Pipeline Watch: Phase III Starts With STR001, Cabometyx And Brazikumab

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. 5 and Oct. 11, 2018, divided by event type.

Events can include Phase II and Phase III clinical 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. 5 to Oct. 11, 2018

Lead company/partner	Compound	Indication	Comments
PHASE III RESULTS PUBLISHED
Boehringer Ingelheim/ Eli Lilly	Jardiance (empagliflozin)	type 2 diabetes and cardiovascular disease	EMPA-REG OUTCOME; Circulation, Oct. 8, 2018.
Bausch Health Companies	Altreno (tretinoin) lotion, 0.05%	moderate-to-severe acne	Journal of Drugs in Dermatology, Oct. 2018.
PHASE III INTERIM/TOP-LINE RESULTS
Johnson & Johnson	Stelara (ustekinumab)	ulcerative colitis	Clinical remission with single iv dose.
Roche Holding AG	Ocrevus (ocrelizumab)	relapsing multiple sclerosis	CHORDS; safe and effective after switch from another disease modifying therapy.
Celgene Corp.	Otezla (apremilast)	scalp psoriasis	STYLE; met primary endpoint.
Venus Remedies Ltd.	Elores (ceftriaxone/ sulbactam/EDTA)	urinary tract infections	PLEA; higher cure rates vs meropenem.
Diurnal Group PLC	Chronocort (hydro- cortisone)	congenital adrenal hyperplasia	DIUR-005; mixed results.
UPDATED PHASE III RESULTS
Protalix BioTherapeutics Inc./Chiesi Farmaceutici SpA	pegunigalsidase alfa	Fabry's disease	BRIDGE; improved renal function.
Paratek Pharmaceuticals Inc.	Nuzyra (omadacycline)	skin and skin structure infections	OASIS-2; improved quality of life.
Ardelyx Inc.	tenapanor	irritable bowel syndrome	T3MPO-3; safe in long-term study.
Johnson & Johnson	Stelara (ustekinumab)	Crohn's disease	IM-UNITI; use of concomitant immunosuppressants not necessary.
Bellerophon Therapeutics Inc.	INOpulse (nitric oxide)	pulmonary arterial hypertension	INOvotion-1; clinical improvements.
Roche Holding AG	Ocrevus (ocrelizumab)	multiple sclerosis	OPERA I, II; ORATORIO; efficacy maintained.
Evofem Biosciences Inc.	Amphora (L-lactic acid, citric acid, potassium bitartrate)	contraception	AMP001; patient comfort confirmed.
ObsEva SA	nolasiban (OBE001)	infertility	IMPLANT2; improved pregnancy rate.
Theravance Biopharma/Mylan	Yupelri (revefenacin)	chronic obstructive pulmonary disease	Positive data, safe CV profile.
Insmed Inc.	Arikayce (amikacin) liposome inhalation	MAC lung disease	Further clinical data collected.
PHASE III COMPLETED
Adamas Pharmaceuticals Inc.	Gocovri (amantadine) extended-release	Parkinson's disease with dyskinesia	EASE LID 2; well tolerated over two years.
PHASE III INITIATED
Strekin AG	STR001	hearing loss	RESTORE; in Europe.
Exelixis	Cabometyx (cabozantinib)	thyroid cancer	COSMIC-311; refractory disease.
Allergan	brazikumab	ulcerative colitis, Crohn's disease	EXPEDITION, INTREPID; versus adalimumab.
Diurnal Group PLC	Chronocort (hydro- cortisone)	congenital adrenal hyperplasia	RESTORE; delayed release formulation.
PHASE III ANNOUNCED
Tyme Technologies Inc.	SM-88	pancreatic cancer	In an adaptive clinical study.
PHASE II SUSPENDED
Menlo Therapeutics Inc.	serlopitant	refractory chronic cough	TUSSIX; failed to show clinical benefit.
PHASE II INTERIM/TOP-LINE RESULTS
Xencor Inc.	XmAb5871	systemic lupus erythematosus	Positive trend in primary endpoint.
Cavion LLC	CX-8998	essential tremor	T-CALM; improved symptoms.
Constellation Pharmaceuticals Inc.	CPI-0610	myelofibrosis	MANIFEST; signs of efficacy.
UPDATED PHASE II RESULTS
Cytokinetics Inc./ Astellas	reldesemtiv	spinal muscular atrophy	Fast skeletal muscle troponin activator.
Audentes Therapeutics Inc.	AT132	X-linked myotubular myopathy	ASPIRO; improved respiratory function.
Amicus Therapeutics Inc.	ATB200/AT2221	Pompe disease	Durable responses.
Acceleron Pharma Inc.	ACE-083	muscular dystrophy	Signs of efficacy.
Mithra Pharmaceuticals	Donesta (estetrol)	menopausal symptoms	E4Relief; promising agent.
Biogen	Spinraza (nusinersen)	spinal muscular atrophy	NURTURE; benefits of treating pre-symptomatic infants.
Loxo Oncology Inc.	LOXO-292	thyroid cancer	LIBRETTO-001; early evidence of activity.
Novavax Inc.	RSV F vaccine	RSV infections	Maternal dosing was immunogenic.
Allergan Inc.	brazikumab	Crohn's disease	Well tolerated.
Arena Pharmaceuticals Inc.	etrasimod	ulcerative colitis	OASIS; clinical responses observed.
Ritter Pharmaceuticals Inc.	RP-G28	lactose intolerance	Gut microbiome adapted.
Mapi Pharma	glatiramer acetate depot	multiple sclerosis	Encouraging results.
GeNeuro SA	GNbAC1	multiple sclerosis	CHANGE-MS; evidence of efficacy.
Targovax AS	ONCOS-102, oncolytic virus	mesothelioma	Signs of efficacy.
PHASE II COMPLETED
Altimmune Inc.	NasoVAX	pandemic flu vaccine	Immune responses observed, well tolerated.
TG Therapeutics Inc.	ublituximab	multiple sclerosis	Safe and effective.
PHASE II INITIATED
Oryzon Genomics SA	vafidemstat	dementia with aggressive episodes	REIMAGINE; in various clinical settings.
Sermonix Pharmaceuticals LLC	lasofoxifene	metastatic breast cancer	With ESR1 gene mutations.
Clene Nanomedicine Inc.	CNM-Au8	optic neuritis	In relapsing multiple sclerosis.
Therachon AG	FE203799	short bowel syndrome	Two studies.
Antibe Therapeutics Inc.	ATB-346	pain	In healthy volunteers.
Sorrento Therapeutics Inc.	STI-A1014	sarcoma, bladder cancer	Dose ranging studies.

Source: Informa Pharma Intelligence's Biomedtracker.

 

 

Marketing Approvals – Oct. 5 to Oct. 11, 2018

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Akcea Therapeutics Inc.	-	Tegsedi (inotersen)	hATTR amyloidosis with polyneuropathy	US	In adults.
Leadiant Biosciences Inc.	-	Revcovi (elapegademase-lvir)	adenosine deaminase severe combined immune deficiency	US	Pediatric and adult patients.
Medical Developments International	-	Penthrox (low- dose methoxy- flurane)	emergency pain relief	Saudi Arabia	By inhalation.
SUPPLEMENTAL REGULATORY APPROVAL
Merck & Co	-	Gardasil 9	HPV vaccine	US	People aged 27 to 45 years.
Ono Pharma	-	Opdivo (nivolumab)	renal cell cancer	South Korea	Combined with Yervoy.
Boehringer Ingelheim	-	Stiolto Respimat (tiotropium/ olodaterol)	COPD including chronic bronchitis and emphysema	US	Inhalation spray.
Johnson & Johnson/ Bayer AG	-	Xarelto (rivaroxaban) plus low-dose aspirin	CV risk reduction in coronary artery disease or peripheral artery disease	US	Based on COMPASS trial.

Source: Biomedtracker.

 

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.