ProQR Therapeutics N.V. (PRQR - Free Report) stock soared past 120% on Wednesday after the company released encouraging top-line results from an early-stage phase I/II study, which evaluated its pipeline candidate QR-110 for an eye disorder — Leber’s congenital amaurosis 10 (LCA10).
The study clinically demonstrated meaningful improvement in vision following treatment with QR-110. Leber’s congenital amaurosis 10 (LCA 10) is the most common genetic cause of blindness in children with severe unmet medical need.
QR-110 is a first-in-class single-stranded ribonucleic acid (RNA) oligonucleotide, which targets LCA 10 due to the p.Cys998X mutation in the CEP290 gene.
Interim data from the phase I/II program showed that treatment with QR-110 led to rapid and sustained progress in vision, measured by visual acuity and the mobility course performance. QR-110 was also well-tolerated with no serious adverse events recorded.
After three months of treatment, approximately 60% patients showed a clinically meaningful response in visual acuity and mobility course endpoints in the trial. The company reported that betterment of vision was observed as early as two months post treatment with QR-110 and was stable of three months.
The results met the company’s expectations and criteria to stop enrollment in and progress to a pivotal phase II/III trial, which is likely to begin in the first half of 2019.
This Netherlands-based small biotech company agreed with the FDA to submit a protocol for the phase II/III study and named the same ILLUMINATE. Simultaneously, the company plans to start a study on pediatric patient population.
Shares of ProQR have skyrocketed 444.2% so far this year against the industry’s decrease of 2.9%.
We would like to remind investors that in June 2016, QR-110 was granted an orphan drug designation by the FDA and the European Commission. Subsequently in May 2017, QR-110 received a fast track designation by the former regulatory body for the treatment of LCA 10.
Apart from QR-110, ProQR has many promising pipeline candidates, namely QR-421a and QR-411 for Usher syndrome type 2A. Both candidates are given an orphan drug status by the FDA as well as the European Commission for Usher sydrome type 2.
Zacks Rank & Other Stocks to Consider
ProQR currently carries a Zacks Rank #2 (Buy). Other top-ranked stocks in the healthcare sector include Gilead Sciences, Inc. (GILD - Free Report) , Ligand Pharmaceuticals Inc. (LGND - Free Report) and Illumina, Inc. (ILMN - Free Report) , all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Gilead Sciences’ earnings estimates have moved 7.7% north for 2018 and 1.9% for 2019 over the past 60 days. The stock has gained 4% year to date.
Ligand’s earnings estimates have been raised 14.4% for 2018 and 3.7% for 2019 over the past 60 days. The stock has surged 85.7% so far this year.
Illumina’s earnings estimates have been revised 12.1% upward for 2018 and 10% for 2019 over the past 60 days. The stock has jumped 58.5% so far this year.
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