Articles from uniQure Inc.

~ Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort ~

LEXINGTON, Mass. and AMSTERDAM, Jan. 08, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the pricing of its underwritten public offering of 4,411,764 of its ordinary shares at a public offering price of $17.00 per share. The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, are expected to be approximately $75 million. All securities to be sold in the offering are being sold by uniQure. In addition, uniQure has granted to the underwriters a 30-day option to purchase up to 661,764 additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is expected to close on or about January 10, 2025, subject to the satisfaction of customary closing conditions.

LEXINGTON, Mass. and AMSTERDAM, Jan. 07, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it has commenced an underwritten public offering of its ordinary shares and pre-funded warrants to purchase its ordinary shares. All securities to be sold in the offering will be offered by uniQure. In addition, uniQure intends to grant the underwriters a 30-day option to purchase up to 15% additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

~ U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval ~

LEXINGTON, Mass. and AMSTERDAM, Nov. 21, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE).

~ Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington’s disease ~

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-ALS.

LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024, uniQure announced the dosing of the first patient in its U.S., multi-center, open-label Phase I/IIa trial of AMT-191.

LEXINGTON, Mass. and AMSTERDAM, Aug. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease, a rare, inherited genetic disease. The Phase I/IIa study is a multi-center, open-label trial being conducted in the United States with two dose-escalating cohorts assessing the safety, tolerability and early signs of efficacy of AMT-191 in individuals with Fabry disease.

~ Announced RMAT designation for AMT-130 in Huntington’s disease and positive interim Phase I/II data demonstrating the slowing of disease progression and reductions in a key biomarker of neurodegeneration; Meeting with FDA expected in the second half of 2024 to discuss potential for expedited clinical development ~

~ uniQure maintains preferential access to industry-leading manufacturing capabilities to support its pipeline of gene therapy candidates ~

~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control ~

~ Proposed divestiture streamlines uniQure’s focus and represents an important milestone in its ongoing effort to significantly reduce operating expenses ~

~ Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 ~

~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~

~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~

LEXINGTON, Mass. and AMSTERDAM, Dec. 08, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the grant of inducement equity awards to newly hired employees.

Patient enrollment expected to begin in first half of 2024

~ Announced FDA clearance of Investigational New Drug (IND) application for AMT-260 in refractory mesial temporal lobe epilepsy ~

LEXINGTON, Mass. and AMSTERDAM, Oct. 24, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium.

~ Reduction of 28% of workforce not related to HEMGENIX® manufacturing obligations; Total cost savings of $180 million to extend cash runway into second quarter of 2027 ~

LEXINGTON, Mass. and AMSTERDAM, Oct. 02, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the grant of inducement equity awards to newly hired employees.

Clinical trial initiation expected in the fourth quarter of 2023

~ Announced encouraging interim analysis data from ongoing Phase I/II clinical trial of AMT-130 in Huntington’s disease showing preservation of function compared to baseline and clinical benefits relative to natural history; both dose cohorts of AMT-130 continue to be generally well-tolerated ~

~ Walid Abi-Saab, M.D., appointed Chief Medical Officer, will bring extensive track record in leading drug development and execution ~

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~

Milestone payment triggered by first commercial sale of HEMGENIX® in U.S. by CSL Behring

LEXINGTON, Mass. and AMSTERDAM, June 16, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the grant of inducement equity awards to Jeannette Potts, the Company’s newly hired Chief Legal and Compliance Officer, and other newly hired employees.

~ uniQure to receive $375 million upfront cash payment ~

~ Clinical update from U.S. Phase I/II trial in Huntington’s disease expected in second quarter 2023 ~

LEXINGTON, Mass. and AMSTERDAM, May 02, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting being held May 16-20 in Los Angeles, California.

LEXINGTON, Mass. and AMSTERDAM, May 02, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the Company granted equity awards to 38 employees as a material inducement to commencing their employment. The equity awards were approved by the Company’s board of directors on April 24, 2023 (the “Grant Date”) in accordance with Nasdaq Listing Rule 5635(c)(4).

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 13, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the Company granted equity awards to 31 employees as a material inducement to commencing their employment. The equity awards were approved by the Company’s board of directors on February 23, 2023 (the “Grant Date”) in accordance with Nasdaq Listing Rule 5635(c)(4).

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 02, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in the following upcoming investor and scientific conferences:

~ Achieved U.S. and European Commission marketing approvals of first and only gene therapyfor adults with hemophilia B ~

~ Data follow the historic approval of the first gene therapy for hemophilia B, which has been shown in clinical trials to reduce the rate of annual bleeds, reduce or eliminate the need for prophylactic therapy and generate elevated and sustained factor IX levels for years after a one-time infusion ~

~ Historic approval represents the first gene therapy in Europe to treat hemophilia B and provides a new treatment option for patients that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and generates elevated and sustained factor IX levels ~

 ~ License of APB-102 further strengthens uniQure’s pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~

LEXINGTON, Mass. and AMSTERDAM, Dec. 20, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the Company granted equity awards to employees as a material inducement to commencing their employment. The equity grants were approved by the Company’s board of directors on December 7, 2022, October 31, 2022, and September 27, 2022 (the “Grant Dates”) each in accordance with Nasdaq Listing Rule 5635(c)(4).

If approved, etranacogene dezaparvovec would be the first licensed gene therapy in Europe for people living with hemophilia B

~ IND Submission and Clinical Development Expected to Begin in 2023 ~

~ Historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and generates elevated and sustained factor IX levels ~

~ Event to be Webcast Live on uniQure’s Corporate Website at 8:30 a.m. EST ~

~ Patient enrollment at the higher dose of AMT-130 to resume in European Phase Ib/II study following Data Safety Monitoring Board recommendation ~~ U.S. and European pre-approval inspections of Company’s Lexington, MA manufacturing site completed with EMA issuing Good Manufacturing Certificate ~~ Virtual investor event focused on next pipeline program, AMT-260 investigational gene therapy for refractory temporal lobe epilepsy, to be held on Tuesday, November 29, 2022 ~

Eight Posters Highlighting Pre-Clinical Programs and Leading Gene Therapy Manufacturing and Administration Capabilities

LEXINGTON, Mass. and AMSTERDAM, Oct. 03, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in the following upcoming investor and scientific conferences: