Research and Development
Our research and development efforts are currently focused on the metabolic dysfunction which we believe is at the heart of three core epidemic diseases – NASH, obesity, and diabetes – each with significant overlap and association with other age-related diseases. In addition, we also continue to explore the potential of our peptides to treat a range of other epidemic diseases with underlying metabolic dysfunction, including cancer, cardiovascular and CNS diseases.
Our core disease focus targets large markets with substantial unmet medical needs. The addressable US adult patient population for NASH ranges between 12 million and 29 million (a wide range, given the current difficulty in diagnosing this disease), and has no approved drugs for its treatment. Obesity has an addressable US adult patient population of 80 million and shares a strong overlap with NAFLD and NASH. The addressable US adult patient population for Type 2 Diabetes is 21 million, and while this market is relatively well established, substantial unmet medical needs remain, with significant additional room for new and effective therapies, either as standalone agents or in combination therapy.
Lead Program CB4211 and MDP Analogs
CB4211 is based on the naturally occurring peptide MOTS-c, an MDP discovered in 2012 by our founders and their academic collaborators, whose research has shown that MOTS-c plays a significant role in the regulation of metabolism. CB4211 is a novel and improved analog of the MOTS-c peptide that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 represents our “first in human MBT drug candidate,” with initiation of clinical trials targeted for mid 2018, and an activity readout expected in early 2019.
Using our technology platform, our team has discovered and identified more than 100 new MDPs encoded within the mitochondrial genome. Novel analogs of these MDPs are currently being evaluated and prioritized for continued development and partnership opportunities. Several of these more advanced new peptides have demonstrated promising therapeutic potential in disease models for diabetes, Alzheimer’s and cancer. Collectively, these novel peptides represent potential treatments for the underlying metabolic dysfunction associated with a wide spectrum of age-related diseases and their comorbidities.