uniQure stock boosted by FDA alignment on accelerated approval for AMT-130

On Tuesday, Raymond (NS:RYMD) James made a significant adjustment to its outlook on uniQure BV (NASDAQ: NASDAQ:QURE), elevating the stock from Outperform to Strong Buy and substantially increasing the price target to $52.00, up from the previous figure of $20.00.

The stock has already shown strong momentum, gaining nearly 14% in the past week. According to InvestingPro data, six analysts have recently revised their earnings estimates upward for the upcoming period. The adjustment follows the biotechnology company's recent announcement regarding its gene therapy product, AMT-130, for the treatment of Huntington's Disease.

uniQure reported that it had obtained agreement from the U.S. Food and Drug Administration (FDA) for an accelerated approval submission of AMT-130. The company highlighted that the composite Unified Huntington's Disease Rating Scale (cUHDRS), compared with a natural history external control, will be used as the intermediate clinical endpoint for the approval process. Notably, the FDA has indicated that no further clinical studies will be needed to back the Biologics License Application (BLA) submission.

The company is poised to discuss additional specifics with the FDA, including technical manufacturing requirements, the statistical analysis plan, and the design for a confirmatory trial or post-marketing obligations, in the first half of 2025. A more detailed update on the filing timeline is anticipated to follow these discussions. InvestingPro analysis shows the company maintains a FAIR overall financial health score, with liquid assets exceeding short-term obligations, though it's currently burning through cash reserves.

The analyst at Raymond James expressed confidence that the existing data on cUHDRS, along with reductions in cerebrospinal fluid neurofilament light chain (CSF NfL), will be sufficient for an accelerated approval of AMT-130. The potential market for Huntington's Disease treatments in the United States is estimated to exceed $2 billion, suggesting substantial growth prospects for uniQure, which currently has a market capitalization of approximately $700 million.

This optimism is captured in the upgraded rating and increased price target. For deeper insights into uniQure's valuation and 10+ additional exclusive analyst tips, visit InvestingPro.

In other recent news, uniQure N.V. has reached an agreement with the U.S. Food and Drug Administration (FDA) on key aspects of an accelerated approval process for its gene therapy, AMT-130, a treatment for Huntington's disease. This development has been positively received by financial services company Stifel. Additionally, the company has initiated a Phase I/II clinical trial for its investigational treatment AMT-162, targeting ALS caused by SOD1 mutations.

Coverage of uniQure has been resumed by Raymond James, assigning an Outperform rating, while H.C. Wainwright and Stifel have maintained a Buy rating on the company. As part of a significant organizational restructuring, uniQure has reduced its workforce by 65% and sold its manufacturing facility in Lexington, Massachusetts, to Genezen.

These recent developments reflect the company's progress in the development of gene therapies, with a focus on AMT-130 for Huntington's disease and AMT-162 for ALS. Further discussions with the FDA are anticipated in the first half of 2025 to discuss the statistical analysis plan and technical requirements for the Biologics License Application submission for AMT-130.

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