Biofrontera AG (FSE: B8F), the specialist for sun-induced skin cancer, presented data from three pivotal Phase III trials for its lead drug candidate BF-200 ALA at the 14th Annual South Beach Symposium. The studies demonstrate very high efficacy, positive safety and excellent aesthetic outcomes that is activated using photodynamic therapy (PDT) for the topical treatment of actinic keratosis (AK), a superficial skin cancer caused by sun damage.
Two of the studies formed the basis for marketing authorization approval of BF-200 ALA as first-line treatment of mild to moderate actinic keratosis on the face and scalp by the European Medicines Agency in December 2011. The third study was performed to strengthen the New Drug Application submitted to the U.S. Food and Drug Administration in July 2015. Filing of the application was accepted in September, since when the FDA undertakes its substantive review. No review issue was reported by the FDA following their mid-term review meeting.
In the three Phase III trials conducted in centers in Germany, Switzerland and Austria, a total of 779 patients with 4 to 8 mild to moderate AK lesions were randomized, with all three studies using red light PDT with LED illumination sources at approximately 635 nanometers (nm).
- A 122-patient Phase III study demonstrated excellent efficacy of BF-200 ALA compared to placebo with a patient complete clearance rate of 87%, compared to placebo response of 13%.1
- A 571-patient Phase III study showed significantly improved patient clearance rates for BF-200 ALA (85%) compared to those of placebo (13%) and methylaminolevulinate (MAL) (68%), which is marketed as Metvix™ and was the current standard of care topical photosensitizing agent used in PDT. A 1-year follow-up showed slightly lower recurrence rates for BF-200 ALA compared to MAL, and PDT side effects were not enhanced using BF-200 ALA.2
- An 86-patient Phase III study used a field directed approach (the entire tube of BF-200 ALA was applied to a field of cancerized skin) and compared efficacy and cosmetic outcome of BF-200 ALA with placebo, finding that 91% percent of the patients completely cleared after a maximum of two treatments with BF-200 ALA, compared to only 22% complete clearance with placebo. With BF-200 ALA treatment, clearance was sustained over one year of follow-up in 63.3% of the patients. Several cosmetic parameters were followed during follow-up and found to continuously improve, for the first time proving the strong skin rejuvenation of PDT in a controlled pivotal trial.3
In addition to the three phase III studies on AK, results of a Phase III study of BF-200 ALA for the treatment of non-aggressive basal cell carcinoma (BCC) showed high clearance rates of all superficial and nodular BCCs in more than 93.4% of the patients.
Prof. Dr. Hermann Lübbert, PhD, CEO of Biofrontera AG, commented, "These results from our three Phase III trials evaluating BF-200 ALA in actinic keratosis are substantial as they demonstrate the clinical viability of our novel combination therapy system. Our photosensitizer agent BF-200 ALA utilizes a unique delivery method through nanoemulsion technology which constitutes a stabilized and penetration-enhancing formulation for use in PDT, making it one of the most effective treatment modalities for the removal AK lesions and BCC tumors."
Dr. Brian Berman, MD, PhD, Professor Emeritus, Dermatology and Cutaneous Surgery at the University of Miami Miller School of Medicine and SBS Session Director and presenter of the oral presentation Futures Symposium: Innovations in Clinical and Aesthetic Dermatology, added, "Skin cancer remains the most diagnosed cancer in the U.S., and can often be fatal, requiring better treatment options and improved outcomes for patients. Innovations in the clinic like BF-200 ALA could offer an effective and safe treatment option. Data from the large 570-patient comparator study in particular demonstrated that BF-200 ALA reaches a significantly superior patient complete clearance rate in AK-treatment as compared to Metvix® while the side-effect profile does not differ. Furthermore, the high therapeutic efficacy of BF-200 ALA becomes particularly evident when treating Olsen grade II lesions and lesions on the scalp."