PTC299 is an orally bioavailable drug designed to modulate RNA-mediated protein expression to inhibit the production of vascular endothelial growth factor (VEGF) in tumors.
PTC has recently completed a phase Ia safety and pharmacokinetic study of PTC299 in healthy volunteers. Preliminary results confirmed that the drug is orally bioavailable, well tolerated, and safely achieves the desired target plasma concentrations.
“The safety and pharmacokinetic data obtained from these trials should allow a prompt transition to phase I/II clinical trials in cancer patients. The advantage of this approach is that we have already established PTC299 dose levels that are likely to be well tolerated and achieve plasma concentrations associated with preclinical testing,” noted Langdon Miller, chief medical officer at PTC Therapeutics.
PTC Therapeutics also said that PTC299 was the second compound that entered clinical development from its post-transcriptional control technologies that identify small molecules which affect RNA biology.