Drug & Device Pipeline News

December 4, 2023

This week’s Pipeline features a phase 2 trial approval for early Alzheimer’s disease, a phase 1 trial start for Type 1 diabetes and an FDA approval for a treatment for HR+ HER2- locally advanced or metastatic breast cancer.

Company	Drug/Device	Medical Condition	Status
Trials Authorized
Carisma Therapeutics	CT-0525	HER2+ solid tumors	IND for a phase 1 trial approved by the FDA
HOOKIPA Pharma	HB-500	HIV	IND for a phase 1 trial approved by the FDA
Nexcella	NXC-201	Relapsed/refractory primary or light chain (AL) amyloidosis	IND for a phase 1 trial approved by the FDA
Ethris	ETH47 inhaled mRNA vaccine	Respiratory viral infections	Approval for a phase 1 trial granted by the UK’s regulatory authority
ProJenX	Prosetin	Amyotrophic lateral sclerosis	Approval for a phase 1 trial granted by Health Canada
TC BioPharm	TCB-008	Relapsed/refractory acute myeloid leukemia	IND for a phase 1b trial approved by the FDA
Gracell Biotechnologies	FasTCAR-T GC012F	Refractory systemic lupus erythematosus	IND for a phase 1/2 trial approved by the FDA
KSQ Therapeutics	KSQ-001EX	Melanoma, head and neck squamous cell carcinoma and non-small cell lung cancer	IND for a phase 1/2 trial approved by the FDA
Senhwa Biosciences	Silmitasertib (CX-4945)	Community-acquired pneumonia caused by viral infection	IND for a phase 2 trial approved by the FDA
Omnix Medical	OMN6	Hospital-acquired bacterial pneumonia or ventilator-associated bacterial pneumonia caused by Acinetobacter baumannii complex	IND for a phase 2 trial approved by the FDA
Lighthouse Pharmaceuticals	LHP588	P. gingivalis-positive Alzheimer's disease	IND for a phase 2 trial approved by the FDA
Alzamend Neuro	AL001	Major depressive disorder	IND for a phase 2a trial approved by the FDA
GRIbio	GRI-0621	Idiopathic pulmonary fibrosis	IND for a phase 2a trial approved by the FDA
INmune Bio	XPro	Early Alzheimer’s disease	Approval for a phase 2 trial granted by France and Spain’s regulatory authorities
RTI Surgical	Cortiva Allograft Dermis	Breast reconstruction	IDE approved by the FDA
Trials Initiated
Asklepios BioPharmaceutical	AB-1005 gene therapy	Multiple system atrophy -parkinsonian type	Initiation of a phase 1 trial
Chengdu Origen Biotechnology	KH631 gene therapy	Wet age-related macular degeneration	Initiation of a phase 1 trial
Longboard Pharmaceuticals	LP659	Rare neuroinflammatory conditions	Initiation of a phase 1 trial
Neumora Therapeutics	NMRA-266	Schizophrenia and other neuropsychiatric disorders	Initiation of a phase 1 trial
OncoNano Medicine	ONM-501	Advanced solid tumors and lymphomas	Initiation of a phase 1 trial
TOLREMO therapeutics	TT125-802	Solid tumors	Initiation of a phase 1 trial
ProMIS Neurosciences	PMN310	Alzheimer’s disease	Initiation of a phase 1a trial
SAB Biotherapeutics	SAB-142	Type 1 diabetes	Initiation of a phase 1 trial in Australia
MicuRx Pharmaceuticals	MRX-5	Nontuberculous mycobacteria infections	Initiation of a phase 1 trial in Australia
OncoResponse	OR502 alone and with anti-PD-1	Advanced solid tumors	Initiation of a phase 1/2 trial
ASC Therapeutics	ASC618 gene therapy	Hemophilia A	Initiation of a phase 1/2a trial
Ceapro	Avenanthramides	Inflammation-based diseases	Initiation of a phase 1/2a trial
Acadia Pharmaceuticals	ACP-204	Alzheimer's disease psychosis	Initiation of a phase 2 trial
CG Pharmaceuticals	Ivaltinostat	Metastatic pancreatic cancer	Initiation of a phase 2 trial
MC2 Therapeutics	MC2-25 VLS	Vulvar lichen sclerosis	Initiation of a phase 2 trial
Skye Bioscience	SBI-100 ophthalmic emulsion	Primary open-angle glaucoma or ocular hypertension	Initiation of a phase 2 trial
Escient Pharmaceuticals	EP262	Moderate-to-severe atopic dermatitis	Initiation of a phase 2a trial
Opsidio	OpSCF	Moderate-to-severe atopic dermatitis	Initiation of a phase 2a trial
ADvantage Therapeutics	AD04	Mild Alzheimer’s disease	Initiation of a phase 2b trial in Europe
Acadia Pharmaceuticals	ACP-101	Hyperphagia in Prader-Willi syndrome	Initiation of a phase 3 trial
Approvals
AstraZeneca	Truqap (capivasertib) plus Faslodex (fulvestrant)	HR+, HER2- locally advanced or metastatic breast cancer with PIK3CA, AKT1 or PTEN alterations	Approved by the FDA
SpringWorks Therapeutics	Ogsiveo (nirogacestat)	Desmoid tumors in adults	Approved by the FDA
Vertex Pharmaceuticals CRISPR Therapeutics	Casgevy (exagamglogene autotemcel)	Sickle cell disease and transfusion-dependent beta thalassemia	Approved by the UK’s MHRA
Astellas Pharma Pfizer	Xtandi (enzalutamide)	Nonmetastatic castration-sensitive prostate cancer with biochemical recurrence at high risk for metastasis	Approved by the FDA for expanded indication
Merck	Keytruda (pembrolizumab)	First-line locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma	Approved by the FDA for expanded indication
Ascendis Pharma	Yorvipath (palopegteriparatide)	Chronic hypoparathyroidism	Approved in the European Union
Everest Medicines	Nefecon	Primary immunoglobulin A nephropathy	Approved in China
Medtronic	Symplicity Spyral renal denervation system	Hypertension	Approved by the FDA