New England Journal of Medicine Publishes Phase 3 Data on B-VEC in Patients with Dystrophic Epidermolysis Bullosa
December 14 2022 - 5:00PM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a
biotechnology company focused on developing and commercializing
genetic medicines for patients with rare diseases, announced today
that data from the pivotal Phase 3 (GEM-3) trial of beremagene
geperpavec (B-VEC) for dystrophic epidermolysis bullosa (DEB) have
been published here in the New England Journal of Medicine (NEJM).
The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC)
for Dystrophic Epidermolysis Bullosa,” will appear in
the December 15, 2022 issue of the NEJM.
In this GEM-3 trial of 31 patients, complete wound
healing at 6 months occurred in 67.4% of B-VEC wounds compared to
21.6% for placebo (difference, 45.8 percentage points; 95%
confidence interval [CI], 23.6 to 68.0; p=0.002). Complete wound
healing at 3 months occurred in 70.6% of the wounds exposed to
B-VEC as compared with 19.7% of those exposed to placebo
(difference, 51.0 percentage points; 95% CI, 29.3 to 72.6;
p=0.0005).
“The impressive phase 3 results with B-VEC are the
best we have seen to date in patients with DEB and, if approved,
B-VEC provides hope for these patients suffering through
debilitating and potentially life-threatening symptoms associated
with the disease, ” said Peter Marinkovich, M.D., Director of
the Blistering Disease Clinic at Stanford Health
Care, Associate Professor of Dermatology at the Stanford
University School of Medicine, primary investigator of the GEM-3
trial and primary author of the manuscript.
The GEM-3 trial was a randomized, double-blind,
intra-patient placebo-controlled multi-center trial designed to
evaluate the efficacy and safety of B-VEC for the treatment of
DEB. In the trial, matched wounds receiving topical B-VEC or
placebo were evaluated in 31 DEB patients over 26 weeks. The
pivotal GEM-3 trial met its primary endpoint of complete wound
healing at six-months and its secondary endpoint of complete wound
healing at three-months. B-VEC was well tolerated, with no
drug-related serious adverse events or discontinuations due to
treatment.
“B-VEC was developed by Krystal scientists as a
potential first-in-class therapy for DEB,” said Suma Krishnan,
President, Research & Development, Krystal
Biotech. “We are working closely with the FDA to get B-VEC
approved and deliver a meaningful benefit to patients with this
debilitating disease.”
The Company received US Food and Drug
Administration (FDA) filing acceptance of its Biologics License
Application (BLA) for B-VEC. The BLA was granted Priority Review
designation and the Prescription Drug User Fee Act action date is
February 17, 2023. The Company has filed the Marketing
Authorization (MA) application with the European Medical Agency
(EMA) and is currently working closely with the EMA through the MA
validation process.
About Dystrophic Epidermolysis Bullosa
(DEB)DEB is a rare and severe disease that affects the
skin and mucosal tissues. It is caused by one or more mutations in
a gene called COL7A1, which is responsible for the production
of the protein type VII collagen (COL7) that forms anchoring
fibrils that bind the dermis (inner layer of the skin) to the
epidermis (outer layer of the skin). The lack of functional
anchoring fibrils in DEB patients leads to extremely fragile skin
that blisters and tears from minor friction or trauma. DEB patients
suffer from open wounds, which leads to skin infections, fibrosis
which can cause fusion of fingers and toes, and ultimately an
increased risk of developing an aggressive form of squamous cell
carcinoma which, in severe cases, can be fatal.
About B-VECB-VEC is an
investigational non-invasive, topical, redosable gene therapy
designed to deliver two copies of the COL7A1 gene when applied
directly to DEB wounds. B-VEC was designed to treat DEB at the
molecular level by providing the patient’s skin cells the template
to make normal COL7 protein, thereby addressing the fundamental
disease-causing mechanism.
The FDA and EMA have each granted B-VEC orphan drug
designation for the treatment of DEB, and the FDA has granted B-VEC
fast track designation and rare pediatric designation for the
treatment of DEB. In addition, the FDA granted Regenerative
Medicine Advanced Therapy (RMAT) to B-VEC for the treatment of DEB
and the EMA granted PRIority MEdicines (PRIME) eligibility for
B-VEC to treat DEB.
About Krystal Biotech, Inc.Krystal
Biotech, Inc. (NASDAQ: KRYS) is a biotechnology company focused on
developing and commercializing genetic medicines for patients with
rare diseases. The Company’s wide-ranging pipeline is based on its
proprietary redosable HSV vector. Headquartered in Pittsburgh,
Pennsylvania, the Company is led by an experienced management team,
is fully-integrated and has core capabilities in viral vector
design, vector optimization, gene therapy manufacturing and
commercialization. For more information, please visit
http://www.krystalbio.com, and follow @KrystalBiotech
on LinkedIn and Twitter.
Forward-Looking StatementsAny
statements in this press release about future expectations, plans
and prospects for the Company, including statements about the
clinical utility of B-VEC and other statements containing the words
“anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,”
“plan,” “predict,” “project,” “target,” “potential,” “likely,”
“will,” “would,” “could,” “should,” “continue,” and similar
expressions, constitute forward-looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including: uncertainties associated with regulatory review
of clinical trials and applications for marketing approvals, the
availability or commercial potential of B-VEC, the sufficiency of
cash resources and need for additional financing and such other
important factors as are set forth under the caption “Risk Factors”
in the Company’s annual and quarterly reports on file with the U.S.
Securities and Exchange Commission. In addition, the
forward-looking statements included in this press release represent
the Company’s views as of the date of this release. The Company
anticipates that subsequent events and developments will cause its
views to change. However, while the Company may elect to update
these forward-looking statements at some point in the future, it
specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing the Company’s views as of any date subsequent to the
date of this press release.
CONTACT:
Investors and MediaMeg
DodgeKrystal Biotechmdodge@krystalbio.comSource: Krystal Biotech,
Inc.
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