Ameluz (BF-200 ALA) plus photodynamic therapy (PDT), known as Ameluz-PDT, has shown a statistically significant improvement in target lesion clearance compared to placebo plus PDT for patients with superficial basal cell carcinoma (sBCC). These findings come from the phase 3 ALA-BCC-CT013 trial, utilizing the BF-RhodoLED lamp for PDT.
The primary endpoint of the trial was a composite of complete histological and clinical clearance of a preselected main target BCC lesion in each patient, measured 12 weeks after the initiation of the last PDT cycle. In the Ameluz arm, involving 145 evaluable patients, the success rate was 65.5%, while the placebo arm, with 42 evaluable patients, showed a rate of only 4.8% (P < .0001).
Efficacy Outcomes
Complete histological clearance was observed in 75.9% of patients in the Ameluz arm compared to 19.0% in the placebo arm. The complete clinical clearance rates were 83.4% and 21.4% in the respective arms. Secondary efficacy parameters also showed highly significant results (P < .0001), including total clearance of all sBCC lesions in 64.1% of patients in the Ameluz arm versus 4.8% in the placebo arm. Patient satisfaction was also notably higher, with 64.3% of patients receiving Ameluz-PDT rating their overall treatment satisfaction and esthetic outcome as very good, compared to 22.2% in the placebo-PDT group.
Management Perspective
"We are delighted that these highly significant results mirror those found in the European studies," said Hermann Luebbert, PhD, chief executive officer and chairman of Biofrontera, in a news release. The company anticipates submitting its dossier to the FDA around the end of the second quarter or early in the third quarter of 2025, following the completion of the 1-year follow-up phase in December 2024.
Trial Design and Patient Population
The double-blind, multicenter ALA-BCC-CT013 trial involved 187 patients with at least one histologically and clinically confirmed sBCC. Participants were at least 18 years old with treatment-naive sBCC lesions on the face, forehead, bald scalp, extremities, or neck/trunk. Lesions near embryonic fusion planes or with aggressive growth patterns were excluded. The diameter of each lesion was at least 0.6 cm, with the entire treatment field not exceeding approximately 20 cm2.
Patients were randomized to receive one cycle of two PDT treatments (Ameluz-PDT or placebo-PDT) one to two weeks apart, with a potential repeat after three months if needed. Secondary efficacy outcomes and safety were also evaluated.
Regulatory Pathway
The database lock for the trial occurred in October 2024. FDA approval consideration requires the final ALA-BCC-CT013 study report and follow-up data obtained one year after the first PDT dose. The last patient treated is expected to complete this follow-up by December 2024.
Clinical Implications
According to Todd Schlesinger, MD, FAAD, FASMS, a board-certified dermatologist, Mohs surgeon, and main contributor to the study, potential FDA approval of Ameluz-PDT could provide a new noninvasive option for patients, potentially reducing scarring, repeated office visits for ionizing radiation, and overall patient burden, while also offering a beneficial cosmetic outcome.
