Pipeline

March 19, 2018

Company	Drug/Device	Medical Condition	Status
XW Laboratories Inc.	XW10172	Narcolepsy	Phase I trials initiated
Cadent Therapeutics	CAD-1883	Neurological (Essential Tremor) and psychiatric disease (Spinocerebellar ataxia)	Phase I trials initiated
Arrowhead Pharmaceuticals, Inc.	ARO-AAT	Alpha-1 liver disease	Phase I trials initiated
Voyager Therapeutics	VY-AADC	Advanced Parkinson’s disease	Phase Ib trials initiated
Protalix Biotherapeutics, Inc.	OPRX-106	Ulcerative colitis	Phase II trials initiated enrolling 24 subjects
Merrimack Pharmaceuticals, Inc.	MM-121	Lung cancer	Phase II trials initiated expanding from 80 to 100 subjects
Biogen	SPINRAZA (nusinersen)	Spinal muscular atrophy (SMA)	Phase II trials initiated enrolling 25 pre-symptomatic infants six weeks old or younger
MyoKardia	Mavacamten	Hypertrophic cardiomyopathy (HCM)	Phase II trials initiated
AM-Pharma	recombinant human Alkaline Phosphatase (recAP)	Acute Kidney Injury (AKI)	Phase II trials initiated enrolling 301 subjects with sepsis
Eleven Biotherapeutics, Inc.	Vicinium	Non-muscle invasive bladder cancer (NMIBC) previously treated with bacillus Calmette-Guérin (BCG)	Phase III trials initiated
AbbVie	Elagolix	Uterine fibroids	Phase III trials initiated
Merck	KEYTRUDA (pembrolizumab)	Advanced cervical cancer with disease progression on or after chemotherapy	Priority Review granted by the FDA
Cerapedics, Inc.	P-15L	Transforaminal lumbar interbody fusion (TLIF) surgery for degenerative disk disease	IDE Approval granted by the FDA
Proteostasis Therapeutics	PTI-428	Cystic fibrosis	Breakthrough Designation granted by the FDA