CB03 / Zhimeng Biopharma - LARVOL DELTA

An Open-label Study to Evaluate Safety, Tolerability, and Efficacy of CB03-154 in Subjects Diagnosed With Epilepsy (clinicaltrials.gov) - P2 | N=144 | Recruiting | Sponsor: Shanghai Zhimeng Biopharma, Inc.

New P2 trial • CNS Disorders • Epilepsy

Zhimeng Biopharma's investigational drug CB03-154 for amyotrophic lateral sclerosis has achieved the first patient first dose milestone in the phase 2/3 pivotal clinical trial (PRNewswire) - "This phase 2/3 clinical trial is a multicenter, randomized, double-blind, placebo-controlled study with open-label extension....This study will enroll approximately 240 patients diagnosed with ALS in about 15 centers in China."

Trial status • Amyotrophic Lateral Sclerosis

Zhimeng Biopharma's Investigational Drug Approved to Initiate Phase 2 Epilepsy Clinical Trial in China (PRNewswire-Asia) - "The upcoming trial is a randomized, double-blind, placebo-controlled, multinational, multicenter study designed to evaluate the clinical efficacy, safety, and tolerability of CB03-154 as an adjunctive therapy in adult patients (18-70 years old) with focal epilepsy."

New P2 trial • Epilepsy

A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov) - P2/3 | N=240 | Not yet recruiting | Sponsor: Shanghai Zhimeng Biopharma, Inc.

New P2/3 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Plasma NfL

Preclinical Overview of CB03 (KCNQ2/3 Channel Opener) for Epilepsy (AES 2024) - "The well-known first-generation KCNQ (or Kv7) opener, Retigabine/Ezogabine (RTG/EZG), was approved by the FDA in 2011 as an anticonvulsant used as adjunctive therapy for partial epilepsies. Preclinical data showed that CB03 was highly selective for KCNQ2/3, inhibited neuronal firing, and was effective in preventing seizures in epileptic animals. CB03 is a potential drug for treating epilepsy."

Preclinical • CNS Disorders • Epilepsy • Mental Retardation • Psychiatry • CACNA1B • CAV2 • CAV3 • KCNQ1OT1 • NAV1

Quantitative Analysis of Continuous EEG in a Phase 1 Study of a Novel Potassium Channel Opener (CB03-154) to Assess the Safety and Tolerability (AES 2024) - "There is no evidence from the qEEG analysis that the study drug (CB03-154) has any adverse brain effects on brain network activity, either when administered acutely or chronically over a two-week period, at the doses tested. Results suggest that CB03-154 should have minimal CNS side-effects at these doses. The lack of frequency spectrum power band effects does not imply a lack of anti-seizure effects at the doses tested, as the changes seen with the older anti-seizure medications relate to their adverse CNS effects not their anti-seizure actions."

Clinical • P1 data • CNS Disorders • Epilepsy

Preclinical Overview of CB03-154 (KCNQ2/3 Channel Opener) for Amyotrophic Lateral Sclerosis (ALS) (ANA 2024) - "KCNQ channel activators act as a positive allosteric modulator of KCNQ potassium (K+) channels leading to an inhibition of high-frequency action potential firing at the initial axon segment. Preclinical efficacy data suggested that CB03-154 is a promising new investigational drug for ALS and warrant further clinical studies in ALS patients."

Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Mental Retardation • Psychiatry

A Study to Evaluate the Safety, Tolerability, and Efficacy of CB03-154 in Adult Patients With Focal Epilepsy (clinicaltrials.gov) - P2 | N=180 | Not yet recruiting | Sponsor: Shanghai Zhimeng Biopharma, Inc.

New P2 trial • CNS Disorders • Epilepsy

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CB03-154 in Healthy Participants in Australia (clinicaltrials.gov) - P1 | N=56 | Completed | Sponsor: Shanghai Zhimeng Biopharma, Inc. | Not yet recruiting ➔ Completed

Trial completion

Zhimeng Biopharma CB03 Received Orphan Drug Designation from the US FDA - A Potassium Ion Channel Opener for Amyotrophic Lateral Sclerosis (PRNewswire) - "Shanghai Zhimeng Biopharma, Inc...announced that one of its innovative small-molecule KCNQ2/3 selective opener compounds (CB03), developed for the treatment of amyotrophic lateral sclerosis (ALS), received orphan drug designation (ODD) from US FDA....A phase 1 study for the evaluation of the safety, tolerability, and pharmacokinetics of CB03 in healthy subjects is currently ongoing in Australia and US. The phase 1 study is expected to complete the dosing phase (both single dose and multiple dose) before the end of 2023."

Orphan drug • Trial status • Amyotrophic Lateral Sclerosis

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CB03-154 in Healthy Participants in Australia (clinicaltrials.gov) - P1 | N=62 | Not yet recruiting | Sponsor: Shanghai Zhimeng Biopharma, Inc.

New P1 trial

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CB03-154 in Healthy Participants (clinicaltrials.gov) - P1 | N=70 | Recruiting | Sponsor: Shanghai Zhimeng Biopharma, Inc.

New P1 trial

Zhimeng Biopharma Announces Dosing of First Subject of Its Novel Antiepileptic Drug Candidate CB03 in First-in-Human Phase I Clinical Trial (PRNewswire) - "Shanghai Zhimeng Biopharma, Inc...announced dosing of the first participant in the US Phase I study in healthy subjects of its innovative small-molecule KCNQ2/3 selective opener (CB03), developed for the treatment of refractory epilepsy."

Trial status • CNS Disorders • Epilepsy