uniQure, a company that researches and develops human gene based therapies, has initiated its phase I clinical trial in acute intermittent porphyria (AIP).

The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the E.C.’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). uniQure was granted orphan drug designation for the treatment of AIP in 2009 from the EMA.

"The start of the AIP phase I study marks the first of four programs that will enter clinical trials over the next 12 months," said Jorn 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."

The phase I will enroll eight patients with severe AIP at two centers: the Clinical University of Navarra in Pamplona, Spain, and the 12 de Octubre University Hospital in Madrid, Spain. 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.

uniQure expects interim results of the phase I in Q3 2013.