TipRanks
Thu, January 21, 2021, 4:34 PM
ProQR (PRQR)
ProQR is a biotechnology company focused on treatments for congenital progressive blindness. Specifically, the firm is working on medications to reverse a group of genetic sight disorders called inherited retinal diseases. These diseases currently have no effective treatments.
The company has a research pipeline of five drug candidates, in varying stages of the research process. The two that are farthest along are QR-110 (Sepofarsen), and QR-421. Of those two, QR-110 is currently in Phase 2/3 studies. This candidate is an RNA therapy designed to correct the most common CEP290 gene mutation causing Leber congenital amaurosis 10 (LCA10). This is a severe genetic retinal disease that affects as many as 3 in 100,000 children.
QR-421 is another RNA therapy, this one focused on exon 13 mutations in the USH2A gene. These mutations cause blindness due to retinitis pigmentosa and/or Usher syndrome. QR-421 is in Phase 1/2 studies, with an aim of restoring lost vision or preventing the loss in the first place.
Covering the stock for JMP, analyst Jonathan Wolleben points to Sepofarsen as a key component of his bullish thesis.
“We continue to feel good about sepofarsen’s chance of success in Illuminate for multiple reasons: 1) Phase 1/2 confirmed the target registrational dose and dosing interval (6 months); 2) patients had clinically significant and durable BCVA improvements after 12 months – pivotal primary endpoint; 3) supportive secondary efficacy measures (FST, mobility); 4) similar responses seen in second treated eyes; 5) long-term safety confirms positive risk/benefit; and 6) Illuminate patient population was enriched based on Phase 1/2 results (baselinevision of >/=hand motion). We assign sepofarsen a 60% POS and model LCA10 as an ~$300M opportunity to PRQR at peak penetration," Wolleben opined.
In line with his upbeat outlook, Wolleben puts a $20 price target on the stock, implying a 384% one-year upside, along with an Outperform (i.e. Buy) rating. (To watch Wolleben’s track record, click here)
All in all, PRQR gets a unanimous Strong Buy rating from the analyst consensus, based on 3 positive stock reviews. Shares are currently trading for $4.13, and their $20.67 average price target is slightly more bullish than Wolleben’s, suggesting an upside of 400% for the coming 12 months. (See PRQR stock analysis at TipRanks)