Pipeline

April 9, 2018

Company	Drug/Device	Medical Condition	Status
Wize Pharma, Inc.	LO2A	moderate to severe Conjunctivochalasis (CCH)	Phase II trials initiated enrolling 62 subjects
Synlogic	SYNB1020	hyperammonemia in patients with cirrhosis	Phase Ib/IIa trials initiated
Amylyx Pharmaceuticals, Inc.	AMX0035	Amyotrophic lateral sclerosis (ALS)	Phase II open-label extension initiated enrolling 132 patients in the U.S.
Rigel Pharmaceuticals	fostamatinib	IgA nephropathy (IgAN)	Phase II topline results announced evaluating subjects in the U.S., Asia and Europe
Kazia Therapeutics Limited	GDC-0084	glioblastoma multiforme (GBM)	Phase II trials initiated
Eli Lilly and Company	Cyramza (ramucirumab)	hepatocellular carcinoma (HCC)	Phase III positive topline results announced evaluating 292 patients across 20 countries in North America, Asia, Europe and Latin America
Allergan, Inc. and Gedeon Richter Ltd.	cariprazine	bipolar I depression	Phase III positive topline results announced evaluating 493 subjects
GenSight Biologics	GS010 (rAAV2/2-ND4)	Leber Hereditary Optic Neuropathy (LHON)	Phase III topline results announced evaluating 37 subjects
Wize Pharma, Inc.	LO2A	dry-eye syndrome (DES) in patients with Sjögren's syndrome	Phase IV trials initiated enrolling 60 subjects
TARIS Biomedical	TAR-200 (GemRIS)	organ-confined or locally-advanced Muscle Invasive Bladder Cancer	Fast Track Designation granted
Tetra Discovery Partners	BPN14770	Fragile X Syndrome	Orphan Drug Designation granted
PharmAbcine Inc.	TTAC-0001	Glioblastoma Multiforme (GBM)	Orphan Drug Designation granted
Scholar Rock	SRK-015	spinal muscular atrophy (SMA)	Orphan Drug Designation granted
AstraZeneca	moxetumomab pasudotox	hairy cell leukemia (HCL)	Priority Review granted
Dr. Reddy’s Laboratories Ltd.	DFN-02	migraine	NDA filed with the FDA
AstraZeneca Pharmaceuticals LP	BYDUREON	type 2 diabetes with inadequate glycemic control	FDA approval granted
Amgen	Blincyto (blinatumomab)	B-cell precursor acute lymphoblastic leukemia (ALL)	FDA approval granted