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uniQure N.V. reports company news on its gene therapy pipeline, regulatory interactions, clinical data, operating results, and capital structure. The company develops genetic medicines for severe diseases, with disclosed programs including AMT-130 for Huntington’s disease, AMT-260 for refractory mesial temporal lobe epilepsy, AMT-191 for Fabry disease, ALS programs, and an approved gene therapy for hemophilia B.
Recurring updates cover FDA and MHRA discussions, clinical-trial observations, exploratory efficacy and safety data, quarterly and annual financial results, debt financing arrangements, corporate presentations, and governance or shareholder matters tied to its Nasdaq-listed public-company status.
uniQure N.V. (NASDAQ: QURE) announced six data presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting from May 16-19, 2022, in Washington, D.C. Highlights include an oral presentation on alpha-synuclein lowering in Parkinson's models and several poster sessions covering advancements in AAV gene therapies. Dr. Ricardo Dolmetsch emphasized the innovative potential of the linQURE® cargo platform in silencing mutant C9orf72 in ALS preclinical models. The event underscores uniQure's commitment to advancing gene therapies for severe medical conditions.
uniQure N.V. (QURE) reported Q1 2022 financial results, with revenues of $1.8 million, up from $0.5 million in Q1 2021, driven by collaboration revenue from CSL Behring. The company received $55 million in milestone payments related to the regulatory progress of etranacogene dezaparvovec for hemophilia B, which was validated by the EMA for accelerated assessment. R&D expenses rose to $45 million, while SG&A expenses decreased to $11 million. The net loss was $46.7 million, or $1.00 per share. Cash and equivalents stood at $524.9 million, projected to fund operations into H1 2025.
CSL Behring's Marketing Authorization Application (MAA) for etranacogene dezaparvovec has been accepted for accelerated assessment by the EMA, potentially making it the first gene therapy for hemophilia B. Supported by the phase 3 HOPE-B study, which showed a 64% reduction in annual bleeds after a single infusion, this breakthrough therapy aims to significantly improve patients' lives. Etranacogene dezaparvovec addresses the underlying genetic cause of hemophilia B, offering hope for a transformative treatment option in the EU and EEA.
uniQure N.V. (NASDAQ: QURE) announced the completion of enrollment for the first two cohorts in its Phase I/II clinical trial of AMT-130, targeting early-stage Huntington's disease. A total of 26 patients were enrolled across two cohorts, with initial results expected in Q2. This trial aims to evaluate the safety and efficacy of AMT-130, a gene therapy designed to reduce harmful protein levels associated with the disease. A third cohort is planned to refine the administration procedure. The trial advances uniQure's commitment to innovative gene therapies addressing critical medical needs.
uniQure N.V. (QURE) reported a significant revenue increase to $524 million for 2021 from $37.5 million in 2020, driven primarily by a $462.4 million license revenue from its CSL Behring agreement. The company ended 2021 with $556 million in cash, sufficient to fund operations into 2025. Positive clinical data from the HOPE-B trial demonstrated the efficacy of etranacogene dezaparvovec for hemophilia B, with a 64% reduction in annualized bleeding rate. Ongoing studies for Huntington’s Disease and new therapies for refractory temporal lobe epilepsy and Fabry disease are advancing, maintaining a strong pipeline.
uniQure announced the dosing of the first two patients in its European Phase Ib/II clinical trial of AMT-130, a gene therapy for Huntington’s disease. The trial will include 15 patients across Poland, the UK, and Germany. Dr. Ricardo Dolmetsch reported that patient enrollment is expected to finish by year-end, with preliminary data anticipated in Q2 2022. The innovative AMT-130 aims to silence the huntingtin gene using a proprietary AAV5 vector, marking a significant step in gene therapy advancements for neurodegenerative disorders.
CSL Behring released positive long-term results from the Phase 3 HOPE-B trial of etranacogene dezaparvovec (EtranaDez), a gene therapy for hemophilia B. The study showed a stable increase in mean Factor IX (FIX) activity, reaching 36.9 IU/dL at 18 months post-infusion. Participants experienced a 64% reduction in annual bleeding rates and 98% discontinued prophylactic treatment. The therapy was generally well-tolerated, though one unrelated death occurred. This data reinforces the potential of EtranaDez to transform hemophilia B management and may accelerate regulatory reviews in the U.S. and Europe.
uniQure N.V. (NASDAQ: QURE) reported positive preliminary data from its Phase I/II trial of AMT-130 for Huntington's disease. Treatment was well tolerated, with no serious adverse events noted in the first four patients at one year of follow-up. Neurofilament Light Chain levels returned to baseline in treated patients. A total of 19 procedures have been completed, and higher-dose cohort enrollment is progressing. An open-label study in Europe has also commenced, aiming to further assess safety and efficacy. Upcoming data releases are expected in 2022 and beyond.
CSL Behring announced that the European Medicines Agency's CHMP has accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorization Application (MAA) for hemophilia B. This gene therapy, currently in Phase 3 trials, could provide a significant long-term treatment option for patients. The accelerated review may cut the evaluation timeline from 210 to 150 days. The company aims to submit the MAA in the first half of 2022, highlighting their commitment to innovative treatments and addressing the unmet needs of hemophilia B patients.
uniQure's etranacogene dezaparvovec gene therapy has achieved significant clinical milestones in the pivotal Phase III HOPE-B trial for hemophilia B. The therapy met its primary endpoint by demonstrating non-inferiority in annualized bleeding rate (ABR) compared to standard FIX prophylactic therapy after 18 months, with an ABR of 1.51 versus 4.19 in the lead-in period. Additionally, it achieved statistical superiority in bleeding rate reduction (p=0.0002). FIX activity remained stable, averaging 36.9% of normal levels at 18 months. CSL Behring plans to file for regulatory approval in the U.S. and EU in 2022.