- UniQure’s AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain.
- The FDA recently said that data from uniQure’s Phase I/II trials were not sufficient and recommended a new randomized Phase III study.
- RBC Capital estimates a 50% chance of eventual approval as Prasad’s exit could reopen a more balanced discussion at the FDA about the risk-benefit profile of AMT-130.
Shares of uniQure N.V. (QURE) surged more than 20% on Monday, following upgrades from Wall Street firms, citing a potentially improving regulatory outlook for its Huntington’s disease therapy.
Wells Fargo upgraded the company to ‘Overweight’ from ‘Equal Weight’ and sharply raised its price target to $60 from $15, according to The Fly. This implies a 243% potential rise from its current price of around $17.5.
Vinay Prasad’s Reported Departure In Spotlight
Wells Fargo said the outlook for uniQure’s experimental gene therapy AMT-130 could improve following reports that Vinay Prasad will leave the U.S. Food and Drug Administration (FDA) next month. Prasad, who serves as both the Chief Medical and Scientific Officer and the Director of the Center for Biologics Evaluation and Research at the FDA, is set to leave next month, according to a Bloomberg report on Friday.
Wells Fargo also noted that strong criticism from the media and the Huntington’s disease community over the FDA’s call for a new Phase 3 trial highlights stakeholder support for the therapy. That backing could strengthen the drug’s chances during review, approval, and commercialization.
AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain. It uses a virus to deliver genetic instructions that help lower the protein. The treatment is administered directly into the striatum, an area of the brain heavily affected by Huntington’s disease, through a surgical procedure.
Separately, RBC Capital Markets upgraded uniQure to ‘Outperform’ from ‘Sector Perform’ and raised its price target to $35 from $11. The firm said Prasad’s exit could reopen a more balanced discussion at the FDA about the risk-benefit profile of AMT-130, estimating about a 50% chance of eventual approval.
FDA Seeks New Randomized Study
The upgrades come after regulatory uncertainty weighed on the company earlier this year. The FDA recently said that data from uniQure’s Phase I/II trials were insufficient to support a marketing application and recommended that the company conduct a new randomized Phase III study, including a controversial sham surgery control group.
uniQure has raised ethical concerns about such a trial design, arguing that it could expose patients to surgical risks without therapeutic benefit. The company plans to request another meeting with the FDA in the second quarter of 2026 to discuss potential study designs.
Despite the regulatory debate, AMT-130 has shown promising early data. In an earlier study, patients receiving a high dose of the gene therapy experienced about a 75% slower rate of disease progression after three years, along with a slower decline in both motor function and cognitive ability.
How Did Stocktwits Users React?
Despite the sharp rally, retail sentiment for QURE on Stocktwits turned ‘bullish’ from ‘extremely bullish’ a day earlier, amid ‘extremely high’ message volumes.
One user expects the stock to surge to $150 if the company receives the FDA approval.
Another user weighed the options present for short sellers and long-term investors.
The stock has declined nearly 28% so far in 2026.
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