Tag: small molecule

Zurich, July 29, 2025 – MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory and infectious diseases, announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA's ODD, underscoring MP1032's potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side effects like osteoporosis, growth stunting, and metabolic issues.

PARIS, FRANCE, 24 July 2025 - Ipsen announced today that the European Commission has approved Cabometyx^® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues.

MN-166 COMBAT ALS trial down to single digit required enrollment while MN-001 hypertriglyceridemia trial in type 2 diabetes patients requires final two patients

Wuppertal, Germany, July 24, 2025 - Aicuris Anti-infective Cures AG today announced the completion of patient enrollment in its pivotal Phase 3 trial, PRIOH-1, evaluating pritelivir for the treatment of refractory and/or treatment resistant herpes simplex virus (HSV) infections in immunocompromised patients. The company’s lead candidate is a small molecule specifically inhibiting the viral helicase-primase complex, which represents a novel mechanism of action distinct from current HSV therapies.

- XIPERE Now Approved for Use in Multiple Global Markets -