uniQure Announces Recommendations from Data Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease | 2020-09-25 | Press Releases – About Your Online Magazine

LEXINGTON, Massachusetts and AMSTERDAM, September 25, 2020 (GLOBE NEWSWIRE) – uniQure N.V. (NASDAQ: QURE), a leading gene therapy company that promotes transformative therapies for patients with serious medical needs, today announced that the independent Data Security Monitoring Council (DSMB) overseeing the AMT- Phase I / II clinical trial 130 for the treatment of Huntington’s disease & CloseCurlyQuote; s found and reviewed the 90-day safety data of the first two patients enrolled in the study. No significant safety concerns were noted to avoid additional dosing, and the next two patients are now cleared for inclusion in the study. The Phase I / II study is a randomized double-blind clinical trial conducted in the United States. One patient was treated with AMT-130 and one patient received imitation surgery.

“We are very pleased with the positive outcome of this first DSMB meeting, & CloseCurlyDoubleQuote; said Ricardo Dolmetsch, president of research and development at uniQure. “We are now going to advance the study and hope to enroll the next two patients as soon as possible. & CloseCurlyDoubleQuote;

About the AMT-130 Phase I / II Clinical Trial

The AMT-130 Phase I / II clinical trial for the treatment of Huntington’s disease & CloseCurlyQuote; s will explore the signs of safety, tolerability and efficacy in 26 patients with early Huntington & CloseCurlyQuote disease randomly manifested for treatment with AMT-130 or an imitation (simulation) surgery. The five-year multicenter study consists of a 12-month blind study period followed by long-term, non-blind follow-up. Patients will receive a single administration of AMT-130 through stereotactic neurosurgical application guided by magnetic resonance enhanced by convection directly into the striatum (caudate and putamen). Additional details are available at www.clinicaltrials.gov (NCT04120493).

AMT-130 is the first uniQure & CloseCurlyQuote clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE ™ platform.

About Huntington’s disease and CloseCurlyQuote; s

Huntington’s disease & CloseCurlyQuote; s is a rare inherited neurodegenerative disease that leads to motor symptoms, including chorea and behavioral abnormalities and cognitive decline, resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with an expansion of the disease-causing CAG repeat in the first exon of the huntingtin gene that leads to abnormal protein production and aggregation in the brain. Despite the clear etiology of Huntington’s disease & CloseCurlyQuote; s, there are currently no approved therapies to delay the onset or slow the progression of the disease & CloseCurlyQuote; s.

About uniQure

uniQure is delivering on the promise of gene therapy – unique treatments with potentially healing results. We are taking advantage of our modular and validated technology platform to rapidly pipeline of proprietary genetic therapies to treat patients with hemophilia B, Huntington’s disease, Fabry’s disease, type 3 spinocerebellar ataxia and other diseases. www.uniQure.com

Forward-looking statements from uniQure

This press release contains forward-looking statements. All statements, except statements of historical facts, are forward-looking statements, often indicated by terms such as “anticipate”, “believe”, “could”, “estimate”, “hope”, “objective”, “intend” “looking forward to “,” can “,” plan “,” potential “,” predict “,” project “,” should “,” will “,” would “and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, if we will be able to enter the next two patients inside the clinical trial . Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our company and the economy in general and the healthcare system, The you clinical development activities, clinical outcomes, collaboration arrangements, regulatory oversight, product marketing and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading “Risk factors” in uniQure & CloseCurlyQuote; s periodic filings of securities, including its Annual Report on Form 10-K filed on March 2, 2020 and Quarterly Report on Form 10-Q filed on July 30 , 2020 . Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements and we assume no obligation to update these forward-looking statements, even if new information is made available in the future.

UniQure contacts:

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Paula Fonseca