Pipeline

July 1, 2019

Company	Drug/Device	Medical Condition	Status
Cyxone	T20K	multiple sclerosis (MS)	Phase I trial initiated enrolling healthy male volunteers in the Netherlands
NantKwest Inc.	PD-L1 t-haNK	solid tumors	Phase I trial initiated
Pepscan	clinical neoantigen peptides	cancer	Phase I trial initiated
I-Mab Biopharma	TJC4	advanced malignant tumors	Phase I trial initiated
Asieris Pharmaceuticals	APL-1202 in combination with BCG (Bacillus Calmette Guerin)	non-muscle invasive bladder cancer (NMIBC)	Phase Ib trial initiated enrolling subjects in China
Precigen, Inc.	PRGN-3006 UltraCAR-T	relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS)	Phase I/Ib trial initiated
Syntrix Pharmaceuticals	SX-682 in combination with KEYTRUDA (pembrolizumab)	metastatic melanoma	Phase I/II trial initiated enrolling 77 subjects at Massachusetts General Hospital and Dana Farber Cancer Institute
Minovia Therapeutics	Mitochondrial Augmentation Therapy (MAT)	Pearson syndrome	Phase I/II trial initiated enrolling seven subjects
Xeris Pharmaceuticals, Inc.	developmental ready-to-use glucagon	Post-bariatric hypoglycemia (PBH) following bariatric surgery	Phase II trial initiated enrolling 12 subjects with PBH
iLiAD Biotechnologies	BPZE1	pertussis	Phase IIb trial initiated
X4 Pharmaceuticals, Inc.	mavorixafor (X4P-001)	WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis)	Phase III trial initiated enrolling 28 subjects in 20 countries
XW Laboratories Inc.	XWL-008	narcolepsy	Orphan Drug designation granted by the FDA
Aridis Pharmaceuticals, Inc.	AR-501	lung infection associated with cystic fibrosis	Orphan Drug designation granted by the FDA
LifeMax Laboratories, Inc.	LM-030	Netherton Syndrome	Orphan Drug designation granted by the FDA
Boehringer-Ingelheim Eli Lilly and Company	empagliflozin	chronic heart failure	Fast Track designation granted by the FDA
Arrowhead Pharmaceuticals Inc.	ARO-AAT	alpha-1 liver disease	Fast Track designation granted by the FDA
ANI Pharmaceuticals, Inc.	Vancomycin Hydrochloride for Oral Solution USP, 250 mg/5 ml	enterocolitis caused by Staphylococcus aureus (including methicillin-resistant strains) and antibiotic-associated pseudomembranous colitis caused by C. difficile	Approval granted by the FDA
Regeneron Pharmaceuticals, Inc. Sanofi	Dupixent (dupilumab)	chronic rhinosinusitis with nasal polyposis (CRSwNP)	Approval granted by the FDA
Dignitana AB	DigniCap Delta	chemotherapy-induced hair loss	Approval granted by the FDA