Pipeline Watch: Phase III Readouts For Talazoparib, Ampion, Avatrombopag

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 Dec. 8 and Dec. 14, 2017, 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 – Dec. 8 to Dec. 14, 2017

Source: Informa Pharma Intelligence’s BiomedTracker.

Lead company/partner	Compound	Indication	Comments
Phase III Results Published
Pfizer Inc.	Trumenba vaccine	meningitis	NEJM, Dec. 14, 2017.
Daiichi Sankyo Co. Ltd.	edoxaban	cancer-associated venous thromboembolism	Hokusai-VTE CANCER; NEJM online, Dec. 12, 2017.
Takeda Pharmaceutical Co. Ltd./Seattle Genetics Inc.	Adcetris (brentuximab vedotin)	Hodgkin lymphoma	Echelon-1; NEJM online, Dec. 10, 2017.
Phase III Completed
Cyclacel Pharmaceuticals Inc.	sapacitabine	acute myeloid leukemia	SEAMLESS; mixed results.
Phase III Interim/Top-line Results
Pfizer Inc.	talazoparib	breast cancer	EMBRACA; PFS extended.
Ampio Pharmaceuticals Inc.	Ampion (LMW human serum albumin fraction)	severe knee osteoarthritis	Met primary endpoint.
Dova Pharmaceuticals	avatrombopag	immune thrombo-cytopenic purpura	Study 302; durable platelet responses.
Glenmark Pharmaceuticals Ltd.	Ryaltris (olopatadine/ mometasone furoate)	allergic rhinitis	Efficacy and safety endpoints met.
Updated Phase III Results
Roche/AbbVie Inc.	Venclexta (venetoclax)	chronic lymphocytic leukemia	MURANO; PFS extended.
Ablynx NV	caplacizumab	thrombotic thromb- ocytopenic purpura	HERCULES; clinically meaningful responses.
Johnson & Johnson/Genmab AS	Darzalex (daratumumab)	multiple myeloma	ALCYONE; improved outcomes.
Emmaus Life Sciences Inc.	Endari (L-glutamine)	sickle cell anemia	Reduced crises.
Geron Corp./[Janssen Biotech NV]	imetelstat	myelodysplastic syndromes	Initial efficacy signs.
Kyowa Hakko Kirin Co. Ltd.	mogamulizumab	cutaneous T-cell lymphoma	MAVORIC; PFS improved, objective responses.
PharmaMar SA	Aplidin (plitidepsin)	multiple myeloma	ADMYRE; PFS and overall survival increased.
Shionogi Inc.	lusutrombopag	thrombocytopenia	Met all endpoints.
Verastem Inc.	duvelisib	chronic lymphocytic leukemia	DUO; improved PFS.
Roche	Hemlibra (emicizumab-kxwh)	hemophilia A with inhibitors	HAVEN 1,2 4; reduced bleeds.
Cytokinetics Inc.	tirasemtiv	amyotrophic lateral sclerosis	VITALITY-ALS; missed primary endpoint.
MediciNova Inc.	ibudilast	amyotrophic lateral sclerosis	Signs of efficacy.
Phase III Initiated
Corbus Pharmaceuticals Holdings Inc.	anabasum	scleroderma	RESOLVE-1; an international study.
Pfizer Inc.	PF-04965842	atopic dermatitis	JADE-Mono-1; a JAK1 inhibitor.
BioMarin Pharmaceutical Inc.	valoctocogene roxaparvovec	hemophila A	GENEr8-1; an open-label study.
Clementia Pharmaceuticals Inc.	palovarotene	fibrodysplasia ossificans progressive	MOVE; a multinational study.
Regeneron Pharmaceuticals Inc./Sanofi	cemiplimab	non-small cell lung cancer	Combined with ipilimumab or chemotherapy.
Phase III Announced
Roche	Tecentriq (atezolizumab)	advanced breast cancer, triple-negative	Impassion132; in early relapsing or recurrent disease.
Sanofi/Alnylam Holding Co.	fitusiran	hemophilia A and B	ATLAS; FDA lifts clinical hold.
Renova Therapeutics	RT-100 AC6 gene therapy	heart failure	FLOURISH; to begin in Q1 2018
Updated Phase II Results
Iovance Biotherapeutics Inc.	Contega (tumor infiltrating lymphocytes)	melanoma	Partial responses.
Valiseek Ltd.	VAL401	non-small cell lung cancer	Improved overall survival.
Alexion Pharmaceuticals Inc.	ALXN1210	paroxysmal nocturnal hemoglobinuria	Reduced hemolysis, well tolerated.
Atara Biotherapeutics Inc.	ATA230	CMV infection	High response rate.
bluebird bio Inc.	LentiGlobin gene therapy	sickle cell disease, beta-thalassemia	Meaningful clinical improvement.
Bristol-Myers Squibb Co.	Opdivo (nivolumab)	Hodgkin's lymphoma	CheckMate 205; stable reductions in tumor burden seen.
Bristol-Myers Squibb Co.	lirilumab	acute myeloid leukemia	EffiKIR; mixed results.
Gilead Sciences Inc.	KTE-C19	acute lymphoblastic leukemia	ZUMA-3; promising efficacy.
GlycoMimetics Inc.	GMI-1271	acute myeloid leukemia	Encouraging efficacy.
Innovation Pharmaceuticals Inc.	brilacidin	oral mucositis	Met primary endpoint.
MorphoSys AG	MOR208	diffuse large B-cell lymphoma	L-MIND; signs of efficacy, well tolerated.
Novartis AG	Kymriah (tisagenlecleucel)	diffuse large B-cell lymphoma	JULIET; high response rates.
Spark Therapeutics Inc./Pfizer Inc.	SPK-9001	hemophilia B	GOLD-B; clinical responses seen.
Proteostasis Therapeutics Inc.	PTI-428	cystic fibrosis	Positive results observed.
Spark Therapeutics Inc.	SPK-8011	hemophilia A	Initial signs of clinical responses.
uniQure NV	AMT-060	hemophilia B	Bleeding reduced.
Acceleron Pharma Inc.	luspatercept	myelodysplastic syndrome	PACE-MDS; responses seen.
Actinium Pharmaceuticals Inc.	Actimab-A (lintuzumab labelled with Ac-225	acute myeloid leukemia	Initial signs of efficacy.
Agios Pharmaceuticals Inc.	AG-348	pyruvate kinase deficiency	Hemoglobin levels increased.
Immune Design Corp.	G100 plus low-dose radiation and pembrolizumab	non-Hodgkin's lymphoma	Robust responses observed.
Incyte Corp.	INCB50465	diffuse B-cell lymphoma	CITADEL-101; clinical responses seen.
Kadmon Corp. LLC	KD025	graft-vs-host disease	Encouraging clinical responses.
Syros Pharmaceuticals Inc.	SY-1425	myelodysplastic syndrome	Shows clinical activity.
BioMarin Pharmaceutical Inc.	valoctocogene roxaparvovec	hemophilia A	Sustained activity.
Global Blood Therapeutics Inc.	voxelotor	sickle cell disease	HOPE-KIDS 1; well tolerated.
Stemline Therapeutics Inc.	SL-401	blastic plasmacytoid dendritic cell neoplasm	Clinical responses seen.
Phase II Interim/Top-line Results
argenx SE	ARGX-113	myasthenia gravis	Clinical responses.
UCB SA	bimekizumab	ankylosing spondylitis	BE AGILE; efficacy noted, well tolerated.
Regeneron Pharmaceuticals Inc./Sanofi	cemiplimab, a PD-1 inhibitor	cutaneous squamous cell carcinoma	EMPOWER-CSCC1; responses seen, rolling BLA started.
AbbVie Inc.	Imbruvica (ibrutinib) plus nivolumab	indolent non-Hodgkin lymphoma, chronic lymphocytic leukemia	Well tolerated, good responses seen.
argenx SE	ARGX-110	acute myeloid leukemia, cutaneous T-cell lymphoma	Signs of clinical efficacy.
Atara Biotherapeutics Inc.	tabelecleucel (ATA-129)	EBV-associated blood cancers	Clinical responses observed.
Bristol-Myers Squibb Co.	Sprycel (dasatinib)	acute lymphoblastic leukemia, pediatric	Survival benefit reported.
Agios Pharmaceuticals Inc.	enasidenib, ivosidenib	acute myeloid leukemia	Preliminary efficacy combined with azacitidine.
Incyte Corp.	INCB057643	advanced cancers	Initial efficacy signals, well tolerated.
Incyte Corp.	INCB52793	advanced cancer	Preliminary signs of efficacy.
Karyopharm Therapeutics Inc.	eltanexor	multiple myeloma	Well tolerated, promising activity.
Magenta Therapeutics Inc.	MGTA-456, expanded cord blood stem cells	hematologic cancers	Rapid and durable engraftment.
Mereo BioPharma Group Ltd.	acumapimod (BCT-197)	chronic obstructive pulmonary disease	AETHER; primary endpoint met.
Ionis Pharmaceuticals Inc./Roche	IONIS-HTTRx	Huntington's disease	Signs of efficacy, well tolerated.
TG Therapeutics Inc.	umbralisib (TGR-1202)	chronic lymphocytic leukemia	Initial signs of efficacy.
AstraZeneca PLC	Calquence (acalabrutinib)	mantle cell lymphoma	Durable clinical responses.
Curis Inc.	CUDC-907	diffuse large B-cell lymphoma	Clinical responses seen.
Karyopharm Therapeutics Inc.	selinexor	multiple myeloma	Promising activity.
Oncopeptides AB	Ygalo (melflufen)	multiple myeloma	HORIZON; responses seen.
X4 Pharmaceuticals Inc.	X4P-001-RD	immunodeficiency	Promising activity.
Kura Oncology Inc.	tipifarnib	peripheral T-cell lymphoma	Encouraging activity.
Merck & Co. Inc.	Keytruda (pembrolizumab)	mediastinal large B-cell lymphoma	KEYNOTE-170; antitumor activity shown.
Phase II Initiation
Constellation Pharmaceuticals Inc.	CPI-1205	prostate cancer	PROSTAR; with enzalutamide or abiraterone.
TapImmune Inc.	TPIV 200	breast cancer	A therapeutic vaccine.
Realm Therapeutics Inc	PRO13	allergic conjunctivitis	Topical eye drops.

Marketing Approvals – Dec. 8 to Dec. 14, 2017

Source: Biomedtracker.

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Pfizer Inc.	-	Ixifi (infliximab-qbtx)	rheumatoid arthritis, Crohn's disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis, plaque psoriasis	US	A biosimilar to Remicade.
Medimetriks Pharmaceuticals Inc.	Grupo Ferrer Internacional SA	Xepi (ozenoxacin)	impetigo	US	A cream formulation.
Sanofi	-	Admelog (insulin lispro)	diabetes, type 1 and 2	US	A follow-on product.
SUPPLEMENTAL REGULATORY APPROVAL
Bayer AG	-	Stivarga (regorafenib)	liver cancer, second line	China	Already approved for GI cancers.
Eisai Co. Ltd.	-	Aricept (donepezil)	severe Alzheimer's disease	China	Already available for mild to moderate disease.
Shire PLC	-	Oncaspar (pegaspargase)	acute lymphoblastic leukemia	EU	A lyophilized formulation.
GlaxoSmithKline PLC	-	Nucala (mepolizumab)	Churg-Strauss syndrome	US	Based on the MIRRA study.

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 .