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ProQR’s QR-421a fast tracked, Phase 1/2 clinical trial in 2109

ProQR Therapeutics N.V. got Fast Track designation from the Food and Drug Administration (FDA) for QR-421a, a first-in-class investigational RNA-based oligonucleotide to tackle the underlying cause of the vision loss related to the Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

To date, there are no approved treatments or products in clinical development that treat the vision loss associated with Usher syndrome type 2, ProQR notes.

ProQR, a company focused on the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, has received orphan drug designation for QR-421a in the United States and the European Union.

Daniel de Boer, Chief Executive Officer of ProQR noted that the patients with Usher syndrome, the leading cause of combined deafness and blindness, currently have no available therapies for their vision loss and this designation emphasizes the high unmet need in this disease. He added that the company is also looking forward to begin enrollment in the Phase 1/2 STELLAR clinical trial in the coming months with preliminary data expected in mid-2019.

The trial was initiated in 2018. The company expect results from multiple dose adaptive trial in the second half of 2020.

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