uniQure (QURE.US) soared due to its gene therapy AMT-130, with Goldman Sachs Group, Inc. optimistic that peak sales could reach $2.5 billion.

Goldman Sachs Group, Inc.'s latest in-depth analysis report focuses on the breakthrough progress and commercial prospects of uniQure NV's gene therapy, AMT-130, in the treatment of Huntington's disease. According to disclosures, the therapy has shown significant efficacy in two dosing groups in clinical trials the high-dose group of 17 patients showed a 75% slower disease progression compared to the natural history, a 60% slowing in total functional capacity score change, and an average decrease of 8.2% in cerebrospinal fluid neurofilament light chain protein levels after 36 months of follow-up. Although direct comparative data is not available for the low-dose group of 12 patients, the overall trend is consistent with the high-dose group. Biomarkers and multiple secondary endpoints support the neuroprotective effect of AMT-130. Recently, the stock has experienced a significant increase due to this therapy news. Under the FDA accelerated approval pathway, uniQure plans to submit a biologics license application in the first quarter of 2026, based on the three-year change data of the Composite Unified Huntington's Disease Rating Scale (CUHDRS) in the high-dose group and using the ENROLL-HD natural history dataset as an external control for propensity score matching analysis. The FDA has recognized their manufacturing process validation pathway and has requested additional full-scale good manufacturing practice batches and individual process performance confirmation batches to support the biologics license application (BLA) submission. In terms of safety, no new severe adverse events have been reported since December 2022, and drug-related adverse reactions have been resolved. In the third cohort of the combination immunosuppressive regimen trial, three severe adverse events fully recovered with supportive care. In terms of commercial prospects, Goldman Sachs Group, Inc.'s model predicts that AMT-130's global peak sales could reach $2.5 billion, with a 90% success rate. uniQure plans to initially focus on marketing to around 6,000 of the approximately 20,000 symptomatic diagnosed patients in the United States, assuming a cumulative penetration rate of about 25%. The company will also hold a pre-BLA meeting with the FDA in the fourth quarter of 2025 to discuss confirmatory study requirements and post-marketing research plans to pave the way for the final product launch. The analysis emphasizes that AMT-130 not only reached statistical significance in efficacy data, but its strategy of submitting a BLA through the accelerated approval pathway has also been recognized by the FDA. Combined with the unmet medical needs in the field of Huntington's disease, this therapy is expected to become a key product that changes the treatment landscape. uniQure is steadily advancing the commercialization process of this innovative therapy through continuous communication with regulatory agencies and rigorous accumulation of clinical data, creating dual value for patients and investors.