Pipeline

January 19, 2020

Company	Drug/Device	Medical Condition	Status
Alucent Biomedical	Natural Vascular Scaffolding technology	peripheral artery disease	phase 1 trial enrolling up to 15 patients across five U.S. research sites
Clover Biopharmaceuticals	SCB-313	malignant pleural effusions	phase 1 trial dosing first patient in China
Tmunity	CART-TnMUC1-01	TnMUC1-positive advanced cancers	phase 1 trial dosing first patient
Cellectis	UCART123	relapsed/refractory acute myeloid leukemia (AML)	phase 1 trial dosing first patient
BioTheryX	BTX-A51	relapsed/refractory AML	phase 1 initiation of patient dosing
Innovation Pharmaceuticals	Brilacidin	ulcerative colitis	phase 1 dosing of first cohort of 3 patients
Retrotope	RT001	Friedreich’s ataxia	phase 2/3 trial dosing of first patient
Zymeworks	ZW25	locally advanced and/or metastatic HER2-positive, HR-positive breast cancer	phase 2 trial initiated, enrolling up to 76 patients in the U.S. and Canada
Hutchison China MediTech	surufatinib	advanced solid tumors	phase 2 trial initiated in China
Akero Therapeutics	AKR-001	non-alcoholic steatohepatitis	ongoing phase 2a trial expanded to include patients with compensated cirrhosis
Tolero Pharmaceuticals	Alvocidib	AML	phase 2 trial first patient dosed
Tyme Technologies	SM-88 (racemetyrosine)	high-risk sarcomas	phase 2 trial first patient dosed
Neuroptika	NRO-1	dry eye disease	phase 2 trial first patient enrolled of 120 patients
ContraFect	exebacase	Staph aureus bacteremia	phase 3 trial first patient dosed, 350 patients
Akcea Therapeutics Ionis Pharmaceuticals	AKCEA-TTR-LRx	transthyretin-mediated amyloid cardiomyopathy	phase 3 trial initiated
Abeona Therapeutics	EB-101	recessive dystrophic epidermolysis bullosa	phase 3 trial initiated, assessing up to 15 patients
Cerevel Therapeutics	tavapadon	Parkinson’s disease	Phase 3 trial initiated, enrolling 1,200 patients
Novavax	NanoFlu	influenza in older adults	Fast-Track designation granted by the FDA
Arena Pharmaceuticals	APD418	decompensated heart failure	Fast-Track designation granted by the FDA
Presage Biosciences	CIVO Technology Platform	cancer	IND approved by the FDA
Lannett Company	cocaine hydrochloride nasal solution	local anesthetic	NDA approved by the FDA
Novo Nordisk	Ozempic (semaglutide)	cardiovascular events in diabetes patients	approved by the FDA for additional indication
Neurelis	Valtoco (diazepam) nasal spray	intermittent, stereotypic episodes of frequent seizure activity	approved by the FDA