Zai Lab Submits NDA for KarXT in Schizophrenia, J&J Reports Positive Data for Guselkumab, and BrainStorm's Debamestrocel Shows Survival Benefit in ALS

Zai Lab's KarXT Meets Primary Endpoint in Phase III Bridging Study for Schizophrenia, Will Submit for Marketing Approval

On October 29, Zai Lab announced the primary data from the Phase III multicenter clinical trial in China evaluating the safety and efficacy of KarXT (xanomeline and trospium chloride). Consistent with previous global clinical trials, this registrational bridging study met its primary endpoint, demonstrating a statistically significant 9.2-point reduction in the Positive and Negative Syndrome Scale (PANSS) total score from baseline at week 5 compared to placebo (-16.9 vs -7.7, p=0.0014).

The study also met all key secondary efficacy endpoints, showing significant improvements in the PANSS Positive Symptoms Subscale score, PANSS Negative Symptoms Subscale score, PANSS Marder Negative Symptoms Factor score, Clinical Global Impression-Severity (CGI-S) score, and the percentage of PANSS responders at Week 5 compared to placebo. The key secondary endpoints were formally tested in a pre-specified order. Zai Lab is actively advancing and plans to submit a New Drug Application for KarXT to the National Medical Products Administration in early 2025.

KarXT is an oral antipsychotic compound composed of xanomeline and trospium. Xanomeline is an agonist of the M1 and M4 subtypes of muscarinic acetylcholine receptors (M-AChR), while trospium is an antagonist of M-AChR. On September 26, the FDA approved the drug for the treatment of schizophrenia in adults.

Nearly 2 Years of Sustained Clinical Remission! Johnson & Johnson's Monoclonal Antibody Therapy Publishes Positive Data

Recently, Johnson & Johnson announced positive results from the Phase 3 GRAVITI clinical study of its interleukin-23 (IL-23) targeted antibody Tremfya (guselkumab). The study showed that, at 48 weeks, adult patients with moderate to severe active Crohn's disease (CD) who received Tremfya as induction and maintenance therapy achieved significant clinical remission and endoscopic response compared to placebo.

GRAVITI is a randomized, double-blind, placebo-controlled Phase 3 study designed to evaluate the efficacy and safety of subcutaneous Tremfya in patients with moderate to severe active Crohn's disease who have had an inadequate response or intolerance to conventional therapies (such as corticosteroids or immunomodulators) or biologics (TNF antagonists or vedolizumab).

Tremfya is a fully human monoclonal antibody. In addition to targeting IL-23, this antibody can also simultaneously bind to the receptor CD64 on IL-23-producing cells. IL-23 is a cytokine secreted by activated monocytes/macrophages and dendritic cells, and it is a driving factor in immune-mediated diseases such as UC.

Cell Therapy Extending the Lives of ALS Patients

On October 29, BrainStorm Cell Therapeutics announced the presentation of expanded study results for its investigational cell therapy, debamestrocel, at the 2024 NEALS (Northeast ALS Consortium) Annual Meeting held online.

In Brainstorm's previous Phase 3 clinical trial, a total of 10 participants with amyotrophic lateral sclerosis (ALS) (6 received debamestrocel, 4 received placebo) were enrolled in the open-label Expanded Access Program (EAP). The study was divided into two 28-week cycles with an interval between the two cycles. Participants received intrathecal injections of debamestrocel every 8 weeks, with up to 6 administrations.

The study results showed that at the last follow-up of the EAP, 9 out of 10 participants survived. The survival curve demonstrated a statistically significant survival advantage for the debamestrocel group compared to the externally matched control group, with a median survival time of 46.6 months in the debamestrocel group versus 41.1 months in the matched control group.