Rhythm Pharmaceuticals, Inc. develops and commercializes therapies for rare neuroendocrine diseases, centered on IMCIVREE (setmelanotide), an MC4R agonist for hyperphagia and severe obesity linked to rare MC4R pathway disorders. Company updates commonly cover U.S. and international commercialization of setmelanotide, regulatory authorizations and label expansions for acquired hypothalamic obesity, Bardet-Biedl syndrome, POMC/PCSK1 deficiency and LEPR deficiency, and safety information tied to approved uses.
Rhythm news also follows clinical data from setmelanotide studies, including acquired hypothalamic obesity and genetically caused MC4R pathway diseases, along with development of investigational MC4R agonists bivamelagon and RM-718 and preclinical small molecules for congenital hyperinsulinism. Recurring corporate items include financial results, specialty-pharmacy access arrangements, healthcare conference presentations and board governance changes.
BOSTON, Dec. 1, 2022 - Rhythm Pharmaceuticals (Nasdaq: RYTM) announced CEO David Meeker will participate in a fireside chat at the BofA Securities 2022 Virtual Biotech SMID Cap Conference on December 8, 2022, at 9:40 a.m. ET. The live audio webcast will be accessible on the Investor Relations section of their website, with a replay available for 30 days post-event. Rhythm focuses on treating severe obesity from rare melanocortin-4 receptor pathway diseases with its FDA-approved medication setmelanotide, which targets specific genetic obesity conditions.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced significant advancements in its Phase 3 clinical trial of IMCIVREE (setmelanotide) for Bardet-Biedl syndrome (BBS), with results published in The Lancet Diabetes and Endocrinology. The trial met its primary and all key secondary endpoints, achieving substantial weight and hunger reductions after one year. Approximately 32.3% of patients aged 12 and older met the primary endpoint. Setmelanotide is now approved as the first therapy for chronic weight management in patients with BBS in the U.S. and EU, addressing a critical need for this rare genetic condition.
Rhythm Pharmaceuticals (RYTM) reported strong demand for IMCIVREE® (setmelanotide), with over 120 prescriptions since FDA approval for Bardet-Biedl Syndrome (BBS). The company successfully completed a $140 million public offering, extending its cash runway into 2025. Setmelanotide received FDA Breakthrough Therapy Designation for hypothalamic obesity, with a Phase 3 trial expected to begin early 2023. Revenue for Q3 2022 reached $4.3 million, up from $1.0 million in Q3 2021, while net loss was $40.9 million, compared to $35.1 million in the prior year.
BOSTON, Nov. 7, 2022 - Rhythm Pharmaceuticals (NASDAQ: RYTM) announced the grant of inducement equity awards to two new employees on November 3, 2022. The total of 23,400 shares comprises 15,600 stock options and 7,800 restricted stock units (RSUs). The stock options have an exercise price of $24.32 and vest over four years, while RSUs vest annually over the same period. These equity awards are part of Rhythm’s 2022 Employment Inducement Plan, adopted on February 9, 2022, aimed at attracting talent to support its mission in treating hyperphagia and severe obesity linked to rare MC4R pathway diseases.
Rhythm Pharmaceuticals presented important data at ObesityWeek® 2022, showcasing the efficacy of setmelanotide in treating Bardet-Biedl Syndrome (BBS). Notably, 84% of participants in a Phase 3 trial showed improvements in weight, hunger, or quality of life. The Clinical Registry Investigating Bardet-Biedl Syndrome (CRIBBS) revealed a positive correlation between hyperphagia severity and body mass index (BMI) in affected children. Furthermore, the research indicates that obesity remains prevalent, with many patients experiencing ongoing weight challenges. Rhythm aims to address hunger and weight issues linked to rare MC4R pathway diseases.
Rhythm Pharmaceuticals announced positive results from its Phase 2 trial of setmelanotide for hypothalamic obesity, showing that 89% of participants achieved a BMI reduction of over 5% at 16 weeks. The therapy also demonstrated sustained weight loss in a long-term extension, with a 21.1% average BMI reduction over 29 weeks and 26.7% over 41 weeks for some patients. Additionally, the FDA granted Breakthrough Therapy designation for setmelanotide, and a Phase 3 trial is set to begin in early 2023 to further evaluate its effectiveness.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announces that setmelanotide has received Breakthrough Therapy Designation from the FDA for treating hypothalamic obesity. This designation accelerates the development and review process for medications targeting serious conditions with unmet needs. Setmelanotide demonstrated significant efficacy in a Phase 2 trial, with all 11 patients achieving a BMI decrease of over 5%, averaging a 17.2% reduction. The company plans to initiate a Phase 3 trial in early 2023 to further evaluate this treatment.
Rhythm Pharmaceuticals (Nasdaq: RYTM) is set to present full data from its Phase 2 clinical trial of setmelanotide for hypothalamic obesity during the Obesity Society’s ObesityWeek 2022 on November 2, 2022. The conference call will include results from 18 patients. Additionally, Rhythm will report its Q3 2022 earnings on November 8, 2022, at 8:00 a.m. ET. Setmelanotide, approved for chronic weight management, targets hyperphagia and severe obesity linked to rare MC4R pathway diseases. Rhythm is actively advancing its clinical programs and has submitted a Type II variation application for setmelanotide in the EU.
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) announced the inaugural International Meeting on Pathway-Related Obesity: Vision of Excellence (IMPROVE) 2022 in Berlin, focused on rare pathway-related obesities driven by MC4R variants. At the event, the company presented data from an exploratory Phase 2 Basket Study evaluating setmelanotide in patients with specific MC4R variants. Results showed that 30.4% of patients in the rescuable cohort achieved significant BMI reduction, while the predictive value of the in vitro assay was limited. Setmelanotide is FDA-approved for chronic weight management in certain genetic obesity cases.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced the appointment of Dana Washburn, M.D., as Senior Vice President of Clinical Development, effective September 26, 2022. Dr. Washburn will lead the clinical program for setmelanotide, a treatment for hyperphagia and severe obesity linked to rare MC4R pathway diseases. With over 20 years of experience in biopharmaceuticals, he previously held leadership roles at Alexion Pharmaceuticals and other organizations. Rhythm is advancing setmelanotide through multiple clinical trials, supported by recent regulatory approvals in the U.S. and Europe.