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Pipeline

December 7, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
Rhizen Pharmaceutical	RP7214	treatment of SARS-CoV-2 infection	IND approved by the FDA
City of Hope	COH04S1	SARS-CoV-2 vaccine	initiation of phase 1 trial
NeoImmuneTech	NT-I7 (efineptakin alfa)	adult patients with mild COVID-19	first patient dosed in phase 1 trial
Capricor Therapeutics	intravenous infusion of CAP-1002	patients with SARS-CoV-2 who require supplemental oxygen	first patients dosed in phase 2 trial
Clear Creek Bio	brequinar	nonhospitalized patients with SARS-CoV-2 who are symptomatic	first patient dosed in phase 2 trial
Edesa Biotech	EB05	hospitalized COVID-19 patients	first patient enrolled in phase 2/3 trial
Algernon Pharmaceuticals	NP-120 (Ifenprodil)	hospitalized patients with confirmed COVID-19	final patient enrolled in phase 2b/3 trial
Apilli Therapeutics	Avigan tablets (favipiravir)	post-exposure prophylaxis for COVID-19	initiation of phase 3 trial
Apilli Therapeutics	Avigan tablets (favipiravir)	patients with mild-to-moderate COVID-19 symptoms	initiation of phase 3 trial
ARCA Biopharma	AB201	patients hospitalized with COVID-19	Fast Track designation granted by the FDA
Mesoblast	remestemcel-L	acute respiratory distress syndrome (ARDS) due to COVID-19 infection	Fast Track designation granted by the FDA
Eli Lilly Incyte	Olumiant (baricitinib) plus remdesivir	hospitalized patients diagnosed with COVID-19 who require supplemental oxygen or ventilation	Emergency Use Authorization (EUA) granted by the FDA
Pfizer BioNTech	COVID-19 mRNA vaccine (BNT162b2)	COVID-19 vaccine	EUA granted in the UK
Regeneron	casirivimab and imdevimab	mild-to-moderate COVID-19 in adults, as well as in pediatric patients at least 12 years of age and weighing at least 88 pounds, who have received positive results of direct SARS-CoV-2 viral testing and are at high risk for progressing to severe COVID-19 and/or hospitalization	EUA granted by the FDA
Other Trials and Actions
F-star Therapeutics	FS222	cancer	clinical trial approved in Spain
Orchard Therapeutics	OTL-200	metachromatic leukodystrophy	IND approved by the FDA
GEMoaB	UniCAR-T-PSMA	advanced solid tumors	initiation of phase 1a trial
NeuroTherapia	NTRX-07	Alzheimer's Disease	completion of phase 1a trial
American Cryostem	ATCell Expanded Autologous, Adipose-Derived Mesenchymal Stem Cells	post-concussion syndrome in retired military and athletes	initiation of phase 1 trial
Bio-Path Holdings	BP1002	refractory/relapsed lymphoma and chronic lymphocytic leukemia	first patient dosed in phase 1 trial
Codagenix	CodaVax-RSV vaccine	prevention of respiratory syncytial virus	patient dosing complete in phase 1 trial
F-star Therapeutics	FS120	advanced cancer	first patient dosed in phase 1 trial
IGC Pharma	IGC-AD1	mild-to-severe dementia due to Alzheimer’s disease	initiation of phase 1 trial
Junshi Biosciences	JS108	advanced solid tumors	first patient dosed in phase 1 trial
MapLight Therapeutics	ML-004	social deficit in autism spectrum disorder	patient dosing complete in phase 1 trial
Nordic Nanovector	Betalutin (177Lu lilotomab satetraxetan)	patients with relapsed/refractory diffuse large B-cell lymphoma not eligible for autologous stem cell transplantation	patient enrollment complete in phase 1 trial
North Sea Therapeutics	SEFA-1024	hypertriglyceridemia	first patient dosed in phase 1 trial
QPex Biopharma	QPX7728	drug resistant bacterial infections	initiation of phase 1 trial
Silicon Therapeutics	SNX281	advanced solid tumors or lymphoma	first patients dosed in phase 1 trial
Phoenix Molecular Designs	PMD-026	triple negative breast cancer	first patients dosed in phase 1b trial
Ascentage Pharma	APG-2575	relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma	first patient dosed in phase 1/2 trial in Europe
Inovio	INO-3107	recurrent respirator papillomatosis	first patient dosed in phase 1/2 trial
PMV Pharmaceuticals	PC14586	advanced solid tumors with the specific p53 Y220C variant	first patient dosed in phase 1/2 trial
RegenxBio	RGX-111	mucopolysaccharidosis Type I	first patient dosed in phase 1/2 trial
Sio Gene Therapies	AXO-AAV-GM1	Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis	first patient dosed in high-dose cohort of phase 1/2 trial
Turnstone Biologics Takeda	RIVAL-01/TAK-605	solid tumors	initiation of phase 1/2 trial
Apellis Swedish Orphan Biovitrum	pegcetacoplan	sporadic amyotrophic lateral sclerosis	first patient dosed in phase 2 trial
Apnimed	AD109	obstructive sleep apnea	first patient dosed in phase 2 trial
Athira Pharma	ATH-1017	mild-to-moderate Alzheimer's disease	first patients dosed in phase 2 trial
Cara Therapeutics	oral Korsuva (difelikefalin tablets)	moderate-to-severe pruritus in atopic dermatitis patients	patient enrollment complete in phase 2 trial
Istari Oncology	PVSRIPO in combination with Keytruda	recurrent glioblastoma multiforme	first patient dosed in phase 2 trial
Oramed	oral insulin capsule ORMD-0801	nonalcoholic steatohepatitis	initiation of phase 2 trial
Rafael Pharma	CPI-613 (devimistat) in combination with hydroxychloroquine	clear cell sarcoma of soft tissue	initiation of phase 2 trial
Verastem Oncology	VS-6766	recurrent low-grade serous ovarian cancer	initiation of phase 2 trial
Abivax	ABX464	ulcerative colitis	patient enrollment complete in phase 2b trial
Ionis Pharma AstraZeneca	ION449 (AZD8233)	dyslipidemia	initiation of phase 2b trial
3S Bio SOBI Selecta Biosciences	SEL-212	chronic refractory gout	initiation of phase 3 trial
Bausch Health and Bausch + Lomb	NOV03 (perfluorohexyloctane)	dry eye disease associated with Meibomian gland dysfunction	initiation of phase 3 trial
Bellerophon Therapeutics	INOpulse	fibrotic interstitial lung disease	first patient enrolled in phase 3 trial
Deciphera Pharmaceuticals	Quinlock	second-line gastrointestinal stromal tumor	patient enrollment complete in phase 3 trial
Insmed	brensocatib	bronchiectasis	first patient dosed in phase 3 trial
Ionis Pharmaceuticals	AKCEA-APOCIII-LRx	adult patients with familial chylomicronemia syndrome	initiation of phase 3 trial
Mycovia Pharmaceuticals	teseconazole (VT-1161)	recurrent vulvovaginal candidiasis	last patient visit complete in phase 3 trial
Oramed	oral insulin capsule ORMD-0801	type 2 diabetes	initiation of phase 3 trial
PTC Therapeutics	vatiquinone (PTC743)	Friedreich ataxia	initiation of phase 3 trial
Phathom Pharmaceuticals	vonoprazan	healing and maintenance of healing of erosive esophagitis; relief of heartburn	patient enrollment complete in phase 3 trial
Pfizer	marstacimab (PF-06741086)	severe hemophilia A or B, with or without inhibitors	first patient dosed in phase 3 trial
Principa Biopharma	rilzabrutinib	patients with moderate-to-severe pemphigus who are either newly diagnosed or relapsing with chronic disease	patient enrollment complete in phase 3 trial
RedHill Biopharma	RHB-204	pulmonary nontuberculous mycobacteria disease caused by Mycobacterium avium Complex (MAC)	initiation of phase 3 trial
Zymeworks	zanidatamab	previously treated HER2 gene-amplified biliary tract cancer	Breakthrough Therapy designation granted by the FDA
Moleculin Biotech	WP1066	diffuse intrinsic pontine glioma, medulloblastoma and atypical teratoid rhabdoid tumor	Rare Pediatric Disease designation granted by the FDA
Asklepios BioPharmaceutical Selecta Biosciences	MMA-101	isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations	Orphan Drug designation granted by the FDA
Catalyst Biosciences	Marzeptacog alfa	episodic bleeding in subjects with hemophilia A or B with inhibitors	Fast-Track designation granted by the FDA
RhoVac	RV001	prostate cancer	Fast-Track designation granted by the FDA
Protagonist Therapeutics	PTG-300	polycythemia vera	Fast-Track designation granted by the FDA
Theralase Technologies	Theralase	Bacillus Calmete Guérin unresponsive nonmuscle-invasive bladder cancer carcinoma In situ	Fast-Track designation granted by the FDA
Alnylam Pharmaceuticals	Oxlumo (lumasiran)	primary hyperoxaluria type 1	approved by the FDA
Armis Biopharma	VeriFixx Small Bone Implant	fixation of osteotomies and reconstruction of the lesser toes following correction procedures for hammertoe, claw toe and mallet toe	approved by the FDA
Cerus Corporation	INTERCEPT Blood System for Cryoprecipitation	treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency	approved by the FDA
Eiger Biopharmaceuticals	Zokinvy (lonafarnib)	Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies	approved by the FDA
Genentech	Xofluza (baloxavir marboxil)	post-exposure prevention of influenza for patients 12 years of age and older after contact with an individual who has the flu	approved by the FDA for expanded indication
Genentech	Gavreto (pralsetinib)	adults and children over 12 years of age with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory	approved by the FDA
Novartis	Xolair (omalizumab)	add-on maintenance treatment of nasal polyps in adults over 18 years of age with inadequate response to nasal corticosteroids	approved by the FDA for expanded indication
Profound Medical Group	Sonalleve	osteoid osteoma	approved by the FDA under a Humanitarian Device Exemption
Rhythm Pharmaceuticals	Imcivree (setmelanotide)	chronic weight management in adults and pediatrics over the age of six with obesity due to proopiomelanocortin, proprotein convertase subtilisin/kexin type 1 or leptin receptor deficiency confirmed by genetic testing	approved by the FDA
Viatris ViiV Healthcare	pediatric dolutegravir tablets for oral suspension, 10 mg	treatment of HIV-1 infected pediatric patients at least 4 weeks old and weighing at least 6.6 pounds	tentative approval granted by the FDA under the U.S. President's Emergency Plan for AIDS Relief
Y-mAbs Therapeutics	Danyleza (naxitamab-gqgk)	patients with relapsed or refractory high-risk neuroblastoma	approved by the FDA