uniQure N.V. (NASDAQ: QURE) is advancing its gene therapy pipeline, with lead candidate AMT-130 showing promising results in Huntington's disease. The company is scheduled to meet with the FDA to discuss potential accelerated development pathways for AMT-130. Recent corporate updates also include the initiation of new clinical studies and strategic capital preservation initiatives.
AMT-130 for Huntington's Disease
uniQure is focusing on AMT-130 for the treatment of Huntington's disease. The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AMT-130, and a Type B meeting with the FDA is scheduled for late November. The company plans to present the latest clinical data and discuss the potential for an expedited development pathway.
In July 2024, uniQure announced positive interim data from the ongoing U.S. and European Phase I/II studies of AMT-130. At 24 months, the data demonstrated a statistically significant, dose-dependent slowing in disease progression measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients receiving the high dose of AMT-130 compared to a propensity score-weighted external control (p=0.007). Additionally, a statistically significant reduction of neurofilament light chain (NfL) in cerebrospinal fluid (CSF) was observed in patients dosed with AMT-130 compared to baseline (p=0.02). AMT-130 has been generally well-tolerated with a manageable safety profile across both doses.
Patient dosing is ongoing in a third cohort of up to 12 patients to further evaluate both doses of AMT-130 together with an immunosuppression regimen, with a focus on evaluating near-term safety and tolerability. Enrollment in this third cohort is expected to be completed in the fourth quarter of 2024. An additional interim update from the Phase I/II clinical trials of AMT-130 is expected in mid-2025, including three years of follow-up data on 21 treated patients.
Expansion of Clinical Studies
uniQure has initiated new Phase I/II clinical studies for other gene therapy candidates:
- AMT-191 for Fabry Disease: The first patient was dosed in August 2024. AMT-191 has been granted Orphan Drug and Fast Track designations. The U.S., multi-center, open-label trial is expected to include up to 12 adult male patients across two dose cohorts.
- AMT-162 for SOD1 Amyotrophic Lateral Sclerosis (ALS): The first patient was dosed in October 2024. The U.S., multi-center, open-label trial is expected to include up to 12 patients across three dose cohorts.
- AMT-260 for Refractory Mesial Temporal Lobe Epilepsy (mTLE): The first patient has been enrolled into the observational phase of the Phase I/II clinical trial. Enrollment has been slower than expected due to restrictive inclusion criteria for the initial sentinel patients. The company is working to activate additional recruitment sites.
Capital Preservation Initiatives
uniQure has taken steps to streamline operations and preserve capital. In July 2024, the company sold its Lexington, MA manufacturing facility to Genezen. In August 2024, an organizational restructuring was announced, expected to eliminate approximately 65% of roles across the organization and reduce recurring cash burn by $70 million per year. These measures have extended the company's cash runway through the end of 2027.
As of September 30, 2024, uniQure held approximately $435 million in cash, cash equivalents, and investment securities. The company retired $50 million of its outstanding debt with Hercules Capital in July 2024, which is expected to reduce annual interest expense by approximately $5 million.
