Lead company/partner	Compound	Indication	Comments
Phase III Results Published
GlaxoSmithKline PLC	Fluarix Tetra (flu vaccine)	influenza prophylaxis in infants	The Lancet Child & Adolescent Health online, March 5, 2018.
Phase III Interim/Top-line Results
Dermira Inc.	olumacostat glasaretil	acne vulgaris	CLAREOS-1, 2; missed co-primary endpoints, program likely to be discontinued.
VBL Therapeutics	VB-111	glioblastoma	GLOBE; missed primary endpoint.
Purdue Pharma LP/Eisai Co. Ltd.	lemborexant	sleep disorders	SUNRISE 1; achieved endpoints.
Vanda Pharmaceuticals Inc.	Hetlioz (tasimelteon)	jet lag disorder	JET8; clinically meaningful effects on sleep.
Esperion Therapeutics Inc.	bempedoic acid	dyslipidemia	CLEAR Tranquility; met primary endpoint.
Glenmark Pharmaceuticals Ltd.	Ryaltris (mometasone/ olopatadine) nasal spray	seasonal allergic rhinitis	Rapidly improved nasal symptoms.
Clearside Biomedical Inc.	suprachoroidal CLS-TA (triamcinolone acetonide)	macular edema in non-infectious uveitis	PEACHTREE; met primary endpoint.
ViiV Healthcare	Tivicay (dolutegravir)	HIV/AIDS with tuberculosis	INSPIRING; well tolerated and effective.
Gilead Sciences Inc.	Biktarvy (bictegravir/ emtricitabine/tenofovir alafenamide)	HIV/AIDS in adolescents	Viral suppression observed.
Updated Phase III Results
Shire PLC	lanadelumab	prevention of hereditary angioedema	HELP; clinically meaningful efficacy.
Aimmune Therapeutics Inc.	AR101	peanut allergy	PALISADE; positive results.
DBV Technologies SA	Viaskin Peanut	peanut allergy	PEPITES; mixed results.
Phase III Initiated
Nightstar Therapeutics Ltd.	NSR-REP1 gene therapy	chorioderemia	STAR; using a AAV2 vector.
Allena Pharmaceuticals Inc.	ALLN-177	enteric hyperoxaluria	URIROX-1; data expected in 2019.
Bioverativ Inc./ Sanofi	BIVV009	cold agglutinin disease	Cardinal, Cadenza; in adult patients.
Insys Therapeutics Inc.	cannabidiol oral solution	infantile spasms	A rare type of epilepsy in very young children.
Clearside Biomedical Inc.	suprachoroidal CLS-TA (triamcinolone acetonide)	macular edema associated with retinal vein occlusion	TOPAZ; combined with an anti-VEGF agent.
Biohaven Pharmaceuticals Holding Co. Ltd.	rimegepant	migraine, acute	A Zydis oral dissolving tablet formulation.
Updated Phase II Results
Ultragenyx Pharmaceutical Inc.	DTX301, gene therapy	ornithine transcarb- amylase deficiency	Promising efficacy, acceptable safety.
Bavarian Nordic AS	MVA-BN RSV, a universal RSV vaccine	RSV infection	Positive data in elderly population.
G1 Therapeutics Inc.	trilaciclib	small cell lung cancer	Favorable signs of efficacy and well tolerated.
GTx Inc.	enobosarm	urinary incontinence	Positive data in women.
Roche/ Ionis Pharmaceuticals Inc.	IONIS-HTTRx	Huntington's disease	Reduced mutant Huntington protein levels.
Phase II Interim/Top-line Results
Merck KGAA	evobrutinib	multiple sclerosis	Reduced CNS lesions.
Zafgen Inc.	ZAF-1061-201	diabetes, type 2	Promising initial results.
Sanifit	SNF472	calciphylaxis	Primary and secondary endpoints met.
Santhera Pharmaceuticals AG	idebenone	multiple sclerosis, primary progressive	No signs of efficacy on disease progression.
Roivant Sciences GMBH	vibegron	overactive bladder	Reduced urgency and well tolerated.
Verona Pharma PLC	RPL554	cystic fibrosis	Improved FEV1.
Reata Pharmaceuticals Inc.	omaveloxolone	mitochondrial myopathies	Initial signs of efficacy.
Immuron Ltd.	IMM-124E	non-alcoholic steatohepatitis (NASH)	Positive results.
Caladrius Biosciences Inc.	CLBS03, autologous cell therapy	diabetes, type 1	T-Rex; well tolerated, positive signs.
MyoKardia Inc.	mavacamten	congestive heart failure	PIONEER-HCM; met primary endpoint.
Phase II Initiation
MaaT Pharma	MaaT013, microbiome enema	acute graft versus host disease	In stem cell transplant patients.
Hutchison China Meditech Ltd.	epitinib	glioblastoma	In China.
Benitec Biopharma Ltd.	BB-401	head and neck cancer	In Australia and Russia.
Ablynx NV	ALX-0171, an inhaled Nanobody	RSV infections	In Japan.
InflaRx GMBH	IFX-1	hidradenitis suppurativa	Its lead compound.

Pipeline Watch: Phase III Starts With NSR-REP1 Gene Therapy, ALLN-177 And BIVV009

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