Inhibikase Therapeutics is a clinical-stage pharmaceutical company focused on developing IKT-001, an oral prodrug of imatinib, for Pulmonary Arterial Hypertension (PAH). PAH is a rare, progressive disease affecting roughly 50,000 Americans, and most existing treatments act as vasodilators that manage symptoms without addressing the underlying disease. Imatinib inhibits tyrosine kinases implicated in the vascular cell proliferation and fibrosis that drive PAH, giving it potential as a disease-modifying therapy. The challenge with imatinib itself was demonstrated in a prior Phase 3 trial, where GI side effects caused high discontinuation rates. IKT-001 is designed to reduce GI toxicity by limiting inhibitory activity at c-Kit while in the gut, converting to active imatinib systemically. Inhibikase is currently running IMPROVE-PAH, an adaptive Phase 3 trial across roughly 180 global sites. The company has no approved products and no revenue, funding operations through equity financing, including a $115M public offering in late 2025. Inhibikase is pursuing FDA approval via the 505(b)(2) pathway, which allows reliance on existing imatinib data. The company has also out-licensed a legacy neurology candidate, risvodetinib, to ABLi Therapeutics for up to $47.5M in milestones plus royalties, and acquired CorHepta Pharmaceuticals to build a broader cardiopulmonary pipeline.
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