uniQure N.V. - QURE STOCK NEWS

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.

uniQure N.V. (NASDAQ: QURE) announced that the U.S. FDA has granted Orphan Drug Designation to AMT-191, their investigational gene therapy for Fabry disease. This follows the dosing of the first patient in a U.S. multi-center, open-label Phase I/IIa trial in August 2024. AMT-191 is a one-time intravenously administered AAV5-based gene therapy targeting the liver to produce GLA protein.

The Phase I/IIa trial will include two cohorts of up to six adult male patients each, with low and high doses. Patients will be followed for 24 months to assess safety, tolerability, and early efficacy signs. The Orphan Drug Designation provides incentives including tax credits, grants, fee waivers, and seven years of market exclusivity upon approval.

uniQure N.V. (NASDAQ: QURE) has initiated dosing in a Phase I/IIa clinical trial of AMT-191 for Fabry disease treatment. The multi-center, open-label trial in the US will assess safety, tolerability, and early efficacy signs in two dose-escalating cohorts. AMT-191 is an AAV5-based gene therapy delivering a galactosidase alpha (GLA) transgene to the liver. It aims to address the α-galactosidase A enzyme deficiency in Fabry patients.

The trial includes two cohorts of up to six adult male patients each, with low (6x10^13 gc/kg) and high (3x10^14 gc/kg) doses administered intravenously. Patients will be followed for 24 months. This milestone aligns with uniQure's goal to advance three new gene therapies into clinical studies this year, alongside programs in Huntington's disease, temporal lobe epilepsy, and SOD1-ALS.

uniQure (NASDAQ: QURE) reported its Q2 2024 financial results and provided a company update. Key highlights include:

1. RMAT designation for AMT-130 in Huntington's disease and positive interim Phase I/II data showing slowing of disease progression.

2. Initiated patient screening for three Phase I/II studies in epilepsy, ALS, and Fabry disease.

3. Announced organizational restructuring, reducing headcount by 65% and lowering cash burn by $75 million annually.

4. Strong cash position of $524 million as of June 30, 2024, expected to fund operations through 2027.

5. Q2 2024 revenue of $11.1 million, up from $2.4 million in Q2 2023.

6. Net loss of $56.3 million ($1.16 per share) in Q2 2024, compared to $68.5 million ($1.44 per share) in Q2 2023.

uniQure has closed the sale of its global manufacturing facility in Lexington, Massachusetts to Genezen, a contract development and manufacturing organization. This strategic move is expected to reduce uniQure's annual cash burn by $40 million and streamline operations while maintaining preferential access to gene therapy manufacturing capabilities.

Key points:

• uniQure becomes a significant shareholder in Genezen
• CEO Matt Kapusta joins Genezen's Board of Directors
• $50 million in outstanding debt retired
• Amin Abujoub appointed as new Chief Technical Operations Officer
• Chief Operating Officer role eliminated

The company aims to focus future investments on projects with potential to increase shareholder value and plans to announce additional cost reduction measures later this quarter.

uniQure announced positive interim data from Phase I/II trials of AMT-130 for Huntington's disease, showing a statistically significant, dose-dependent slowing in disease progression. At 24 months, high-dose patients demonstrated an 80% slowing of disease progression compared to a propensity score-weighted external control. Additionally, a significant reduction in CSF neurofilament light protein (NfL) levels was observed. The trials also indicated that AMT-130 remains well-tolerated with no new serious adverse events. uniQure aims to meet with the FDA in the second half of 2024 to discuss expedited clinical development pathways for AMT-130.

uniQure announced the sale of its manufacturing facility in Lexington, Massachusetts, to Genezen for $25 million, consisting of $12.5 million in Series C Preferred Stock and a $12.5 million convertible note. This divestiture will allow uniQure to reduce its annual cash burn by $40 million and is part of a broader strategy to cut operating expenses. Genezen will now manufacture HEMGENIX® and support uniQure's pipeline programs, with the majority of current employees transitioning to Genezen. The deal is expected to close in early Q3 2024, and uniQure will use the proceeds to repay $50 million in debt, further saving on interest expenses.

Genezen announced its acquisition of uniQure's commercial gene therapy operations in Lexington, MA. The acquisition includes a state-of-the-art, commercially-licensed viral vector manufacturing facility, enhancing Genezen's ability to provide late-phase and commercial gene therapy development services. The Lexington site will become Genezen's global AAV center of excellence, complementing its existing operations in Indianapolis. Genezen will also enter into strategic supply agreements for uniQure's clinical portfolio and CSL's HEMGENIX® product. The transaction is supported by additional growth equity from Ampersand Capital Partners.

uniQure has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy, AMT-130, for Huntington’s disease. This designation is based on 24-month interim data from Phase I/II clinical trials released in December 2023 and represents a significant milestone.

AMT-130 is the first therapy for Huntington’s disease to receive RMAT designation, facilitating closer collaboration with the FDA and potentially expediting its development and approval process. This decision underscores AMT-130's potential to address the unmet medical needs of Huntington’s disease patients.

uniQure plans to present updated interim data from ongoing Phase I/II studies in mid-2024, including follow-up results on 29 treated patients, 21 of whom will have data spanning at least two years.