ProQR reveals positive results from Phase 1/2 trial of Sepofarsen
Category: #health  By Paroma Bhattacharya  Date: 2019-10-12
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ProQR reveals positive results from Phase 1/2 trial of Sepofarsen

Leber congenital amaurosis (LCA) is a very rare eye condition that can be seen in newborn babies. Currently, the disease doesn’t have a permanent therapy, however, many pharma companies are now making some advancements to treat this disease.

ProQR Therapeutics N.V., a biotechnology company based in Netherlands, is one such firm that has recently revealed encouraging top-line findings from the PQ-110-001 study, a dose range finding, first-in-human trial Phase 1/2 trials of sepofarsen (QR-110) in patients suffering from LCA10 (Leber’s congenital amaurosis 10) due to the mutation of p.Cys998X in CEP290 gene.

Speaking on the move, Stephen R. Russell, MD, Principal Investigator, the University of Iowa, said that the trial has established that patients getting sepofarsen showed a clinically meaningful development in vision, meanwhile in a few situations the patient showed a groundbreaking improvement in vision which could be said as life-changing.

As per David Rodman, M.D., Executive Vice President of Research & Development, ProQR, the company is delighted with the reported data from the Phase 1/2 trial, where in LCA10 patients demonstrated a durable and rapid improvement in vision after regular dosage of sepofarsen.

Moreover, the positive data from this trial further strengthens the company’s trust in the design of the Phase 2/3 study, which might act as the primary registration trial for the drug program, adds Rodman

Reportedly, ProQR has also prior made some advancements in this field when it revealed that sepofarsen was cleared for the PRIME (PRIority Medicines) program by the EMA (European Medicines Agency).

Commenting on the approval, Daniel A. de Boer, Chief Executive Officer, ProQR, said that the agency’s decision to provide PRIME access to its therapy further establishes the grueling demand for modern medicine to treat LCA10 patients, which simplifies the rigorous selection process of the therapy.

Furthermore, the company hopes to expand sepofarsen’s reach to patients of LCA10 as soon as possible, he added.

Source Credit: https://ir.proqr.com/news-releases/news-release-details/proqr-announces-positive-top-line-results-phase-12-study



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Paroma Bhattacharya

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Paroma Bhattacharya

Paroma currently works as a content developer for Algosonline, MSF and a series of alike platforms. Fortified with a post-graduation degree in Journalism and Mass Communication, she delved head long into a writing career, creating resourceful and information enriched ...

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