Previously, the researchers initiated a Phase 2 trial (NCT05312710) evaluating the safety and efficacy of neoadjuvant APG-157 in patients with newly diagnosed, locally advanced head and neck cancer of the oral cavity and/or oropharynx.

According to a press release from developer Aveta Biomics, the FDA has granted the experimental immuno-oncology drug APG-157 fast-track designation as a neoadjuvant treatment for patients with head and neck cancer.¹

The developers designed APG-157 to have a dual mechanism of action by selectively inducing apoptosis of cancer cells while simultaneously reprogramming the immune environment. The active ingredient, an orally administered lozenge, is derived from Curcuma longa, which is normally used as a source of the spice turmeric.² The researchers hypothesized that administering APG-157 as a neoadjuvant treatment could reduce the need for extensive surgery and other intensive therapies in patients with head and neck cancer.

“There is an urgent need for new treatments for (patients with) head and neck cancer, many of whom have poor survival and significant morbidity rates after current standard treatments, including surgery, radiation therapy and chemotherapy/targeted therapies,” Parag Mehta, PhD, CEO of Aveta Biomics, said in the press release.¹ “The rare granting of Fast Track designation for neoadjuvant treatment of head and neck cancer underscores FDA’s recognition of APG-157’s potential role in delivering meaningful treatment benefits for these patients.”

Previously, the researchers initiated a Phase 2 trial (NCT05312710) evaluating the safety and efficacy of neoadjuvant APG-157 in patients with newly diagnosed, locally advanced head and neck cancer of the oral cavity and/or oropharynx.³

“Oral cancer affects the tongue, mouth and throat. It often occurs in people who smoke or have the human papillomavirus…Surgery in the mouth or tongue is painful and recovery is long,” said Dr. Marilene B. Wang, head and neck surgeon and professor in the division of head and neck surgery at the University of California, Los Angeles School of Medicine, in a press release about the study.² “We found that APG-157 shrinks small mouth tumors and may stabilize the growth of large tumors. For smaller tumors, the amount of radiation or chemotherapy may be reduced or minor surgery may be performed.”

In this study, an estimated total of 24 patients will receive APG-157 at a dose of 100 mg, given as 2 lozenges 3 times daily. Treatment with APG-157 will continue for 4 to 6 weeks before patients receive other therapies.

The primary endpoint of the study is the ability of APG-157 to affect tumor size as assessed by MRI with or without contrast and PET/CT imaging. Secondary endpoints include changes in immunohistochemical profile, changes in cytokine levels, and changes in cell-free RNA biomarkers in blood and saliva from baseline to the end of treatment.

Patients aged 18 years and older with confirmed squamous cell carcinoma of the oral cavity or oropharynx and newly diagnosed stage I to IV squamous cell carcinoma of the head and neck are eligible to participate in the study. Patients who subsequently receive local therapy with curative surgery with or without radiation or purely palliative radiotherapy are also eligible to participate in the study.

Patients for whom definitive local therapy is available less than 4 weeks after initial diagnosis or who have received chemotherapy or radiotherapy within 8 weeks prior to study entry are excluded from the study. Recurrent or metastatic cancer, dental abscesses, bleeding gums or tooth cracks, and a fracture of the lower or upper jaw within 8 weeks prior to study entry are also reasons for exclusion from the study.

“This Fast Track approval, based on our extensive Phase 1 and Phase 2 data, accelerates our ability to make our promising first-line therapy available to all (patients with) newly diagnosed, locally advanced head and neck cancer in a frontline setting,” said Karim Malek, MD, MTh, MBA, Chief Medical Officer at Aveta Biomics.¹

References

1. FDA grants Fast Track designation to Aveta Biomics’ drug APG-157 for neoadjuvant treatment of head and neck cancer. Press release. Aveta Biomics. August 22, 2024. Retrieved August 23, 2024. https://tinyurl.com/4kya8f55
2. VA clinical trial of a lozenge with natural active ingredient shows promising results and safety in the fight against oral cancer. Press release. U.S. Department of Veterans Affairs. September 2, 2022. Retrieved August 23, 2024. https://tinyurl.com/56jp3pr4
3. Safety and efficacy of APG-157 in head and neck cancer. ClinicalTrials.gov. Updated August 14, 2024. Accessed August 23, 2024. https://tinyurl.com/2atkk3t3