Pipeline

August 17, 2020

Company	Drug/Device	Medical Condition	Status
COVID-19 Trials and Actions
SAB Biotherapeutics	SAB-185	COVID-19	first patient dosed in phase 1 trial
Arcturus Therapeutics	ARCT-021 investigational vaccine	COVID-19	patient dosing complete in first cohort of phase 1/2 trial
Kamada	plasma-derived immunoglobulin (IgG) product	hospitalized, non-ventilated COVID-19 patients with pneumonia	first patient enrolled in phase 1/2 trial
Biophytis	Sarconeos (BIO101)	COVID-19 patients with respiratory failure	approval for a phase 2/3 trial granted by the Brazilian Health Regulatory Agency
Romark	NT-300 (nitazoxanide extended-release tablets)	mild or moderate COVID-19	initiation of phase 3 trial
Organicell Regenerative Medicine	Organicell Flow	mild to moderate respiratory distress due to COVID-19	two emergency INDs approved by the FDA
Baxter	HF20 Set and ST Set used in continuous renal replacement therapy (CRRT)	renal replacement patients of low weight and low blood volume who cannot tolerate a larger extracorporeal circuit volume in an acute care environment during the COVID-19 pandemic	Emergency Use Authorization (EUA) granted by the FDA
Helix	Helix COVID-19 next-generation sequencing test	SARS-CoV-2 in upper respiratory specimens	EUA granted by the FDA
Appili Therapeutics	favipiravir	COVID-19	IND approved by the FDA
Other Trials and Actions
Altimmune	NasoShield, a single dose intranasal anthrax vaccine	anthrax exposure	patient enrollment complete in phase 1b trial
Arrowhead Pharmaceuticals	ARO-ENaC	cystic fibrosis	first patients dosed in phase 1/2 trial
Oncopeptides	melflufen and dexamethasone	patients with immunoglobulin light chain (AL) amyloidosis following at least one prior line of therapy	first patient enrolled in phase 1/2 trial
Sapience Therapeutics	ST101	adults with unresectable and metastatic solid tumors who are not eligible for other therapies or have progressed on prior therapies	first patient dosed in phase 1/2 trial
Vaccibody AS Nektar Therapeutics	bempegaldesleukin with VB10.NEO personalized neoantigen cancer vaccine	advanced squamous cell carcinoma of the head and neck	first patient dosed in phase 1/2a trial
BioPath Holdings	prexigebersen (BP1001) in combination with frontline therapy decitabine and venetoclax	acute myeloid leukemia	enrollment and dosing of the first patient in stage 2 of phase 2 trial
Gyroscope Therapeutics	GT005	geographic atrophy secondary to dry age-related macular degeneration	initiation of phase 2 trial
Eloxx Pharmaceuticals	ELX-02	cystic fibrosis	enrollment resumed in phase 2 trial
Treadwell Therapeutics	CFI-400945 in combination with durvalumab	advanced or metastatic triple negative breast cancer	initiation of phase 2 study
Seelos Therapeutics	SLS-005 (trehalose)	amyotrophic lateral sclerosis	approval of phase 2b/3 study granted by the FDA
Boehringer Ingelheim	nintedanib	children and adolescents age 6 to 17 with clinically significant fibrosing interstitial lung disease	initiation of phase 3 trial
Fibrogen	Pamrevlumab combined with systemic corticosteroids	nonambulatory patients with Duchenne muscular dystrophy	initiation of phase 3 trial
Rafael Pharmaceuticals	CPI-613 (devimistat) in combination with modified FOLFIRINOX (mFFX)	metastatic pancreatic cancer	patient enrollment complete in phase 3 trial
American Gene Technologies	AGT103-T	HIV	IND approved by the FDA
India Globalization Capital	IGC-AD1	mild to severe dementia due to Alzheimer’s disease	IND approved by the FDA
Kahr Medical	DSP107	advanced solid tumors	IND approved by the FDA
LogicBio	LB-001 (in vivo gene editing program)	pediatric patients with methylmalonic acidemia	IND approved by the FDA
Tonix Pharmaceuticals	TNX-102 SL	alcohol use disorder	IND approved by the FDA for phase 2 trial
Aquestive Therapeutics	AQST-108 oral sublingual systemic epinephrine film	allergic reactions (Type 1), including anaphylaxis	Fast Track designation granted by the FDA
Kazia Therapeutics	paxalisib (formerly GDC-0084)	diffuse intrinsic pontine glioma	Rare Pediatric Disease designation granted by the FDA
Bayer	Lampit (nifurtimox)	pediatric patients (birth to age 17) for the treatment of Chagas disease (American trypanosomiasis) caused by Trypanosoma cruzi (T. cruzi).	approved by the FDA
Guardant Health	Guardant360 CDx	comprehensive genomic profiling in patients with solid tumors	approved by the FDA
Genentech PTC Therapeutics	Evrysdi (risdiplam)	spinal muscular atrophy in adults and children age two months and older	approved by the FDA
joimax	Intrac electromagnetic navigation tracking and control system	minimally invasive spinal surgery	approved by the FDA
Life Spine	PLATEAU-X Ti Lateral Lumbar Spacer System	surgical treatment of spinal disorders	approved by the FDA
NS Pharma	Viltepso (viltolarsen) injection	patients with Duchenne muscular dystrophy who are amenable to exon 53 skipping therapy	approved by the FDA
Trevena	Olinvyk (oliceridine)	moderate to severe acute pain in adults, where the pain is severe enough to require an intravenous opioid and for whom alternative treatments are inadequate	approved by the FDA
ViiV Healthcare Shionogi	Dovato (dolutegravir/lamivudine)	complete regimen for the treatment of HIV-1 infection to replace the current antiretroviral (ARV) regimen in adults who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable ARV regimen with no history of treatment failure and no known resistance to the individual components of Dovato	approved by the FDA for expanded indication