Investment firm H.C. Wainwright has reiterated its Buy rating and set a price target of $70 per share for uniQure BV (NASDAQ: QURE), signaling a robust confidence in the gene therapy company's prospects amid ongoing regulatory evaluations. This price objective suggests an upside exceeding 200% relative to uniQure's current share price of $22.69, aligning with consensus analyst recommendations that remain favorably inclined. Data from InvestingPro indicates price targets among analysts vary widely, from a low of $33.05 up to a high of $96.43.
The reaffirmation came following a detailed January 14 meeting with uniQure’s executive team, including Chief Medical Officer Walid Abi-Saab, Chief Customer and Strategy Officer Kylie O’Keefe, and Senior Director of Investor Relations and Communications Chiara Russo. The discussions addressed multiple regulatory scenarios pertinent to AMT-130, the company’s investigational gene therapy candidate targeting Huntington's disease.
H.C. Wainwright emphasized that the forthcoming FDA Type A meeting is a pivotal near-term event that could unlock resolution in the current regulatory impasse, potentially reinstating an accelerated approval path for AMT-130. The firm highlighted that uniQure’s leadership conveyed strong confidence in the therapy's demonstrated efficacy, safety profile, and durability of effect, noting that the regulatory debates appear more focused on the timeline for generating confirmatory data than on foundational concerns about the treatment itself.
Financially, uniQure continues to face headwinds. InvestingPro data reveals the company operates with negative gross profit margins and is projected to remain unprofitable this year. Nevertheless, uniQure maintains robust financial flexibility, exemplified by a current ratio of 7.12, reflecting that short-term obligations are more than covered by liquid assets, which tempers concerns about near-term liquidity risks.
Additionally, H.C. Wainwright pointed to the FDA’s recently issued draft guidance dated January 12 regarding clinical trial designs for rare diseases, which endorses the use of external data sources, such as natural history studies, for primary efficacy inference. This development may favorably influence uniQure’s regulatory strategy and bolster its prospects for approval.
Market participants should monitor how the evolving regulatory landscape might affect uniQure, particularly as the company prepares to release its upcoming earnings report on March 2. The stock has experienced notable volatility but appreciated roughly 47.5% over the past half-year. UniQure’s current market capitalization stands near $1.41 billion.
In related recent developments, uniQure is actively engaging with the U.S. Food and Drug Administration concerning its Biologics License Application (BLA) data for AMT-130. The scheduled Type A meeting aims to review the application data that could support an accelerated approval pathway. However, the FDA has communicated challenges; according to minutes from a prior pre-BLA meeting, the agency indicated that existing Phase I/II data may be insufficient to approve AMT-130 at this juncture.
This regulatory feedback prompted some market analysts to adjust their outlooks. For instance, Mizuho reduced its price target for uniQure from $60 to $33 while maintaining an Outperform rating on the stock. Contrastingly, Cantor Fitzgerald reaffirmed its Overweight rating and kept a $55 price target, acknowledging the complexities in forecasting uniQure’s trajectory amidst ongoing regulatory and market challenges. These developments underscore the nuanced environment uniQure navigates in securing regulatory approvals for its investigational therapy.