Pipeline

April 30, 2018

Company	Drug/Device	Medical Condition	Status
AOBiome Therapeutics	Ammonia Oxidizing Bacteria (AOB) intranasal spray	episodic migraine	Phase II trial initiated enrolling 303 subjects
Mithra	Donesta	Vasomotor Symptoms (VMS) in post-menopausal women	Phase II top-line results announced evaluating 257 subjects
Concert Pharmaceuticals	CTP-543	moderate-to-severe alopecia areata	Phase IIa trial initiated evaluating 90 subjects
Aldeyra Therapeutics, Inc.	topical ocular reproxalap	allergic conjunctivitis	Phase III trial initiated enrolling 300 subjects
Catalyst Pharmaceuticals, Inc.	Firdapse (amifampridine phosphate)	MuSK antibody positive Myasthenia Gravis (MuSK-MG)	Phase III trial initiated enrolling 60 subjects in the U.S. and Italy
Pfizer Inc.	TRUMENBA (Meningococcal Group B Vaccine)	Active immunization to prevent invasive disease caused by Neisseria meningitides group B (MenB) in children ages 1 through 9 years	Breakthrough Therapy Designation granted by the FDA
Lumendi, LLC	DiLumen C2	second-generation endoscopic accessory indicated to ensure complete positioning of an endoscope in the large intestine and assist with optical visualization, diagnosis and endoscopic treatment	510(k) clearance granted by the FDA
Varian	Calypso Anchored Beacon transponder	tumor detection	510(k) clearance granted by the FDA
Amerigen Pharmaceuticals Limited and Dipharma S.A.	Miglustat 100 mg capsules	Adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option	aNDA filed with the FDA
Sage Therapeutics	intravenous formulation of brexanolone (SAGE-547)	postpartum depression (PPD)	NDA filed with the FDA
Veloxis Pharmaceuticals A/S	de novo indication of ENVARSUS XR (tacrolimus extended-release tablets)	Prophylaxis of organ rejection in kidney transplant patients	sNDA filed with the FDA
Abeona Therapeutics Inc.	ABO-102	AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA)	RMAT Designation granted by the FDA
MeiraGTx Limited	AAV-RPGR	X-linked retinitis pigmentosa (SLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene	Fast Track Designation granted by the FDA
Polyphor	balixafortide (POL6326) in combination with eribulin	patients with HER2-negative metastatic breast cancer who previously received at least two chemotherapeutic regiments in the metastatic setting	Fast Track Designation granted by the FDA
QIAGEN	PartoSure	preterm birth	FDA approved