BioTheryX, Inc., founded by the drug development team behind the remarkable IMiDs® franchise of compounds, is applying its extensive and proven commercial success to deliver efficacious therapies to patients with unmet medical needs.
The identification of Cereblon (an E3 ligase) as a key target of the IMiDs® (the most successful class of anti-cancer drug in the world) showed that targeting protein degradation is a viable drug development strategy that can lead to enormous commercial success. With unmatched commercial and scientific experience, the BioTheryX drug development team is building a set of high value assets in protein modulation known as Protein Homeostatic Modulators (PHM®). These novel small molecule cereblon binders have clinically relevant substrate degradation profiles that offer vast therapeutic opportunities.
While PHM® compounds have significant potential as novel small molecule therapeutics; they have a broad range of molecular orientations when bound to cereblon, providing a new level of structural control in the creation of chimeric molecules that degrade high-value clinical targets with unrivaled probability of success and robust commercial freedom to operate. Our lead PHM®-based chimeras have shown excellent preclinical efficacy.
A critical approach in curing leukemias such as AML is eradication of the leukemic or cancer stem cells. Our most advanced clinical lead is a novel kinase inhibitor that blocks a specific leukemic stem cell target as well as a super-enhancer target preventing transcription of key oncogenic genes. This lead molecule has demonstrated remarkable preclinical animal efficacy implying the eradication of AML stem cells, and the potential for cures in certain malignancies. Preclinical toxicology showed good tolerance at therapeutic doses.
“The platform discovered by BioTheryX is exciting and broad, and can be applied to any substrate at will. It opens a wide window to the development of novel therapeutic modalities to a broad array of diseases, including malignancies and neurodegenerative disorders. Therefore, we are excited to be part of this novel and promising emerging approach" -Professor Aaron Ciechanover, Nobel Laureate in Chemistry (2004) for the Discovery of Ubiquitin Mediated Protein Degradation