Gene therapy biotech sees its stock rocket higher on promising results for rare cases of butterfly disease
Shares of Krystal Biotech took off this morning $KRYS after the little biotech reported promising results from its gene therapy to treat a rare skin disease called epidermolysis bullosa.
Focusing on an update with 4 new patients, researchers spotlighted the success of KB103 in closing some stubborn wounds. Krystal says that of 4 recurring and 2 chronic skin wounds treated with the gene therapy, the KB103 group saw the closure of 5. The 6th — a chronic wound, defined as a wound that had remained open for more than 12 weeks — was partially closed. That brings the total so far to 8 treated wounds, with 7 closures.
At 90 days, meanwhile, all 3 wounds in the placebo group remained open.
Significantly, one patient dropped out of the study after 30 days, which Krystal says was due to difficulty in traveling to the trial site. Investors, though, weren’t complaining. They drove the biotech’s shares up 35% in early trading on Monday.
The kids who suffer from epidermolysis bullosa are sometimes called “butterfly children,” to illustrate a rare genetic condition that causes the skin to remain extremely fragile, blistering and cracking at a touch. It’s caused by mutations in the COL7A1 gene encoding type VII collagen, or COL7.
KB103 — now headed for a Phase III start before the end of this year — was selected by the EMA for its PRIME designation, intended to speed along experimental drugs after demonstrating some promise. And the FDA has now followed up with its own special designation of the therapy for its Regenerative Medicine Advanced Therapy program.
The disease has defeated several attempts to come up with new therapies for it.
“New treatments for patients suffering from EB are desperately needed, especially one that provides a convenient, painless way to administer treatment for patients suffering with this debilitating disease and to reduce their travel burden,” said Peter Marinkovich, associate professor of dermatology, Stanford University and principal investigator in the GEM study. “These exciting data in the Phase 1/2 trials are supportive of this very promising new approach for treating this debilitating disease.”
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