Pipeline Watch: Phase III Starts With VX-445, Reproxalap And AVXS-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 April 20 and April 26, 2018, 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 – Apr. 20 to Apr. 26, 2018

Lead company/partner	Compound	Indication	Comments
PHASE III INTERIM/TOP-LINE RESULTS
Merck & Co. Inc.	relebactam plus imipenem/cilastatin	carbapenem-resistant infections	RESTORE-IM1; favorable efficacy and side effect responses.
Alexion Pharmaceuticals Inc.	ALXN1210, every eight weeks	paroxysmal nocturnal hemoglobinuria	Switching endpoints non-inferior to Soliris.
AveXis Inc.	AVXS-101, gene therapy	spinal muscular atrophy, type 1	STRIVE; signs of efficacy.
GW Pharmaceuticals PLC	Epidiolex (cannabidiol) oral solution	Lennox-Gastaut and Dravet syndromes	GWPCARE5; clinical improvements.
AbbVie Inc.	upadacitinib	rheumatoid arthritis	SELECT-SUNRISE; met primary endpoint, in Japanese patients.
AstraZeneca PLC	Imfinzi (durvalumab) with or without tremelimumab	non-small cell lung cancer (NSCLC), third-line	ARCTIC; combination missed endpoints, monotherapy encouraging.
UPDATED PHASE III RESULTS
Biogen Inc.	Spinraza (nusinersen)	spinal muscular dystrophy	SHINE; benefits sustained.
Alder Biopharmaceuticals Inc.	eptinezumab	episodic migraine	PROMISE1; increased migraine-free intervals, quality of life.
Teva Pharmaceutical Industries Ltd.	fremanezumab	chronic migraine	HALO CM, EM; efficacy confirmed.
Biohaven Pharmaceuticals Holding Co. Ltd.	rimegepant	migraine	Durability of clinical effects.
Neurocrine Biosciences Inc.	Ingrezza (valbenazine)	tardive dyskinesia	Kinect 3, 4; improved symptoms.
Flexion Therapeutics Inc.	Zilretta (triamcinolone acetonide) Inj	osteoarthritis	Robust treatment responses.
Zogenix Inc.	ZX008 (low-dose fenfluramine)	Dravet syndrome	Reduced seizures, well tolerated.
Celgene Corp.	ozanimod	multiple sclerosis	RADIANCE, SUNBEAM; reduced relapse rates.
Ionis Pharmaceuticals Inc.	inotersen	hATTR amyloidosis polyneuropathy	Benefits observed, sustained response.
Paratek Pharmaceuticals Inc.	omadacycline	community-acquired bacterial pneumonia	OPTIC, OASIS 2; high response rate, non-inferior to moxifloxacin.
Achaogen Inc.	plazomicin	urinary tract infections	EPIC, CARE; high response rates, well tolerated.
Melinta Therapeutics Inc.	Vabomere (meropenem and vaborbactam)	bacterial infections	TANGO II, efficacy in patients with co-morbidities.
PHASE III INITIATED
Vertex Pharmaceuticals Inc.	VX-445, tezacaftor and ivacaftor	cystic fibrosis	A triple regimen.
Aldeyra Therapeutics Inc.	reproxalap	conjunctivitis, allergic	Topical ocular administration.
AveXis Inc.	AVXS-101, gene therapy	pre-symptomatic spinal muscular atrophy types 1,2,3	SPRINT; a one-time dose.
PHASE III ANNOUNCED
Synthetic Biologics Inc.	ribaxamase (SYN-004)	Clostridium difficile associated diarrhea	In the US.
PHASE II SUSPENDED
Prothena Corp. PLC	NEOD001	AL amyloidosis	PRONTO; missed endpoints.
PHASE II INTERIM/TOP-LINE RESULTS
Daiichi Sankyo Co. Ltd.	DS-5141	Duchenne muscular dystrophy	Signs of efficacy, well tolerated.
Mirati Therapeutics Inc.	sitravatinib plus nivolumab	NSCLC	Signs of efficacy.
Mirati Therapeutics Inc.	mocetinostat plus durvalumab	NSCLC	Signs of efficacy.
Tesaro Inc.	Zejula (niraparib)	ovarian cancer, heavily pretreated	QUADRA; active in BRCA mutation negative patients.
Neuraltus Pharmaceuticals Inc.	NP001	amyotrophic lateral sclerosis	Missed primary and secondary endpoints.
Biocad	BCD-085	psoriasis, ankylosing spondylitis	Durable clinical responses.
UPDATED PHASE II RESULTS
Acceleron Pharma Inc.	AC-083	muscular dystrophy	Dose dependent responses seen.
Centrexion Therapeutics Corp.	CNTX-4975	arthritis pain	TRIUMPH; improved chronic pain.
Medivir AB	MIV-711	osteoarthritis	Reduced disease progression.
Roche	RG7916	spinal muscular atrophy	JEWELFISH; signs of efficacy, well tolerated.
Roche	RG6206	Duchenne muscular dystrophy	Signs of efficacy.
Vaccibody AS	VB10.16	precancerous cervical neoplasia	Immune responses observed.
Vertex Pharmaceuticals Inc.	VX-445, VX-659, in triple combinations	cystic fibrosis	Well tolerated, improved endpoints.
Catabasis Pharmaceuticals Inc.	edasalonexent	Duchenne muscular dystrophy	MoveDMD; positive effects observed.
Zynerba Pharmaceuticals Inc.	ZYN-002 (transdermal cannabidiol)	focal seizures	STAR 2; improved seizure control.
Agilis Biotherapeutics LLC	AGIL-AADC gene therapy	AADC deficiency	Improved motor function.
MediciNova Inc.	MN-166 (ibudilast)	progressive multiple sclerosis	SPRINT-MS; reduced rate of progression of whole brain atrophy.
Rebiotix Inc.	RBX2660	Clostridium difficile infections	Well tolerated and effective.
Ionis Pharmaceuticals Inc.	IONIS-HTT-Rx	Huntington's disease	Lowered mutant huntingtin levels.
TG Therapeutics Inc.	ublituximab	multiple sclerosis	Signs of efficacy.
AzurRx BioPharma Inc.	MS1819	exocrine pancreatic insufficiency	Positive interim data.
SK Biopharmaceuticals Co. Ltd.	cenobamate	epilepsy	Reduced seizure frequency.
Molecular Partners AG	MP0250	multiple myeloma	Initial signs of efficacy.
Summit Therapeutics PLC	ezutromid	Duchenne muscular dystrophy	PhaseOut DMD; sings of efficacy.
PHASE II COMPLETED
argenx SE	efgartigimod	generalized myasthenia gravis	Encouraging efficacy.
PHASE II INITIATION
Durect Corp.	DUR-928	alcoholic hepatitis	Dose escalation.
PaxVax Inc.	chikungunya vaccine	chikungunya	Licensed from the NIAID.
Aclaris Therapeutics Inc.	ATI-502	alopecia	A JAK 1/3 inhibitor.
AOBiome	ammonia oxidizing bacteria	episodic migraine prevention	Intranasal spray.
Axsome Therapeutics Inc.	AXS-05 (bupropion and dextromethorphan)	smoking cessation	Fixed dose combination.
Oramed Pharmaceuticals Inc.	ORMD-0801 (oral insulin) capsule	diabetes, type 2	A 90-day pivotal study.

Source: Informa Pharma Intelligence’s BiomedTracker.

 

 

Marketing Approvals – Apr. 20 to Apr. 26, 2018

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Pfizer Inc.	-	Mylotarg (gemtuzumab ozogamicin)	acute myeloid leukemia	EU	Previously untreated disease.
SUPPLEMENTAL REGULATORY APPROVAL
Bristol-Myers Squibb Co.	-	Opdivo (nivolumab)	various indications	EU	Two- and four-week dosing schedules.
Sunovion Pharmaceuticals Inc.	-	Latuda (lurasidone)	bipolar depression	Canada	In adolescents.
GlaxoSmithKline PLC	-	Trelegy Ellipta (fluticasone/ umeclidinium/vilanterol)	chronic bronchitis, emphysema, COPD exacerbations	US	Use in a broader population of COPD patients.
Otsuka Holdings Co. Ltd.	-	Jynarque (tolvaptan)	to slow kidney function decline	US	In patients with autosomal dominant polycystic kidney disease.
Helsinn Group	-	Akynzeo (fosnetupitant/ palonosetron), iv	chemotherapy induced nausea and vomiting	US	With dexamethasone.

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.