All of our current research and development programs, including our five clinical product candidates, 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 can address targets not amenable to intervention by traditional carbon based compounds. Second, boron’s reactivity allows boron based compounds to interact with a biological target to create a change that is specific to a particular disease or condition.
Despite the ubiquity of boron in the environment, research to evaluate the therapeutic promise of boron based compounds has been limited. This lack of research activity was primarily due to the availability of carbon based natural products as the starting points for small molecule drug discovery. In addition, the evaluation of boron based compounds as product candidates has been hampered by an insufficient understanding of the physical properties necessary to provide boron based compounds with the chemical and biological attributes required of pharmaceutical therapies. Boron based compounds have also been historically difficult to synthesize.
Significant advances have recently been made in the science and practice of boron based drug discovery. We have developed an expertise and understanding of the interactions of boron based compounds with key biological targets relevant to treating human disease. This know-how has enabled the efficient optimization of disease modifying properties for our lead compounds and their rapid progression from the research stage into clinical trials.
Additionally, we have discovered new methods of synthesis of 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 that have not been effectively addressed by carbon based compounds and develop antibiotic or antifungal therapies that kill pathogens that have become resistant to existing drugs.
Broad utility across multiple disease areas. In addition to our core therapeutic areas of dermatology and anti-infectives, our compounds have exhibited extensive preclinical activity in multiple disease areas, including diabetes, cancer and ophthalmic indications.
Rapid and efficient synthesis of drug-like compounds. Our recent 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. These advances have made manufacturing of boron based compounds feasible on a commercial scale.
Unencumbered intellectual property landscape. We believe the intellectual property landscape for boron based pharmaceutical products is relatively unencumbered compared to that of carbon based products, providing an attractive opportunity for us to build our intellectual property portfolio.
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