Study to Evaluate Safety and Efficacy of Drug Seeking to Treat Those with Mild Cognitive Impairment or Mild Dementia
HALLE (SAALE), Germany and San Diego, CA – USA, 20 March 2019 – Probiodrug AG, a clinical stage biopharmaceutical company developing novel therapeutic solutions to treat Alzheimer’s disease (AD) (Euronext Amsterdam: PBD) and the Alzheimer’s Disease Cooperative Study (ADCS), announced today that the National Institutes of Health (NIH) is funding in part a US Phase 2b core program to evaluate the efficacy and safety of Probiodrug’s PQ912 in patients with mild cognitive impairment (MCI) or mild dementia due to AD with an NIH Research Project (R01) grant expected to total 15 million USD over four years.
The grant was awarded by the National Institute on Aging, part of the NIH, for the project A Seamless Phase 2A-B Randomized Double-Blind Placebo-Controlled Trial to Evaluate the Efficacy and Safety of PQ912 in Patients with Early Alzheimer’s Disease. The funding is under award number R01AG061146 to the University of California, San Diego.
Probiodrug is the sponsor of the U.S. study led by study principal investigator Howard Feldman, MD, Director of the ADCS, a national consortium of clinical sites, based at the University of California, San Diego.
“We are very grateful to the NIH for supporting this research which will advance a novel experimental therapeutic approach for those suffering from mild cognitive impairment and mild dementia due to early Alzheimer’s,” said Howard Feldman, MD, Professor of Neurosciences and Director of the ADCS, based at the University of California, San Diego. “This new medication PQ 912 will allow us to target several disease mechanisms, including the buildup of a particularly toxic form of amyloid beta peptides as well as the modulation of the neuroinflammatory process that occurs in the disease. We look forward to being able to measure the sufficiency of dosing, against the disease targets, an opportunity we often do not achieve in clinical trials and which will help definitively address whether this approach works.”
The study is designed as a randomized, double-blind, placebo-controlled trial evaluating 460 study participants from centers in the U.S. and Canada. Patients will be treated 1:1 with either PQ912 – a specific small molecule inhibitor of Glutaminyl-Cyclase – or with a placebo, for a duration of 18 months.
“We strongly believe in our first-in-class approach for early Alzheimer’s disease intervention and hope that the funding awarded by the NIH in PQ912 will motivate other investors to support us in this important research,” said Dr. Ulrich Dauer, CEO of Probiodrug. “The support of the NIH will provide important data for our therapeutic concept.”
The NIH grant will partially fund the U.S. study and the remaining budget will be covered by Probiodrug.
* The content of this press release is solely the responsibility of the author and does not necessarily represent the official views of the National Institutes of Health.
For more information, please contact:
Dr. Ulrich Dauer, CEO
The Alzheimer’s Disease Cooperative Study
Howard Feldman, MD
MC Services AG
Anne Hennecke, Susanne Kutter
Tel: +49 (0) 211 529 252 27
Notes to Editors:
About Probiodrug AG
Headquartered in Halle (Saale), Germany, Probiodrug AG (Euronext Amsterdam: PBD) is a clinical stage biopharmaceutical company focused on the development of new therapeutic products for the treatment of Alzheimer’s disease (AD). Probiodrug has identified a new therapeutic concept linked to disease initiation and progression. The development approaches are targeting a key neuro-/synaptotoxic component of the pathology, pyroglutamate-Abeta (pGlu-Abeta) as a therapeutic strategy. Its lead product, PQ912, has successfully completed a Phase 2a (SAPHIR) study. The company’s pipeline also includes PBD-C06, an anti-pGlu-Abeta-specific monoclonal antibody, in preclinical development. Probiodrug has medical use and composition of matter patents related to the inhibition of QC and anti-pGlu-Abeta-specific monoclonal antibodies, and has, in the Company’s view, a leading position in this field of research.
PQ912, is a first in class, highly specific and potent inhibitor of Glutaminyl Cyclase (QC), the enzyme catalyzing the formation of synaptotoxic pGlu-Abeta. PQ912 has shown therapeutic effects in AD animal models. A Phase-1 study in healthy young and elderly volunteers revealed a dose dependent exposure and showed good safety and tolerability up to the highest dose resulting in >90% target occupancy in the spinal fluid. In June 2017, Probiodrug announced top-line data of the Phase-2a SAPHIR trial of PQ912 and presented the study results at CTAD 2017. Results strongly support (a) the hypothesis of pGlu-Abeta being synaptotoxic and (b) the therapeutic concept pursued by Probiodrug. The study provides important guidance how to move forward with the development of PQ912 as a disease-modifying drug for AD. Altogether, the results make the program highly attractive for further development; the company has initiated the preparation of a Phase 2b core program.
About Alzheimer’s disease
Alzheimer’s disease is a neurological disorder, which is the most common form of dementia. Today, 50 million people live with dementia worldwide, and this number is projected to treble to more than 152 million by 2050. Dementia also has a huge economic impact. Alzheimer’s has an estimated, global societal cost of US$ 1 trillion, and it will become 2 trillion dollar disease by 2030. (World Alzheimer Report 2018).
The Alzheimer’s Disease Cooperative Study
The Alzheimer’s Disease Cooperative Study (ADCS) was founded by the late Leon Thal, MD, a world leader in Alzheimer’s research, to promote the discovery, development, and testing of new drugs for the treatment of AD. It is part of a larger AD research and treatment effort at UC San Diego, which includes the Shiley-Marcos Alzheimer’s Disease Research Center. Studies of AD and other neurodegenerative disorders at UC San Diego are part of the clinical and bench strength of the UC System.
The ADCS was formed in 1991 as a cooperative agreement between the National Institute on Aging (NIA) and the University of California, San Diego.
About NIH Research Project Grant Program (R01)
The Research Project Grant (R01) is the original and historically oldest grant mechanism used by NIH. The R01 provides support for health-related research and development based on the mission of the NIH. The grant supports a discrete, specified, circumscribed project to be performed by the named investigator(s) in an area representing the investigator’s specific interest and competencies, based on the mission of the NIH.
The NIH is comprised of Institutes and Centers that support specific areas of health-related research and almost all Institutes and Centers at the NIH fund R01 grants. Research grant applications are assigned to an Institute or Center based on receipt and referral guidelines, and many applications are assigned to multiple Institutes and Centers as interdisciplinary and multidisciplinary research is encouraged.
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.