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...
treatment News
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)...
SOUTH SAN FRANCISCO, Calif. — Rigel Pharmaceuticals, Inc. today announced a collaboration with CONNECT, an international collaborative network of pediatric cancer centers, to conduct a Phase 2 clinical trial to evaluate REZLIDHIA® (olutasidenib) in combination with temozolomide as maintenance therapy in newly diagnosed pediatric and young adult patients with high-grade glioma...
SOUTH SAN FRANCISCO, Calif. — Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced a peer-reviewed publication in Leukemia & Lymphoma on data from an analysis of the Phase 2 study evaluating REZLIDHIA® (olutasidenib), a potent, selective, oral, small-molecule inhibitor of mutant isocitrate dehydrogenase-1 (mIDH1), in patients with mIDH1 acute myeloid leukemia (AML) who were relapsed/refractory...
SOUTH SAN FRANCISCO, Calif. — Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced that its oral Syk inhibitor, R788, is being evaluated in a Phase 2 clinical trial funded, designed and implemented by the National Cancer Institute (NCI), part of the U.S. National Institutes of Health. This open-label, single arm clinical...
Riociguat may be a useful option for patients with pulmonary arterial hypertension (PAH) in whom phosphodiesterase-5 inhibitor (PDE5i) treatment is insufficient, according to the results of a recent study published in the journal Lancet Respiratory Medicine. Researchers evaluated clinical improvement, defined as an absence of clinical worsening and prespecified improvements in...