On August 15, 2024 DermBiont, a clinical-stage biotechnology company that is advancing first-in-class topical therapeutics to address patient needs in rare disease oncology and aesthetic dermatology, reported positive initial data from its ongoing open label, multi-center Phase 2a study for the treatment of basal cell carcinoma (BCC) (Press release, DermBiont, AUG 15, 2024, View Source [SID1234645951]). As a result, DermBiont has reprioritized SM-020, a first-in-class, patient-applied, and topical kinase inhibitor, for the development of two lead orphan disease oncology indications: treatment of locally advanced basal cell carcinoma (laBCC) and prevention of BCC in Gorlin Syndrome patients.

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DermBiont’s Phase 2a open-label trial is actively enrolling and treating up to 30 subjects with one to five superficial, nodular, or infiltrating primary BCCs. Lesions are being treated with SM-020 1% gel BID (twice daily) for 28 days, with the primary endpoint based on percent change from baseline in greatest tumor diameter at week 6. The safety of the drug product is being evaluated based on frequency of adverse events (AEs) and application site reactions (ASRs).

At the time of this release, seven subjects with a total of nine BCCs (1 superficial, 4 nodular, and 4 infiltrating) have been treated and show a prompt (within days of starting treatment) and robust clinical response to the application of SM-020 1% gel, clearly demonstrating the disease modifying potential of SM-020. All nine tumors have also demonstrated a partial or total reduction in clinically visible tumor.

Dr. Karl Beutner, M.D., Ph.D., and CEO of DermBiont said: "We are excited about these early results in BCC and the potential for a topical product for the treatment of laBCC and the prevention of BCCs in Gorlin Syndrome. An effective topical treatment would be welcomed by patients with laBCC given the limitations and side effects of the currently approved oral hedgehog inhibitors, while also providing patients with Gorlin Syndrome with an alternative to expensive and painful surgical procedures to remove chronic BCCs that result in significant scarring, require post-operative wound care, and lead to significant down time."

SM-020 has been well tolerated to date with no drug-related adverse events and only rare and transient primarily mild-to-moderate severity application site reactions. The same drug product has also been shown to be extremely well tolerated by subjects with seborrheic keratoses (SK), having been administered by over 100 subjects with SKs twice daily for at least 28 days in prior clinical trials.

Emma Taylor, M.D. and Chief Medical Officer at DermBiont, added: "SM-020 in vitro is highly potent at killing BCC cells and has a well-defined mechanism of action that hits established tumorigenesis pathways known to be the cause of BCCs, so the robust response that we are seeing in the clinic is not surprising."

Basal Cell Carcinoma is the most common cancer with over three million cases annually in the United States alone. A BCC that is at least 10mm in diameter and that has either failed or is not appropriate for conventional treatment is considered a laBCC. In the United States, over 10,000 individuals suffer from laBCC annually, at least 10,000 individuals suffer from Gorlin Syndrome, and more than 30,000 people suffer from high frequency sporadic BCC, with tens of thousands of individuals being diagnosed with multiple basal cell carcinomas annually.

In parallel to ongoing pivotal trial-enabling nonclinical and CMC scale-up work, DermBiont plans to seek Orphan Drug and Fast Track Designations for the treatment of laBCC and the prevention of BCC in patients with Gorlin Syndrome, to be followed by pivotal trials in two orphan indications for the approval of SM-020 for treatment of laBCC and prevention of BCC in Gorlin Syndrome patients. Following the reprioritization of SM-020 for laBCC and Gorlin Syndrome, Dermbiont is evaluating options for its SK program.