Pipeline

June 10, 2019

Company	Drug/Device	Medical Condition	Status
Cellenkos, Inc.	CK0801	bone marrow failure syndromes, including aplastic anemia, hypoplastic myelodysplasia and primary myelofibrosis	Phase I trial initiated
Xencor, Inc.	XmAb22841	advanced solid tumors	Phase I trial initiated
Alnylam Pharmaceuticals, Inc.	ALN-AGT	hypertension	Phase I trial initiated enrolling 168 subjects
UCB Neuropore Therapies, Inc.	UCB0599	Parkinson’s disease	Phase Ib trial initiated
Amphera B.V.	MesoPher	pancreatic cancer	Phase II trial initiated enrolling 10 adult subjects with surgically resected pancreatic cancer who have received adjuvant standard of care treatment
Immunovant	IMVT-1401	moderate-to-severe active Graves' ophthalmopathy (GO)	Phase II trial initiated enrolling eight subjects
Rejenevie Therapeutics	AR-100 immune restoration therapy	immunology	Phase II trial initiated enrolling 20 healthy subjects 55 years of age or older in Freeport, Bahamas
Ironwood Pharmaceuticals, Inc. Allergan PLC	MD-7246	abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D)	Phase II trial initiated enrolling 400 subjects
Verona Pharma PLC	metered-dose inhaler formulation of ensifentrine (RPL554)	moderate-to-severe chronic obstructive pulmonary disease (COPD)	Phase II trial initiated enrolling 36 subjects at two sites in the UK
Minoryx Therapeutics	leriglitazone (MIN-102)	Friedreich’s Ataxia	Phase II trial initiated enrolling 36 subjects 12 years of age or older at numerous sites across Europe
GENFIT	elafibranor	nonalcoholic fatty liver disease (NAFLD)	Phase II trial initiated enrolling 16 subjects with NAFL as identified with magnetic resonance spectroscopy (H-MRS)
Arcutis, Inc.	ARQ-151	atopic dermatitis	Phase IIa trial initiated
Akero Therapeutics, Inc.	AKR-001	non-alcoholic steatohepatitis (NASH)	Phase IIa trial initiated enrolling 80 subjects
Ansun Biopharma, Inc.	DAS181	lower respiratory tract parainfluenza virus	Phase III trial initiated enrolling immunocompromised subjects at sites in the U.S., Europe and Asia
Eyenovia Inc.	MicroPine	pediatric progressive myopia	Phase III trial initiated enrolling 400 subjects ages three to 12 years of age
Incyte	pemigatinib (INCB54828)	cholangiocarcinoma	Phase III trial initiated enrolling 432 subjects
Allergan plc Gedeon Richter PLC	VRAYLAR (cariprazine)	manic or mixed episodes associated with bipolar I disorder in adults	sNDA approval granted by the FDA
Cadent Therapeutics	CAD-1883	spinocerebellar ataxia (SCA)	Orphan Drug designation granted by the FDA
Bayer	Aliqopa (copanlisib)	marginal zone lymphoma (MZL)	Breakthrough Therapy designation granted by the FDA
Imara, Inc.	IMR-687	sickle cell disease	Fast Track designation granted by the FDA
NovoCure	NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy	malignant pleural mesothelioma (MPM)	Approval granted by the FDA
AveXis Inc.	Zolgensma (onasemnogene abeparvovec-xioi)	spinal muscular atrophy (SMA) in children less than two years of age	Approval granted by the FDA
Novartis	Piqray (alpelisib) tablets in combination with the endocrine therapy fulvestran	hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated advanced or metastatic breast cancer in postmenopausal women and men	Approval granted by the FDA
Novartis	Jakafi (ruxolitinib)	steroid-refractory acute GVHD in adult and pediatric subjects 12 years and older	Approval granted by the FDA
Celgene Corporation	REVLIMID (lenalidomide) in combination with a rituximab product (R²)	previously treated follicular lymphoma (FL) or marginal zone lymphoma (MZL) in adults	Approval granted by the FDA