Pipeline

September 9, 2019

Company	Drug/Device	Medical Condition	Status
Inhibrx Inc.	INBRX-101	alpha-1 antitrypsin deficiency (AATD)	Phase 1 trial initiated
Ribon Therapeutics	RBN-2397	cancer	Phase 1 trial initiated enrolling 2,397 subjects with various tumor types, including squamous cell carcinoma of the lung
Infinity Pharmaceuticals Arcus Biosciences	IPI-549 in combination with Arcus’ AB298 and Doxil	advanced triple negative breast cancer (TNBC)	Phase 1/1b trial initiated
Hutchison China MediTech Limited	HMPL-689	relapsed or refractory lymphoma	Phase 1/1b trial initiated
Rocket Pharmaceuticals, Inc.	RP-L201	severe Leukocyte Adhesion Deficiency-I (LAD-I)	Phase 1/2 trial initiated enrolling nine pediatric subjects globally
Infinity Pharmaceuticals Genentech	IPI-549 in combination with Tecentriq and Abraxane (nab-paclitaxel)	front-line triple negative breast cancer (TNBC)	Phase 2 trial initiated
OBI Pharma	OBI-999	Globo H	IND approval granted by the FDA
Magenta Therapeutics	MGTA-456	metabolic disorders	RMAT designation granted by the FDA
Prescient Metabiomics	LifeKit Prevent Colorectal Neoplasia Test	colorectal cancer (CRC)	Breakthrough Device designation granted by the FDA
Ra Pharmaceuticals, Inc.	zilucoplan	myasthenia gravis	Orphan Drug designation granted by the FDA
ApiFix Ltd.	Minimally Invasive Deformity Correction (MID-C) system	progressive adolescent idiopathic scoliosis (AIS)	HDE approval granted by the FDA
US WorldMeds, LLC	MYOBLOC (rimabotulinumtoxinB) injection	chronic sialorrhea	sBLA approval granted by the FDA