Iecure Inc. has received clearance from the Australian Therapeutic Goods Administration (TGA) of the company’s clinical trial notification (CTN) for ECUR-506 (formerly GTP-506), an investigational therapy in development for the treatment of ornithine transcarbamylase (OTC) deficiency in pediatric (or neonatal) patients.

Fibroblast activation protein (FAP) is a serine protease that is highly overexpressed in the stroma of most epithelial-derived tumors – including pancreatic, breast and colorectal cancers – in the context of cancer-associated fibroblasts (CAFs).

Simultaneous inhibition of phosphoinositide 3-kinase (PI3K) and histone deacetylase (HDAC) is considered a good strategy in cancer treatment, although current PI3K/HDAC dual inhibitors have limitations, such as low selectivity and moderate toxicity levels.

A new degrader strategy has been previously proposed to mitigate platelet toxicity associated with Bcl-xL degraders. This strategy consists of selectively degrading Bcl-xL by the von Hippel-Lindau protein (VHL) E3 ligase in tumor cells, but not in platelets, which minimally express VHL. DT-2216 was developed as the first Bcl-xL degrader of this kind; however, this clinical candidate has still shown some platelet toxicity in vivo.

Additional early-stage research and drug discovery news in brief, from: Ankyra Therapeutics, Kineta, Transcenta, Transcode Therapeutics.