Pipeline

January 18, 2021

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
Codagenix Serum Institute of India	COVI-VAC	single-dose, intranasal, live attenuated vaccine against SARS-CoV-2	first patient dosed in phase 1 trial
DalCor Pharmaceuticals	dalcetrapib	oral anti-viral treatment for COVID-19	initiation of phase 2 trial
Innovation Pharmaceuticals	Brilacidin	hospitalized COVID-19 patients	Fast-Track designation granted by the FDA for phase 2 trial
PerkinElmer	PerkinElmer New Coronavirus Nucleic Acid Detection Kit	testing of individuals without COVID-19 symptoms	Emergency Use Authorization (EUA) granted by the FDA
Quanterix	Simoa SARS-CoV-2 N Protein Antigen Test	detection of SARS-CoV-2 virus nucleocapsid protein	EUA granted by the FDA
Other Trials and Actions
Editas Medicine	EDIT-301	sickle cell disease	IND approved by the FDA
Vor Biopharma	VOR33	acute myeloid leukemia	IND approved by the FDA
Chipscreen Biosciences	chiauranib	second-line treatment of small-cell lung cancer	IND approved by China’s National Medical Products Administration (NMPA)
Gracell Biotechnologies	GC007g, allogeneic donor-derived anti-CD19 chimeric antigen receptor (CAR)-T cell therapy	B-cell acute lymphoblastic leukemia	IND approved by China’s NMPA
aTyr Pharma Kyorin Pharma	ATYR1923 (KRP-R120)	interstitial lung disease	completed last subject visit in phase 1 trial
Jounce Therapeutics	JTX-8064 as a monotherapy and in combination with either JTX-4014 or Keytruda	advanced solid tumors	initiation of phase 1 trial
Jupiter Orphan Therapeutics	JOTROL	rare diseases, including ataxias, lysosomal storage disorders and mitochondrial diseases	first patient dosed in phase 1 trial
OX2 Therapeutics	immunotherapy	recurrent glioblastoma	first patient dosed in phase 1 trial
Rezolute	RZ402	diabetic macular edema	initiation of phase 1 trial
Clearside Biomedical	CLS-AX (axitinib injectable suspension)	neovascular age-related macular degeneration (wet AMD)	first patients enrolled in phase 1/2a trial
Felix Biotechnology	YPT-01	chronic P. aeruginosa infections in cystic fibrosis	initiation of phase 1/2 trial
Terns Pharmaceuticals	TERN-101	nonalcoholic steatohepatitis	patient enrollment complete in phase 2a trial
Adverum Biotechnologies	ADVM-022	diabetic macular edema	patient enrollment complete in phase 2 trial
Cytokinetics	CK-274	hypertrophic cardiomyopathy	first patient dosed in cohort 2 of phase 2 trial
Immutep	eftilagimod alpha	head and neck cancer	patient enrollment complete in phase 2 trial
Cara Therapeutics	oral Korsuva (difelikefalin)	moderate-to-severe pruritus in patients suffering from notalgia paresthetica	initiation of phase 2 trial
OncoSec Medical	TAVO (tavokinogene telseplasmid) plus Opdivo (nivolumab)	neoadjuvant therapy prior to surgery in patients with operable, locally or regionally advanced melanoma	first patient dosed in phase 2 trial
Redhill Biopharma Cosmo Pharmaceuticals	Rifamycin SV-MMX 600mg	diarrhea-predominant irritable bowel syndrome	completion of phase 2 trial
REMD Biotherapeutics	volagidemab (REMD-477)	type 1 diabetes	patient enrollment complete in phase 2 trial
Mirum Pharmaceuticals	volixibat	primary sclerosing cholangitis	first patient enrolled in phase 2b trial
RhoVac	RV001	advanced prostate cancer	first patient enrolled in phase 2b trial
Kintara Therapeutics	VAL-083	newly diagnosed and recurrent glioblastoma	initiation of patient recruitment in phase 2/3 trial
Prilenia Therapeutics	pridopidine	amyotrophic lateral sclerosis	first patient enrolled in phase 2/3 trial
Arcutis Biotherapeutics	topical roflumilast cream (ARQ-151)	atopic dermatitis	initiation of two phase 3 trials
Blue Earth Diagnostics	18F-fluciclovine, a positron emission tomography imaging radiopharmaceutical	detecting recurrent brain metastases	first patient dosed in phase 3 trial
Denovo Biopharma	DB102 (enzastaurin)	patients with newly diagnosed glioblastoma multiforme	first patient dosed in phase 3 trial
Organogenesis	ReNu, cryopreserved amniotic suspension allograft	management of symptoms associated with knee osteoarthritis	first patient enrolled in phase 3 trial
Pfizer	PF-06939926	ambulatory male patients, ages 4 through 7, with Duchenne muscular dystrophy	first patient dosed in phase 3 trial
Rockwell Medical Wanbang Biopharmaceuticals	Triferic Dialysate	patients with chronic kidney disease requiring hemodialysis	first patient dosed in phase 3 trial
TauRx Pharmaceuticals	hydromethylthionine	mild cognitive impairment and mild-to-moderate Alzheimer’s disease	patient enrollment complete in phase 3 trial
Orchard Therapeutics	OTL-200, ex vivo autologous hematopoietic stem cell gene therapy	early-onset metachromatic leukodystrophy	Regenerative Medicine Advanced Therapy designation granted by the FDA
Gemini Therapeutics	GEM103	age-related macular degeneration	Fast-Track designation granted by the FDA
Leading Biosciences	LB1148	postoperative gastrointestinal dysfunction associated with pediatric heart surgery	Fast-Track designation granted by the FDA
Merus	Zenocutuzumab	patients with metastatic solid tumors harboring NRG1 gene fusions (NRG1+ cancers) that have progressed on standard-of-care therapy	Fast-Track designation granted by the FDA
Abbott	i-STAT Alinity TBI plasma test	rapid handheld traumatic brain injury blood test	approved by the FDA
ClearMind Biomedical	Axonpen System	illumination and visualization of intracranial tissue and fluids and the controlled aspiration of tissue and/or fluid during surgery	approved by the FDA