The biotech’s stock rose more than 60% as fresh data appeared to alleviate some investor concerns about the therapy’s effectiveness and reliability.
Shares of UniQure rose more than 60% Tuesday morning after the Netherlands-based biotechnology company gave an updated look at clinical trial results for its gene therapy for Huntington’s disease.
UniQure has been periodically releasing data from two small trials that are testing a low and high dose of the therapy, called AMT-130, in patients with early Huntington’s disease. By December of last year, the company said both doses appeared to be preserving or improving neurological function.
Though UniQure had described those results as “very promising,” investors weren’t so convinced. AMT-130 is designed to thwart the production of huntingtin — a protein that, while normally helpful for brain function, can mutate, become toxic and ultimately cause Huntington’s. But on that measure of reducing mutant huntingtin, the therapy’s performance hasn’t been consistent. The low dose has also sometimes seemed more effective than the high dose.
UniQure’s latest update pertains to results gathered as of March 31. By then, the company had two years of data from 21 participants who received AMT-130, including 12 on the low dose and nine on the high. Previously, UniQure shared data from 18 months of follow-up for the high-dose arm.
According to UniQure, the new results show AMT-130 is decelerating the progression of Huntington’s, and doing so in a “dose-dependent” manner — meaning the higher dose has been more effective. The company said that at 24 months, the larger dose had slowed disease progression by 80% compared to a “natural history cohort” of patients with early Huntington’s disease who were similar to the study participants. Patients receiving that dose had about the same motor and cognitive function as when the study began.
The lower dose, meanwhile, slowed the disease by 30% at the two-year mark. The comparison to the natural history cohort is a “post hoc” analysis, which are typically considered less statistically rigorous.
Additionally, UniQure said participants receiving its therapy experienced a significant reduction in “neurofilament light chain,” a protein that research suggests is indicative of brain and nervous system damage. Levels in the fluid surrounding patients’ brains and spines dropped an average of 11% two years after treatment, compared to baseline levels.
That finding may prove valuable, since neurofilament has become a useful litmus test for drugs targeting neurodegenerative diseases. Biogen, notably, secured approval for the ALS medicine Qalsody because of its effects on this protein.
Earlier data from UniQure’s trial had found average neurofilament levels declined 6.6% at 30 months for participants on the low dose and barely at all at the 18-month mark for those on the high dose.
Even though investors had “very low expectations” for UniQure’s program, the fresh cut of data are “interesting and “broadly look favorable,” according to Paul Matteis, an analyst at the investment bank Stifel.
The biggest question now, Matteis argues, is how the company intends to get its therapy onto the market.
UniQure wants to meet with the Food and Drug Administration before the end of the year to present the updated data and “discuss potential expedited clinical development pathways and accelerated approval.” It also expects to present safety data in the first half of 2025.
UniQure plans on presenting another interim analysis from its studies of AMT-130 in mid-2025, an update which will include three-year data.
