uniQure (QURE, Financial) has achieved a significant milestone with the U.S. Food and Drug Administration granting Breakthrough Therapy designation for its investigational treatment AMT-130. This therapy is designed to address Huntington’s disease, a challenging inherited neurodegenerative condition that currently lacks disease-modifying treatments.
This new designation adds to a series of recognitions AMT-130 has received, including Regenerative Medicine Advanced Therapy, Orphan Drug, and Fast Track status from the FDA. These acknowledgments highlight the potential of AMT-130 to significantly impact the treatment of this rare disorder.
The support for the Breakthrough Therapy designation stems from promising clinical data gathered during the ongoing Phase I/II trials. In July 2024, uniQure released interim findings demonstrating that the treatment has a dose-dependent effect in slowing the disease's progression. This conclusion was based on comparing the cUHDRS scores of patients receiving AMT-130 to those in a propensity-weighted natural history cohort.
So far, AMT-130 has been administered to 45 patients, marking a substantial step forward in investigating a new therapeutic option for those affected by Huntington’s disease.
Wall Street Analysts Forecast
Based on the one-year price targets offered by 10 analysts, the average target price for uniQure NV (QURE, Financial) is $36.70 with a high estimate of $70.00 and a low estimate of $13.00. The average target implies an upside of 290.84% from the current price of $9.39. More detailed estimate data can be found on the uniQure NV (QURE) Forecast page.
Based on the consensus recommendation from 14 brokerage firms, uniQure NV's (QURE, Financial) average brokerage recommendation is currently 1.8, indicating "Outperform" status. The rating scale ranges from 1 to 5, where 1 signifies Strong Buy, and 5 denotes Sell.
Based on GuruFocus estimates, the estimated GF Value for uniQure NV (QURE, Financial) in one year is $13.56, suggesting a upside of 44.41% from the current price of $9.39. GF Value is GuruFocus' estimate of the fair value that the stock should be traded at. It is calculated based on the historical multiples the stock has traded at previously, as well as past business growth and the future estimates of the business' performance. More detailed data can be found on the uniQure NV (QURE) Summary page.
