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LEXINGTON, Mass. and AMSTERDAM - uniQure N.V. (NASDAQ: QURE), a gene therapy company, has announced progress in its regulatory discussions with the U.S. Food and Drug Administration (FDA) for AMT-130, a gene therapy candidate for Huntington’s disease. The company has aligned with the FDA on key aspects of its Biologics License Application (BLA) expected to be submitted in the first quarter of 2026.
The recent Type B meetings with the FDA have resulted in agreement on several components of the statistical analysis plan and Chemistry, Manufacturing, and Controls (CMC) information to support the BLA for AMT-130. The FDA supports the use of the Unified Huntington’s Disease Rating Scale (cUHDRS) as an acceptable clinical endpoint for accelerated approval. The primary efficacy analysis will compare the 3-year change in cUHDRS in high-dose AMT-130 patients with an external control arm adjusted by propensity score.
The external control data will be derived from the ENROLL-HD study, a comprehensive natural history study of Huntington’s disease, which has enrolled approximately 33,000 patients. This dataset is expected to provide a robust sample size, reducing variability and enhancing the robustness of the analysis.
The company also plans to submit updated statistical analysis and CMC plans to the FDA in the second quarter of 2025, followed by initiating a Process Performance Qualification (PPQ) run, presenting top-line Phase I/II data, and holding a pre-BLA meeting with the FDA later in the year. While InvestingPro analysis indicates the company is currently burning through cash, it maintains a strong liquidity position with a current ratio of 11.99, ensuring adequate resources for its clinical development programs.
uniQure aims to provide the first disease-modifying treatment for Huntington’s disease and has received the FDA’s Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy designations for AMT-130.
The company is conducting Phase I/II clinical studies to evaluate the safety, tolerability, and exploratory efficacy of AMT-130 in the United States and Europe. The studies involve a one-time administration of the therapy directly into the striatum of patients with early manifest Huntington’s disease.
Huntington’s disease is a rare, inherited neurodegenerative disorder that results in progressive physical and mental deterioration. Despite its clear genetic etiology, there are currently no approved therapies to delay onset or slow progression of the disease.
This update is based on a press release statement from uniQure N.V.
In other recent news, uniQure has made significant strides with its gene therapy programs. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to uniQure’s AMT-130 for Huntington’s disease, highlighting its potential to address this serious condition. This designation, along with others such as Regenerative Medicine Advanced Therapy and Fast Track, is based on promising interim data from ongoing Phase I/II trials, which suggest AMT-130 could slow disease progression. Analysts from H.C. Wainwright have reaffirmed their Buy rating and a $70 price target for uniQure, reflecting confidence in the company’s financial position and the prospects of its gene therapy pipeline. The firm sees the Breakthrough Therapy designation as a critical milestone, potentially accelerating the approval process and commercial launch of AMT-130 by 2026.
Additionally, uniQure announced an amended employment agreement with CEO Matthew Kapusta, which includes a base salary of $676,700 and provisions for performance bonuses and long-term incentives. In the event of termination or a change in control, Kapusta is entitled to severance benefits, including salary continuation and accelerated vesting of equity awards. The company’s recent advancements, including the FDA designation and leadership stability, underscore its commitment to developing innovative treatments for severe diseases. As uniQure progresses with its clinical trials and regulatory interactions, the market will closely watch the development of its promising gene therapy candidates.
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