Verseon’s drug discovery program, based on an industry-leading in silico platform, progresses four programs with multiple novel compounds in anticoagulation, hereditary angioedema (HAE), diabetic macular edema (DME), and oncology.
Anticoagulant Program
Verseon has submitted the Phase I protocol for its lead precision oral anticoagulant (PROAC) candidate, VE-1902, and expects to start first-in-human trials in Q3 2018.
Precision oral anticoagulants (PROACs) are potent, highly selective, reversible covalent inhibitors of thrombin. They have shown excellent efficacy and low bleeding risk in multiple preclinical studies but do not disrupt platelet function. Therefore, they may be the first oral anticoagulants suitable for long-term anticoagulant-antiplatelet combination therapy. Such treatment is particularly relevant for tens of millions of patients worldwide with acute coronary syndrome or non-valvular atrial fibrillation comorbid with coronary artery disease.
The second PROAC candidate in development, VE-2851, is expected to enter the clinic in 2019.
Diabetic Macular Edema (DME) program
Verseon also detailed progress with its oral plasma kallikrein inhibitor candidates for the treatment of diabetic macular edema (DME). These small-molecule drug candidates are suitable for oral dosing and could replace or complement the current standard DME treatment, which is recurrent eye injections. An oral treatment would also be suitable for ongoing prophylactic treatment.
Numerous preclinical studies have demonstrated the efficacy and oral bioavailability of several of Verseon’s DME drug candidates, and the company expects to nominate the first candidate for development in Q4 2018.
Hereditary Angioedema (HAE) Program
Verseon is also developing a number of oral plasma kallikrein inhibitors for the treatment of hereditary angioedema (HAE), a rare and sometimes life-threatening genetic disease characterized by recurring episodes of swelling. Recent research has shown that plasma kallikrein is an important target central to the HAE pathway.
Verseon’s candidates have demonstrated good pharmacokinetics suitable for oral dosing, as well as efficacy in a key preclinical model. Further development is planned to optimize the pharmacokinetic profiles of these candidates and to test for in vivo efficacy.
Solid Tumor Program
Finally, Verseon is developing a class of next-generation chemotherapy agents for the treatment of multidrug resistant cancers. These small-molecule tubulin inhibitors have shown nanomolar in vitro potency against a range of cancer cell lines in preclinical studies. Further preclinical testing showed that the tubulin inhibitors are only weakly affected by common modes of drug resistance.
Verseon will continue to optimize several candidates with both low nanomolar activity against various cancer cell lines and insensitivity to major tumor resistance mechanisms. These candidates will be taken forward for further development.
