Pipeline Watch: Phase III Starts For BL-8040, Trigriluzole, MIN-101

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. 15 and Dec. 21, 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. 15 to Dec. 21, 2017

Source: Informa Pharma Intelligence’s BiomedTracker.

Lead company/partner	Compound	Indication	Comments
Phase III Results Published
Motif Bio PLC	iclaprim (iv)	skin and skin structure infections	REVIVE-1; Clinical Infectious Diseases, Dec. 21, 2017.
Gilead Sciences Inc.	momelotinib	myelofibrosis	Simplify 2; The Lancet Hematology, Dec. 20, 2017.
Roche	Tecentriq (atezolizumab)	bladder cancer	IMvigor211; The Lancet, Dec. 18, 2017.
Allergan PLC	Avycaz (ceftazidime plus avibactam)	hospital acquired pneumonia	REPROVE; The Lancet, Dec. 15, 2017.
AstraZeneca PLC	AZD8931	colorectal cancer	FOCUS4-D; The Lancet Gastrotenterology & Hepatology, Dec. 15, 2017.
Phase III Interim/Top-line Results
AbbVie Inc.	upadacitinib, an oral JAK1 selective inhibitor	rheumatoid arthritis	SELECT-MONOTHERAPY; met all endpoints.
Gedeon Richter PLC/Allergan PLC	Vraylar (cariprazine)	bipolar I depression	RGH-MD-54; positive results.
Merck & Co. Inc.	Keytruda (pembrolizumab)	gastric cancer as second-line therapy	KEYNOTE-061; missed primary endpoint.
Shire PLC	SHP609 (idursulfase) by intrathecal administration	Hunter syndrome and cognitive impairment in children	Missed endpoints; no impact on iv use in current Hunter syndrome indications.
Phase III Initiated
BioLineRx Ltd.	BL-8040	mobilizing stem cells in multiple myeloma	GENESIS; in addition to G-CSF in transplant patients.
Biohaven Pharmaceuticals Holding Co. Ltd.	trigriluzole	obsessive-compulsive disorder	At 35 sites in the US.
Minerva Neurosciences Inc.	MIN-101	schizophrenia	For negative symptoms.
Incyte Corp.	epacadostat	renal cell cancer	KEYNOTE-679/ECHO-302; as first line therapy.
Novo Nordisk AS	Tresiba (insulin degludec)	type 1 diabetes	EXPECT; in pregnant women.
VBI Vaccines Inc.	Sci-B-Vac vaccine	hepatitis B	PROT ECT, CONSTANT; a third-generation product.
Phase III Announced
Verrica Pharmaceuticals Inc.	VP-102	molluscum contagiosum	A double-blind study.
Apellis Pharmaceuticals Inc.	APL-2	geographic atrophy	Associated with age-related macular degeneration.
Eyenovia Inc.	phenylephrine plus tropicamide	mydriasis	MicroStat; to dilate pupils.
Eyenovia Inc.	MicroProst (Gla203)	glaucoma	a micro-formulation of a prostaglandin.
Updated Phase II Results
Abivax	ABX464	HIV/AIDS	ABX464-005; HIV reservoir reduction observed.
Ascend Biopharmaceuticals Ltd.	ASN-002, interferon- gamma gene therapy	nodular basal cell carcinoma	Clinical responses seen.
Phase II Completed
RXi Pharmaceuticals Corp.	RXI-109, a self-delivering RNAi	wound healing	Improved scar appearance, well tolerated.
Phase II Interim/Top-line Results
UCB SA	bimekizumab	psoriatic arthritis	BE ACTIVE; skin and joint symptoms improved.
Galera Therapeutics Inc.	GC4419, a dismutase mimetic	severe oral mucositis in head and neck cancer	Reduced symptoms.
Phase II Initiation
Gemphire Therapeutics Inc.	gemcabene	non-alcoholic steatohepatitis (NASH)	A proof of concept study.
Ironwood Pharmaceuticals Inc.	IW-1701	sickle cell anemia	STRONG-SCD; once daily use.
Minerva Neurosciences Inc.	seltorexant	major depression	Versus quetiapine.
AiVita Biomedical	CLBS20	ovarian cancer	An immunotherapy.
Aclaris Therapeutics Inc.	ATI-50002	vitiligo	A JAK inhibitor.
Insys Therapeutics Inc.	cannabidiol	childhood absence epilepsy	An oral solution.
La Jolla Pharmaceutical Co.	LJPC-401	hemochromatosis	In hereditary disease.
Teva Pharmaceutical Industries Ltd.	fremanezumab	post-traumatic headache	With persistent headache.

Marketing Approvals – Dec. 15 to Dec. 21, 2017

Source: Biomedtracker.

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Spark Therapeutics Inc.	-	Luxturna (voretigene neparvovec-rzyl)	Leber's congenital amaurosis	US	For adults and children.
La Jolla Pharmaceutical Co.	-	Giapreza (angiotensin II)	hypotension/ septic shock	US	By iv infusion.
AEterna Zentaris Inc.	-	Macrilen (macimorelin)	adult growth hormone deficiency	US	a diagnostic.
Carmel Biosciences Inc.	Medicure Inc.	Prexxartan (valsartan)	congestive heart failure	US	First oral liquid form.
Aerie Pharmaceuticals Inc.	-	Rhopressa (netarsudil)	glaucoma	US	Ophthalmic solution .
Aclaris Therapeutics Inc.	-	Eskata (hydrogen peroxide)	raised seborrheic keratoses	US	Topical solution.
SUPPLEMENTAL REGULATORY APPROVAL
Roche	-	Alecensa (alectinib)	non-small cell lung cancer	EU	In ALK-positive disease.
Bristol-Myers Squibb Co.	-	Opdivo (nivolumab)	adjuvant therapy in completely resected melanoma	US	In patients with lymph node involvement or metastatic disease.
Roche	-	Perjeta (pertuzumab) plus trastuzumab and chemo	adjuvant therapy of HER2-positive early breast cancer	US	In patients at high risk of recurrence .
Exelixis Inc.	Ipsen	Cabometyx (cabozantinib)	renal cell carcinoma, first-line	US	In previously untreated advanced disease.
Portola Pharmaceuticals Inc.	-	Bevyxxa (betrixaban)	venous thromboembolism	US	A single-drug regimen.
Pfizer Inc.	Avillion	Bosulif (bosutinib)	chronic myeloid leukemia, first--line	US	In Ph-positive disease as an accelerated review.

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 .