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ProQR (PRQR) Begins Dosing in Phase I/II Eye Disorder Study

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ProQR Therapeutics N.V. PRQR announced that it has dosed the first patient in a phase I/II study, Aurora, evaluating its investigational RNA-based candidate QR-1123 for the treatment of patients with autosomal dominant retinitis pigmentosa (adRP). Initial data from the study is expected in 2021.

The Aurora study will enroll up to 35 adult patients with adRP due to the P23H mutation in the rhodopsin (RHO) gene. Per the company, the study will include single-dose escalation (open label) and multiple-dose escalation (double-masked) groups where intravitreal injections of QR-1123 or sham procedures will be given in one eye. The aim of the study is to evaluate the safety and tolerability of the candidate.

Notably, last October, ProQR in-licensed QR-1123 from Ionis Pharmaceuticals (IONS - Free Report) .

In November 2019, the FDA granted Orphan Drug designation to QR-1123 for treating adRP due to the P23H mutation. Earlier in September, the regulatory body granted a Fast Track status to QR-1123 for the same indication.

We remind investors that previously in August, the FDA cleared the investigational new drug application for QR-1123 to treat adRP due to the P23H mutation. The indication currently has no approved therapy.

Shares of ProQR have declined 44.4% so far this year against the industry’s increase of 6.8%.


ProQR is engaged in developing transformative RNA medicines for addressing severe genetic rare diseases, such as adRP, Leber’s congenital amaurosis 10 (LCA10) and Usher syndrome.

Apart from QR-1123, the company is developing Sepofarsen (QR-110) for LCA10 and QR-421a for Usher syndrome type 2.

Sepofarsen is being evaluated in the ongoing phase II/III Illuminate study for LCA10. Top-line data from the same is expected during the first half of 2021. QR-421a is currently being developed in the ongoing phase I/II Stellar study for treating patients with Usher syndrome type 2. Interim data from the same is awaited in the first quarter of 2020.

Zacks Rank & Stocks to Consider

ProQR currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Anika Therapeutics Inc. ANIK and Guardant Health, Inc. GH, both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings estimates have moved 16% north for 2019 and 17.4% for 2020 over the past 60 days. The stock has soared 61.4% year to date.

Guardant Health’s loss per share estimates have narrowed 30.7% for 2019 and 8.5% for 2020 over the past 60 days. The stock has skyrocketed 88.3% so far this year.

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