Endocrine signaling pathways use peptide hormones to regulate many physiological functions including growth, energy utilization, metabolism, gastrointestinal function and stress responses. Diseases arise when these pathways are disrupted by injury, surgery, genetic mutations, tumors (such as NETs), inflammation or senescence.
Typical treatment of endocrine diseases has historically entailed administration of unphysiological doses of hormones or antagonists to replace, supplement or block endogenous biology. The goal of the therapeutic intervention is to restore homeostasis and appropriate endocrine function.
An attraction in developing drugs for endocrine diseases is that the biological pathways are largely well understood, and the disease targets are appreciably validated. A common challenge is that native hormones have relatively short time-action and therefore require frequent administration or pump-based infusion, and often in large amounts. The therapeutic window can be appreciably narrow such that the dose required to achieve full efficacy is insufficiently separated from the tolerated dose required to adequately restore homeostasis and proper biological function.
To overcome these challenges, MBX Biosciences is advancing novel peptide hormone analogs by applying its world-class chemistry to address rare endocrine diseases. Our first disease indications are poorly managed with conventional drugs, but possess highly validated molecular targets to support drug discovery and development.