Ameluz-PDT Leads to Target Lesion Clearance in Superficial Basal Cell Carcinoma

Todd Schlesinger, MD, FAAD, FASMS

Ameluz (BF-200 ALA) plus photodynamic therapy (PDT; Ameluz-PDT) performed with the BF-RhodoLED lamp generated a statistically significant improvement in target lesion clearance outcomes vs placebo plus PDT (placebo-PDT) in patients with superficial basal cell carcinoma (sBCC), according to topline data from the phase 3 ALA-BCC-CT013 trial (NCT03573401).¹

The trial’s primary end point was a composite of complete histological and clinical clearance of 1 preselected “main target” BCC lesion in each patient measured 12 weeks after the initiation of the last PDT cycle. Among 145 evaluable patients in the Ameluz arm, the success rate of this end point was 65.5% compared with 4.8% among 42 evaluable patients in the placebo arm (P < .0001).

Complete histological clearance of these lesions was observed in 75.9% of patients in the Ameluz arm vs 19.0% of those in the placebo arm. The complete clinical clearance rates were 83.4% and 21.4% in these respective arms.

The outcomes for all other secondary efficacy parameters were highly significant (P < .0001). These outcomes included total clearance of all sBCC lesions in 64.1% of patients in the Ameluz arm compared with 4.8% of those in the placebo arm. Furthermore, 64.3% of patients who received Ameluz-PDT rated their overall treatment satisfaction and esthetic outcome as very good compared with 22.2% of those who received placebo-PDT.

“We are delighted that these highly significant results mirror those found in the European studies,” Hermann Luebbert, PhD, chief executive officer and chairman of Biofrontera, stated in a news release. “Once we finish the 1-year follow-up phase in December [2024], we will be in a position to submit our dossier to the FDA around the end of [the second quarter or early in the third quarter] of 2025”.

The double-blind, multicenter ALA-BCC-CT013 trial evaluated the safety and efficacy of Ameluz-PDT vs placebo-PDT in 187 patients with at least 1 histologically and clinically confirmed sBCC. Eligible patients included those at least 18 years of age with at least 1 treatment-naive sBCC lesion in the face/forehead, bald scalp, extremities, and/or neck/trunk. Lesions were not permitted to be within the embryonic fusion planes, such as within 2 cm of the hair zone or on the ears.² Eligible sBCCs lacked histological evidence of aggressive growth patterns. The diameter of each eligible lesion was at least 0.6 cm, and the entire treatment field could not be larger than approximately 20 cm2.

Patients were randomly assigned to receive 1 cycle of 2 PDT treatments (either Ameluz-PDT or placebo-PDT) 1 to 2 weeks apart. Treatment was repeated after 3 months if required.^1,2

Secondary efficacy outcomes and safety were also evaluated.^2,3

The database lock for this trial occurred in October 2024.3 For Ameluz-PDT to be considered for FDA approval for patients with sBCC, the regulatory agency needs to receive the final ALA-BCC-CT013 study report, as well as follow-up data obtained 1 year after the trial’s first PDT dose. The last patient treated in the trial is expected to complete this follow-up period by December 2024.

“I am excited that the potential FDA approval [of Ameluz-PDT] may provide me with a new noninvasive option for my patients that may not involve potential scarring or repeated office visits for ionizing radiation, which could therefore lead to reduced patient burden and a beneficial cosmetic outcome,” Todd Schlesinger, MD, FAAD, FASMS, a board-certified dermatologist at Epiphany Dermatology in Charleston, South Carolina, a Mohs surgeon, and main contributor to the study, added in the news release.¹

References

1. Biofrontera Inc. announces highly significant results in phase 3 study of Ameluz-photodynamic therapy (PDT) regarding the treatment of superficial basal cell carcinoma (sBCC). News release. Biofrontera Inc. October 31, 2024. Accessed November 1, 2024. https://investors.biofrontera-us.com/full-news/?qm-storyId=5894271814242812
2. Study to evaluate the safety and efficacy of BF-200 ALA (Ameluz) and BF-RhodoLED in the treatment of superficial basal cell carcinoma (sBCC) with photodynamic therapy (PDT). ClinicalTrials.gov. Updated June 26, 2024. Accessed November 1, 2024. https://clinicaltrials.gov/study/NCT03573401
3. Biofrontera Inc. announces database lock of phase 3 study of Ameluz-photodynamic therapy (PDT) in the treatment of superficial basal cell carcinoma (sBCC). News release. Biofrontera Inc. October 14, 2024. Accessed November 1, 2024. https://investors.biofrontera-us.com/full-news/?qm-storyId=8891234282776144