A Conversation with CEO Cory Nicholas on Neurona Therapeutics’ Recent RMAT Designation

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At the California Institute for Regenerative Medicine (CIRM), we enjoy highlighting achievements that come out of the research we support.

We recently sat down with Cory Nicholas, PhD, CEO and Co-Founder of Neurona Therapeutics, to discuss an important milestone for the clinical-stage company.

Neurona recently received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for NRTX-1001, a CIRM-funded neural cell therapy aimed at treating drug-resistant focal epilepsy. This RMAT designation validates the Company's preliminary clinical data and underscores the potential of NRTX-1001 to suppress seizures long-term.

In this interview, Dr. Nicholas discusses the impact of the RMAT designation, the potential of NRTX-1001, and what it could mean for patients and the future of regenerative medicine.

Some responses have been edited for clarity and brevity.

Q: Can you describe the research you and your team are doing at Neurona for neural cell therapies and why you're so passionate about it?

Our work at Neurona Therapeutics focuses on the development of allogeneic, or off-the-shelf, regenerative cell therapies, meaning therapies that are manufactured from a single cell source for all patients eligible to receive them. Neurona's therapies are designed to be administered once as a single-dose treatment to specifically address neurological disorders. Epilepsy is our lead indication, and we're committed to creating safe and effective treatments for patients with drug-resistant forms of this serious disorder.

My passion for the neural cell therapy field began a long time ago, influenced by personal experiences with family members suffering from various diseases, including stroke. My journey studying stem cell and developmental biology eventually led me to focus on brain development and repair, where I saw tremendous potential for impact in neurology.

I became fascinated with the idea of using stem cell technologies to improve health and began exploring ways to translate that to the clinic. Throughout my career, I've had the privilege of working with terrific mentors and like-minded colleagues who shared a similar vision. With this foundation, and the passionate team of innovators we've assembled that has been instrumental to Neurona's success, we've been able to push the boundaries of what's possible.

Q: Can you please tell us about Neurona's NRTX-1001 cell therapy? Can you explain how the cell therapy works to a lay audience?

NRTX-1001 is a cell therapy that involves the administration of inhibitory neurons into the nervous system, aimed at restoring balance within the brain for individuals with drug-resistant focal epilepsy.

Drug-resistant epilepsy is characterized by uncontrolled seizures caused by a hyperactive imbalance in the brain's circuitry. The core idea behind our therapy is to supply the missing inhibitory elements to rebalance these circuits.

By administering these inhibitory neurons into targeted parts of the brain, we aim to provide the necessary "brake" that is missing in patients with epilepsy. Once these neurons are in place, they are designed to effectively rebalance the brain's circuits and stop the seizures.

This approach not only addresses the symptoms but targets the underlying cause of the seizures, offering hope for a long-term solution for those with this form of epilepsy.

Q: Why is the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for Neurona's NRTX-1001 cell therapy significant for patients with epilepsy?

The RMAT designation for our NRTX-1001 therapy is a pivotal moment and recognition of Neurona's progress toward the potential treatment of people living with uncontrolled seizures. Preliminary data from the first patients treated in the ongoing open-label Phase 1/2 clinical trial are showing encouraging signs that NRTX-1001 is well-tolerated and has the potential to significantly reduce seizures. The RMAT designation now enables additional dialogue with the FDA to explore potential ways to accelerate clinical development, which is encouraging for individuals and families impacted by this disorder.

There is a common misconception that epilepsy is just a nuisance. This is categorically false. Epilepsy is a serious, and unfortunately all too common, neurological disorder that can significantly impair one's quality of life and can also be fatal. Over 3 million Americans have epilepsy, and, despite over 30 anti-seizure medications on the market, 56 percent of adults living with epilepsy continue to have uncontrolled seizures.

Seizures, whether frequent or rare, can compromise one's independence, prevent people from driving, working, and supporting dependents, and even cause dangerous situations like car accidents, falls, and sudden unexpected death. In addition, drug-resistant seizures and their medications can cause neurocognitive impairment (memory loss), mood disorders (depression, anxiety), sleep disruption, drowsiness, social isolation, and psychosis.

Q: How does NRTX-1001 differ from the current standard of treatments?

Current standard treatments for the drug-resistant epilepsy population are not very effective and/or can have severe side effects. Some patients may undergo lobectomy surgery to remove the epileptic region of the brain, which carries enormous risks and potential for permanent loss of brain function, including neuropsychological deficits.

We believe cell therapy offers an alternative solution. Our approach aims to regenerate these crucial inhibitory neurons affected in epilepsy, restoring balance to the brain's circuits, without the significant adverse effects often caused by the removal or destruction of brain tissue, and possibly even restoring lost function. In sum, NRTX-1001 has the potential to address a key unmet need for a safer and more effective alternative.

This is just the first step, but it holds great promise for filling a critical gap in treatment options for people and families around the world struggling with drug-resistant seizures.

Q: What are some milestones Neurona is hoping to achieve next for NRTX-1001?

We are conducting clinical trials to assess the safety, efficacy, and durability of NRTX-1001. Ten patients with epilepsy on one side of the brain, called unilateral temporal lobe epilepsy, have been treated so far, and we are now enrolling an expansion cohort of six more patients.

We are also beginning to look at additional focal epilepsy populations that could possibly benefit from NRTX-1001 and have recently begun a second clinical trial in ten patients with drug-resistant epilepsy on both sides of the brain, called bilateral temporal lobe epilepsy. The bilateral study is just starting, and from there, we plan to expand to focal epilepsy disorders in other regions of the brain.

Initially, our efforts are focused on epilepsy. However, we are performing preclinical research to evaluate the utility of NRTX-1001 and other neural cell types for disorders with similar hyperactive neural circuit pathology. This includes subsets of Alzheimer's disease, chronic neuropathic pain, and Parkinson's disease, where we see the potential for our inhibitory cell therapy approach to similarly quiet, rebalance, and repair hyperactive neural circuits.

We are excited about the possibilities and are committed to advancing our research for the prospective benefit of patients living with these challenging neurological conditions.

Q: How has CIRM funding supported the advancement of Neurona's NRTX-1001 cell therapy?

CIRM's support has been vital in addressing epilepsy and critical to the advancement of NRTX-1001, truly supporting us from bench to bedside. This support dates back nearly 20 years, starting with our initial grants at UCSF and continuing with subsequent grants to the Company after Neurona's launch in 2015. In the beginning, human pluripotent stem cells were a new frontier, and CIRM's backing was instrumental in accelerating our research.

We would not be where we are today without CIRM's support. Neurona and our regenerative cell therapy approach remain true to CIRM's original intent, involving many years of hard work and dedication. We are very grateful to CIRM and their team for their unwavering support.

Learn more about Neurona Therapeutics at their website www.neuronatherapeutics.com.

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