NeuroGenetic Pharmaceuticals Completes Phase 1 Clinical Trials for NGP 555 to Treat and Prevent Alzheimer’s Disease; Shown as Safe and Well-Tolerated in Healthy Volunteers

SAN DIEGO--()--NeuroGenetic Pharmaceuticals, Inc. (NGP), a privately held biopharmaceutical company focused on Alzheimer’s disease (AD) therapeutics, has completed Phase 1 clinical trials for safety, pharmacokinetics, and biomarker changes in amyloid peptides in the cerebrospinal fluid. This first in human clinical trials with NGP 555 was funded by an R01 grant from the National Institute of Aging (NIA). NGP 555 is a proprietary “first in class” small molecule for the treatment and prevention of Alzheimer’s disease. NGP 555 acts by shifting the production of Abeta42 (a disease-causing form of amyloid) to shorter non-toxic forms of amyloid so is called a Gamma-secretase modulator (GSM). NGP 555 is taken orally as a capsule once daily.

In general, NGP 555 was safe and well-tolerated in the Phase 1 study with dose-dependent plasma exposure. NGP completed Phase 1a studies in a single ascending dose clinical trial with NGP 555 in young, healthy subjects dosed at 25 mg – 300 mg. The 14-day Phase 1b multiple ascending dose clinical trial was a randomized, placebo-controlled, double-blind study in healthy volunteers (40-65 years old) dosed at 100 – 400 mg. Analysis of Abetaallofoms in cerebrospinal fluid was accomplished using Mesoscale ELISA technology in a subset of subjects.

NGP 555 is believed to act by decreasing the levels of the “plaque-forming amyloid”, Abeta42, while increasing shorter, non-toxic forms such as Abeta37. Proof of target engagement was established in this small study with subjects showing a 51% favorable change in Abeta37/Abeta42 ratios at Day 14 compared to baseline pre-drug levels for 400 mg (2 subjects) and 36% for 200 mg (4 subjects) versus 2% for placebo (1 subject). In addition, a pharmacokinetic relationship was established with CSF biomarker changes.

NeuroGenetic published pre-clinical findings on NGP 555 in the Alzheimer’s & Dementia Journal: Translation Research and Clinical Intervention (online version published Oct 14, 2016).

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 and cognitive decline, such as NGP 555, would represent an important advance in our ability to prevent AD and hinder its progression to dementia. 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.

About Alzheimer’s disease

The Alzheimer’s Association ( 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 selecting an early stage patient population increase the likelihood of success for preventative amyloid-based therapies such as NGP 555.


NeuroGenetic Pharmaceuticals, Inc.
Dr. William T. Comer, CEO
Nuffer, Smith, Tucker PR for NeuroGenetic Pharmaceuticals, Inc.
Tom Gable

Release Summary

NeuroGenetic Pharmaceuticals has completed Phase 1 clinical trials for safety, pharmacokinetics, and biomarker changes in amyloid peptides in the cerebrospinal fluid.

NeuroGenetic Pharmaceuticals, Inc.