LUND, Sweden, July 18, 2024 (GLOBE NEWSWIRE) -- Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing drugs for the treatment of rare and severe primary mitochondrial disease, today announced a positive outcome of the interim analysis for FALCON, the potentially registrational study evaluating KL1333 in patients with primary mitochondrial disease. The study evaluates fatigue and myopathy as alternative independent primary endpoints, only one of which is required for a successful study readout. The independent Data Monitoring Committee (DMC) recommended continuing the study without modification. The favorable recommendation by the DMC to continue the FALCON study with a total of 180 patients validates the overall study design and confirms the strong safety profile of KL1333.
Renowned Rare-Disease Drug Developer Emil Kakkis, M.D., Ph.D., to Join Actio Biosciences Board of Directors
SHANGHAI, July 16, 2024 /PRNewswire/ -- Mabwell (688062.SH), an innovation-driven biopharmaceutical company with entire industry chain, announced that its self-developed novel B7-H3-targeting ADC (R&D code: 7MW3711) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), for the treatment of small cell lung cancer.
BETHESDA, Md., July 15, 2024 /PRNewswire/ -- A new survey of 800 rare disease (RD) patients and caregivers from the U.S., United Kingdom (UK), Spain, and Germany, was conducted by Avant Health and Rare Patient Voice between February and April 2024. 66% of RD patients report taking up to five years to get the RD diagnosed; this ranged between 74% (U.S) and 56% (UK). These results were part of a survey asking RD patient/caregiver opinions about their diagnostic journey, access to healthcare, and perspectives on living with a RD.
PARAMUS, N.J., July 10, 2024 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that its Board of Directors has appointed a new President at its US headquarters. Yukiteru Sugiyama, Ph.D. replaces retiring president Tsugio Tanaka, MSc as of June 30, 2024.
LOUISVILLE, Ky., July 10, 2024 /PRNewswire/ -- PharmassêtX Inc., a late-preclinical stage pharmaceutical development company, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to epigallocatechin gallate (EGCG), a well-characterized component of green tea that the Company is developing as a high-purity, high-potency drug for the treatment of inflammatory bowel disease (IBD).
PRINCETON, N.J., July 9, 2024 /PRNewswire/ -- 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 an interim update on the open-label, investigator-initiated study (IIS) evaluating extended HyBryte™ treatment for up to 12 months in patients with early-stage cutaneous T-cell lymphoma (CTCL). The trial is sponsored by Ellen Kim, MD, Director, Penn Cutaneous Lymphoma Program, Vice Chair of Clinical Operations, Dermatology Department, and Professor of Dermatology at the Hospital of the University of Pennsylvania who was a leading enroller in the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study for the treatment of early-stage CTCL.
NEW YORK, July 08, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of...