About Cathepsin S & Autoimmune Diseases

Cathepsin S plays a key role in the pathology of autoimmune disease and in the maintenance of neuropathic pain. Genetic and pharmacologic inhibition of cathepsin S results in amelioration of disease in pharmacology models.

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Cathepsin S inhibitor: VBY-129 for Treatment of Neuropathic Pain:

VBY-129, is a potent and selective cathepsin S inhibitor. Cathepsin S appears to be a key mediator of a process pivotal in developing neuropathic pain, and by inhibiting cathepsin S, VBY-129 should prevent or substantially reduce pain.

We have completed Phase 1 clinical trials in healthy volunteers, evaluating the safety and pharmacokinetics of the drug by oral administration. Biomarkers used in these trials confirmed robust inhibition of cathepsin S using a once dialing dosing regimen. In addition, VBY-129 was well tolerated at all doses we tested.

Once-daily dosing of VBY-129 achieves target concentration for inhibition of cathepsin S and blocks invariant chain processing in PBMCs from Phase I subjects

We believe VBY-129 has the potential to more effectively treat neuropathic pain without the side effects experienced with existing therapies, such as opioid drugs. We retain worldwide rights to VBY-129 in all indications. More than 15 million people in the United States and Europe suffer from chronic neuropathic pain. According to Datamonitor, in 2014, sales of neuropathic pain therapies are projected to exceed $2.4 billion in the United States.

VBY-129 for Treatment of Crohn’s Disease:

Cathepsin S also appears to be a critical enzyme in the modulation of the immune system and the development of autoimmune diseases. Of the autoimmune diseases, we have chosen to focus first on Crohn’s disease due to the elevated levels of cathepsin S observed in patients and the effectiveness of VBY-129 in a widely-used preclinical mouse model of Crohn’s disease.

We believe VBY-129 has the potential to treat Crohn’s disease in a more effective manner than current therapies and more convenient manner than current biological therapies. Crohn’s disease affects approximately 900,000 people in the United States and Europe. In 2013, Crohn’s disease therapies generated sales of more than $2.9 billion in the United States.