Pipeline Watch: Phase III Studies of CSL112 And RT-100 Imminent

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. 1 and Dec.7, 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. 1 to Dec. 7, 2017

Source: Informa Pharma Intelligence’s BiomedTracker.

Lead company/partner	Compound	Indication	Comments
Phase III Suspended
Sanofi	Clostridium difficile vaccine	recurrent C difficile infection	Cdiffense; Interim analysis indicated unlikely to meet endpoint.
Phase III Interim/Top-line Results
AbbVie Inc./Boehringer Ingelheim GMBH	risankizumab	psoriasis	IMMhance; met primary endpoints.
GlaxoSmithKline PLC	Shingrix vaccine	chickenpox, shingles prevention	ZOE-HSCT; positive in autologous stem cell transplant.
AbbVie Inc.	Imbruvica (ibrutinib)	Waldenstrom macroglobulinemia	INNOVATE; improved PFS.
Revance Therapeutics Inc.	daxibotulinumtoxinA (RT002)	glabellar lines	SAKURA 1, 2; positive results.
Akili Interactive Labs Inc.	AKL-T01	attention deficit hyperactivity disorder	STARS-ADHD; A digital medicine.
UCB SA	Vimpat (lacosamide)	focal seizures in children	Reduced seizure frequency.
Bristol-Myers Squibb Co.	Opdivo (nivolumab)	advanced non-small cell lung cancer (NSCLC), previously treated	CheckMate078; met endpoint of overall survival benefit in China.
Vertex Pharmaceuticals Inc.	Kalydeco (ivacaftor)	cystic fibrosis	ARRIVAL; positive results in children aged one to two years.
Updated Phase III Results
Novartis AG	isqali (ribociclib)	advanced breast cancer, HR+, HER2-	MONALEESA-7; positive in premenopausal women.
Roche	Tecentriq (atezolizumab)	advanced NSCLC, first-line	IMpower150; clinically meaningful PFS benefit.
Pfizer Inc.	Ibrance (palbaciclib)	advanced breast cancer, HR+, HER2-	PALOMA-2; positive results.
Novo Nordisk AS	Xultophy (insulin degludec/liraglutide)	diabetes, type 2	DUAL VII; a once-daily option.
Ultragenyx Pharmaceutical Inc./Kyowa Hakko Kirin Co. Ltd.	burosumab (KRN23)	X-linked hypophosphatemia	Sustained clinical improvement after 48 weeks.
Neurocrine Biosciences Inc.	Ingrezza (valbenazine)	tardive dyskinesia	KINECT 4; improved symptoms long term.
Phase III Initiated
La Jolla Pharmaceutical Co.	LJPC-401 (synthetic human hepcidin)	beta thalassemia	To reduce cardiac iron levels.
Phase III Announced
CSL Ltd.	CSL112 (apolipoprotein A-I)	atherosclerosis	AEGIS-II; will take around four years.
Renova Therapeutics	RT-100 (a gene therapy)	congestive heart failure	FLOURISH; by intracoronary inj.
Iterum Therapeutics Ltd.	sulopenem etzadroxil	urinary and reproductive tract infections	An oral penem antibiotic.
Phase II Suspended
Teva Pharmaceutical Industries Ltd./Active Biotech AB	Nerventra (laquinimod)	primary progressive multiple sclerosis	ARPEGGIO; primary endpoint on brain atrophy not met.
Updated Phase II Results
Sage Therapeutics Inc.	SAGE-217	major depressive disorder	Durable responses, well tolerated.
Adaptimmune Therapeutics PLC	NY-ESO SPEAR T-cells	multiple myeloma	Complete and partial responses observed.
Puma Biotechnology Inc.	Nerlynx (neratinib)	HER2+-early stage breast cancer	CONTROL; adjuvant treatment of diarrhea.
Immunovaccine Inc.	DPX-Survivac plus epacadostat, low-dose cyclophosphamide	advanced ovarian cancer	Partial responses, well tolerated.
OncBioMune Pharmaceuticals Inc.	ProscaVax (PSA plus IL-2 and GM-CSF)	prostate cancer	Durable responses observed.
BioLineRx Ltd.	BL-8040 plus cytarabine	acute myeloid leukemia	Positive overall survival data.
Bayer AG/Loxo Oncology Inc.	larotrectinib	solid tumors	SCOUT; durable responses in children.
Neuralstem Inc.	NSI-189	major depression	Antidepressant and cognitive benefits.
Ra Pharmaceuticals Inc.	RA-101495	paroxysmal nocturnal hemoglobinuria	Sustained responses, no ADR concerns.
Zynerba Pharmaceuticals Inc.	ZYN002 (transdermal cannabidiol)	focal epilepsy	STAR 1, 2; clinically meaningful responses.
Phase II Completed
Immunomedics Inc.	sacituzumab govitecan	breast cancer, triple negative, relapsed/ refractory metastatic	Tumor shrinkage and durable clinical responses seen.
Phase II Interim/Top-line Results
Adocia SAS	BioChaperone Lispro U100 (insulin)	diabetes, type 1	Positive result versus Novolog and Fiasp.
Madrigal Pharmaceuticals Inc.	MGL-3196	non-alcoholic steatohepatitis (NASH)	Reduced liver fat levels, improved liver enzymes.
PaxVax Inc.	Vaxchora (CVD 103-HgR)	cholera in endemic settings	Shows utility of single higher doses.
Galectin Therapeutics Inc.	GR-MD-02	NASH	Mixed results but did improve portal hypertension.
Ironwood Pharmaceuticals Inc.	IW-1973	diabetic nephropathy	Improved metabolic parameters.
Tioga Pharmaceuticals Inc.	asimadoline	atopic dermatitis	Reduced itching.
CASI Pharmaceuticals Inc.	ENMD-2076	breast cancer	Durable clinical activity.
Phase II Initiation
Minerva Neurosciences Inc.	seltorexant	insomnia disorder	In adults and elderly subjects.
ObsEva SA	OBE022	preterm labor	PROLONG; prostaglandin antagonist.
Realm Therapeutics	PR022	atopic dermatitis	A topical gel.
Aveo Pharmaceuticals Inc./Biodesix Inc.	ficlatuzumab	head and neck cancer	With and without cetuximab.
Kalytera Therapeutics	cannabidiol	graft-vs-host disease	Multiple doses.

Marketing Approvals – Dec. 1 to Dec. 7, 2017

Source: Biomedtracker.

Lead Company	Partner Company	Drug	Indication	Market	Comments
REGULATORY APPROVAL
Novo Nordisk AS	-	Ozempic (semaglutide)	diabetes, type 2	US	Once-weekly, an adjunct to diet and exercise.
Sunovion Pharmaceuticals Inc.	_	Lonhala Magnair (glycopyrrolate) inhalation	chronic obstructive pulmonary disease	US	For twice daily use; the first nebulized LAMA for COPD.
Mylan NV	Biocon Ltd.	Ogivri (trastuzumab-dkst)	breast and gastric cancer, HER2+	US	Herceptin biosimilar.
Indivior PLC	-	Sublocade (buprenorphine)	opioid use disorder	US	Once-monthly sc injection.
Novaliq GMBH	-	NovaTears + Omega-3	dry eye disease	EU	A lipid layer stabilizer.
Grupo Biotoscana SL	-	Harvoni (ledipasvir/ sofosbuvir)	hepatitis C	Brazil	Launch expected in Q2 2018.
H. Lundbeck AS	Takeda	Brintellix (vortioxetine)	major depressive disorder	China	Launch expected Q2 2018.
Novartis AG	-	Rydapt (midostaurin)	acute myeloid leukemia	Canada	Based on the Ratify study.
Merck KGAA	-	Mavenclad (cladribine)	multiple sclerosis	Canada	As monotherapy.
SUPPLEMENTAL REGULATORY APPROVAL
Roche	-	Avastin (bevacizumab)	glioblastoma	US	Provisional approval converted to full approval.
Amgen Inc.	-	Repatha (evolocumab)	heart attack, stroke and coronary revascularization prevention	US	Based on the Fourier study.
Eli Lilly & Co.	-	Taltz (ixekizumab)	psoriatic arthritis	US	In adults.

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.