Wilmington, Delaware – The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to IKT-001, an investigational formulation of imatinib developed by Inhibikase Therapeutics for the treatment of pulmonary arterial hypertension (PAH).
The designation is intended to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the United States. It provides benefits such as tax credits, waiver of certain FDA fees, and, upon approval, seven years of market exclusivity.
According to Inhibikase, approximately 50,000 people in the U.S. are living with PAH. “The grant of orphan drug designation for IKT-001 by FDA is another important milestone for Inhibikase and reflects the high unmet medical need among the approximately 50,000 people suffering from PAH in the United States,” said Mark Iwicki, the company’s CEO.
Improving on Earlier Imatinib Therapy
PAH is a progressive disease characterized by elevated blood pressure in the pulmonary arteries, which places excessive strain on the heart. The condition results from abnormal growth of cells lining the pulmonary blood vessels, leading to narrowing of the arteries and symptoms such as shortness of breath, fatigue, and reduced exercise capacity.
Imatinib, a tyrosine kinase inhibitor originally developed to treat certain cancers, has previously shown promise in PAH. Novartis investigated an oral formulation, imatinib mesylate, which improved exercise capacity in clinical studies but was ultimately discontinued because of significant safety concerns.
IKT-001 is designed to overcome those limitations. The investigational therapy contains an inactive precursor that is converted into active imatinib after administration, with the goal of delivering the drug’s therapeutic benefits while reducing adverse effects.
According to Inhibikase, preclinical studies demonstrated improvements in pulmonary vascular remodeling and blood flow markers associated with PAH, while showing a lower potential for gastrointestinal toxicity compared with imatinib mesylate.
“We believe that IKT-001 has the potential to become the first once-daily oral antiproliferative therapy for PAH, offering meaningful benefits for patients,” Iwicki said.
Phase 3 Trial in Progress
IKT-001 is currently being evaluated in the Phase 3 IMPROVE-PAH trial (NCT07365332), a randomized, placebo-controlled study expected to enroll nearly 500 adults aged 18 to 75 with PAH who are receiving stable background therapy.
The trial consists of two parts. The first will assess the drug’s effect on pulmonary vascular resistance, an invasive measure of blood flow through the lung arteries, while the second will evaluate its impact on PAH symptoms and clinical outcomes.
The IMPROVE-PAH study is currently enrolling participants at clinical sites in Massachusetts and Kentucky.
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