uniQure advances gene therapy trial for Fabry disease

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, announced today the completion of enrollment for the first cohort in a Phase I/IIa trial of AMT-191, an investigational treatment for Fabry disease. Following a safety assessment by the Independent (LON:IOG) Data Monitoring Committee (IDMC), which found no significant safety concerns, the company has been advised to proceed with dosing the second cohort. According to InvestingPro data, the company’s stock has shown remarkable momentum, posting a 115% gain over the past six months.

Fabry disease is a genetic disorder characterized by the buildup of lipids in various cells, leading to a range of symptoms affecting multiple systems in the body. Current treatments involve bi-weekly enzyme replacement therapy, which has limited effectiveness for many patients. While uniQure maintains a strong liquidity position with a current ratio of 6.51, InvestingPro analysis indicates the company is quickly burning through cash as it advances its clinical programs.

AMT-191, a novel AAV5-based gene therapy, aims to address this unmet medical need by using a proprietary promoter to deliver a galactosidase alpha (GLA) transgene to the liver, potentially offering a one-time intravenous treatment alternative. The first cohort of the trial involved up to six adult male patients, and the second cohort is expected to begin enrollment in the first quarter of 2025.

The trial assesses the safety, tolerability, and preliminary efficacy of AMT-191 by measuring the expression of the lysosomal enzyme aGLA-A. The U.S. Food and Drug Administration has granted AMT-191 Orphan Drug status and Fast Track designation, recognizing the urgency of developing treatments for rare diseases with significant unmet needs.

uniQure’s Chief Medical (TASE:PMCN) Officer, Dr. Walid Abi-Saab, expressed optimism about the initial findings and the IDMC’s recommendation, reinforcing the company’s commitment to advancing this clinical program. The company’s broader pipeline includes gene therapies for Huntington’s disease, refractory temporal lobe epilepsy, ALS, and other serious conditions.

The information in this article is based on a press release statement from uniQure Inc. and is intended to provide a factual update on the company’s progress with its AMT-191 clinical trial for Fabry disease. Analysts maintain a bullish outlook on uniQure, with price targets ranging from $19.48 to $68.75, suggesting significant upside potential. For comprehensive financial analysis and additional insights, including 12 more exclusive ProTips, visit InvestingPro.

In other recent news, uniQure BV has seen significant developments in its gene therapy programs. H.C. Wainwright has reaffirmed its confidence in uniQure by maintaining a Buy rating and raising the price target to $70. This development follows the firm’s promising discussions with uniQure’s top executives regarding the AMT-130 program, a potential treatment for Huntington’s disease. Additionally, the company has announced a public offering of over 4 million shares at $17.00 each, expecting to raise approximately $75 million.

The U.S. Food and Drug Administration (FDA) has agreed to an accelerated approval process for AMT-130, a development that has been positively received by financial services companies like Raymond (NSE:RYMD) James, Stifel, and RBC Capital Markets. TD Cowen, through its analyst Joseph Thome, has outlined anticipated catalysts for 2025 that are expected to generate significant investor interest, including clinical trial data releases and regulatory milestones across various firms.

Mizuho (NYSE:MFG) Securities has increased its price target for uniQure’s stock to $20.00, reflecting growing confidence in uniQure’s AMT-130. However, Mizuho noted that the market has already priced in the potential for accelerated approval of AMT-130. Lastly, Goldman Sachs maintained its Neutral rating on uniQure shares, citing the FDA’s review of AMT-130 data. These recent developments reflect uniQure’s progress in gene therapy, particularly with AMT-130 for Huntington’s disease and AMT-162 for ALS.

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