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Pipeline

Providing Novel Therapies for Patients with Neurodegenerative Diseases

Drug Target Drug Candidate Mortality 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 complete
Phase 1/1b Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Oncology
BCR-Abl IkT-001Pro Small molecule Stable-phase CML (orphan indication)
Preclinical development Phase in progress
Phase 1/1b Phase not started
Phase 2 Phase not started
Phase 3 Phase not started
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-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:

Synuclein toxicity, neurodegeneration, motor dysfunction and neuroinflammation are the four major disease pillars for Parkinson’s Disease suppressed by IkT-148009. View Candidate

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 Candidate

IkT-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).

The prodrug technology adds a linker to the kinase inhibitor and suppresses on-dosing side effects.
View Candidate