Natural product molecules (and derivatives) are important for the development of modern drugs, as they play a vital role in the pipeline for new therapeutics. For example, >100 FDA-approved drugs are derived from tetracyclic terpenoids, which can be metabolized by the body into steroids. Despite this success, there remain considerable limitations to broad medicinal exploitation of the class due to lingering scientific challenges associated with compound availability. Carbacyclix has developed a concise asymmetric route to forging natural and unnatural (enantiomeric) C19 and C20 tetracyclic terpenoid skeletons suitable to drive medicinal exploration. While efforts have been focused on establishing the chemical science, early investigations reveal that the emerging chemical technology can deliver compositions of matter that are potent and selective agonists of the estrogen receptor beta.
Tuberculosis (TB) is the leading infectious disease cause of death worldwide, including in persons with HIV infection. Drug-resistant TB, an increasingly global epidemic, requires prolonged treatment with toxic, expensive drugs. BCG, the only vaccine currently in use for the prevention of TB, is very effective for the first few years of life but loses efficacy after 10-15 years. The only new vaccine to have shown efficacy in humans in a fully-powered Phase 3 trial is the booster vaccine being developed by investigators at Dartmouth College. DAR-901 is the most advanced and promising candidate in the global portfolio. Pre-clinical studies include a tuberculosis challenge study indicating DAR-901 is superior to the BCG booster vaccine.