Drug & Device Pipeline News

October 2, 2023

This week’s Pipeline features a phase 1 trial approval for Alpha-1 antitrypsin deficiency, trial initiations for spinal muscular atrophy, spinal cord injury and obstructive sleep apnea, and another FDA approval for blockbuster diabetes drug Jardiance.

Company	Drug/Device	Medical Condition	Status
Trials Authorized
Krystal Biotech	KB408	Alpha-1 antitrypsin deficiency	IND for a phase 1 trial approved by the FDA
Akeso	AK117 plus azacitidine	Newly diagnosed higher-risk myelodysplastic syndromes	IND for a phase 2 trial approved by the FDA
AltruBio	ALTB-268	Ulcerative colitis	IND for a phase 2 trial approved by the FDA
Aligos Therapeutics	ALG-055009	Nonalcoholic steatohepatitis	IND for a phase 2a trial approved by the FDA
Trials Initiated
Entrada Therapeutics	ENTR-601-44	Duchenne muscular dystrophy in patients amenable to exon 44 skipping	Initiation of a phase 1 trial in the UK
Cantex Pharmaceuticals	Azeliragon	Treatment of patients refractory to first-line treatment of metastatic pancreatic cancer	Initiation of a phase 1/2 trial
Smart Immune	SMART101 allogeneic cell therapy	Acute leukemia and myelodysplasia syndrome	Initiation of a phase 1/2 trial in France
BioInvent	BI-1808	Advanced malignancies	Initiation of phase 2a portion of a phase 1/2a trial
Gracell Biotechnologies	GC012F	Relapsed/refractory multiple myeloma	Initiation of a phase 1b/2 trial
Armata Pharmaceuticals	AP-SA02	Staphylococcus aureus bacteremia	Initiation of phase 2a portion of a phase 1b/2a trial
NervGen Pharma	NVG-291	Spinal cord injury	Initiation of a phase 1b/2a trial
Kallyope	K-757 and K-833	Obesity and type 2 diabetes	Initiation of a phase 2 trial
NMD Pharma	NMD670	Spinal muscular atrophy	Initiation of a phase 2 trial
Zucara Therapeutics	ZT-01	Preventing nighttime hypoglycemia in type 1 diabetics	Initiation of a phase 2a trial
Cantex Pharmaceuticals	Azeliragon	Decreasing the incidence of acute kidney injury and other life-threatening complications in patients hospitalized for COVID-19	Initiation of phase 3 portion of a phase 2/3 trial
Apnimed	AD109 (aroxybutynin/atomoxetine)	Obstructive sleep apnea	Initiation of a phase 3 trial
YS Biopharma	PIKA vaccine	Rabies	Initiation of a phase 3 trial
CymaBay Therapeutics	Seladelpar	Cirrhosis due to primary biliary cholangitis	Initiation of a phase 3b/4 trial
Approvals
Viatris Ocuphire Pharma	Ryzumvi (phentolamine ophthalmic solution) 0.75 percent	Pharmacologically induced mydriasis produced by adrenergic agonists or parasympatholytic agents	Approved by the FDA
Boehringer Ingelheim Eli Lilly	Jardiance (empagliflozin)	Chronic kidney disease	Approved by the FDA for new indication
Saptalis Pharmaceuticals	Likmez (metronidazole oral suspension)	Bacterial infections	Approved by the FDA for new formulation
AbbVie	Tepkinly (epcoritamab	Relapsed/refractory diffuse large B-cell lymphoma in adults	Approved by the European Commission