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uniQure publishes 36-week follow-up data from AMT-061 study and AMT-060 long-term follow-up data in patients with haemophilia B

FIX exercise Up to 54% regular, average 45% regular, after 36 weeks
After administration of AMT-061 in Part IIb study

– Medical benefit and tolerability of AMT-060 Preserved in all patients
] After as much as 3.5 years of follow-up

LEXINGTON, Mass and AMSTERDAM, The Netherlands, July 06, 2019 (GLOBE NEWSWIRE) – uniQure NV (NASDAQ: QURE), a leading gene remedy firm that develops transformative remedies for patients with critical medical needs, at the moment up to date medical data from three patients who have been coated by the corporate's ongoing Part IIb study AMT-061 . studied AAV5 -based gene remedy containing patented protected FIX-Padua for patients with extreme and reasonably extreme haemophilia B. In addition, the company introduced as much as three.5 years of follow-up data from 10 patients in the AMT-060 part I / In its first-generation gene remedy in the remedy of haemophilia B. These medical data have been introduced on Saturday, July 6 in oral shows at the 27th International Congress of Trombosis and Hemostasis (ISTH), which takes place in Melbourne, Australia.

AMT-061 (AAV5-Padua hFIX variant) enhanced vector for gene switch in adults with extreme or average to extreme haemophilia B: follow-up o 36 weeks

AMT-061 step IIb Research is open, single dose, single-handed, multi-centered in the US. Three patients with extreme haemophilia (endogenous factor IX (FIX) activity less than 1%) have been enrolled and acquired one intravenous infusion of 2 x 1013 vc / kg. Previous to administration of AMT-061, all three patients had low AAV5 neutralizing antibodies, however weren’t excluded from the study. Within the Part IIb study, patients have been monitored for 52 weeks to guage using FIX exercise, bleeding rates and FIX alternative remedy, and monitored for 5 years to assess the security of AMT-061.

Oral presentation in ISTH 36-week follow-up data show that each one three patients have continued to increase FIX levels after single-dose administration of AMT-061, and that two out of three patients keep FIX exercise in the traditional vary. The imply FIX activity in three patients after 36 weeks of dosing was 45% of regular when the first affected person reached 54% of FIX exercise, the opposite affected person achieved 30% FIX exercise and the third patient had FIX exercise. 51% normal. The second and third patients had previously failed and excluded the second gene therapy study as a result of totally different AAV vectors have been beforehand neutralizing antibodies. The reported FIX activity was measured using activated partial partial thromboplastin time (aPTT) carried out in a central laboratory.

None of the patients in the study experienced vital lack of FIX exercise, reported potential bleeding occasions, or needed FIX substitution remedy for bleeding. One patient underwent an present situation in the hip surgical procedure and was treated perioperatively with a short-acting agent alternative. The investigator reported this as a critical opposed occasion not related to AMT-061.

”These up to date data additional present that single-dose administration of AMT-061 is nicely tolerated and has the potential to extend FIX activity in normal haemophilia B residents,” Matt Kapusta uniQure Group managing director. “We are nonetheless very happy with these outcomes, which, based on this data report, show that FIX activity is steadily growing with out bleeding, and no FIX substitution remedy or immunosuppression is needed. We consider that AMT-061 has the potential to be the first-rate and best-ranked gene therapy for patients with haemophilia B, and we’re nonetheless targeted on our aim of collaborating in the continued HOPE-B key study by the top of this yr.

Everlasting expression of FIX and upkeep of bleeding and FIX after AMT-060 gene therapy after as much as 3.5 years of follow-up

In the ongoing I / II study of AMT-060, all 10 patients nonetheless have long-term relevant medical impact, including steady improve in FIX exercise and enchancment in disease as measured by decreased use of FIX substitution therapy and decreased bleeding frequency. After as much as three.5 years of follow-up, the AMT-060 continues to be protected and properly tolerated and no new critical negative effects have occurred and inhibitors haven’t developed because the final reported data.

All 5 patients on the second dose website 2 x 1013 gc / kg are nonetheless freed from routine FIX substitution remedy as much as 3 years after remedy. Over the past 12 months, the typical annual bleeding fee was zero.7 bleeding, which is 83% decrease than the yr earlier than. Throughout the identical interval, using FIX substitution remedy decreased by 96% compared to the pre-treatment yr. The typical annual regular state FIX activity over 3 years was 7.9%, in contrast with 7.1% in the primary yr and 8.4% in the second yr.

AMT-060 is the first-generation gene therapy of uniQue consisting of the AAV5 vector. transporting the gene cassette with the wild-type FIX gene. The AMT-060 Part I / II Experiment Details are part of the AMT-061 Advertising Authorization Assertion.

”The AMT-060 Part I / II study additional demonstrates the long-term safety and tolerability of the uniQure gene remedy vessel in haemophilia B,” stated Professor Frank WG Leebeek, MD, Ph.D. Institution of Erasmus University Medical Middle in Rotterdam, the Netherlands. “We’ve got now proven evidence of sustainable medical advantages comparable to continued FIX activity, improved phenotype of disease and a big reduction in spontaneous bleeding as much as three.5 years after administration. This info could be very encouraging for uniQure hemophilia B program.

About AMT-061

AMT-061 consists of an AAV5 viral vector having a gene cassette having a patent-protected variant of factor IX (FIX-Padua) Padua. In america and Canada, uniQure owns several patents issued masking a wide range of strategies of treating bleeding issues, together with haemophilia B, using AAV gene therapy with the FIX-Padua variant. Other patents pending in the European Union

AAV5-based gene therapies have been proven to be protected and properly tolerated in many medical trials, including 4 uniQure studies in 25 patients with haemophilia B and different indications. Not one of the patients treated in medical trials with the Company's AAV5 gene therapy have skilled any cytotoxic T cell mediated immune response to the capsid. As well as, preclinical and medical data point out that AAV5-based gene therapies may be clinically efficient in patients with pre-existing AAV5 antibodies, probably growing patient compliance as in comparison with other gene remedy product candidates.

Pivotal Part III HOPE-B Study

The central Part III HOPE-B study is a multinational, multicentre, open, single-arm study to assess the security and efficacy of AMT-061. About 55 adults with haemophilia B, categorised as critical or reasonably severe, are included in a six-month measurement period, during which they continue to make use of their current remedy requirement to determine baseline control. After a six-month transition period, patients will receive one intravenous dose of AMT-061 at a dose of two x 1013 gc / kg confirmed in the Part IIb study. Affected person Dosage for HOPE-B Core Analysis is now ongoing

The primary endpoint of the study is predicated on the extent of FIX exercise achieved after administration of AMX-061, and the secondary endpoints measure the annual use of FIX alternative remedy, annual leakage charges and security

HOPE-B Patients collaborating in a central study are examined for the presence of present neutralizing AAV5 antibodies, however are usually not excluded from the check on the idea of their titers. Previous uniQure studies recommend that AAV5 gene therapies could be viable remedies for at the least 97% of patients

About Part I / II AMT-060

Part I / II Study is Open – Multicentre Study Including 10 patients who acquired a 30-minute intravenous dose of AMT-060 with out prophylactic use of corticosteroids. The study has two dose groups, each with 5 patients, the first cohort receiving 5 × 1012 gc / kg and the second cohort 2 × 1013 gc / kg. 9 patients have been categorised as severe (<1% FIX) with haemophilia. One affected person with a low dose cohort had a average / severe (1.5% FIX activity) phenotype.

About uniQure

uniQure produces a promise of gene therapy – individual remedies with probably healing effects. We will make the most of our modular and validated know-how surroundings in advance for the development of gene therapies owned and operated by the pipeline for the remedy of haemophilia B, haemophilia A, Huntington's illness, Fabry's disease, spinocerebellar ataxia sort three and cardiovascular disease. www.uniQure.com

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This press launch accommodates forward-looking statements. All statements aside from historical details are forward-looking statements typically expressed by terms resembling "anticipate", "believe", "could", "estimate", "wait", "goal", "intend", "look forward", "can "," plan "," potential "," predict "," project "," should "," will "," would "and comparable expressions. Future statements are based mostly on administration's beliefs and assumptions and on the knowledge out there to administration only in this press launch. These forward-looking statements embrace, but usually are not limited to, Part IIb analysis, the power of AMT-061 to offer all haemophilia B customers with access to a one-off remedy that can normalize FIX activity or remove the need to substitute remedy or scale back or get rid of the danger of immune responses that require immunosuppression or might lead to loss of efficacy, the power of AMT-061 to be first-class or best-in-class gene therapy for patients with haemophilia B, the power of AMT-061 to offer sustained medical advantages akin to continuous FIX activity, improved illness phenotypes or a big discount in spontaneous bleeding, our capacity to enroll in an ongoing HOPE-B core study by the top of this yr, our means to realize a few of the deliberate near future or different milestones, the danger of ongoing or deliberate medical research and / or improvement and regression. product candidates approval. Precise outcomes might differ significantly for the explanations anticipated in these forward-looking statements for a variety of reasons, including, with out limitation, the dangers related with our companions' medical improvement actions, medical outcomes, collaborative preparations, M&A and strategic modifications, regulatory oversight, product commercialization, and mental property rights, in addition to dangers, uncertainties, and others. Elements described in the “Risk Factors” part of the UniQure Quarterly Report on Type 10-Q, filed April 29, 2019. These dangers, uncertainties, and different elements, you shouldn’t rely unnecessarily on these forward-looking statements, and usually are not obliged to replace these forward-looking statements; even if new info becomes obtainable in the longer term.

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