GlobeNewswire Press

  1. BioCryst's Oral BCX7353 Meets Primary Endpoint In Phase 3 APeX-2 Trial 5/21/2019

    BioCryst Pharmaceuticals, Inc.(Nasdaq: BCRX) today announced that the randomized (n=121), double-blind, placebo-controlled, Phase 3 APeX-2 trial of once-daily, oral BCX7353 for the prevention of hereditary angioedema (HAE) attacks achieved its primary endpoint for both dose levels (110 mg and 150 mg), with the 150 mg dose reducing the attack rate in HAE patients by 44 percent (p<0.001) compared to placebo.

  2. Abeona Therapeutics Announces FDA Clearance Of Investigational New Drug Application For ABO-202 Gene Therapy In CLN1 Disease 5/21/2019

    Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company is cleared to begin a Phase 1/2 clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease, following acceptance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA).

  3. Genocea Announces Positive Interim Immunogenicity Data From GEN-009 Neoantigen Vaccine Phase 1/2a Clinical Trial 5/16/2019

    Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing personalized cancer immunotherapies, today announced the first clinical results from its ongoing Phase 1/2a trial for GEN-009, the company’s lead neoantigen vaccine candidate.

  4. Mustang Bio And Nationwide Children's Hospital Receive Orphan Drug Designation For MB-108 (Oncolytic Virus C134) For The Treatment Of Malignant Glioma 5/16/2019

    Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, and Nationwide Children’s Hospital (“Nationwide Children’s”), one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MB-108 (oncolytic virus C134) for the treatment of malignant glioma, a type of brain cancer with a median survival of less than 18 months. Nationwide Children’s has exclusively licensed oncolytic virus C134 to Mustang.

  5. Axovant Announces Dosing Of First Patient In Clinical Program For AXO-AAV-GM1, A Novel Gene Therapy For GM1 Gangliosidosis 5/16/2019

    Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced dosing of the first patient in a clinical study of AXO-AAV-GM1 (also known as AAV9-GLB1), an investigational gene therapy for the treatment of GM1 gangliosidosis.

  6. DEINOVE Is Now Ready To Start Phase II Clinical Trial For Its Antibiotic Compound DNV3837 5/16/2019

    DEINOVE (Euronext Growth Paris: ALDEI), a French biotech company that uses a disruptive approach to develop innovative antibiotics and bio-based active ingredients for cosmetics and nutrition, announced that all the conditions are in place for the upcoming start of the Phase II trial testing the antibiotic candidate DNV3837 for the treatment of Clostridioides difficile infections.

  7. GENFIT: Positive 36-Month DSMB Recommendation For Continuation Of Phase 3 RESOLVE-IT Study Of Elafibranor In NASH 5/14/2019

    GENFIT (Euronext: GNFT – Nasdaq: GNFT) a late-stage biopharmaceutical company dedicated to the discovery and development of innovative therapeutic and diagnostic solutions in metabolic and liver related diseases, today announced that the Data Safety Monitoring Board (DSMB) issued a new positive recommendation for the continuation, without any modifications, of the RESOLVE-IT Phase 3 trial evaluating elafibranor in NASH.

  8. Myovant Sciences Announces Positive Phase 3 Results From LIBERTY 1 Study Evaluating Once Daily Relugolix Combination Therapy In Women With Uterine Fibroids 5/14/2019

    Myovant Sciences (NYSE: MYOV), a clinical-stage healthcare company focused on developing and commercializing innovative therapies for women’s health and prostate cancer, today announced that LIBERTY 1, the first of two Phase 3 studies of once daily relugolix combination therapy met its primary efficacy endpoint and six key secondary endpoints in women with uterine fibroids.

  9. resTORbio Announces Initiation Of Phase 3 Clinical Program Of RTB101 In Clinically Symptomatic Respiratory Illness 5/9/2019

    resTORbio, Inc. (Nasdaq: TORC), today announced the initiation of PROTECTOR 1, the first Phase 3 trial of RTB101, an orally administered, small molecule, potent inhibitor of target of rapamycin complex 1 (TORC1).

  10. Merus Announces First Patient Treated In Phase 1 Clinical Trial Of MCLA-145 For Advanced Solid Tumors 5/9/2019

    Merus N.V. (Nasdaq: MRUS), a clinical-stage immuno-oncology company developing Biclonics®, innovative full-length human bispecific antibody therapeutics, today announced that the first patient has been treated in its Phase 1 trial evaluating safety, tolerability, and preliminary efficacy of MCLA-145 for the treatment of patients with advanced solid tumors. MCLA-145 is a potential first-in-class PD-L1 x CD137 Biclonics® being developed in collaboration with Incyte (NASDAQ:INCY) for the treatment of solid tumors.