December 11, 2012
uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient. The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the European Commission's Seventh Framework Program with the aim to develop a gene therapy for the treatment of AIP, a rare and devastating disease caused by mutations in the porphobilinogen deaminase gene (PBGD). AIP can be life-threatening and the long-term effects include irreversible nerve damage, liver cancer and kidney failure. uniQure was granted orphan drug designation for the treatment of AIP in 2009 from the European Medicines Agency.
"The start of the AIP Phase I study marks the first of four programs that will enter clinical trials over the next 12 months," says Jörn Aldag, CEO of uniQure. "After AIP we expect clinical trials to be initiated in Parkinson's disease, hemophilia B, and Sanfilippo B. After many years of building and developing our capabilities and competencies, and the approval in November of Glybera for LPLD as the first gene therapy in the Western world, we are highly motivated to expedite the clinical development of our other advanced gene therapies."
About the AIP Phase I study
The Phase I will enroll eight patients with severe AIP at two centers: the Clinical
University of Navarra
, and the 12 de Octubre University Hospital,,
. The study's primary objective is the assessment of safety and determination of the maximum tolerated dose. Secondary objectives include tolerability of treatment, pharmacokinetics, changes in the levels of surrogate markers of activity including porphobilinogen (PBG) and delta-aminolevulinic acid (ALA), and assessment of symptom control, neuro-psychological changes and quality of life. All patients will be followed for one year, and the interim results of the Phase I are expected in Q3 2013.
About acute intermittent porphyria