Uniqure Stock Price, News & Analysis

uniQure (NASDAQ: QURE) has announced it will report its first quarter 2025 financial results on Friday, May 9, 2025, before market open. The company will host its inaugural quarterly earnings conference call at 8:30 a.m. ET on the same day. This initiative to begin regular earnings calls comes as uniQure advances its pipeline and AMT-130 toward a BLA submission and potential commercialization, aiming to enhance engagement with the investment community.

The event will be accessible via webcast through uniQure's website, with a 90-day replay available afterward. Phone participants must pre-register through an online form to receive dial-in details and are advised to join 15 minutes before the start time.

uniQure (NASDAQ: QURE) has received FDA Breakthrough Therapy designation for AMT-130, its gene therapy treatment for Huntington's disease, a rare inherited neurodegenerative disorder with no current disease-modifying therapies.

The designation is based on promising interim data from Phase I/II trials showing dose-dependent slowing of disease progression. The company presented 24-month data in July 2024, demonstrating meaningful disease progression slowdown based on cUHDRS scores compared to natural history.

AMT-130 has already received Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations. The treatment has been administered to 45 patients to date, with additional regulatory guidance on the Biologics License Application expected in Q2 2025.

uniQure (NASDAQ: QURE) reported its 2024 financial results and company progress. Key highlights include FDA alignment on Accelerated Approval pathway for AMT-130 in Huntington's disease and completion of patient enrollment in its third cohort Phase I/II study. The company initiated dosing in Phase I/II studies for multiple programs including AMT-260 for epilepsy, AMT-191 for Fabry disease, and AMT-162 for SOD1-ALS.

Financial position shows cash and equivalents of $367.5 million as of December 31, 2024, plus $80.7 million from recent financing, expected to fund operations into second half of 2027. Revenue increased to $27.1 million in 2024 from $15.8 million in 2023. The company reported a net loss of $239.6 million ($4.92 per share) compared to $308.5 million ($6.47 per share) in 2023.

CSL Behring announced four-year results from the pivotal HOPE-B study of HEMGENIX, their gene therapy for hemophilia B. The data demonstrates sustained efficacy and safety through four years post-treatment. Key findings include:

- 94% of patients remained free of continuous factor IX prophylaxis
- Mean factor IX activity levels maintained at 37% through four years
- Approximately 90% reduction in mean adjusted annualized bleeding rate from lead-in to year four
- Joint bleeds reduced from 2.34 to 0.09 mean ABR

The Phase III HOPE-B trial involved 54 adult male participants with severe or moderately severe hemophilia B. No serious adverse events related to HEMGENIX were reported, with 96% of treatment-related adverse events occurring in the first six months. HEMGENIX, approved by FDA in 2022, is the first gene therapy for hemophilia B that can treat patients with and without AAV5 neutralizing antibodies.

uniQure (NASDAQ: QURE) has announced the completion of enrollment in the first cohort of its Phase I/IIa trial for AMT-191, a gene therapy treatment for Fabry disease. The Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients and found no significant safety concerns, recommending progression to the second cohort.

The company plans to begin enrollment for the second dose cohort in Q1 2025. AMT-191 is an AAV5-based gene therapy utilizing a proprietary promoter to deliver a galactosidase alpha (GLA) transgene targeting the liver to produce GLA protein. This one-time intravenous treatment aims to address Fabry disease, a condition where GLA gene variants cause α-galactosidase A enzyme deficiency, leading to lipid accumulation in multiple cell types and resulting in a multi-system disorder.

uniQure (NASDAQ: QURE) announced a favorable recommendation from the Independent Data Monitoring Committee (IDMC) for its Phase I/II EPISOD1 clinical trial of AMT-162, a gene therapy for SOD1-ALS. The IDMC reviewed 28-day safety data from the first study cohort and found no significant safety concerns, recommending progression to the second cohort.

AMT-162 is an AAVrh10-based gene therapy designed to reduce mutated SOD1 protein expression through miRNA. The therapy targets SOD1-ALS, where patients produce misfolded SOD1 protein that damages motor neurons, leading to muscle weakness and eventual death. The treatment is administered intrathecally as a one-time dose, aiming to slow or stop SOD1-ALS progression. The company plans to begin enrollment for the second dose cohort in Q1 2025.