uniQure Proclaims Up to date Preliminary AMT-191 Section I/IIa Knowledge Exhibiting Sustained Will increase in α-Gal A Enzyme Exercise in Sufferers with Fabry Illness – Worldwide Dispatch

~ Supraphysiological expression of α-Gal A exercise maintained for over a 12 months in longest handled affected person as of knowledge cutoff date ~

~ Steady Lyso-Gb3 ranges maintained post-dosing, no matter enzyme alternative remedy standing throughout all cohorts ~

~ Six of 11 sufferers have discontinued enzyme alternative remedy as of knowledge cutoff date ~

LEXINGTON, Mass. and AMSTERDAM, Feb. 06, 2026 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a number one gene remedy firm advancing transformative therapies for sufferers with extreme medical wants, in the present day introduced up to date preliminary security and exploratory efficacy knowledge from 11 sufferers in its Section I/IIa trial of AMT-191, an investigational AAV gene remedy for the remedy of Fabry illness. The up to date knowledge was offered on the WORLDSymposium in San Diego, California.

As of the January 8, 2026 research knowledge cutoff date, all 11 sufferers within the three dose cohorts (6×10¹³ genome copies/kilogram (gc/kg), 4×10¹³ gc/kg and 2×10¹³ gc/kg) had elevated α-galactosidase A (α-Gal A) exercise.

Importantly, dose-dependent elevations had been noticed throughout the three dose ranges with α-Gal A exercise starting from 0.34- to 82.2-fold above imply regular degree¹ on the lowest dose, 1.6- to 312.52-fold on the mid dose, and 27.7- to 223.7-fold on the highest dose. These will increase had been sturdy for the measured time interval starting from the longest follow-up interval of greater than a 12 months in a handled affected person (high-dose cohort) to the shortest follow-up interval of 4 months in a handled affected person (mid-dose cohort).

Six of 11 dosed sufferers had been withdrawn from enzyme alternative remedy (ERT) having met a pre-specified standards together with elevated α-Gal A exercise. Steady plasma lyso-Gb3 ranges had been maintained post-dose throughout all dose cohorts, no matter ERT standing by means of the cutoff date.

“These up to date preliminary knowledge reinforce our confidence within the organic exercise of AMT-191, together with sustained and dose-dependent will increase in α-Gal A exercise throughout all dose cohorts of the handled sufferers,” acknowledged Walid Abi-Saab, M.D., chief medical officer of uniQure. “Whereas the research stays ongoing, we consider the preliminary knowledge collected are supportive of the potential for AMT-191 as a one-time administered gene remedy for folks residing with Fabry illness, and we stay up for offering extra updates on this system.”

AMT-191 continued to point out a manageable security profile. No Severe Opposed Occasions (SAEs) associated to AMT-191 have been noticed on the 4×10¹³ gc/kg and 2×10¹³ gc/kg doses. Two sufferers on the 4×10¹³ gc/kg dose skilled asymptomatic Grade 3 liver enzyme elevations.   Each had been confirmed as dose-limiting toxicity following an Unbiased Knowledge Monitoring Committee overview, and per protocol, the corporate has paused extra dosing within the mid- and high-dose cohorts pending additional analysis. Each sufferers have responded to corticosteroid remedy and stay in observe up.

No extra SAEs have been noticed on the 6×10¹³ gc/kg dose past the 5 beforehand reported in two sufferers: two SAEs unrelated to AMT-191 (stroke, diplopia), two associated SAEs (chest ache, elevated troponin), and one probably associated SAE (leptomeningeal enhancement). As beforehand reported, one affected person on the 6×10¹³ gc/kg dose skilled an asymptomatic, Grade 3 liver enzyme elevation that totally resolved with a restricted course of corticosteroid remedy.

These up to date knowledge had been offered on the twenty second Annual WORLDSymposium throughout a poster presentation (Poster Ref: LB-07) on February 3, 2026. An oral presentation will happen on Friday, February 6, 2026 within the session from 10:30-11:30 a.m. Pacific Time.  The presentation can even be accessible on uniQure’s web site on the Occasions & Shows web page after the oral presentation.

In regards to the Section I/IIa Scientific Program of AMT-191

The Section I/IIa medical trial of AMT-191 is a multi-center, open-label trial being carried out in the USA consisting of three dosing cohorts of three or extra grownup male sufferers every receiving an intravenous infusion of AMT-191. Sufferers weren’t excluded from the trial based mostly on pre-existing neutralizing anti-bodies to AAV5. Sufferers proceed to obtain their common enzyme alternative remedy till assembly withdrawal standards and can be adopted for a interval of 24 months. The trial will discover the protection, tolerability, and early indicators of efficacy by measuring the expression of lysosomal enzyme α-Gal A. Extra particulars can be found on www.clinicaltrials.gov (NCT06270316).

AMT-191 has been granted each Orphan Drug and Quick Observe designation by the U.S. Meals and Drug Administration.

About Fabry Illness

Fabry illness is an X-linked genetic lysosomal storage dysfunction brought on by a deficiency of the α-galactosidase A (α-Gal A) enzyme, resulting in poisonous accumulation of globotriaosylsphingosine (lyso-Gb3) that may injury the kidneys, coronary heart, nervous system, eyes, intestine and pores and skin. It’s estimated that sort 1 basic Fabry illness impacts a minimum of one in 40,000 males and roughly one in 20,000 females, and kind 2 Fabry illness might happen in some populations as often as 1 in 1,500 to 4,000 males. The present commonplace of look after Fabry illness is bi-weekly infusions of enzyme alternative remedy, a remedy with restricted effectiveness in lots of sufferers on account of poor cross-correction, with inefficient clearance of substrates within the goal organs, particularly the kidney and the coronary heart.

About uniQure

uniQure is delivering on the promise of gene remedy – single remedies with doubtlessly healing outcomes. The approvals of uniQure’s gene remedy for hemophilia B – an historic achievement based mostly on greater than a decade of analysis and medical growth – signify a serious milestone within the area of genomic medication and ushers in a brand new remedy method for sufferers residing with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the remedy of sufferers with Huntington’s illness, refractory temporal lobe epilepsy, ALS, Fabry illness, and different extreme ailments. www.uniQure.com

uniQure Ahead-Trying Statements

This press launch accommodates forward-looking statements. All statements aside from statements of historic reality are forward-looking statements, which are sometimes indicated by phrases similar to “anticipate,” “consider,” “might,” “set up,” “estimate,” “anticipate,” “purpose,” “intend,” “stay up for”, “might,” “plan,” “potential,” “predict,” “challenge,” “search,” “ought to,” “will,” “would” and related expressions. Ahead-looking statements are based mostly on administration’s beliefs and assumptions and on data accessible to administration solely as of the date of this press launch. Examples of those forward-looking statements embrace, however aren’t restricted to, statements relating to: the potential of AMT-191 to be a one-time administered gene remedy for folks residing with Fabry illness; and plans to supply extra AMT-191 program updates. uniQure’s precise outcomes might differ materially from these anticipated in these forward-looking statements for a lot of causes. These dangers and uncertainties embrace, with out limitation: dangers related to the medical outcomes and the event and timing of uniQure’s packages; the danger that extra affected person knowledge grow to be accessible that leads to completely different findings than that offered in preliminary or interim knowledge; uniQure’s interactions with regulatory authorities, which can have an effect on the initiation, timing and progress of medical trials and pathways and timing for regulatory approval; uniQure’s capacity to proceed to construct and preserve the corporate infrastructure and personnel wanted to attain its targets; uniQure’s effectiveness in managing present and future medical trials and regulatory processes; the continued growth and acceptance of gene therapies; uniQure’s capacity to reveal the therapeutic advantages of its gene remedy candidates in medical trials; uniQure’s capacity to acquire, preserve and defend mental property; and uniQure’s capacity to fund its operations and to boost extra capital as wanted. These dangers and uncertainties are extra totally described beneath the heading “Danger Elements” in uniQure’s periodic filings with the U.S. Securities & Change Fee (“SEC”), together with its Annual Report on Type 10-Ok filed with the SEC on February 27, 2025, its Quarterly Reviews on Type 10-Q filed with the SEC on Might 9, 2025, July 29, 2025 and November 10, 2025, and in different filings that uniQure makes with the SEC every now and then. Given these dangers, uncertainties and different components, you shouldn’t place undue reliance on these forward-looking statements, and uniQure assumes no obligation to replace these forward-looking statements, even when new data turns into accessible sooner or later.

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¹ Regular vary (1.38 – 8.66 nmol); imply regular of three.57 nmol