Company Product Description Indication Status
American Gene Technologies International Inc., of Rockville, Md. AGT103-T Lentiviral vector-based gene therapy HIV infection FDA cleared IND for phase I study; enrollment expected to begin in September 2020, with initial data by year-end 2020
Arvelle Therapeutics GmbH, of Zug, Switzerland Cenobamate Sodium channel inhibitor Epilepsy U.K. MHRA granted promising innovative medicine designation to treat drug-resistant focal-onset seizures in adults
Atyr Pharma Inc., of San Diego, and Kyorin Pharmaceutical Co. Ltd., of Tokyo ATYR-1923 (KRP-R120) Fusion protein comprising immuno-modulatory domain of histidyl tRNA synthetase fused to FC region of a human antibody Interstitial lung disease Kyorin received Japanese approval for start of phase I study in healthy male volunteers in Japan
Clearside Biomedical Inc., of Alpharetta, Ga. CLS-AX (axitinib injectable suspension) Tyrosine kinase inhibitor; pan-VEGF inhibitor Neovascular age-related macular degeneration FDA cleared IND for phase I/IIa study, expected to begin by year-end 2020
Equillium Inc., of La Jolla, Calif. Itolizumab (EQ-001) Monoclonal antibody targeting CD6-ALCAM pathway COVID-19 Submitted request to FDA for pre-IND meeting to review proposal for initiating clinical trial in hospitalized patients with COVID-19
Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. Pedmark (sodium thiosulfate) Otoprotectant Ototoxicity FDA issued CRL for NDA covering intravenous administration to prevent ototoxicity associated with cisplatin chemotherapy in patients ≥1 month to 18 years of age with localized, non-metastatic solid tumors; company plans to request type A meeting
Mersana Therapeutics Inc., of Cambridge, Mass. XMT-1536 Sodium phosphate cotransporter 2B inhibitor Ovarian cancer FDA granted fast track designation to treat platinum-resistant high-grade serous disease
Mesoblast Ltd., of Melbourne, Australia Ryoncil (remestemcel-L) Mesenchymal stem cell therapy Graft-vs.-host disease FDA Oncologic Drugs Advisory Committee meeting scheduled for Aug. 13, 2020, will include non-voting morning session to discuss characterization and quality attributes of study drug and afternoon session to discuss phase III results and supporting data in BLA submission, with vote on whether available data support efficacy in pediatric patients with steroid-refractory acute disease
Neurogene Inc., of New York Adeno-associated viral vector-based gene therapy CLN5 gene stimulator Batten disease FDA granted orphan drug designation to treat CLN7 form of disease
Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, unit of Chiesi Farmaceutici SpA, of Parma, Italy Pegunigalsidase alfa Alpha-galactosidase stimulator Fabry disease FDA accepted BLA, granting priority review and setting Jan. 27, 2021, PDUFA date; no advisory committee meeting expected

Notes

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

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