Pipeline

September 14, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
Leukemia and Lymphoma Society	acalabrutinib	patients with hematological cancers who have tested positive for COVID-19	initiation of phase 1 trial
Celularity	(CYNK-001) human placental hematopoietic stem cell derived NK cells	adults with COVID-19	first patient dosed in a Phase 1/2 trial
Kamada	anti-SARS-CoV-2 plasma-derived hyperimmune immunoglobulin (IgG) product	hospitalized, nonventilated COVID-19 patients with pneumonia	enrollment complete in phase 1/2 trial
SpyBiotech and the Serum Institute of India	novel virus-like particle (VLP) vaccine targeting COVID-19	COVID-19	first patients dosed in a Phase 1/2 trial
Aldeyra Therapeutics	ADX-629	adult patients hospitalized for COVID-19	IND approved by the FDA for phase 2 trial
Other Trials and Actions
Translate Bio	MRT5005	cystic fibrosis	patient enrollment and dosing resumed in phase 1/2 trial
Allena Pharmaceuticals	ALLN-346	hyperuricemia in patients with gout and advanced chronic kidney disease	first patient dosed in phase 1 trial
Flexion Therapeutics	FX201	osteoarthritis	first patient treated in second cohort of phase 1 trial
Scholar Rock	SRK-181 in combination with anti-PD-(L)1 therapy	locally advanced or metastatic solid tumors	first patient dosed in Part A2 of phase 1 trial
Viking Therapeutics	VK0214	X-linked adrenoleukodystrophy	initiation of phase 1 trial
Zion Pharma	ZN-A-1041	brain metastases accompanying HER2+ advanced breast cancer	initiation of phase 1 trial
Compugen	COM701, Opdivo (nivolumab) and BMS-986207	advanced solid tumors	first patient dosed in phase 1/2 trial
89bio	BIO89-100	severe hypertriglyceridemia	initiation of phase 2 trial
Clene Nanomedicine	CNM-Au8	early symptomatic ALS patients	patient enrollment complete in phase 2 trial
Goldfinch Bio	GFB-887	focal segmental glomerulosclerosis (FSGS) and diabetic nephropathy	first patients dosed in phase 2 trial
Intensity Therapeutics Merck	INT230-6 combined with Keytruda (pembrolizumab)	colon cancer (non-MSI high), pancreatic and bile duct cancer, and squamous-cell carcinoma	first patients dosed in phase 2 expansion trial
Lexicon Pharmaceuticals	LX9211	diabetic peripheral neuropathic pain	first patients dosed in phase 2 trial
MyoKardia	danicamtiv (formerly MYK-491)	patients with primary dilated cardiomyopathy thought to be caused by genetic mutations of the sarcomere	first patient dosed in phase 2 trial
REGENXBIO	suprachoroidal delivery of RGX-314 using the SCS Microinjector	wet age-related macular degeneration	first patient dosed in phase 2 trial
AZTherapies	ALZT-OP1a	mild- to moderate-stage ALS	first patient dosed in phase 2a trial
LIPAC Oncology	LiPax (paclitaxel)	patients with low-grade highly recurrent non-muscle-invasive bladder cancer	completion of phase 2a trial
Chi-Med	fruquintinib	metastatic colorectal cancer	initiation of phase 3 trial
Oncopeptides	melflufen	relapsed refractory multiple myeloma	enrollment complete in phase 3 trial
Apros Therapeutics	APR003	advanced unresectable colorectal cancer with malignant liver lesions	IND approved by the FDA
Atara Biotherapeutics	ATA2271	advanced mesothelioma	IND approved by the FDA
Biohaven Pharma	oral zavegepant (formerly BHV-3500)	migraine	IND approved by the FDA
Caribou Biosciences	CB-010	relapsed/refractory B-cell non-Hodgkins lymphoma	IND approved by the FDA
InspireMD	CGuard Embolic Prevention System	prevention of stroke caused by carotid artery disease	Investigational Device Exemption approved by the FDA
Precision Biosciences	PBCAR269A	relapsed/refractory multiple myeloma	Fast Track designation granted by the FDA
Tern Pharmaceuticals	TERN-201	non-alcoholic steatohepatitis	Fast Track designation granted by the FDA
Ascentage Pharma	APG-2575	chronic lymphocytic leukemia	Orphan Drug designation granted by the FDA
AlphaMab Oncology	KN046	thymic epithelial tumors	Orphan Drug designation granted by the FDA
Kleo Pharmaceuticals	KP1237	multiple myeloma	Orphan Drug designation granted by the FDA
Peptilogics	PLG0206	prosthetic joint infections	Orphan Drug designation granted by the FDA
BeyondSpring	Plinabulin	chemotherapy-induced neutropenia	Breakthrough Therapy designation granted by the FDA
Clarity Pharmaceuticals	64Cu-SARTATE	diagnostic for the clinical management of neuroblastoma	Rare Pediatric Disease designation granted by the FDA
Alydia Health	Jada System	control and treatment of postpartum hemorrhage and abnormal postpartum uterine bleeding	approved by the FDA
Athena Bioscience	Qdolo (tramadol hydrochloride) Oral Solution 5mg/1mL C-IV	management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate	approved by the FDA
Eli Lilly	Trulicity (dulaglutide)	type 2 diabetes	additional doses approved by the FDA
Genentech	Gavreto (pralsetinib)	metastatic rearranged during transfection (RET) fusion-positive non-small-cell lung cancer	approved by the FDA
GlaxoSmithKline Innoviva	Trelegy Ellipta (fluticasone furoate / umeclidinium / vilanterol)	asthma in patients aged 18 years and older	approved by the FDA for additional indication