Innovative Phase II study design of Glutaminylcyclase Inhibitor PQ912 in early Alzheimer's disease
Poster presentation at CTAD, San Diego, 8-10 December 2016
HALLE (SAALE), Germany, 8 December 2016 - Probiodrug AG (Euronext Amsterdam: PBD), a biopharmaceutical company developing novel therapeutic solutions to treat Alzheimer's disease (AD), announced today that the study design of the ongoing Phase IIa SAPHIR trial comparing PQ912 to placebo will be presented as a poster on Thursday, December 8, 2016 at the 9 th Clinical Trials on Alzheimer's disease ( CTAD ) meeting in San Diego, USA.
The SAPHIR study is a 3 month study in treatment naïve patients with early AD. PQ912 targets the inhibition of the Glutaminylcyclase (QC) resulting in a reduction of the production of neurotoxic pyroGlu-Abeta (pGlu-Abeta) and related oligomers. PQ912 has been extensively investigated in Phase 1 Multiple ascending dose studies (MAD) showing good tolerability and a dose dependent QC-inhibition in the spinal fluid.
The SAPHIR study has been designed and is conducted in collaboration with Philip Scheltens, M.D., Ph.D., the VUmc Amsterdam (NL) and the CRO Julius Clinical ( NL).
The primary objective of the SAPHIR study is to investigate the safety of PQ912 in the target population and the secondary objective is to assess the pharmacodynamic profile. The publication at CTAD reveals that the study applies a series of methodological innovations which in this combination has not been executed before in an early AD study. Specific in and exclusion criteria based on diagnostic biomarkers of Abeta and tau were required to be met by all patients to ensure a high confidence of the diagnosis of early AD. Mini-Mental State Examination (MMSE) and Cogstate test battery assessments at baseline are monitored blindly every 30 patients to ensure consistency and reliability of ratings. A number of exploratory endpoints like EEG, fMRI and a series of CSF based biomarkers including QC-activity, pGlu-Abeta, Abeta oligomers, neurogranin as well as inflammation markers are centrally analysed. Based on an exploratory analysis of 86 randomised patients, a low standard deviation for the Neuro-psychological test battery and functional EEG at baseline has been observed.
Prof Philip Scheltens, Director of the Alzheimer Center at the VUmc in Amsterdam and Chairman of the SAPHIR study, said: "The combination of in and exclusion criteria together with the primary and innovative exploratory outcome parameter in the SAPHIR study is unique for an early AD study. We are excited to see the full results in the second quarter of 2017."
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For more information, please contact:
Probiodrug Dr Konrad Glund, CEO Email: contact@probiodrug.de
Hume Brophy Conor Griffin, Alexia Faure, Alex Protsenko Tel: +44 (0) 20 7862 6381 Email: probiodrug@humebrophy.com
The Trout Group Tricia Truehart Tel: +1 (646) 378-2953 Email: ttruehart@troutgroup.com
Notes to Editors: About Probiodrug AG Headquartered in Halle (Saale), Germany, Probiodrug AG (Euronext Amsterdam: PBD) is a biopharmaceutical company focused on the development of new therapeutic products for the treatment of Alzheimer's disease.
Founded in 1997, the company successfully developed a novel therapeutic concept for diabetes - the DP4 inhibitors - which provided the basis for a novel class of antidiabetics - the gliptins. Its core capabilities are based on its long-standing expertise in the elucidation of the structure and function of enzymes involved in the modification of proteins and peptides, which play a central role in pathological conditions.
Today Probiodrug's aim is to become a leading company in the development of Alzheimer's disease treatments and to thereby provide a better life for Alzheimer's disease patients. It has identified a new therapeutic concept linked to disease initiation and progression. The development approaches are targeting pyroglutamate-Abeta (pGlu-Abeta) as a therapeutic strategy to fight Alzheimer's disease. The Company has medical use and composition of matter patents related to the inhibition of Glutaminyl Cyclase (QC) and anti-pGlu-Abeta- specific monoclonal antibodies, providing it, in the Company's view, with a leading position in this field of research.
Probiodrug's lead product candidate, PQ912, is a highly specific and potent inhibitor of Glutaminyl Cyclase (QC), which has shown therapeutic effects in Alzheimer's animal models. PQ912 is currently in a Phase 2a study, the SAPHIR trial. In a preceding Phase 1 study with healthy young and elderly volunteers, PQ912 has shown to be safe and well tolerated and also revealed high QC-inhibition.
About Alzheimer's disease Alzheimer's disease is a neurological disorder, which is the most common form of dementia, and ultimately leads to death. Because Alzheimer's disease cannot be cured and is degenerative, the affected patients must increasingly rely on others for assistance. . Today, 47 million people live with dementia worldwide, and this number is projected to treble to more than 131 million by 2050, as populations age. Dementia also has a huge economic impact. Alzheimer's has an estimated, global societal cost of US$ 818 billion, and it will become a trillion dollar disease by 2018. (World Alzheimer Report 2016).
Forward Looking Statements Information set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgment of Probiodrug AG as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.
