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 the Company presented new data on the compassionate use of
topical beremagene geperpavec (B-VEC) to treat a patient with
dystrophic epidermolysis bullosa (DEB) with recurrent cicatrizing
conjunctivitis at the Association for Research in Vision and
Ophthalmology (ARVO) 2023 Annual Meeting on April 23, 2023.
“DEB is a devastating disease with limited treatment options,
and there is a substantial population of DEB patients with ocular
complications for which treatment options are limited and often
include surgery,” said Alfonso L. Sabater, M.D., PhD, Assistant
Professor of Clinical Ophthalmology at the Bascom Palmer Eye
Institute at the University of Miami Miller School of Medicine. “It
is exciting to potentially advance a topical treatment for patients
with ocular complications associated with DEB.”
The data presented describes the first application of B-VEC to
treat ocular complications in a patient with DEB under a
compassionate use program. The patient presented with cicatrizing
conjunctivitis and underwent surgical symblepharon lysis with
pannus removal in the right eye. B-VEC was administered to the
patient’s right eye at regular intervals following surgery in
addition to routine post-surgical management.
B-VEC was well tolerated and associated with full corneal
healing by 3 months as well as significant visual acuity
improvement from hand motion to 20/40 at 7 months, the latest time
point of the on-going treatment effect evaluation.
A
B
Figure 1: Slit lamp pictures of the right eye.
A: Baseline ankyloblepharon. The visual acuity was
hand motion (HM) B: Ocular surface of the right
eye 6 months after the surgery after B-VEC applications.
No drug-related adverse events (AE) have been observed. Two non
drug related, serious AEs were reported: 1) Prolonged
hospitalization due to complications post-gastrointestinal surgery,
and 2) Prolonged hospitalization due to complications
post-esophageal dilation. B-VEC treatment was not interrupted
during either event.
Ocular complications are common in patients with DEB, with over
half of the patients diagnosed with recessive DEB potentially
affected. Typical ocular manifestations include corneal abrasion,
as well as corneal scarring, pannus, eyelid ectropions and
blisters.1,2 There are no specific FDA-approved treatment options
for ocular manifestations of DEB.3
“Ocular complications impose a heavy burden on DEB patients.
Based on this promising initial data, we plan to engage with
regulatory authorities and explore how we can expand the utility of
B-VEC to address this urgent need,” said Suma Krishnan, President,
Research & Development, Krystal Biotech. “We are also excited
about the implications for our platform as this clinical data,
together with ongoing preclinical studies evaluating intravitreal
and subretinal routes of delivery to the eye, suggests significant
potential to treat multiple ocular diseases with few or no
treatment options.”
The poster was available to conference attendees and is
available on the Investor section of the Company’s website.
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 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 Krystal Biotech, Inc., including statements about our
plans to engage with regulatory authorities to explore how we can
expand the utility of B-VEC; the significant potential of our
platform to treat multiple ocular diseases with few or no treatment
options; 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 release.
CONTACTInvestors and
Media:
Meg Dodge
Krystal Biotech
mdodge@krystalbio.com
1. Tang JY, Marinkovich MP, Lucas E, et al. A systematic
literature review of the disease burden in patients with recessive
dystrophic epidermolysis bullosa. Orphanet J Rare Dis. 2021
Apr 13; 16(1): 175. doi: 10.1186/s13023-021-01811-7.
2. Tong L, Hodgkins PR, Denyer J, et al. The eye in
epidermolysis bullosa. Br J Ophthalmol. 1999 Mar; 83(3):
323-6. doi:10.1136/bjo.83.3.323.
3. Chen VM, Mehta N, Robbins CC, et al. Anterior-segment
spectral domain optical coherence tomography in epidermolysis
bullosa. Ocul Surf. 2020 Oct; 18(4): 912-919. doi:
10.1016/j.jtos.2020.08.010
Photos accompanying this announcement are available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/dd1859a6-21d6-4bee-84d1-1cd6b7052ea5
https://www.globenewswire.com/NewsRoom/AttachmentNg/0ef5004a-2f1b-4cc7-98ec-d8fda41d606c
Krystal Biotech (NASDAQ:KRYS)
Historical Stock Chart
From Sep 2024 to Oct 2024
Krystal Biotech (NASDAQ:KRYS)
Historical Stock Chart
From Oct 2023 to Oct 2024