Ensemble's platform is a product engine for developing Ensemblins, which are synthetically engineered macrocycles – a class of compounds that have traditionally been obtained from natural sources, resulting in over 100 marketed macrocycle drugs. Now, Ensemble is the first company to successfully and reliably engineer and exploit synthetic macrocycle drugs.
Macrocyclic drugs have unique features:
These unique features result in benefits in three distinct areas of value for Ensemblins:
- Their ring structure confers conformational rigidity, the ability to cross cell membranes (to address intra-cellular targets), and the potential to bind to disease-relevant proteins.
- Ensemblins are synthetic macrocycles with a ring of 14 or more atoms that are designed with properties of binding affinity, target selectivity, and metabolic stability.
- They are larger than traditional small molecules giving them greater ability to bind selectively to extended protein surfaces.
- These combined properties enable their ability to address more difficult drug discovery targets such as those involving protein-protein interactions, both inside and outside cells.
- Distinct pharmaceutical properties: Ensemblins can have traditional small molecule drug-like oral bioavailability enabling convenient oral administration.
- Target affinity and selectivity: Ensemblins are able to bind to protein targets with surfaces that are not addressed by conventional small molecules thus opening up a range of current unexploited targets.
- Versatility: Ensemble can engineer Ensemblins to bind to a variety of targets for different disease indications, e.g., antibiotics, immune modulators, anticancer.
To date, Ensemble has developed a pipeline of Ensemblin drug series that target both biologic-accessible targets (i.e., oral anti-inflammatory such as RA, Crohn's) and undruggable intracellular targets (i.e., BCL- cancer, PTP1B – diabetes, obesity, cancer). These include:
- Disruptors of protein-protein interactions
- Novel protease inhibitors
- Novel phosphatase inhibitors