Shares of ProQR Therapeutics N.V. (PRQR) have gained 22 percent over the last 10 trading days as the Company gears up to report findings from a planned three-month interim analysis of its trial, dubbed STELLAR, on March 31, 2020.
The STELLAR trial is a phase I/II study of RNA-based oligonucleotide drug candidate QR-421a in adults with Usher syndrome type 2 and non-syndromic retinitis pigmentosa.
Usher syndrome is a rare genetic disorder primarily characterized by combined deafness and blindness. Currently, there are no available therapies for this condition. There are three types of Usher syndrome – Type 1, Type 2 and Type 3.
Patients with Usher syndrome type 2 (USH2), the most common type of Usher syndrome, have a moderate to severe hearing impairment from birth and commonly experience the first symptoms of night blindness in their second decade of life, which progresses to complete blindness by the third or fourth decade of life. The progressive vision loss in these patients is known as retinitis pigmentosa (RP).
The STELLAR study aims to find out if different dose levels of QR-421a given as a single intravitreal injection in one eye are safe in patients with Usher syndrome type 2 or RP due to mutations in exon 13 of the USH2A gene.
Also in the Company’s pipeline are Sepofarsen for Leber’s congenital amaurosis 10; QR-1123 for autosomal dominant retinitis pigmentosa and QR-504a for Fuchs Endothelial Corneal Dystrophy.
— Sepofarsen (QR-110) is under a phase II/III trial, for Leber’s congenital amaurosis 10, dubbed ILLUMINATE. Top-line data from this study is expected during the first half of next year.
Leber congenital amaurosis, or LCA, is a rare disorder affecting the retina of the eye and Leber congenital amaurosis 10 is a subtype of LCA.
— Sepofarsen is also under an international phase Ib/II open-label extension study in patients with Leber’s congenital amaurosis 10 (LCA10), dubbed INSIGHT. Updated data from the INSIGHT extension study is expected in the second half of this year.
— QR-1123 is under a phase I/II trial in patients with autosomal dominant retinitis pigmentosa, dubbed AURORA. Initial data from this study are expected in 2021.
— QR-504a for Fuchs Endothelial Corneal Dystrophy is expected to advance into clinical development this year.
ProQR had cash and cash equivalents of €112.0 million at the end of the year 2019.
PRQR has traded in a range of $4.83 to $16.07 in the last 1 year. The stock closed Friday’s trading at $7.18, up 9.28%.
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