What's The Thesis On ProQR Now, After Promising Phase 1 Data?

ProQR Therapeutics NVPRQR
, a nano-cap pre-clinical stage bio pharmaceutical company engaged in the discovery and development of ribonucleic acid-based therapeutics for the treatment of severe genetic disorders,
announced the completion of a phase 1b study Monday, which helped boost shares of ProQR higher by 4 percent early Tuesday morning. ProQR's phase 1b safety and tolerability clinical trial of QR-010, a novel investigational RNA therapeutic in subjects with cystic fibrosis, saw favorable tolerability across all doses coupled with no drug related adverse events, Leerink's Joseph Schwartz commented in a research report. Perhaps more important, the company reported improvements in exploratory endpoints, including improvements in patient symptoms according to the CFQ-R (12.5mg dose, p=0.0072) and a 10.9 percent (p=0.0461) improvement in percent predicted forced expiratory volume. Schwartz maintains an Outperform rating on ProQR's stock with a price target boosted from $10 to $12.

The data report should be viewed as a "positive validation" for the company's RNA repair platform for two reasons, the analyst noted. First, it validates clinical safety and tolerability, and second, it gives the company the opportunity to now evaluate its platform in a bigger and more severe CF population group. However, the company has yet to confirm any future developments but it is evaluation collaborations as a combo therapy or as a monotherapy.

Yet despite the encouraging data, the analyst still sees a 30 percent probability of success which does mark an increase from a prior estimate of just 20 percent. Also, the company's peak penetration now stands at 33 percent which is lower from a prior 45 percent estimate to reflect the "increasingly competitive CF landscape."

At time of publication, shares of ProQR were down 10.76 percent at $5.76.

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Posted In: BiopharmaceuticalJoseph SchwartzLeerinkRNARNA TherapeuticAnalyst ColorBiotechLong IdeasNewsAnalyst RatingsMoversTrading IdeasGeneral