Pipeline Watch: Risankizumab, Viaskin Peanut, Symtuza 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 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 Oct. 20 and Oct. 26, 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 – Oct. 20 and Oct. 26, 2017

Source: Informa Pharma Intelligence’s BiomedTracker.

Lead company/partner	Compound	Indication	Comments
Phase III Results Published
Melinta Therapeutics Inc.	Baxdela (delafloxacin)	skin and skin structure infections	PROCEED; Journal of Antimicrobial Chemotherapy, Oct. 5, 2017.
Alexion Pharmaceuticals Inc.	Soliris (eculizumab)	myasthenia gravis	REGAIN; The Lancet Neurology, Oct. 20, 2017.
Updated Phase III Results
MedDay Pharmaceuticals	Qizenday (biotin, high-dose)	multiple sclerosis, progressive	Decreased whole brain and grey matter volumes.
Novartis AG	Gilenya (fingolimod)	multiple sclerosis	PARADIGMS; reduced relapses in children.
Novartis AG	siponimod	multiple sclerosis, secondary progressive	EXPAND; reduced MRI activity, slowed brain volume loss.
Trevena Inc.	Olinvo (olicerdine)	acute pain	APOLLO-1,2; reduced pain.
Shionogi Inc.	lusutrombopag	thrombocytopenia	L-Plus 2; met primary endpoint.
Boehringer Ingelheim GMBH	nintedanib	idiopathic pulmonary fibrosis	IMPULSIS-ON Ext.; durable long-term efficacy.
Dova Pharmaceuticals Inc.	avatrombopag	thrombocytopenia associated with chronic liver disease	ADAPT 1, 2; well tolerated and effective.
Phase III Interim/Top-line Results
AbbVie Inc.	risankizumab	psoriasis	ultIMMa-1, -2, IMMvent; met all co-primary endpoints .
DBV Technologies SA	Viaskin Peanut	peanut allergy	PEPITES; narrowly missed primary endpoint, showed clinical promise.
Johnson & Johnson	Symtuza (darunavir, cobicistat, tenofovir alafenamide and emtricitabine)	HIV/AIDS	AMBER; effective as one tablet once-daily.
Vertex Pharmaceuticals Inc.	Orkambi (lumacaftor/ ivacaftor)	cystic fibrosis, with 2 copies of F508del mutation	Met primary endpoint, well tolerated.
Vertex Pharmaceuticals Inc.	VX-661	cystic fibrosis	Plus ivacaftor, mixed results.
Amgen Inc.	Kyprolis (carfilzomib) once weekly with dexamethasone	multiple myeloma, relapsed and refractory	A.R.R.O.W.; better efficacy than twice weekly.
Evoke Pharma Inc.	Gimoti (metoclopramide) nasal	gastroparesis, diabetic	Similar systemic exposure to reference drug.
Phase III Initiated
Horizon Pharma PLC	teprotumumab	Graves' ophthalmopathy	OPTIC; in patients with active thyroid eye disease.
United Therapeutics Corp.	Aurora-GT, cell therapy	pulmonary arterial hypertension	SAPPHIRE; transfected cells to rebuild lung tissue.
BeyondSpring Inc.	plinabulin	chemotherapy induced neutropenia	Taking place in China.
Tricida Inc.	TRC101	metabolic acidosis	In chronic kidney disease.
Phase III Announced
Alkermes PLC	Aristada (aripiprazole lauroxil)	schizophrenia	As a two-monthly injection.
Phase II Suspended
Global Blood Therapeutics Inc.	voxelotor (GBT440)	idiopathic pulmonary fibrosis	Insufficient efficacy; trials continue in sickle cell disease.
Updated Phase II Results
Omeros Corp.	OMS721	hemolytic uremic syndrome	Symptoms resolved.
Spring Bank Pharmaceuticals Inc.	inarigivir soproxil	hepatitis B	ACHIEVE; active antiviral with novel action.
Intercept Pharmaceuticals Inc.	Ocaliva (obeticholic acid)	primary sclerosing cholangitis	AESOP; efficacy encouraging.
Replicor Inc.	REP 2139 Mg	hepatitis B	REP 401; infection controlled.
NGM Biopharmaceuticals Inc.	NGM282	non-alcoholic steatohepatitis (NASH)	Reduces hepatic steatosis.
TG Therapeutics Inc.	ublituximab	multiple sclerosis	Rapid and robust B cell depletion and clinical stability.
CymaBay Therapeutics Inc.	seladelpar	primary biliary cholangitis	Well tolerated, potent anti-cholestatic effects.
Arbutus Biopharma Corp.	ARB-1467	hepatitis B	Reduced HBV surface antigen.
Progenics Pharmaceuticals Inc.	Azedra (iobenguane I-131)	neuroendocrine tumors	Tumor responses seen.
Ocera Therapeutics Inc.	ornithine phenylacetate (OCR-002)	hepatic encephalopathy	STOP-HE; clinical benefits observed.
Phase II Completed
Albireo Pharma Inc.	A4250	primary biliary cholangitis and hepatic fibrosis	Reduces pruritus, serum bile acid in children.
Phase II Interim/Top-line Results
MediciNova Inc.	MN-166 (ibudilast)	multiple sclerosis, progressive	SPRINT-MS; positive data.
Vaxart Inc.	H1N1 flu oral tablet	seasonal flu	Positive efficacy, well tolerated.
Vertex Pharmaceuticals Inc./Parion Sciences Inc.	VX-371	cystic fibrosis	Well tolerated, mixed results.
Brickell Biotech Inc.	sofpironium bromide (BBI-4000)	axillary hyperhidrosis	Positive results.
Gilead Sciences Inc.	GS-0976	NASH	Reduced hepatic steatosis, well tolerated.
Ergomed PLC	PeproStat (HXSA902)	surgical bleeding	Met primary efficacy endpoint, well tolerated.
Achillion Pharmaceuticals Inc./Johnson & Johnson	odalasvir, AL-335, simeprevir (JNJ-4178)	hepatitis C	OMEGA-1; well tolerated and effective.
Albireo Pharma Inc.	A4250	primary biliary cholangitis	Improved pruritus.
Myr GmbH	Myrcludex B	hepatitis D	Reduced viral titers, well tolerated.
Eiger BioPharmaceuticals Inc.	PEG-interferon lambda	hepatitis D	Encouraging efficacy signs, well tolerated.
Bayer AG	vilaprisan	uterine fibroids	ASTERIOD 2; clinical benefits, well tolerated.
Phase II Initiation
Menlo Therapeutics Inc.	serlopitant	chronic cough	TUSSIX; in refractory disease.
Apexigen Inc./Boehringer Ingelheim GMBH	APX005M	esophageal cancer	A CD40 agonistic antibody.
Neurocrine Biosciences Inc.	Ingrezza (valbenazine)	Tourette syndrome	T-Force Gold, results in late 2018.
Biogen	Tysabri (natalizumab)	drug resistant focal epilepsy	OPUS; as adjunct therapy.
Sienna biopharma	pegcantratinib	pruritus	A topical product.

Marketing Approvals – Oct. 20 to Oct. 26, 2017

Source: Biomedtracker.

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
GlaxoSmithKline PLC	-	Shingrix vaccine	chickenpox, shingles prophylaxis	US	In adults aged 50 years and older.
Norgine Pharma	-	Plenvu (polymers)	bowel cleansing	Iceland, UK	In adults.
Swedish Orphan Biovitrum AB	Bioverativ Inc.	Alprolix (eftrenona-cog-alfa)	hemophilia B	Saudi Arabia	In all age groups.
Swedish Orphan Biovitrum AB	-	Orfadin (nitisinone)	tyrosinemia	Algeria, Tunisia	First in North Africa.
SUPPLEMENTAL REGULATORY APPROVAL
Janssen Biotech Inc.	-	Simponi Aria (golimumab)	psoriatic arthritis, ankylosing spondylitis	US	Already available for rheumatoid arthritis.
AstraZeneca PLC	-	Bydureon (exenatide) BCise	diabetes, type 2	US	Once weekly single dose autoinjector.
Merz GMBH & Co. KGAA	-	Xeomin (inocobotul- iniumtoxinA	certain foot deformities in cerebral palsy	Russia	Based on trials in Russia.
Shire PLC	-	Firazyr (icatibant)	hereditary angioedema	EU	In children aged 2 years and older.
Tesaro Inc.	Opko Health Inc.	Varubi (rolapitant) IV	preventing delayed nausea and vomiting	US	Associated with cancer chemo. An oral version already available.
Alexion Pharmaceuticals Inc.	-	Soliris (eculizumab)	generalized myasthenia gravis in AChR-antibody positive	US	Already approved in other indications.

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 .