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Drug pipeline 1Q20

The US Food and Drug Administration (FDA) kept up its pace with nine approvals. Notable firsts include Tazverik for epithelial sarcoma (the first small-molecule inhibitor of the histone methyltransferase EZH2) and Tepezza for thyroid eye disease (the first approved monoclonal antibody (mAb) targeting insulin-like growth factor receptor; IGF-1R). Mesoblast’s mesenchymal stem cell therapy (remestemcel-L) for pediatric graft-versus-host disease and GlaxoSmithKline’s first-in-class small molecule HIV attachment inhibitor targeting glycoprotein 120 (gp120) come before the FDA next quarter. Clinical trial results of two oligonucleotide drugs showed a cholesterol-lowering antisense oligonucleotide drug and a small-interfering RNA (siRNA) drug gave improvements over statin alone. The COVID-19 pandemic is affecting the drug pipeline; Bristol-Myers Squibb’s Zeposia is among several drug launches delayed.

Historic US regulatory approvals by drug class

Biologics approvals held steady in 2019.

*New molecular entity (NME) class includes mainly small-molecule drugs, but also steroid, synthetic peptide, and mixed compounds, excluding new formulations. **Partial year to March 31.Source: BioMedTracker, a service of Sagient Research (

Upcoming catalysts (3Q20)

Drug/company Indication Drug information
Fostemsavir tromethamine/GlaxoSmithKline HIV/AIDS 8/5/2020 FDA PDUFA date for this small-molecule HIV attachment inhibitor that interferes with viral gp120 protein binding to CD4+ cells
Lisocabtagene maraleucel/Bristol-Myers Squibb Diffuse large B-cell lymphoma, non-Hodgkin's lymphoma 8/17/2020 FDA PDUFA date for these autologous chimeric antigen receptor (CAR) modified T cells with 1:1 ratio of CD4+ and CD8+ cells
Margetuximab/MacroGenics Breast cancer 8/19/2020 FDA PDUFA date for this chimeric Fc modified IgG1 mAb targeting epidermal growth factor receptor 2 (EGFR-2)
Valoctocogene roxaparvovec/BioMarin Hemophilia A 8/12/2020 FDA PDUFA date for adeno-associated virus 5 gene therapy vector containing a B-domain-deleted factor VIII gene with a liver-specific promoter
Inebilizumab/Viela Bio Neuromyelitis optica (Devic’s syndrome 6/13/2020 FDA PDUFA date for this humanized IgG1-κ mAb against CD38
Satralizumab/Roche Neuromyelitis optica (Devic’s syndrome) 5/31/2020 FDA PDUFA date for this humanized IgG2 mAb against interleukin-6 receptor
Remestemcel-L/Mesoblast Graft-versus-host disease 9/30/2020 FDA PDUFA date for mesenchymal stem cells isolated from the bone marrow of autologous donors
  1. Source: BioMedTracker, a service of Sagient Research (

Notable clinical trial results

Drug/company Indication Drug information
Inclisiran/The Medicines Company Dyslipidemia/hypercholesterolemia 3/18/2020 In three phase 3 trials of this siRNA targeting proprotein convertase subtilisin–kexin type 9 mRNA, twice-yearly dosing led to durable low-density lipoprotein C reductions compared with placebo (N. Engl. J. Med., 2020)
Pelacarsen/Novartis (Ionis) Cardiovascular disease 1/1/2020 In a randomized, double-blind, placebo-controlled, dose-ranging phase 2 trial of patients with established heart disease, this N-acetylgalactosamine-conjugated 2′-O-methoxyethyl phosphorothioate antisense oligonucleotide designed to suppress lipoprotein(a) mRNA resulted in dose-dependent decreases in lipoprotein levels (80% at highest dose compared with 6% in controls) (N. Engl. J. Med. 382, 244–255, 2020)
Nemolizumab/Galderma Pruritus 2/20/2020 In a 12-week, randomized, double-blind, phase 2 trial of humanized IgG2 mAb directed against interleukin-31 receptor-α, which blocks signaling from IL-31, patients on drug had 53% reduction in severity compared with 20% with placebo (N. Engl. J. Med. 382, 706–716, 2020)
PTI-125/Cassava Sciences Alzheimer’s disease 2/7/2020 In a phase 2a open-label trial of this small-molecule binder of filamin A (a scaffolding protein required for the toxic signaling of β-amyloid), multiple biomarkers showed improvement after 28 days (J. Prev. Alzheimers Dis. 2020)
RV521/ReViral Respiratory syncytial virus (RSV) 1/27/2020 In a randomized trial, this small-molecule viral fusion inhibitor of RSV F protein reduced viral load and disease severity in healthy adults challenged with RSV (Antimicrob. Agents Chemother. 64, e01884-19, 2020)
  1. Source: BioMedTracker, a service of Sagient Research (

Notable drug approvals

Drug/company Indication Drug information
Tazverik (tazemetostat)/Royalty Pharma, Epizyme Epithelial sarcoma 1/23/2020 FDA granted accelerated approval for this first-in-class small molecule inhibitor of methyltransferase EZH2, which catalyzes the trimethylation of Lys27 on histone H3
Ayvakit (avapritinib)/Blueprint Medicines Gastrointestinal stromal tumor 1/9/2020 FDA approved this selective inhibitor of KIT and platelet-derived growth factor-α (PDGFRα) for patients harboring PDGFRA exon 18 mutations, including PDGFRA D842V mutation.
Tepezza (teprotumumab)/Horizon Therapeutics Thyroid eye disease 1/21/2020 FDA approved this human IgG1 mAb that targets IGF-1R
Nexletol (bempedoic acid)/Esperion Therapeutics Dyslipidemia/hypercholesterolemia 2/21/2020 FDA approved this small molecule inhibitor of ATP citrate lyase, upstream from statins
Nurtec ODT (rimegepant)/Biohaven Pharmaceuticals Migraine and other headaches 2/27/2020 FDA approved this small-molecule, orally dissolving calcitonin gene-related peptide (CGRP) receptor antagonist
Isturisa (osilodrostat)/Recordati Cushing’s syndrome 3/6/2020 FDA approved this small-molecule inhibitor of aldosterone synthase
Sarclisa (isatuximab)/Sanofi Multiple myeloma 3/2/2020 FDA approved this humanized IgG1 mAb against CD38
Zeposia (ozanimod)/Bristol-Myers Squibb Multiple sclerosis 3/25/2020 FDA approved this selective sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator
  1. Source: BioMedTracker, a service of Sagient Research (

Notable regulatory setbacks

Drug/company Indication Drug information
AXO-AAV-GM2/Axovant GM2 gangliosidoses (Tay-Sachs disease, Sandhoff disease, AB variant) 1/13/2020 FDA put a clinical hold on this adeno-associated virus (AAV) serotype 2 gene therapy that delivers β-hexosaminidase α and β subunit genes (HEXA and HEXB) via two coadministered AAVrh8 vectors delivered directly to the central nervous system due to CMC and device-related issues.
PledOx/Pled Pharma Chemotherapy-induced peripheral neuropathy 1/23/2020 FDA put a clinical hold on phase 3 clinical trial of this manganese superoxide dismutase mimic due to a few adverse effects, while trials continue in Europe and Asia
LB-0001/LogicBio Therapeutics Organic acidemias 2/10/2020 FDA put a clinical hold on this recombinant adeno-associated viral vector with a human methylmalonyl-CoA mutase gene for unspecified clinical and non-clinical reasons
Concizumab/Novo Nordisk Hemophilia A and B 3/16/2020 FDA put a clinical hold on a two phase 3 and one phase 2 trial of this humanized IgG4 mAb specific for the K2 domain of tissue factor pathway 1 (TFP1) inhibitor owing to three non-fatal thombic events — the fifth such TFP1 inhibitor to fail.
  1. Source: BioMedTracker, a service of Sagient Research (

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DeFrancesco, L. Drug pipeline 1Q20. Nat Biotechnol 38, 519–520 (2020).

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