uniQure shares tumbled by more than 40% shortly at the market open on Wednesday after it released mixed results from a Phase 1/2 clinical trial involving its Huntington’s disease treatment AMT-130.
The company reported that AMT-130, a one-time administered investigational gene therapy, continues to be well-tolerated with a manageable safety profile in both its low-dose and high-dose cohort.
Patients in both dose cohorts showed improvements in total motor score, total functional capacity, and the composite United Huntington’s Disease Rating Scale compared to natural history, uniQure said.
However, the trial’s biomarker results were mixed. Patients in both dose cohorts experienced a transient increase in cerebrospinal fluid (CSF) neurofilament light chain (NfL) which peaked about one month after the administration of AMT-130.
CSF NfL for the low-dose cohort decreased by a mean of 12.9% compared to baseline, while the high-dose cohort demonstrated a mean increase of 51.5% compared to baseline at the 12-month mark.
In the control group, the mean CSF NfL was 6.8% below baseline after 12 months.
Additionally, Mutant Huntingtin protein (mHTT), which was below baseline for the low-dose cohort and increased by 4.7% in the control group, showed a mean increase of 39.7% after 12 months for the high-dose group.
The company described the data as “promising” and said it supported the continued clinical development of AMT-130 and that it was pursuing regulatory interactions to discuss its late-stage development.
“We plan to engage with regulators to advance this promising clinical program as we collect more data from these patients and from our European study,” uniQure president of research and development Dr. Ricardo Dolmetsch commented.
uniQure stock was down 43.9% at US$10.90 on Wednesday morning.
Contact the author at emily.jarvie@proactiveinvestors.com
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