Providing Novel Therapies for Patients with Neurodegenerative Diseases
Drug Target | Drug Candidate | Modality | Disease indication | Preclinical development | Phase 1/1b | Phase 2 | Phase 3 |
---|---|---|---|---|---|---|---|
Neurodegeneration | |||||||
c-Abl | IkT-148009 | Small molecule | Parkinson's Disease: Treatment Naive |
Preclinical development Phase complete
|
Phase 1/1b Phase complete
|
Phase 2 Phase in progress
|
Phase 3 Phase not started
|
c-Abl | IkT-148009 | Small molecule | Parkinson's Disease: Early Stage |
Preclinical development Phase complete
|
Phase 1/1b Phase complete
|
Phase 2 Phase in progress
|
Phase 3 Phase not started
|
c-Abl | IkT-148009 | Small molecule | Neurogenic Constipation |
Preclinical development Phase complete
|
Phase 1/1b Phase complete
|
Phase 2 Phase in progress
|
Phase 3 Phase not started
|
c-Abl | IkT-148009 | Small molecule | Dysphagia |
Preclinical development Phase complete
|
Phase 1/1b Phase complete
|
Phase 2 Phase in progress
|
Phase 3 Phase not started
|
c-Abl | IkT-148009 | Small molecule | Multiple System Atrophy |
Preclinical development Phase in progress
|
Phase 1/1b Phase not started
|
Phase 2 Phase not started
|
Phase 3 Phase not started
|
Oncology | |||||||
BCR-Abl | IkT-001Pro | Small molecule | Stable-phase CML (orphan indication) |
Preclinical development Phase complete
|
Phase 1/1b Phase complete
|
Phase 2 Phase complete
|
Phase 3 Phase in progress
|
Research Phase | |||||||
c-Abl | IkT-148x | Small molecule | Dementia with Lewy Body |
Preclinical development Phase in progress
|
Phase 1/1b Phase not started
|
Phase 2 Phase not started
|
Phase 3 Phase not started
|
c-Abl | IkT-148x | Small molecule | Multiple System Atrophy |
Preclinical development Phase in progress
|
Phase 1/1b Phase not started
|
Phase 2 Phase not started
|
Phase 3 Phase not started
|
c-Abl | IkT-1427 | Small molecule | Progressive multifocal leukoencephalopathy |
Preclinical development Phase in progress
|
Phase 1/1b Phase not started
|
Phase 2 Phase not started
|
Phase 3 Phase not started
|
IkT-148009
A Novel, Selective, Oral c-Abl Kinase Inhibitor
IkT-148009 is our lead product candidate to treat Parkinson’s and related diseases. Emerging from our RAMP™ discovery engine, IkT-148009 is currently in a Phase 1b study in Parkinson’s patients evaluating the safety, tolerability and pharmacokinetics of oral, once a day IkT-148009 therapy at three escalating doses given for seven days. Exploratory endpoints include assessing motor and non-motor function in the brain and gastrointestinal tract. In a Phase 1 clinical study of 88 older and elderly healthy volunteers, IkT-148009 displayed an excellent safety profile up to 325 mg given once daily or 25 mg daily for seven days with no clinically significant adverse events observed. In a Phase 1b clinical study ongoing, IkT-148009 displayed the same excellent safety profile as seen in healthy volunteers and similar drug pharmacology.
In validated animal models of progressive, alpha-synuclein-dependent disease, IkT-148009 that was given therapeutically halted disease progression and led to functional recovery in under eight weeks of oral treatment. Remarkably, IkT-148009 nearly cleared pathological alpha-synuclein from the affected neurons and the gastrointestinal tract in a manner that correlated with functional recovery. We believe this is the first time this has been observed for any oral therapy attempting to treat Parkinson’s disease in a living organism. The four major disease pillars of Parkinson’s are suppressed by IkT-148009 in multiple validated animal models include:


IkT-1427
A Novel, Selective, Potentially Antiviral c-Abl Kinase Inhibitor
IkT-1427 is in early pre-clinical development as an antiviral drug substance to suppress replication of the John Cunningham Virus (JCV), the cause of Progressive Multifocal Leukoencephalopathy (PML). View CandidateIkT-001Pro
A Prodrug of the Anticancer Agent Imatinib Mesylate (marketed at Gleevec®)
IkT-001Pro is the first product candidate that deploys our prodrug technology. A prodrug is a precursor formulation of an active drug substance that enhances specific properties to improve drug delivery in patients. We have developed a platform prodrug technology that adds a small linker to a known or novel kinase inhibitor and suppresses on-dosing side effects common to drugs in this class, particularly gastrointestinal disturbance (nausea, diarrhea and/or vomiting).
