Pipeline

March 8, 2021

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
eXIthera Pharmaceuticals	EP-7041	thrombosis prevention in critically ill patients with COVID-19	IND approved by the FDA for phase 2 trial
Sorrento Therapeutics	intranasal STI-2099 (COVIDROPS)	mild COVID-19	IND approved by the FDA
Altimmune	AdCOVID	single-dose intranasal COVID-19 vaccine	commencement of patient enrollment in phase 1 trial
AIVITA Biomedical	AV-COVID-19	personalized COVID-19 vaccine	completion of phase 1 trial
Novavax Takeda	NVX-CoV2373	COVID-19 vaccine	first patients dosed in phase 2 trial in Japan
Johnson & Johnson	single-shot COVID-19 vaccine	COVID-19	Emergency Use Authorization granted by the FDA
Other Trials and Actions
Flexion Therapeutics	FX301	postoperative pain in patients undergoing bunionectomy	IND approved by the FDA
Molecular Targeting Technologies	EBTATE (177Lu-DOTA-EB-TATE)	neuroendocrine tumors	IND approved by the FDA
QureBio (no website available)	Q-1802	advanced solid tumors	IND approved by the FDA
TissueTech	TTBT01	dry eye disease	IND approved by the FDA for phase 2 trial
Hangzhou Sciwind Biosciences	XW003	nonalcoholic steatohepatitis	IND approved by China’s National Medical Products Administration
Wellmarker Bio	WM-S1-030	advanced cancer	trial approved by the Australian Therapeutic Goods Administration
Innovent	IBI322	advanced malignancies	first patient dosed in phase 1a trial in the U. S
Prometheus Biosciences	PRA023	inflammatory bowel disease	first patients dosed in multiple ascending-dose (MAD) portion of phase 1a trial
AbFero Pharmaceuticals	SP-420	myelodysplastic and myelofibrosis patients with transfusional iron overload	initiation of phase 1 trial
Apollomics	APL-106 (uproleselan injection)	relapsed or refractory acute myeloid leukemia	first patient enrolled in phase 1 trial in China
Forendo Pharma	FOR-6219	endometriosis	completion of phase 1 trials
Kymera Therapeutics	KT-474	atopic dermatitis or hidradenitis suppurativa	initiation of patient dosing in single ascending-dose (SAD) portion of phase 1 trial
Pipeline Therapeutics	PIPE-307	multiple sclerosis	initiation of phase 1 trial
Clearside Biomedical	CLS-AX (axitinib injectable suspension)	neovascular age-related macular degeneration	completion of patient dosing in first cohort of phase 1/2a trial
Cyclo Therapeutics	Trappsol Cyclo	Niemann-Pick disease type C1	completion of last patient last visit in phase 1/2 trial
Kronos Bio	KB-0742	MYC gene-amplified solid tumors	first patient dosed in phase 1/2 trial
Transgene BioInvent	BT-001	solid tumors	first patients enrolled in phase 1/2 trial
Dicerna Roche	RG6346	chronic hepatitis B virus infection	initiation of phase 2 trial
Highlight Therapeutics	BO-112	patients with unresectable or metastatic melanoma that have previously progressed to checkpoint inhibitors	first patients enrolled in phase 2a trial
Annexon	ANX007	geographic atrophy	first patients dosed in phase 2 trial
Assembly Biosciences Arbutus Biopharma	vebicorvir in combination with AB-729	chronic hepatitis B infection	initiation of phase 2 trial
BioMarin Pharma	vosoritide	achondroplasia	patient enrollment complete in phase 2 trial
BiomX	BX001	mild-to-moderate acne	first patient dosed in phase 2 trial
BioXcel Therapeutics	BXCL501	delirium-related agitation	initiation of phase 2 trial
Marker Therapeutics	MT-401	patients with acute myeloid leukemia following an allogeneic stem-cell transplant in both the adjuvant and active disease settings	first patient treated in phase 2 trial
Ocular Therapeutix	OTX-DED (dexamethasone intracanalicular ophthalmic insert)	short-term treatment for signs and symptoms of dry eye disease	first patient dosed in phase 2 trial
Pharvaris	PHVS416	hereditary angioedema due to C1-inhibitor deficiency type 1 and 2	first patient dosed in phase 2 trial
Longeveron	Lomecel-B infusion	aging frailty	completion of phase 2b trial
Seres Therapeutics	SER-287	mild-to-moderate ulcerative colitis	completion of patient enrollment in phase 2b trial
ADial Therapeutics	AD04	alcohol use disorder in persons with certain target genotype	50 percent enrollment reached in phase 3 trial
I-Mab	eftansomatropin alfa (TJ101)	pediatric growth hormone deficiency	first patient dosed in phase 3 trial in China
Exicure	cavrotolimod (AST-008)	Merkel cell carcinoma	Orphan Drug designation granted by the FDA
Oblato	OKN-007	diffuse intrinsic Pontine glioma	Fast-Track designation granted by the FDA
Anuncia	ReFlow System Mini	cerebrospinal fluid disorders requiring shunting	Breakthrough Device designation granted by the FDA
MedAlliance	SELUTION SLR, sustained limus release DEB catheter	atherosclerotic lesions in native coronary arteries	Breakthrough Device designation granted by the FDA
AbbVie	Humira (adalimumab)	moderate-to-severe active ulcerative colitis in children age 5 and older	approved by the FDA for expanded indication
Bridge BioPharma Origin Biosciences	Nulibry (fosdenopterin) for injection	molybdenum cofactor deficiency (MoCD) type A	approved by the FDA
KemPharm	Azstarys (serdexmethylphenidate)	attention deficit hyperactivity disorder (ADHD) in patients age 6 and older	approved by the FDA
Oncopeptides	Pepaxto (melphalan flufenamide	relapsed or refractory multiple myeloma	approved by the FDA
Pfizer	Lorbrena (lorlatinib)	first-line treatment of people with anaplastic lymphoma kinase (ALK)-positive nonsmall-cell lung cancer	approved by the FDA for expanded indication
Sarepta Therapeutics	Amondys 45 (casimersen)	Duchenne muscular dystrophy patients with a confirmed mutation amenable to exon 45 skipping	approved by the FDA
Accufix Surgical	Accu-Joint system	patients with degenerative and posttraumatic arthritis in the metatarsophalangeal joint	approved by the FDA
Vascular Grafts Solutions	VIOLA device	clampless proximal anastomosis in coronary artery bypass grafting	approved by the FDA