ADX71149 for epilepsy

ADX71149 for the treatment of epilepsy.

Our partnered drug candidate, ADX71149, is a novel orally active mGlu2 PAM for Epilepsy,  an indication with a large commercial opportunity as existing therapies frequently provide ineffective control of symptoms or have side effects that discourage adherence. Our partner, Janssen, is conducting a placebo-controlled Phase 2 proof of concept clinical trial of ADX71149 in epilepsy patients since June 2021. Cohort 1 have completed  the study. Cohort 2, testing a different dose of ADX71149 has completed recruitment and we expect to announce results of the study in Q2 2024.

Under our agreement with Janssen, they are responsible for financing the development and commercialization, if any, of ADX71149.


Epilepsy is one of the most common serious neurological disorders, affecting about 65 million people globally (Thurman et al. 2011). It affects 1% of the population by age 20 and 3% of the population by age 75 (Holmes et al. 2008). Epilepsy describes a condition in which a person has recurrent seizures due to a chronic, underlying process. Epilepsy refers to a clinical phenomenon rather than a single disease entity, since there are many forms and causes. Epilepsy is defined by any of the following conditions: (1) at least two unprovoked (or reflex) seizures occurring >24 hours apart; (2) one unprovoked (or reflex) seizure and a probability of further seizures similar to the general recurrence risk (at least 60%) after two unprovoked seizures, occurring over the next 10 years; (3) diagnosis of an epilepsy syndrome (Fisher et al. 2014). The synaptic vesicle protein 2A, or SV2A has been identified as a broad spectrum anti‑convulsant target in models of partial and generalized epilepsy, and studies in animal models and human tissue suggest that changes in the expression of SV2A are implicated in epilepsy (Mendoza‑Torreblanca et al. 2013; Kaminski et al. 2012). SV2A ligands include levetiracetam (Lynch et al. 2004), which is an anti‑epileptic drug commercialized under trademark Keppra®, is approved in Europe and the USA as a monotherapy or add‑on therapy in patients diagnosed with epilepsy.


Our partner, Janssen, has completed Phase 1 and two Phase 2a clinical trials in schizophrenia and anxious depression, respectively. Janssen has announced that ADX71149 has been extensively profiled in preclinical models of epilepsy showing both standalone activity and in combination with SV2A ligands including Keppra. Janssen has patented the combination of mGlu2 PAM with SV2A ligands for the treatment of epilepsy. The placebo-controlled Phase 2 proof of concept clinical trial of in epilepsy patients since June 2021. On November 14, 2023, we announced that the last patient has been randomized in Cohort 2 and results evaluating the efficacy, safety and tolerability of ADX71149 in combination with levetiracetam or brivaracetam from patient Cohort 1 and Cohort 2 are anticipated for the second quarter of 2024.

More information on the study can be found with identifier NCT04836559

We believe that, subject to regulatory approval, ADX71149 may provide a substantial benefit to patients.