Pipeline

April 22, 2019

Company	Drug/Device	Medical Condition	Status
Vaxart, Inc.	bivalent norovirus vaccine	norovirus	Phase Ib trial initiated
Zymeworks, Inc.	ZW25	HER2-positive metastatic gastric, gastroesophageal junction, and esophageal adenocarcinomas	Phase II trial initiated
Cortexyme, Inc.	COR388	mild to moderate Alzheimer’s disease (AD)	Phase II/III trial initiated enrolling more than 570 subjects at more than 90 sites across the U.S. and Europe
CStone Pharmaceuticals	CS1001 in combination with oxaliplatin and capecitabine (XELOX) chemotherapy	unresectable, locally advanced, or metastatic gastric adenocarcinoma (GC) and gastro-esophageal junction (GEJ) adenocarcinoma	Phase III trial initiated in China
Alnylam Pharmaceuticals, Inc.	lumasiran	primary hyperoxaluria type 1 (PH1)	Phase III trial initiated enrolling 8 subjects aged 6 years and younger
Ultragenyx Pharmaceutical Inc.	UX007	long-chain fatty acid oxidation disorders (LC-FAOD)	Rare Pediatric Disease designation granted by the FDA
Elucent Medical	EnVisio Surgical Navigation System	breast tumors	510(k) clearance granted by the FDA
Aprea Therapeutics	APR-246	Myelodysplastic Syndromes (MDS)	Fast Track designation granted by the FDA
Ultragenyx Pharmaceutical Inc.	UX007	long-chain fatty acid oxidation disorders (LC-FAOD)	Fast Track designation granted by the FDA
Janssen Pharmaceuticals	Balversa (erdafitinib)	locally advanced or metastatic bladder cancer	Breakthrough Therapy designation granted by the FDA
Viracta Therapeutics, Inc.	nanatinostat in combination with valganciclovir	post-transplant lymphoproliferative disorder (PTLD), plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma	Orphan Drug designation granted by the FDA
Aprea Therapeutics	APR-246	Myelodysplastic Syndromes (MDS)	Orphan Drug designation granted by the FDA
Gelesis	PLENITY (Gelesis100)	weight management	Clearance to market granted by the FDA
Circassia Pharmaceuticals, Inc.	DUAKLIR PRESSAIR (aclidinium bromide and formoterol fumarate)	chronic obstructive pulmonary disease (COPD)	Approval granted by the FDA
Intact Vascular, Inc.	Tack Endovascular System (6F)	peripheral arterial disease (PAD)	Approval granted by the FDA