Pipeline Watch: Kisqali, SI-6603, Cosentyx Phase III Readouts

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 more 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 Nov. 3 and Nov. 9, 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 – Nov. 3 to Nov. 9, 2017

Source: Informa Pharma Intelligence’s BiomedTracker.

Lead company/partner	Compound	Indication	Comments
Phase III Results Published
Otsuka Pharmaceutical Co. Ltd.	tolvaptan	polycystic kidney disease, autosomal dominant	REPRISE; NEJM online, Nov. 4, 2017.
Sanofi/Drugs for Neglected Diseases initiative	fexinidazole	human African trypanosomiasis	The Lancet online, Nov. 4, 2017.
Vertex Pharmaceuticals Inc.	VX-661 plus ivacaftor	cystic fibrosis	EVOLVE, EXPAND; NEJM online, Nov. 3, 2017.
Phase III Interim/Top-line Results
Novartis AG	Kisqali (ribociclib)	advanced breast cancer, premenopausal	MONALEESA-7; PFS improved in HR+/HER2- disease.
Otonomy Inc.	Otividex (dexamethasone)	Meniere's disease	AVERTS-2; achieved primary endpoint.
Seikagaku Corp./Ferring BV	SI-6603 (condoliase)	lumbar disc herniation	Missed primary endpoint.
Novartis AG	Cosentyx (secukinumab)	psoriatic arthritis	FUTURE 5; reduced symptoms, joint damage.
TaiGen Biotechnology Co. Ltd.	Taigexyn (nemonoxacin), iv	pneumonia, community-acquired	Met co-primary endpoints.
Updated Phase III Results
Eli Lilly & Co.	Taltz (ixekizumab)	psoriatic arthritis, psoriasis	SPIRIT-P2, IXORA-S; sustained improvement.
AbbVie Inc.	upadacitinib	rheumatoid arthritis	SELECT-BEYOND, -NEXT; symptoms improved.
AMAG Pharmaceuticals Inc.	Feraheme (ferumoxytol)	iron-deficiency anemia	FIRM; reduction in hypophosphatemia.
UCB SA	Evenity (romosozumab) then denosumab	post-menopausal osteoporosis	FRAME Ext; reduced fracture risk.
Boehringer Ingelheim GMBH	Cyltezo (adalimumab-adbm) biosimilar	rheumatoid arthritis, switch study	VOLTAIRE-RA; efficacy similar to Humira.
Celltrion Inc.	CT-P10 (rituximab) biosimilar	rheumatoid arthritis, switch study	Comparable to reference rituximab.
Bristol-Myers Squibb Co.	Opdivo (nivolumab)	renal cell carcinoma	CheckMate 025; durable overall survival rate at 3 years.
Alexion Pharmaceuticals Inc.	Kanuma (sebelipase alfa)	lysosomal acid lipase deficiency	Infants survived beyond one year of age.
Kyowa Hakko Kirin Co. Ltd.	evocalcet	secondary hyper-parathyroidism	Non-inferior to cinacalcet.
Ardelyx Inc.	tenapanor	hyperphosphatemia	Decreased potassium, well tolerated.
Clovis Oncology Inc.	Rubraca (rucaparib)	ovarian cancer	ARIEL3; Improved PFS.
Phase III Initiated
Sanofi	Praluent (alirocumab)	homozygous familial hypercholesterol- emia	In patient aged more than 12 years.
Zosano Pharma Corp.	M207 (zolmitriptan)	migraine	A long-term safety study.
Alnylam Holding Co.	givosiran	acute hepatic porphyria	ENVISION; in more than 20 countries.
Esperion Therapeutics Inc.	bempedoic acid/ ezetimibe	hypercholesterolemia and atherosclerosis	A non-statin therapy.
Pharmacosmos AS	Monofer (iron isomaltoside)	hypophosphatemia induced by iron therapy	PHOSHARE; versus Injectafer /Ferinject.
Stealthyx Therapeutics Ltd.	elamipretide	primary mitochondrial myopathy	MMPOWER-3; daily sc injections.
Sequential Medicine Ltd.	SM-1 (zolpidem, lorazepam, diphenhydramine)	transient insomnia	A randomized study.
Phase III Announced
Novartis AG/Amgen Inc.	erenumab	migraine	EMPOwER; once monthly sc dosing.
Strekin AG	STR001	sudden hearing loss	In adults.
Odonate Therapeutics llc	tesetaxel plus capecitabine	HR+/HER2- advanced breast cancer	CONTESSA; in patients previously taxane treated.
Dova Pharmaceuticals Inc.	avatrombopag	thrombocytopenia	Open label study.
Updated Phase II Results
OncoSec Medical Inc.	ImmunoPulse IL-12 plus pembrolizumab	melanoma	Robust responses, tumors became "hot".
Cara Therapeutics Inc.	CR845	arthritis pain, pruritus	Pain and itch reduced.
Corbus Pharmaceuticals Holdings Inc.	resunab	dermatomyositis, scleroderma	Clinical benefits observed.
Immune Design Corp.	CMB305 plus atezolizumab	sarcoma	Greater clinical benefit with combo.
Johnson & Johnson	Tremfya (guselkumab)	psoriatic arthritis	Benefits on joint symptoms.
OrthoTrophix Inc.	TPX-100	osteoarthritis, knee	Sustained knee function improved.
Selecta Biosciences Inc.	SEL-212	gout	Reduced flare rate, well tolerated.
Centrexion Therapeutics Corp.	CNTX-4975	osteoarthritis, knee	TRIUMPH; improved knee function.
Galapagos NV	filgotinib	rheumatoid arthritis	DARWIN3; durable responses.
Merck KGAA/Bristol-Myers Squibb Co.	atacicept	systemic lupus erythematosus	ADDRESS II; signs of efficacy.
Quest PharmaTech Inc.	oregovomab	ovarian cancer	Signs of clinical benefit.
Samumed	SM04690	osteoarthritis, knee	Evidence of cartilage regeneration.
Cognition Therapeutics Inc.	CT1812	Alzheimer's disease	Encouraging effects on biomarkers.
Omeros Corp.	OMS721	IgA nephropathy	Clinical improvements recorded.
Pharnext SA	PXT864	Alzheimer's disease	PLEODIAL-II; stabilized cognition disability.
Reata Pharmaceuticals Inc./Kyowa Hakko Kirin Co. Ltd.	bardoxolone methyl	Alport syndrome, chronic kidney disease	CARDINAL, TSUBAKI; met primary endpoint.
Acadia Pharmaceuticals Inc.	pimavanserin	Alzheimer's disease psychosis	Improved psychosis symptoms.
Alnylam Holding Co.	lumasiran	primary hyperoxaluria type 1	Well tolerated, signs of benefit.
ChemoCentryx Inc.	avacopan	ANCA-associated vasculitis	CLEAR; reduced kidney inflammation.
Retrophin Inc.	sparsentan	focal segmental glomerulosclerosis	DUET; sustained improvement, well tolerated.
Quark Pharmaceuticals Inc.	QPI-1002	acute kidney failure after cardiac surgery	Positive clinical results.
SAGE Therapeutics Inc.	SAGE-217	Parkinson's disease	Improved tremor, well tolerated.
Phase II Interim/Top-line Results
Millendo Therapeutics Inc.	ATR-101	congenital adrenal hyperplasia	Well tolerated, promising efficacy.
AbbVie Inc./Boehringer Ingelheim GMBH	risankizumab	psoriatic arthritis	Positive new results.
Calithera Biosciences Inc.	CB-839 plus nivolumab	melanoma, NSCLC, renal cell carcinoma	Some durable responses, well tolerated.
Centrexion Therapeutics Corp.	CNTX-4975 (capsaicin)	neuropathic pain	Rapid and prolonged pain relief.
Pique Therapeutics Inc.	PT-107 (allogeneic whole cell vaccine)	NSCLC	Improved median overall survival.
Flex Pharma Inc.	FLX-787	amyotrophic lateral sclerosis	Reduced cramping and pain.
Johnson & Johnson /Bristol-Myers Squibb Co.	Stelara (ustekinumab)	active lupus	Signs of efficacy.
Merck KGAA	sprifermin	osteoarthritis	FORWARD; met primary endpoint.
AbbVie Inc.	upadacitinib	rheumatoid arthritis	BALANCE-EXTEND; no unexpected safety signals.
Phase II Initiation
BeiGene (Beijing) Co. Ltd.	BGB-3111	indolent non-Hodgkin's lymphoma	An international open-label study.
Oncoceutics Inc.	ONC201	brain cancer, histone-mutant	In patients with the histone H3 K27M mutation.
Horama SAS	HORA-PDE6b, gene therapy	retinitis pigmentosa	By subretinal administration.

Marketing Approvals – Nov. 3 to Nov. 9, 2017

Source: Biomedtracker.

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Norgine BV	-	Plenvu (NER1006)	bowel cleansing	Belgium, Denmark	Prior to bowel procedures.
SUPPLEMENTAL REGULATORY APPROVAL
Genentech Inc.	-	Alecensa (alectinib)	ALK-positive metastatic NSCLC as detected by an FDA approved test	US	As first line therapy.
Otsuka Holdings Co. Ltd.	Lundbeck	Abilify Maintena (aripiprazole) once monthly	bipolar I disorder	Canada	Maintenance monotherapy.
Keryx Biopharmaceuticals Inc.	-	Auryxia (ferric citrate)	iron deficiency anemia in patients requiring dialysis	US	In patients with chronic kidney disease.
Roche	-	Zelboraf (vemurafenib)	Erdheim-Chester disease	US	In patients with BRAF V600 mutation.
UCB SA	-	Vimpat (lacosamide)	partial onset seizures in pediatric patients	US	As oral formulations.

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 .