Wolfe Research initiated coverage of uniQure BV (NASDAQ: QURE) on Monday and assigned the stock a Peerperform rating, according to the research note. At the time of the initiation the shares were trading at $24.64, representing a market capitalization of $1.53 billion. The stock has risen 12.5% over the past week and gained 113% over the last 12 months.
The initiation highlights uniQure's work with adeno-associated virus, or AAV, gene therapy - a modality the firm describes as partially validated given that multiple products have been developed using the approach. Wolfe Research pointed specifically to uniQure's lead investigational program, AMT-130, which has shown encouraging treatment effects in Phase 1/2 studies in patients with Huntington's disease.
Despite the encouraging clinical signal, Wolfe Research emphasized that uniQure must identify a clear regulatory route with the U.S. Food and Drug Administration. The firm noted that Huntington's disease presents a challenging regulatory and clinical environment, given its unmet medical needs, and said a disease-modifying therapy such as AMT-130 would be well received by the patient community. Wolfe Research estimated that, if approved, AMT-130 could generate U.S. peak sales in excess of $1.5 billion for Huntington's disease.
Given remaining uncertainty around the regulatory path, the analyst team said it prefers to remain on the sidelines until additional clarity emerges. The note added that updates related to a Type A meeting with regulators could arrive in the coming days, a development the firm flagged as potentially material to its view.
Separately, uniQure disclosed encouraging preliminary results from a Phase I/IIa study of AMT-191, its gene therapy candidate for Fabry disease. The company reported that all 11 patients treated in the trial demonstrated elevated alpha-galactosidase A, or alpha-Gal A, activity. In the highest dose cohort, increases in enzyme activity ranged from 27.7-fold to 223.7-fold above normal levels. The company also said the longest-treated patient maintained supraphysiological enzyme expression for more than one year, signaling sustained activity following dosing.
Market watchers have supplied mixed views. H.C. Wainwright reiterated a Buy rating on uniQure and set a $70.00 price target after discussions with company management about potential regulatory scenarios for AMT-130. By contrast, analysis on a separate platform indicated the stock currently appears overvalued relative to its own Fair Value estimate, while also noting the company’s strong cash position and other financial health metrics.
Investors have also been notified of an April 13, 2026 deadline to file lead plaintiff applications in a securities class action lawsuit. The complaint alleges that uniQure and certain executives failed to disclose material information during a specified period, asserting violations of federal securities laws. The presence of active litigation adds a legal dimension to the company’s outlook.
In sum, analysts are weighing promising clinical data against unresolved regulatory and legal questions. AMT-130’s early clinical performance and the AMT-191 enzyme-activity findings strengthen uniQure’s clinical narrative, but the market and research community are looking to forthcoming regulatory interactions and filings for clearer signals on approval likelihood and commercial prospects.
Key points
- Wolfe Research initiated coverage with a Peerperform rating while citing partial validation of the AAV gene therapy approach and encouraging Phase 1/2 AMT-130 data in Huntington's disease.
- uniQure reported strong preliminary AMT-191 data for Fabry disease, with all 11 patients showing elevated alpha-Gal A activity and sustained supraphysiological expression in the longest-treated patient.
- Regulatory clarity with the FDA - including outcomes from an anticipated Type A meeting - and legal developments are the near-term events likely to influence sentiment and valuation.
Risks and uncertainties
- Regulatory path risk - Wolfe Research explicitly noted that uniQure needs to determine an FDA approval pathway for AMT-130, and the firm is waiting for greater clarity.
- Valuation risk - third-party analysis cited in the coverage suggests the stock appears overvalued relative to its Fair Value estimate, indicating potential downside if expectations are not met.
- Legal risk - investors were reminded of an April 13, 2026 deadline to apply to be lead plaintiff in a securities class action alleging failure to disclose material information, introducing litigation uncertainty.
The developments described above reflect a company with promising clinical data but with outstanding regulatory and legal questions that market participants will monitor closely in the near term.