All of our current research and development programs are based upon compounds that have been internally discovered using our boron chemistry platform.
Our technology platform is based on the use of boron chemistry to develop novel therapies. Boron is a naturally occurring element that is ingested frequently through consumption of fruits, vegetables, milk and coffee. Boron has two attributes that we believe provide compounds with drug-like properties. First, boron-based compounds have a unique geometry that allows them to have two distinct shapes, giving boron-based drugs the ability to interact with biological targets in novel ways and to address targets not amenable to intervention by traditional carbon-based compounds. Second, boron’s reactivity as compared to carbon allows us to design molecules that can hit targets that are difficult to inhibit with carbon chemistry.
Despite the ubiquity of boron in the environment, researchers have faced challenges in evaluating boron-based compounds as product candidates due to previously limited understanding of the physical properties necessary to provide boron-based compounds with the chemical and biological attributes required of pharmaceutical therapies as well as difficulty in chemical synthesis.
We have developed expertise and an understanding of the interactions of boron-based compounds with key biological targets relevant to treating disease. This know-how is primarily related to methods for modulating boron’s reactivity to optimize reactions with the target and minimize unwanted chemical reactivity. Our advances have enabled the efficient optimization of disease-modifying properties for our first approved product, KERYDIN, and our lead development candidate, crisaborole, as they have progressed from the research stage into the clinical stage, and, in the case of KERYDIN, the commercial stage.
Additionally, we have discovered new methods of synthesizing boron compounds, allowing for the creation of new compound families with broad chemical diversity and retention of drug-like properties. These new compound families expand the universe of biological targets that can be addressed by small molecule, boron-based compounds.
We believe our focus on boron-based chemistry provides us with multiple advantages in the small molecule drug discovery process. These advantages include:
Novel access to biological targets.
Due to the unique geometry and reactivity of boron-based molecules, our boron-based compounds are able to modulate existing biological targets and can address targets not amenable to intervention by traditional carbon-based compounds. This may enable us to treat diseases in addition to onychomycosis that have not been effectively addressed by carbon-based compounds.
Broad utility across multiple disease areas.
Our compounds have exhibited extensive preclinical activity in multiple disease areas, including inflammatory, fungal and bacterial diseases, which are our core areas of focus, as well as in neglected diseases and applications in animal health.
Rapid and efficient synthesis of drug-like compounds.
Our proprietary technological advances in the synthesis of boron-based compounds coupled with our rational drug design capabilities, have enabled us to rapidly create large families of boron-based compounds with drug-like properties. We believe that these advances have made manufacturing of boron-based compounds economical on a commercial scale.
intellectual property landscape.
We believe the intellectual property landscape for boron-based pharmaceutical products is less encumbered compared to that of carbon-based products, providing an attractive opportunity for us to build our intellectual property portfolio.