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ProQR Initiates Dosing in Phase II/III Eye Disorder Study

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ProQR Therapeutics N.V. (PRQR - Free Report) announced that it has started dosing patients in the phase II/III study – ILLUMINATE – evaluating its RNA-based candidate sepofarsen (QR-110) for an eye disorder, Leber’s congenital amaurosis 10 (LCA10).

The ILLUMINATE study will enroll 30 patients in three separate study arms and evaluate two active doses of sepofarsen and a sham control over a period of 24 months for treating LCA10 in patients over eight years of age with certain mutation in the CEP290 gene. The primary endpoint of the study is improvement in best corrected visual acuity compared to baseline, which is set at 3.0 LogMAR or better.

The company has designed ILLUMINATE study to serve as registration study, which will allow the data from it to be used for filing regulatory application seeking approval for the candidate.

Leber’s congenital amaurosis 10 is the most common genetic cause of blindness in children with severe unmet medical need.

Shares of ProQR have declined 17.6% so far this year against the industry’s increase of 8.6%.


We remind investors that ProQR announced favorable data last year from a phase I/II study evaluating sepofarsen as a treatment for LCA10. The study clinically demonstrated meaningful improvement in vision following treatment with the candidate.

We would like to remind investors that in June 2016, sepofarsen was granted an orphan drug designation by the FDA and the European Commission. Subsequently, in May 2017, sepofarsen received a fast track designation by FDA for the treatment of LCA 10.

Apart from sepofarsen, ProQR has many promising pipeline candidates, namely QR-421a and QR-411 for Usher syndrome type 2A. Both candidates have been given an orphan drug status by the FDA as well as the European Commission for Usher sydrome type 2.


Zacks Rank & Stocks to Consider

ProQR currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector include Fibrocell Science Inc. , Celsion Corp. and Exelixis, Inc. (EXEL - Free Report) , all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Fibrocell Science’s loss estimates have narrowed from $2.68 to $1.38 for 2019 and from $2.55 to $1.32 for 2020 over the past 60 days. The stock has gained 69.3% year to date.

Celsion’s loss estimates have narrowed from 94 cents to 78 cents for 2019 and 2020 over the past 60 days. The stock has surged 59.6% so far this year.

Exelixis’ earnings estimates have increased from 98 cents to $1.08 for 2019 and from $1.12 to $1.30 for 2020 over the past 60 days. The stock has rallied 16.9% so far this year.

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