Gene therapy company uniQure announced Tuesday “ongoing evidence” of a clinical benefit for its Huntington’s disease treatment. However, investors were not impressed as the stock dropped nearly 20%.
Courtesy of uniQure
Biotech uniQure unveiled more data on Tuesday surrounding its candidate known as AMT-130, a potential treatment for Huntington’s disease. The company touted the Phase I/II interim data, updated at 30 months for those taking the low dose and 18 for those taking a high amount, stating that it has shown “ongoing evidence of potential dose-dependent clinical benefit.”
The results were apparently not what Wall Street hoped for, with uniQure’s stock price dropping 19% on Tuesday.
When measured on the composite Unified Huntington’s Disease Rating Scale, the candidate showed a “favorable difference” of 0.39 points at 30 months and 1.24 points for the low and high doses, respectively.
For the total functional capacity metric, a difference was noted in 0.95 points at 30 months in low-dosage patients and 0.49 points at 18 months in those taking the higher dose. The total motor score for AMT-130 showed a difference of 2.80 points at 30 months for the lower dosage amount and 1.70 points for the higher dose at 18 months.
However, in the neurofilament light chain (NfL), the low-dosage amounts were recorded at 6.6% below the baseline, while a decline was also marked in the higher dosage. Also, changes in total brain volume had “trended below natural history” in patients after surgery, and these changes are not clinically meaningful or associated with an increase in neurodegeneration.
“The clinical assessment trends in the ongoing studies of AMT-130 look very promising and continue to show disease stability in Huntington’s disease patients treated with this one-time administered gene therapy, several of whom have now been followed for more than two years,” uniQure CMO Walid Abi-Saab said in a statement. “We are observing favorable trends in evaluation of motor skills, functional independence, and composite rating scores compared to a non-concurrent criteria-matched natural history cohort.”
However, four serious adverse events related to the drug were recorded in the high-disease group, including central nervous system inflammation and severe headache.
The results are not deterring uniQure and will start the enrollment process for a third cohort of around 12 patients to investigate both doses in combination with a perioperative immune suppression to evaluate safety. In the first quarter of 2024, uniQure is planning to start the regulatory process and present another clinical update from Phase I/II at mid-year.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
