A Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity

A Phase 2, Single-arm, Open-label Study to Assess Efficacy and Safety of LB54640 in Patients With Obesity Due to POMC, PCSK1, or LEPR Deficiency.

The purpose of the study was to assess the efficacy of LB54640 in participants with rare genetic disorders of obesity

Patients with obesity due to POMC, PCSK1 or LEPR deficiency will be enrolled to receive LB54640 by oral administration for 16 weeks. After 16 weeks of treatment, the participant may enter a long term extension phase and receive treatment for up to 1 year.

Inclusion Criteria:

• Participants aged ≥12 years at the time of enrollment and diagnosed with genetic obesity due to POMC, PCSK1, or LEPR deficiency mutations.
• Obesity is defined as BMI ≥30 kg/m2 for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants <18 years of age.

Exclusion Criteria:

• Participants with recent (within 2 months, prior to Screening and up to enrollment) intensive diet/exercise regimen that resulted in >2% weight loss, use of medication to treat obesity (within 3 months of first dose of LB54640 with certain exceptions), or weight loss surgery within 6 months prior to Screening or prior surgery which resulted in >10% weight loss with no evidence of weight regain are excluded
• History of major surgical procedure
• Weight loss surgery within the previous 6 months
• Any history of a suicide attempt, or any suicidal behavior
• HbA1c >10.9%
• Fasting glucose level >270 mg/dL