Uniqure unveiled mixed results for its study of a Huntington's disease treatment on Wednesday, and QURE stock crashed.
The biotech company hopes its gene therapy will be a one-time treatment for Huntington's, a progressive disease that causes nerve cells in the brain to break down and die. A group of patients who received a low dow of the drug, known as AMT-130, showed functional improvements after two years. The high-dose group showed similar improvements a year following treatment.
But biomarker results were mixed as the high-dose group experienced increases in troublesome proteins a year following treatment.
On today's stock market, QURE stock plummeted 40.2% to close at 11.62. Shares hit their lowest point in six years.
QURE Stock: Mixed Functional, Biomarker Results
Six patients received the low dose of the Huntington's disease treatment, while 10 received the high dose.
On average, patients in both groups showed improvements in motor skills, functional capacity and total symptoms compared to the expected natural course of Huntington's disease.
But Uniqure also examined a protein called neurofilament light chain. A heightened level of this protein in a patient's cerebral spinal fluid is associated with worsening neurodegenerative disease. The low-dose group saw an average decrease of 12.9% after two years. But the high-dose group experienced a 51.5% increase after a year. Those levels seemingly backed off over time, however.
Uniqure also measured mutant Huntingtin protein, which plays a key role in driving the disease. Patients in the low-dose group experienced an 8.1% decrease in this protein after two years. But the high-dose group had a 39.7% increase compared to a jump of 4.7% for the placebo group.
RBC Capital Markets analyst Luca Issi kept his outperform rating and 52 price target on QURE stock.
"Overall, data (are) not perfect but we are encouraged by the functional data and like that Uniqure is planning to engage the regulators soon," he said in a report.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
