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

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