UCB has entered into a definitive agreement to acquire Neurona Therapeutics, including lead asset NRTX-1001, adding to its epilepsy portfolio.  

Neurona Therapeutics is a clinical-stage biotherapeutics company focused on advancing regenerative cell therapies for epilepsies and other disorders of the nervous system. 

UCB will pay $650 million upfront and up to $500 million in potential future milestone payments. 

Jean-Christophe Tellier, Chief Executive Officer at UCB, said: “For more than 30 years, UCB has helped shape the modern epilepsy landscape. Bringing NRTX‑1001 into our portfolio allows us to extend that legacy into the era of regenerative medicine. We believe this therapy has the potential to provide durable targeted repair of the nervous system following a single dose and could represent a major step forward for people living with mesial temporal lobe epilepsy.” 

Regenerative pluripotent stem cell technology 

The Neurona Therapeutics platform uses regenerative pluripotent stem cell technology to deliver cells aimed at structurally and functionally restoring compromised neural circuitry.  

NRTX‑1001 is currently being investigated in Phase I/II clinical trials which are evaluating the safety, tolerability, and effects on seizure frequency in drug-resistant unilateral and bilateral mesial temporal lobe epilepsy (mTLE) with and without Mesial Temporal Sclerosis (MTS).  

Administered as a minimally invasive single dose directly into the brain, this approach introduces cells that produce the inhibitory neurotransmitter, gamma-aminobutyric acid (GABA), to repair and rebalance the overactive neural networks and potentially provide durable seizure reduction. 

Based on encouraging preliminary data, NRTX-1001 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for drug‑resistant mesial temporal lobe epilepsy (MTLE) and PRIME (Priority Medicines) designation from the EMA for adults with drug‑resistant focal epilepsy. 

Cory Nicholas, Co‑founder and CEO of Neurona Therapeutics, said: “We are grateful to UCB for their investment and support these past years, and we are delighted for the opportunity to work hand-in-hand with the broader UCB family to bring transformative cell therapies to patients around the world.”