Objective:

To evalute the safety, tolerability and clinical benefit of risvodetinib (IkT-148009) in untreated Parkinson's disease.

Background:

Risvodetinib is an oral, selective inhibitor of non-receptor Abelson Tyrosine Kinases (c-Abl).  In pre-clinical animal models, risvodetinib was shown to halt disease progression and reverse functional loss with substantial reduction of alpha-synuclein aggregate pathology.

Design/Methods:

Risvodetinib was given once daily at 50, 100 or 200 mg, all believed to be therapeutic doses. 120 patients were randomized 1:1:1:1 across the three doses or placebo for 12 weeks and subsequently rolled into a 12 month extension study with the placebo group moving onto the top therapeutic dose. Safety and tolerability were measured by evaluation of frequency and severity of treatment-emergent adverse events, measures of cardiovascular safety, standard laboratory analyses and monitoring for changes in vision. A hierarchy of 15 secondary endpoints evaluated the potential benefit of risvodetinib on measures of motor and non-motor function in the Central and Enteric Nervous Systems. Biomarker analysis using CSF/plasma assessment by seed-amplification assay and skin biopsy complement functional assessments.

Results:

As of this writing the trial is ongoing with 32 participants in consenting, screening or enrolled. 197 potential participants have been identified using a proprietary pre-qualification portal that allows pre-qualified participants to communicate directly with trial sites. By the time this presentation will be made public, it is anticipated that the trial will be fully enrolled and blinded or unblinded data evaluating treatment benefit and safety/tolerability of risvodetinib (IkT-148009) over 12 weeks of once daily dosing will be discussed.

Conclusions:

Data from a 12 week double-blinded, placebo controlled trial will be presented providing a first look into whether a treatment validated in models of heritable and sporadic PD can impact disease in human Parkinson's.