Enterprise Therapeutics Doses First Person with Cystic Fibrosis in Phase 2 Trial for Novel Therapy ETD001
23 Julio 2024 - 4:57AM
Business Wire
- Trial aims to deliver clinical proof-of-concept and assess the
safety profile of novel long acting ENaC inhibitor ETD001
- ETD001, a CFTR mutation agnostic approach to CF, is focussed on
providing a novel treatment for members of the CF community that do
not benefit from currently available mutation-targeted
therapies
Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical
company dedicated to the discovery and development of novel
therapies to improve the lives of those suffering from respiratory
disease, today announced dosing of the first person with cystic
fibrosis (pwCF) in its Phase 2a trial of ETD001.
ETD001, a low molecular weight compound with first-in-class
potential, targets the epithelial sodium channel (ENaC) in the
airway epithelium to increase the hydration and clearance of mucus.
The Phase 2a trial aims to deliver clinical proof-of-concept and to
assess the safety profile of ETD001 in the 10% of pwCF with the
highest unmet medical need. The study will be performed at sites
located in the UK, Germany, France and Italy and will assess lung
function (FEV1) in pwCF who are either ineligible for or are not
receiving CFTR modulators.
CF is estimated to affect over 100,000 people worldwide, with an
average life expectancy of only 50 years. Failed mucociliary
clearance and mucus congestion in the lungs of pwCF leads to cycles
of infection and inflammation and an ongoing decline in lung
function. Increasing fluid volume in the lung by inhibiting ENaC
with ETD001 will hydrate mucus, improve clearance, reduce mucus
congestion, and is expected to drive substantial improvements in
lung function. ETD001 has previously demonstrated a strong safety
profile in healthy participants in a Phase 1 trial and has been
shown to be long-acting in pre-clinical studies.
Dr John Ford, CEO, Enterprise Therapeutics, said: “The
dosing of the first person with CF in our Phase 2a trial of ETD001
represents an incredible milestone, testament to Enterprise’s
dedication to advancing a novel approach to treating pwCF with the
highest unmet medical need. ETD001 has already demonstrated an
excellent safety profile in healthy participants, as well as a
pharmacokinetic (PK) profile consistent with a long lung residency.
We look forward to progressing ETD001 through Phase 2 trials and
beyond.”
Paul Russell, Head of Development, Enterprise Therapeutics,
commented: “By targeting the underlying mechanisms of mucus
congestion in the lungs through ENaC inhibition, ETD001 has the
potential to be a transformative respiratory therapeutic. This is
not only for the ~10% of pwCF who are not genetically suited to, or
do not benefit from CFTR modulators, but also those suffering from
other muco-obstructive lung diseases such as COPD and asthma. The
commencement of our Phase 2a trial brings us an exciting step
closer to realising that potential.”
Dr Renu Gupta, CMO, Enterprise Therapeutics, said: “We
are grateful to the pwCF participating in our Phase 2 study of
ETD001, and to the clinical investigators for achieving this major
milestone. We are hopeful that our steadfast commitment to
advancing this innovative ENaC targeting molecule, along with our
partnerships with the CF community, will lead to a treatment that
will vastly improve the lives of people living with CF.”
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Katie Odgaard Zyme Communications Tel: +44 (0)7787 502 947
Email: katie.odgaard@zymecommunications.com