Pipeline

August 24, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
Algernon Pharmaceuticals	NP-120 (Ifenprodil)	hospitalized patients with confirmed COVID-19	first patient enrolled in the U.S. in phase 2b/3 trial
Enzychem LifeSciences	EC-18	patients with acute respiratory distress syndrome (ARDS) due to COVID-19 pneumonia	IND approved by the FDA for phase 2 trial
Accelerate Diagnostics	BioCheck SARS-CoV-2 IgM and IgG Combo Test and fully automated MS-Fast instrument	COVID-19 antibody test	Emergency Use Authorization (EUA) granted by the FDA
LumiraDx	LumiraDx SARS-CoV-2 antigen test	detection of SARS-CoV-2 antigens	EUA granted by the FDA
Other Trials and Actions
CARsgen Therapeutics	CT041	claudin18.2-positive solid tumors	IND approved by the FDA
Acepodia	ACE1702	HER2-expressing solid tumors	first patients dosed in phase 1 trial
Arrowhead Pharmaceuticals	ARO-HIF2	clear-cell renal cell carcinoma	first patients dosed in phase 1b trial
Enterprise Therapeutics	ETD002	all cystic fibrosis patients, independent of genotype	first patients dosed in phase 1 trial
Epirium Bio	EPM-01	Becker muscular dystrophy	enrollment of first patients in phase 1 trial
Immunic	IMU-856	diarrhea-predominant irritable bowel syndrome (IBS-D), ulcerative colitis (UC) and Crohn's disease	dosing of first patients in phase 1 trial
I-Mab	plonmarlimab (TJM2)	rheumatoid arthritis	dosing of first patient in phase 1b trial
Pluristem Therapeutics	PLX-R18	incomplete hematopoietic recovery following hematopoietic cell transplantation	patient enrollment complete in phase 1 trial
Ultimovacs ASA	UV1 in combination with pembrolizumab	metastatic malignant melanoma	patient enrollment complete in phase 1 trial
4D Molecular Therapeutics	4D-125	X-linked retinitis pigmentosa	initiation of phase 1/2 trial
Trefoil Therapeutics	TTHX1114	regenerative treatment of corneal endothelial dystrophies	initiation of phase 1/2 trial
Alkermes	ALKS 4230	advanced, malignant solid tumors	initiation of phase 2 trial
Amplyx	MAU868	BK viremia in kidney transplant recipients	dosing of first patient in phase 2 trial
Ascletis Pharma	ASC22 (Envafolimab)	chronic hepatitis B	dosing of first patient in phase 2a trial
AzurRx	MS1819	cystic fibrosis patients with exocrine pancreatic insufficiency	dosing of first patients in phase 2b trial
Imara	IMR-687	adult patients with sickle cell disease	dosing of first patient in phase 2b trial
Neuroptika	NRO-1	dry eye disease	patient enrollment complete in phase 2 trial
Verona Pharma	pressurized metered-dose inhaler (pMDI) formulation of ensifentrine	moderate to severe chronic obstructive pulmonary disease	initiation of phase 2 trial
BioLineRx	motixafortide (BL-8040)	mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma	patient enrollment complete in phase 2 trial
Endospan	NEXUS Aortic Arch Stent Graft System	thoracic aortic arch disease	IDE approved by the FDA
4D Molecular Therapeutics	4D-310	Fabry Disease	Fast Track designation granted by the FDA
Akeso Biopharma	AK104	patients with recurrent or metastatic squamous cervical cancer who have disease progression on or after platinum-based chemotherapy	Fast Track designation granted by the FDA
Precision Biosciences	PBCAR0191	advanced B-cell precursor acute lymphoblastic leukemia	Fast Track designation granted by the FDA
Mustang Bio	MB-107	X-linked severe combined immunodeficiency in newly diagnosed infants	Rare Pediatric Disease designation granted by the FDA
Scholar Rock	SRK-015	spinal muscular atrophy	Rare Pediatric Disease designation granted by the FDA
Genentech	Enspryng (satralizumab-mwge)	adults with AQP4 antibody-positive neuromyelitis optica spectrum disorder	approved by the FDA
RapidAI	Rapid LVO	detecting suspected large vessel occlusions	approved by the FDA