POMC

Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update

Retrieved on: 
Thursday, February 22, 2024

-- 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

“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

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

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

-- 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.

Rhythm Pharmaceuticals Presents Data from its Long-term Extension Study of Setmelanotide Showing Sustained and Deepened BMI Reduction in Patients with Hypothalamic Obesity at One Year at ObesityWeek® 2023

Retrieved on: 
Tuesday, October 17, 2023

BOSTON, Oct. 17, 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 several data presentations showing that setmelanotide therapy resulted in sustained and deepened weight loss in patients with severe obesity caused by rare MC4R pathway diseases during The Obesity Society’s Annual Meeting at ObesityWeek®.

Key Points: 
  • Rhythm and its collaborators delivered a total of six presentations at the conference in Dallas from October 14 to 17.
  • In addition, the Company will host an investor conference call and webcast to discuss these data presentations on Wednesday, October 18, 2023, at 8:00 a.m.
  • This conference call will be accessible under “Events & Presentations” in the Investor Relations section of the Company’s website at www.rhythmtx.com.
  • A replay will be available on the Rhythm website for 30 days following the presentation.

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

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 Announces Six Abstracts to be Presented at The Obesity Society’s Annual Meeting at ObesityWeek ®

Retrieved on: 
Tuesday, October 10, 2023

The lead author on this research is Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, NY.

Key Points: 
  • The lead author on this research is Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, NY.
  • Rhythm will post these data presentations on the Company’s website on the “Publications and Presentations” page following the conference.
  • This conference call will be accessible under “Events & Presentations” in the Investor Relations section of the Company’s website at www.rhythmtx.com .
  • A replay will be available on the Rhythm website for 30 days following the presentation.

Rhythm Pharmaceuticals Presents New Data at ESPE 2023

Retrieved on: 
Friday, September 22, 2023

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 Reports Second Quarter 2023 Financial Results and Business Update

Retrieved on: 
Tuesday, August 1, 2023

-- 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.