UniQure stock soars on Huntington’s gene therapy data

Shares of UniQure surged over 60% on Tuesday morning following the Netherlands-based biotech company's latest update on its gene therapy for Huntington's disease. The company has been sharing data from two small-scale trials evaluating both low and high doses of the therapy, known as AMT-130, in patients with early stages of the disease. By the end of last year, UniQure indicated that both dosage levels appeared to maintain or enhance neurological function.

Despite UniQure's optimistic portrayal of these findings, investors had remained skeptical. AMT-130 aims to inhibit the production of huntingtin, a protein that is typically beneficial for brain function but can mutate and become toxic, leading to Huntington's disease. However, the therapy's efficacy in reducing mutant huntingtin has been inconsistent, with the low dose sometimes showing greater effectiveness than the high dose.

The latest update from UniQure concerns data collected up to March 31. At this point, the company had accumulated two years of data from 21 participants who received AMT-130—12 on the low dose and nine on the high dose. Previously, UniQure had disclosed data from an 18-month follow-up for the high-dose group.

According to UniQure, the new results demonstrate that AMT-130 is slowing the progression of Huntington's in a dose-dependent manner, meaning the higher dose has proven more effective. At the 24-month mark, the higher dose had reportedly slowed disease progression by 80% in comparison to a natural history cohort of patients with early Huntington's disease, who were similar to the study participants. Those receiving the higher dose maintained roughly the same motor and cognitive functions as when the study commenced. In contrast, the lower dose slowed the disease by 30% over the same period. The comparison to the natural history cohort is a post hoc analysis, which tends to be less statistically robust.

Additionally, UniQure reported that participants receiving AMT-130 experienced a notable reduction in neurofilament light chain, a protein indicative of brain and nervous system damage. Levels of this protein in the cerebrospinal fluid dropped by an average of 11% two years post-treatment compared to baseline levels. This finding is significant as neurofilament light chain has become an important biomarker for therapies targeting neurodegenerative diseases. For example, Biogen received approval for its ALS treatment, Qalsody, owing to its effect on this protein.

Earlier data from UniQure’s trials had shown that neurofilament levels declined by an average of 6.6% at 30 months for participants on the low dose, with little to no reduction observed at the 18-month mark for those on the high dose.

Despite initially low expectations from investors regarding UniQure's program, the new data appears promising and generally positive, as noted by Paul Matteis, an analyst at the investment bank Stifel. The primary question now is how UniQure plans to bring its therapy to market.

UniQure aims to meet with the Food and Drug Administration (FDA) before the end of the year to present the updated data and discuss potential accelerated clinical development and approval pathways. The company also plans to present safety data in the first half of 2025. Additionally, UniQure anticipates releasing another interim analysis from its AMT-130 studies in mid-2025, which will include three-year data.