Company Product Description Indication Status
Ambrx Inc., of San Diego ARX-788 HER2 receptor-targeting antibody-drug conjugate Metastatic breast cancer FDA granted fast track designation as monotherapy to treat those who received 1 or more prior anti-HER2 regimens in metastatic setting
Antengene Corp. Ltd., of Shanghai ATG-010 (Xpovio, selinexor) Exportin 1 inhibitor Multiple myeloma; diffuse large B-cell lymphoma NDA submitted to South Korean Ministry of Food and Drug Safety for treatment, in combination with low-dose dexamethasone, of adults with relapsed/refractory (r/r) MM and as monotherapy for adults with r/r DLBCL
Arcturus Therapeutics Holdings Inc., of San Diego ARCT-021 Anti-SARS-CoV-2 mRNA vaccine COVID-19 infection FDA cleared IND for phase II study expected to enroll 600 participants to inform dose selection for phase III
Atara Biotherapeutics Inc., of South San Francisco Tab-Cel (tabelecleucel) Anti-Epstein-Barr virus allogeneic T-cell therapy  Lymphoproliferative disease BLA submission for treatment of EBV-positive post-transplant disease not yet initiated, pending procedural decision from FDA regarding presentation of historical non-pivotal data
Ayala Pharmaceuticals Inc., of Rehovot, Israel AL-102 Gamma secretase inhibitor  Desmoid tumors At end-of-phase I meeting, FDA authorized company to proceed with pivotal phase II/III study that may potentially serve as registrational trial; study in adults and adolescents, known as Ringside, expected to begin in first half of 2021
Bioniz Therapeutics Inc., of Irvine, Calif. BNZ-1 IL-2/IL-9/IL-15 inhibitor Cutaneous T-cell lymphoma Based on FDA feedback at end-of-phase II meeting, including guidance on trial design and NDA submission, pivotal phase III trial in people with relapsed/refractory disease expected to begin in second half of 2021
Bristol Myers Squibb Co., of New York Opdivo (nivolumab)  PD-1 inhibitor Gastric and gastroesophageal junction cancer; esophageal adenocarcinoma EMA validated type II variation MAA, initiating centralized review of agent, in combination with fluoropyrimidine- and platinum-based combination chemotherapy, in adults with first-line advanced or metastatic disease
Bristol Myers Squibb Co., of New York Opdivo (nivolumab)  PD-1 inhibitor Esophageal or gastroesophageal junction cancer EMA validated type II variation MAA, initiating centralized review as adjuvant treatment in adults with residual pathologic disease after neoadjuvant chemoradiotherapy and resection
Forge Biologics Inc., of Columbus, Ohio FBX-101 AAV gene therapy Krabbe disease FDA cleared IND for phase I/II Reskue trial, expected to begin screening for enrollment in January 2021
Inozyme Pharma Inc., of Boston INZ-701 Ectonucleotide pyrophosphatase-PDE-1 stimulator ENPP1 deficiency FDA cleared IND application and U.K. MHRA authorized CTA for phase I/II trial in affected adults, expected to initiate enrollment in first half of 2021
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-9001 (icosapent + docosapentaenoic acid + heneicosapentaenoic acid) Omega-3 fatty acid-based therapeutic Hypertriglyceridemia; lipid metabolism disorder FDA conditionally accepted Lypdiso as proposed brand name
Passage Bio Inc., of Philadelphia PBGM-01 GLB1 gene stimulator Infantile GM1 gangliosidosis FDA cleared IND application for phase I/II Imagine-1 study, expected to dose first participant in first quarter of 2021
Pfizer Inc., of New York, and Opko Health Inc., of Miami Somatrogon Growth hormone ligand Growth hormone deficiency FDA accepted BLA filing, setting PDUFA date in October 2021
Provention Bio Inc., of Red Bank, N.J. Teplizumab Anti-CD3 monoclonal antibody Type 1 diabetes FDA accepted BLA filing and granted priority review, assigning PDUFA date of July 2, 2021

Notes

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