Uniqure Stock Price, News & Analysis

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.

uniQure (Nasdaq: QURE) has announced the pricing of its public offering of 4,411,764 ordinary shares at $17.00 per share, expecting to raise approximately $75 million in gross proceeds before deducting underwriting costs and expenses. The company has also granted underwriters a 30-day option to purchase up to 661,764 additional shares at the same price.

The offering is expected to close around January 10, 2025. Leerink Partners, Stifel, and Guggenheim Securities are serving as bookrunning managers, while Chardan and H.C. Wainwright & Co. are acting as lead managers for the offering. The shares are being offered through an automatically effective shelf registration statement filed with the SEC on January 7, 2025.

uniQure (Nasdaq: QURE) has announced the launch of an underwritten public offering of ordinary shares and pre-funded warrants to purchase ordinary shares. The gene therapy company plans to grant underwriters a 30-day option to purchase up to an additional 15% of ordinary shares at the public offering price, less underwriting discounts and commissions.

Leerink Partners is serving as the bookrunning manager for the proposed offering. The securities will be offered through an automatically effective shelf registration statement on Form S-3 filed with the SEC on January 7, 2025. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

uniQure has reached an agreement with the FDA on key elements for an Accelerated Approval pathway for AMT-130, their Huntington's disease treatment. The FDA agreed that data from ongoing Phase I/II studies compared to natural history external control can serve as the primary basis for a Biologics License Application (BLA), eliminating the need for additional pre-submission studies.

The FDA accepted the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an intermediate clinical endpoint and acknowledged that reductions in neurofilament light chain (NfL) in cerebrospinal fluid may support evidence of therapeutic benefit. This follows the RMAT designation granted in May 2024 and promising interim data from July 2024 showing durable, dose-dependent slowing of disease progression.

uniQure has announced the dosing of the first patient in its GenTLE Phase I/IIa clinical trial of AMT-260, targeting refractory mesial temporal lobe epilepsy (MTLE). The trial evaluates a one-time gene therapy treatment using an AAV9 vector that delivers engineered miRNAs to reduce GluK2 protein expression, which is believed to trigger seizures in MTLE patients. The study will include two dose cohorts of six patients each across 10 active sites, with two additional sites planned by end of 2024. Preclinical studies showed AMT-260 reduced seizure frequency in a dose-dependent manner and decreased GluK2 expression in both animal models and patient tissue samples.

uniQure reported Q3 2024 financial results with a strong cash position of $435.2 million, extending runway through 2027. The company announced positive interim data from AMT-130 trials in Huntington's disease, showing significant disease progression slowing. A Type B FDA meeting is scheduled for Q4 2024 to discuss potential expedited development. The company initiated patient dosing in new Phase I/II studies for SOD1-ALS and Fabry disease treatments.

Q3 net loss was $44.4 million ($0.91 per share), improved from $89.6 million loss in Q3 2023. The company completed organizational restructuring, including the sale of its Lexington facility, expected to reduce annual cash burn by $70 million and eliminate 65% of roles.

uniQure N.V. (NASDAQ: QURE) has announced the dosing of the first patient in the Phase I/II clinical trial of AMT-162 for the treatment of SOD1-ALS, a rare inherited form of amyotrophic lateral sclerosis. The EPISOD1 trial is a multi-center, open-label study with three dose-escalating cohorts, designed to assess the safety, tolerability, and exploratory efficacy of AMT-162.

AMT-162 is an AAVrh10-based gene therapy that aims to knock down the expression of mutated SOD1 protein, which is toxic to motor neurons. The therapy, administered intrathecally as a one-time treatment, has received Orphan Drug status and Fast Track designation from the FDA.

The trial will be conducted in the United States, with four active sites and plans to activate seven more by Q1 2025. It will measure neurofilament light chain and SOD1 protein levels as exploratory efficacy markers.