Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a
biotechnology company focused on developing and commercializing
genetic medicines for patients with debilitating diseases,
announced today that the first patient has been dosed at the Cystic
Fibrosis Institute of Chicago in the Company’s Phase 1 CORAL-1/US
study evaluating KB407, an engineered HSV-1-based,
aerosol-delivered, mutation agnostic, genetic medicine for the
treatment of patients with cystic fibrosis (CF).
“By delivering full-length copies of CFTR directly to the lung
via nebulization, KB407 has the potential to address the basic
genetic defect present in cystic fibrosis,” said Steven R. Boas,
M.D., Director of the Cystic Fibrosis Institute and Professor of
Pediatrics at Northwestern University Feinberg School of
Medicine. “As this therapy does not depend on the type of CFTR
mutation present, KB407 may potentially benefit all individuals
affected by cystic fibrosis.”
“Dosing our first patient in the KB407 Phase 1 clinical trial is
an exciting step forward for the company and for the patients we
aim to benefit,” said Hubert Chen, M.D., Senior Vice President of
Clinical Development at Krystal Biotech. “Not only is the Phase 1
study designed to provide key insights into the safety and efficacy
of KB407, it is also a critical step in expanding our vector
platform to tissues beyond the skin. We look forward to continued
enrollment into the Phase 1 study with anticipated data in
2024.”
CORAL-1/US Study Overview
The CORAL-1/US study is a multicenter, dose-escalation trial of
KB407 in patients (n~20) with CF regardless of their underlying
genotype. Each administration of KB407 will be nebulized in under
30 minutes. This study will include three cohorts and enroll five
subjects each in the first two cohorts and ten subjects in the last
cohort.
Cohort 1 participants will receive a single administration of
KB407 on Day 0, and Cohort 2 participants will receive
administrations of KB407 on Day 0 and Day 1. Cohorts 1 and 2 will
be conducted on an open-label basis and up to 3 subjects in each of
the first two cohorts may receive concurrent CFTR modulator
therapy. Cohort 3 participants will be randomized 4:1 to receive
administrations of KB407 or placebo on Day 0, Day 1, Day 2, and Day
3. Cohort 3 will follow a randomized, placebo-controlled, and
double-blind design and will enroll only subjects who are not
otherwise eligible for CFTR modulators. A Data Monitoring Committee
will conduct a safety review after each cohort before proceeding to
the next one.
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 the percent predicted forced
expiratory volume in one second (ppFEV1). Vector shedding and
biodistribution will also be assessed in blood, urine, buccal, and
sputum samples. The CORAL-1/US study also includes a bronchoscopy
sub-study for assessment of CFTR transgene expression in the
airways at both the nucleic acid and protein levels. At select
sites, subjects may undergo an optional bronchoscopy 24 to 96 hours
after the last dose of KB407. The bronchoscopy will include
bronchial brushings and endobronchial biopsies.
Details about the Phase 1 study can be found at
www.clinicaltrials.gov under NCT identifier NCT05504837.
About KB407KB407 is an investigational,
redosable gene therapy designed to molecularly correct the
underlying cause of CF by delivering two copies of
the CFTR gene directly to the airways via nebulization.
By enabling the expression of full-length, healthy CFTR protein in
the lung, treatment with KB407 has the potential to restore proper
anion transport within transduced cells to correct the lung
manifestations of the disease. Because KB407 is engineered to
enable expression of wild-type CFTR protein, treatment is agnostic
to patients’ underlying CFTR mutations. The US Food and Drug
Administration and the European Commission have granted orphan drug
designation for KB407 to treat patients with CF.
About Cystic Fibrosis
CF is a genetic disease caused by mutations in the CFTR gene
which result in dysfunctional or absent CFTR protein, and buildup
of mucus in the lungs that leads to persistent lung infections and
progressive pulmonary disease. The Cystic Fibrosis Foundation
estimates that there are close to 40,000 children and adults living
with CF in the US, and an estimated 105,000 people diagnosed with
CF across 94 countries. People of every racial and ethnic group are
affected by this debilitating disease.
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 approved 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 commercial-stage fully integrated
company focused on developing genetic medicines for patients with
debilitating diseases. VYJUVEKTM is the Company’s first
commercial product, the first-ever redosable gene therapy, and the
only medicine approved by the FDA for the treatment of dystrophic
epidermolysis bullosa. The Company presently has three candidates
in clinical development and a wide-ranging pipeline that is powered
by its proprietary, redosable HSV-1 gene delivery platform.
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, the expansion of the Company’s platform to
tissues beyond the skin, the potential of KB407 to treat all
patients with cystic fibrosis, the timing of reporting results from
the Phase I clinical trial of KB407, 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 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.
CONTACTInvestors and Media:Meg
Dodge
Krystal
Biotech
mdodge@krystalbio.com
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