Probiodrug AG, a biopharmaceutical company developing novel therapeutic solutions to treat Alzheimer’s disease (AD), announced that the first patient has been enrolled in the Phase IIa “SAPHIR” clinical study of its lead product PQ912 at the Alzheimer Centre, VU Medical Centre (VUmc), Amsterdam.
PQ912 is a novel pathology-related drug candidate with disease modifying potential for AD. It is a small molecule that targets glutaminyl cyclase (QC), an enzyme that catalyses the formation of pyroglutamate-Abeta (pGlu-Abeta), a highly toxic Abeta variant. This modified peptide triggers the formation of toxic soluble Abeta oligomers, which are a key culprit in the disease pathology. Probiodrug discovered QC and has developed QC-inhibition as a therapeutic concept for AD to the current stage. PQ912 is the first QC-inhibitor being tested in patients.
The Phase IIa clinical trial ‘Safety and Tolerability of PQ912 in Subjects with Early Alzheimer’s disease (SAPHIR)’ is a randomised, double-blind multicentre study which plans to enrol a total of 110 patients with early stage Alzheimer’s disease. It will be led by internationally renowned experts in AD in five European countries at 14 sites.
The primary endpoint of the trial is the safety and tolerability of PQ912 compared with placebo over a three-month treatment period. Additionally, a set of exploratory readouts comprising of cognitive tests, functional assessments by EEG and functional MRI and new molecular biomarkers in CSF will be used to evaluate the compound’s effect on the pathology of the disease. First data of the SAPHIR study are expected mid 2016.
Professor Philip Scheltens, Director of the Alzheimer Centre at VUmc and Chairman of the SAPHIR study, said: “Our team at the Alzheimer Centre is very excited and highly dedicated to carrying out this important novel trial in Alzheimer’s disease. Its design and intervention make this trial unique, and all investigators are eager to see the outcomes.”
Inge Lues, Chief Development Officer at Probiodrug, commented: “The start of the SAPHIR study is an important milestone for Probiodrug. The design of this study results from close collaboration with Professor Scheltens and his team at the Alzheimer Centre and builds on PQ912’s strong Phase I data package. We believe PQ912 offers a promising and innovative approach to treating Alzheimer’s disease and hope to show therapeutic potential for novel QC-inhibitors in this first-in-patient clinical trial.”
The SAPHIR study follows an exploratory Phase I study in which PQ912 was proven to be safe and well tolerated and showed good pharmacokinetic profile resulting in effective brain concentration and target occupancy.
