Lead company/partner	Compound	Indication	Comments
PHASE III SUSPENDED
Teva Pharmaceutical Industries Ltd.	fremanezumab	migraine	ENFORCE; endpoints unlikely to be met for chronic cluster headache.
PHASE III RESULTS PUBLISHED
Fennec Pharmaceuticals Inc.	sodium thiosulfate	hearing loss - chemotherapy-induced	PEDMARK study in the NEJM.
Gilead Sciences Inc.	Biktarvy (bictegravir/ emtricitabine/tenofovir alafenamide)	HIV/AIDS	GS-US-380-1844 non-inferiority trial in The Lancet.
Gilead Sciences	Biktarvy (bictegravir/ emtricitabine/tenofovir alafenamide)	HIV/AIDS	GS-US-380-1878 non-inferiority trial published in The Lancet HIV.
PHASE III INTERIM/TOP-LINE RESULTS
Nanobiotix SA	NBTXR3	sarcoma	Act.In.Sarc trial; positive results for soft tissue sarcoma.
Shire PLC	Cinryze (IV) (C1 esterase inhibitor [human])	hereditary angioedema (HAE)	Study 0624-301 pediatric study. Lessened the severity of attacks.
Novo Nordisk AS	oral semaglutide	Type 2 diabetes	PIONEER 4 and PIONEER 7; positive results.
Bristol-Myers Squibb Co.	Orencia (abatacept)	lupus nephritis	Failed to meet its primary endpoint.
UPDATED PHASE III RESULTS
Rigel Pharmaceuticals Inc..	Tavalisse (fostamatinib)	immune thrombocytopenic purpura (ITP)	Study 049; represents a treatment option for adult ITP.
Alexion Pharmaceuticals Inc.	ravulizumab (ALXN1210)	paroxysmal nocturnal hemoglobinuria (PNH)	Noninferiority to eculizumab on primary and key secondary endpoints.
Geron Corp.	imetelstat	myelodysplastic syndrome	IMerge study; encouraging results.
Johnson & Johnson	esketamine	major depressive disorder	SUSTAIN-1 AND -2, TRANSFORM-2 AND -3; updates at ECNP.
Alnylam Pharmaceuticals Inc.	patisiran	transthyretin-related hereditary amyloidosis	APOLLO; positive results for primary endpoint.
Celgene Corp.	Pomalyst (pomalidomide)	multiple myeloma	OPTIMISMM: significant and clinically meaningful PFS improvement.
CSL Ltd.	Privigen (immune globulin)	chronic inflammatory demyelinating polyneuropathy	PATH; significantly reduced relapse rate.
Daiichi Sankyo Co. Ltd.	quizartinib	acute myelogenous leukemia	QuANTUM-R; significantly prolonged OS.
Novartis AG	Cosentyx (secukinumab)	axial spondyloarthritis	MEASURE 1 and 2; sustained improvement.
Novartis AG	Kymriah (tisagenlecleucel)	diffuse large B-cell lymphoma	JULIET study; strong efficacy with durable responses.
Seattle Genetics Inc.	Adcetris (brentuximab)	Hodgkin's lymphoma	ECHELON; improved outcomes for advanced patients.
UCB SA	Cimzia (certolizumab pegol)	psoriatic arthritis	RAPID-PsA; long-term positive results.
AbbVie Inc.	Venclexta (venetoclax)	multiple myeloma	MURANO trial; encouraging findings.
Amgen Inc.	Evenity (romosozumab)	osteoporosis	FRAME Study; substantially higher BMD T-score increases after one year.
Celgene Corporation	Revlimid (lenalidomide)	indolent non-Hodgkin's lymphoma	RELEVANCE; lenalidomide plus rituximab vs rituximab + chemotherapy not superior but different toxicity profile.
Novartis	Jakavi (ruxolitinib)	myelofibrosis	JUMP; may lead to greater benefits.
Novartis	Cosentyx (secukinumab) sc	psoriatic arthritis	FUTURE 5 study.
Novartis	biosimilar Erelzi (etanercept), GP2015	rheumatoid arthritis (RA)	EQUIRA; positive switching data in moderate-to-severe RA.
Novartis	biosimilar Zessly (infliximab).	rheumatoid arthritis	REFLECTIONS B537-02 study; positive switching data.
Verastem Inc.	duvelisib	chronic lymphocytic leukemia/small cell lymphocytic Lymphoma	DUO Extension study; further support oral duvelisib monotherapy as an effective oral treatment option.
PHASE III INITIATED
Blueprint Medicines Corp.	avapritinib	gastrointestinal stromal tumor (GIST)	VOYAGER Phase III compared with regorafenib in advanced GIST.
ReVance Therapeutics Inc.	daxibotulinumtoxinA for Injection (RT002)	cervical dystonia	ASPEN Phase III clinical program
Idorsia Pharmaceuticals Ltd.	aprocitentan	hypertension	PRECISION for resistant hypertension in adults.
Marinus Pharmaceuticals Inc.	ganaxolone	seizure disorders (epilepsy)	Marigold Study oral ganaxolone in children and young adults with CDKL5 deficiency disorder.
Agios Pharmaceuticals Inc.	AG-348	metabolic	ACTIVATE in pyruvate kinase deficiency; positive data.
Idorsia Pharmaceuticals	Pivlaz (clazosentan)	hemorrhagic stroke / intracerebral hemorrhage	REACT; prevention of clinical deterioration due to vasospasm-related delayed cerebral ischemia in patients following an aneurysmal subarachnoid hemorrhage.
AROG Pharmaceuticals, LLC	crenolanib	acute myelogenous leukemia	ARO021 to compare the efficacy of crenolanib versus midostaurin combined with standard chemotherapy.
PHASE III ANNOUNCED
Kala Pharmaceuticals Inc.	KPI-121 0.25%	dry eye	STRIDE 3 (STRIDE - Short Term Relief In Dry Eye), to begin Q3 2018.
Rhythm Pharmaceuticals Inc.	setmelanotide	obesity	A combined pivotal Phase III trial of setmelanotide in BBS and Alström Syndrome.
PHASE II INTERIM/TOP-LINE RESULTS
Heron Therapeutics Inc.	HTX-011	postsurgical pain	Study 209 (in total knee arthroplasty) and Study 211 (in breast augmentation); primary endpoints achieved in both studies.
[C#200900382:argenx SE]	efgartigimod	pemphigus vulgaris	Well-tolerated with no severe or serious study drug-related adverse events.
[CerebralRx Ltd.]	CT001	seizure disorders (epilepsy)	Preliminary clinical proof of concept data.
Sarepta Therapeutics Inc.	microdystrophin gene therapy program	Duchenne muscular dystrophy	Phase I/IIa increase in α-Sarcoglycan expression from baseline.
hVIVO	Flu-v	influenza (including vaccines)	Phase IIb Study 003; successful results.
ObsEva SA	OBE2109	endometriosis	EDELWEISS Phase IIb clinical trial; 12-week data positive.
Bristol-Myers Squibb Co.	Empliciti (elotuzumab)	multiple myeloma	ELOQUENT-3 trial, highly significant 46% reduction in risk of progression or death.
Cytokinetics Inc.	reldesemtiv (CK-2127107)	spinal muscular atrophy	CY 5021 trial; successful results
Verastem Inc.	duvelisib	chronic lymphocytic leukemia (CLL)	Phase Ib/II study; an effective regimen for the initial therapy of younger, fit CLL patients.
MorphoSys AG	MOR208	chronic lymphocytic leukemia/small cell lymphocytic lymphoma	Phase II COSMOS trial; potential medical application in additional B-cell malignancies.
Tolero Pharmaceuticals	alvocidib	acute myelogenous leukemia	Zella 201, an ongoing Phase II study; encouraging activity.
Acceleron Pharma Inc.	luspatercept	myelodysplastic syndrome	Phase II PACE-MDS Study; increased response rates were seen.
Akari Therapeutics PLC	Coversin	paroxysmal nocturnal hemoglobinuria	COBALT, met its primary endpoint.
UPDATED PHASE II RESULTS
Catalyst Biosciences Inc.	CB 2679d/ISU304	severe hemophilia B	Production of neutralizing antibodies against CB 2679d.
Rhythm Pharmaceuticals	setmelanotide	obesity	Phase II basket studies; efficacy in Bardet-Biedl Syndrome; proof-of-concept achieved in Alström syndrome.
TG Therapeutics Inc.	ublituximab	multiple sclerosis	ULTIMATE I & II; favorable clinical and MRI outcomes at week 52.
BioLineRx Ltd.	BL-8040	acute myelogenous leukemia (AML)	Significantly improved overall survival of r/r AML patients.
Roche	risdiplam (RG7916)	spinal muscular atrophy	FIREFISH study; promising results.
TG Therapeutics Inc.	TGR-1202 (umbralisib)	chronic lymphocytic leukemia (CLL)	Safe and effective in a KI intolerant CLL population.
Wilson Therapeutics AB	WTX101 (bis-choline tetrathiomolybdate)	Wilson disease	Study 201; improved neurological status and disability.
AROG Pharmaceuticals, LLC	crenolanib	acute myelogenous leukemia	ARO-006; positive results for younger patients.
bluebird bio Inc.	LentiGlobin	anemia	Phase I/II Northstar (HGB-204) study; positive data.
Epizyme Inc.	tazemetostat	indolent non-Hodgkin's lymphoma	Generally well tolerated with meaningful clinical activity and durability of response.
Erytech Pharma SA	ERY-ASP	acute myelogenous leukemia	ENFORCE; addition of eryaspase did not improve survival in this difficult-to-treat patient population
Global Blood Therapeutics Inc.	voxelotor	sickle cell anemia	HOPE-KIDS 1 Study; sustained and durable improvements in hemoglobin levels.
Johnson & Johnson	Tremfya (guselkumab)	psoriatic arthritis	Phase IIa 2001; substantial benefits
Johnson & Johnson	Stelara (ustekinumab)	systemic lupus erythematosus (SLE)	Efficacy in pts with active SLE and was well-tolerated. May work via a novel mechanism of action in SLE.
Molecular Partners AG	MP0250	multiple myeloma	Acceptable safety profile and promising activity in realspsed/refractory MM patients.
Nohla Therapeutics Inc.	dilanubicel (NLA101)	acute myelogenous leukemia	Significantly improved hematopoietic recovery.
Novartis AG	Tasigna (nilotinib)	chronic myelogenous leukemia	ENESTop and ENESTfreedom; long-term results positive.
Oncopeptides AB	Ygalo	multiple myeloma	HORIZON trial; encouraging results.
Ra Pharmaceuticals Inc.	RA101495	paroxysmal nocturnal hemoglobinuria	Appears safe and well-tolerated; support Phase III trial.
Rigel Pharmaceuticals Inc.	Tavalisse (fostamatinib)	anemia	SOAR; markedly improved Hgb levels in some patients with warm antibody autoimmune hemolytic anemia.
Selecta Biosciences Inc..	SEL-212	gout	Well tolerated, mitigated immunogenicity, and sustained control of serum uric acid levels.

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