Phase 3 Study of Ameluz-PDT in Superficial Basal Cell Carcinoma Reaches 1-Year Follow-Up to Support FDA sNDA Submission

Basal Cell Carcinoma | Maris– stock.adobe.com

All patients enrolled onto the phase 3 ALA-BCC-CT013 study (NCT03573401), which is evaluating treatment with the investigational photodynamic therapy (PDT) Ameluz alongside the BF-RhodoLED lamp in patients with superficial basal cell carcinoma (sBCC), have completed 1-year follow-up as of December 2024, according to an announcement from Biofrontera.¹

One-year follow-up data will support the company’s submission of a supplemental new drug application to the FDA, which is anticipated for the third quarter of 2025. Patients with sBCC lesions treated during the study will continue to be followed for another 4 years.

“We were delighted with the highly statistically significant results for the primary and secondary end points that we communicated [in 2024],” Hermann Luebbert, PhD, chief executive officer and chairman of Biofrontera, stated in the news release. “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.”

Prior results from ALA-BCC-CT013 were reported in October 2024, and they showed that statistical significance was reached for all primary and secondary efficacy end points with PDT plus Ameluz (P <.0001).² Complete clinical and histological clearance of a preselected “main target” BCC lesion—the study’s primary end point—was observed among 65.5% of evaluable patients in the PDT and Ameluz arm (n = 145) 12 weeks after initiation of the last PDT cycle; among evaluable patients in the placebo and PDT arm (n = 42), the success rate was 4.8%.

Furthermore, 75.9% and 19.0% of patients in each respective arm achieved complete histological clearance. The complete clinical clearance rates were 83.4% and 21.4%, respectively.

Total clearance of all sBCC lesions was achieved by 64.1% of patients in the Ameluz/PDT arm vs 4.8% of patients in the placebo/PDT arm. A total of 64.3% and 22.2% of patients, respectively, reported their overall treatment satisfaction and aesthetic outcome as “very good” or “good."

“We routinely use PDT in our institution for the treatment of actinic keratoses” M. Shane Chapman, MD, an investigator for ALA-BCC-CT013, added in a news release.¹ “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”. Chapman also serves as chair of the Department of Dermatology at Dartmouth Hitchcock Medical Center and the Geisel School of Medicine at Dartmouth.

Trial Overview

The double-blind, placebo-controlled, multicenter ALA-BCC-CT013 study enrolled 187 patients at least 18 years of age with at least 1 sBCC lesion likely to be histologically confirmed.³ To be eligible for the study, at least 1 treatment-naive sBCC lesion in the face/forehead, bald scalp, extremities, and/or neck/trunk was required, eligible lesions needed to be at least 0.6 cm in size, and the entire treatment field could not exceed approximately 20 cm². Patients also needed to be willing to undergo up to 4 cycles of PDT within 3.5 months, have no significant physical abnormalities within the potential treatment field, abstain from extensive sunbathing, and be willing to stop using moisturizers or any other cosmetics within the treatment field.

Key exclusion criteria included hypersensitivity to 5-aminolevulinic acid (5-ALA), current treatment with immunosuppressive therapy, or evidence of clinically significant unstable medical conditions.

Upon enrollment, patients were randomly assigned to receive PDT in addition to either topical BF-200 Ameluz containing 7.8% 5-ALA or a topically applied vehicle containing no active ingredient. PDT treatments were administered 1 to 2 weeks apart, and lesions that were not completely resolved after 3 months were retreated.¹

The trial’s primary end point was the complete clinical and histological response of a patient’s main target lesion at 12 weeks following the start of their last PDT cycle including treatment of the main target lesion.³ Main target lesion clinical response rate, main target lesion histological response rate, the patient complete clinical response rate, and patient satisfaction with aesthetic outcomes and treatment, served as secondary end points.

References

1. Biofrontera Inc. announces achievement of key milestone in phase 3 study of Ameluz®-Photodynamic Therapy (PDT) in the treatment of superficial basal cell carcinoma (sBCC). News release. Biofrontera. January 8, 2025. Accessed January 8, 2025. https://investors.biofrontera-us.com/full-news/?qm-storyId=8216409730342465
2. 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. October 31, 2024. Accessed January 8, 2025. https://investors.biofrontera-us.com/full-news/?qm-storyId=5894271814242812
3. 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 January 8, 2025. https://clinicaltrials.gov/study/NCT03573401