Pipeline

August 3, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
Denali Therapeutics Sanofi	DNL758 (SAR443122)	hospitalized adult patients with severe COVID-19 lung disease	first patients dosed in phase 1b trial
AI Therapeutics	LAM-002A	patients with confirmed COVID-19	initiation of phase 2 trial
Apogenix	asunercept	patients with severe COVID-19	clearance to initiate a phase 2 trial in Russia
Constant Therapeutics	TXA127	patients with COVID-19 who require oxygen but are not in the ICU	initiation of phase 2 trial
Pfizer BioNTech	BNT162b2 vaccine candidate	COVID-19	initiation of phase 2/3 trial
RedHill Biopharma	opaganib (Yeliva)	patients hospitalized with severe COVID-19 and pneumonia requiring treatment with supplemental oxygen	initiation of phase 2/3 trial
Moderna	mRNA-1273 vaccine candidate	COVID-19	dosing of first patients in phase 3 trial
ReAlta Life Sciences	RLS-0071	acute lung injury secondary to COVID-19	IND approved by the FDA
Color	COVID-19 testing without clinical monitoring	COVID-19	Emergency Use Authorization (EUA) granted by the FDA
Truvian Sciences	Easy Check COVID-19 IgM/IgG antibody test	COVID-19	EUA granted by the FDA
Gilead Sciences	remdesivir	patients 12 years and older with a body weight of at least 40 kg with severe symptoms of COVID-19 who have pneumonia and require extra oxygen to help them breathe	approved by Health Canada
Other Trials and Actions
4-D Molecular Therapeutics	4D-110	choroideremia	dosing of first patient in phase 1 trial
Chi-Med	HMPL-306	hematological malignancies	initiation of phase 1 trial
Cleave Therapeutics	CB-5339	patients with relapsed/refractory acute myeloid leukemia or relapsed/refractory intermediate or high-risk myelodysplastic syndrome	dosing of first patient in phase 1 trial
Pipeline Therapeutics	PIPE-505	sensorineural hearing loss associated with hearing speech in noisy environments	initiation of phase 1/2a trial
Repare Therapeutics	RP-3500	solid tumors with specific genome instability-related genomic alterations including those in ATM gene	dosing of first patient in phase 1/2 trial
Acurx Pharmaceutical	ibezapolstat	clostridioides difficile Infection	initiation of phase 2 trial
Amgen Arrowhead	AMG 890	patients with elevated levels of lipoprotein (a) (Lp(a))	initiation of phase 2 trial
Iterion Therapeutics	tegavivint	desmoid tumors	enrollment of first patients in phase 2a trial
NGM Biopharma	NGM621	patients with geographic atrophy secondary to age-related macular degeneration	initiation of phase 2 trial
Corcept Therapeutics	relacorilant combined with nab-paclitaxel (Abraxane)	metastatic, platinum-resistant ovarian cancer	patient enrollment complete in phase 2 trial
Corcept Therapeutics	relacorilant	Cushing’s syndrome caused by adrenal adenomas or hyperplasia	enrollment of first patient in phase 3 trial
Krystal Biotech	beremagene geperpavec (KB103)	dystrophic epidermolysis bullosa	initiation of phase 3 trial
Carisma Therapeutics	CT-0508	recurrent or metastatic HER2 overexpressing solid tumors after failure of approved HER2 targeted agents	IND approved by the FDA
NeoImmuneTech	NT-I7 (efineptakin alfa)	patients with locally recurrent squamous cell carcinoma of head and neck undergoing salvage surgery	IND approved by the FDA
Momenta Pharmaceuticals	nipocalimab	prevention of hemolytic disease of the fetus and newborn	Rare Pediatric Disease designation and Orphan Drug designation granted by the FDA
Protara Therapeutics	TARA-002	lymphatic malformations	Rare Pediatric Disease designation granted by the FDA
Imara	IMR-687	beta-thalassemia	Rare Pediatric Disease designation and Fast Track designation granted by the FDA
Black Diamond Therapeutics	BDTX-189	adult patients with solid tumors harboring an allosteric HER2 mutation or an EGFR or HER2 Exon 20 insertion mutation who have progressed following prior treatment and who have no satisfactory treatment options	Fast Track designation granted by the FDA
Checkmate Pharmaceuticals	CMP-001 in combination with nivolumab or pembrolizumab	initial treatment of patients with unresectable Stage III or Stage IV melanoma to prolong the time to disease progression and treatment of patients with unresectable or metastatic melanoma refractory to prior anti-PD-1 blockade to improve the overall tumor response rate	Fast Track designation granted by the FDA
Sarepta Therapeutics	SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)	Duchenne Muscular Dystrophy	Fast Track designation granted by the FDA
Inovio	INO-3107	recurrent respiratory papillomatosis	Orphan Drug designation granted by the FDA
Merus N.V.	Zenocutuzumab	pancreatic cancer	Orphan Drug designation granted by the FDA
Ocugen	OCU400	RHO mutation-associated retinal degeneration	Orphan Drug designation granted by the FDA
Regulus Therapeutics	RGLS4326	autosomal dominant polycystic kidney disease	Orphan Drug designation granted by the FDA
Vita Therapeutics	VTA-11	Duchenne Muscular Dystrophy	Orphan Drug designation granted by the FDA
Merck	MK-6482	patients with von Hippel-Lindau disease-associated renal cell carcinoma with nonmetastatic RCC tumors less than three centimeters in size, unless immediate surgery is required	Orphan Drug designation and Breakthrough Therapy Drug designation granted by the FDA
Takeda	pevonedistat	patients with higher-risk myelodysplastic syndromes	Breakthrough Therapy Drug designation granted by the FDA
AstraZeneca	Breztri Aerosphere (budesonide/glycopyrrolate/formoterol fumarate)	chronic obstructive pulmonary disease	approved by the FDA
Kite Pharma/Gilead Sciences	Tecartus (brexucabtagene autoleucel)	relapsed or refractory mantle cell lymphoma	approved by the FDA
Stratus Medical	Vesta RF Cannula	radiofrequency heat lesion procedures for the relief of pain	approved by the FDA