Shares of Krystal Biotech Inc (NASDAQ: KRYS) more than doubled on Monday after the gene therapy company said its pivotal GEM-3 trial for VYJUVEK reported positive topline results.
GEM-3 trial met its primary and secondary endpoint
According to Krystal Biotech, the study involving its topical treatment for Dystrophic Epidermolysis Bullosa (Dystrophic EB) achieved the primary and secondary endpoint. In the press release, COO 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.
VYJUVEK is different from the current standard of care as it treats Dystrophic EB at the molecular level. The topical gene therapy did not present any serious adverse effects during the trial.
The sharp increase in the stock price today added millions to Krystal Biotech’s market cap that now stands at $1.92 billion.
BLA to be submitted to the FDA next year
Krystal Biotech expects biologics license application for the treatment to be submitted to the U.S. FDA by mid-2022. Shortly after, it added, it will apply for marketing authorisation in Europe.
With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.
Krystal Biotech is a Pittsburgh-headquartered pivotal-stage gene therapy company focused on treatments for rare diseases. Including a nearly 150% surge on Monday, the stock is now up 60% year-to-date.
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