Company Product Description Indication Status
Abbvie Inc., of North Chicago, and Calibr, the translational arm of The Scripps Research Institute CLBR-001 + SWI-019 CAR T-cell therapy B-cell malignancies FDA granted fast track designation
Aegle Therapeutics Corp., of Boston AGLE-102 Extracellular vesicle therapy Dystrophic epidermolysis bullosa FDA granted fast track status
Akari Therapeutics plc, of New York Nomacopan C5 complement inhibitor Bullous pemphigoid Completed scientific advice meeting with EMA in advance of starting a phase III pivotal study; agency agreed to company’s proposal for placebo-controlled trial with primary endpoint of disease remission on minimal corticosteroids
Baylx Inc., of Irvine, Calif. BX-U001  Non-frozen human umbilical cord tissue mesenchymal stem cells COVID-19 FDA cleared the IND for a phase I/IIa trial in about 30 patients with moderate to severe acute respiratory distress syndrome
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. ION-373 Antisense therapeutic designed to stop mutated gene from producing excess GFAP Alexander disease FDA granted orphan designation
Irlab Therapeutics AB, of Stockholm Mesdopetam (IRL-790) Dopamine D3 receptor antagonist Parkinson’s disease FDA accepted IND for inclusion of U.S. patients in planned phase IIb/III study
Kadmon Holdings Inc., of New York Belumosudil (KD-025) ROCK2 inhibitor Chronic graft-vs.-host disease Submitted NDA to FDA; application reviewed under Real-Time Oncology Review program
Pfizer Inc., of New York PF-06939926 Gene therapy Duchenne muscular dystrophy FDA granted fast track designation
Provention Bio Inc., of Red Bank, N.J. Teplizumab (PRV-031) Anti-CD3 monoclonal antibody Delay or prevention of type 1 diabetes Submitted clinical module of BLA to FDA for use in at-risk individuals; final modules, including CMC module, expected to be submitted in the fourth quarter of 2020
Solid Biosciences Inc., of Cambridge, Mass. SGT-001 DMD gene stimulator Duchenne muscular dystrophy FDA lifted clinical hold on Ignite DMD phase I/II trial; dosing expected to resume in first quarter of 2021
Teneobio Inc., of Newark, Calif. TNB-486 Bispecific T-cell engaging antibody B-cell non-Hodgkin lymphoma FDA cleared the IND for phase I studies
Zosano Pharma Corp., of Fremont, Calif. Qtrypta (zolmitriptan transdermal microneedle system) 5-HT 1b receptor agonist; 5-HT 1d receptor agonist Acute migraine Received discipline review letter (DRL) from FDA in connection with 505(b)(2) NDA, describing 2 concerns with respect to clinical pharmacology section, specifically high plasma concentrations of zolmitriptan observed in 5 study subjects from 2 pharmacokinetic studies and differences in zolmitriptan exposures observed between subjects receiving different lots in clinical trials; approval by the PDUFA date of Oct. 20, 2020, is not expected given the DRL


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