Rhythm Pharmaceuticals, Inc.
Biopharmaceutical company developing precision therapies for rare neuroendocrine diseases caused by impairment of the MC4R pathway. Operates clinical development programs across phases, maintains a de-identified sequencing database to support variant interpretation and patient identification, conducts CMC and cGMP operations for peptide therapeutics, and provides patient support and post‑approval safety programs. Maintains global regulatory and compliance functions and an active clinical pipeline including injectable and oral MC4R‑targeted candidates and discovery work in congenital hyperinsulinism.
Industries
Nr. of Employees
medium (51-250)
Rhythm Pharmaceuticals, Inc.
Patents
Method of treating melanocortin-4 receptor-associated disorders in heterozygous carriers
US-12655178-B2
View DetailsMethod of treating melanocortin-4 receptor-associated disorders in heterozygous carriers
US-11702448-B2
View Details
Method of treating melanocortin-4 receptor-associated disorders in heterozygous carriers
US-12655178-B2
View DetailsMethod of treating melanocortin-4 receptor-associated disorders in heterozygous carriers
US-11702448-B2
View DetailsProducts
setmelanotide (MC4R receptor agonist) injection
Sterile peptide solution for subcutaneous administration developed to activate the melanocortin‑4 receptor pathway to reduce excess body weight and hyperphagia in select rare genetic and syndromic conditions.
Investigational oral MC4R agonist (development-stage)
Orally administered MC4R‑specific agonist in clinical development intended to avoid hyperpigmentation and evaluated for hypothalamic obesity indications.
Investigational long‑acting weekly MC4R agonist (development-stage)
Weekly dosing MC4R‑specific agonist in clinical development designed for extended dosing intervals and reduced hyperpigmentation risk for neuroendocrine obesity indications.
Discovery pipeline for congenital hyperinsulinism (CHI)
Early‑stage discovery programs evaluating candidate therapeutics for congenital hyperinsulinism to address persistent hypoglycemia in affected patients.
setmelanotide (MC4R receptor agonist) injection
Sterile peptide solution for subcutaneous administration developed to activate the melanocortin‑4 receptor pathway to reduce excess body weight and hyperphagia in select rare genetic and syndromic conditions.
Investigational oral MC4R agonist (development-stage)
Orally administered MC4R‑specific agonist in clinical development intended to avoid hyperpigmentation and evaluated for hypothalamic obesity indications.
Investigational long‑acting weekly MC4R agonist (development-stage)
Weekly dosing MC4R‑specific agonist in clinical development designed for extended dosing intervals and reduced hyperpigmentation risk for neuroendocrine obesity indications.
Discovery pipeline for congenital hyperinsulinism (CHI)
Early‑stage discovery programs evaluating candidate therapeutics for congenital hyperinsulinism to address persistent hypoglycemia in affected patients.
Services
Design and execution of multi‑phase clinical trials (including pediatric studies), dose titration and efficacy/safety assessments for therapies targeting MC4R‑pathway disorders and related neuroendocrine conditions.
Use of a de‑identified sequencing database and biochemical screening to support identification and interpretation of variants relevant to MC4R‑pathway diseases and to inform clinical development decisions.
Programs providing education, injection training, access support and caregiver resources for patients receiving therapy for rare MC4R‑pathway diseases.
Corporate compliance program implementation, regulatory submission support and post‑approval safety reporting to meet global regulatory requirements.
Operational review and processing of physician requests for access to investigational therapies for eligible patients outside of clinical trials.
Provision of scientific priorities and administrative support for IIS focused on natural history, genotype‑phenotype correlations, energy expenditure, hyperphagia and real‑world evidence.
Design and execution of multi‑phase clinical trials (including pediatric studies), dose titration and efficacy/safety assessments for therapies targeting MC4R‑pathway disorders and related neuroendocrine conditions.
Use of a de‑identified sequencing database and biochemical screening to support identification and interpretation of variants relevant to MC4R‑pathway diseases and to inform clinical development decisions.
Programs providing education, injection training, access support and caregiver resources for patients receiving therapy for rare MC4R‑pathway diseases.
Corporate compliance program implementation, regulatory submission support and post‑approval safety reporting to meet global regulatory requirements.
Operational review and processing of physician requests for access to investigational therapies for eligible patients outside of clinical trials.
Provision of scientific priorities and administrative support for IIS focused on natural history, genotype‑phenotype correlations, energy expenditure, hyperphagia and real‑world evidence.
Expertise Areas
- Clinical trial management for rare neuroendocrine diseases
- MC4R‑pathway therapeutic development across peptide and small‑molecule modalities
- Pediatric clinical development and dosing titration
- Genetic sequencing and variant interpretation for rare obesity
Key Technologies
- De‑identified DNA sequencing database
- Genetic epidemiology and variant interpretation
- Peptide therapeutic development
- Oral small‑molecule MC4R agonist development
News & Updates
Regulatory approval in the U.S. for a therapy targeting MC4R‑pathway‑related obesity due to certain genetic deficiencies.
European regulatory authorization for the MC4R‑pathway therapy for specific genetic obesity indications.
Regulatory approval widened to include Bardet‑Biedl syndrome for treatment of hyperphagia and severe obesity.
Strategic acquisitions and early clinical milestones including acquisition of assets and first dosing of additional clinical candidates expanding the development portfolio.
Company lists completed Phase 3 programs and ongoing Phase 2 studies across MC4R‑targeted therapies and indications.
Regulatory approval in the U.S. for a therapy targeting MC4R‑pathway‑related obesity due to certain genetic deficiencies.
European regulatory authorization for the MC4R‑pathway therapy for specific genetic obesity indications.
Regulatory approval widened to include Bardet‑Biedl syndrome for treatment of hyperphagia and severe obesity.
Strategic acquisitions and early clinical milestones including acquisition of assets and first dosing of additional clinical candidates expanding the development portfolio.
Company lists completed Phase 3 programs and ongoing Phase 2 studies across MC4R‑targeted therapies and indications.