BL-1020, an oral antipsychotic agent that reduces dopamine activity and enhances GABAA activity, for the treatment of schizophrenia

In schizophrenia, a psychiatric disorder that affects approximately 1% of the global population and is associated with substantial disability, a significant proportion of patients (usually estimated to be at least 30%) fail to respond to treatment, and many patients have difficulty continuing treatment because of side effects. At the time of publication, all available medications for schizophrenia targeted dopamine and other monoamine neurotransmitters. However, a substantial amount of research suggests that patients with schizophrenia have underlying deficits within the GABA neurotransmitter system. BL-1020, being developed by BioLineRx Ltd under license from Tel Aviv University Ltd and Bar-Ilan Research & Development Co Ltd, is a novel compound consisting of the well-established antipsychotic drug perphenazine and GABA. Preclinical studies of BL-1020 indicated that the compound penetrated the brain and was efficacious in rodents, with a significant reduction in side effects compared with the administration of perphenazine. BL-1020 was well tolerated in all clinical trials conducted, and clinically meaningful improvements were demonstrated in phase II trials in patients with schizophrenia. Further data from phase II and subsequent phase III trials will be required to derive conclusions for BL-1020 regarding overall efficacy.