Pipeline Watch: Phase III Progress With Bempedoic Acid, Lefamulin, And Ibrutinib Combo

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 May 18 and May 24, 2018, divided by event type.

Events can include Phase II and Phase III 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 – May 18 to May 24, 2018

Lead company/partner	Compound	Indication	Comments
PHASE III RESULTS PUBLISHED
Sanofi/  Regeneron Pharmaceuticals Inc.	Dupixent (dupilimub)	asthma	LIBERTY ASTHMA QUEST, VENTURE; NEJM, May 21, 2018.
AstraZeneca PLC	tralokinumab	asthma	STRATOS 1, 2; The Lancet Respiratory Medicine, May 20, 2018.
PHASE III INTERIM/TOP-LINE RESULTS
Esperion Therapeutics Inc.	bempedoic acid	lipid lowering in statin intolerant patients	Study 3; positive top-line results.
Nabriva Therapeutics PLC	lefamulin, oral	community-acquired pneumonia	LEAP-2; positive results.
AbbVie Inc./ Janssen Biotech Inc.	Imbruvica (ibrutinib) plus Gazyva (obinutuzumab)	chronic lymphocytic leukemia, first-line	iLLUMINATE; met primary PFS endpoint.
GlaxoSmithKline PLC	Nucala (mepolizumab)	asthma, severe eosinophilic	COLUMBA; long-term safety and efficacy supported.
UroGen Pharma Ltd.	MitoGel (mitomycin)	urothelial cancer, upper tract	OLYMPUS; durable complete responses.
Trevena Inc.	oliceridine	acute pain following colorectal surgery	Well tolerated in open label safety study.
Avenue Therapeutics Inc.	iv tramadol	pain following surgery	Effective and well tolerated.
UPDATED PHASE III RESULTS
Merck & Co. Inc.	Keytruda (pembrolizumab)	squamous non-small cell lung cancer (NSCLC)	KeyNote-407; PFS and overall survival improved.
Sesen Bio Inc.	Vicinium (antibody drug conjugate)	invasive bladder cancer	VISTA; effective and well tolerated.
Roche	Hemlibra (emicizumab-kxwh)	hemophilia A	HAVEN 3, 4; prevented bleeding.
Shire PLC	Vonvendi (von Willebrand factor)	von Willebrand disease in surgical patients	Effective and well tolerated.
Shire PLC	Natpara (rhPTH(1-84))	hypoparathyroidism	RACE; well tolerated and effective in five-year data.
AstraZeneca PLC	Bevespi Aerosphere (glycopyrrolate/ formoterol fumarate)	chronic obstructive pulmonary disease	PINNACLE-4; improved lung function.
Circassia Pharmaceuticals PLC	Duaklir Pressair (aclidinium/ formoterol)	chronic obstructive pulmonary disease	AMPLIFY; improved symptoms.
AstraZeneca PLC	Fasrena (benralizumab)	severe asthma	ZONDA; reduced oral steroids, improved lung function.
PHASE III ANNOUNCED
Otsuka Holdings Co. Ltd./ Lundbeck Inc.	Rexulti (brexpiprazole)	agitation associated with Alzheimer's disease	A 12-week study.
MyoKardia Inc.	mavacamten	cardiomyopathies	EXPLORER-HCM.
PHASE II INTERIM/TOP-LINE RESULTS
Clementia Pharmaceuticals Inc.	palovarotene	fibrodysplasia ossificans progressive	Reduced new bone growth, well tolerated.
GlaxoSmithKline PLC	nemiralisib	chronic obstructive pulmonary disease	Improved lung function.
Vanda Pharmaceuticals Inc.	Hetlioz (tasimelteon)	jet lag disorder	JET; effective in transatlantic travel.
Allergy Therapeutics PLC	Pollinex Quattro Grass (grass allergens)	seasonal allergic rhinitis	Dose response observed.
AstraZeneca PLC	AZD8871	chronic obstructive pulmonary disease	Bronchodilation sustained, improved symptoms.
MimiVax	SurVaxM	glioblastoma	Survival benefit and well tolerated.
Bristol-Myers Squibb Co.	pentraxin-2	idiopathic pulmonary fibrosis	Positive treatment effect observed.
Rocket Pharmaceuticals	RP-L102, gene therapy	Fanconi anemia	Restoring bone marrow function.
UPDATED PHASE II RESULTS
[AIT Therapeutics Inc.]	nitric oxide inhalation	respiratory tract infections	Improved six-minute walking distance.
BioMarin Pharmaceutical Inc.	valoctocogene roxaparvovec, gene therapy	hemophilia A	Reducing bleeding, well tolerated.
Spark Therapeutics Inc./ Pfizer Inc.	SPK-9001	hemophilia B	Signs of efficacy.
Kadmon Gene Therapy Holdings Ltd.	KD025	idiopathic pulmonary fibrosis	Well tolerated, signs of efficacy.
FibroGen Inc.	pamrevlumab	idiopathic pulmonary fibrosis	PRAISE; slowed disease progression.
Mereo BioPharma Group PLC	acumapimod	chronic obstructive pulmonary disease	AETHER; signs of efficacy.
Verona Pharma PLC	RPL554	chronic obstructive pulmonary disease	Improved lung function.
Prometic Life Sciences Inc.	PBI-4050	idiopathic pulmonary fibrosis	Promising efficacy.
Roivant Sciences GMBH	vibegron	overactive bladder	Durable efficacy.
Fibrocell Science Inc.	FCX-007, gene therapy	epidermolysis bullosa	Positive efficacy trends, well tolerated.
Abeona Therapeutics Inc.	ABO-102, gene therapy	Sanfilippo A syndrome	Clinical effects observed, well tolerated.
Abeona Therapeutics Inc.	ABO-101, gene therapy	Sanfilippo B syndrome	Signs of efficacy, well tolerated.
uniQure NV	AMT-060, gene therapy	hemophilia B	Clinical benefits observed.
Glenmark Pharmaceuticals Ltd.	GBR-830, anti-OX40 MAb	atopic dermatitis	Clinical effects observed.
GTx Inc.	enobosarm	urinary incontinence	Durable responses.
RXi Pharmaceuticals Corp.	Samcyprone	common warts	Therapeutic responses seen.
PHASE II INITIATION
Biophytis SA	Sarconeos	sarcopenia	SARA-INT; results by end of 2019.
Sotio Group/ Targovax ASA	DCVAC/PCa plus ONCOS-102	prostate cancer	SP015; an active immunotherapy.
bionomics Ltd.	BNC210	generalized anxiety disorder	In the elderly.
Acceleron Pharma Inc.	sotatercept	pulmonary arterial hypertension	PULSAR; a double-blind study.
PanOptica Inc.	PAN-90806	wet age-related acute macular degeneration	A novel eye drop formulation.
XBiotech Inc.	RA-18C3	atopic dermatitis	In moderate to severe disease.
Inovio Pharmaceuticals Inc.	VGX-3100	HPV infection	Related to anal dysplasia.

Source: Informa Pharma Intelligence’s BiomedTracker.

 

 

Marketing Approvals – May 18 to May 24, 2018

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Sun Pharmaceutical Industries Ltd.	-	Yonsa (abiraterone acetate)	prostate cancer	US	For metastatic disease.
Dova Pharmaceuticals Inc.	-	Doptelet (avatrombopag)	thrombocytopenia	US	In patients with chronic liver disease undergoing surgery or dentistry.
AstraZeneca PLC	-	Lokelma (sodium zirconium cyclosilicate)	hyperkalemia	US	In adults.
Sandoz International GMBH	-	Zessly (infliximab)	multiple indications	EU	A biosimilar.
ViiV Healthcare / Janssen Pharmaceutical Cos.	-	Juluca (dolutegravir/ rilpivirine)	HIV infections	EU, Canada	In adults.
SUPPLEMENTAL REGULATORY APPROVAL
Amgen Inc.	-	Prolia (denosumab)	glucocorticoid-induced osteoporosis	US	In mean and women at high risk of fracture.

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.