Investigational Alzheimer’s Therapy NNI-362 Receives FDA Approval to Enter Phase 1 Clinical Studies

The U.S. Food and Drug Administration has approved Neuronascent’s investigational new drug (IND) application for NNI-362, its therapy candidate for Alzheimer’s disease, allowing it to move ahead with a Phase 1a clinical trial, the company announced.

The first-in-human trial will evaluate the safety of NNI-362 in healthy volunteers, ages 50 to 72 years. The study, to be conducted at a single clinical site, will also assess how the therapeutic agent is absorbed, distributed, broken down, and eliminated by the body, or its pharmacokinetics.

Researchers will administer single, multiple, and increasing doses of NNI-362 in approximately 56 healthy older participants.

“Given the many recent disappointments in Alzheimer’s trials targeting amyloid, it is essential to pursue innovative treatment targets and strategies, especially if such a treatment is a non-invasive therapy that could safely produce new neurons in aged Alzheimer’s patients,” R. Scott Turner, MD, PhD, clinical monitor of the trial and the director of the Memory Disorders Program at Georgetown University, said in a press release.

NNI-362 is an orally available therapeutic candidate that is being developed to selectively stimulate neuronal progenitor cells to transform into fully functioning neurons through a process called neurogenesis.

In old age, neurogenesis slows down, contributing to the cognitive decline that commonly affects most elderly people. In a chronic neurodegenerative condition such as Alzheimer’s disease, this process occurs much rapidly, resulting in brain cell death and progressive loss of memory, thinking, and behavior changes.

Early interventions developed for Alzheimer’s disease only focused on protecting the existing brain cells in the patients, but these treatments are insufficient in halting the progression of this chronic condition, according to the release.

Neuronascent’s neuron regeneration approach is aimed at not only replacing the lost neurons but also supporting their survival, expanding the treatment options available to reverse Alzheimer’s disease progression and improve cognitive function.

“The brain of an Alzheimer’s disease patient appears incapable of replacing those lost neurons without therapeutic intervention,” said Judith Kelleher-Andersson, PhD, the founder and CEO of Neuronascent.

Preclinical studies with animal models of Alzheimer’s disease have shown that treatment with NNI-362 could markedly increase the number of neurons in regions of the brain responsible for memory in elderly mice. They also provided supporting information that proved that the development of new neurons and level of cognitive function in aged mice after oral treatment with NNI-362 was very similar to that in young animals, further supporting NNI-362’s ability to reverse Alzheimer’s progression.

“This ability to reverse age and disease-related cognitive and new neuron deficits following oral NNI-362 administration is indeed what we expect to see in Alzheimer’s patients, who already have cognitive loss due to neuron degeneration and the ‘co-morbidity of age,’” Kelleher-Andersson said.

Neuronascent recently received a $2.25 million grant from the National Institute on Aging at the National Institutes of Health to support the Phase 1a trial of NNI-362.