A Huntington’s gene therapy by UniQure has made waves after slowing disease progression by 75% in a mid-stage trial.
During the Phase I/II study (NCT05243017), patients treated with a high dose of AMT-130 – otherwise known as ifezuntirgene inilparvovec – experienced a 0.38 reduction in unified Huntington’s disease rating scale (cUHDRS) scores at 36 months.
This cUHDRS reduction was significantly less than the 1.52 decline seen in the external control group.
cUHDRS is a common measure of Huntington’s disease progression, and it considers four key aspects of cognitive and motor function to quantify the severity of a patient’s deterioration.
The disease-modifying therapy (DMT) also slowed the deterioration of patients’ total functional capacity (TFC) at 36 months, with treated individuals seeing a mean reduction of 0.36 from baseline as opposed to the 0.88 drop observed in the control group.
Meanwhile, AMT-130 demonstrated favourable trends towards other secondary measures of disease progression, with patients given the drug consistently outranking their control peers in cognitive and motor function tests.
This includes the symbol digit modalities test (SDMT), the Stroop word reading test (SWRT) and total motor score (TMS).
AMT-130 was also generally well tolerated, with the majority of serious treatment-emergent adverse events (TEAEs) occurring within the first few weeks post-administration. In a 24 September investor call, Uniqure also noted that these events were fully resolved with the use of steroids or supportive care.
UniQure eyes AMT-130 approval
Following the positive trial results, UniQure will submit a biologics licence application (BLA) for AMT-130 to the US Food and Drug Administration (FDA) by Q1 2026. If all goes to plan, the Dutch biotech anticipates that the drug will launch later that year.
UniQure’s CMO Walid Abi-Saab said: “We are incredibly excited about these topline results and what they may represent for individuals and families affected by Huntington’s disease.
“These findings demonstrate AMT-130’s potential to fundamentally transform the treatment landscape for Huntington’s disease following a one-time, precision-delivered dose.”
It appears that investors are just as enthusiastic about AMT-130’s market potential, as UniQure’s stock value shot up nearly 250% after the results debuted – going from $13.66 at market close on 23 September to $47.50 at market close on 24 September.
If AMT-130 were to secure FDA clearance, it would be the first ever therapy approved for the treatment of Huntington’s disease, which currently impacts one in every 10,000 to 20,000 people in the US alone.
Huntington's disease causes nerve cells in the brain to decay over time, impacting a person's movements, thinking ability and mental health.
Upon its potential approval, analysts at GlobalData forecast that the drug will make $362m for UniQure by 2031 – a 1,710% surge from the $20m predicted in 2026.
UK biotech back on the map?
While the positive results of the Phase I/II study on AMT-130 will be very welcome news for Huntington’s patients, it will also be a leg up for UK biotech, as the gene therapy was evaluated at the University College London (UCL).
In a 24 September LinkedIn post,, senior life sciences recruiter Richard Hendry said: “News from UCL and uniQure about their successful trial in Huntington’s disease proves the UK is pivotal to advancing medication.
“What this [the Phase I/II results] shows is that the UK is punching above its weight in biotech innovation. With the right support and investment … we’re helping bring life-changing treatments to patients who desperately need them.”
Earlier this month, the Association of the British Pharmaceutical Industry (ABPI) released a report stating the UK is at risk of losing its world-leading life sciences status due to investment being captured elsewhere on the international stage.
Life sciences foreign direct investment into the UK fell by 58% from £1.9bn in 2021 to £795m in 2023. It comes as no surprise that the UK’s rank in inward life sciences FDI amongst comparator countries dropped from second in 2021 to seventh in 2023.
