Inhibikase Therapeutics (IKT) Corporate presentation summary

Disease overview and unmet need

• Pulmonary arterial hypertension (PAH) is a rare, progressive, and life-threatening disease with ~30% five-year mortality, reduced quality of life, and high economic burden despite 15 approved vasodilators and only one anti-proliferative agent.

• The $8.3B PAH market is dominated by vasodilators that do not address the underlying disease mechanism, highlighting a significant unmet need for disease-modifying therapies.

• PAH is driven by uncontrolled vascular cell proliferation, leading to vascular remodeling, increased pulmonary artery pressure, right heart failure, and death.

Product and mechanism

• IKT-001 is a novel oral pro-drug of imatinib, engineered for improved gastrointestinal (GI) tolerability and optimal efficacy in PAH.

• Imatinib inhibits tyrosine kinase activity of PDGFR and c-kit, blocking cell signaling that drives vascular remodeling in PAH.

• Preclinical studies show IKT-001 reverses PAH in animal models and demonstrates 18-fold reduced c-kit inhibition versus imatinib, potentially reducing GI side effects.

• IKT-001 is stable in the gut and rapidly converts to imatinib in the blood, minimizing GI exposure and supporting increased tolerability.

Clinical data and rationale

• Imatinib demonstrated statistically significant improvements in 6-minute walk distance (6MWD) and pulmonary vascular resistance (PVR) in Phase 3 IMPRES, but high discontinuation rates due to GI adverse events limited approval.

• Patients able to sustain a 400mg dose of imatinib showed greater improvements in 6MWD (45m) and PVR.

• Contemporary studies confirm dose-dependent efficacy and disease modification, with improved tolerability through dose adjustments.

• IKT-001 is designed to overcome prior limitations by improving GI tolerability, enabling higher sustained dosing and potentially better outcomes.