1st patient dosed in 201 trial of IkT

Inhibikase study to test safety of oral therapy at 22 US sites

by Margarida Maia, PhD | May 19, 2023

Inhibikase Therapeutics, which is recruiting up to 120 untreated Parkinson's disease patients across the U.S. to test its oral therapy IkT-148009, announced that its 201 trial program just dosed its first participant.

The Phase 2 trial (NCT05424276) is assessing the safety and tolerability of IkT-148009 versus a placebo in people with Parkinson's, ages 30 to 75, who have received no prior treatment. The study also will evaluate the therapy's pharmacokinetics, meaning its movement into, through, and out of the body.

The medication will be given by mouth as a gelatin capsule once daily at a dose of 50, 100 and 200 mg for 12 weeks, or about three months.

Information on contacts and locations for the 22 U.S. study sites is available on the trial page; not all locations have started recruiting. The trial, which started this month, is slated to run through October.

"Dosing of the first patient in our ‘201’ trial completes the first milestone in our revamped Phase 2 program evaluating the clinical benefit of IkT-148009 for the treatment of Parkinson's disease and related disorders," Milton H. Werner, PhD, Inhibikase's president and CEO, said in a press release.

IkT-148009 had been placed on a clinical hold late last year by the U.S. Food and Drug Administration (FDA) due to safety concerns. The issues stemmed from the use of the highest dose of 200 mg/day, with the FDA seeking additional safety and pharmacological data on the therapy.

That clinical hold was partly lifted in January, after the higher dose was found to reach steady levels in the body after four days without causing any serious side effects in healthy volunteers.

The company has shared the new data with the FDA as it seeks clearance to resume testing of this highest dose.

Inhibikase designed its therapy to treat the cause of Parkinson's, a disease characterized by the death and dysfunction of neurons, or nerve cells, in a part of the brain that controls movement and coordination.

The symptoms of Parkinson's are due to the accumulation of alpha-synuclein protein, which forms toxic aggregates or clumps that eventually result in the death of dopaminergic neurons. These nerve cells communicate with other neurons by releasing a signaling molecule called dopamine.

IkT-148009 is a small molecule designed to get into the brain and block the activity of Abelson tyrosine kinase (c-Abl), a protein known to play a role in regulating how neurons respond to damage. Blocking c-Abl is expected to halt disease progression and reverse the loss of dopamine-secreting neurons.

"Our research has validated the critical role that c-Abl plays in the initiation and progression of Parkinson's disease, as well as the potential of IkT-148009 as a promising new approach to disease modification," Werner said.

The 201 study also will look at preliminary efficacy, including measures of motor and non-motor symptoms, and the levels of phosphorylated alpha-synuclein in the skin and cerebrospinal fluid — the liquid surrounding the brain and spinal cord. A recent study showed this type of analysis can detect Parkinson's disease with high accuracy.