A Huntington鈥檚 gene therapy by 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 鈥� otherwise known as ifezuntirgene inilparvovec 鈥� experienced a 0.38 reduction in unified Huntington鈥檚 disease rating scale (cUHDRS) scores at 36 months.
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This cUHDRS reduction was significantly less than the 1.52 decline seen in the external control group.
cUHDRS is a common measure of progression, and it considers four key aspects of cognitive and motor function to quantify the severity of a patient鈥檚 deterioration.
The disease-modifying therapy (DMT) also slowed the deterioration of patients鈥� 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.
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By GlobalDataThis 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鈥檚 CMO Walid Abi-Saab said: 鈥淲e are incredibly excited about these topline results and what they may represent for individuals and families affected by Huntington鈥檚 disease.
鈥淭hese findings demonstrate AMT-130鈥檚 potential to fundamentally transform the treatment landscape for Huntington鈥檚 disease following a one-time, precision-delivered dose.鈥�
It appears that investors are just as enthusiastic about AMT-130鈥檚 market potential, as UniQure鈥檚 stock value shot up nearly 250% after the results debuted 鈥� 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鈥檚 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 鈥� 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鈥檚 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 ,, senior life sciences recruiter Richard Hendry said: 鈥淣ews from UCL and uniQure about their successful trial in Huntington鈥檚 disease proves the UK is pivotal to advancing medication.
鈥淲hat 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 鈥� we鈥檙e helping bring life-changing treatments to patients who desperately need them.鈥�
Earlier this month, the Association of the British 色界吧 Industry (ABPI) released a report stating the UK is at risk of losing its world-leading life sciences status due to investment being .
Life sciences foreign direct investment into the UK fell by 58% from 拢1.9bn in 2021 to 拢795m in 2023. It comes as no surprise that the UK鈥檚 rank in inward life sciences FDI amongst comparator countries dropped from second in 2021 to seventh in 2023.
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