New Phase 3 VIITAL Data Confirms Efficacy of Abeona’s Pz-cel for RDEB

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New data from the Phase 3 VIITAL study (NCT04227106) has been published in The Lancet, showcasing the safety and efficacy of Abeona Therapeutics’ prademagenezamikeracel (pz-cel; Zevaskyn) for recessive dystrophic epidermolysis bullosa (RDEB).¹

Jean Tang, MD, PhD, professor of dermatology at the Stanford University School of Medicine, principal investigator of the VIITAL study, and lead author of The Lancet publication, shared her thoughts in a statement.

"People with RDEB endure incredibly fragile skin, leading to severe, painful wounds that can last for years, and lead to systemic complications impacting the length and quality of life. Our VIITAL study showed ZEVASKYN was well tolerated and significantly improved wound healing and pain in large chronic RDEB wounds after just a single treatment. We hope sharing these important findings, including our work published in The Lancet, raises awareness for RDEB and helps improve the lives of patients,” Tang said.¹

RDEB is a rare, inherited skin disorder characterized by mutations in the COL7A1 gene. This mutation disrupts the production of functional collagen VII, a critical protein needed to anchor the dermal and epidermal layers of the skin together. As a result, patients suffer from years of severe skin fragility, leading to chronic blistering, persistent wounds, debilitating pain, risk of life-threatening infections, and a heightened susceptibility to aggressive squamous cell carcinoma. There is currently no cure for RDEB.

VIITAL was an intra-patient randomized, open-label, controlled, Phase 3 study that included 11 patients with RDEB and focused on 43 pairs of their large chronic wounds. Both co-primary endpoints of wound healing and pain reduction were met when compared to matched untreated (control) wounds. Over 80% of wounds were healed by 50% or more from baseline after 24 weeks, compared to only 16% of control wounds (p < 0.0001).

The mean change in wound pain, measured by the Wong-Baker FACES scale, was -3.1 in treated wounds and -0.9 in control wounds. Greater reductions in itch and blistering were also noted, with 65% of treated wounds healing by 75% or more at the conclusion of the trial. No serious adverse events or cases of squamous cell carcinoma were reported, aligning with the previous phase 1/2a study of pz-cel (NCT01263379).

Pz-cel represents a novel therapeutic approach. It is created by harvesting a patient’s own skin cells, correcting the genetic defect ex vivo with a functional COL7A1 gene, and then expanding these corrected cells into sheets of keratinocytes. These gene-corrected cell sheets are surgically applied to chronic wound sites in a one-time procedure, where they express collagen VII locally, promoting more durable skin adhesion and wound healing. In April, it became the first and only autologous cell-based gene therapy product approved by the US Food and Drug Administration (FDA) to treat RDEB wounds with a single application.²

“The publication of our VIITAL study results in The Lancet, a respected global medical journal, marks a significant achievement as our U.S. launch of ZEVASKYN is underway, with the first patient expected to be treated in the third quarter of 2025,” Vish Seshadri, chief executive officer of Abeona, said in a press release. “We deeply appreciate the invaluable participation of patients and their families in the VIITAL study, along with the scientific community's critical contributions to advancing effective treatment options for RDEB.”¹

References

1. Abeona Therapeutics® Announces Publication in The Lancet of Phase 3 VIITAL™ Study Data in Recessive Dystrophic Epidermolysis Bullosa. News release. Abeona Therapeutics. Published June 24, 2025. Accessed June 26, 2025. https://investors.abeonatherapeutics.com/press-releases/detail/311/abeona-therapeutics-announces-publication-in-the-lancet

2. US FDA approves zevaskyn (prademagenezamikeracel), the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). News release. Abeona Therapeutics. Published April 29, 2025. Accessed June 26, 2025. https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel