Panoptes` current focus is on its first clinical candidate PP-001. PP-001 intends to offer patients therapies that are highly efficacious and well tolerated and thus provide a better quality of life.The clinical candidate PP-001 was discovered by a structure-based docking approach with several million virtual compounds. A highly diverse subset of approximately 300 hits was synthesized and used as starting point for hit-to-lead optimization through medicinal chemistry. PP-001 is the result of a lead optimization program and was chosen for further development based on target efficacy and pharmacokinetic properties. PP-001 is a highly specific nanomolar inhibitor of an essential enzyme of the de novo pyrimidine pathway leading to inhibition of expression of IFN-γ and IL-17, two hallmark cytokines of Th1 and Th17 cells responsible for autoimmune uveitis and other inflammatory diseases. Further, PP-001 reduces the host cell pyrimidine pool, which leads to inhibition of replication of many viruses especially adenovirus serotypes causing the highly contagious viral form of conjunctivitis. PP-001 showed in vivo proof of concept for uveitis:
- In two different rat models for uveitis, PP-001 completely ameliorates clinical and histological symptoms of the disease.
- In addition, a therapeutic approach with PP-001 prevents relapses of experimental autoimmune uveitis.
- Highly stable and soluble small molecule with established and cost efficient GMP synthesis process (1kg scale)
- PP-001 can be developed as an oral, topical or local (e.g. intravitrial) formulation
- Single dose studies in rats and repeat dose studies in mice did not show signs of intolerability