Phase 3 Findings on Birch Triterpenes for Epidermolysis Bullosa, with Anna Bruckner, MD

The rare and severe dermatologic condition known as epidermolysis bullosa (EB) is a group of severe genetic disorders impacting patients’ skin, promoting fragility and open wounds. Erosions, cutaneous and mucosal blistering, and impaired healing are all manifestations of the disease.

A phase 3 study titled EASE was conducted to evaluate the long-term efficacy and safety of birch triterpenes (Filsuvez), also referred to as Oleogel-S10, among individuals with dystrophic EB (DEB) and junctional EB (JEB).¹ After the EASE trial’s 24-month, open-label phase, the HCPLive team spoke with trial investigator Anna Bruckner, MD, professor of dermatology and pediatric dermatologist at the University of Colorado, to discuss new findings.

“This follows on the heels of a 90-day randomized control trial that looked at patients with epidermolysis bullosa who received [birch triterpenes] to treat the wounds that are associated with EB,” Bruckner explained. “The primary patient population was mainly patients with recessive dystrophic EB, but the study also included patients with dominant dystrophic and junctional EB.”

Bruckner highlighted the design of the EASE trial and its open-label extension trial. Those in the treatment cohorts of the double-blind phase entered the single-arm open-label phase, with all EB partial thickness wounds being treated with birch triterpenes. The endpoints of the study included data on adverse events, shifts in body surface area percentage of wounds, wound infection maximum severity, EB Disease Activity and Scarring Index (EBDASI), pruritus, disease severity, pain, and quality of life.

“The way that this therapy works is that it's a topical product that's applied to all of the open wound areas when the individuals’ bandages are changed,” Bruckner said. “And the major findings that the open-label study showed were that there was a sustained reduction overall in terms of wound burden for these patients and that, overall, the therapy is very safe…”

EASE’s findings on birch triterpenes were noted by Bruckner as encouraging, given the positive long-term safety profile of the treatment and the sustained reduction the investigators had observed in patients’ wound burden over at least 24 months of therapy.

The study demonstrated that while adverse events (AEs) were noted among 77.1% of subjects, such events were typically mild-to-moderate. Bruckner’s team found severe and serious AEs in 18.0% and 24.4% of participants, respectively.

“I think it is also important to keep in mind that…these patients do tend to have a lot of medical needs, and the nature of EB means that they're going to have problems with infections or other things that could be going on,” Bruckner said. “But thankfully, this open-label extension did not show that there was an increased risk of problems with treated wounds, such as infections and whatnot. So I think that was very reassuring. Also, there was not an increased risk for other complications that we think about with long-term wounds in patients with EB such as such squamous cell carcinoma.”

For additional information on these new data, view the full interview segment posted above.

The quotes contained in this summary were edited for clarity.

Bruckner highlighted the following disclosures for this interview: Amryt Pharma / Chiesi (contracted research, advisor/consultant, speaker); Castle Creek / Fibrocell (contracted research, advisor/consultant); Krystal Biotech (consultant); Phoenix Tissue Repair (contracted research); Phoenicis Therapeutics (formerly ProQR/Wings) (contracted research); RheaCell (contracted research); Twi Biotechnology (contracted research, consultant).

References

1. Murrell DF, Bodemer C, Bruckner AL, Cunningham T, Davis C, Fernández MF, Kiritsi D, Maher L, Sprecher E, Torres Pradilla M, Kern JS. Long-term safety and efficacy of Oleogel-S10 (birch bark extract) in epidermolysis bullosa: 24-month results from the Phase III EASE Study. Br J Dermatol. 2025 Jan 16:ljaf022. doi: 10.1093/bjd/ljaf022. Epub ahead of print. PMID: 39821055.