Full Press Release Details
Krystal Biotech Announces Positive Interim Results from Placebo-Controlled Phase 1/2 Clinical Trial of KB103
KB103 is Krystal s first off-the-shelf , topical, gene
therapy candidate to treat patients suffering from Dystrophic Epidermolysis Bullosa.
Results on 2 patients met all primary efficacy (presence of
functional COL7 expression, observation of NC1 and NC2 reactive anchoring fibrils and continued expression following repeat administration) and safety endpoints (no adverse events, inflammation or irritation) in topically administered KB103 wounds.
With respect to secondary endpoints topically administered KB103 wounds closed in 2 weeks and continue to stay closed to date. Topically
administered placebo administered wounds took 10 weeks to close in patient 1 and did not completely close throughout the study in patient 2.
when administered intradermally to intact skin shows presence functional COL7 expression and anchoring fibrils in both patients.
study is anticipated to be complete in 1H 2019 and a pivotal Phase 3 study is anticipated to commence in 2H 2019.
Management and the Principal
Investigator to host a webcast at 8:45 AM EST webcast/conference call details below.
PITTSBURGH, October 15, 2018 Krystal
Biotech, Inc. (Nasdaq:KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases, today announced positive interim results from its ongoing placebo-controlled
Phase 1/2 clinical trial of KB103.
GEM Study Status update
Two adult Recessive Dystrophic Epidermolysis Bullosa ( RDEB ), NC1[+] patients aged 35 and 28 years old have completed treatment with topical KB103
to date. The patients were re-dosed during the study. In each patient, two wounds with an approximate surface area of 10 cm2 were randomized to receive
either topical KB103 or placebo (for a total of 4 wounds evaluated). KB103 was also injected intradermally in both patients to intact skin to evaluate the mechanism of action of KB103 and validate molecular correction. Both KB103-treated wounds and
intradermally injected intact skin were biopsied to assess functional COL7 expression and anchoring fibril formation. KB103-treated wounds were also evaluated for clinically relevant healing compared to placebo-treated wounds and to baseline. They
were also monitored for general and recombinant viral infection and autoimmune response at each evaluation.
Results on 2 patients demonstrate a
meaningful clinical benefit and suggest that KB103 can afford a simple, convenient, painless way to administer treatment for patients suffering with this debilitating disease, said Dr. Peter Marinkovich, MD, Associate Professor of
Dermatology, Stanford University and Principal Investigator in the GEM study. These early data are encouraging and we look forward to continuing the study in pediatric populations.
Results on 2 patients met
all primary efficacy (presence of functional COL7 expression, observation of NC1 and NC2 reactive anchoring fibrils and continued expression following repeat administration) and safety endpoints (no adverse events, inflammation or irritation) in
topically administered KB103 wounds.
With respect to secondary endpoints topically administered KB103 wounds closed in 2 weeks and
continue to stay closed to date. Topically administered placebo wounds took 10 weeks to close in patient 1 and did not completely close throughout the study in patient 2.
KB103 when administered intradermally to intact skin on both patients shows presence functional COL7 expression and anchoring fibrils.
Management, along with the Principal Investigator on the Study Dr. Peter Marinkovich, MD, Stanford University, will elaborate on the clinical data
during a webcasted conference call today at 8:45 am EST. The dial-in details of the call are
+1-786-815-8673, conference ID: 4487935. The live webcast can be accessed at https://edge.media-server.com/m6/p/5d7wxq73
About Krystal Biotech
Krystal Biotech, Inc.
(NASDAQ:KRYS) is a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases. For more information, please visit http://www.krystalbio.com.
KB103 is Krystal s lead product
candidate that seeks to use gene therapy to treat dystrophic epidermolysis bullosa, or DEB, an incurable skin blistering condition caused by a lack of collagen in the skin. KB103 is a replication-defective,
non-integrating viral vector that has been engineered employing Krystal s STAR-D platform to deliver functional human COL7A1 genes directly to the patients
dividing and non-dividing skin cells. HSV-1 is Krystal s proprietary vector that can penetrate skin cells more efficiently than other viral vectors. Its
high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.
About Dystrophic Epidermolysis Bullosa
epidermolysis bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen protein in the skin. It is caused by mutations in the gene coding for type VII collagen, or COL7, a major component of anchoring
fibrils, which connect the epidermis to the underlying dermis, and provide structural adhesion between these skin layers in a normal individual. The lack of COL7 in DEB patients causes blisters to occur in the dermis as a result of separation from
the epidermis. This makes the skin incredibly fragile, leading to blistering or skin loss at the slightest friction or knock. It is progressive and incredibly painful.
The most severe form of DEB is recessive DEB, or RDEB, which is caused by null mutations in the COL7A1 gene. DEB also occurs in the form of dominant DEB, or
DDEB, which is considered to be a milder form of DEB. There are no known treatments affecting the underlying cause of either form of the disease, and the current standard of care for DEB patients is limited to palliative
treatments. Krystal is developing KB103 for the treatment of the broad DEB population, including both recessive and dominant forms of the disease.
Forward-Looking Statements
This press release on
announcing positive interim data from Krystal s phase 1/2 trial evaluating KB103 in patients suffering from Dystrophic epidermolysis bullosa, or DEB, contains forward-looking statements regarding matters that are not historical
facts, including statements relating to the Company s clinical trials, including plans to commence a pivotal Phase 3 study in 2H 2019. There can be no assurance that the data contained in these results will be replicated in additional current
and future patients enrolled in this or any future trial or that these results will prove clinically meaningful in the development of KB103 as a potential drug. Because such statements are subject to risks and uncertainties, actual results may
differ materially from those expressed or implied by such forward-looking statements. Words such as anticipates, plans, expects, intends, will, potential, hope and
similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon current expectations of the Company and involve assumptions that may never materialize or may prove to be incorrect. Actual
results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. Detailed information regarding factors that may cause actual results to differ
materially from the results expressed or implied by statements in this press release relating to the Company may be found in the Company s periodic filings with the Securities and Exchange Commission, including the factors described in the
section entitled Risk Factors in its annual report on Form 10-K for the fiscal year ended December 31, 2017, and supplemented from time to time and the Company s Quarter Reports on Form 10-Q and other filings submitted by the Company to the SEC, copies of which may be obtained from the SEC s website at www.sec.gov. The parties do not undertake any obligation to update forward-looking
statements contained in this press release.
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