June 25, 2025 -- Amicus Therapeutics (Nasdaq: FOLD) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease (LOPD). "We are delighted that we will now be able to offer a compelling new treatment option to patients living with late-onset Pompe disease in Japan. We are grateful to the MHLW and to Japan’s Pompe community, including the patients,

June 25, 2025 — Alvotech (NASDAQ: ALVO), a global biotech company specializing in the development and manufacturing of biosimilar medicines for patients worldwide, Kashiv Biosciences LLC (“Kashiv”), a fully integrated biopharmaceutical company headquartered in New Jersey, US, and Advanz Pharma Holdco Limited (“Advanz Pharma”), a UK-headquartered global pharmaceutical company with a strategic focus on specialty, hospital, and rare disease medicines, today announced positive topline results from a

Riliprubart earns orphan drug designation in the US for antibody-mediated rejection in solid organ transplantation Ongoing phase 2 study evaluating riliprubart for the potential prevention and treatment of active antibody-mediated rejection in kidney transplant recipients Riliprubart was also designated orphan drug for the investigational use in chronic inflammatory demyelinating polyneuropathy in the US and EU June 25, 2025. The US Food and Drug Administration (FDA) has granted orphan drug des

- Near doubling of 1-year overall survival (OS), increased median OS of 4 months (12.5 vs 8.5 months), and 43% reduction in risk of death in patients treated with at least one cycle (4 weeks) of elraglusib plus gemcitabine/nab-paclitaxel (GnP) vs GnP alone - Patients with liver metastases experienced a 2.5x improvement in 1-year OS with a 38% reduction in risk of death when treated with elraglusib plus GnP June 24, 2025 -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”)

Good Tolerability and Robust Tumor Uptake were Observed in TNBC Animal Models Auger Emitters Offer the Potential Benefit of Enhanced Cytotoxicity, Safety and Ease-of-Use June 24, 2025 -- Cellectar Biosciences, Inc. (Nasdaq: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of drugs for the treatment of cancer, today announced that the company has submitted a protocol with the U.S. Food and Drug Administration (FDA) for a Phase 1b Dose Finding study

Submission Seeks Approval of GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Comprehensive Data Package Includes Positive Results from Phase 3 STRIVE-ON Safety Trial of GTx-104 Submission Has the Potential to Trigger Exercise of up to $7.6 million in Gross Proceeds from 2023 Financing Warrants Tied to FDA Acceptance of NDA for Review June 25, 2025 -- Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage, biopharma company

- End-of-Phase 2 Meeting with FDA for Mild-to-Moderate Alzheimer’s Disease Scheduled - - Applications for IND and Breakthrough Status Filed for Dementia with Lewy Bodies - June 25, 2025 -- Cognition Therapeutics, Inc., (the Company or Cognition) (NASDAQ: CGTX), a clinical stage company developing drugs that treat neurodegenerative disorders, announced that the company will conduct an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) on July 9, 2025. During the meeting, the

June 17, 2025 -- Medicovestor, Inc., a biotechnology company advancing next-generation antibody-drug conjugates (ADCs), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ADoBind MC001, its lead candidate for the treatment of pancreatic cancer. Pancreatic cancer is among the most aggressive and fatal malignancies, with a five-year survival rate under 10% and limited treatment advances over the past decades. ADoBind MC001 is designed to

Two-part clinical study programme to evaluate change in lean and fat mass in patients receiving two different doses of S-pindolol benzoate whilst 1) receiving the GLP-1 agonist semaglutide; and 2) after cessation of semaglutide treatment Initial proof-of-concept data expected in second half of 2026 25 June 2025. Actimed Therapeutics Ltd (“Actimed”), a UK based clinical stage specialty pharmaceutical company focused on the treatment of cancer cachexia and other muscle wasting disorders, today an

Biodexa Pharmaceuticals PLC (“Biodexa” or “the Company”), (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, is pleased to announce the activation of the first clinical study site for its Serenta trial in patients with familial adenomatous polyposis (FAP). The trial, which is now enrolling, represents a significant milestone in the development of a potential new treatment option for FAP.