{
  "id": 19541294,
  "title": "uniQure at Stifel 2026 Healthcare Conference: AMT-130 nears key test",
  "url": "https://urgent.news/2026/11/11/uniqure-at-stifel-2026-healthcare-conference-amt-130-nears-key-test",
  "topic": "finance",
  "section": "Finance & Markets",
  "published": "2026-11-11T19:39:37.000Z",
  "source": {
    "name": "Investing.com",
    "slug": "investing-com",
    "url": "https://www.investing.com/news/transcripts/uniqure-at-stifel-2026-healthcare-conference-amt130-nears-key-test-93CH-5019758"
  },
  "original_language": "en",
  "account": "On 11 November 2026, uniQure, a company developing experimental gene therapy for Huntington's disease, attended the Stifel 2026 Healthcare Conference to discuss advancements in its AMT-130 clinical program. The FDA had accepted uniQure's biologics license application (BLA) for AMT-130 with priority review, but an advisory committee meeting was scheduled before a decision could be made. Management described AMT-130 as the first disease-modifying treatment candidate for Huntington's disease, with latest data supporting its case for approval. However, the company noted the need for a confirmatory study and acknowledged the challenges of building surgical capacity and the limitations of external-control data at longer follow-up periods.\n\nThe FDA's acceptance of the BLA was based on 36-month efficacy data from the phase I/II study, focusing on the primary endpoint Total Functional Capacity. This endpoint measures how well patients manage daily life, with treated patients losing less than 0.1 points on average over 48 months compared to about 1.0 points in untreated patients. This difference equates to approximately 60% slowing of disease progression. For the composite Unified Huntington's Disease Rating Scale (cUHDRS), high-dose patients showed more than a 1-point improvement versus low-dose patients, suggesting a dose response and demonstrating about 44% slowing of disease progression.\n\nUniQure is preparing for an advisory committee meeting and a larger confirmatory phase III study, which will enroll about 200 patients and randomly assign them to AMT-130 or standard of care. The study will not use a sham procedure. While the external-control data have limitations, the company argues that the three-year data are the most robust basis for the filing, acknowledging a known issue in neurodegenerative disease studies where a portion of the data points are missing at longer time points due to patient discontinuation and missed visits.",
  "summary": null,
  "key_points": [],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 1,
    "also_reported_by": []
  },
  "ai_generated": true,
  "disclaimer": "Summaries, key points and the editor’s take are written by software from other outlets’ reporting and may contain errors — always check the linked original."
}