Drug & Device Pipeline News

September 18, 2023

This week’s Pipeline features the first phase 3 trial approval for sarcopenia, a phase 1 trial start for neurological and neuropsychiatric conditions, and an FDA approval for a device to treat chronic limb-threatening ischemia.

Company	Drug/Device	Medical Condition	Status
Trials Authorized
Gain Therapeutics	GT-02287	GBA1-Parkinson’s disease	Approval for a phase 1 trial granted by Australia’s regulatory authority
Biosyngen	BRL03	Lung cancer, gastric cancer and other advanced solid tumors	IND for a phase 1/2 trial approved by the FDA
Tango Therapeutics	TNG348	BRCA1/2-mutant HRD+ cancers	IND for a phase 1/2 trial approved by the FDA
Aston Sci.	AST-201 (ovarian cancer vaccine) plus chemotherapy	Newly diagnosed stage III advanced ovarian cancer following surgical intervention	IND for a phase 2 trial approved by the FDA
Biophytis	Sarconeos (BIO101)	Sarcopenia	IND for a phase 3 trial approved by the FDA
Trials Initiated
Myeloid Therapeutics	MT-302	Advanced or metastatic epithelial tumors	Initiation of a phase 1 trial
Neurocrine Biosciences	NBI-1117570	Neurological and neuropsychiatric conditions	Initiation of a phase 1 trial
Replicate Bioscience	RBI-4000 (rabies vaccine)	Rabies	Initiation of a phase 1 trial
Vincerx Pharma	VIP943	Relapsed/refractory acute myeloid leukemia, myelodysplastic syndrome and B-cell acute lymphoblastic leukemia	Initiation of a phase 1 trial
Nuvectis Pharma	NXP900	Advanced solid tumors	Initiation of a phase 1a trial
Arbutus Biopharma	AB-101	Hepatitis B virus	Initiation of a phase 1a/1b trial
BioInvent International	Keytruda (pembrolizumab) plus BI-1206	Solid tumors	Initiation of a phase 1/2 trial
Daiichi Sankyo	DS-3939	Advanced solid tumors including non-small cell lung, breast, urothelial, ovarian, biliary tract and pancreatic cancer	Initiation of a phase 1/2 trial
Opus Genetics	OPGx-LCA5	Leber congenital amaurosis associated with mutations in the LCA5 gene	Initiation of a phase 1/2 trial
4D Molecular Therapeutics	4D-150	Diabetic macular edema	Initiation of a phase 2 trial
AnHeart Therapeutics	Safusidenib	Grades 2 or 3 recurrent/progressive mIDH1 glioma	Initiation of a phase 2 trial
Q32 Bio Horizon Therapeutics	Bempikibart	Severe alopecia areata	Initiation of a phase 2 trial
SciSparc	SCI-110	Tourette syndrome	Initiation of a phase 2b trial in Israel
VBI Vaccines	VBI-1901 vaccine	First recurrent glioblastoma	Initiation of a phase 2b trial
AN2 Therapeutics	Epetraborole	Treatment-refractory mycobacterium avium complex (MAC) lung disease	Initiation of phase 3 part of phase 2/3 trial
Invivyd	VYD222	Prevention of symptomatic COVID-19	Initiation of a phase 3 trial
Approvals
BioLineRx	Aphexda (motixafortide) plus filgrastim	Stem cell mobilization for multiple myeloma patients	Approved by the FDA
Pfizer BioNTech	Comirnaty (COVID-19 vaccine, mRNA) 2023-2024 formulation	COVID-19	Approved by the FDA for new formulation
Moderna	Spikevax (COVID-19 vaccine, mRNA)	COVID-19	Approved by the FDA for new formulation
BioRay Pharmaceutical	Anruixi (zuberitamab)	CD20-positive diffuse large B-cell lymphoma	Approved in China
LimFlow	LimFlow System	Chronic limb-threatening ischemia in patients with no suitable endovascular or surgical revascularization options	Approved by the FDA