Pharma Focus America

Inhibikase Therapeutics Announces FDA has Lifted the Full Clinical Hold on IkT-148009 in Multiple System Atrophy

Thursday, March 09, 2023

Inhibikase Therapeutics, Inc., a clinical-stage pharmaceutical company focused on developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease, Parkinson's-related disorders, and other diseases associated with Abelson Tyrosine Kinases, has announced that the U.S. Food and Drug Administration (FDA) has lifted the full Clinical Hold on IkT-148009, the company's c-Abl inhibitor, in Multiple System Atrophy (MSA). This regulatory clearance allows Inhibikase Therapeutics to proceed with its plans for a future Phase 2 clinical trial in MSA.

Milton H. Werner, Ph.D., President and Chief Executive Officer of Inhibikase Therapeutics, expressed gratitude for the FDA's expeditious review of their response to the Clinical Hold on IkT-148009 in MSA. He emphasized that preclinical models have demonstrated the therapeutic potential of IkT-148009 in MSA, just as it has shown promise in their work on Parkinson's disease. One of the ongoing model studies has exhibited significant neuroprotective benefits in response to c-Abl inhibition by IkT-148009. With the Clinical Hold lifted and the Investigational New Drug (IND) application now open, the company is looking forward to completing these studies before initiating the Phase 2a trial in MSA patients.

IkT-148009 is a potent and selective brain-penetrant c-Abl tyrosine kinase inhibitor that has shown the ability to halt disease progression, protect and restore lost neurons, and clear the underlying protein pathology in animal studies of Parkinson's disease. Multiple System Atrophy (MSA) is a rare form of Parkinsonism that affects a different part of the brain and progresses three times faster than typical Parkinson's disease. In previously published work, Inhibikase has demonstrated that MSA may also be initiated by c-Abl modification of alpha-synuclein aggregates in the brain. Ongoing animal models of MSA have shown that IkT-148009 provides significant neuroprotective benefits, preventing functional loss in mice during a seven-week, once-daily dosing regimen. The company plans to assess the functional benefits of IkT-148009 over an additional three months to determine if it leads to the clearance of alpha-synuclein aggregate pathology in the treated animals.

The upcoming Phase 2 clinical trial, known as the '202' trial, will evaluate the safety, tolerability, and pharmacokinetics of IkT-148009 in MSA patients over a six-month period of once-daily dosing at one of two oral doses. Secondary and exploratory endpoints will assess clinical benefits using a modified version of the Total Unified MSA Rating Scale (UMSARS), quality of life assessment, severity of symptoms related to orthostatic hypotension, and levels of neurofilament light chain in peripheral blood and spinal fluid. Furthermore, biomarkers of treatment efficacy will be explored by measuring levels of phosphorylated alpha-synuclein in spinal fluid, peripheral blood, and skin. The trial will also monitor the clinical effect on the progression of atrophy using MRI.

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