FDA grants orphan drug status for uveal melanoma drug

Uveal melanoma

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for iOnctura’s IOA-244, which is in development for solid and hematologic malignancies including uveal melanoma. 

Uveal melanoma is a disease in which cancer originates in the tissues of the eye, causing symptoms such as blurred vision or a dark spot on the iris.  

When the cancer metastases, which it does in approximately 50% of patients, there are limited treatment options and projected overall survival is only a year. 

A PI3Kδ inhibitor, IOA-244 (roginolisib) is being investigated in the DIONE-01 trial, a two-part, first-in-human Phase I study. Part A of the study investigated the safety and pharmacokinetics of continuous daily dosing of IOA-244 at 10, 20, 40 and 80mg.  

Part B is an ongoing cohort-expansion of the biologically-effective dose (BED) of 80mg in solid and hematologic malignancies including a recently opened non-Hodgkin ́s lymphoma cohort. 

Across all patients treated to date, roginolisib given at the BED showed less than 5% Grade 3 or Grade 4 toxicities, with these toxicities being transient in nature. There have been no dose-limiting drug reductions or interruptions and long- term (over six months) administration of IOA-244 is well tolerated. 

Catherine Pickering, Chief Executive Officer of iOnctura, said: “We are delighted to provide these positive updates on IOA-244, our lead clinical programme. These data demonstrate for the first time that a semi-allosteric inhibitor of PI3Kδ can be given to patients safely for long durations with no serious adverse events.  

“We are excited to take IOA-244 forwards into a monotherapy registration study in uveal melanoma and to further explore its potential both in lymphoma and solid tumours such as NSCLC.” 

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