uniQure Announces Dosing of First Patient in Phase I/II

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) — uniQure NV (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with serious medical needs, today announced that the first patient has been treated 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 multicenter, open-label, Phase I/II study being conducted in the United States with three dose-escalating cohorts to evaluate the safety, tolerability, and exploratory evidence of efficacy of AMT-162 in individuals with SOD1-ALS .

“We are pleased to announce the first patient dosing of AMT-162, our experimental gene therapy for the treatment of SOD1-ALS, a debilitating, degenerative and fatal disease,” said Walid Abi-Saab, MD, Chief Medical Officer of uniQure. “The initiation of this study marks the advancement of our third gene therapy program in the clinic with this study design and continues our goal of rapidly generating proof-of-concept data using proven biomarkers to bring treatments to patients as quickly as possible. ” possible. We believe our novel AAV-based gene therapy candidate can provide the convenience of single dosing with the potential for a differentiated efficacy profile required for such a devastating disease.”

AMT-162 is an experimental AAVrh10-based gene therapy that expresses a miRNA designed to suppress expression of the mutant SOD1 protein. Patients with SOD1-ALS express a misfolded SOD1 protein. This misfolded protein is toxic to motor neurons and causes degeneration that, over time, leads to muscle weakness, loss of function and eventual death. AMT-162 may provide a novel, one-time, intrathecally administered approach to slowing or stopping the progression of SOD1-ALS. AMT-162 has received both orphan drug designation and fast track designation from the U.S. Food and Drug Administration.

The EPISOD1 Phase I/II clinical trial of AMT-162 is being conducted in the United States. The multicenter, open-label study consists of three cohorts of up to four patients each who will receive a short course of immunosuppression before and after an intrathecal infusion of AMT-162. The study will examine the safety and tolerability of AMT-162 and identify early signs of effectiveness by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein. There are currently four active locations in the US and plans to activate seven additional locations by the first quarter of 2025. Further details can be found at www.clinicaltrials.gov (NCT06100276).

About amyotrophic lateral sclerosis (ALS), caused by mutations in superoxide dismutase 1 (SOD1)
SOD1-ALS is a rare, progressive and fatal neurodegenerative disease that causes loss of motor neurons in the brain and spinal cord. As ALS progresses, sufferers experience muscle weakness and atrophy, causing them to lose the ability to move their arms and legs, speak, swallow, and eventually breathe, leading to respiratory failure. Based on a 2021 study in Neuroepidemiology, it is estimated that there are approximately 170,000 people with ALS worldwide, with SOD1 mutations accounting for 2% of this population. According to the National Institute of Neurological Disorders and Stroke, the average life expectancy of those diagnosed with ALS is three to five years from the onset of symptoms.

About uniQure

uniQure delivers on the promise of gene therapy – single treatments with potentially curative results. The approval of uniQure’s gene therapy for hemophilia B – a historic achievement based on more than a decade of research and clinical development – represents an important milestone in the field of genomic medicine and initiates a new treatment approach for patients with hemophilia. uniQure is currently developing a pipeline of proprietary gene therapies to treat patients with Huntington’s disease, refractory temporal lobe epilepsy, ALS, Fabry disease and other serious diseases. www.uniQure.com

Forward-Looking Statements by uniQure

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements and are often identified by words such as “anticipate,” “believe,” “could,” “determine,” “estimate,” “expect.” ”, “Target” etc. are marked. “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “will,” “would,” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements regarding the availability of proof-of-concept data in the Company’s open-label Phase I/II clinical trial for SOD1-ALS in the United States; the effectiveness of the EPISOD1 study design; and the potential efficacy profile of AMT-162 through single administration with the ability to slow or stop the progression of SOD1-ALS. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, but are not limited to, risks related to the Company’s clinical results and the development and timing of its programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to establish and maintain the corporate infrastructure and personnel necessary to achieve its objectives; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefit of its gene therapy candidates in clinical trials; the Company’s ability to obtain, retain and protect its intellectual property; and the Company’s ability to finance its operations and raise additional capital as needed. These risks and uncertainties are described in more detail under the heading “Risk Factors” in the Company’s periodic filings with the U.S. Securities and Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed on February 28, 2024. its quarterly reports on Form 10-Q filed on May 7, 2024 and August 1, 2024, and other filings the Company makes from time to time with the SEC. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company undertakes no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure contacts:
FOR INVESTORS:	FOR MEDIA:
Chiara Russo	Tom Malone
Direct: 617-306-9137	Direct: 339-970-7558
Cell: 617-306-9137	Cell: 339-223-8541
[email protected]	[email protected]

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