New York, NY – November 26, 2013 – CereSpir™ Incorporated, a company developing CHF 5074, a first-in-class microglial modulator, as a potential treatment for Alzheimer’s disease, announced today Richard Margolin, MD, has joined the Company as Vice President, Clinical Development. Dr. Margolin will lead CereSpir’s Phase 3 program for CHF 5074, which includes two clinical trials anticipated to begin in 2014, and will report directly to Daniel G. Chain, PhD, Chairman, President and Chief Executive Officer.
“Dr. Margolin is a recognized leader in the study of Alzheimer’s disease. His extensive career at the front line of the industry’s efforts to develop new therapeutic options for Alzheimer’s disease will be critically valuable to us,” stated Dr. Chain. “I have known Dr. Margolin for many years, and I am very pleased to be working with him as a partner while we advance CHF 5074 into the final phase of clinical development. We are designing an optimal clinical trial strategy that takes advantage of the latest insights in the field and regulatory guidelines that were established to improve the likelihood of success for companies developing drugs for Alzheimer’s disease.”
“CHF 5074 is a promising compound for the potential treatment of early stage AD, including people with Mild Cognitive Impairment (MCI) who have a widely recognized genetic risk factor, the apolipoprotein E4 gene (ApoE4),” commented Dr. Margolin. “The compound appears to act on several therapeutic targets, especially microglia, which are a key component of the brain’s innate immune system. Evidence suggests CHF 5074 may reduce amyloid deposition, inflammation and tau-associated pathology, thus reducing irreversible neurodegeneration. Notably, the Phase 2a clinical data obtained in MCI patients builds on the compound’s robust preclinical and Phase 1 data and gives me hope it may succeed as a therapeutic agent for this condition. CHF 5074’s proposed mechanism of action also provides a plausible basis by which it could delay or prevent the onset of symptomatic AD in high-risk individuals.”
Dr. Margolin joins CereSpir from Janssen Alzheimer Immunotherapy R&D, where he served as Senior Director, Biomarkers, playing a key role in executing biomarker sub-studies associated with the bapineuzumab Phase 3 program conducted collaboratively by Janssen and Pfizer. Previously, he held positions with Merck Research Laboratories and i3 Research, and for many years prior to entering the pharmaceutical industry, Dr. Margolin had leadership positions in academic medicine, first at Vanderbilt University and then at the Keck USC School of Medicine. Dr. Margolin has served as PI or co-PI for over 50 clinical trials targeting AD, MCI, and other CNS indications, and was site PI for the NIH-sponsored Alzheimer’s Disease Cooperative Study. He has authored or co-authored over 60 scientific publications. Dr. Margolin is board-certified in psychiatry. He obtained a Bachelor’s degree from Harvard University, a Medical Degree from the University of California, Irvine, and trained in psychiatry at Vanderbilt.
About CHF 5074
CHF 5074 is a small molecule with a unique microglial modulating mechanism of action that selectively reduces their pro-inflammatory activity while increasing their ability to remove neurotoxic amyloid beta (“Aβ”) aggregates in the brain by phagocytosis. Microglia are small cells that travel through the brain to remove waste products, such as amyloid aggregates that cause inflammation and irreversible damage to nerve cells. Chronic microglia dysfunction is increasingly believed to play an important role at the very beginnings of Alzheimer’s disease.
The results from Chiesi Farmaceutici SpA’s human clinical studies corroborate a large body of data from published preclinical studies. In Alzheimer’s disease transgenic mouse models, CHF 5074 was shown to reduce neuroinflammation, inhibit brain amyloid β plaque deposits, reduce tau pathology, and reverse associated memory deficits. These findings indicate CHF 5074 acts simultaneously on several important therapeutic targets, and this neuroprotective multi-target approach may translate into preventing the memory loss that is the hallmark of Alzheimer’s disease.
About Alzheimer’s disease, ApoE4, and Mild Cognitive Impairment
Alzheimer’s disease (AD) is the most prevalent neurodegenerative disease and the leading cause of dementia among the elderly. More than 5 million people in the U.S. have been diagnosed with AD, and the associated costs of care exceeds $200 billion annually. AD is expected to afflict 100 million people globally by 2050, and due to the lack of effective treatments, it represents one of the world’s greatest unmet medical needs.
Individuals who have one copy of the ApoE4 gene are three times more likely to develop AD than those who do not carry ApoE4. People who have two copies of the ApoE4 gene have approximately nine times greater risk. Carrying the ApoE4 gene is associated with a more aggressive form of AD that is believed to involve a strong inflammatory response.
Mild Cognitive Impairment (MCI) is a condition in which people experience cognitive changes that are serious enough to be noticed by themselves or others, but the changes are not severe enough to interfere sufficiently with daily life or independent function for them to be diagnosed with dementia. People with MCI who carry the ApoE4 gene have an increased risk of developing Alzheimer’s or another type of dementia.
About CereSpir Incorporated
CereSpir is dedicated to preserving the primary essence of each person, his or her memories. The Company holds the exclusive worldwide development and commercialization rights to CHF 5074, a novel, first-in-class small molecule microglial modulator, from Chiesi Farmaceutici S.p.A. Upon receiving further input from the U.S. Food and Drug Administration, CereSpir anticipates initiating a Phase 3 clinical trial to determine if CHF 5074 has the potential to become the first approved therapy for people with the ApoE4 genotype and Mild Cognitive Impairment (MCI). The Company’s ultimate goal is to obtain approval to treat patients with CHF 5074 before amyloid plaques develop as a prevention strategy for Alzheimer’s disease.