Krystal Biotech Inc. (NASDAQ: KRYS) has announced encouraging topline results from its GEM-3 study of experimental berenmagene geperpavec (B-VEC), currently known as VYJUVEK for dystrophic Epidermolysis Bullosa (dystrophic EB) treatment.
VYJUVEK met the primary outcome
The trial’s primary outcome compared complete wound healing of topical VYJUVEK to placebo at six-month intervals and found it to be statistically significant. VYJUVEK is the only non-invasive, redosable, and topical gene therapy under development and the first genetically corrected method to treat dystrophic EB that has passed a double-blind Phase 3 trial.
Krystal Chief Operating Officer and Founder Suma Krishnan said, “Dystrophic Epidermolysis Bullosa is referred to as ‘the worst disease you’ve never heard of’ because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEKTM which showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds.”
Stanford University’s Associate Dermatology Professor and Bullous Disease Clinic Director Dr. Peter Marinkovich commented, “Today’s positive B-VEC results represent the culmination of years of study on the molecular basis and genetic correction of this disease. Finally, dystrophic EB patients may have an easily administered genetically targeted therapy which has been shown to promote durable wound healing in this clinical trial. This is a long overdue milestone for patients living with this disease, and one that has potential to drastically change the treatment paradigm.”
Krystal to file BLA in1H 2022
Based on the results, the company plans to submit a Biologics License Application with the US FDA in 1H 2022 as the first step in the execution of its global commercialization and regulatory strategy. In addition, Krystal will file an MAA in Europe after the BLA, with other regulatory paths stills under consideration.
CEO Krish Krishnan said, “These pivotal data provide important validation of our redosable gene delivery technology, emboldening us to expand our pipeline to address other genetic skin diseases, continue to explore the potential in genetic lung diseases, and invest in growing the platform capability to address new organ systems as well.”
