Analyst Lights Up Over ProQR Blindness Trial Results

ProQR Therapeutics (PRQR) shares surged 62% in Thursday trading, one day after the Leiden, Netherlands-based biopharmaceutical company announced positive Phase 1/2 clinical results for its QR-421a treatment for Usher Syndrome (a rare genetic disorder that causes deafness, night-blindness, loss of peripheral vision, and can also cause loss of balance).

As the company explained, QR-421a was observed to be “well tolerated” in patients receiving it, and “demonstrated a concordant benefit in multiple measures of vision, including best corrected visual activity (BCVA), static perimetry, and retinal imaging (OCT).”

With these results in hand, ProQR is ready to proceed to Phase 2/3 trials, and expects to begin these before the end of the year.

H.C. Wainwright analyst Andrew Fein found the initial data set from the QR-421a study “positive” for the treatment of patients experiencing early to moderate symptoms of Usher Syndrome, and opined that the data also look promising for treatment of more advanced cases of the disease as well.

“[We] believe the current data are sufficient for planning of parallel Phase 2/3 registrational trials… From a safety standpoint, specifically keeping in mind that both cataracts and cystoid macular edema (CME) occur in >30% of patients in the normal course of disease, we view it as a positive that only one patient was identified to have worsening of pre-existing cataracts in their treated and untreated eye and deemed not to be treatment related, and no new occurring cases of CME were observed. We believe the safety and tolerability profile overall, are encouraging for advancing into the Phase 2/3 trials,” Fein commented.

It’s worth pointing out, of course, that the Phase 1/2 clinical trials that generated this week’s data involved only a very small sample size — 14 patients. The Phase 2/3 trials envisioned for later this year will be substantially larger — approximately 100 patients each, and thus generate statistically more reliable data.

Now what does this mean for the stock?

Fein rates PRQR a Buy along with a $20 price target. This figure implies ~147% upside from current levels. (To watch Fein’s track record, click here)

As Fein explains his reasoning, he anticipates that in fiscal year 2028, ProQR stock will sell for 25 times the $4.86 per share in profits that he expects it to earn that year — so roughly $121.50 per share seven years from now. But if you discount that valuation back to what ProQR should cost today, it works out to a share price of ~$20.

Overall, the consensus sentiment matches well with Fein’s bullish stance, with TipRanks analytics showing PRQR as a Strong Buy. The 12-month average target of $20 is congruent with Fein’s. (See PRQR stock analysis on TipRanks)

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Disclaimer: The opinions expressed in this article are solely those of the featured analyst. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.