Bright Minds Biosciences Launches Phase 2 Trial of BMB-101 for Epilepsy

Bright Minds Biosciences Inc., a biotechnology company listed on NASDAQ under the ticker symbol DRUG, has announced the commencement of the BREAKTHROUGH study. This open-label Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of BMB-101, a highly selective 5-HT2C receptor agonist, in adult patients suffering from classic Absence Epilepsy and Developmental Epileptic Encephalopathy (DEE).

The BREAKTHROUGH study is structured as a basket clinical trial, focusing on individuals diagnosed with either Absence Epilepsy (including those with Eyelid Myoclonia) or DEE. The trial aims to involve 20 adult participants aged between 18 and 65 years. These epilepsy disorders are known for their refractory seizures, which often do not respond to existing treatments.

The study will begin with a 4-week baseline period where each participant’s seizure activity and EEG patterns will be recorded to establish a baseline. Following this, the Absence Epilepsy group will undergo an 8-week treatment phase, while the DEE group will have a 12-week treatment period with BMB-101. After the treatment phase, there will be a 4-week follow-up period to observe any lasting effects after stopping the drug.

The primary objectives are to assess the drug's safety, tolerability, and efficacy. The main efficacy endpoints include changes in the frequency of generalized spike-wave discharges (GSWD) on a 24-hour EEG for participants with Absence Epilepsy, and changes in seizure frequency recorded in a daily seizure diary for DEE participants, compared to the baseline period.

An open-label extension trial lasting at least another 12 months will be available for all subjects who respond positively to BMB-101, subject to their physician's approval.

Ian McDonald, Chief Executive Officer of Bright Minds Biosciences, expressed optimism about BMB-101, highlighting its unique pharmacological profile and potential to be a best-in-class 5-HT2C agonist. McDonald emphasized that the Phase 1 trial demonstrated central target engagement and provided substantial data on 5-HT2C's role in refractory epilepsies. He believes BMB-101 could significantly impact the DEE and Absence Epilepsy communities and offer a new standard of care for a broader range of epilepsy patients who experience drug resistance.

Bright Minds Biosciences is committed to developing a pipeline of novel treatments for neurological disorders with significant unmet needs. The company's financial position is expected to support the completion of the BREAKTHROUGH study and sustain operations through 2026. This financial stability will enable the company to maintain momentum in its clinical programs and explore additional indications for BMB-101 and other assets in their pipeline.

The company is also investigating the use of 5-HT2C compounds in managing eating disorders and obesity. Additionally, Bright Minds is advancing its 5-HT2A and 5-HT2A/C programs focused on neuropsychiatric disorders like major depressive disorder, treatment-resistant depression, and generalized anxiety disorder.

On September 25, 2024, Bright Minds Biosciences will host an investor call to discuss the BREAKTHROUGH study. The call will include insights from key opinion leaders in the field of epilepsy. Registration for the call can be done through the Bright Minds Biosciences website, and a replay will be available after the event.

BMB-101 is a novel 5-HT2C Gq-protein biased agonist designed for chronic treatment of neurological disorders where tolerance and drug resistance are common issues. It works exclusively via the Gq-protein signaling pathway and avoids beta-arrestin activation, minimizing the risk of receptor desensitization and tolerance development. In preclinical studies, BMB-101 has shown efficacy in animal models of Dravet Syndrome and generalized seizures.

In Phase 1 clinical studies, BMB-101 was administered to 64 healthy volunteers in Single Ascending Dose (SAD), Multiple Ascending Dose (MAD), and food-effects studies. The drug was found to be safe and well-tolerated at all doses, with no Serious Adverse Events (SAEs) reported. Adverse Events (AEs) were mild and consistent with on-target effects for serotonergic drugs. Robust central target engagement was confirmed through extensive target-engagement studies using fluid and physical biomarkers.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!