PCSK1

Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update

Retrieved on: 
Thursday, February 22, 2024
EMANATE, New Drug Application, Investment, Obesity, BMI, Webcast, Initiate, BBS, Stage 2, Hyperpigmentation, Documentation, Polyphagia, European, GAAP, European Medicines Agency, PCSK1, Type, Type 2, G&A, LG Chem, BV, Completeness, Shanda, Bardet–Biedl syndrome, Clinical trial, LEPR, Medicaid, IND, Prescription, Research, Hunger, Acquisition, Quarter, EMA, MC4R, FDA, Prevalence, PMDA, Patient, POMC, Body mass index, Pharmaceutical industry

-- Acquired global rights to oral MC4R agonist LB54640 from LG Chem --

Key Points: 
  • ET --
    BOSTON, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2023.
  • Fourth Quarter and Full Year 2023 Financial Results:
    Cash Position: As of December 31, 2023, cash, cash equivalents and short-term investments were approximately $275.8 million, as compared to $333.3 million as of December 31, 2022.
  • S,G&A Expenses: S,G&A expenses were $32.4 million in the fourth quarter of 2023 and $117.5 million for the year ended December 31, 2023, compared to $26.3 million in the fourth quarter of 2022 and $92.0 million for the year ended December 31, 2022.
  • ET today to review its fourth quarter and year end 2023 financial results and recent business activities.

Rhythm Pharmaceuticals Announces Positive Reimbursement Decision in Italy for IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome

Retrieved on: 
Wednesday, February 7, 2024
Visual impairment, European, Deficit spending, International, Polydactyly, Obesity, POMC, LEPR, Patient, Prevalence, BBS, Polyphagia, Gazzetta Ufficiale, Rhythm, Hunger, Medicine, MC4R, PCSK1, Bardet–Biedl syndrome, Hypogonadism, Pharmaceutical industry

“We are pleased that AIFA has recognized the need for a precision medicine for obesity and control of hunger in BBS,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm.

Key Points: 
  • “We are pleased that AIFA has recognized the need for a precision medicine for obesity and control of hunger in BBS,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm.
  • BBS is also associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.
  • In Italy, the Company estimates the prevalence of BBS to be between 1/125,000 and 1/160,000, with approximately 200 identified.
  • AIFA previously approved reimbursement for IMCIVREE for the treatment of obesity and control of hunger associated with biallelic proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or biallelic leptin receptor (LEPR) deficiency.

Rhythm Pharmaceuticals and LG Chem Life Sciences Enter Agreement for Rhythm to Acquire Global Rights to Oral MC4R Agonist LB54640

Retrieved on: 
Thursday, January 4, 2024
Blood pressure, MC4R, Bardet–Biedl syndrome, Food, Hyperpigmentation, Body mass index, Heart rate, Polyphagia, Chemistry, Chair, Weight loss, POMC, LG Chem, SIGNAL, MD, Pharmacokinetics, Obesity, PCSK1, Patient, Safety, BBS, LEPR, Pharmaceutical industry

BOSTON, Jan. 04, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced it has entered into a global licensing agreement with LG Chem, Ltd. ("LG Chem"), a leading global company headquartered in South Korea that specializes in life sciences as one of its core businesses, for LB54640, an investigational oral small molecule melanocortin-4 receptor (MC4R) agonist now in Phase 2 clinical trials.

Key Points: 
  • “We are excited to build upon the strong chemistry and early translational work performed by LG Chem, a highly regarded company with deep and broad experience and expertise in the pharmaceutical business.
  • LB54640 also demonstrated a favorable safety profile in the trial, with no changes in blood pressure or heart rate observed and no hyperpigmentation observed.
  • In addition, LB54640 has received orphan drug designation from the U.S. Food and Drug Administration for leptin receptor (LEPR) deficiency and proopiomelanocortin(POMC).
  • Also under the terms of the agreement, Rhythm has agreed to pay LG Chem up to $205 million upon achieving certain regulatory and sales milestones, as well as royalties.

Rhythm Pharmaceuticals Announces Completion of Screening for Enrollment in Setmelanotide Phase 3 Hypothalamic Obesity Trial and Additional Updates

Retrieved on: 
Thursday, January 4, 2024
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BOSTON, Jan. 04, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today provided a business update and announced it has completed screening for enrollment in the pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity.

Key Points: 
  • Rhythm closed screening and anticipates overenrolling the ongoing phase 3 hypothalamic obesity trial with more than 140 patients consented and going through active screening and baseline assessments.
  • In this trial, patients with acquired hypothalamic obesity aged 4 years or older will be randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration.
  • Rhythm estimates the prevalence of BBS to be approximately 700 people in Spain with approximately 100 patients identified.
  • The Company anticipates beginning Phase 1 in-human trials in the first half of 2024, including a multiple-ascending dose study in patients with hypothalamic obesity.

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 Reports Second Quarter 2023 Financial Results and Business Update

Retrieved on: 
Tuesday, August 1, 2023
ENDO, LEPR, Completeness, GCC, Marketing, Food and Drug Administration, Prescription, G&A, Ratio, Bardet–Biedl syndrome, CHI, Data analysis, Acquisition, Caregiver burden, Patient, Investment, Hyperpigmentation, Body mass index, Ageing, R, POMC, MC4R, BMI, FDA, IND, Caregiver, D, Polyphagia, Interest, Publication, Webcast, Obesity, MC1R, Clinical trial, Orphanet Journal of Rare Diseases, Date, Rare, Phase 3, PCSK1, Physician, Food, Pharmaceutical industry, Nursing, Life insurance, Video game, Reinsurance, Cryptocurrency, BBS, Rhythm, S

-- Enrollment ongoing in pivotal Phase 3 trial evaluating setmelanotide in hypothalamic obesity; based on rapid progress, study now expected to be fully enrolled by the end of 2023 --

Key Points: 
  • ET --
    BOSTON, Aug. 01, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global 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 reported financial results and provided a business update for the second quarter ended June 30, 2023.
  • R&D Expenses: R&D expenses were $33.5 million in the second quarter of 2023, as compared to $31.5 million in the second quarter of 2022.
  • S,G&A Expenses: S,G&A expenses were $30.0 million for the second quarter of 2023, as compared to $22.3 million for the second quarter of 2022.
  • ET today to review its second quarter 2023 financial results and recent business activities.

Rhythm Pharmaceuticals and Genpharm Announce Collaboration to Provide IMCIVREE® (setmelanotide) to Patients in Gulf Cooperation Council Countries

Retrieved on: 
Monday, May 22, 2023
Polyphagia, LEPR, Obesity, International, Prevalence, GCC, Bardet–Biedl syndrome, POMC, BBS, Patient, PCSK1, European, United Kingdom, MC4R, Gulf Cooperation Council, Rare, Heads of International Research Organizations, Pharmaceutical industry

Under the terms of this exclusive agreement, Genpharm will distribute IMCIVREE to patients in GCC countries for the treatment of hyperphagia and obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed biallelic pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.

Key Points: 
  • Under the terms of this exclusive agreement, Genpharm will distribute IMCIVREE to patients in GCC countries for the treatment of hyperphagia and obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed biallelic pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.
  • IMCIVREE is approved for these indications in the United States, Canada, the United Kingdom and the European Union.
  • Providers in the region now have the ability to prescribe IMCIVREE to qualifying patients who they believe will benefit from the therapy.
  • “Due to many factors, GCC countries have an increased prevalence of certain genetic diseases, creating a significant demand for precision therapies, like IMCIVREE, that address illnesses at their root cause,” said Kamel Ghammachi, Genpharm Chairman.

Rhythm Pharmaceuticals Announces Health Canada Approval of IMCIVREE® Setmelanotide Injection for Weight Management in Bardet-Biedl Syndrome or Genetically-confirmed Biallelic POMC, PCSK1, or LEPR Deficiency

Retrieved on: 
Monday, May 8, 2023
LEPR, Hospital for Sick Children, Faculty, University of Alberta, Benzyl alcohol, Priority review, Obesity, Depression, University, Weight loss, Bardet–Biedl syndrome, Pigment, POMC, BBS, Very early onset inflammatory bowel disease, Arousal, Publication, Patient, PCSK1, The Hospital, Medicine, Hypothalamus, Nausea, Suicidal ideation, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, Health Canada, Hunger, Human, Skin, Pharmaceutical industry, Nursing, Endocrinology, MC4R

BOSTON, May 08, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company, today announced that Health Canada has approved IMCIVREE® (setmelanotide solution for subcutaneous injection) for weight management in adult and pediatric patients 6 years of age and older with obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed biallelic pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency due to variants interpreted as pathogenic, likely pathogenic, or of uncertain significance. Please consult the IMCIVREE product monograph available at www.rhythmtx.ca.

Key Points: 
  • Setmelanotide has been shown to reduce the weight of patients living with BBS or POMC, PCSK1 or LEPR deficiency.
  • The trials met all primary endpoints and key secondary endpoints among patients at 52 weeks on therapy.
  • Results from these trials were featured in several publications, including the peer-reviewed journal The Lancet Diabetes and Endocrinology .
  • The most common adverse reactions were skin hyperpigmentation, injection site reactions and nausea.

Rhythm Pharmaceuticals Reports First Quarter 2023 Financial Results and Business Update

Retrieved on: 
Tuesday, May 2, 2023
LEPR, Polyphagia, Obesity, Food, International, Completeness, Body mass index, Bardet–Biedl syndrome, Food and Drug Administration, Clinical trial, Prescription, G&A, Advances in Therapy, Research, Ratio, POMC, BBS, CHI, Data analysis, Phase 3, Patient, Investment, Physician, PCSK1, National Association of Statutory Health Insurance Physicians, Webcast, MC4R, Ageing, R, BMI, FDA, Hunger, Acquisition, Human, Congenital hyperinsulinism, D, Pharmaceutical industry, Cryptocurrency, Rhythm, S

-- Management to host conference call today at 8:00 a.m. ET --

Key Points: 
  • ET --
    BOSTON, May 02, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global 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 reported financial results and provided a business update for the first quarter ended March 31, 2023.
  • R&D Expenses: R&D expenses were $37.9 million in the first quarter of 2023, as compared to $32.5 million in the first quarter of 2022.
  • S,G&A Expenses: S,G&A expenses were $24.6 million for the first quarter of 2023, as compared to $21.4 million for the first quarter of 2022.
  • ET today to review its first quarter 2023 financial results and recent business activities.