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ProQR's Usher Syndrome Candidate Gets Orphan Drug Status

ProQR Therapeutics N.V. PRQR announced that its key pipeline candidate, QRX-411, has been granted orphan drug designation by the FDA and European Medicines Agency (EMA) for the treatment of retinitis pigmentosa. The candidate also targets Usher syndrome, which causes deafness and blindness due to genetic defects in the Usher gene.

ProQR Therapeutics’ share price has been flat in the last six months, underperforming the Zacks classified Medical - Biomedical and Genetics industry’s increase of 2.7% in the same time frame.

We note that orphan drug designation is granted to drugs capable of treating diseases that affect less than 200,000 people in the U.S. The status makes QRX-411 eligible for seven years and 10 years of marketing exclusivity in the U.S. and the EU, respectively, following an approval for Usher syndrome. The designation also makes the company eligible for certain other benefits, including tax credits related to clinical trial expenses, exemption from the FDA-user fee and assistance from the FDA in clinical trial design.

QRX-411 targets severe genetic retinal diseases for which there are no treatments available to restore vision or impede progression of the disease. QRX-411 targets the mutations in the USH2A gene, specifically the pseudo-exon 40 mutation. The candidate has shown positive pre-clinical data in patients with fibroblasts and the optic cup model for mRNA restoration.

However, QRX-411 is not the only drug that ProQR is developing. The company is targeting various genetic retinal conditions for which no therapies are currently available. The company is developing therapies for all of the ophthalmology indications using its novel RNA oligonucleotide drug discovery platform.

The company is currently investigating QR-110 in a Phase 1/2 clinical study in Leber`s congenital amaurosis Type 10. The product has already received orphan drug designation in both the US and Europe, and has also been granted Fast Track status by the FDA. ProQR Therapeutics is also developing QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.

Moreover, ProQR Therapeutics is developing QRX-421 for Usher syndrome Type II, targeting Exon 13 mutations in the USH2A gene.

Zacks Rank & Stocks to Consider

ProQR Therapeutics currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the healthcare sector include Enzo Biochem, Inc. ENZ, Sanofi SNY and Merck & Company, Inc. MRK. Enzo Biochem sports a Zacks Rank #1 (Strong Buy) while Sanofi and Merck carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Enzo Biochem’s loss estimates have narrowed from 12 cents to 7 cents for 2017 and from 11 cents to 3 cents for 2018 over the last 30 days. The company came up with positive earnings surprise in the last four quarters, with an average beat of 55.83%. The share price of the company is up 61.9% so far this year.

Merck’s earnings estimates have remained unchanged for 2017 and moved up from $4.16 to $4.18 for 2018 over the last 30 days. The company came up with positive earnings surprise in the last four quarters, with an average beat of 4.36%. The share price of the company is up 9% so far this year.

Sanofi’s earnings estimates have moved up from $3.08 to $3.18 for 2017 and from $3.26 to $3.30 for 2018 over the last 60 days. The company came up with positive earnings surprise in two of the last four quarters, with an average beat of 5.10%. The share price of the company is up 18.3% so far this year.

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