Espoo, Finland – Orion presented last week at the 2024 ASCO GU Cancers Symposium a poster with additional data from the ongoing Phase II CYPIDES trial evaluating the safety and efficacy of ODM-208 (or MK-5684), an investigational, oral CYP11A1 inhibitor, in heavily pretreated patients with metastatic castration-resistant prostate cancer (mCRPC)...
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QUINCY, Mass. – The Orphan Drug Act (ODA) celebrates its birthday on January 3. The year 2024 marks 41 years of revolutionizing drug development for rare diseases and helping millions of patients in the process. More than 25 million Americans have some kind of rare disease, defined as a condition...
ELK GROVE VILLAGE, Ill. — Orsini Specialty Pharmacy has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUA™ (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN). WAINUA is a subcutaneous injection that can be self-administered,...
ELK GROVE VILLAGE, Ill. – Orsini Specialty Pharmacy announced today that it is now dispensing tiopronin, the first available generic version, in the US, of THIOLA® (tiopronin). Tiopronin is a reducing and complexing thiol indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation...
MENLO PARK, Calif. — OrsoBio, a clinical-stage biopharmaceutical company developing treatments for obesity and related metabolic disorders, today announced positive topline results from a Phase 2a proof-of-concept study (NCT06564584) evaluating TLC-2716, the Company’s oral, liver-targeted liver X receptor (LXR) inverse agonist, in patients with severe hypertriglyceridemia (SHTG) and metabolic dysfunction–associated...
NESS ZIONA, Israel — Orvida Pharma (formerly Kamari Pharma), a privately held clinical-stage biotechnology company developing first and best-in-class treatments for rare and severe genetic skin diseases, today announced favorable safety results from the Phase 1a portion of its Phase 1a/1b clinical trial evaluating KM-023, a potential first-in-class oral TRPV3 inhibitor. The...
MADRID, Spain and CAMBRIDGE, Mass. — Oryzon Genomics, S.A., a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today that the final results of an observational clinical study aimed at psychometrically characterizing individuals with Phelan-McDermid syndrome (PMS) carrying deletions or pathogenic variants...
MADRID, Spain and CAMBRIDGE, Mass. — Oryzon Genomics, S.A. (ISIN Code: ES0167733015, Ticker: ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today announced that the European Medicines Agency (EMA) has authorized its Clinical Trial Application (CTA) to initiate a Phase II study of iadademstat, Oryzon’s potent and...
MADRID, Spain and CAMBRIDGE, Mass. — Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and global leader in epigenetics, today announced that the United States Patent and Trademark Office (USPTO) has granted U.S. patent US12,564,559 B2, relating to therapeutic combinations of iadademstat, Oryzon’s potent and selective LSD1...
New York, NY – OS Therapies Inc. (NYSE American: OSTX), the world leader in listeria-based cancer immunotherapies, is honored to celebrate the passage of the Mikaela Naylon Give Kids A Chance Act. Mikaela, like so many other children, courageously battled pediatric cancer – specifically Osteosarcoma. Her legacy through this legislation...