ProQR's Genetic Disorder Candidate Gets Orphan Drug Status

ProQR Therapeutics N.V. (NASDAQ:PRQR) announced that the FDA has granted orphan drug designation to its genetic disorder candidate, QR-313, for the treatment of dystrophic epidermolysis bullosa ("DEB").

ProQR’s share price has increased 2.1% so far this year, underperforming the industry’s rise of 15.3% in the same time frame.

The company has already obtained orphan drug designation for its four other candidates, including its usher syndrome candidate, QRX-421, earlier this month.

We note that orphan drug designation is granted to drugs capable of treating diseases that affect less than 200,000 people in the United States. The status makes QR-313 eligible for seven years of marketing exclusivity in the country, following an approval for DEB. 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.

QR-313 is a RNA-based therapy for treating DEB, which is caused by mutations in exon 73 of the COL7A1 gene. The mutation result in the loss of functional collagen type VII protein that makes the skin fragile.

ProQR is also engaged in developing RNA-based therapies for other genetic rare diseases including cystic fibrosis (QR-010) and Leber’s congenital amaurosis (QR-110).

In a restructuring move, the company spun off its central nervous system ("CNS") disorders focused company, Amylon Therapeutics, in late August. Amylon will develop RNA-based therapies for the treatment of a rare genetic disease, which leads to strokes in mid-adulthood. ProQR will own majority ownership stake in the newly formed, privately-held company.

Zacks Rank & Key Picks

ProQR has a Zacks Rank #2 (Buy). A few other stocks to consider in the biotech sector include Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) , Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and PDL BioPharma, Inc. (NASDAQ:PDLI) , While Alexion and Regeneron sport a Zacks Rank #1 (Strong Buy), PDL BioPharma carries a Zacks Rank #2. You can see the complete list of today’s Zacks #1 Rank stocks here.

Alexion Pharmaceuticals’ earnings per share estimates have moved up 5.3% to $5.60 for 2017 and 7.6% to $7.06 for 2018 over the last 60 days. The company delivered positive earnings surprises in the trailing four quarters, with an average beat of 11.12%. Share price of the company has increased 16.8% year to date.

Regeneron’s earnings per share estimates have increased 16.7% to $14.99 for 2017 and 8.6% to $16.64 for 2018 over the last 60 days. The company pulled off positive earnings surprises in two of the trailing four quarters, with an average beat of 10.11%. Share price of the company has increased 17.5% year to date.

PDL BioPharma’s earnings per share estimates increased 263% to 69 cents for 2017 and 174% to 63 cents for 2018 over the last 60 days. The company has topped estimates in two of the trailing four quarters with an average of 33.79%. Shares are up 55.2% so far this year.

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