Krystal Biotech Submits Biologics License Application to U.S. FDA Seeking Approval of B-VEC for the Treatment of Patients with Dystrophic Epidermolysis Bullosa
22 June 2022 - 9:00PM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in
redosable gene therapy, announced today the submission of a
Biologics License Application (BLA) to the U.S. Food and Drug
Administration (FDA) seeking approval of B-VEC (beremagene
geperpavec) for the treatment of patients with dystrophic
epidermolysis bullosa (DEB). B-VEC is an investigational
non-invasive, topical gene therapy designed to treat DEB at the
molecular level by providing the patient’s skin cells with two
copies of the COL7A1 gene to make functional COL7 protein, thereby
addressing the fundamental disease-causing mechanism.
“The unmet medical need for DEB patients remains very high and
our relentless pursuit of a treatment for this disease continues
with the same sense of urgency that we have always had since the
founding of Krystal Biotech,” said Suma Krishnan, President of
Research & Development. “We look forward to working with the
FDA in its review of our BLA submission.”
The BLA submission for B-VEC is supported by data from two
placebo controlled clinical trials - the GEM-3 trial (NCT04491604)
and the GEM-1/2 trial (NCT03536143).
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.
The GEM-1/2 trial was a randomized, open-label, intra-patient
placebo-controlled single-center trial designed to evaluate
efficacy (mechanistic and clinical) and safety of B-VEC for the
treatment of DEB. In the trial, matched wounds receiving topical
B-VEC or placebo were evaluated in nine recessive dystrophic
epidermolysis bullosa patients over 12 weeks. Both mechanistic and
clinical endpoints were met. No serious or severe B-VEC-related
adverse events or systemic drug exposure were noted. Results from
the GEM-1/2 trial of B-VEC for the treatment of DEB were published
in Nature Medicine. The publication provides a
comprehensive analysis of the data from the GEM-1/2 trial showing
that repeat topical applications of B-VEC were well tolerated and
associated with durable wound closure, full-length cutaneous type
VII collagen (COL7) expression, and anchoring fibril assembly with
minimal reported adverse events.
In addition to submitting the BLA to the FDA, the Company has
continued to engage in dialog with regulatory authorities in other
markets, including Europe and Japan. The Company anticipates
submission of a marketing authorization application with the
European Medical Agency (EMA) in 2H 2022.
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-VEC
B-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 functional 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 pivotal-stage gene
therapy company leveraging its proprietary, redosable gene therapy
platform and in-house manufacturing capabilities to develop
life-changing medicines for patients with serious diseases,
including rare diseases in skin, lung, and other areas. For more
information please visit http://www.krystalbio.com, and follow
@KrystalBiotech on LinkedIn and Twitter.
Forward-Looking Statements
Any statements in this press release about future expectations,
plans and prospects for the Company, including statements about the
timing of the submission of the Company’s EMA marketing
authorization application, 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 product candidates
including 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.
CONTACTS:
Investors and Media:
Meg DodgeKrystal Biotechmdodge@krystalbio.com
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