Anterogen's Stem Cell Therapy ALLO-ASC-SHEET Fails to Meet Primary Endpoint in US Phase 2 DFU Trial

Anterogen announced disappointing results from its U.S. Phase 2 trial (DFU-102) evaluating ALLO-ASC-SHEET for the treatment of Wagner Grade 1 diabetic foot ulcers (DFU). The stem cell therapy failed to demonstrate superiority over conventional treatment, marking another setback in the company's clinical development program.

The randomized trial enrolled 66 patients, equally divided between the ALLO-ASC-SHEET treatment arm and a control arm receiving standard hydrogel sheet dressing. After 12 weeks of treatment, the study's primary endpoint of complete wound closure was not met, with the treatment group showing a lower success rate of 45.7% compared to 60.0% in the control group (p=0.2503).

Treatment Profile and Development History

ALLO-ASC-SHEET is an innovative allogeneic adipose-derived stem cell therapy delivered in sheet form. The treatment was developed to address the limitations of conventional dressings and antibiotics, aiming to promote cell regeneration and tissue healing in conditions such as dystrophic bullous epidermolysis bullosa (DEB) and diabetic foot ulcers.

The development journey has been challenging, with setbacks in both domestic and international trials. Prior to this latest disappointment, the company had already experienced a failure in meeting primary endpoints in a Korean Phase 3 trial (DFU-301). Despite receiving FDA approval for Phase 1/2a studies in November 2016 and securing a waiver to proceed directly to Phase 2 based on Korean Phase 2 data, the treatment has struggled to demonstrate statistical significance in larger trials.

Strategic Pivot to More Severe Cases

In response to these results, Anterogen has announced a strategic shift in focus toward treating more severe cases. The company is currently conducting an additional U.S. Phase 2 trial (DFU-103) specifically targeting Wagner Grade 2 patients, who present with deeper ulcers involving muscles and ligaments and face higher infection risks.

"We plan to obtain therapeutically confirmatory results from the ongoing U.S. phase 2 (DFU-103) in Wagner Grade 2 patients," Anterogen stated in their disclosure. The outcome of this ongoing trial is likely to be crucial in determining the future development pathway for ALLO-ASC-SHEET.

Corporate Background

The trial results come amid significant changes in Anterogen's corporate structure. The company's historical ties with Bukwang Pharmaceutical, once a major shareholder, have been severed. Bukwang, which initially held an 18.71% stake, gradually reduced its position starting in October 2018 and completely divested by 2020, ending what had been a close relationship initiated through Anterogen's founder, Lee Sung-koo, a former Bukwang CEO.