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) announced positive topline results from its pivotal Phase 3 trial of setmelanotide for Bardet-Biedl syndrome (BBS) and Alström syndrome. The study achieved its primary endpoint, with 34.5% of participants showing at least a 10% weight reduction. All responders were BBS patients, while none of the Alström participants met this endpoint. Rhythm plans to submit a supplemental New Drug Application (sNDA) to the FDA for BBS in the second half of 2021. The trial enrolled 32 BBS and 6 Alström patients, and setmelanotide was generally well tolerated with mild side effects.
Rhythm Pharmaceuticals (Nasdaq:RYTM) announced the appointments of Camille L. Bedrosian, M.D., and Lynn Tetrault, J.D., to its Board of Directors. Both leaders bring extensive experience in the pharmaceutical industry. Bedrosian, with a strong background in rare diseases, is EVP and CMO at Ultragenyx Pharmaceutical, while Tetrault has over two decades at AstraZeneca, including roles in executive leadership. Their addition is expected to enhance Rhythm's operational leadership and its commitment to developing therapies for rare genetic diseases of obesity.
Rhythm Pharmaceuticals announced FDA approval for IMCIVREE™ (setmelanotide) for chronic weight management in patients aged 6 or older with obesity caused by POMC, PCSK1, or LEPR deficiencies, confirmed by genetic testing. This marks the first therapy approved for these rare genetic diseases, expected to be commercially available in Q1 2021. The approval was based on two trials showing substantial weight loss in patients, alongside the issuance of a Rare Pediatric Disease Priority Review Voucher. The company aims to further explore the treatment's potential in related conditions.
Rhythm Pharmaceuticals announced interim Phase 2 data for setmelanotide, showing that its once-weekly formulation has safety and efficacy comparable to daily doses in treating obesity. Additionally, long-term extension study data indicated durable weight loss and decreased hunger over three years. The FDA has granted Breakthrough Therapy designation for setmelanotide, which targets rare genetic obesity disorders. The company aims for FDA approval for POMC and LEPR deficiency obesity by November 27, 2020, while maintaining a focus on developing precision therapies for patients with severe obesity.
Rhythm Pharmaceuticals reported its third-quarter 2020 financial results, highlighting key developments in its drug candidate setmelanotide. The FDA's PDUFA goal date for setmelanotide's approval for POMC and LEPR deficiency obesities is November 27, 2020. The company appointed Jennifer Chien and Yann Mazabraud to lead global commercial strategies. R&D expenses decreased to $23 million from $26.6 million year-over-year, while net loss narrowed to $33.8 million. As of September 30, 2020, cash reserves stood at $201.8 million, sufficient to fund operations through 2021.
Rhythm Pharmaceuticals released crucial findings from two significant Phase 3 studies indicating that setmelanotide effectively reduced weight and hunger in patients with POMC and LEPR deficiency obesity. Published in The Lancet Diabetes & Endocrinology, the studies showed 80% of participants with POMC deficiency and 45% with LEPR deficiency achieved at least a 10% weight loss. The FDA has accepted the New Drug Application for setmelanotide, with a PDUFA goal date set for November 27, 2020. These results underscore a promising treatment avenue for rare obesity disorders.
Rhythm Pharmaceuticals (Nasdaq:RYTM) has appointed Jennifer Chien as Executive Vice President, Head of North America, effective November 9, 2020. Ms. Chien, with over 20 years in rare diseases, will co-lead global commercial strategies alongside newly appointed EVP, Head of International, Yann Mazabraud. This leadership change comes as Rhythm prepares for the potential approval of setmelanotide, a therapy for rare genetic obesity disorders. The FDA has set a PDUFA date of November 27, 2020, for Rhythm's New Drug Application for setmelanotide.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced the appointment of Yann Mazabraud as Executive Vice President, Head of International, effective October 5, 2020. This organizational change aims to enhance the company's global strategy, particularly for its lead product, setmelanotide, which targets severe obesity linked to rare genetic disorders. Dr. Meeker praised Mazabraud's extensive experience in the biopharmaceutical sector. Additionally, Nithya Desikan, the previous CCO, will leave to pursue other opportunities, while a search for a Head of North American Operations is underway.
BOSTON, Sept. 09, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals (Nasdaq: RYTM) announced participation in two investor conferences this September. At the Morgan Stanley 18th Annual Global Healthcare Conference on September 16, 2020, David Meeker, M.D., will speak at 3:30 p.m. ET. On September 17, 2020, CFO Hunter Smith will present at the Cantor Virtual Global Healthcare Conference at 2:00 p.m. ET. Live webcasts of both events will be accessible on the company's website, with replays available for 30 days following each presentation.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced a corporate overview presentation at the Canaccord Genuity 40th Annual Growth Conference on August 13, 2020, at 9:30 a.m. ET. The company's leadership, including David Meeker, M.D., and Hunter Smith, will present insights on developing therapies for rare genetic disorders of obesity. Rhythm's investigational drug, setmelanotide, has FDA filing acceptance for certain obesity conditions with a PDUFA date of November 27, 2020. A live audio webcast will be accessible on their website, with a replay available for 30 days.