AIPGENE Consortium Presents 1 Year Follow-Up Clinical Data from Acute Intermittent Porphyria Phase I Clinical Trial Using AAV5-PBGD Gene Therapy Candidate

--Top-line Results Confirming Safety, Liver Transfection Presented at ESGCT Conference--

THE HAGUE, the Netherlands, Oct. 27, 2014 (GLOBE NEWSWIRE) -- The AIPGENE Consortium, a pan-European collaboration supported by the Seventh Framework Programme, and uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, today announced the top-line one year follow-up analysis of a completed Phase I dose-escalation clinical trial testing an AAV5-PBGD gene therapy candidate (AMT-021) in Acute Intermittent Porphyria (AIP) patients. The data were presented on Saturday, October 25, at the European Society of Gene and Cell Therapy (ESGCT) and Netherlands Society of Gene and Cell Therapy (NVGCT) Collaborative Congress by Dr. Gloria González-Aseguinolaza, the Scientific Coordinator of the AIPGENE Consortium. The preliminary analysis of the data confirm the safety and successful transduction of patient's liver cells with the porphobilinogen deaminase gene (PBGD) using uniQure's proprietary AAV5 viral vector, as previously indicated in the interim analysis presented in May 2014.

AIP is a rare and devastating monogenic disease caused by mutations in the PBGD gene. AIP can be life threatening and the long-term effects include irreversible nerve damage, liver cancer and kidney failure. AMT-021 consists of the PBGD gene encapsulated in the AAV5 viral vector and is designed to enable transfection of the PBGD gene into patient's liver cells through a single therapeutic intervention.

"We have gathered critical clinical experience that will contribute greatly to the further understanding of this complex disease," said Dr. González-Aseguinolaza, Director of Gene Therapy and Regulated Expression Programme at the Center for Applied Medical Research (CIMA) of the University of Navarra, Spain. "I speak for all of the collaboration members in thanking the Seventh Framework Programme for supporting our study and the advancement of a gene therapy approach to help AIP patients."

"The AIPGENE collaboration has achieved its primary goal in completing a successful Phase I study, providing first and foremost the safety data required for additional clinical study," said Dr. Harald Petry, Chief Scientific Officer of uniQure. "uniQure will work with the consortium to complete the final analysis of the data for publication and to define what the next steps for the further development of AMT-021 should be."

In the multicenter, open label, single dose, dose-ranging Phase I clinical trial, a total of eight patients in four dosing cohorts received AMT-021 doses of up to 2 x 1013 gc/kg. The trial reported no safety concerns from any patient; in particular, there was no evidence of cellular immune response against the AAV5 vector or the PBGD transgene. Humoral responses against AAV5 were detected in all patients, as expected. Vector genomes were detected in patients' liver biopsies obtained one year after vector injection indicating that AMT-021 sustainably transduced human liver cells.

The Phase I trial was conducted by Digna Biotech as part of the AIPGENE Consortium. In addition to Digna, CIMA and uniQure, the consortium included the Clínica Universidad de Navarra/University of Navarra, Karolinska University Hospital, the National Center for Tumour Diseases, Heidelberg, and Servicio Madrileño de Salud, Madrid.

About AIP

Acute Intermittent Porphyria (AIP) is a rare metabolic liver disorder resulting from mutations in the porphobilinogen deaminase, or PBGD, gene, which encodes for the enzyme involved in the production of heme in the body. Insufficient activity of this protein leads to an accumulation of toxic metabolites, resulting in a wide variety of serious clinical problems, including acute, severe abdominal pain, muscular weakness and an array of neurologic manifestations, including psychiatric episodes, seizures and coma. In the majority of cases, precipitating factors, such as hormonal fluctuations, infections, drugs and dietary changes, trigger attacks. Long-term consequences may include irreversible nerve damage, liver cancer and kidney failure. Patients with AIP experience regular hospitalizations and extremely poor quality of life. Acute attacks can be life threatening. Current therapies include intravenous administration of heme and carbohydrate loading, which aim to treat the symptoms only and do not prevent attacks. In some cases, AIP patients require liver transplants.


This Collaborative Project involves seven partners from four different European countries and is related to the theme HEALTH.2010.2.4.4-1: Clinical development of substances with a clear potential as orphan drugs. The AIPGENE project is co-funded by the European Commission within the 7th Framework Programme for Research. The project is coordinated by CIMA, a European Center for Applied Medical Research, and began in 2004. The international team of experts involved in the AIPGENE consortium brings together complementary scientific disciplines to implement a new therapeutic option for AIP patients.

About uniQure

uniQure is delivering on the promise of gene therapy through single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease-modifying therapies to patients with severe disorders. We are engaged in multiple partnerships and have obtained regulatory approval of our lead product, Glybera, in the European Union for a subset of patients with LPLD.

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Source: uniQure