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On Wednesday, H.C. Wainwright reaffirmed its Buy rating and $70.00 price target for uniQure BV (NASDAQ:QURE), representing significant upside from the current price of $11.33. According to InvestingPro data, the stock has shown high price volatility, delivering a remarkable 125% return over the past year despite recent market fluctuations. The firm’s analyst, Patrick Trucchio, highlighted several upcoming milestones for the company that could positively impact its valuation. Trucchio pointed out the anticipated regulatory update on AMT-130 in the second quarter of 2025, as well as pivotal Phase 1/2 data expected in the third quarter of the same year. These developments could pave the way for an accelerated approval pathway for Huntington’s disease treatment.
Trucchio emphasized the significance of the FDA’s agreement on key elements of an accelerated approval path for AMT-130, describing it as a "transformational moment" that could de-risk the program and potentially hasten commercialization by several years. The analyst’s optimism extends beyond the Huntington’s disease program, as uniQure is advancing its gene therapy pipeline with treatments for other conditions, such as Fabry disease, refractory mesial temporal lobe epilepsy, and SOD1-ALS.
Multiple data readouts for these programs are expected throughout 2025 and 2026. Trucchio’s analysis suggests that uniQure’s current valuation, with a market cap of $613 million, does not fully reflect the progress and potential success of its clinical pipeline, particularly in relation to its Huntington’s disease program. While the company reported revenue growth of 71% in the last twelve months, InvestingPro data shows it’s currently burning through cash with an EBITDA of -$169 million. Nevertheless, with a strong current ratio of 9.74 and a cash runway projected to last into the second half of 2027, H.C. Wainwright sees uniQure’s stock as significantly undervalued.
The affirmation of the Buy rating by H.C. Wainwright comes at a critical time for uniQure, as the company is on the brink of potential breakthroughs in gene therapy that could transform treatment options for several diseases. The financial firm’s confidence aligns with the broader analyst consensus, which remains strongly bullish with price targets ranging from $18.39 to $70.89. For deeper insights into uniQure’s financial health and additional analysis, investors can access over 10 exclusive ProTips and comprehensive metrics through InvestingPro.
In other recent news, CSL (OTC:CSLLY) Behring has confirmed the long-term efficacy and safety of HEMGENIX, a gene therapy for hemophilia B. The four-year results from the HOPE-B study demonstrated sustained factor IX activity levels and a significant reduction in the annualized bleeding rate, with 94% of patients remaining free of continuous prophylaxis treatment by the fourth year. No serious adverse events were linked to the treatment, and it was generally well-tolerated. Meanwhile, uniQure has completed enrollment for the first cohort in a Phase I/IIa trial of AMT-191, a gene therapy for Fabry disease, and plans to proceed with the second cohort following a positive safety assessment. The U.S. Food and Drug Administration has granted AMT-191 Orphan Drug status and Fast Track designation. In a separate development, H.C. Wainwright raised its price target for uniQure to $70, maintaining a Buy rating, highlighting optimism about the AMT-130 program for Huntington’s disease. Additionally, uniQure announced a public offering of shares priced at $17 each, aiming to raise approximately $75 million. TD Cowen has identified uniQure’s upcoming accelerated approval application for AMT-130 as a key 2025 catalyst to watch in the biotech sector.
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