Shares of ProQR Therapeutics N.V. (NASDAQ: PRQR) jumped early on Thursday after the company announced results from its cystic fibrosis (CF) study. Essentially, the company said that the study met its primary endpoint in this cohort as measured by a change in total chloride response following four weeks of treatment with QR-010. In the compound heterozygous ∆F508 cohort, no meaningful difference was found. QR-010 was observed to be safe and well-tolerated in both cohorts.
The company also announced that clinical study PQ-010-001 completed all four single-dose cohorts. PQ-010-001 is a placebo-controlled Phase 1b study in subjects with CF homozygous for ∆F508. QR-010 was observed to be safe and well-tolerated in all four single dose cohorts.
The multiple dose cohorts in this study are ongoing and topline safety, tolerability and exploratory efficacy data from this study are expected in mid-2017.
For just a little background: QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the DF508 mutation. The DF508 mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally.
Noreen R. Henig, M.D., chief development officer of ProQR, commented:
Patients with CF feel and do better when the CFTR protein channel works more normally. Our important first step in helping patients with CF was to demonstrate that QR-010 could restore CFTR function in patients with CF due to ∆F508, the most common mutation. Our proof-of-concept NPD study did exactly that in CF patients homozygous for ∆F508; it demonstrated that CFTR protein channels are active in this cohort following administration of QR-010 as measured by the total chloride response. Having achieved this major step, we have increased confidence in QR-010’s potential to make a meaningful clinical impact for patients and will move forward with an aggressive development plan.
After ProQR closed Wednesday up over 11% at $6.25, shares opened on Thursday at $8.50, an additional gain of more than 38%. The consensus analyst price target is $16.04, and a 52-week trading range of $3.48 to $16.23.