Inhibikase Cleared to Expand Phase 1 IkT-148009 Study
Inhibikase Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to start a Phase 1 clinical trial evaluating its investigational, once-daily, oral medicine IkT-148009 in patients with Parkinson’s disease.
The clearance comes after the agency reviewed the safety, tolerability and pharmacokinetic data from a Phase 1 study in healthy volunteers. Pharmacokinetics refers to how a compound moves into, through, and out of the body.
“The extension of our Phase 1 study of IkT-148009 for the treatment of Parkinson’s disease into patients is an important milestone for Inhibikase,” Milton Werner, PhD, president and CEO of Inhibikase, said in a press release.
IkT-148009 is a small molecule that blocks the action of a protein that has been implicated in the development of Parkinson’s disease. That protein, called c-Abl (short for Abelson tyrosine kinase), may be used as a therapeutic target with the goal of halting disease progression and even reversing functional loss, the company says. C-Abl kinase is thought to play a critical role in monitoring damage to neurons in and outside the brain, and regulating biological pathways responsible for protecting neurons from dying.
Studies in animal models have shown that once-daily treatment with IkT-148009 can halt and reverse functional loss in both the brain and gastrointestinal tract, the company reports.
“In validated preclinical animal models, IkT-148009 was able to drive functional recovery in the brain and gastrointestinal tract, clear pathologic alpha-synuclein aggregates, and block neurodegeneration and neuroinflammation. With the initiation of our Phase 1b extension study in Parkinson’s patients, we are excited that we may be closer to finally impacting this devastating disease that affects millions of people worldwide,” Werner said.
The Phase 1 study (NCT04350177), which began dosing healthy volunteers ages 45 to 70 earlier this year, is investigating the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of IkT-148009 versus a placebo for up to seven days. So far, 42 people have received IkT-148009 in doses ranging from 12.5 to 100 mg, which were found to be sufficient to achieve high levels of the medicine in the body. No clinically relevant side effects were noted.
Now, the Phase 1b study — an extension of the Phase 1 study — will focus on the safety, tolerability, and pharmacokinetics of IkT-148009 for up to 14 days. A 13-week toxicology study is currently ongoing, and Inhibikase expects to file its results to the FDA for review in August this year. If given clearance, those results may inform dosing for longer periods of up to three months.
The Phase 1b study also will assess measures of cognition, motor function, and gut motility, as well as clearance of aggregates of alpha-synuclein — a hallmark of Parkinson’s disease — as a result of treatment with Ikt-148009.