Lead company/partner	Compound	Indication	Comments
Updated Phase III Results
GW Pharmaceuticals PLC	Epidiolex (cannabidiol)	Lennox-Gastaut syndrome	GWPCARE3; further positive results.
Gilead Sciences Inc.	Vemlidy (tenofovir alafenamide)	hepatitis B	Safe and effective, as was switching from tenofovir disoproxil fumarate.
Phase III Interim/Top-line Results
AbbVie Inc.	veliparib (a PARP inhibitor)	squamous non-small cell lung cancer (NSCLC), triple negative breast cancer	Missed primary endpoints .
AbbVie Inc./Enanta Pharmaceuticals Inc.	glecaprevir/pibrentasvir	hepatitis C and compensated cirrhosis	EXPEDITION-1; 99% SVR at 12 weeks .
Argos Therapeutics Inc.	rocapuldencel-T (AGS-003) plus sunitinib	renal cell cancer	ADAPT; mixed results but will continue the study .
Mateon Therapeutics	Zybrestat (CA4P; fosbretabulin tromethamine)	ovarian cancer	FOCUS; PFS favored CA4P, no safety issues.
Motif Bio PLC	iclaprim	skin and skin structure infections	REVIVE-1; non-inferior to vancomycin .
Ultragenyx Pharmaceutical Inc./Kyowa Hakko Kirin Co. Ltd.	burosumab (KRN23)	X-linked hypophosphatemia	CL303; increased serum phosphorus, the primary endpoint .
Roche	emicizumab	hemophilia A	HAVEN 2; in pediatric patients, reduced bleeds .
Phase III Initiated
Boehringer Ingelheim GMBH	nintedanib	progressive fibrosing interstitial lung disease	In conditions other than idiopathic pulmonary fibrosis.
Phase III Announced
R-Pharm CJSC	olokizumab	rheumatoid arthritis	CREDO 4; in patients with moderate to severe active disease.
OxThera AB	Oxabact (freeze dried live Oxalobacter bacteria)	hyperoxaluria	OC5-DB-02; to enrol 18 patients.
Leo Pharma AS	LEO 43204 (ingenol disoxate) gel	actinic keratosis	Effect on skin neoplasia.
ProMetic Life Sciences Inc.	PBI-4050	idiopathic pulmonary fibrosis	In patients treated with nintedanib.
SynCore Biotechnology Co. Ltd.	SB05 (EndoTAG-1; lipids loaded with paclitaxel)	pancreatic cancer	Combined with gemcitabine.
Phase II Suspended
Circassia Pharmaceuticals PLC	ToleroMune (house dust mite allergen)	house dust mite allergy	Missed primary endpoint on allergy symptoms and use of rescue medication
OncoMed Pharmaceuticals Inc.	tarextumab	small cell lung cancer	PINNACLE; no effect on PFS or overall survival.
Updated Phase II Results
Eiger BioPharmaceuticals Inc.	Sarasar (lonafarnib)	hepatitis D	LOWR-4; with ritonavir, reduced HDV RNA levels.
Aurinia Pharmaceuticals Inc.	Luveniq (voclosporin)	lupus nephritis	AURA-LV; continued to meet primary endpoints.
Merck & Co. Inc.	MK-3682 plus grazoprevir, ruzasvir	hepatitis C	Effective and well tolerated.
Replicor Inc.	REP-2139-Mg, REP- 2055	hepatitis B	Effective and well tolerated.
Phase II Interim/Top-line Results
Arrowhead Pharmaceuticals Inc./Alnylam Pharmaceuticals Inc.	ARC-520, ARC-521	hepatitis B	RNA interference, significantly reduced HBV viral antigens.
Rebiotix Inc.	RBX2660 (microbiota suspension)	recurrent C difficile infection	PUNCH; open-label study, well tolerated, met primary endpoint, reduced recurrence.
Phase II Initiation
Allergan PLC	cenicriviroc	non-alcoholic steatohepatitis (NASH)	With Novartis’s FXR agonist, LJN452 .
AOBiome	B244 (nitric oxide-delivering bacteria)	hypertension	Patient enrolment completed
Medicenna Therapeutics Inc.	MDNA55	malignant glioma	Results expected first half of 2018.
GeNeuro SA	GNbAC1 (a MAb against MSRV-Env)	type 1 diabetes	Already being evaluated in multiple sclerosis.
AbbVie Inc./Galapagos NV	ABBV-2222	cystic fibrosis	In patients homozygous for the F508del mutation.
Bavarian Nordic AS	ProstVac (rilimogene) with ipilimumab and nivolumab	prostate cancer	In patients with localized disease.
Immunovaccine Inc.	DPX-E7 cancer vaccine	HPV-associated cancers	With low-dose cyclophosphamide.
Oxis International Inc.	OXS-1550	hematologic cancer	An antibody-drug conjugate.

Pipeline Watch: Dismay With Veliparib, Tarextumab, House Dust Mite Allergens

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