MC4R

Rhythm Pharmaceuticals Reports First Quarter 2024 Financial Results and Business Update

Retrieved on: 
화요일, 5월 7, 2024
EMANATE, POMC, FDA, Sale, BV, Shanda, Investment, PES, Body mass index, New Drug Application, BMI, LEPR, LG Chem, LG, Research, Weight, BBS, Bardet–Biedl syndrome, Webcast, MC4R, Prescription, Obesity, Completeness, Patient, Hyperpigmentation, Acquisition, EMA, Polyphagia, Marketing, SIGNAL, Annual general meeting, G&A, The Lancet, Pharmaceutical industry

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

Key Points: 
  • ET --
    BOSTON, May 07, 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 first quarter ended March 31, 2024.
  • This pivotal trial is progressing as planned, and we anticipate dosing the first patients with hypothalamic obesity in Japan in the second quarter of 2024.
  • Financial Guidance: For the year ending December 31, 2024, Rhythm anticipates approximately $250 million to $270 million in Non-GAAP Operating Expenses.
  • ET today to review its first quarter 2024 financial results and recent business activities.

Rhythm Pharmaceuticals Presents Data from Phase 3 Pediatrics Trial at Pediatric Endocrine Society Annual Meeting

Retrieved on: 
월요일, 5월 6, 2024
POMC, FDA, Shanda, PES, Body mass index, Food, New Drug Application, BMI, LEPR, Weight loss, BBS, Bardet–Biedl syndrome, MC4R, Obesity, Patient, Rhythm, Annual general meeting, Proopiomelanocortin, Pharmaceutical industry

BOSTON, May 06, 2024 (GLOBE NEWSWIRE) -- 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 Company delivered three presentations – one oral and two posters – at The Pediatric Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in Chicago, IL.

Key Points: 
  • BOSTON, May 06, 2024 (GLOBE NEWSWIRE) -- 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 Company delivered three presentations – one oral and two posters – at The Pediatric Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in Chicago, IL.
  • In the oral presentation, Rhythm showcased data that was previously announced on December 6, 2023, from its 52-week, Phase 3 pediatrics trial in patients between the ages of 2 and younger than 6 years (N=12) with Bardet-Biedl syndrome (BBS) or obesity due to proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency.
  • Treatment with setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in body mass index (BMI).
  • Rhythm also presented two additional poster presentations at PES 2024:

Endevica Bio drug candidate shows success in weight loss in new study

Retrieved on: 
화요일, 4월 23, 2024
Cancer, Diabetes, Weight loss, ESMO, Research, MC4R, ASCO, Obesity, Patient, Metabolism, Quality of life, Rat, Dietary supplement

Additionally, the treatment of A07D was found to provide durable weight loss even after cessation of treatment – suggesting that this approach avoids the problem of weight loss plateau and weight rebound seen with use of GLP1s alone.

Key Points: 
  • Additionally, the treatment of A07D was found to provide durable weight loss even after cessation of treatment – suggesting that this approach avoids the problem of weight loss plateau and weight rebound seen with use of GLP1s alone.
  • Results from the study support the idea that weight-regulating effects such as those advanced by Endevica Bio to address conditions such as involuntary cancer-related weight loss, can also be harnessed to regulate weight in several disease areas in which excess weight may be detrimental, including diabetes and obesity.
  • Meanwhile, Endevica Bio's most advanced drug candidate in the weight modulation space, TCMCB07 (B07), will be entering Phase 2 studies this summer.
  • "We're thrilled with the research results showing our drug candidate could potentially improve the lives of millions of people who seek to lose weight," said Russell Potterfield, CEO and Executive Chairman of Endevica Bio.

Rhythm Pharmaceuticals Secures $150 Million in Convertible Preferred Stock Financing

Retrieved on: 
월요일, 4월 1, 2024
Patient, Sale, LLP, Investment, Latham & Watkins, Ropes & Gray, MC4R, Security (finance)

The transaction is expected to close on or about April 15, 2024, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The transaction is expected to close on or about April 15, 2024, subject to the satisfaction of customary closing conditions.
  • Holders of the Preferred Stock will be entitled to a 6% cumulative annual dividend, commencing on the second anniversary of closing.
  • The Preferred Stock will vote with the common stock on an as-converted basis, subject to satisfaction of certain antitrust-related conditions.
  • The Company has agreed to grant the investors certain registration rights with respect to the common stock underlying the Preferred Stock.

Rhythm Pharmaceuticals Announces First Patient Dosed in Phase 1 Trial Evaluating RM-718, a Weekly MC4R-specific Agonist

Retrieved on: 
월요일, 3월 25, 2024
Human, Obesity, Hyperpigmentation, Hunger, Patient, Sigma receptor, Polyphagia, Safety, Pharmacokinetics, SAD, MC4R, Pharmaceutical industry

BOSTON, March 25, 2024 (GLOBE NEWSWIRE) -- 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 1 clinical trial of RM-718, an investigational, weekly melanocortin-4 receptor (MC4R)-specific agonist designed to be MC1R-sparing and to potentially avoid hyperpigmentation.

Key Points: 
  • “We are excited to begin clinical development of RM-718, our next-generation MC4R agonist,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • In addition, RM-718 was designed to provide a weekly dosing regimen and to avoid hyperpigmentation.”
    This Phase 1 trial is a three-part study to evaluate safety, tolerability and pharmacokinetics (PK).
  • Cohorts in Parts A and B are double-blind, placebo-controlled, and randomized 2:1.
  • Study participants will receive one weekly dose of either RM-718 or placebo in Part A, four weekly doses of either RM-718 or placebo in Part B, and four weekly doses of open-label RM-718 in Part C. RM-718 or placebo doses are administered weekly via subcutaneous injection.

Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update

Retrieved on: 
목요일, 2월 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 Clinical Development Plan of Setmelanotide for Hypothalamic Obesity in Japan

Retrieved on: 
목요일, 2월 22, 2024
Incidence, EMA, MC4R, Obesity, FDA, Growth, Astrocytoma, Polyphagia, Prevalence, Hunger, PMDA, European Medicines Agency, Weight gain, Patient, Brain, Craniopharyngioma, Pharmaceutical industry

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 announced plans to add a cohort of patients with hypothalamic obesity in Japan to its ongoing global Phase 3 clinical trial of setmelanotide, with dosing expected to begin in the third quarter of 2024.

Key Points: 
  • “Following constructive discussions with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), we are pleased to have developed a clear and efficient plan to support the potential approval of setmelanotide for hypothalamic obesity in Japan,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • The condition most frequently follows the growth and surgical removal or other treatment of craniopharyngioma, astrocytoma, or other rare brain tumors.
  • These individuals often experience rapid weight gain, a reduction in energy expenditure, and an increase in hunger leading to severe obesity within six to 12 months following the hypothalamic lesions.
  • Rhythm also announced today that it completed enrollment in the pivotal, 120-patient cohort in its global Phase 3 trial of setmelanotide in hypothalamic obesity with patients, aged 4 years or older with hypothalamic obesity randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration.

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: 
수요일, 2월 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: 
목요일, 1월 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 New Employment Inducement Grants

Retrieved on: 
금요일, 12월 15, 2023
MC4R, Polyphagia, Obesity, Patient, Compensated emancipation, Employment, Pharmaceutical industry

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 Rhythm’s board of directors on February 9, 2022.
  • The RSUs vest over four years, with 25% of the shares vesting on each anniversary of the applicable date of hire, subject to each such employee’s continued employment on each vesting date.