Rhythm Pharmaceuticals, Inc. (NASDAQ: RYTM)

Rhythm Pharmaceuticals, Inc. (Rhythm), a company operating within the biopharmaceutical industry, is dedicated to transforming the lives of patients and their families living with rare neuroendocrine diseases. The organization's primary business activities revolve around the development of therapies for rare MC4R pathway diseases, including obesity caused by genetic variants in the POMC, PCSK1, LEPR, and other genes.

IMCIVREE, Rhythm's lead asset, is an MC4R agonist approved by the U.S. FDA for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS), or Bardet-Biedl syndrome (BBS). IMCIVREE is the first-ever therapy developed for patients with certain rare diseases that is approved or authorized in the United States, European Union (EU), Great Britain, Canada, and other countries and regions.

Rhythm generates revenue through the sale of IMCIVREE and its pipeline of new drug candidates, including RM-718, an investigational, MC4R-specific agonist designed for weekly administration, and LB54640, an investigational, oral MC4R agonist. The company's customer base includes patients with rare MC4R pathway diseases, healthcare providers, and pharmaceutical companies.

The company's MC4R pathway program is designed to expand the total number of patients who would benefit from setmelanotide therapy or who the company believes could be addressed by one of its new drug candidates. The program focuses on developing therapies for rare MC4R pathway diseases. Rhythm estimates that there are approximately 4,600 to 7,500 patients in the United States with obesity due to biallelic POMC, PCSK1, or LEPR deficiencies, and BBS, and approximately 5,000 to 10,000 patients in the United States with hypothalamic obesity.

Currently available therapies for general obesity have limited efficacy and do not specifically address the impaired signaling in the MC4R pathway. Rhythm believes that its therapies, including IMCIVREE, have the potential to provide clinically meaningful improvements in obesity and hyperphagia by restoring lost function in the MC4R pathway. The company's competitive position is based on its focus on developing therapies for rare MC4R pathway diseases, its proprietary sequencing data, and its estimated responder rates to setmelanotide therapy.

Rhythm has entered into several licensing agreements, including one with Ipsen Pharma S.A.S. for the use of Camurus' drug delivery technology, FluidCrystal, to formulate setmelanotide. The company has also entered into an Exclusive License Agreement with RareStone Group Ltd. for the development, manufacture, and commercialization of IMCIVREE in China.

The biotechnology and pharmaceutical industries are intensely competitive and subject to rapid and significant technological change. Rhythm has competitors with general obesity medications in a number of jurisdictions, many of which have substantially greater name recognition, commercial infrastructures, and financial, technical, and personnel resources than the company. However, Rhythm's focus on rare MC4R pathway diseases and its proprietary data provide a unique position within the industry.