UniQure’s Stock Soars After Promising Gene Therapy Results
UniQure’s stock experienced a significant surge of over 200% on Wednesday after announcing that its experimental gene therapy effectively treated Huntington’s disease.
Huntington’s disease is a degenerative neurological condition that gradually impairs brain function, affecting both movement and cognitive abilities. Remarkably, a high dosage of UniQure’s AMT-130 was found to slow down the disease’s progression by 75% over three years.
Dr. Sarah Tabrizi, who leads the Huntington’s Disease Center at University College London, described the findings as “groundbreaking.” She remarked, “These results are the most compelling we’ve seen so far in this field, highlighting the urgent need for effective treatments and showing that AMT-130 could significantly slow down progression — bringing hope to those affected by this tragic disease.”
The stock soared to 47.50, marking a 247.7% increase and reaching its highest point in nearly five years.
Significant Improvements Noted
Patients treated with the gene therapy displayed noteworthy enhancements on the composite Unified Huntington’s Disease Rating Scale (cUHDRS), which is designed to evaluate disease progression in early to moderate cases of Huntington’s disease.
Additionally, after 36 months, patients experienced lower levels of neurofilament light chain (NfL) in their cerebrospinal fluid. NfL serves as a biomarker indicating potential central nervous system damage. UniQure also confirmed that the gene therapy was well tolerated.
The company plans to submit an application for FDA approval in the first quarter of 2026.
Following a discussion with Huntington’s disease specialists, Leerink Partners analyst Joseph Schwartz raised his price target for UniQure stock from 48 to 68. He characterized the data as “definitive” and noted that the gene therapy is “clearly impactful” in slowing progression.
Schwartz mentioned, “We anticipate growing excitement as a leading expert in Huntington’s disease referred to AMT-130 data as ‘game-changing,’ describing it as a ‘beacon of hope’ for patients and a ‘significant step’ towards a licensed disease-modifying therapy for Huntington’s disease.” He estimates an 85% probability that AMT-130 will receive FDA approval and a 50% chance for endorsement from European regulators.
