Biofrontera Inc. has announced the completion of the 1-year follow-up for all patients enrolled in its Phase 3 study (ALA-BCC-CT013) evaluating Ameluz in combination with RhodoLED photodynamic therapy (PDT) for the treatment of superficial basal cell carcinoma (sBCC). This milestone supports the planned submission of a supplemental New Drug Application (sNDA) to the FDA in the third quarter of 2025, potentially expanding the label for Ameluz to include treatment of this common cutaneous malignancy.
The double-blind, randomized, placebo-controlled, multi-center study enrolled 187 patients with clinically and histologically confirmed sBCC. Participants received either Ameluz-PDT or placebo-PDT in one or two cycles, administered 1-2 weeks apart. Lesions not fully resolved after three months were retreated. The primary endpoint was the complete clinical and histological clearance of a preselected main target lesion at 12 weeks following the last PDT cycle.
Significant Efficacy Demonstrated
In October 2024, Biofrontera reported highly statistically significant results (p < 0.0001) for all primary and secondary endpoints. Specifically, 65.5% of evaluable patients in the Ameluz-PDT arm (n = 145) achieved complete clinical and histological clearance of the main target lesion, compared to only 4.8% in the placebo-PDT arm (n = 42). Secondary endpoints also showed significant improvements in the Ameluz-PDT group, including higher rates of complete histological clearance (75.9% vs. 19.0%) and complete clinical clearance (83.4% vs. 21.4%).
Total clearance of all sBCC lesions was observed in 64.1% of patients treated with Ameluz-PDT, compared to 4.8% in the placebo group. Patient satisfaction was also notably higher in the Ameluz-PDT arm, with 64.3% reporting their overall treatment satisfaction and aesthetic outcome as “very good” or “good,” versus 22.2% in the placebo arm.
Expert Commentary
"We were delighted with the highly statistically significant results for the primary and secondary endpoints that we communicated [in 2024],” stated Dr. Hermann Luebbert, CEO and Chairman of Biofrontera Inc. “The completion of the 1-year follow-up is a crucial milestone in our path to an FDA submission in 2025 and potentially expanding our label to the treatment of a cutaneous malignancy. It demonstrates our continued investment in PDT and supports our vision of partnering with the dermatology community to improve patient care.”
Dr. Shane Chapman, Chair of the Department of Dermatology at Dartmouth Hitchcock Medical Center and an investigator for the study, added, “We routinely use PDT in our institution for the treatment of actinic keratoses. We were impressed with the results of the 12-week data, and I look forward to being able to offer Ameluz-PDT as a treatment option for my patients with sBCC.”
Addressing a Significant Unmet Need
Basal cell carcinoma (BCC) is the most common form of skin cancer, with over 3 million cases diagnosed annually in the U.S. alone. Superficial BCC represents a significant subset of these cases. While generally slow-growing and rarely metastatic, untreated BCC can become locally invasive, causing destruction of skin, tissue, and bone.
Long-term follow-up is crucial in studies of sBCC treatments due to the risk of local recurrence and the potential for new skin cancer development. Patients in the ALA-BCC-CT013 study will be followed for a total of five years to assess the durability of Ameluz-PDT and monitor for any recurrence.
