FOR IMMEDIATE RELEASE

January 09, 2017

 

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, January 09, 2017 – 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 (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 selecting an early stage patient population increase the likelihood of success for preventative amyloid-based therapies such as NGP 555.

CONTACTS:

Dr. William T. Comer, CEO, NeuroGenetic Pharmaceuticals, Inc. Phone: 858-735-5892
Email: wtcomer@neuro-genx.com

MEDIA:

Tom Gable
Nuffer, Smith, Tucker PR for NeuroGenetic Pharmaceuticals, Inc. Phone: 619-251-3881
Email: tg@nstpr.com

November 10, 2015

NeuroGenetic Pharmaceuticals Awarded $2.5 Million Grant From NIA to Conduct Clinical Trials for NGP 555 to Treat and Prevent Alzheimer’s Disease; Reports Initial Results of Phase 1a Clinical Trial

SAN DIEGO, Nov. 10, 2015 – 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.

NeuroGenetic Pharmaceuticals Receives $2.5 Million NIA Grant for Alzheimer’s Research

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.

CONTACTS:

Dr. William T. Comer, CEO, NeuroGenetic Pharmaceuticals, Inc. Phone: 858-735-5892
Email: wtcomer@neuro-genx.com

MEDIA:

Tom Gable
Nuffer, Smith, Tucker PR for NeuroGenetic Pharmaceuticals, Inc. Phone: 619-251-3881
Email: tg@nstpr.com

Paige Nordeen
Phone: 619-296-0605, Ext. 255 Email: pn@nstpr.com

September 29, 2014

FDA Approves NeuroGenetic Pharmaceuticals Application To Begin Clinical Trials for its NGP 555 Compound to Treat and Prevent Alzheimer’s Disease

SAN DIEGO, Sept. 29, 2014 – The U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application from NeuroGenetic Pharmaceuticals, Inc. (NGP) to begin clinical trials on its NGP 555 compound to treat and prevent Alzheimer’s disease (AD). The approval follows successful completion of all preclinical phases under a fast-track grant from the National Institute of Neurologic Disease and Stroke (NINDS).

Dr. William T. Comer, CEO of NGP, a privately held biopharmaceutical company focused on Alzheimer’s disease therapeutics, said NGP completed pre-clinical toxicology and safety studies of NGP 555 under Small Business Innovation Research (SBIR) grants from the NINDS, grant no. 1U44NS073133-01A1 totaling $3.4 million. The efficacy studies 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 (AD).

“NGP 555 prevented the formation of and the deposition of amyloid plaques, thereby precluding neuronal cell death and the dementia associated with AD,” said Comer.

NGP 555, a proprietary “first in class” molecule for the treatment/prevention of Alzheimer’s disease, is a gamma-secretase modulator (GSM) demonstrating good brain penetration and targeting the gamma-secretase complex, a key enzyme in the amyloid pathway. A novel solid dosage form in capsules is available for once daily oral dosing, Comer said.

Comer said NGP anticipates it will begin Phase I human clinical trials by the end of 2014.

About NeuroGenetic Pharmaceuticals, Inc.

NeuroGenetic Pharmaceuticals, Inc. (NGP), San Diego, is a biopharmaceutical discovery and development company founded in 2009 to develop innovative drug therapies for Alzheimer’s disease. NGP 555 is its first compound approved by the FDA to enter clinical trials. Website: http://www.neuro-genx.com/

About Alzheimer’s disease

Alzheimer’s disease is a progressive and fatal brain disease with 5.5 million Americans and up to 30 million worldwide currently living with the disease. The NIH reports that unless the disease can be effectively prevented, the number of people with AD will increase significantly. The FDA acknowledges that advanced stages of Alzheimer’s dementia cannot be treated effectively, and the cognitive impairment must be diagnosed early before the amyloid plaques are well developed. The disease must be prevented, and recent efforts have focused on early diagnosis of amyloid deposits and cognitive impairment so that a drug like NGP 555 can be taken early in the course of the disease and retard the course of disease.

CONTACTS

Dr. William T. Comer, CEO, NeuroGenetic Pharmaceuticals, Inc.
Phone: 858-735-5892
Email: wtcomer@neuro-genx.com

Tom Gable
Gable PR, for NeuroGenetic Pharmaceuticals, Inc.
Phone: 619-284-1714
Email: tom@gablepr.com

January 4, 2012

 

Abbott Biotech Ventures Invests in NeuroGenetic Pharmaceuticals;
Advancing Research on Therapy for Alzheimer’s Disease

SAN DIEGO — NeuroGenetic Pharmaceuticals, Inc. (NGP), a privately held biopharmaceutical company focused on Alzheimer’s disease (AD) therapeutics, has received an investment from Abbott Biotech Ventures. The amount of the investment was not disclosed. Dr. William T. Comer, president and CEO of NGP said, “The funding will expedite the development of our lead candidate, NGP 555, for the prevention of AD, including achieving our immediate goal of initiating clinical trials.”
Earlier, NGP announced that it has been awarded a Small Business Innovation Research (SBIR) fast-track grant from the National Institutes of Health (NIH) for preclinical work on NGP 555. This NIH grant has provided $3.3M from July 2011 through July 2014.

July 2011

In July 2011 NeuroGenetic Pharmaceuticals was awarded a Small Business Innovation Research Fast-track grant from the National Institute of Neurological Disorders and Stroke (NINDS) for a cooperative agreement. The grant will fund the preclinical development and Investigational New Drug application for the clinical candidate NGP 555. The first phase award is $288,000 with future awards up to $1 million per year for three years for milestone-based achievements. NGP 555 is a proprietary “first-in-class” molecule for the prevention/treatment of Alzheimer’s Disease which targets the amyloid pathway in a selective manner precluding side effects seen with other amyloid-based therapies.

Sept. 8, 2010

 

NeuroGenetic Pharmaceuticals announces studies showing its proprietary compound reduces brain plaques associated with Alzheimer’s disease

Data published in Neuron demonstrates proof of concept in long term prevention of disease pathology in a mouse model, without GI side effects

SAN DIEGO — In the Sept 9, 2010 issue of Neuron, NeuroGenetic Pharmaceuticals, Inc. (NGP) reports proof of concept studies that show its proprietary compound, NGP 555, is effective in preventing the amyloid pathology of Alzheimer’s disease (AD) in a transgenic mouse model. The study further demonstrates that following chronic treatment with the gamma secretase modulator (GSM) compound from NGP, the mice were devoid of gastrointestinal side effects, an adverse finding commonly associated with gamma secretase inhibitors (GSIs).
A major pathological hallmark of Alzheimer’s disease is an abundance of neuritic plaques in key areas of the brain involved in memory and cognition. Decades of studies have confirmed that Αβ42 forms the “seed” of these amyloid plaques, which gradually accumulate in the brain and induce neuronal cell death in the underlying brain tissue. This “toxic” molecule is generated by a stepwise process involving a pivotal enzyme, gamma secretase. Modification of gamma secretase activity to decrease production of Αβ42, thereby reducing the deposits of Αβ42 -seeded plaques, would be beneficial for the prevention of Alzheimer’s disease-related pathology.
“We are pleased to make public these data on our gamma secretase modulator, NGP 555,” said Dr. William T. Comer, President and CEO of NeuroGenetic Pharmaceuticals. “Deposition of amyloid plaques can precede dementia by many years, and the progression of plaques to dementia reflects neuronal loss which is irreversible. We believe that halting this gradual progression of AD from pathology to dementia represents a major unmet need, especially given the growth of an aging population and the enormous cost to society for care and hospitalization. Recent advances in the use of Αβ biomarkers in the cerebrospinal fluid and brain scans should permit early diagnosis of AD pathology and allow us to show that NGP 555 prevents the amyloid pathology.”
The work published in Neuron is the first to describe these mechanistically and biochemically distinct GSM compounds and how they provide a more selective mechanism than GSIs. The key advantages of these small molecules include reduction of the “toxic” form of beta amyloid (Αβ42), direct binding to components of the gamma secretase complex, and excellent brain exposure. The paper demonstrated that NeuroGenetic Pharmaceuticals’ approach of gamma secretase modulation allows for selective reduction of Αβ42 and amyloid pathology. Oral administration of NGP 555 (identified as compound 4 in Neuron) in transgenic AD mice resulted in a dose-related lowering of both plasma and brain Αβ42. Chronic daily administration for 7 months led to significant reduction in both diffuse and neuritic plaques, without the GI-related side effects found with GSI compounds, according to the paper in Neuron. The work, conducted by researchers at TorreyPines Therapeutics (TPTX) in collaboration with academic institutions, concludes that these types of GSM compounds warrant further investigation as a potentially safe and effective approach for prevention of AD.
“This study links Αβ biomarker and pathology findings with a mechanistic understanding of how our compounds selectively target a key enzyme involved in the pathology of Alzheimer’s disease,” said Maria Z. Kounnas, Ph.D., lead author on the study and vice president of Alzheimer’s Research at NGP. “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.”
For further information, email the Media Contact listed below (tom@gablepr.com) for a copy of the full paper or read after Sept. 9 in Neuron at: http://www.cell.com/neuron/current

About Alzheimer’s disease

The Alzheimer’s Association (www.alz.org) describes Alzheimer’s as a progressive and fatal brain disease, with as many as 5.3 million Americans and up to 30 million worldwide currently living with the disease. The National Institute of Health reports that unless the disease can be effectively treated or prevented, the number of people with AD will increase significantly. The number of people age 65 and older in the U.S. is expected to grow from 39 million in 2008 to 72 million in 2030, with the number of people with AD doubling every 5-year interval beyond age 65, according to the NIH. For further information:
http://www.nia.nih.gov/Alzheimers/AlzheimersInformation/GeneralInfo/

About NeuroGenetic Pharmaceuticals, Inc.

NeuroGenetic Pharmaceuticals, Inc. (NGP) is a biopharmaceutical discovery and development company founded in 2009 which is focused on developing innovative drug therapies for use in the treatment of neurodegenerative disorders such as Alzheimer’s disease. Based in San Diego, Calif., the company’s next objective is to obtain an Investigational New Drug approval for its clinical candidate, NGP 555. This compound is expected to prevent the deposition of amyloid plaques in the brain, thereby precluding neuronal cell death and the dementia associated with AD. Future clinical trials will utilize specific Αβ biomarkers and/or brain scanning as an early diagnostic and to monitor drug efficacy in clinical trials. NGP licensed the GSM intellectual property from TPTX and expanded its portfolio to include issued patents in the US, Europe, China, India, Japan, Australia, and other countries. For further information, see www.neurogeneticpharmaceuticals.com

CONTACTS

Dr. William T. Comer, CEO, NeuroGenetic Pharmaceuticals, Inc.
Phone: 858-735-5892

MEDIA CONTACT

Tom Gable
Gable PR
Phone: 877-281-5888
Email: tom@gablepr.com