Pipeline Watch: Phase III Misses For Fovista, Istradefylline And Vonapanitase

Pipeline Watch has been updated to bring greater depth and detail to a potential therapeutic product’s progress through the R&D pipeline. The table brings you a snapshot of selected late-stage clinical trial events in the pharma and biotech industries.

The table records clinical developments using data from Informa Pharma Intelligence’s Biomedtracker. It lists drugs for which an important event was recorded between Dec. 9 and Dec. 15, 2016, divided by event type.

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

A companion resource, Pink Sheet’s regulatory-focused FDA Performance Tracker, follows regulatory milestones such as new submissions and user fee calculations, complete response letters and breakthrough therapy designations.

Pipeline Watch – Dec. 9 to Dec.15, 2016

Source: Informa Pharma Intelligence’s Biomedtracker.

Lead company/partner	Compound	Indication	Comments
Phase III Suspended
Ophthotech Corp.	Fovista (pegpleranib) added to ranibizumab	wet age-related macular degeneration	OPH1002, 1003; primary endpoints not met.
Phase III Results
Astellas Pharma Inc./Pfizer Inc.	Xtandi (enzalutamide)	metastatic castration-resistant prostate cancer	Phase IV PLATO study; when added to abiraterone and prednisone, Xtandi did not meet primary endpoint.
Phase III Interim/Top-line Results
Kyowa Hakko Kirin Co. Ltd.	istradefylline (KW-6002)	Parkinson’s disease	The adenosine A2A receptor antagonist did not meet its primary endpoint.
Proteon Therapeutics Inc.	vonapanitase	chronic kidney disease	PATENCY-1; missed primary efficacy endpoint, positive secondary endpoints.
Achaogen Inc.	plazomicin	complicated urinary tract infections, serious carbapenem-resistant infections (CREs)	EPIC, CARE; met primary endpoints in UTIs, and mortality reduction favored plazomicin in CREs.
Synergy Pharmaceuticals Inc./Ironwood Pharmaceuticals Inc.	plecanatide	irritable bowel syndrome with constipation	IBS-C; met its primary endpoint.
Phase III Initiated
BioMarin Pharmaceutical Inc./Chugai Pharmaceutical Co. Ltd.	vosoritide	achondroplasia	An analog of C-type natriuretic peptide, evaluated in children.
Shanghai Henlius Biotech	trastuzumab biosimilar (HLX02)	breast cancer	Taking place in China.
Phase III Announced
Atara Biotherapeutics Inc.	ATA-129 (allogeneic EBV-specific cytotoxic T-lymphocytes)	posttransplant lymphoproliferative disorder	MATCH, ALLELE; after hematopoetic cell or solid organ transplants.
Phase II Suspended
Genticel SA	ProCervix (GTL001)	HPV16/18 infected women	Efficacy similar to placebo; program ended.
BioDelivery Sciences International Inc.	clonidine gel	painful diabetic neuropathy	Efficacy against pain relief similar to placebo.
Phase II Results
Axovant Sciences Ltd.	intepirdine	Alzheimer’s disease	New analysis suggests reduced progression.
Phase II Interim/Top-line Results
Pulmagen Therapeutics (Asthma) Ltd./Teijin Pharma Ltd.	PTR-36 (ADC3680)	eosinophilic asthma	An oral CRTh2 antagonist, showed initial signs of efficacy.
Centrexion Therapeutics Corp.	CNTX-4975	Pain associated with knee osteoarthritis	Positive results against primary and secondary endpoints.
RXi Pharmaceuticals Corp./Hapten Pharmaceuticals	Samcyprone (diphenylcyclopropenone)	cutaneous warts	60% of patients had clearance of warts.
aTyr Pharma Inc.	Resolaris (a natural muscle homeostasis protein)	limb girdle muscular dystrophy	Signals of improved muscle strength, well tolerated.
Revance Therapeutics Inc.	daxibotulinumtoxinA	cervical dystonia	Positive effects on dystonia symptoms.
Pharnext SAS	PXT864	Alzheimer’s disease	PLEODIAL-I, II; promising signs of efficacy.
Eiger BioPharmaceuticals	exendin 9-39	post-bariatric hypoglycemia	Reduced magnitude of hypoglycemia, well tolerated.
Eisai Co. Ltd./Merck & Co. Inc.	Halaven (eribulin) with Keytruda (pembrolizumab)	triple negative breast cancer	KEYNOTE-150; some complete and partial responses.
Orphazyme APS	arimoclomol	amyotrophic lateral sclerosis	Preliminary evidence of efficacy.
OrthoTrophix	TPX-100 (23-amino acid peptide)	knee osteoarthritis	Sustained benefits in knee function.
Phase II Initiation
Galena Biopharma Inc.	NeuVax (nelipepimut-S)	breast cancer (ductal carcinoma in situ)	VADIS; to evaluate any immune response.
MorphoSys AG	MOR208 plus idelalisib	chronic lymphocytic leukemia	COSMOS; in relapsed or refractory disease.
SAGE Therapeutics Inc.	SAGE-217	Parkinson’s disease, essential tremor	Will also be evaluated in mood disorders.
Zealand Pharma AS	ZP4207 (dasiglucagon)	type 1 diabetes	Used in a dual hormone artificial pancreas system.

Marketing Approvals – Dec. 9 to Dec. 15, 2016

Source: Biomedtracker.

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Pfizer Inc.	-	Eucrisa (crisaborole) ointment	mild to moderate eczema	US	A topical twice-daily PDE4 inhibitor.
CSL Behring		Afstyla (Factor VIII,rDNA)	hemophilia A	Canada	In adults and children.
Eli Lilly & Co.	Boehringer Ingelheim GMBH	Synjardy XR (empagliflozin plus metformin) tablets	type 2 diabetes	US	Extended-release tablets.
Vericel Corp.	-	MACI (autologous chrondrocytes on porcine collagen membrane)	cartilage defects of the knee	US	First FDA approved scaffold product.
Ferring BV	Roche	Rekovelle (follitropin delta)	assisted reproduction	EU	Dosing regimen is individualized.
Gilead Sciences Inc.	-	Descovy (emtricitabine, tenofovir alafenamide)	HIV/AIDS	Japan	In two strengths.
Biogen	AbbVie Inc.	Zinbryta (daclizumab)	active relapsing multiple sclerosis	Canada	A once-monthly injection.
SUPPLEMENTAL REGULATORY APPROVAL
Novartis AG	Genmab AS	Arzerra (ofatumumab)	chronic lymphocytic leukemia	EU	In combination with fludarabine and cyclophosphamide.
ACCELERATED/CONDITIONAL APPROVAL
Intercept Pharmaceuticals Inc.	-	Ocaliva (obeticholic acid)	primary biliary cholangitis	EU	Already approved in the US.

Data is 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.