$1.8 Million in New Funding Supports Clinical Stage Treatments

The Alzheimer’s Drug Discovery Foundation (ADDF) announces nearly $1.8 million in new funding, which reflects our commitment to advancing drugs in or near human clinical trials. All five funded programs are potential treatments for Alzheimer’s disease and other forms of dementia.

Dr. Howard Fillit, Founding Executive Director and Chief Science Officer of the ADDF, noted: “We have been steadily increasing our funding for clinical stage treatments. Many promising programs are now reaching clinical trials, and the ADDF is doing all we can to help them succeed.” 

TREATMENTS

CLINICAL

Barbara Borroni, MD, University of Brescia
Non-invasive Brain Stimulation to Restore Cortical Plasticity and Connectivity in Genetic and Sporadic FTD
$90,000
Dr. Borroni and her team discovered that people with a disease-causing mutation for frontotemporal degeneration (FTD) have impairment in specific brain circuits nearly two decades before the onset of symptoms. Non-invasive techniques such as transcranial direct current stimulation (tDCS) have been shown to modulate brain circuits in healthy subjects and improve cognitive function in patients with neurodegenerative disorders. With this award, Dr. Borroni will conduct a clinical trial to explore the effects of repeated sessions of tDCS in patients with FTD and in presymptomatic carriers with a genetic mutation for FTD, who already show impairment in brain circuit functioning. This is the first ever trial to test a non-pharmacological approach to FTD. This award was made through The Fund to Treat FTD, in collaboration with the Association for Frontotemporal Degeneration.

Roberta Diaz Brinton, PhD, University of Arizona
Allopregnanolone Novel Patentable Formulations to Advance Commercialization
$150,000
Dr. Brinton is currently preparing for a phase 2 clinical trial of allopregnanolone, which is on track to become the first neuroregenerative therapy for Alzheimer’s disease. Allopregnanolone is a naturally occurring neuro-steroid that spurs a process called neurogenesis, which involves regenerating lost neurons to restore cognitive function. The ADDF has supported Dr. Brinton’s development of this therapy for over a decade. This grant supports the creation of a patentable formulation of allopregnanolone. This will allow Dr. Brinton to commercialize her therapy if future clinical trials are successful. The funding was awarded as part of Dr. Brinton’s receipt of the 2017 Goodes Prize for Excellence in Alzheimer’s Drug Discovery.

John Olichney, MD, University of California Davis School of Medicine
Manufacture and Testing of GMP Grade Senicapoc for a Phase 2 Clinical Trial Repurposing for Prodromal & Mild Alzheimer’s Disease
$434,991
Microglia are the brain’s immune cells and, when overactive, can cause damaging inflammation. Preclinical research showed that drugs that block the calcium-activated potassium channel KCa3.1 reduce the activation of microglia and associated inflammation and may help protect the brain from Alzheimer’s-related damage.  Dr. Olichney and an interdisciplinary team of researchers at UC Davis have identified an existing KCa3.1 blocker called Senicapoc, which had excellent safety and tolerability in prior clinical trials for sickle cell anemia and asthma. Senicapoc (or other KCa3.1 channel blockers) have not yet been tested in persons with Alzheimer’s disease. With this funding, the team will manufacture clinical-grade Senicapoc and perform stability testing required for submission of an Investigational New Drug application to the FDA. If the application is approved, they plan to move quickly to begin a phase 2 clinical trial.

Sharon Rosenzweig-Lipson, PhD, AgeneBio Inc.
Accelerating Manufacture of Clinical Supplies for Initiation of Patient Enrollment in the HOPE4MCI Phase 3 Trial
$798,087
Amnestic mild cognitive impairment (aMCI), a condition in which memory is worse than expected for a person's age, is recognized as an early stage of Alzheimer's disease with a high risk of progression to dementia. Currently there is no FDA-approved treatment for aMCI. AgeneBio developed the therapy AGB101 to slow progression in mild cognitive impairment (MCI) due to Alzheimer's disease. It targets hippocampal hyperactivity, which is characteristic of the aMCI stage of the disease and predicts progression to a diagnosis of dementia. The company is now preparing for a phase 3 trial of AGB101 in 2018. The FDA has reviewed the Phase 3 clinical protocol and the manufacturing plan and supports its implementation. This award from the ADDF will support the manufacture, testing, validation, packaging, and distribution of study medication to accelerate the start of the phase 3 trial.

PRECLINICAL

Grace Stutzmann, PhD, NeuroLucent
Ryanodine Receptor Inhibitors as a Treatment for Alzheimer’s Disease
$308,985
Dr. Stutzmann is targeting early abnormalities in neurons, specifically increases in calcium levels. Increased calcium can become toxic to neurons and lead to impaired synaptic function and structure, which harms memory. Dr. Stutzmann found that increases in calcium within neurons are triggered by the ryanodine receptor, and drugs designed to inhibit the receptor can correct the problem. She recently founded the biotechnology company, NeuroLucent LLC, to further develop ryanodine receptor drugs. With this funding, Dr. Stutzmann and team will design, test, and optimize a series of small molecule drug candidates for the purpose of stabilizing dysregulated calcium signaling within brain cells in patients with early Alzheimer’s or mild cognitive impairment.