Krystal Biotech: Topical gene therapy shows positive results in phase 3 trial of dystrophic epidermolysis bullosa wound healing


Approximately one in every 20,000 children born in the U.S. is affected by epidermolysis bullosa (EB), a rare and debilitating connective tissue disorder with many genetic and symptomatic variations. Patients with EB can experience significantly diminished quality of life.

Prominent symptoms of EB include extremely fragile skin that causes painful blisters and ruptures and wounds to the skin, which are prone to infection. Internal organs and bodily systems can be seriously affected. EB can lead to squamous cell carcinoma, which in some cases can be deadly before patients reach the age of 30 years.

Vyjuvek was effective in healing the majority of dystrophic epidermolysis bullosa wounds in a phase 3 trial, Krystal Biotech announced in a press release.

B-VEK has been granted orphan drug designation by both the FDA and European Medicines Agency. The FDA has also given the drug fast-track designation and rare pediatric designation for the treatment of dystrophic epidermolysis bullosa.

Krystal plans to file a Biologics License Application with the FDA in the first half of 2022.

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Where was this DD yesterday?? :broken_heart:

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Attached are the supporting materials from todays webcast in PDF format.
Nov28FINAL - slides GEM-3 topline.pdf (1.4 MB)

A stock offering was also announced today - likely to capitalize on the positive results of the study: Krystal Biotech Announces Proposed $200 Million Public Offering of Common Stock

The Company and certain selling stockholders intend to grant the underwriters a 30-day option to purchase up to an additional $30 million of the shares of the Company’s common stock offered in the public offering. The offering is subject to market and other conditions, and there can be no assurances as to whether or when the offering may be completed, or as to the actual size or terms of the offering. All of the shares to be sold in the proposed offering will
be sold by the Company other than up to 200,000 shares of the Company’s common stock that may be sold by the selling stockholders in connection with the exercise of the underwriters’ option to purchase additional shares.

It looks like they are using the proceeds to try to prepare to for possible commercialization of their treatment, as well as advancing development on current pipeline.

Krystal Biotech Announces Proposed $200 Million Public Offering of Common Stock.pdf (31.4 KB)

Ticker: KRYS

This popped today, but may still have more to give.
I wouldn’t chase it now though. If you plan to, watch how it moves in pre-market and look for an entry near or below VWAP.


Ortex data…