FDA grants orphan drug status to experimental PAH treatment
Inhibikase says oral therapy could provide benefits with reduced safety risks
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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to IKT-001, an experimental formulation of imatinib that Inhibikase Therapeutics is developing to treat pulmonary arterial hypertension (PAH).
The FDA gives orphan drug designation to investigational medications that aim to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. The designation aims to offer additional incentives to drug developers investing in rare disease treatments, where a small patient population can make it hard to recoup development costs and turn a profit.
The designation offers Inhibikase perks including tax breaks and fee waivers, as well as a guaranteed seven years of market exclusivity if the FDA ultimately approves IKT-001.
“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,” Mark Iwicki, CEO of Inhibikase, said in a company press release.
PAH is a chronic disorder marked by abnormally elevated pressure in the vessels that carry blood from the heart to the lungs, which strains the heart and leads to symptoms such as shortness of breath. In PAH, cells lining these blood vessels grow abnormally, narrowing the vessels and increasing blood pressure.
Preclinical data show promise
Imatinib, a small molecule that blocks cell growth, was originally developed as an anticancer agent. Novartis previously developed an oral formulation of the molecule, imatinib mesylate, as a potential treatment for PAH. Clinical trials indicated that imatinib mesylate improved exercise capacity in people with PAH, but that formulation also caused substantial safety problems. Safety issues ultimately led Novartis to pull the plug on its development.
IKT-001 contains an inactive precursor molecule that’s converted into active imatinib once it’s inside the body. Inhibikase is developing the therapy with the goal of offering benefits similar to those of imatinib mesylate, but with fewer safety risks. According to Iwicki, preclinical data “demonstrated improvements in pulmonary vascular and [blood flow] markers of PAH, together with a lower potential for [digestive] toxicity compared to imatinib mesylate.”
“We believe that IKT-001’s potential to be the first once-daily oral [medication that blocks cell growth] may offer significant potential benefits to the PAH patient population,” Iwicki said.
Inhibikase is sponsoring a two-part Phase 3 clinical trial called IMPROVE-PAH (NCT07365332) that aims to test IKT-001 against a placebo in nearly 500 adults with PAH, ages 18 to 75, who are on stable treatment with available PAH therapies. The first part of the study will evaluate IKT-001’s effects on an invasive measure testing resistance in lung blood vessels, while the second will look at how the therapy affects PAH symptoms. The trial is currently enrolling at sites in Massachusetts and Kentucky.
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