Lead company/partner	Compound	Indication	Comments
PHASE III RESULTS PUBLISHED
Shionogi & Co. Ltd./ Roche	Xofluza (baloxavir marboxil)	flu	CAPSTONE 1; NEJM, Sept. 6, 2018.
Bausch Health Companies Inc.	loteprednol etabonate ophthalmic gel	pain after cataract surgery	Study 842; Journal of Cataract and Refractive Surgery, Sept. 2018.
PHASE III INTERIM/TOP-LINE RESULTS
AstraZeneca plc	anifrolumab	systemic lupus erythematosus	TULIP 1: missed primary endpoint.
EyeGate Pharmaceuticals Inc.	EGP-437	anterior uveitis	Not non-inferior to standard of care.
Histogenics Corp.	NeoCort cell therapy	knee cartilage injury	Some clinically meaningful outcomes.
Recro Pharma	N1539 (meloxicam iv)	post-surgery pain	Pain relieved and well tolerated.
UPDATED PHASE III RESULTS
bluebird bio	Lenti-D gene therapy	adrenoleukodystrophy	Starbeam; reduced disease progression.
Biohaven Pharma Holding Co. Ltd.	rimegepant	migraine	Well tolerated, rapid and sustained pain relief.
PHASE III INITIATED
Biogen Inc.	Cirara (iv glibencla- mide)	ischemic stroke, severe	CHARM; for treatment and prevention.
argenx NV	efgartigimod	myasthenia gravis	In patients with generalized disease.
NeuroRx Inc.	NRX-101 (D-cyclo-serine/lurasidone)	bipolar disorder with acute suicidal ideation and behavior	SBD-ASIB; a fixed dose oral combination.
PHASE III ANNOUNCED
AstraZeneca	AZD3759	non-small cell lung cancer (NSCLC)	Asia (EGFR Mut+); as first-line therapy.
ProQR Therapeutics NV	QR-110	Leber's congenital amaurosis	ILLUMINATE; a double blind 12-month study.
Xenon Pharmaceuticals Inc.	XEN496 (ezogabine)	KCNQ2 epileptic encephalopathy	A rare pediatric disorder.
PHASE II INTERIM/TOP-LINE RESULTS
Gilead Sciences Inc./ Galapagos NV	filgotinib	ankylosing spondylitis	TORTUGA; met primary endpoint.
BioCryst Pharmaceuticals Inc.	BCX7353	hereditary angioedema	ZENITH-1; signs of efficacy, well tolerated.
Tesaro Inc.	Zejula (niraparib) plus TSR-042	NSCLC, first-line	JASPER; objective responses seen.
Synlogic Inc.	SYNB1618	phenylketonuria	Well tolerated, signs of efficacy.
NeuroRx Inc.	NRX-101 (D-cyclo-serine/lurasidone)	bipolar disorder with acute suicidal ideation and behavior	STABIL-B; encouraging efficacy data, well tolerated.
Abivax SA	ABX464, oral	ulcerative colitis	101EU; effective and well tolerated.
iXBiopharma Ltd.	Wafermine (ketamine) sublingual wafer	acute pain post bunionectomy	Pain relief observed, well tolerated.
Checkpoint Therapeutics Inc.	CK-101	NSCLC	Encouraging safety and efficacy data.
Arrowhead Pharmaceuticals Inc.	ARO-HBV	hepatitis B	Signs of efficacy, well tolerated.
UPDATED PHASE II RESULTS
Sangamo Therapeutics Inc.	SB-913	Hunter syndrome (MPS II)	CHAMPIONS; reduced glycosaminoglycans.
ProQR Therapeutics NV	QR-110	Leber's congenital amaurosis	Improved vision, well tolerated.
Aeglea BioTherapeutics Inc.	pegzilarginase	arginase 1 deficiency	Reduced plasma arginine, well tolerated.
Altimmune Inc.	NasoVAX, intranasal	pandemic flu	mucosal immune responses seen.
Spectrum Pharmaceuticals Inc.	poziotinib	NSCLC	Encouraging safety and efficacy data.
BeyondSpring Pharmaceuticals Inc.	plinabulin	prevention of docetaxel-induced thrombocytopenia	Protective-1; superior efficacy to standard of care.
PHASE II INITIATED
Inovio Pharmaceuticals Inc.	INO-4700	MERS infection prevention	Taking place in South Korea.
Thrombolytic Science LLC	HisproUK (tPA and prourokinase)	ischemic stroke	A sequential dual treatment.
Verastem Inc.	duvelisib plus venetoclax	chronic lymphocytic leukemia	At the Dana Farber Cancer Institute.
Synthetic Biologics Inc.	SYN-010 (lovastatin lactone)	irritable bowel syndrome	A modified release reformulation.

Pipeline Watch: Phase III Starts in Ischemic Stroke, Myasthenia Gravis, Bipolar Depression

More from Pipeline Watch

More from R&D