Neurona Therapeutics: $120 Million Raised To Advance Pipeline Of Regenerative Cell Therapy Candidates For Chronic Neurological Disorders

By Amit Chowdhry ● Feb 9, 2024

Neurona Therapeutics (a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders) announced the successful completion of a $120 million financing co-led by Viking Global Investors and Cormorant Asset Management with participation from new and existing investors, including The Column Group, LYFE Capital, Schroders Capital, Willett Advisors, Ysios Capital Partners, Euclidean Capital, SymBiosis, Alexandria Venture Investments, Berkeley Frontier Fund, Sphera Biotech Master Fund LP, Spur Capital Partners, UCB Ventures, and UC Investments.

The proceeds from the financing will be used to advance the company’s pipeline of wholly-owned, off-the-shelf cell therapies for multiple indications, including its lead investigational candidate, NRTX-1001. NRTX-1001 is being evaluated in an ongoing open-label, single-arm Phase I/II clinical trial for the treatment of drug-resistant mesial temporal lobe epilepsy (MTLE) and has potential application in Alzheimer’s disease and other disorders of the nervous system.

The data from the first cohort of five subjects in Neurona’s ongoing clinical trial of NRTX-1001 were presented in December 2023 at the Annual Meeting of the American Epilepsy Society. And the five subjects entered the study with a history of seizure activity that was not controlled by anti-seizure medications.

These subjects have received a one-time administration of NRTX-1001 as well as temporary immunosuppression to promote the long-term persistence of the cell therapy. And the first two subjects, who had 32 and 14 seizures per month during the six-month baseline, respectively, continued to report a >95% reduction from baseline in overall seizure counts more than one year after NRTX-1001 administration, with elimination of their more severe focal impaired-awareness seizures.

The first subject in the clinical trial was discontinued the immunosuppression regimen, as planned, and has reported durable seizure reduction with no rebound in seizure frequency to date. And the second subject in the clinical trial has been completely seizure-free for the past six months and recently discontinued immunosuppression, as planned. Per protocol, the modality-specific cognitive tests administered at six months post-treatment, and subsequently at three-month intervals thereafter, revealed improvements on some measures, and no deterioration in performance, by the first and second subjects.

The remaining three subjects were treated with NRTX-1001 more recently and was followed for three months post-administration. And two of the three subjects demonstrated reduced monthly seizure frequencies of 76% and 87% from baseline levels (26 and 30 seizures per month, respectively) since the first month, with elimination of their more severe focal impaired-awareness seizures. One subject – who entered the trial with baseline seizure activity of two seizures per month – had not yet responded and experienced variability in seizure count that was consistent with their disease history. Of note, NRTX-1001 required up to 5-7 months from administration for maximum efficacy to become apparent in preclinical studies, reflecting the time required for the transplanted cells to functionally integrate and mature.

NRTX-1001 was well-tolerated in all subjects to date. And adverse events have primarily been mild to moderate and typical of those associated with the temporary immunosuppression regimen, which resolved in the first subject upon discontinuation of the immunosuppressant drugs. No severe adverse events from the cell therapy, delivery procedure, or immunosuppression regimen were reported thus far in the ongoing clinical trial. And one serious adverse event, a status epilepticus seizure cluster, was reported but was consistent with the patient’s medical history before treatment.

In November 2023, the Data Safety Monitoring Board overseeing the Phase I/II trial cleared continuing enrollment for a second cohort of five patients who are expected to receive a one-time administration of the higher dose level of NRTX-1001. And the first patient in this open-label cohort has since received the higher dose of cells on-target and has not reported serious adverse events to date.

KEY QUOTES:

“We are excited to co-lead this financing with Viking and other committed investors to help advance Neurona’s cell therapies to address unmet needs in chronic neurological disorders. Neurona has pioneered development of a fully-differentiated cell therapy for drug-resistant focal epilepsy that is designed to be disease-modifying, repairing the affected neural network, and is yielding very promising initial clinical data. As a neurologist, I am particularly encouraged by the data generated thus far, suggesting that NRTX-1001 has the potential to provide seizure control and preserve neurocognitive function, which would be a game-changer for the field.”

– Raymond Kelleher, M.D., Ph.D., managing director of Cormorant and a neurologist at Massachusetts General Hospital, Harvard Medical School, who will be joining the Neurona Board of Directors

“This financing is a testament to the hard work and dedication of the Neurona team, commitment of our collaborators, and encouraging preliminary data from the first patients in the ongoing clinical trial of NRTX-1001 cell therapy. We are grateful for the significant investment from this reputable syndicate of new and existing investors. It signifies the conviction that Neurona’s cell therapies have the potential to transform the treatment of previously refractory, devastating neurological disorders. This funding will support ongoing and planned clinical studies of NRTX-1001 for drug-resistant epilepsies and Alzheimer’s disease, as well as the advancement of our other cell therapy candidates towards the clinic for additional neurological indications.”

– Cory R. Nicholas, Ph.D., Neurona’s chief executive officer and co-founder