Krystal Biotech’s (NASDAQ:KRYS) stock more than doubled on November 29 after the company said its pivotal, phase III trial of B-VEC met designated primary and secondary endpoints.
B-VEC was tested to treat the rare, genetic skin disease dystrophic epidermolysis bullosa (DEB). Krystal said B-VEC achieved the trial’s primary endpoint of complete wound healing at six-month timepoints and the secondary endpoint of complete wound healing at three-month timepoints.
SanaCurrents issued a report forecasting a positive outcome for Krystal’s GEM-3 trial on July 16, 2021, when the stock traded at $63.57 per share. Despite the bear market for small biotechs during 2021, which pushed Krystal shares down to $40 per share at close on November 26, Krystal opened Monday, November 29 at $102 per share.
Many dermatology diseases are treated with topical drugs, often with poor results. Krystal’s B-VEC likewise adopts a topical approach but B-VEC works as a gene therapy, not topical drug therapy. Or, more precisely, a gene delivery therapy.
The distinction proved important, as B-VEC achieved a genetically corrective approach to treating DEB non-invasively while avoiding the side effects induced by many prior gene therapies. B-VEC, now named Vyjuvek, becomes the first, non-invasive and re-dosable gene therapy in development, according to Krystal. The company plans to advance is gene delivery technology to treat six additional medical conditions.
SanaCurrents’ MACE score appears as an image and the abstract from the July 16 report is below.
Abstract
Dystrophic epidermolysis bullosa (DEB) is a debilitating genetic condition in which a missing protein called type 7 collagen (COL7) causes the skin’s upper layer, the epidermis, to disconnect from its lower layer, the dermis. Krystal’s B-VEC therapy attempts to replace the COL7 protein by delivering a COL7A1 gene to the site of the wound.. Krystal expects to report topline results of a 31-patient, phase III B-VEC trial before the end of 2021.