PharmaAkea has completed a Phase 1 clinical trial indicating that its PAT-1251 therapy binds with an enzyme associated with pulmonary fibrosis.
That suggests that it could be an effective treatment for the lung disease. The company will test that hypothesis in further trials.
PharmaAkea has already reported that PAT-1251 reduced fibrosis, or scarring, in mice with interstitial pulmonary fibrosis, or IPF.
PAT-1251 inhibits the LOXL2 enzyme, whose levels are elevated in the blood serum of people with IPF.
LOXL2 increases the stiffness of the extracellular matrix, a structure that supports cells. The increased stiffness leads to an increase in cells that promote scarring. By inhibiting the activity of LOXL2, PAT-1251 decreases fibrosis.
San Diego-based PharmaAkea conducted the Phase 1 trial (NCT02852551) to evaluate the safety, pharmacokinetics and pharmacodynamics of PAT-1251 in healthy individuals, and their ability to tolerate The study was also designed to determine the maximum dose people could tolerate. Pharmacokinetics refers to the body’s effect on a drug, while pharmacodynamics refers to the drug’s effect on the body.
Results indicated that PAT-1251 was generally safe and that healthy people tolerate it well. Researchers will use the findings to decide what dose of PAT-1251 to use in a Phase 2 trial expected to start 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,” Robert Williamson, CEO of PharmAkea, said in a press release.
PharmAkea presented Phase 1 and animal-study findings on PAT-1251 at the IPF Summit in Boston, Aug. 21-23. The poster session was titled “PAT-1251, a Mechanism-Based Small Molecule Inhibitor of LOXL2, as a Potential Anti-Fibrotic Treatment for IPF.”
The animal studies showed that PAT-1251 significantly reduced fibrosis in a mouse model of IPF.