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PRQR

ProQR Therapeutics

$6.23

-0.07 (-1.11%)

16:12
10/15/19
10/15
16:12
10/15/19
16:12

ProQR Therapeutics gets RPD from FDA for sepofarsen to treat LCA 10

ProQR Therapeutics announced that it received Rare Pediatric Disease, RPD,designation from the U.S. Food and Drug Administration for sepofarsen for the treatment of Leber's congenital amaurosis 10, LCA10, the most common cause of blindness due to genetic disease in children. Sepofarsen, QR-110, is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of LCA10 due to the p.Cys998X mutation in the CEP290 gene. The RPD designation provides priority review by the FDA to encourage treatments for rare pediatric diseases. Under the RPD program, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review by the FDA of a subsequent marketing application for a different product

PRQR ProQR Therapeutics
$6.23

-0.07 (-1.11%)

02/20/19
SBSH
02/20/19
NO CHANGE
SBSH
Buy
Citi opens 90-day catalyst watch on ProQR Therapeutics
Citi analyst Yigal Nochomovitz opened a 90-day catalyst watch on ProQR Therapeutics in anticipation of the interim Phase 1/2 Wings data for QR-313 in dystrophic epidermolysis bullosa. On positive data, or evidence of exon skipping in biopsies and evidence of skin lesion/blister resolution, the analyst sees the stock up 15%-25%. On negative data, he sees downside of 10%-15%. Nochomovitz has a Buy rating on ProQR Therapeutics.
03/27/19
CANT
03/27/19
NO CHANGE
Target $40
CANT
Overweight
ProQR Therapeutics spin-out 'smart, but not surprising,' says Cantor Fitzgerald
Cantor Fitzgerald analyst Eliana Merle thinks ProQR Therapeutics' spin-out of its dystrophic epidermolysis bullosa program is a "smart, but not surprising," strategic move. Given that the company has communicated it wants to focus on its ophthalmology programs, investors are unlikely to find this announcement about the skin program spin-out surprising, Merle tells investors in a research note. The analyst keeps an Overweight rating on ProQR Therapeutics with a $40 price target.
10/09/19
CANT
10/09/19
NO CHANGE
Target $40
CANT
Overweight
ProQR shares could more than double on tomorrow's data, says Cantor Fitzgerald
Cantor Fitzgerald analyst Eliana Merle reiterates an Overweight rating on ProQR Therapeutics with a $40 price target ahead of tomorrow's data update on lead program QR-110, which is in development for LCA10, a rare genetic form of blindness. Investors view ProQR and QR-110 as a "show-me story," but these data could lift a "significant overhang" around platform safety and serve as a "key piece of clinical de-risking" for QR-110's Phase 2/3 trial under way, Merle tells investors in a research note partially titled "A double by this time tomorrow?". The analyst thinks the stock could trade up 50%-150% tomorrow versus downside to its cash position of ~$2 per share, or ~65%.
10/10/19
SBSH
10/10/19
NO CHANGE
Target $40
SBSH
Buy
ProQR Therapeutics price target raised to $40 from $34 at Citi
Citi analyst Yigal Nochomovitz raised his price target on ProQR Therapeutics to $40 from $34, stating that he disagree's with the market's initial take on the company's updated data on sepofarsen. Given his view that the 12-month data validate the treatment benefits observed at 3 months, Nochomovitz increased his odds on the success for sepofarsen in patients with Leber's congenital amaurosis 10 to 70% from 50% and keeps his Buy rating on the stock. In afternoon trading, ProQR shares are down 26c, or 4%, to $5.95.

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