LB Pharmaceuticals announces positive schizophrenia trial data

The study achieved its main goal by showing that LB-102 significantly improved symptoms in patients with schizophrenia after 4 weeks of treatment. The 50 mg dose reduced symptoms by 5 points compared to the placebo, with strong evidence showing it worked. The 75 mg dose showed a 4.7-point reduction, while the highest dose of 100 mg led to an even greater 6.8-point improvement in symptoms. These results suggest LB-102 is effective in treating schizophrenia and warrant further study. 

Heather Turner, Chief Executive Officer of LB Pharmaceuticals, said, “The efficacy, safety, and tolerability data observed in this study reinforce the potential of LB-102 to provide a first-in-class benzamide option for patients in the U.S. with acute schizophrenia. Based on these findings, we plan to advance LB-102 into Phase III, explore its potential in additional psychiatric indications, and pursue the global development of a long-acting injectable formulation.”

Safety profile and next steps

The safety profile of LB-102 was promising, with a low incidence of extrapyramidal symptoms (EPS), few adverse events related to elevated prolactin, and minimal QT interval prolongation – which are common side effects of D2 antagonists. Additionally, the average weight gain in the treated subjects was 2 kg, a typical side effect seen with many antipsychotics. Across the 251 dosed patients, one case of sedation was reported.

Dr Anna Eramo, Chief Medical Officer of LB Pharmaceuticals, emphasised the strength of the study, saying, “This trial, designed to be considered a registrational trial in both size and statistical powering, provided high-quality data to inform our clinical path forward. We observed a clinically meaningful effect size across all three treatment arms, underscoring the clinical and statistical strength of the efficacy findings. We were pleased to see a generally safe and well-tolerated therapeutic profile across all three doses with the potential to further increase the dosage in future studies. Taken together, the strength of our findings in efficacy, safety, and tolerability underpins the potential of LB-102 to provide a much-needed treatment option for patients with schizophrenia.”

Dr John M. Kane, Professor of Psychiatry at the Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, noted the importance of these findings for patients with schizophrenia. He stated, “Today, people living with schizophrenia frequently switch between medications in search of a treatment that adequately addresses their symptoms while being tolerable and safe for long-term use. These data highlight the potential of LB-102 to provide a new option for patients in the U.S. as the first-in-class benzamide antipsychotic with favourable efficacy, safety and tolerability results and convenient once-daily dosing.”

With these positive results in hand, LB Pharmaceuticals plans to engage with regulatory authorities to finalise the design of its Phase III trial and expects to launch this programme later in 2025 or early 2026.