PharmAkea announced that it has successfully completed a Phase 1 single and multiple ascending dose (SAD/MAD) clinical study with PAT-1251, its novel, small-molecule LOXL2 inhibitor being developed for the treatment of IPF and other fibrotic diseases.

The study results indicate that PAT-1251 was generally well tolerated and a maximum tolerated dose (MTD) was achieved. A proprietary target engagement assay was used successfully to demonstrate that PAT-1251 binds the LOXL2 enzyme in plasma. After successful completion of the 6- and 9-month toxicology studies in Q4 2017, PAT-1251 will be ready to enter Phase 2 clinical development in early 2018.

"Based on its unique mechanism of action, PharmAkea's LOXL2 inhibitor, PAT-1251, has the potential to improve lung function in patients suffering from IPF, a debilitating lung disorder in which novel agents with improved tolerability, efficacy, and long-term safety profiles are needed," said Robert Williamson, CEO of PharmAkea.

The aim of the completed study was to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of oral ascending doses of PAT-1251 in healthy subjects.  The data from these studies will inform the dose selection and study design for a subsequent Phase 2 proof-of-concept study in IPF.