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The FDA granted Breakthrough Therapy designation to IDE196 for the neoadjuvant treatment of primary uveal melanoma when enucleation has been recommended.
Treatment with IDE196 (darovasertib) has been granted Breakthrough Therapy designation by the United States Food and Drug Administration (FDA) for the neoadjuvant treatment of adult patients with primary uveal melanoma for whom enucleation has been recommended, according to a news release from IDEAYA Biosciences, Inc.
The Breakthrough Therapy designation from the FDA follows a Fast Track designation granted by the regulatory agency for the potential first-in-class protein kinase C (PKC) inhibitor, IDE196, in combination with Xalkori (crizotinib) for adult patients being treated for metastatic uveal melanoma, in which, a phase 2/3 registration-enabling trial of the IDE196 and Xalkori combination in 1L HLA-A2-negative metastatic uveal melanoma is ongoing.
According to the official FDA website, Breakthrough Therapy designation is meant to expedite the process of a drug in development; Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
The news release adds that Breakthrough Therapy designation facilitates more intensive FDA guidance, cross-disciplinary collaboration and eligibility for rolling submission and priority review.
"We are pleased to receive FDA Breakthrough Therapy designation as we prepare to advance neoadjuvant IDE196 into a potential phase 3 registrational trial in patients with primary uveal melanoma. This designation highlights the potential of monotherapy IDE196 in a patient population with significant unmet medical need where there are currently no FDA-approved systemic therapies," Dr. Darrin Beaupre, chief medical officer of IDEAYA Biosciences, said in the news release.
Enucleation: surgery to remove an eye.
Plaque brachytherapy: radiation therapy used to treat eye tumors.
Progression-free survival: the length of time during and after treatment that a patient lives with the disease but it does not get worse.
Overall survival: the length of time from the start of treatment that patients diagnosed with the disease are still alive.
Neoadjuvant uveal melanoma is projected to effect approximately 12,000 patients annually in North America, Europe and Australia, the news release states, making this a high unmet medical need, as no systemic therapies are yet approved by the FDA. Therefore, investigators continue to investigate the potential treatment option for patients within this population.
The Breakthrough Therapy designation application for IDE196 was supported by updated interim clinical data from an ongoing phase 2 open-label trial evaluating treatment with the agent as a standalone therapy before surgery (neoadjuvant treatment) for patients with localized uveal melanoma. In September 2024, the company reported that IDE196 led to an 82% tumor shrinkage rate and a 61% eye preservation rate in patients with uveal melanoma.
As part of the Breakthrough Therapy designation application, updated clinical data on neoadjuvant UM were submitted, including information on treatment effectiveness, safety, reduction in radiation exposure, eye preservation and improvements in vision. The company plans to present these results at a medical conference in 2025. Additional clinical updates on the treatment, including median overall survival data from the phase 2 IDE196-001 trial, are planned for release in mid-to-late 2025.
A key data readout on median progression-free survival from the phase 2/3 trial evaluating the combination of IDE196 and Xalkori in the first line is anticipated by the end of 2025. In addition, the company plans to launch a phase 3 randomized trial. The release goes on to share that the potential phase 3 registrational study would evaluate treatment with IDE196 for patients with uveal melanoma who are eligible for enucleation or plaque brachytherapy; these patients would subsequently be split for treatment between two cohorts.
"We are targeting to present the updated phase 2 clinical data in neoadjuvant UM that was provided as part of the Breakthrough Therapy designation application at multiple medical conferences in 2025," Yujiro S. Hata, president and CEO of IDEAYA Biosciences, concluded in the release.
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