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LB54640

Phase 2

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment28

FDA Designations

No designations recorded

Clinical trial landscape

LB54640 · 1 trial · 1 indication

Phase 2 1
NCT06046443A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic ObesityHypothalamic Obesity
COMPLETED28 Analytics
PHASE2COMPLETED
A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity
Hypothalamic ObesityUnlock trial analytics

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 to 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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Frequently asked questions about LB54640

What is LB54640 used for?

LB54640 is an investigational small molecule being developed for the treatment of hypothalamic obesity, a form of obesity that results from damage to the hypothalamus. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.

How does LB54640 work?

LB54640 is a small molecule designed to address hypothalamic obesity, though its specific molecular target has not been disclosed. The drug is being studied for its ability to manage weight in patients with this condition, which is characterized by weight gain due to hypothalamic damage.

Who is developing LB54640?

LB54640 is being developed by Rhythm Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RYTM. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with hypothalamic obesity.

What phase is LB54640 in?

LB54640 is in Phase 2 clinical development. A Phase 2 trial has been completed, and the drug remains investigational, meaning it has not yet received approval from the U.S. Food and Drug Administration or other regulatory bodies for commercial use.

What clinical trials is LB54640 in?

LB54640 has been studied in one completed Phase 2 clinical trial, identified as NCT06046443. This randomized, double-blind, placebo-controlled study enrolled 28 participants aged 12 years and older with hypothalamic obesity and was conducted in the United States and United Kingdom.

Is LB54640 the same as any other drug?

No alternative names for LB54640 have been reported. The drug is identified solely by its code name LB54640 in clinical trial registries and by its developer, Rhythm Pharmaceuticals.