WOBURN, Mass. — Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RGT-61159 for the...
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Data are being presented at the 30th Annual Congress of the European Hematology Association 2025 (EHA2025) WOBURN, Mass. — Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today the presentation...
PRINCETON, N.J. — Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the results from its Phase 2a proof of concept study evaluating SGX945 (dusquetide) in the treatment of Behçet’s...
BOSTON – Rhythm (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced recent progress in the clinical development of the company’s lead product candidate, setmelanotide, a first-in-class melanocortin-4 receptor (MC4R) agonist. “We are pleased to announce important progress across our clinical...
BOSTON, Mass. — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced it has received orphan drug designation from Japan’s Ministry of Health, Labour and Welfare (MHLW) for setmelanotide as a treatment for acquired hypothalamic...
BOSTON, Mass. — 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 the National Institute for Health and Care Excellence (NICE) has issued guidance that recommends IMCIVREE® (setmelanotide) as an option for...
BOSTON, Mass. — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced positive preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS). The Company also announced plans to...
BOSTON, Mass. — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced the Company will hold a conference call and webcast on Monday, April 7, 2025 at 8:00 a.m. ET to disclose topline results from...
BOSTON, Mass. — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced positive topline results from its Phase 2 trial evaluating bivamelagon (formerly LB54640), an investigational oral melanocortin-4 receptor (MC4R) agonist, in patients with acquired...
TOKYO, Japan – RIBOMIC, Inc. (TOKYO:4591), a clinical-stage pharmaceutical company specializing in aptamer therapeutics, has conducted a Phase 2 clinical trial of umedaptanib pegol (anti-FGF2 aptamer) in pediatric patients (ages 5-14) with achondroplasia (ACH). The company announced today that the administration of the high-dose (0.6 mg/kg) subcutaneous injection (once biweekly)...