Patient Resources for Risvodetinib (IkT-148009)
Risvodetinib (IkT-148009) is a potent small-molecule medication designed and engineered as chronically administered oral medication that targets the underlying biological mechanism leading to Parkinson’s disease. The goal of the medication is to halt and reverse the loss of function for dopamine-secreting neurons in the brain and GI tract.
Completed
Risvodetinib (IkT-148009)-101 ("the 101 Trial")
This Phase 1/1b study investigated the safety and tolerability of the drug Risvodetinib (IkT-148009) in healthy elderly volunteers (55 to 70 years old) and in Parkinson’s patients. It also looked at the pharmacology of Risvodetinib (IkT-148009) in the body. This first-in-human study was designed in 3 parts. In Part A, healthy participants were administered a single, oral dose of Risvodetinib (IkT-148009) or placebo between 12.5 mg and 325 mg. In Part B, healthy participants were administered an oral dose of Risvodetinib (IkT-148009) once a day for 7 days between 12.5 mg and 200 mg. In Part C, Parkinson’s patients were administered an oral dose of Risvodetinib (IkT-148009) once a day for 7 days at 50 mg or 100 mg. The outcomes of these studies were reported in the Journal of Parkinson’s Disease in January, 2024. See the Publications page for this report.
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Active
Risvodetinib (IkT-148009)-201 (“the 201 Trial”) in Parkinson’s disease
This Phase 2 study is investigating the safety and tolerability of the c-Abl inhibitor Risvodetinib (IkT-148009) at three doses for 12 weeks in 120 untreated Parkinson’s patients (Hoehn & Yahr < 3.0, ages 30 to 80 years old) who do not currently take any medications to control their disease. Primary endpoints are safety and tolerability, as this study represents the first long-term dosing study of Risvodetinib in this patient population. Secondary endpoints will evaluate a hierarchy of motor and non-motor features in the central nervous system, evaluates sleep, swallowing, digestion and defecation. Exploratory endpoints include evaluation of disease pathology in skin and spinal fluid. Thirty-two sites in the U.S. are participating in this study. The study is expected to be extended for an additional 12 months following the 12 week double-blinded period.
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Planned
Risvodetinib (IkT-148009)-202 (“the 202 Trial”) in Multiple Systems Atrophy
This Phase 2 study will investigate the safety, tolerability and efficacy of Risvodetinib (IkT-148009) at two doses in patients with Multiple Systems Atrophy (MSA). Primary endpoints are safety and tolerability, as this study represents the first long-term dosing study in this patient population. Secondary endpoints evaluate a hierarchy of motor and non-motor features in the central nervous system and GI tract. Patients enrolled in this study will be allowed to continue on their current treatment regimen to control symptoms of the disease. Twenty-fives sites in 4 E.U. countries and the U.S. are planned for this study.
Completed
IkT-001Pro-501 (“the 501 Trial”) in Chronic Myelogenous Leukemia
This bioequivalence study will evaluate four doses of IkT-001Pro, the prodrug of imatinib mesylate, in two parts. Part A will evaluate four doses of 001Pro to match the pharmacokinetics of imatinib mesylate 400 mg in healthy volunteers. Part B will confirm the dose in a larger population of healthy volunteers. Part C measured bioequivalence at steady-state for 600 mg imatinib. Part D plans to measure bioequivalence of IkT-001Pro for imatinib mesylate 800 mg. One site in the U.S. is being used for this study.
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Planned
IkT-001Pro-701 (“the 701 Trial”) in Pulmonary Arterial Hypertension
This Phase 2/3 program will evaluate two doses of imatinib delivered as IkT-001Pro in WHO functional class II through IV patients who currently receive standard-of-care and will exclude the use of anticoagulants. The first part of this trial will evaluate cardiac hemodynamics with a primary endpoints measuring safety, tolerability and the improvement in Pulmonary Vascular Resistance (PVR) over 24 weeks. Several secondary endpoints will evlaute the improvement in other cardiac functions along with improvement in the 6 Minute Walking Distance (6MWD). If patients demonstrate an acceptable safety and tolerability profile, the trial will be amended to roll into a Phase 3 trial with primary endpoint in the 6MWD. The trial is currently planned with 70 participants at each of two dosing of IkT-001Pro plus a placebo group. The IND to open clinical development is expected to be filed by early 3Q24.