IOA-244 for Uveal Melanoma Earns Orphan Drug Designation


In early January 2023, Healio reported that IOA-244, a treatment designed for uveal melanoma, was granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA). Orphan Drug designation is granted to drugs or biologics that treat, prevent, or diagnose rare conditions – those affecting fewer than 200,000 Americans. The drug developer – in this case, iOnctura – also earns benefits such as fee waivers, tax credits, and seven years of market exclusivity upon drug approval. 

iOnctura describes IOA-244 as:

the only semi-allosteric PI3Kδ inhibitor, with a unique chemical structure and binding mode. IOA-244 is currently in Phase I/II clinical development in indications burdened by immune mediated resistance and a high intrinsic expression of PI3Kδ in cancer cells and tumor-infiltrating cells.

Within these development phases, IOA-244 (with an intended name of roginolisib) is being evaluated in the Phase 1 DIONE-01 study. The first portion of the study will focus on understanding the safety, tolerability, and pharmacokinetics of 10, 20, 40, or 80mg IOA-244. Alternatively, the second portion will explore the efficacy of 80mg IOA-244 for other oncological problems. 

Thus far, IOA-244 has been shown to be relatively safe and well-tolerated. We look forward to seeing the insights gleaned from this, and future, research. 

What is Uveal Melanoma?

Uveal melanoma may also be called ocular melanoma. Regardless of the name, this cancer develops in melanin-producing cells in the iris, choroid layer, or ciliary body (all of these are in the uvea, or the middle section of the eye). In rarer cases, ocular melanoma may form in the outermost layer of the eye. While doctors are unsure of the exact cause of uveal melanoma, they do know that DNA errors occur in healthy eye cells. Additional risk factors include being Caucasian, older age, certain inherited skin disorders or genetic mutations, UV light exposure, and light eye color. 

In many cases, people are asymptomatic. If and when symptoms appear, they can include:

  • Growing dark spots on the iris
  • A sensation of flashes or dust specks in the eye
  • Loss of peripheral vision
  • Blurred vision
  • Changes in pupil shape 
  • Glaucoma (complication)
  • Vision loss (complication)

Currently, treatment options include radiation, cold treatments, photodynamic therapy, laser therapy, and surgery. Those with uveal melanoma should speak with their care team about their best treatment options.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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