Proopiomelanocortin

Rhythm Pharmaceuticals Announces Updates on MC4R Pathway Programs at R&D Event

Retrieved on: 
Wednesday, December 6, 2023
Bardet–Biedl syndrome, Quality of life, Genotype, Food, Stage 2, Hyperpigmentation, Life, Shanda, Polyphagia, FDA, Type, Weight loss, POMC, BMI, European, Disease, MC1R, University of Florida, MD, Pediatric endocrinology, New Drug Application, University, SIM1, Obesity, TBX3, Endocrinology, PCSK1, Human, Patient, Proopiomelanocortin, Hunger, BBS, LEPR, Pharmaceutical industry

-- Company to host R&D webcast today at 8:00 a.m. ET--

Key Points: 
  • “Today, we are excited to showcase our commitment to understanding the MC4R pathway and developing the next generation of therapies,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • The hyperphagia and severe obesity of rare genetically-caused MC4R pathway diseases can present early in life, and these data show potential efficacy in patients younger than 6.
  • A live webcast of the R&D call will be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/ .
  • The archived webcast will be available on Rhythm Pharmaceuticals’ website approximately two hours after the conference call and will be available for 30 days following the call.

LG Chem Announces Initiation of Two Phase 2 Trials (ROUTE & SIGNAL) of Novel Oral MC4R Agonist LB54640 in Patients with Rare Forms of Genetic Obesity

Retrieved on: 
Tuesday, October 17, 2023
Research, FDA, Genetics, Chemicals, Plastics, Clinical Trials, Manufacturing, Biotechnology, Pharmaceutical, Health, Science, Pharmacokinetics, Obesity, POMC, Weight loss, LEPR, Safety, Proopiomelanocortin, PCSK1, LG Chem, Genetic disorder, Body mass index, Patient, Polyphagia, Pharmaceutical industry, MC4R

LG Chem is making progress in the development of the world's first oral treatment for rare forms of obesity.

Key Points: 
  • LG Chem is making progress in the development of the world's first oral treatment for rare forms of obesity.
  • LG Chem Life Sciences today announced the initiation of two phase 2 clinical trials of LB54640, the company’s novel oral small molecule melanocortin-4 receptor (MC4R) agonist, which is in development for the potential treatment of certain rare forms of obesity.
  • The Phase 2 ROUTE trial is a single-arm, open-label study that will evaluate the safety, tolerability, pharmacokinetics, and weight loss efficacy of LB54640.
  • Participants will receive one of three doses of LB54640 by oral administration once daily for up to 52 weeks.

Rhythm Pharmaceuticals Presents New Data at ESPE 2023

Retrieved on: 
Friday, September 22, 2023
Polyphagia, LEPR, Adolescent medicine, University of Alberta, Risk, Obesity, University, Pediatrics, Metabolic syndrome, Doctor of Philosophy, Genetics, Pediatric endocrinology, Bardet–Biedl syndrome, ESPE, Endocrine Society, POMC, ROAD, BBS, Early-onset Alzheimer's disease, Patient, Division, Endocrinology, University of Ulm, Medicine, Body mass index, MC4R, European Society for Paediatric Research, Imperial College London, Proopiomelanocortin, Vaccine, Nursing, Rhythm, The Hague

BOSTON, Sept. 22, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting being held September 21-23, 2023 in The Hague, Netherlands.

Key Points: 
  • BOSTON, Sept. 22, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting being held September 21-23, 2023 in The Hague, Netherlands.
  • “We are excited to deliver multiple presentations at ESPE 2023 including statistics that deepen the understanding of the genetics of obesity from our Rare Obesity Advanced Diagnosis™ (ROAD) testing program as we learn more about the importance of genetics in patients with early-onset, severe obesity,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • “Frequency of Obesity-Related Gene Variants in a European Population with Early-Onset, Severe Obesity,” as presented orally by Jesús Argente M.D., Ph.D., Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain, shows that among individuals with early-onset, severe obesity, approximately 31.4% carried variants believed to be associated with obesity.
  • In addition, Rhythm is hosting a satellite symposium at ESPE 2023, titled, “Hyperphagia and early-onset, severe obesity: The role of precision medicine in the treatment of leptin melanocortin-4 receptor (MC4R) pathway diseases.”

Rhythm Pharmaceuticals Announces New Employment Inducement Grants

Retrieved on: 
Wednesday, December 7, 2022
Bardet–Biedl syndrome, Benzyl alcohol, Weight loss, Allergy, Depression, Fertility, Hunger, Obesity, Professional Football Compensation Committee, Toxicity, Growth, Medicines and Healthcare products Regulatory Agency, Priapism, COVID-19, MHRA, Genetic testing, Diagnosis, Employment, Excipient, Food, FDA, MC4R, Safety, PCSK1, Skin, Pregnancy, EC, Plasma, LEPR, Milk, Cyprus Securities and Exchange Commission, Child, Polyphagia, DNA, VUS, Heart rate, Financial compensation, Alström syndrome, Patient, United, Proopiomelanocortin, RSU, Pigment, Metabolic acidosis, BBS, Data analysis, Blood pressure, Risk, Pharmaceutical industry, Medicine, Birth control, POMC, Setmelanotide, European

About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, 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). The European Commission (EC) and Great Britain’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 genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

Key Points: 

    Rhythm Pharmaceuticals to Present at BofA Securities 2022 Virtual Biotech SMID Cap Conference

    Retrieved on: 
    Thursday, December 1, 2022
    United, Skin, Hunger, Risk, Pregnancy, Allergy, EC, All rights reserved, Patient, Chair, Metabolic acidosis, Bardet–Biedl syndrome, Polyphagia, Conference, Pigment, Priapism, VUS, Bank of America, Benzyl alcohol, Alström syndrome, Obesity, FDA, Plasma, COVID-19, Growth, LEPR, Excipient, Diagnosis, Fertility, Weight loss, Medicines and Healthcare products Regulatory Agency, Food, Setmelanotide, DNA, Toxicity, Proopiomelanocortin, Webcast, Depression, Genetic testing, Data analysis, Safety, MC4R, BBS, Milk, Child, PCSK1, MHRA, Blood pressure, Heart rate, Pharmaceutical industry, Medicine, Birth control, Rhythm, European, POMC

    About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, 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). The European Commission (EC) and Great Britain’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 genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

    Key Points: 
    • A replay of the webcast will be available on the Rhythm website for 30 days following the presentation.
    • 2022 Rhythm Pharmaceuticals, Inc. All Rights Reserved.
    • Inthe United StatesandEurope, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
    • This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

    Rhythm Pharmaceuticals Announces New Employment Inducement Grants

    Retrieved on: 
    Monday, November 7, 2022
    Pigment, Plasma, VUS, Growth, Private Securities Litigation Reform Act, Excipient, Proopiomelanocortin, Benzyl alcohol, Blood pressure, PCSK1, Bardet–Biedl syndrome, Nasdaq, Professional Football Compensation Committee, Toxicity, Population, Weight loss, Risk, Time, Financial compensation, Pregnancy, Skin, Obesity, Depression, United, COVID-19, Alström syndrome, BBS, Product, DNA, Genetic testing, Milk, Hunger, GLOBE, Priapism, RSU, FDA, Patient, Medicines and Healthcare products Regulatory Agency, Data analysis, Heart rate, Fertility, Child, Safety, Adult, MC4R, Food, Diagnosis, Cyprus Securities and Exchange Commission, Metabolic acidosis, LEPR, EC, Allergy, Employment, MHRA, Polyphagia, Pharmaceutical industry, Dietary supplement, Medicine, Birth control, Setmelanotide, European, POMC

    About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, 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). The European Commission (EC) has 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.The UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) authorized setmelanotide for the treatment of obesity and the control of hunger associated with 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 genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

    Key Points: 
    • These inducement stock options and inducement RSUs are subject to the terms of the Rhythm Pharmaceuticals, Inc. 2022 Employment Inducement Plan (the "Inducement Plan").
    • The Inducement Plan is used exclusively for the grant of equity awards to individuals as an inducement material to the employees entering into employment with Rhythm pursuant to Nasdaq Listing Rule 5635(c)(4).
    • The Inducement Plan was adopted by Rhythms board of directors on February 9, 2022.
    • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases.

    Rhythm Pharmaceuticals Announces New Employment Inducement Grants

    Retrieved on: 
    Thursday, October 6, 2022
    Skin, Polyphagia, Diarrhea, Abdominal pain, Depression, Financial compensation, BBS, Obesity, Pigment, Vomiting, Proopiomelanocortin, EC, Sexual arousal, Suicidal ideation, DNA, Fetus, European Commission, Regulation of tobacco by the U.S. Food and Drug Administration, Senior, Private Securities Litigation Reform Act, Incidence, RSU, Genetic testing, Hunger, GLOBE, FDA, Bardet–Biedl syndrome, Nausea, MHRA, Diagnosis, Employment, Risk, Professional Football Compensation Committee, Headache, Alström syndrome, Data analysis, Cyprus Securities and Exchange Commission, Medicines and Healthcare products Regulatory Agency, LEPR, Patient, PCSK1, Nasdaq, Food, MC4R, Setmelanotide, VUS, Adult, Safety, Pregnancy, COVID-19, Pharmaceutical industry, Birth control, POMC

    These inducement stock options and inducement RSUs are subject to the terms of the Rhythm Pharmaceuticals, Inc. 2022 Employment Inducement Plan (the "Inducement Plan").

    Key Points: 
    • These inducement stock options and inducement RSUs are subject to the terms of the Rhythm Pharmaceuticals, Inc. 2022 Employment Inducement Plan (the "Inducement Plan").
    • The Inducement Plan is used exclusively for the grant of equity awards to individuals as an inducement material to the employees entering into employment with Rhythm pursuant to Nasdaq Listing Rule 5635(c)(4).
    • The Inducement Plan was adopted by Rhythms board of directors on February 9, 2022.
    • To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

    Rhythm Pharmaceuticals Presents New Disease Burden Findings and Analyses of Setmelanotide Data in Children and Adolescents with Rare MC4R Pathway Diseases at ESPE 2022

    Retrieved on: 
    Monday, September 19, 2022
    Adult, Bardet–Biedl syndrome, Endocrine Society, Leptin receptor, Microdeletion syndrome, Department, MC4R, FDA, UCL Great Ormond Street Institute of Child Health, MD, Pediatrics, Adolescence, Poster, European Society for Medical Oncology, Obesity, Patient, Mental health, BBS, POMC, Very early onset inflammatory bowel disease, Nasdaq, Ageing, Caregiver, EC, Polyphagia, ESPE, LEPR, GLOBE, Pediatric endocrinology, Disease, Pharmaceutical industry, Residential care, Setmelanotide, Pediatric nursing, Proopiomelanocortin, Rhythm

    BOSTON, Sept. 19, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, announced the presentation of new findings on the burden of hyperphagia and obesity on patients with Bardet-Biedl syndrome (BBS) and their caregivers during the 60th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE 2022) held in Rome on Sept. 15 to 17.

    Key Points: 
    • Rhythm and its collaborators delivered one poster and three oral presentations during ESPE 2022, including data from new analyses that showed setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients with rare MC4R pathway diseases across three separate pivotal trials.
    • CARE-BBS participants at the time of the survey were residents of the United States (n=60), Canada (n=62), United Kingdom (n=59) or Germany (n=61).
    • In addition, the findings illustrate how the negative impact of hyperphagia on caregivers affects family dynamics, emotional well-being, and caregiver employment.
    • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases.

    Rhythm Pharmaceuticals Announces New Employment Inducement Grants

    Retrieved on: 
    Wednesday, September 7, 2022
    COVID-19, Safety, Setmelanotide, BBS, Cyprus Securities and Exchange Commission, Depression, GLOBE, EC, Adult, Suicidal ideation, Nausea, Patient, Obesity, Regulation of tobacco by the U.S. Food and Drug Administration, Private Securities Litigation Reform Act, Fetus, Genetic testing, Incidence, Food, DNA, LEPR, Headache, Bardet–Biedl syndrome, Pigment, Alström syndrome, Nasdaq, FDA, Vomiting, Proopiomelanocortin, PCSK1, Employment, Professional Football Compensation Committee, RSU, Medicines and Healthcare products Regulatory Agency, Diarrhea, Skin, Data analysis, MHRA, European Commission, Polyphagia, Risk, VUS, Sexual arousal, Diagnosis, Abdominal pain, Financial compensation, MC4R, Hunger, Pregnancy, Pharmaceutical industry, Birth control, POMC

    About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, 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). The European Commission (EC) has 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. The UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) authorized setmelanotide for the treatment of obesity and the control of hunger associated with 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 genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

    Key Points: 
    • These inducement stock options and inducement RSUs are subject to the terms of the Rhythm Pharmaceuticals, Inc. 2022 Employment Inducement Plan (the "Inducement Plan").
    • The Inducement Plan is used exclusively for the grant of equity awards to individuals as an inducement material to the employees entering into employment with Rhythm pursuant to Nasdaq Listing Rule 5635(c)(4).
    • The Inducement Plan was adopted by Rhythms board of directors on February 9, 2022.
    • To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

    PreventionGenetics Receives FDA Approval for its Companion Diagnostic Genetic Test as a Class II Medical Device

    Retrieved on: 
    Thursday, June 30, 2022
    LEPR, FDA, Patient, Arousal, Lists of diseases, Benzyl alcohol, Suicidal ideation, Abdominal pain, Adult, FDA Center for Devices and Radiological Health, Skin, CDRH, Device, Risk, Vomiting, Genetic testing, VUS, Depression, Obesity, Prevalence, Proopiomelanocortin, Cdx, PCSK1, BBS, Food, Sexual arousal, Headache, Center, Companion diagnostic, Pigment, FACMG, Alström syndrome, Incidence, Diarrhea, Food and Drug Administration Amendments Act of 2007, Physician, Pregnancy, Nausea, Fetus, Medical device, Vaccine, Birth control, Pharmaceutical industry, POMC, Doctor of Philosophy

    MARSHFIELD, Wis., June 30, 2022 /PRNewswire/ -- PreventionGenetics, a subsidiary of Exact Sciences Corp., announced today that it was recently granted marketing authorization for the POMC/PCSK1/LEPR companion diagnostic (CDx) genetic test as a Class II device by the Center for Devices and Radiological Health (CDRH), a division of the Food and Drug Administration (FDA). PreventionGenetics, an accredited germline DNA testing laboratory offering genetic testing to patients and physicians around the world, developed the test in collaboration with Rhythm Pharmaceuticals, Inc.

    Key Points: 
    • PreventionGenetics, an accredited germline DNA testing laboratory offering genetic testing to patients and physicians around the world, developed the test in collaboration with Rhythm Pharmaceuticals, Inc.
    • The POMC/PCSK1/LEPR companion diagnostic (CDx) genetic test is for in vitro diagnostic use only and must be ordered by a qualified professional in accordance with clinical laboratory regulations.
    • For more information on PreventionGenetics and the Companion Diagnostic test, visit https://www.preventiongenetics.com/sponsoredTesting/Rhythm/CDX .
    • To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .