Dystrophic Epidermolysis Bullosa Treatment Receives IND Clearance


Dystrophic epidermolysis bullosa treatment receives IND clearance.

This morning, Krystal Biotech announced that the US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance to its dystrophic epidermolysis bullosa (DEB) treatment, KB103.

KB103 recently received Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA) for the treatment of DEB. In an exclusive interview with Rare Disease Report®, chairman and chief executive officer of Krystal Biotech, Krish Krishnan, discussed KB103’s drug mechanisms and projected timeline.

"Clearance of the IND by the FDA is the first of several important milestones for the KB103 program,” he said in a recent statement. “I am proud of the efforts of the Krystal team that has worked diligently to bring KB103 into the clinic in a timely manner. We are excited about the possibility of providing a simple topically-applied treatment to fundamentally treat this debilitating disease.”

KB103 is a replication-defective, non-integrating viral vector that has been engineered to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is the replication-deficient, non-integrating viral vector that is able to penetrate skin cells more efficiently than other viral vectors. Due to its high payload capacity, it is able it to accommodate large or multiple genes. In addition, its low immunogenicity makes it an appropriate choice for direct and repeated delivery to the skin (in the form of a cream).

DEB is a debilitating, genetic condition that causes the skin to be very fragile and blister easily. The slightest of injuries, such as bumping or rubbing, can cause the skin to break and blister. DEB is caused by a mutation in the gene called COL7A1. When mutated, COL7A1 doesn’t produce the required protein collagen VII to create the anchoring fibers that hold the skin together.

The Phase 1/2 clinical study of KB103 is expected to start in May 2018 and will be a single site study conducted at Stanford University. The release date for the study’s initial data is expected by the end of 2018.

Suma Krishnan, founder and chief operating officer of Krystal, expressed her excitement for the drug’s potential. "We are excited about KB103 entering the clinic. Our goal is to demonstrate that KB103 could truly be a first-in-class potent treatment for DEB that has the potential to change the lives of people affected by this condition,” she said. “We plan on enrolling patients in this Phase 1/2 study as soon as we can and hope to have a meaningful data readout shortly."

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