IQ-AI's subsidiary Imaging Biometrics LLC has submitted an application to the U.S. Food and Drug Administration (FDA) for Breakthrough Therapy Designation for its oral gallium maltolate (GaM) therapy, the London-based medical services company announced on May 2, 2025.
The application follows promising preliminary results from a Phase 1 trial evaluating GaM as a potential treatment for adult patients with recurrent or refractory glioblastoma (GBM) IDH-wildtype. Early findings suggest an overall survival of 34.1 months from initial diagnosis, substantially exceeding the median survival of 14-15 months typically reported for patients receiving standard care treatment, which includes surgical resection, radiation, and chemotherapy.
"Our decision to apply for Breakthrough Therapy Designation reflects our optimism in the potential of oral GaM as a treatment for recurrent or refractory glioblastoma IDH-wildtype," said Trevor Brown, CEO of IQ-AI.
Understanding Glioblastoma and Current Treatment Challenges
Glioblastoma is an aggressive form of brain cancer with limited treatment options and poor prognosis. Standard of care typically includes maximal safe surgical resection followed by radiation therapy and chemotherapy with temozolomide. Despite this multimodal approach, recurrence is common, and effective treatments for recurrent disease remain an urgent unmet need.
The IDH-wildtype classification represents the most common and aggressive molecular subtype of glioblastoma, accounting for approximately 90% of cases. These tumors typically demonstrate resistance to conventional therapies, contributing to their poor prognosis.
Breakthrough Therapy Designation Implications
Breakthrough Therapy Designation is granted to therapies that demonstrate substantial improvement over existing treatments based on preliminary clinical evidence. While IQ-AI has already received Fast Track Designation for GaM, the Breakthrough Therapy Designation would provide more intensive FDA guidance and senior-level organizational support throughout the development process.
The FDA is required to respond to IQ-AI's application within 60 days. If granted, this designation could potentially accelerate the development and review timeline for GaM, bringing this promising treatment to patients more quickly.
Phase 1 Trial Details and Cautious Optimism
While the preliminary survival data from the Phase 1 trial appears promising, the company acknowledges that caution is appropriate when interpreting these results due to the limited size of the trial population. Phase 1 trials typically focus on safety and dosing rather than efficacy, with small patient cohorts.
Nevertheless, the substantial difference between the observed 34.1-month overall survival and the typical 14-15 month survival with standard treatment suggests potential clinical significance that warrants further investigation in larger trials.
Gallium Maltolate: Mechanism and Potential
Gallium maltolate is an orally administered compound that leverages gallium's ability to mimic iron in biological systems. Cancer cells, including glioblastoma cells, typically have increased iron requirements compared to normal cells to support their rapid proliferation.
By competing with iron for binding to proteins involved in cellular proliferation, gallium can potentially disrupt critical processes in cancer cells while having minimal effects on normal cells. Additionally, gallium compounds have demonstrated anti-inflammatory and immunomodulatory properties that may contribute to their therapeutic effects.
The oral administration route represents a significant advantage for brain tumor patients, potentially improving quality of life by reducing the need for invasive procedures or frequent hospital visits for intravenous treatments.
Next Steps in Development
While awaiting the FDA's decision on Breakthrough Therapy Designation, IQ-AI and Imaging Biometrics are likely continuing their clinical development program for GaM. Typically, this would include planning for larger Phase 2 trials to further evaluate efficacy and refine dosing strategies, followed by pivotal Phase 3 trials if earlier results remain promising.
The company has not yet disclosed specific plans for these next-phase trials, including potential patient enrollment targets, trial design, or timeline projections. However, the regulatory designations already secured and being pursued suggest a commitment to advancing this therapy through the clinical development and regulatory approval processes.
