Neurona Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for NRTX-1001 in Focal Epilepsy
June 18 2024 - 4:05PM
Neurona Therapeutics, a clinical-stage biotherapeutics company
advancing regenerative cell therapies for disorders of the nervous
system, today announced that the U.S Food and Drug Administration
(FDA) has granted the company’s lead product candidate, NRTX-1001,
the RMAT expedited program designation for drug-resistant mesial
temporal lobe epilepsy (MTLE).
“We’re pleased the FDA has recognized the
potential of NRTX-1001, and we see the RMAT designation as
validating both our positive initial data as well as our novel
regenerative approach to treating focal epilepsy,” said Cory R.
Nicholas, Ph.D., Neurona’s Co-Founder and Chief Executive Officer.
“In the near-term, we are focused on completing the ongoing Phase
1/2 study and engaging with the FDA to discuss our product
development plan. The Neurona team is committed to bringing our
cell therapy to people living with drug-resistant focal epilepsy as
safely and expeditiously as possible.”
“NRTX-1001 represents a potential paradigm shift
in the treatment of drug-refractory epilepsy where the current
standard of care involves surgical removal or ablation of the
affected brain region,” said Kim J. Burchiel, M.D., Head of
Functional Neurosurgery at Oregon Health Sciences University, who
treated the second subject in the Phase 1/2 trial. “Unlike current
surgical options, which are destructive to brain tissue and can
result in serious adverse effects including neurocognitive
impairment, NRTX-1001 cell therapy has shown the potential to
control seizures without such adverse effects. If these findings
are confirmed, NRTX-1001 would satisfy a major unmet medical need
for people with drug-resistant epilepsy.”
NRTX-1001 is a regenerative cell therapy
candidate derived from human pluripotent stem cells. The fully
differentiated neural cells, called interneurons, secrete the
inhibitory neurotransmitter gamma-aminobutyric acid (GABA).
Delivered as a one-time administration, the human interneurons are
intended to integrate, persist, and provide long-term GABAergic
inhibition to repair hyper-excitable neural networks. NRTX-1001 is
currently being studied for safety and efficacy in two ongoing open
label multicenter Phase 1/2 trials for drug-resistant unilateral
mesial temporal lobe epilepsy (unilateral MTLE) and for
drug-resistant bilateral mesial temporal lobe epilepsy (bilateral
MTLE), with neocortical focal epilepsy and other indications
planned in the future.
Managed by the FDA’s Center for Biologics,
Evaluation and Research (CBER), the RMAT program is dedicated to
advancing investigational regenerative medicines, including cell
therapies, that are intended to treat, modify, reverse, or cure
serious diseases where preliminary clinical evidence indicates that
the regenerative medicine therapy has the potential to address
unmet medical needs. Investigational therapies that receive RMAT
designation are eligible to receive expedited development review
and planning guidance from senior CBER managers. RMAT also creates
a pathway for early discussions about potential surrogate endpoints
and ways to support accelerated approval.
About Neurona’s Clinical Trial of
NRTX-1001 for Unilateral Mesial Temporal Lobe Epilepsy
The ongoing multicenter Phase 1/2 clinical trial
is designed to evaluate the safety and efficacy of a single
administration of NRTX-1001 for drug-resistant unilateral MTLE. The
first stage of the trial is an open-label dose-escalation study in
10 subjects, recently expanded to up to 16 subjects, with 8
patients to be treated at a starting dose and 8 at a higher dose.
Patients receive a single administration of NRTX-1001 and are
monitored for safety and effects on their epilepsy disease
symptoms. Patient recruitment is underway at epilepsy centers
across the United States. For more information, please visit
www.clinicaltrials.gov (NCT05135091). The first stage of the
clinical trial is supported by the California Institute for
Regenerative Medicine (CIRM; CLIN2-13355).
About Mesial Temporal Lobe
Epilepsy
An estimated three million Americans have
epilepsy, and 25 to 35 percent live with ongoing seizures, despite
treatment with approved drugs, illustrating a significant unmet
medical need in this community. MTLE is a common type of focal
epilepsy in adults and primarily affects the internal structures of
the temporal lobe, where seizures often begin in a structure called
the hippocampus. For people with MTLE and seizures that are
resistant to drugs, epilepsy surgery - where the damaged temporal
lobe is surgically removed or ablated by laser - can be an option.
However, the current surgical options are not available or
effective for all patients, are tissue-destructive, and can have
significant adverse effects.
About Neurona Therapeutics
Neurona is developing allogeneic, off-the-shelf,
regenerative neural cell therapy products with curative potential
to provide long-term repair of the nervous system after a single
administration. The company’s lead product NRTX-1001, comprising
GABAergic interneurons, is currently being studied for safety and
efficacy in two ongoing open label multicenter Phase 1/2 trials
(NCT05135091) for drug-resistant unilateral mesial temporal lobe
epilepsy and (NCT06422923) for drug-resistant bilateral mesial
temporal lobe epilepsy, with neocortical focal epilepsy and other
indications planned in the future. A positive clinical update from
the Phase 1/2 trial in unilateral TLE was presented at the American
Academy of Neurology 2024 Annual Meeting. In February 2024, Neurona
raised $120 million in a private financing co-led by Viking Global
Investors and Cormorant Asset Management. For more information
about Neurona, visit:
www.neuronatherapeutics.com.
Media Contact:Elizabeth Wolffe, Ph.D.Wheelhouse
LSAlwolffe@wheelhouselsa.com
Investor Contact:Laurence WattsNew Street
Investor Relationslaurence@newstreetir.com