Full Press Release Details
Krystal Biotech Announces Positive Results from Phase 2 Clinical Trial
( GEM-2 study ) of KB103 and Receives Regenerative Medicine Advanced Therapy ( RMAT ) Designation from FDA for KB103
PITTSBURGH, June 24,
2019 Krystal Biotech, Inc. (Nasdaq: KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from rare dermatological diseases, today announced positive results from its Phase 2
placebo-controlled clinical trial of KB103 (GEM-2) study and an update on results from Phase1 (GEM-1) study.
GEM-2 Study of KB103
In the Phase 2 trial, four additional patients (two adults (ages 22 and 19) and two pediatric (ages 14 and 15)) with severe generalized recessive dystrophic
epidermolysis bullosa ( RDEB ) were enrolled in December 2018. Prior to dosing, three wounds of size up to 20cm2 were selected on each patient for the trial and subsequently randomized
to receive either KB103 or placebo in a 2:1 (KB103: placebo) ratio. A total of four KB103-treated recurring wounds and two KB103-treated chronic wounds were included in the trial results. Having established the presence of type VII collagen (COL7)
and anchoring fibrils in the Phase 1 portion of the trial and acknowledged by the FDA, the endpoints of the Phase 2 portion were revised toward a focus on clinical improvement.
One of the four patients (aged 19) voluntarily dropped out of the trial after 30 days due to an inability to travel to the clinical trial site. The dropout
was unrelated to any safety or efficacy issues with KB103. Data from this patient was excluded from the overall analysis. The Phase 2 analysis was conducted on remaining three patients (six KB103 treated wounds and three placebo treated wounds) in
the Phase 2 trial. With respect to the six KB103 treated wounds on patients enrolled for the duration of the Phase 2 trial, two were categorized as chronic and four as recurring based on patient reporting. Chronic wounds remain open for greater than
12 weeks while recurring wounds open and close spontaneously.
Earlier in the Phase 1 trial, two adult patients with severe generalized RDEB were
evaluated. In each patient, two wounds with an approximate surface area of 10 cm2 were randomized to receive either topical KB103 or placebo.
Clinical Data Update
from all patients in Phase 2 trial at 90 day timepoint show that KB103 was well tolerated. No serious adverse events, and no drug-related adverse events were reported. No inflammation or irritation was observed in KB103-treated wounds. In addition,
no antibody response was noted for COL7.
Clinical Endpoint: Percent of Wound Closure
Clinical Endpoint: Time to 100% wound closure
Clinical Endpoint: Duration of wound closure
Mechanistic Endpoints:
New treatements 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 Dr. Peter Marinkovich, M.D., 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.
Ongoing and next steps
enrolled and increased frequency of dosing on two additional patients to study chronic wounds in more detail and in preparation of designing a robust pivotal trial. We anticipate commencing the Phase 3 pivotal trial before year end 2019 and will
provide a clinical update on these additional patients prior to commencing a pivotal trial.
KB103 s RMAT designation follows our
recent announcement of the Priority Medicines, or PRIME designation received from the EMA, and we look forward to collaborating closely with the EMA and initiating a clinical trial in the EU in the upcoming months. The RMAT pathway is analogous to
the Breakthrough Therapy designation designed for traditional drug candidates and medical devices and was specifically created by the U.S. Congress in 2016 to get important new cell therapy and gene therapy products to the patient earlier. Just like
the Breakthrough designation, it allows companies developing regenerative medicine therapies to interact with the FDA more frequently in the clinical testing process, and RMAT-designated products may be eligible for priority review and accelerated
Management, along with the principal investigator on the study Dr. Peter Marinkovich, M.D., Stanford University, will
elaborate on the clinical data during a webcasted conference call today at 8:45 a.m. EST. To join online please click:
https://www.netroadshow.com/nrs/home/#!/?show=ef9bf999 or www.netroadshow.com and enter the entry code: KRYS2019 or visit
www.netroadshow.com and enter the entry code: KRYS2019
*Dial in details will appear after you ve launched the presentation and agreed
If you require assistance or do not have access to a computer or mobile device, please join by phone: U.S. Toll
Free: 1.877.820.2105 or International: +1.470.279.7412 and enter the entry code: KRYS2019
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 Regenerative Medicine Advanced Therapy
Established under the 21st Century Cures Act, RMAT designation is a program designed to expedite the development and approval
of regenerative medicine products, including gene therapy products. An investigational therapy is eligible for the RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and
preliminary clinical evidence indicates a potential to address unmet medical needs for that disease or condition. The designation includes all the benefits of the FDA s Fast Track and Breakthrough Therapy designations and enables the ability to
work more closely and frequently with the FDA to discuss surrogate or intermediate endpoints to support the potential acceleration of approval and satisfy post-approval requirements.
About the Priority Medicines (PRIME) Initiative
PRIME is a program launched by the European Medicines Agency (EMA) to enhance support for the development of medicines that target an unmet medical
need. This voluntary program is based on enhanced interaction and early dialogue with developers of promising medicines, to optimize development plans and speed up evaluation so these medicines can reach patients earlier. Through PRIME, the EMA
offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine s benefits and risks and enable accelerated assessment of medicines applications. The goal of the initiative is to help patients
benefit as early as possible from therapies that may significantly improve their quality of life.
About Dystrophic Epidermolysis Bullosa
Dystrophic 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 trial 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.
LifeSci Public Relations
Source: Krystal Biotech, Inc.