— LB54640 has shown targeted effect on MC4R without hyperpigmentation —
BOSTON, MA, USA I July 23, 2024 I Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the first patients have been dosed in the Company’s Phase 2 clinical trial evaluating LB54640, an investigational oral melanocortin-4 receptor (MC4R) agonist, in hypothalamic obesity.
“Based on LG Chem’s significant preclinical work and encouraging results from a Phase 1 trial, we believe LB54640 has the potential to be an effective oral therapy for treating MC4R pathway diseases without hyperpigmentation,” said David Meeker, M.D., Chair, Chief Executive Officer and President of Rhythm Pharmaceuticals. “We look forward to advancing this molecule with the goal of offering a full portfolio of treatment options to patients struggling with hyperphagia and severe obesity and ensuring they get the treatment that is right for them.”
The Phase 2 trial is a randomized, placebo-controlled, double-blind study to assess the effect of LB54640 on safety, weight reduction, hunger, and quality of life in patients 12 years of age and older (N=28) with hypothalamic obesity. Patients will take an oral daily dose of either LB54640 (low, middle, or high dose) or placebo for 14 weeks. The primary endpoint of the study is the change from baseline in body mass index after 14 weeks of treatment, and patients may continue on therapy for up to 52 weeks.
Rhythm entered into a global licensing agreement with LG Chem, Ltd. (“LG Chem”) for LB54640 in January 2024. In a Phase 1 trial in healthy overweight adults, LB54640 demonstrated dose-dependent weight reduction and a favorable safety profile with no changes in blood pressure or heart rate observed and no hyperpigmentation observed.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.
Setmelanotide Indication
In the United States, setmelanotide is indicated 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 BBS.
In the European Union, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. In Europe, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
SOURCE: Rhythm Therapeutics
Post Views: 6,781
— LB54640 has shown targeted effect on MC4R without hyperpigmentation —
BOSTON, MA, USA I July 23, 2024 I Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the first patients have been dosed in the Company’s Phase 2 clinical trial evaluating LB54640, an investigational oral melanocortin-4 receptor (MC4R) agonist, in hypothalamic obesity.
“Based on LG Chem’s significant preclinical work and encouraging results from a Phase 1 trial, we believe LB54640 has the potential to be an effective oral therapy for treating MC4R pathway diseases without hyperpigmentation,” said David Meeker, M.D., Chair, Chief Executive Officer and President of Rhythm Pharmaceuticals. “We look forward to advancing this molecule with the goal of offering a full portfolio of treatment options to patients struggling with hyperphagia and severe obesity and ensuring they get the treatment that is right for them.”
The Phase 2 trial is a randomized, placebo-controlled, double-blind study to assess the effect of LB54640 on safety, weight reduction, hunger, and quality of life in patients 12 years of age and older (N=28) with hypothalamic obesity. Patients will take an oral daily dose of either LB54640 (low, middle, or high dose) or placebo for 14 weeks. The primary endpoint of the study is the change from baseline in body mass index after 14 weeks of treatment, and patients may continue on therapy for up to 52 weeks.
Rhythm entered into a global licensing agreement with LG Chem, Ltd. (“LG Chem”) for LB54640 in January 2024. In a Phase 1 trial in healthy overweight adults, LB54640 demonstrated dose-dependent weight reduction and a favorable safety profile with no changes in blood pressure or heart rate observed and no hyperpigmentation observed.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.
Setmelanotide Indication
In the United States, setmelanotide is indicated 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 BBS.
In the European Union, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. In Europe, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
SOURCE: Rhythm Therapeutics
Post Views: 6,781
