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.
Rhythm Pharmaceuticals (RYTM), a commercial-stage biopharmaceutical company specializing in rare neuroendocrine diseases, has announced its upcoming participation in the Oppenheimer 35th Annual Healthcare Life Sciences Conference. David Meeker, M.D., who serves as Chair, President and Chief Executive Officer, will engage in a fireside chat on Wednesday, February 12, 2025, at 8:40 a.m. ET.
The presentation will be accessible via webcast in the Investor Relations section of Rhythm's website under 'Events & Presentations' at www.rhythmtx.com. Interested parties can access a replay of the webcast on the company's website for 30 days following the presentation.
Rhythm Pharmaceuticals (RYTM) announced preliminary Q4 2024 net revenues of approximately $42 million from global IMCIVREE sales, up 26% from Q3 2024, and full-year 2024 revenues of approximately $130 million compared to $77.4 million in 2023. U.S. sales contributed about 74% of Q4 and 73% of full-year revenues.
The company reported several pipeline developments: completion of enrollment in the Japanese cohort of Phase 3 trial for acquired hypothalamic obesity (HO), with topline data expected in H1 2025; completion of enrollment in two substudies of Phase 3 EMANATE trial for MC4R pathway diseases; and plans to initiate a new Phase 2 trial for Prader-Willi syndrome in Q1 2025. The company is also advancing trials for bivamelagon and RM-718 in HO treatment.
Rhythm Pharmaceuticals (NASDAQ: RYTM) announced FDA approval for expanded indication of IMCIVREE® (setmelanotide) to treat children as young as 2 years old with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed POMC, PCSK1, and LEPR deficiencies. IMCIVREE is the first precision medicine targeting MC4R pathway impairment, addressing hyperphagia and obesity in these rare conditions.
The approval follows successful clinical trials showing significant weight and hunger reduction. The drug was previously approved for patients 6 years and older, and has received marketing authorization in the UK and EU for patients as young as 2 years. BBS affects approximately 4,000-5,000 people in the US, while POMC, PCSK1, and LEPR deficiency obesities affect 600-2,500 people.
Rhythm Pharmaceuticals (NASDAQ: RYTM) announced that the UK's MHRA has expanded marketing authorization for IMCIVREE (setmelanotide) to treat obesity and control hunger in patients as young as 2 years old with Bardet-Biedl syndrome (BBS) or POMC, PCSK1, or LEPR deficiency. Previously approved for patients 6 years and older in 2022, IMCIVREE received NICE recommendation for NHS reimbursement in May 2024 for BBS patients aged 6-17. The drug will be available through the Medicines for Children program by year-end. A similar application is under FDA priority review with a PDUFA date of December 26, 2024.
Rhythm Pharmaceuticals (NASDAQ: RYTM) presented new real-world data at ESPE 2024 showing positive results from setmelanotide treatment in pediatric patients with hypothalamic obesity. Four pediatric patients achieved >5% weight reduction at three months, with weight decreases ranging from 5.2% to 9.6%. The patients included two cases each of acquired and congenital hypothalamic obesity, treated under France's early-access program. All patients remain on therapy with no new safety signals observed.
The company also announced plans for a new 34-week substudy to evaluate setmelanotide in 39 patients with congenital hypothalamic obesity, with first patient enrollment expected in Q1 2025. Rhythm maintains its timeline for topline data from its pivotal Phase 3 trial in acquired hypothalamic obesity in H1 2025.
Rhythm Pharmaceuticals (RYTM) published results from its Phase 3 VENTURE trial evaluating setmelanotide in children aged 2-5 years with rare genetic obesity disorders. The study, published in The Lancet Diabetes & Endocrinology, showed significant results: 83% of patients achieved BMI Z-score reduction, with an 18% mean decrease in BMI from baseline. 91% of caregivers reported reduced hunger in patients. The treatment was generally well-tolerated with no serious adverse events. The FDA has granted Priority Review for label expansion with a PDUFA date of December 26, 2024.
Rhythm Pharmaceuticals (Nasdaq: RYTM) has granted inducement restricted stock units (RSUs) to three new employees, totaling 15,750 shares of common stock. The grants were approved on November 7, 2024, under the company's 2022 Employment Inducement Plan. The RSUs will vest over four years, with 25% vesting annually on each hire date anniversary, contingent on continued employment. These grants comply with Nasdaq Listing Rule 5635(c)(4) and are specifically designed as employment inducements.
Rhythm Pharmaceuticals (RYTM), a global commercial-stage biopharmaceutical company specializing in rare neuroendocrine diseases, has announced its participation in two upcoming investor conferences. David Meeker, Chair, President and CEO, will participate in fireside chats at the Guggenheim Securities Healthcare Innovation Conference on November 11 and the Stifel 2024 Healthcare Conference on November 18, both at 11:30 a.m. ET. The presentations will be webcasted and available on the company's website under the Investor Relations section, with replays accessible for 30 days.
Rhythm Pharmaceuticals (RYTM) reported Q3 2024 global sales of IMCIVREE® at $33.3 million, up 14% from Q2 2024. Early-access program data showed adult patients with acquired hypothalamic obesity achieved 12.8% mean BMI reduction after three months. The FDA accepted sNDA for Priority Review to expand IMCIVREE label for patients as young as 2 years, with PDUFA date of Dec. 26, 2024. Q3 net loss was $45.0 million ($0.73 per share). The company has $298.4 million cash on hand, expected to support operations into 2026.
Rhythm Pharmaceuticals (RYTM) presented five new data sets at ObesityWeek® 2024, showcasing positive results for setmelanotide therapy across multiple studies. Real-world data showed a mean BMI decrease of 12.8% in adult patients with acquired hypothalamic obesity after three months, extending to 21.3% at six months. The Phase 2 DAYBREAK trial demonstrated a 12.4% mean BMI reduction in patients with rare MC4R pathway diseases after 40 weeks. Notable results included a 23.8% BMI reduction in pediatric patients aged 2-6 years with BBS or POMC/LEPR Deficiency at 18 months. Analysis of 43,000 genetic samples revealed associations between genetic variants and early-onset obesity.