Krystal Biotech Announces FDA Acceptance of KB407 IND Application for Cystic Fibrosis Clinical Trial
August 01 2022 - 5:39PM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in
redosable gene therapy, today announced that the United States Food
and Drug Administration (FDA) has accepted its Investigational New
Drug (IND) application to evaluate KB407 in a clinical trial for
cystic fibrosis (CF).
KB407 is a modified HSV-1 vector carrying two copies of the
cystic fibrosis transmembrane conductance regulator (CFTR) gene to
the respiratory cells in the lungs. By inducing expression of full
length, normal CFTR protein in the lung, treatment with KB407 has
potential to restore ion and water flow into and out of lung cells
to correct the lung manifestations of the disease in patients
regardless of their underlying genetic mutation.
CF is a genetic disease, affecting people of every racial and
ethnic group, by causing a buildup of mucus in the lungs leading to
persistent lung infections and progressive pulmonary disease.
Sources including the Cystic Fibrosis Foundation indicate that
there are close to 40,000 children and adults living with cystic
fibrosis in the US, and an estimated 105,000 people diagnosed with
CF across 94 countries.
“We are excited to advance KB407, our investigational gene
therapy for patients with CF, into the clinic. It is designed to
treat the root cause of the disease regardless of an individual
patient’s mutation by giving the body instructions to produce its
own functional protein,” said Hubert Chen, M.D., Senior Vice
President of Clinical Development at Krystal Biotech. Dr. Chen
continued, “In addition, this IND acceptance represents an
important milestone for us as it allows us to demonstrate the power
of our platform to deliver genes, beyond skin cells, to respiratory
cells.”
On July 1, 2022, the Company submitted an IND application to
initiate the Phase 1 clinical trial of KB407. At the end of the
30-day review period, the Company received notification that the
FDA accepted the IND allowing the Phase 1 clinical trial to begin.
The Company anticipates initiating the clinical trial in 2H
2022.
Phase 1 Trial OverviewThe Phase 1 clinical
trial will utilize nebulized administration to deliver KB407 in up
to 20 adults with CF. The study will enroll 3 cohorts sequentially
to evaluate ascending doses of KB407. The primary endpoint of the
trial will be the safety and tolerability of nebulized KB407.
Changes in lung function from baseline will be assessed by forced
expiratory volume in one second.
About KB407KB407 is an investigational,
redosable gene therapy designed to correct the underlying cause of
CF by delivering two copies of the CFTR gene directly to the airway
epithelial cells when delivered via a nebulizer. By inducing
expression of full length, normal CFTR protein in the lung,
treatment with KB407 has potential to restore ion and water flow
into and out of lung cells to correct the lung manifestations of
the disease in patients regardless of their underlying genetic
mutation.
About Cystic FibrosisCystic fibrosis is caused
by genetic mutations that result in dysfunctional or absent CFTR
protein and is the most common fatal inherited disease in the
United States. Lack of functional CFTR in secretory airway
epithelial cells causes dehydrated mucus buildup in the lungs,
pancreas, and other organs. This mucus buildup in the lungs leads
to loss of lung function, and eventually, respiratory failure.
According to the US Cystic Fibrosis Foundation, the median age
at death for patients with CF in the United States was
34.1 years in 2020.
Although CFTR modulators are effective in patients with certain
CFTR mutations, patients may still experience pulmonary symptoms
requiring treatment. Importantly, approximately 10-15% of CF
patients harbor genetic mutations that are not expected to be
responsive to current therapies and currently have no available
disease-modifying treatment options, representing a significant
unmet need.
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 Krystal Biotech, Inc., including statements about the clinical
utility of KB407 and the expected timing of the KB407 Phase 1
clinical trial program in the U.S., 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: the availability or commercial potential of
product candidates including KB407, 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.
CONTACTS:Investors and
Media:Meg DodgeKrystal Biotechmdodge@krystalbio.comSource:
Krystal Biotech, Inc.
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