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Part C: RM-718

Phase 1

Hypothalamic Obesity | Small molecule | Rare Disease |Rhythm Pharmaceuticals, Inc.|Last Updated: Dec 22, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment150

FDA Designations

RMAT

Clinical trial landscape

Part C: RM-718 · 1 trial · 3 indications

Phase 1 1
NCT06239116A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway ImpairmentHypothalamic Obesity
RECRUITING150 Analytics
PHASE1RECRUITING
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Hypothalamic ObesityUnlock trial analytics

Study Endpoints

Primary Endpoints

Parts A, B, C, D: Safety and Tolerability Assessed by Number of Study Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 through the Safety-Follow-up call (up to Day 43 for all Part A cohorts, up to Day 70 for all Part B cohorts, up to Day 140 for Part C cohort, up to Day 210 for Part D cohort)

Secondary Endpoints

AUCtau measurement of RM-718
up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cmax measurement of RM-718
up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
Cmin measurement of RM-718
up to 168 hours post-dose on Day 1 (Parts A, B and C) and 168 hours post-dose on Day 22 (Parts B and C).
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
RM-718 (Cohort A1)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A2)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A3)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A4)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A5)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A6)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A7)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A8)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort A9)EXPERIMENTALSingle dose of RM-718 (4) or placebo (2)
RM-718 (Cohort B1)EXPERIMENTALMultiple ascending doses of RM-718 (4) or placebo (2)
RM-718 (Cohort B2)EXPERIMENTALMultiple ascending doses of RM-718 (4) or placebo (2)
RM-718 (Cohort B3)EXPERIMENTALMultiple ascending doses of RM-718 (4) or placebo (2)
RM-718 (Cohort B4)EXPERIMENTALMultiple ascending doses of RM-718 (4) or placebo (2)
RM-718 (Cohort B5)EXPERIMENTALMultiple ascending doses of RM-718 (4) or placebo (2)
RM-718 (Cohort B6)EXPERIMENTALMultiple ascending doses of RM-718 (4) or placebo (2)
RM-718 (Cohort C1)EXPERIMENTALMultiple ascending doses of RM-718 (30)
RM-718 (Cohort D1)EXPERIMENTALMultiple ascending doses of RM-718 (up to 30)

Interventions

NameTypeDescription
Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)DRUGSingle ascending dose of RM-718 or placebo (matched to specific RM-718 Part A dose cohort)
Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)DRUGMultiple ascending doses of RM-718 or placebo (matched to specific RM-718 Part B dose cohorts)
Part C: RM-718DRUGMultiple ascending doses of RM-718
Part D: RM-718DRUGMultiple ascending doses of RM-718
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Eligibility Criteria

Age Range12 Years to 65 Years
SexALL
Healthy VolunteersYes
Study Sites7

Key Inclusion Criteria: Parts A and B: * Male and female subjects in good health aged 18-55 years of age at Screening. * Body mass index (BMI) ≥30 kg/m2. * Subjects who are medically healthy with normal or clinically insignificant screening results. * Subjects must use a highly effective form of c...

Countries:United States
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Frequently asked questions about Part C: RM-718

What is RM-718 used for?

RM-718 is an investigational small molecule being developed for hypothalamic obesity, a rare disease. It is also being studied in patients with Prader-Willi syndrome and other MC4R pathway impairments. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes RM-718?

RM-718 is being developed by Rhythm Pharmaceuticals, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol RYTM. The company is conducting a Phase 1 clinical trial of RM-718 in the United States.

What phase is RM-718 in?

RM-718 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 trial is actively recruiting participants in the United States.

What clinical trials is RM-718 in?

RM-718 is being studied in a Phase 1 clinical trial with the identifier NCT06239116. This trial is titled 'A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment' and is recruiting 150 participants aged 12 years and older in the United States.

Is RM-718 being studied in healthy volunteers?

Yes, RM-718 is being studied in healthy volunteers as part of its Phase 1 clinical trial. The trial also includes patients with hypothalamic obesity, Prader-Willi syndrome, and other MC4R pathway impairments. The study is randomized, double-blind, and controlled.