Pipeline

August 31, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
ReiThera	GRAd-COV2	COVID-19 vaccine	initiation of phase 1 trial
SAB Biotherapeutics	SAB-185	COVID-19	dosing of first patient in phase 1b trial
Mereo Biopharma	alvelestat	hospitalized patients with moderate to severe COVID-19 respiratory disease	initiation of phase 1b/2 trial
Novavax	NVX-CoV2373	COVID-19 vaccine	initiation of phase 2 portion of phase 1/2 trial
Laurent Pharmaceuticals	LAU-7b	COVID-19	approval from the FDA to begin patient enrollment in phase 2 trial
Partner Therapeutics	inhaled Leukine (sargramostim, rhu-GM-CSF)	hospitalized COVID-19 patients with acute hypoxemia	first patient enrolled in phase 2 trial
Capricor Therapeutics	CAP-1002	COVID-19	IND approved by the FDA for phase 2 trial
Fluidigm	Advanta Dx SARS-CoV-2 RT-PCR Assay	extraction-free saliva-based test to detect SARS CoV 2 virus	Emergency Use Authorization (EUA) granted by the FDA
Other Trials and Actions
Aquestive Therapeutics	AQST-108, oral sublingual film formulation of epinephrine	allergic reactions, including anaphylaxis	initiation of phase 1 trial
Celldex Therapeutics	CDX-527	advanced or metastatic solid tumors that have progressed during or after standard of care therapy	initiation of phase 1 trial
Cerecor	CERC-002	adults with moderate-to-severe Crohn’s disease who have previously failed treatment with anti-TNF agents	patient enrollment resumed in phase 1b trial
CohBar	CB4211	nonalcoholic steatohepatitis and obesity	first subjects dosed in phase 1b stage of phase 1a/1b trial
Debiopharm	Debio 1124	advanced, unresectable pulmonary and extrapulmonary small cell carcinoma	first patient dosed in phase 1 trial
Krystal Biotech	KB301	acne scars and facial wrinkles	initiation of phase 1 trial
Silverback Therapeutics	SBT6050	HER2-expressing solid tumors	initiation of phase 1 trial
Oncolytics Biotech	pelareorep combined with retifanlimab	unresectable locally advanced or metastatic triple-negative breast cancer	dosing of first patient in phase 2 trial
Mitsubishi Tanabe Pharma	edaravone (MT-1186)	amyotrophic lateral sclerosis	patient enrollment resumed in phase 3 trial
Protalix Biotherapeutics	pegunigalsidase alfa (PRX–102)	Fabry disease	completion of treatment in phase 3 trial
NeoImmuneTech	NT-I7 (efineptakin alfa) and Kymriah (tisagenlecleucel)	relapsed/refractory large B cell lymphoma	IND approved by the FDA
REGENEX Bio	suprachoroidal delivery of RGX-314	diabetic retinopathy	IND approved by the FDA
Metacrine	MET409	nonalcoholic steatohepatitis	Fast-Track designation granted by the FDA
Rocket Pharmaceuticals	RP-L401 (lentiviral vector-based gene therapy)	infantile malignant osteopetrosis	Fast-Track designation granted by the FDA
Turning Point Therapeutics	repotrectinib	patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK tyrosine kinase inhibitors (TKIs) and have no satisfactory alternative treatments	Fast-Track designation granted by the FDA
Kazia Therapeutics	paxalisib (GDC-0084)	malignant glioma, including diffuse intrinsic pontine glioma	Orphan Drug designation granted by the FDA
Taysha Gene Therapies	TSHA-101 (AAV9-based gene therapy)	GM2 gangliosidosis	Orphan Drug designation and Rare Pediatric Disease designation granted by the FDA
AmideBio	AB-G023	congenital hyperinsulinism	Rare Pediatric Disease designation granted by the FDA
Editas Medicine	EDIT-301	sickle cell disease	Rare Pediatric Disease designation granted by the FDA
BrainsWay	deep transcranial magnetic stimulation system	short-term smoking cessation	approved by the FDA
Cassiopea	Winlevi (clascoterone cream 1%)	acne in patients 12 years and older	approved by the FDA
Gyroscope Limited	Orbit Subretinal Delivery System	microinjection into the subretinal space at the back of the eye	approved by the FDA
Janssen Amgen	Darzalex (daratumumab) in combination with Kyprolis (carfilzomib) and dexamethasone	patients with relapsed/refractory multiple myeloma who have received one to three previous lines of therapy	approved by the FDA for expanded indication
Novartis	Kesimpta (ofatumumab)	relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease	approved by the FDA
Pelvital	Flyte	strengthening of pelvic floor muscles for women with stress urinary incontinence	approved by the FDA
Recordati Rare Diseases	Cystadrops (cysteamine ophthalmic solution) 0.37%	cystinosis	approved by the FDA
SurGenTec	OsteoFlo NanoPutty- Quadphasic Synthetic Bone Graft	bone grafts	approved by the FDA