Company Product Description Indication Status
4D Molecular Therapeutics Inc., of Emeryville, Calif 4D-310 Gene therapy expressing GLA Fabry disease FDA granted fast track designation
Akeso Biopharma Inc., of Los Angeles AK-104 Anti-PD-1/CTLA4 bispecific antibody Recurrent or metastatic squamous cervical cancer after platinum-based chemotherapy FDA granted fast track designation
Antengene Corp., of Shanghai ATG-017 Inhibitor of extracellular signal-regulated kinase 1 and 2 Advanced solid tumors and hematological malignancies Australian Therapeutic Goods Administration authorized the start of a phase I study
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Vosoritide Analogue of C-type natriuretic peptide Achondroplasia EMA validated the MAA
Enzychem Lifesciences Corp., of Englewood, N.J. EC-18 CCL26 gene inhibitor; TLR modulator Acute respiratory disease syndrome due to COVID-19 pneumonia FDA accepted the IND for a 60-patient phase II study with a primary endpoint of patients alive and free of respiratory failure through day 28
Genentech, of South San Francisco, a unit of Roche Holding AG Xolair (omalizumab) Monoclonal antibody targeting immunoglobulin E Moderate to severe persistent asthma and chronic idiopathic urticaria FDA accepted the supplementary BLA for prefilled syringes across all approved U.S. indications; decision expected by the first quarter of 2021
Hoth Therapeutics Inc., of New York Biolexa Antimicrobial Mild to moderate atopic dermatitis Submitted study for ethics approval from the Bellberry Human Research Ethics Committee in Australia
Micurx Pharmaceuticals Inc., of Foster City, Calif. MRX-8 Polymyxin antimicrobial Infections caused by multidrug-resistant gram-negative pathogens FDA cleared the IND for a phase I study
NS Pharma Inc., of Paramus, N.J., a unit of Nippon Shinyaku Co. Ltd. Viltepso (viltolarsen) DMD gene modulator Duchenne muscular dystrophy amenable to exon 53 skipping FDA granted an accelerated approval for the drug
Passage Bio Inc., of Philadelphia PBGM-01 Gene therapy expressing b-gal GM1 gangliosidosis Submitted IND to FDA for a phase I/II study; agency placed the application on clinical hold pending additional risk assessments of the intra-cisterna magna injection delivery method
Scholar Rock Holding Corp., of Cambridge, Mass. SRK-015 Inhibits activation of myostatin Spinal muscular atrophy FDA granted rare pediatric disease designation
TG Therapeutics Inc., of New York Umbralisib Inhibitor of PI3K-delta and CK1-epsilon Marginal zone lymphoma after at least 1 prior anti-CD20-based regimen and follicular lymphoma after at least 2 prior systemic therapies FDA accepted the NDA for a priority review; PDUFA action date of Feb. 15, 2021

Notes

For more information about individual companies and/or products, see Cortellis.

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