Rhythm Pharmaceuticals, Inc.
Rhythm Pharmaceuticals is a global biopharmaceutical company dedicated to transforming the lives of patients with rare neuroendocrine diseases through rapid advancement of care and precision medicines that address the root cause. The company focuses on developing therapies for rare diseases such as hyperphagia and obesity caused by impairments in the MC4R pathway, with a mission to enable patients to live their fullest lives through stigma-free care.
Industries
Nr. of Employees
medium (51-250)
Products
IMCIVREE (setmelanotide) injection
An approved MC4R-pathway agonist injection for chronic weight management in patients with certain monogenic or syndromic forms of obesity caused by specific genetic variants, indicated for adults and pediatric patients 6 years and older where appropriate genetic testing shows relevant variants.
Pipeline MC4R-targeted candidates (investigational)
A portfolio of investigational MC4R-targeted assets in development, including orally administered and weekly-dosed MC4R-specific agonists and early-stage discovery programs for additional rare endocrine diseases such as congenital hyperinsulinism.
IMCIVREE (setmelanotide) injection
An approved MC4R-pathway agonist injection for chronic weight management in patients with certain monogenic or syndromic forms of obesity caused by specific genetic variants, indicated for adults and pediatric patients 6 years and older where appropriate genetic testing shows relevant variants.
Pipeline MC4R-targeted candidates (investigational)
A portfolio of investigational MC4R-targeted assets in development, including orally administered and weekly-dosed MC4R-specific agonists and early-stage discovery programs for additional rare endocrine diseases such as congenital hyperinsulinism.
Services
Clinical trial program
Design and conduct of multi-center clinical trials for therapies targeting rare neuroendocrine and MC4R pathway diseases, including Phase 1–3 studies and special cohort management.
Expanded access (compassionate use) program
Assessment and management of expanded access requests for investigational therapies when clinical trial participation is not possible and no comparable treatment exists.
Investigator-initiated and real-world study support
Support for external investigator-initiated studies in areas such as natural history, genotype–phenotype correlations, energy expenditure, hyperphagia, and real-world use of therapies.
Patient support program (education and access)
Programs providing education, resources, and access assistance tailored for patients and caregivers affected by rare MC4R pathway diseases.
Clinical trial program
Design and conduct of multi-center clinical trials for therapies targeting rare neuroendocrine and MC4R pathway diseases, including Phase 1–3 studies and special cohort management.
Expanded access (compassionate use) program
Assessment and management of expanded access requests for investigational therapies when clinical trial participation is not possible and no comparable treatment exists.
Investigator-initiated and real-world study support
Support for external investigator-initiated studies in areas such as natural history, genotype–phenotype correlations, energy expenditure, hyperphagia, and real-world use of therapies.
Patient support program (education and access)
Programs providing education, resources, and access assistance tailored for patients and caregivers affected by rare MC4R pathway diseases.
Expertise Areas
- Clinical trial management for rare diseases
- Genetics-driven precision medicine
- MC4R pathway drug discovery
- Peptide and small-molecule therapeutic development
Key Technologies
- DNA sequencing data platform
- Genotype-driven patient selection
- Peptide therapeutics
- Oral small-molecule MC4R agonists