BioTuesdays

Krystal Biotech initiates Phase 3 dystrophic epidermolysis bullosa trial

Krystal Biotech Logo

Krystal Biotech (NASDAQ:KRYS) initiated a Phase 3 clinical trial evaluating beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB).

DEB is a rare and severe monogenic skin disease with no currently-approved treatments. B-VEC is a topical, re-dosable gene therapy designed to treat both the dominant and recessive forms of the disease.

The trial will enroll some 30 DEB patients aged 6 months or older, who will be treated once weekly for six months with either B-VEC or placebo.

The trial’s primary outcome measure is complete wound healing from baseline in B-VEC treated wounds compared with placebo at weeks 20, 22 and 24.

“The initiation of the pivotal study marks an important milestone toward our goal of a potential painless, convenient, and corrective approach to treat patients suffering from this debilitating disease,” Suma Krishnan, Krystal Biotech’s founder and COO, said in a statement. 

“We look forward to having a home dosing protocol in place in the upcoming months that, we believe, would further improve the quality of life of our patients and their caregivers,” she added.

Sign up for the BioTuesdays weekly newsletter

Get the latest news on the healthcare industry’s corporate and clinical developments, executive moves, and market updates. Every Tuesday, in your inbox.