Welcome to our dedicated page for Rhythm Pharmaceu news (Ticker: RYTM), a resource for investors and traders seeking the latest updates and insights on Rhythm Pharmaceu stock.
Rhythm Pharmaceuticals (RYTM) is a commercial-stage biopharmaceutical company pioneering precision medicines for rare neuroendocrine diseases, including genetic obesity disorders. This page aggregates all official announcements, press releases, and verified news coverage related to RYTM's therapeutic advancements and operational developments.
Investors and stakeholders will find timely updates on regulatory milestones, clinical trial outcomes, financial disclosures, and strategic partnerships. The curated collection prioritizes primary-source materials including FDA submissions, research publications, and executive communications to ensure informational accuracy.
Track RYTM's progress in developing MC4R pathway therapies like IMCIVREE® (setmelanotide), with updates spanning global approvals, research expansions, and market access initiatives. Content is organized chronologically to facilitate efficient monitoring of the company's progress in addressing rare genetic conditions.
Bookmark this page for direct access to Rhythm Pharmaceuticals' latest verified updates. For complete market analysis, combine these resources with broader sector research and regulatory filings.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced positive results from its Phase 2 Basket Study, demonstrating proof-of-concept for setmelanotide in patients with genetic obesity disorders. The study found that 34.3% of patients with HET obesity achieved significant weight loss. Updated data suggests that 100,000-200,000 patients in the U.S. could benefit from this treatment. Additionally, the pivotal Phase 3 trial for Bardet-Biedl Syndrome showed significant improvements in pediatric patients’ BMI-Z scores. Rhythm plans to initiate further studies to expand setmelanotide's application.
Rhythm Pharmaceuticals (Nasdaq: RYTM) will host a virtual event on January 26, 2021, to present updates on its exploratory Phase 2 Basket Study and genetic sequencing efforts. Management will discuss data for setmelanotide in individuals with heterozygous obesity linked to genetic variants in POMC, PCSK1, or LEPR genes, as well as SRC1 and SH2B1 deficiency. The company has sequenced samples from approximately 37,500 individuals with severe obesity to enhance understanding and diagnosis of these conditions.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $100M. This voucher was granted by the FDA following the approval of IMCIVREE™ (setmelanotide) for chronic weight management in patients with genetic deficiencies causing severe obesity. The sale will provide non-dilutive capital to further develop setmelanotide, which has been approved for patients aged 6 and older. The upfront payment is conditioned on customary closing requirements, with an expected transaction completion following U.S. antitrust clearance.
On January 5, 2021, Pharvaris announced the appointment of David Meeker, M.D., as Chair of the Board and Robert Glassman, M.D., as Director. Dr. Meeker, currently CEO of Rhythm Pharmaceuticals (RYTM), brings extensive experience in rare disease drug development, while Dr. Glassman, a venture partner at OrbiMed Advisors, offers financial expertise. Pharvaris aims to enhance treatment options for hereditary angioedema through novel oral bradykinin-B2-receptor antagonists. The leadership changes are expected to bolster the company's strategic initiatives and clinical programs.
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.
