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LB54640

Phase 2

Hypothalamic Obesity | Small molecule | Metabolic |Rhythm Pharmaceuticals, Inc.|Last Updated: Feb 12, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment28
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06046443A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic ObesityPHASE2 COMPLETED 28Jul 11, 2024Feb 9, 2026Feb 12, 202610 United States, United Kingdom
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Study Endpoints
Primary Endpoints
Mean percentage change in BMI
From Baseline to Week 14
Secondary Endpoints
Frequency and severity of adverse events (AEs)
From Baseline to Week 52
Mean change in BMI
Baseline to Weeks 6, 10, 14
Proportion of patients who achieve ≥5% reduction in body weight
Baseline to Week 14
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LB54640 Low doseEXPERIMENTALParticipants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio.
LB54640 Middle doseEXPERIMENTALParticipants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio.
LB54640 High doseEXPERIMENTALParticipants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio.
PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio.
Open-label LB54640EXPERIMENTALEligible participants who provide consent to continue into the open-label portion of the study will receive open-label LB54640.
Interventions
NameTypeDescription
LB54640DRUGOral daily administration
PlaceboDRUGPlacebo matched to LB54640
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites10

Key Inclusion Criteria: * Documented evidence of acquired hypothalamic obesity (HO) * Age 12 years and older * Weight gain associated with the hypothalamic injury and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients \<18 years of age * Male and f...

Countries:United StatesUnited Kingdom
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