4DMT Receives Rare Pediatric Disease Designation from FDA for Aerosolized 4D-710 for Treatment of Cystic Fibrosis Lung Disease
January 23 2024 - 8:00AM
4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company),
a leading clinical-stage genetic medicines company focused on
unlocking the full potential of genetic medicines to treat large
market diseases, today announced that the U.S. Food and Drug
Administration (FDA) has granted Rare Pediatric Disease Designation
(RPDD) for the Company’s product candidate aerosolized 4D-710 for
treatment of cystic fibrosis (CF) lung disease. 4D-710 is being
evaluated in the Phase 1/2 AEROW clinical trial in people with CF
who are not eligible for, or cannot tolerate any of, the currently
approved CFTR modulators.
RPDD may be granted to investigational drugs and biologics
designed to address serious or life-threatening diseases primarily
affecting individuals 18 years of age and under, and meeting the
definition of “rare disease or condition” (affects less than
200,000 individuals in the U.S.). If the drug or biologic is
approved for marketing, 4DMT may qualify for a priority review
voucher that may be transferred or sold to another sponsor.
“The Rare Pediatric Disease Designation is a very important
regulatory milestone in our development path for 4D-710 and
highlights the urgent need for novel therapeutic options for people
living with CF lung disease, including children, especially those
who are not eligible for currently available disease modifying
therapies,” said David Kirn, M.D., Co-founder and Chief Executive
Officer of 4DMT. “Based on the CFTR expression levels and clinical
activity seen to-date, we are excited about the potential for
4D-710 to transform the lives of people with CF with an effective
and durable treatment option. We continue to enroll our AEROW
clinical trial and work with the CF Foundation and regulators to
identify the most efficient path to advance this therapy, with
preliminary feedback expected this quarter.”
About Cystic Fibrosis Lung Disease and
4D-710
Cystic fibrosis is an inherited, progressive disease caused by
mutations in the CFTR gene. It affects the lungs, pancreas, and
other organs. According to the CF Foundation, nearly 40,000 people
in the United States and more than 105,000 people worldwide are
living with cystic fibrosis, with approximately 1,000 new cases of
cystic fibrosis diagnosed in the United States each year. Lung
disease is the leading cause of morbidity and mortality in people
with cystic fibrosis. Cystic fibrosis causes impaired lung
function, inflammation and bronchiectasis and is commonly
associated with persistent lung infections and repeated
exacerbations due to the inability to clear thickened mucus from
the lungs. People with cystic fibrosis require lifelong treatment
with multiple daily medications. The complications of the disease
result in progressive loss of lung function, increasing need for IV
antibiotics and hospitalizations, ultimately leading to end-stage
respiratory failure.
4D-710 is comprised of our targeted and evolved next generation
vector, A101, and a codon-optimized CFTR∆R transgene. 4D-710 has
the potential to treat a broad range of people with cystic
fibrosis, independent of the specific CFTR mutation, and is
designed for aerosol delivery to achieve CFTR expression within
lung airway epithelial cells. 4D-710 is being initially developed
for the approximately 15% of people whose disease is not amenable
to existing CFTR modulator medicines (based on variant-eligibility
and/or drug intolerance) targeting the CFTR protein. In people with
CFTR mutations whose disease is amenable to modulator medicines,
the improvement in lung function is incomplete and is variable. We
therefore expect to potentially develop 4D-710 in this broader
population, as a single agent and/or in combination with CFTR
modulator small molecule medicines.
About 4DMT
4DMT is a leading clinical-stage genetic medicines company
focused on unlocking the full potential of genetic medicines to
treat large market diseases in ophthalmology and pulmonology.
4DMT’s proprietary invention platform, Therapeutic Vector
Evolution, combines the power of the Nobel Prize-winning
technology, directed evolution, with approximately one billion
synthetic AAV capsid-derived sequences to invent customized and
evolved vectors for use in our wholly owned and partnered product
candidates. Our product design, development, and manufacturing
engine helps us efficiently create and advance our diverse product
pipeline with the goal of revolutionizing medicine with potential
curative therapies for millions of patients. Currently, 4DMT is
advancing five clinical-stage and two preclinical product
candidates, each tailored to address rare and large market diseases
in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is
also advancing programs in CNS through a gene editing partnership.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™,
and the 4DMT logo are trademarks of 4DMT.
All of our product candidates are in clinical or preclinical
development and have not yet been approved for marketing by the FDA
or any other regulatory authority. No representation is made as to
the safety or effectiveness of our product candidates for the
therapeutic uses for which they are being studied.
Learn more at www.4DMT.com and follow us on LinkedIn.
Forward Looking Statements:
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, implied and
express statements regarding the therapeutic potential, and
clinical benefits of 4DMT’s product candidates, as well as the
plans, announcements and related timing for the clinical
development of 4D-710. The words "may," “might,” "will," "could,"
"would," "should," "expect," "plan," "anticipate," "intend,"
"believe," “expect,” "estimate," “seek,” "predict," “future,”
"project," "potential," "continue," "target" and similar words or
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Any forward looking statements in this press
release are based on management's current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including risks and
uncertainties that are described in greater detail in the section
entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent
Quarterly Report on Form 10-Q as well as any subsequent filings
with the Securities and Exchange Commission. In addition, any
forward-looking statements represent 4D Molecular Therapeutics'
views only as of today and should not be relied upon as
representing its views as of any subsequent date. 4D Molecular
Therapeutics explicitly disclaims any obligation to update any
forward-looking statements. No representations or warranties
(expressed or implied) are made about the accuracy of any such
forward looking statements.
Contacts:
Media:
Katherine SmithInizio Evoke
CommsKatherine.Smith@inizioevoke.com
Investors:
Julian PeiHead of Investor Relations and Corporate
CommunicationsInvestor.Relations@4DMT.com267-644-5097
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