Pipeline

September 17, 2018

Company	Drug/Device	Medical Condition	Status
Amryt	AP101	Epidermolysis Bullosa, a genetic disorder that causes skin and lining of internal organs to blister and tear	Granted Investigational New Drug clearance by FDA to open U.S. sites as part of EASE Phase III clinical trial
Fibrocell Science, Inc.	FCX-013	Moderate to severe localized scleroderma	Granted Fast Track designation by the FDA
Loxo Oncology, Inc.	LOXO-292	Metastatic RET-fusion-positive non-small cell lung cancer; RET-mutant medullary thyroid cancer	Granted Breakthrough Therapy designation by the FDA
Genesys Spine	AIS-C Stand-Alone System a first of its kind, non-screw based, zero-profile, direct-anterior interbody system	Cervical spine surgery	Granted FDA clearance
Perrigo Company plc	OTC equivalent of Imodium® Multi-Symptom Relief (loperamide hydrochloride and simethicone tablets, 2 mg/125 mg)	Diarrhea plus bloating, pressure and cramps	Granted approval by the FDA
Concentric Analgesics, Inc.	CA-008, a non-opioid therapeutic	Post-surgical pain	Granted Breakthrough Therapy designation by the FDA
physIQ	AFib detection analytics engine	Atrial Fibrillation	Granted FDA 510(k) clearance
Foamix Pharmaceuticals, Ltd.	FMX101, tpical minocycline foam	Moderate to severe acne	Met endpoints in Phase III trial
Merck KGaA and Pfizer	Combination of Bavencio (avelumab) and Inlyta (axitinib)	Advanced renal cell carcinoma	Therapy significantly improved progression-free survival in Phase III trial
Vital Therapies	VT-308	Severe alcoholic hepatitis	Failed Phase III clinical trial
Gilead Sciences and Galapagos NV	Filgotinib	Moderate to severe active rheumatoid arthritis	FINCH 2 Phase III trial successfully met primary endpoints
Bristol-Myers Squibb	BMS-986165	Moderate to severe plaque psoriasis	Hit efficacy endpoints in Phase II clinical trial
Amygdala Neurosciences	ANS-6637	Alcohol use disorder	Initiated Phase II clinical trial with NIAAA
Abeona Therapeutics, Inc.	Gene therapy product ABO-101 (AAV-NAGLU)	MPS IIIB (Sanfilippo syndrome type B)	Initiated Phase I/II clinical trial in Spain
EpicGenetics, Inc.	Tuberculosis vaccine, BCG	Fibromyalgia	Launching Phase II clinical trial with Mass General Hospital
Checkpoint Therapeutics, Inc.	CK-101 (also known as RX518), a third-generation epidermal growth factor receptor tyrosine kinase inhibitor (TKI)	Non-small cell lung cancer	Positive preliminary safety and efficacy data announced from an ongoing Phase I/II clinical trial