Krystal Biotech has developed a gene therapy, called VYJUVEK, which is designed for use in patients with Dystrophic EB (DEB) to heal chronic wounds.
They have recently announced the positive news that VYJUVEK has passed Phase 3 Clinical Trials, as it has been successful in meeting its targets for healing wounds after both 3 months and 6 months of use. By passing Phase 3 Clinical Trials, VYJUVEK has also proven to have no serious side effects.
VYJUVEK is a potential topical treatment that is applied directly to the wound; therefore, it is not invasive. The treatment is designed to deliver two copies of the COL7A1 gene directly to wounds. It then has the potential to treat DEB at a molecular level by helping the patient’s skin cells to make COL7 protein as normal. This therefore targets the cause of the disease.
31 patients took part in the trial with following results:
67% of patients who were treated with VYJUVEK achieved complete wound healing after 6 months, compared to only 22% who were treated with a placebo.
71% of patients who were treated with VYJUVEK achieved complete wound healing after only 3 months, compared to 20% who were treated with a placebo.
VYJUVEK was well tolerated. No drug-related serious adverse events or discontinuations due to treatment were reported. One mild drug-related adverse event was reported during the trial.
Below you can see the effect VYJUVEK had on wounds compared to the treatment used by the placebo group.
“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,” said Dr. Peter Marinkovich, M.D., Bullous Disease Clinic Director and Associate Professor of Dermatology at Stanford University.
Next Steps for Krystal Biotech
• File a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in the first half of 2022
• Submit a Marketing Authorization Application (MAA) in Europe shortly after the BLA.
• Krystal will continue to manufacture VYJUVEKTM using the commercial scale process at its in-house cGMP manufacturing facility, ANCORIS, which was designed to support potential launch.
• The Company is currently constructing its second, larger, facility ASTRA, which is expected to come on-line in 2022 to help support a potential global launch and the pipeline.