A new experimental drug, emiltatug ledadotin (Emi-Le), has shown encouraging results for patients with aggressive adenoid cystic carcinoma (ACC), a rare cancer that currently has no approved systemic treatments. Findings revealed that the B7-H4–targeted antibody-drug conjugate produced strong tumor responses in patients with advanced disease.
Among 32 patients with particularly aggressive ACC, nearly 47% experienced tumor shrinkage, including one complete response, while more than 81% achieved disease control. The median progression-free survival reached 7.8 months, significantly longer than the historical average of 2 to 3 months for similar patients. Responses typically appeared within 2.7 months and lasted a median of 6.4 months.
The treatment demonstrated a manageable safety profile in 180 patients, with no treatment-related deaths and no severe grade 4 or 5 side effects. The most common adverse events were mild fatigue, nausea, temporary liver enzyme increases, and proteinuria. Importantly, the drug caused very low rates of nerve damage, blood-cell suppression, eye problems, hair loss, or lung inflammation.