Bardet–Biedl syndrome

Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update

Retrieved on: 
Thursday, February 22, 2024
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-- 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
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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 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
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-- 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 Receives Positive CADTH Reimbursement Recommendation for IMCIVREE® (setmelanotide)

Retrieved on: 
Thursday, November 2, 2023
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BOSTON, Nov. 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 announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended that IMCIVREE® (setmelanotide) be reimbursed by CADTH-participating public drug plans, with conditions, for weight management in adult and pediatric patients six years of age and older with obesity due to Bardet-Biedl syndrome (BBS).

Key Points: 
  • People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
  • BBS may also be associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.
  • “This recommendation represents a significant milestone for Canadian patients living with obesity due to BBS.
  • We appreciate CADTH’s recommendation and their recognition of the rare genetic nature of BBS, the unmet needs of these patients and that IMCIVREE is the only available therapy that targets the underlying disease mechanism of BBS,” said Carol Stiff, General Manager of Rhythm Pharmaceuticals Canada Inc.

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
BMI, Obesity, POMC, Weight loss, LEPR, Bardet–Biedl syndrome, Setmelanotide, MC4R, BBS, Conference call, Disease, WHO, World Health Organization, Hypothalamic disease, Conference, Polyphagia, NIH, Birth control, Pharmaceutical industry, Nursing, Rhythm, Patient

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.

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

Retrieved on: 
Tuesday, October 10, 2023
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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
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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 ICD-10 Diagnosis Code Established for Bardet-Biedl Syndrome

Retrieved on: 
Wednesday, September 6, 2023
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The new code, Q87.83 Bardet-Biedl syndrome (BBS), takes effect Oct. 1, 2023.

Key Points: 
  • The new code, Q87.83 Bardet-Biedl syndrome (BBS), takes effect Oct. 1, 2023.
  • “Finding and connecting patients, families and physicians across the country has been extremely challenging because of the lack of a dedicated ICD-10 code as patients with BBS are lumped in with many other genetic syndromes,” said Tim Ogden, President of the BBS Foundation.
  • “We believe this new disease specific ICD-10 code marks an important advance for the care of patients with BBS,” said Jennifer Chien, Rhythm Executive Vice President and Head of North America.
  • People living with BBS also may experience cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.