Pipeline Watch: Phase III Starts With Murepavadin, Infigratinib And Mavacamten

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

The progression of investigational products through a series of increasingly sophisticated and targeted clinical trials is a well-tried and tested route for the development of new medicines for commercialization. Knowing the stage that an investigational medicine has reached is information of great interest to competing companies, regulators, patients and payers.

The table below records the past week's changes to the progress of selected potential new products through the clinical trial pipeline, using data from Informa Pharma Intelligence's Biomedtracker. It lists drugs for which an important event was recorded between Oct. 19 to Oct. 25, 2018, divided by event type.

Events can include Phase II and Phase III clinical trial initiations, development discontinuations, and the release of top-line and updated clinical trial results. A brief summary of product approvals is also included.

A companion resource, the Pink Sheet's regulatory-focused FDA Performance Tracker, follows regulatory milestones such as submissions, user fee dates, complete response letters and approvals.

 

Pipeline Watch – Oct. 19 to Oct. 25, 2018

Lead company/partner	Compound	Indication	Comments
PHASE III RESULTS PUBLISHED
AstraZeneca plc/ Merck & Co.	Lynparza (olaparib)	ovarian cancer, advanced BRCA-mutated	SOLO-1; NEJM, Oct. 21, 2018.
Pfizer Inc.	Ibrance (palbociclib) plus fulvestrant	metastatic breast cancer, HR+, HER-	PALOMA-3; NEJM, Oct. 20, 2018.
Eli Lilly	Taltz (ixekizumab)	ankylosing spondylitis	COAST-W; Arthritis & Rheumatology, Oct. 20, 2018.
Eli Lilly	Taltz (ixekizumab)	ankylosing spondylitis	COAST-V, The Lancet, Oct. 22, 2018.
Johnson & Johnson	Zytiga (abiraterone) plus radiotherapy	prostate cancer	STAMPEDE; The Lancet, Oct. 21, 2018.
Takeda	Velcade (bortezomib)	mantle cell lymphoma, front-line	The Lancet Oncology, Oct. 19, 2018.
Novartis AG/Amgen Inc.	Aimovig (erenumab)	refractory migraine	LIBERTY; The Lancet, Oct. 22, 2018.
PHASE III INTERIM/TOP-LINE RESULTS
Bayer/Orion Pharma	darolutamide	prostate cancer	ARAMIS; extended metastasis-free survival.
H. Lundbeck A/S	Lu AF35700	schizophrenia, treatment-resistant	DayBreak; missed primary endpoint.
Syndax Pharmaceuticals	entinostat with exemestane	breast cancer	E2112; mixed results.
Amgen Inc.	Enbrel (etanercept)	psoriatic arthritis	SEAM-PsA; better than methotrexate.
Genmab/Johnson & Johnson	Darzalex (daratumumab)	multiple myeloma, front line	Cassiopeia; met primary endpoint.
UPDATED PHASE III RESULTS
Johnson & Johnson	Tremfya (guselkumab)	moderate to severe psoriasis	VOYAGE-1; stable clearance at 3 years.
Takeda	Alunbrig (brigatinib)	NSCLC, with CNS metastasis	ALTA-1L; improved intracranial efficacy.
Bayer	Xofigo (radium-223) plus abiraterone	prostate cancer, metastatic castration- resistant	ERA-223; did not improve efficacy, higher fracture rate.
PharmaMar	Zepsyre (lurbinectedin)	ovarian cancer, platinum-resistant	CORAIL; mixed results.
Roche AG	Tecentriq (atezolizumab) plus Abraxane	advanced breast cancer, triple negative, first-line	IMpassion 130; improved outcomes.
HUYA Bioscience	chidamide (HBI-8000) plus exemestane	breast cancer	Benefit on PFS.
Novartis AG	alpelisib plus fulvestrant	breast cancer	SOLAR-1; increased median PFS.
Eli Lilly	Cyramza (ramucirumab) plus docetaxel	bladder cancer	RANGE; prolonged PFS.
Takeda	Entyvio (vedolizumab) subcutaneous	ulcerative colitis	VISIBLE 1; achieves and maintains remission.
Merk KGaA/Pfizer	Bavencio (avelumab)	renal cell cancer	JAVELIN Renal 101; met primary endpoint.
Otsuka/Servier	Lonsurf (trifluridine/ tipracil)	gastric cancer	TAGS; met primary endpoint.
Novartis AG	Cosentyx (secukinumab)	psoriatic arthritis	FUTURE 5; sustained clinical responses at 52 weeks.
AbbVie Inc.	upadacitinib	rheumatoid arthritis, inadequate or naïve to methotrexate	SELECT-COMPARE, -EARLY; improved symptoms.
Sellas Life Sciences Group Inc.	NeuVax (nelipeptimut-S)	breast cancer	Clinical benefit shown.
Gilead Sciences/ Galapagos	filgotinib	rheumatoid arthritis	FINCH 2; improved symptoms.
Bristol-Myers Squibb	Opdivo (nivolumab) plus Yervoy (ipilimumab)	renal cell carcinoma	CheckMate-214; treatment-free survival improved.
Novartis AG	Cosentyx (secukinumab)	ankylosing spondyl- arthritis	MEASURE 2; sustained benefit after 4 years.
Merck & Co	Keytruda (pembrolizumab)	head and neck cancer, first line	KeyNote-048; durable responses.
FibroGen	roxadustat	anemia due to chronic renal failure, dialysis- dependent and independent	Improved symptoms in China studies.
Merz	Xeomin (incobotulin- umtoxinA)	chronic sialorrhea	SIAXI; sustained improvements.
Regeneron Pharmaceuticals Inc.	Eylea (aflibercept)	diabetic retinopathy, non-proliferative	PANORAMA; improved symptoms.
PHASE III COMPLETED
Pfizer/Eli Lilly	tanezumab	osteoarthritis pain	Pain reduced.
PHASE III INITIATED
Polyphor AB	murepavadin	nosocomial pneumonia	PRISM-UDR; due to P. aeruginosa.
QED Therapeutics	infigratinib	biliary tract cancer	Compared to standard of care.
MyoKardia Inc	mavacamten	hypertrophic cardio- myopathy	MAVA-LTE; long-term safety study.
MitoTech SA/Essex Bio-Technology	Visomitin (skQ1)	dry eye disease	In moderate to severe disease.
PHASE III ANNOUNCED
Syndax Pharmaceuticals Inc.	entinostat with pembrolizumab	NSCLC	In PD-L1 refractory disease.
Alnylam Pharmaceuticals Inc.	lumasiran	hyperoxaluria	ILLUMINATE-B; in children.
PHASE II INTERIM/TOP-LINE RESULTS
Johnson & Johnson	Ad26.Mos4.HIV vaccine, tetravalent	HIV/AIDS	TRAVERSE; promising immune responses.
Intra-Cellular Therapies Inc.	ITI-214	Parkinson's disease	Signs of improvement, well tolerated.
UCB SA/Biogen Inc.	dapirolizumab pegol	systemic lupus erythematosus	Mixed results.
AbbVie Inc.	upadacitinib	ulcerative colitis	U-ACHIEVE; positive results.
Clovis Oncology	Rubraca (rucaparib)	prostate cancer	TRITON2; promising results.
Takara Bio	C-REV (canerpaturev) plus ipilimumab	metastatic melanoma	Encouraging clinical activity.
Incyte/Novartis AG	capmatinib	NSCLC	GEOMETRY mono-1; positive results.
Exelixis Inc.	Cabometyx (cabozantinib)	sarcoma	Meaningful clinical activity.
Agenus Inc.	AGEN2034, AGEN1884	cervical cancer	Early signals of activity.
Merck & Co.	Keytruda	bladder cancer, high-risk, non-muscle	KeyNote-057; encouraging activity.
Medicenna Therapeutics Corp.	MDNA55	glioblastoma	Clinical responses.
GlaxoSmithKline PLC/ MorphoSys AG	GSK3196165	rheumatoid arthritis	Clinically meaningful benefits.
AstraZeneca plc	Imfinzi (durvalumab)	small cell lung cancer	BALTIC; encouraging antitumor activity.
MC2 Therapeutics A/S	PADciclo (ciclosporin)	dry eye disease	Clinical improvement reported.
Bristol-Myers Squibb	ipilimumab plus nivolumab	colorectal cancer	NICHE; tumor responses associated with neoadjuvant use.
AbbVie Inc/ Boehringer Ingelheim	risankizumab	Crohn's disease	Sustained effects after long-term therapy.
Eisai/Purdue	lemborexant	sleep disorder	Study 202; improved sleep patterns.
UPDATED PHASE II RESULTS
Merck KGaA	tepotinib	liver cancer	Encouraging signs of efficacy.
Stemline Therapeutics Inc.	SL-701	brain cancer	Antitumor activity, well tolerated.
Glenmark Pharmaceuticals Ltd.	GBR 830	atopic dermatitis	Promising results.
BioLineRx Ltd.	BL-8040 plus pembrolizumab	pancreatic cancer	COMBAT/KeyNote-202; encouraging disease control.
Idera Pharmaceuticals Inc.	tilsotolimod plus ipilimumab, pembrolizumab	melanoma	ILLUMINATE 204; durable responses.
X4 Pharmaceuticals Inc.	X4P-001 plus nivolumab	clear cell renal cell cancer	Improved clinical responses.
QED Therapeutics	infigratinib	bile duct cancer	Clinical responses.
Dynavax Technologies Corp.	SD-101 with pembrolizumab	melanoma, head & neck cancer	SYNERGY-001, KeyNote-184; encouraging results.
Innate Pharma	monalizumab with cetuximab	head & neck carcinoma	Durable responses.
Bristol-Myers Squibb	Opdivo plus ipilimumab	advanced bladder cancer	CheckMate-032; improved PFS.
Adaptimmune Therapeutics plc	MAGE-A10, -A4, SPEAR T-cells	NSCLC, solid tumors	Signs of antitumor activity.
G1 Therapeutics Inc.	trilaciclib	small cell lung cancer	Immune system benefits.
Mologen AG	lefitolimod	small cell lung cancer	IMPULSE; positive results.
Tesaro Inc.	Zejula (niraparib)	ovarian cancer, fourth line	QUADRA; durable responses.
Arena Pharmaceuticals Inc.	etrasimod	severe ulcerative colitis	OASIS; clinical responses.
Genmab/Seattle Genetics	tisotumab vedotin	cervical cancer	Encouraging activity.
Celtaxsys Inc.	acebilustat	cystic fibrosis	EMPIRE CF; improved lung exacerbations.
AstraZeneca PLC	AZD6738 plus durvalumab	solid tumors	Signals of clinical activity.
Leap Therapeutics Inc.	DKN-01 plus pembrolizumab	esophageal cancer	Early efficacy signals.
Bayer AG/Loxo Oncology Inc.	larotrectinib	TRK fusion cancers	SCOUT, NAVIGATE; consistent clinical efficacy.
Incyte Corp.	pemigatinib	cholangiocarcinoma	FIGHT-202; initial signs of efficacy.
Athenex Inc.	Oraxol (HM30181A and oral paclitaxel)	breast cancer	Encouraging efficacy.
Merck KGaA	M7583	diffuse large B-cell lymphoma	Evidence of clinical benefit.
Johnson & Johnson/ MorphoSys	Tremfya (guselkumab)	dactylitis in psoriatic arthritis	Resolved symptoms.
Merus NV	MCLA-128	solid tumors	Activity seen.
Aveo Oncology/EUSA Pharma	tivozanib plus nivolumab	renal cell carcinoma	TiNivo; favorable efficacy and safety.
Ionis Pharmaceuticals Inc./AstraZeneca plc	danvatirsen plus durvalumab	head and neck cancer	SCORES; clinical responses seen.
Protagonist Therapeutics Inc.	PTG-100	ulcerative colitis	PROPEL; signals of clinical efficacy.
Samamed LLC	SM04690	osteoarthritis	Improvements seen.
ImmunoGen Inc.	mirvetuximab soravtansine plus pembrolizumab	ovarian cancer	FORWARD II; initial clinical activity.
Intra-Cellular Therapies Inc.	ITI-214	Parkinson's disease	Improved motor symptoms.
Epizyme Inc.	tazemetostat	solid tumors	Promising activity.
Corbus	lenabasum	dermatomyositis, scleroderma	Reduced T-cell inflammation.
CytomX Therapeutics Inc	CX-072	solid tumors	PROCLAIM-072; responses observed.
Bristol-Myers Squibb	nivolumab plus ipilimumab	metastatic colorectal cancer, first-line	CheckMate-142; durable clinical benefit.
Kura Oncology	tipifarnib	squamous cell carcinoma with HRAS mutations	Positive clinical data.
Mirati Therapeutics	sitravatinib plus nivolumab	NSCLC	Significant clinical activity.
Johnson & Johnson	Stelara (ustekinumab)	systemic lupus erythematosus	Sustained effect on disease activity.
Amo Pharma	AMO-02	muscular dystrophy	Clinical benefits seen.
OrthoTrophix Inc.	TPX-100	knee osteoarthritis	knee cartilage structure improved.
Xencor Inc.	XmAb5871	systemic lupus erythematosus	Supports further evaluation.
Bristol-Myers Squibb	BMS-986165	plaque psoriasis	Clinical efficacy noted.
UCB SA	bimekizumab	psoriatic psoriasis	BE ACTIVE; achieved primary endpoint.
Galera Therapeutics Inc.	avasopasem manganese	radiation induced oral mucositis	Reduced symptoms.
Selecta Biosciences Inc.	SEL-212	gout	Reduced symptoms.
Neurotrope Inc.	bryostatin-1	Alzheimer's disease	Encouraging data.
TaiwanJ Pharmaceuticals	JKB-122	non-alcoholic steatohepatitis (NASH)	Improved symptoms.
Beigene	zanubrutinib	chronic lymphocytic leukemia	Durable clinical responses seen.
Eisai/Biogen	BAN2401	Alzheimer's disease	Study 201; reduced brain amyloid.
Akebia Therapeutics Inc.	vadadustat	anemia in chronic renal failure, dialysis- independent	CI-0021, CI-0022; symptoms improved.
Ovid Therapeutics	OV101	Angelman syndrome	STARS; improved symptoms.
Horizon Pharma	teprotumumab	active thyroid eye disease	Improved symptoms.
Alnylam Pharmaceuticals Inc.	lumasiran	hyperoxaluria	Reduced oxalate levels.
PHASE II INITIATED
AstraZeneca plc	Tagrisso (osimertinib)	NSCLC	ORCHARD; in advanced disease.
Twi Pharmaceuticals	AC-203	epidermolysis bullosa	Inherited disease.
Kazia Therapeutics Ltd.	GDC-0084	brain cancer	With Dana-Farber Cancer Institute.
Merck KGaA	M7824	NSCLC	In PD-L1 expressers.
Santhera Pharmaceuticals	POL6014	cystic fibrosis	Multiple ascending dose study.
Axovant Sciences	AXO-LENTI-PD, gene therapy	Parkinson's disease	Initial data expected in 2019 first half.
Laurent Pharmaceuticals Inc.	LAU-7b	cystic fibrosis	APPLAUD; effects on lung function.

Source: Informa Pharma Intelligence's Biomedtracker.

 

 

Marketing Approvals – Oct. 19 to Oct. 25, 2018

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Roche	Shionogi	Xofluza (baloxavir marboxil)	influenza	US	Single oral dose.
Spectrum Pharmaceuticals	-	Khapzory (levoleucovorin)	rescue therapy	US	In various cancers.
Zai Lab	Tesaro Inc.	Zejula (niraparib)	ovarian cancer	Hong Kong	In adult patients.
Ethypharm	-	Baclocur (baclofen)	alcohol dependence	France	To reduce alcohol consumption.
SUPPLEMENTAL REGULATORY APPROVAL
Sanofi Inc.	Regeneron Inc.	Dupixent (dupilumab)	asthma, moderate to severe	US	Add-on maintenance therapy.

Source: Biomedtracker.

 

Data are from Biomedtracker, the pharmaceutical and biotechnology industries' window into the FDA approval process, offering real-time intelligence for clinical and regulatory events. For more information on the research covered in this article, click here.