Pipeline

October 5, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
Covistat/Ensysce Biosciences	oral nafamostat	acute respiratory failure due to COVID-19	initiation of phase 1 trial
Covaxx	UB-612	COVID-19 vaccine	first patient dosed in phase 1 trial
Stemedica Cell Technologies	intravenous allogeneic mesenchymal stem cells	patients with moderate to severe lung injury due to COVID-19	IND approved by the FDA for phase 2 trial
Windtree Therapeutics	lyo lucinactant	COVID-19 associated lung injury and acute respiratory distress syndrome	IND approved by the FDA for phase 2 trial
FSD Pharma	FSD201 (ultramicronized palmitoylethanolamide)	hospitalized patients with COVID-19	initiation of phase 2 trial
Novavax	NVX-CoV2373 (COVID-19 vaccine)	COVID-19	initiation of phase 3 trial
Organicell Regenerative Medicine	Zofin acellular therapy	patients with SARS-CoV-2 who have mild to moderate COVID-19, or who are judged by a healthcare provider to be at high risk of progression to moderate disease	expanded access protocol granted by the FDA
ADial Pharmaceuticals	Assure/FaStep COVID-19 IgG/IgM Rapid Test Device	antibody point-of-care test for COVID-19	Emergency Use Authorization (EUA) granted by the FDA
Cepheid	Xpert Xpress SARS-CoV-2/Flu/RSV molecular diagnostic test	qualitative detection of the viruses causing COVID-19, Flu A, Flu B, and RSV infections from a single patient sample	EUA granted by the FDA
Hologic	Panther Fusion SARS-CoV-2 assay	testing of individuals without symptoms or other reasons to suspect COVID-19 infection	EUA granted by the FDA
Quotient	MosaiQ COVID-19 antibody test	detection of COVID-19 antibodies	EUA granted by the FDA
Other Trials and Actions
Aerie Pharmaceuticals	AR-15512 (TRPM8 agonist) eye drop	dry eye disease	IND approved by the FDA
IRLAB	mesdopetam (IRL790)	levodopa-induced dyskinesias in Parkinson's disease	IND approved by the FDA
Inversago Pharma	INV-101	Prader-Willi syndrome and non-alcoholic steatohepatitis	initiation of phase 1 trial
Kuur Therapeutics	KUR-502	relapsed/refractory CD19-positive malignancies	first patients dosed in phase 1 trial
Prelude Therapeutics	PRT1419	relapsed/refractory hematologic malignancies	first patients dosed in phase 1 trial
Zucara Therapeutics	ZT-01	type 1 diabetes	first patient dosed in phase 1 trial
BioMarin	BMN 307	phenylketonuria	first patient dosed in phase 1/2 trial
Codiak Biosciences	exoSTING	advanced metastatic or recurrent, injectable solid tumors	first patients dosed in phase 1/2 trial
Aptinyx	NYX-2925	fibromyalgia	patient recruitment recommenced in phase 2 trial
Calithera Biosciences	telaglenastat (CB-839)	nonsmall-cell lung cancer	first patient dosed in phase 2 trial
Concert Pharmaceuticals	CTP-692	schizophrenia	patient enrollment complete in phase 2 trial
Ocular Therapeutics	OTX-CSI (cyclosporine intracanalicular insert)	dry eye disease	first patients dosed in phase 2 trial
Aurinia	voclosporin ophthalmic solution	dry eye syndrome	last patient study visit in phase 2/3 trial
CiVi Biopharma/Eicos Sciences	CIVI030 (intravenous iloprost)	systemic sclerosis	patient recruitment resumed in phase 3 trial
Eisai	lorcaserin	Dravet syndrome	initiation of phase 3 trial
Harbour BioMed	batoclimab (HBM 9161)	myasthenia gravis (MG) and adult immune thrombocytopenia	first patient dosed in phase 3 trials
Infinity	eganelisib (IPI-549) in combination with a checkpoint inhibitor and chemotherapy	patients with inoperable locally advanced or metastatic triple-negative breast cancer, in the first-line setting	Fast-Track designation granted by the FDA
AbbVie	elezanumab (ABT-555)	spinal cord injury	Orphan Drug and Fast-Track designations granted by the FDA
Neurophth Therapeutics	NR082 (rAAV2-ND4, NFS-0)	Leber's Hereditary Optic Neuropathy associated with ND4 mutation	Orphan Drug designation granted by the FDA
CSL Behring	Haegarda (C1 Esterase Inhibitor Subcutaneous [human]	routine prophylaxis to prevent hereditary angioedema attacks in patients 6 years of age and older	approved by the FDA for expanded indication
Eton Pharmaceuticals	Alkindi Sprinkle (hydrocortisone) oral granules	replacement therapy for adrenocortical insufficiency in children under 17 years of age	approved by the FDA
GlaxoSmithKline	Nucala (mepolizumab)	hypereosinophilic syndrome (HES) without an identifiable non-blood-related cause of the disease in adults and children 12 years of age and older	approved by the FDA for expanded indication
Janssen	Simponi Aria (golimumab)	active polyarticular juvenile idiopathic arthritis and active psoriatic arthritis in patients 2 years of age and older	approved by the FDA
Masimo	Rad-G Pulse Oximeter	pulse oximetry and respiration rate monitoring	approved by the FDA
Pfizer	Xeljanz (tofacitinib)	children and adolescents 2 years and older with active polyarticular-course juvenile idiopathic arthritis	approved by the FDA for expanded indication
Shionogi	Fetroja (cefiderocol)	hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia	approved by the FDA for expanded indication
Vertex Pharmaceuticals	Kalydeco (ivacaftor)	children with cystic fibrosis ages four months to less than six months old who have at least one mutation in their CFTR gene	approved by the FDA for expanded indication