Lead company/partner	Compound	Indication	Comments
PHASE III SUSPENDED
Aquinox Pharmaceuticals Inc.	rosiptor	interstitial cystitis/ bladder pain syndrome	LEADERSHIP 301; primary endpoint missed.
PHASE III RESULTS PUBLISHED
Astellas PharmaInc./Pfizer Inc.	Xtandi (enzalutamide)	castration-resistant prostate cancer	PROSPER; NEJM, June 28, 2018.
Hutchison MediPharma Ltd.	fruquintinib	colorectal cancer	FRESCO; JAMA, June 26, 2018.
PHASE III INTERIM/TOP-LINE RESULTS
Acceleron Pharma Inc./Celgene Corp.	luspatercept	myelodysplastic syndorome	MEDALIST; met primary and secondary endpoints.
Global Blood Therapeutics Inc.	voxelotor	sickle cell anemia	HOPE; primary endpoint achieved.
Cara Therapeutics Inc.	Korsuva (difelikefalin), intravenous	pain during abdominal surgery	Met primary endpoint, reducing pain and PONV.
Boehringer Ingelheim GMBH/ Eli Lilly & Co.	Jardiance (empagliflozin), with insulin	diabetes, type 1	EASE-2, -3; positive top-line data.
AstraZeneca PLC	Farxiga (dapagliflozin), with insulin	diabetes, type 1	DEPICT-2; improved glycemic control.
Roche	Tecentriq (atezolizumab) plus chemo	small cell lung cancer, extensive stage	IMpower133; improved overall survival and PFS.
Amgen Inc.	Repatha (evolocumab), monthly	diabetes, type 2 and dyslipidemia	BANTING; reduced cholesterol.
Xeris Pharmaceuticals Inc.	glucagon, rescue pen	hypoglycemia in diabetes, type 1	Positive data.
Eli Lilly & Co.	Taltz (ixekizumab)	radiographic axial spondyloarthritis	COAST-W; met primary and major secondary endpoints.
Novo Nordisk AS	semaglutide, oral	diabetes, type 2	PIONEER 3; reduced weight, HbA1c levels in long-term study.
Amgen Inc.	ABP710, biosimilar infliximab	rheumatoid arthritis	Non-inferior to Remicade.
AstraZeneca PLC/Merck & Co. Inc.	Lynparza (olaparib)	ovarian cancer, first-line maintenance	SOLO 1; PFS improved.
Ferring BV/ Merck & Co. Inc./WHO	Pabal (carbetocin), heat stable	acute hemorrhage after childbirth	CHAMPION; prevented excessive bleeding.
UPDATED PHASE III RESULTS
Biohaven Pharmaceuticals Holding Co. Ltd.	rimegepant	migraine	Study 301, 302; relieves pain, restores function.
Edge Therapeutics Inc.	EG-1962	subarachnoid hemorrhage	NEWTON 2; clinical benefits seen.
Eli Lilly & Co.	Emagality (galcanezumab-gnlm)	episodic cluster headache prevention	Positive clinical results.
AstraZeneca PLC	Farxiga (dapagliflozin), with insulin	diabetes, type 1	DEPICT-1; reduced glycemic variability.
Lexicon Pharmaceuticals Inc./ Sanofi	Zynquista (sotagliflozin) with insulin	diabetes, type 1	inTandem; reduced A1C levels.
Catabasis Pharmaceuticals Inc.	edasalonexent	Duchenne muscular dystrophy	MoveDMD; signs of disease slowing.
Novo Nordisk AS	semaglutide, oral	diabetes, type 2	PIONEER 2,4,7; updated clinical data.
Pfizer Inc.	Ibrance (palbociclib) with fulvestrant	HR+, HER2- metastatic breast cancer	PALOMA-3; positive trend in overall survival data.
Array Biopharma Inc.	encorafenib, binimetinib, cetuximab	metastatic colorectal cancer, BRAF-mutant	BEACON; improved efficacy outcomes.
Novo Nordisk AS	semaglutide, oral	diabetes, type 2	PIONEER 1; reduced blood sugar.
Sunovion Pharmaceuticals Inc.	APL-130277 (apomorphine) sublingual film	Parkinson's disease	CTH300; effective against off-episodes.
PHASE III INITIATED
uniQure NV	AMT-061	hemophilia B	HOPE-B; a gene therapy.
Ritter Pharmaceuticals Inc.	RP-G28	lactose intolerance	Liberatus; a placebo controlled study.
Apellis Pharmaceuticals Inc.	APL-2	paroxysmal nocturnal hemoglobinuria	PEGASUS; compared with eculizumab.
MyoKardia Inc./Sanofi	mavacamten	symptomatic obstructive hypertrophic cardiomyopathy	EXPLORER-HCM; a placebo-controlled study.
Aurinia Pharmaceuticals Inc.	voclosporin	lupus nephritis	AURORA 2; extension study.
PHASE III ANNOUNCED
Infant Bacterial Therapeutics AB	IBP-9414	necrotizing enterocolitis	In premature infants.
BioCryst Pharmaceuticals Inc.	BCX7353	hereditary angioedema	APeX-J; to take place in Japan.
Novo Nordisk AS	semaglutide	obesity	STEP, SELECT; to start in 2018.
Novo Nordisk AS	semaglutide, oral	diabetes, type 2	Cardiovascular outcomes study.
PHASE II SUSPENDED
Summit Therapeutics PLC	ezutromid	Duchenne muscular dystrophy	PhaseOut DMD; primary endpoint missed.
Merrimack Pharmaceuticals Inc.	istiratumab	pancreatic cancer	CARRIE; missed primary endpoint.
PHASE II INTERIM/TOP-LINE RESULTS
AbbVie Inc./ Galapagos NV	GLPG2737	cystic fibrosis	PELICAN; achieved efficacy endpoint, well tolerated.
Biocad	BCD-085, an IL-17 inhibitor	psoriasis, ankylosing spondylitis	Effective and sustained effects..
Gemphire Therapeutics Inc.	gemcabene	severe hyper- triglyceridemia	INDIGO-1; significant lowering of TG levels.
Nippon Shinyaku Co. Ltd.	NS-065/NCNP-01	Duchenne muscular dystrophy	Efficacy signals observed.
TrovaGene Inc.	PCM-075	acute myeloid leukemia	Treatment effect observed.
Apellis Pharmaceuticals Inc.	APL-2	complement deficiencies	Early signs of efficacy.
Reveragen BioPharma Inc.	vamorolone	Duchenne muscular dystrophy	Improved clinical symptoms.
Novelion Therapeutics Inc.	metreleptin	obesity	Decreased weight.
Sophiris Bio Inc.	topsalysin	prostate cancer, localized	Mixed results, clinical responses seen.
AstraZeneca PLC	MEDI0382	diabetes, type 2	Reduced blood glucose and body weight.
UPDATED PHASE II RESULTS
BerGenBio ASA	bemcentinib with Keytruda (pembrolizumab)	non-small cell lung cancer	Clinical responses observed.
Enteris BioPharma Inc.	Ovarest (leuprolide), oral	endometriosis	Positive results.
Stemline Therapeutics Inc.	Elzonris (tagraxofusp)	blastic plasmacytoid dendritic cell neoplasm	Efficacy shown.
vTv Therapeutics Inc.	TTP399	diabetes, type 1	Simplici-T1; trend towards glycemic control.
Zafgen Inc.	ZGN-1061	diabetes, type 2	Positive proof of concept study.
Ironwood Pharmaceuticals Inc.	praliciguat	diabetic nephropathy and hypertension	Well tolerated, signs of efficacy.
BeyondSpring Inc.	plinabulin	chemotherapy induced neutropenia	Study 105; reduced bone pain.
Roivant Sciences GMBH	LGD-6972	diabetes, type 2	Decreased HbA1c, well tolerated.
PHASE II INITIATION
BioAtla LLC	BA3021	solid tumors	BA3021-001; antibody drug conjugate.
Cocrystal Pharma Inc.	CC-31244	hepatitis C	With approved drugs.
ProQR Therapeutics BV	QR-313	epidermolysis bullosa	WINGS; placebo controlled study.
Rafael Pharmaceuticals Inc.	CPI-613	Burkitt lymphoma	In relapsed or refractory disease.
Relmada Therapeutics Inc.	dextromethadone	major depressive disorder	Adjunctive therapy.
Aclaris Therapeutics Inc.	ATI-50001	hair loss	JAK inhibitor.
Atossa Genetics Inc.	endoxifen	mammographic breast density	Topical therapy.
Galapagos NV/Servier SA	GLPG1972	osteoarthritis	ROCCELLA; a global study.
Oncology Venture APS/Medical Prognosis Institute AS	2X-121 DRP	breast cancer	PARP inhibitor.
Aurinia Pharmaceuticals Inc.	voclosporin	focal segmental glomerulosclerosis, lupus nephritis	Open-label study.
CellCentric Ltd.	CCS1477	prostate cancer, late stage	p300/CBP inhibitor.
Novartis AG	177Lu-PSMA-SR6	prostate cancer	PROfind, OROter; open-label studies.

Pipeline Watch: Phase III Trials Initiated For AMT-061, RP-G28 And APL-2

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