October 28, 2019, Andover, MA and Tempe, AZ – Ora and SDC congratulate ProQR Therapeutics N.V. (Nasdaq: PRQR) on their recent announcement of positive topline results from the PQ-110-001 study of sepofarsen (QR-110). In a press release on October 10, 2019, ProQR announced that the Phase 1/2 dose range finding, first-in-human trial of sepofarsen (QR-110) in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene garnered positive results including rapid, significant and durable improvements in vision at twelve months and concordant improvement in key secondary outcome measures. ProQR also announced that the target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile.
The study was conducted by Ora, Inc., the world’s leading full-service ophthalmic CRO and product development firm. Keith Lane, MBA, Executive Director and Therapeutic Area Head at Ora, commented “We congratulate the ProQR Therapeutics team for achieving such a significant restoration of vision in LCA Type 10 patients. This treatment has potential to dramatically improve patient quality of life. On behalf of the team at Ora Inc., we are proud to be a part of the sepofarsen program.”
Biostatistics, clinical data management, and electronic data capture (EDC) services for the Phase 1/2 study were performed by SDC, a specialized data services CRO and partner company of Ora, Inc. “We are proud to be part of the clinical development team for the sepofarsen program. The positive topline data reported this month are an encouraging step forward for the genetic rare disease community affected by LCA10,” said Dale W. Usner, PhD, CSO & Sr. VP, Strategic Scientific Consulting at SDC.
For more information, please see ProQR full and original press release dated October 10, 2019: ProQR Announces Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients.