18, 2019 at a price of $5.50 per share with full exercise of underwriters’ option to purchase 1,363,636 additional ordinary shares. Net proceeds totaled approximately €49.3 million. With the addition of this capital ProQR’s operations are funded into 2022.
- At September 30, 2019, prior to the offering on October 18th, ProQR held cash and cash equivalents of €74.8 million, compared to €105.6 million at December 31, 2018. The cash balance at September 30, 2019 excludes the net proceeds of €49.3 million from the underwritten offering of ordinary shares in October 2019. Net cash used in operating activities during the three-month period ended September 30, 2019 was €8.8 million, compared to €4.3 million for the same period last year.
Research and development costs increased to €11.1 million for the quarter ended September 30, 2019 compared to €6.3 million for the same period last year due to increased clinical trial activity.
General and administrative costs increased to €2.9 million for the quarter ended September 30, 2019 compared to €2.6 million for the same period last year.
Net loss for the three-month period ended September 30, 2019 was €12.2 million or €0.31 per share, compared to a €6.0 million loss or €0.18 per share for the same period last year.
In October 2019 the Company sold an aggregate of 10.5 million ordinary shares, with net proceeds to the Company of €49.3 million.
For further financial information for the period ended September 30, 2019, please refer to the financial statements appearing at the end of this release.
Sepofarsen (QR-110) is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.
QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation from the FDA.
QR-1123 is a first-in-class investigational RNA-based oligonucleotide that was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye and received IND clearance in August 2019.
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.