Founder’s Message

Judith Kelleher-Andersson, Ph.D.
Founder, President and Chief Executive Officer (CEO)

“A therapy that is only neuroprotective will never be sufficient to halt or reverse neurodegenerative disease progression”. I came to this realization after working in the neuroscience field for over 20 years as a graduate student, as a post-graduate fellow and after working at three biotech companies, where I directed the discovery of therapeutics aimed at Alzheimer’s disease and other neurodegenerative disorders.

In chronic neurodegenerative disorders, like Alzheimer’s and Parkinson’s disease, much more is required from a therapeutic, than just reducing the loss of the remaining neurons. In founding Neuronascent, my aim was to discover novel therapies that would increase the number of new adult-born neurons in brain regions where neurons had been lost due to age-related disease. Further, a “neuron regeneration” therapy should be orally deliverable, rather than an invasive brain injection of either whole cells or of gene therapies.

Regenerative medicine is not a new field, and through cellular and tissue transplant, regeneration has been successful, one example being bone marrow transplantation for cancer patients. Yet, this regenerative success, using cells or viral vector injection has not been as successful for brain deficits. This is due in part to safety considerations, as well as the in-ability to obtain an appropriate number of functioning new neurons that integrate successfully into the brain, since the brain has a few different cell types (e.g., neurons, astrocytes and oligodendrocytes).

If a regenerative therapy is not an invasive cell injection, but instead is an oral medicine, that easily crosses the barrier between blood and brain, then the above concerns should not apply. An oral medicine can take advantage of endogenous neuronal progenitors, found in select niches of the adult/aging brain. Neuronal progenitors are naturally pushed to become new neurons, to respond to injury. We now believe, there are indeed ways to achieve a heightened response to the diseased brain’s own “compensatory response” to injury. In other words, the brain itself, even under chronic neurodegenerative conditions, has all the “fire-fighting equipment” ready to reduce/replace dying neurons with new neurons. Yet, without therapeutic help, the water is only trickling through the fire hose, even while the fire continues to rage and spread. Neuronascent’s discovery program was able to increase the rate of water flow, i.e., greater formation of new neurons, and to ensure that the water reaches the right location to douse the fire, i.e., neurons go to site of injury and survive to functionality. The discovery of this non-invasive, neuron-regenerative enhancement is what makes Neuronascent’s technology so unique.

Discovering therapies of this nature may sound unachievable, but through Neuronascent’s novel screening process, we were able to identify small molecule therapeutics that promoted significant new neurons, and additionally ensured these new neurons survived to functionality under neurodegenerative conditions. These small molecule therapeutics proved successful not only on human neuronal progenitor cells in a lab dish, but showed that same neuron regenerative capacity in animal models of chronic neurodegenerative diseases.

Today our oral therapeutic candidates are optimized for efficacy and fully patented around the world. Further, these orally available candidates have been found to be safe in all testing to date. Our lead neuron regenerative drug for Alzheimer’s and for Parkinson’s disease has now completed Phase 1a first in human trials where we are able to confirm a safety profile in healthy aged subjects as we showed in animals (National Institute of Aging 1R01 AG056561 grant awarded). This should allow for further testing in Alzheimer’s patients, with the first neuron regenerative therapy, NNI-362. With a further NIA grant award and potential investment dollars, Neuronascent aims to test NNI-362 in both mild to moderate Alzheimer’s patients and in early Parkinson’s patients, to reverse disease progression.

Neuronascent is a small corporation, that we believe now has an over-sized opportunity to improve the well-being of millions of patients already suffering from chronic neurodegenerative disorders that lack of sufficient disease-modifying therapeutic options. As we move toward this goal, we have built a strong track record of meeting critical drug-development milestones, of importance for investors and future pharmaceutical partners.

Judith Kelleher-Andersson, Ph.D.
Founder and CEO