SAN DIEGO--(BUSINESS WIRE)--NeuroGenetic Pharmaceuticals, Inc. (NGP), a privately held biopharmaceutical company focused on Alzheimer’s disease (AD) therapeutics, has been awarded an R01 grant from the National Institute of Aging (NIA) in the amount of $2.5 million for first in human clinical trials with NGP 555. NGP 555 is a proprietary “first in class” small molecule for the treatment and prevention of Alzheimer’s disease. NGP 555 is an amyloid modulator showing good brain penetration in pre-clinical models and is available in capsules for once daily dosing.
The grant funds pilot clinical trials for Alzheimer’s disease. NGP has completed the Phase 1a double blind, placebo controlled, single ascending dose clinical trial with NGP 555. Five cohorts of young, healthy subjects were dosed with 25 mg – 300 mg. All doses were safe and well-tolerated with dose-dependent plasma exposure. The 14-day Phase 1b multiple ascending dose clinical trial is expected to begin next month.
About NeuroGenetic Pharmaceuticals, Inc.
NeuroGenetic Pharmaceuticals, Inc. (NGP), a biopharmaceutical discovery and development company founded in 2009, is developing innovative drug therapies for Alzheimer’s disease. Based in San Diego, Calif., the company’s clinical trials utilize specific amyloid biomarkers and/or brain scanning as an early diagnostic and to monitor drug efficacy in clinical trials. Combining early disease identification with a treatment capable of preventing AD-related pathology, such as NGP 555, would represent an important advance in our ability to prevent AD or hinder its progression to dementia. Clearly, the earlier AD is detected and treated, the better the likelihood of a good outcome.
NGP 555, a proprietary “first in class” molecule for the treatment/prevention of Alzheimer’s disease, is a gamma-secretase modulator targeting the γ-secretase complex, a key enzyme in the amyloid pathway. The compound has excellent brain penetration and is devoid of side-effects seen with other potential amyloid therapies such as gamma-secretase inhibitors and monoclonal antibodies. Efficacy studies in animals showed beneficial and chronic effects on amyloid biomarkers, pathology and cognition while lacking the side effects of other compounds and mechanisms for preventing Alzheimer’s disease. Based on these studies, this compound is expected to prevent the formation of Aβ42 and the deposition of amyloid plaques in the human brain, thereby precluding neuronal cell death and the dementia associated with AD.
About Alzheimer’s disease
The Alzheimer’s Association (www.alz.org) describes Alzheimer’s disease as a progressive and fatal brain disease, with as many as 5.5 million Americans and up to 30 million worldwide currently living with the disease. Without effective prevention and treatment, the number of people with AD will increase significantly. The pathology burden of plaques and tangles in the brain may begin as much as 10 to 20 years before dementia can be detected, a progression from pathology to dementia currently representing a substantial unmet medical need for an aging population and a huge future burden for society cost of treatment and hospitalization. Advances in assessing genetic risk, biomarker profile in spinal fluid, and brain scanning to predict who will develop Alzheimer’s disease (AD) and when it may occur allow preventative AD-therapeutics to be tested efficiently in the clinic. This newfound ability to diagnose and monitor AD-progression makes the need for safe and effective drugs that can stop/prevent AD-plaque formation and dementia onset even more critical.