UniQure on Tuesday released new two-year data on its Huntington’s disease gene therapy called AMT-130, sending its stock $QURE up by more than 50%.
Interim results from the Phase 1/2 trials showed nine patients who received the high dose of AMT-130 saw their disease slow by about 80% compared to a natural history patient group. The result was statistically significant, with a p-value of p=0.007.
Additionally, levels of neurofilament in patients’ cerebrospinal fluid fell by an average of 11% among all 21 individuals who took either the low or high dose of AMT-130. That figure compared to a 26% increase in another natural history study.
Walid Abi-Saab“With the majority of our patients having reached the important two-year time point, we could now conduct, for the first time, robust post-hoc statistical analyses yielding nominal p-values to better understand the potential efficacy of AMT-130 in Huntington’s disease,” CMO Walid Abi-Saab said on a conference call Tuesday morning.
AMT-130 is an AAV5 gene therapy delivered directly to the brain containing a microRNA that targets exon 1 of the huntingtin gene. UniQure says the one-time administration and potential long-term effects help AMT-130 differentiate itself from other treatments in development.
Researchers measured disease progression using the composite Unified Huntington’s Disease Rating Scale, or cUHDRS. Among the nine patients who received the high dose, cUHDRS scores fell by an average of 0.2 points, compared to falling by 1 point in the natural history studies, which contained 154 patients.
In the low-dose group, 12 patients saw their cUHDRS scores fall by an average of 0.7 points. The figure represented a 30% slowing of disease, but the result was not statistically significant, with a p-value of p=0.21.
Expectations for Tuesday’s update had been low, Stifel analyst Paul Matteis wrote Tuesday morning, due to previous data releases raising more questions than answers. UniQure’s shares fell more than 40% in June 2023 after data for another endpoint measuring patients’ total motor scores disappointed investors, leading to a major restructuring last October.
But the two-year data, combined with a “backdrop of an increasingly flexible regulatory environment for gene therapies,” give the program some potential, Matteis wrote in a note.
It’s still not yet clear how uniQure will move forward with AMT-130, as the company needs to discuss future trial plans with the FDA. An accelerated approval is potentially on the table, CEO Matt Kapusta said. But uniQure could also partner with a larger company if it needs to run a large pivotal trial.
“If we’re going to be looking at hundreds of millions of dollars and five to six years of a Phase 3 study, that is something that is likely going to be beyond our means, at least today,” Kapusta said.
For now, uniQure is planning to meet with the FDA before the end of the year. The company is also expecting to release 36-month data in the middle of 2025.