First gene therapy for prevalent pathologies in neurology receives FDA IND clearance - EG 427 initiating first-in-human clinical study
June 24 2024 - 2:00AM
- EG110A to start
Phase 1b/2a clinical study in Neurogenic Detrusor Overactivity
(NDO) patients
- NDO is a severe
bladder condition affecting millions of patients living with Spinal
Cord Injury, Multiple Sclerosis, Parkinson’s disease and other
neurological disorders
- Other severe
pathologies of the bladder to be investigated following this
study
- Preclinical data
show EG110A’s potential for long-lasting efficacy on urinary
continence and to address shortcomings of existing therapies
- Opens pathway for
advancing localized gene therapy in large neurology disease
populations, based on the company’s proprietary vector
platform
Paris, France, June 24, 2024 –
EG 427, a biotechnology company leading the development of pinpoint
DNA medicines for prevalent diseases in neurology based on its
unique non-replicative HSV-1 vector platform, announces today that
it has received Investigational New Drug (IND) clearance from the
U.S. Food and Drug Administration (FDA) for EG110A, a gene therapy
for the treatment of Neurogenic Detrusor Overactivity (NDO) in
Spinal Cord Injury (SCI) patients. The phase 1b/2a study is being
initiated in 2 leading US institutions.
EG110A is a non-replicating HSV-1 vector that
has been designed to selectively silence the signals of key bladder
sensory neurons responsible for the bladder muscle overactivity,
whilst preserving motor neuron and retaining normal bladder
function.
NDO is a common urinary bladder dysfunction
caused mainly by SCI and other neurodegenerative diseases, such as
Multiple Sclerosis or Parkinson’s disease. NDO causes uncontrolled
urinary incontinence, risk of kidney damage as well as urinary
tract infections than can lead to death in 5-10% of the SCI
population. NDO affects most (70-84%) patients with SCI1, an
estimated total of 300,000-400,000 worldwide. It has a significant
impact on their quality of life. The European Association of
Urology recently estimated that incontinence caused by NDO and
other indications, such as overactive bladder, represents a growing
economic burden of over €40 billion per year in Europe2.
“NDO is a serious bladder condition, for whom
treatment options are limited,” said Cornelia Haag-Molkenteller,
MD, PhD, Chief Medical Officer of EG 427. “Preclinical results have
shown clear evidence that suggests EG110A could offer significant
medical improvement over existing therapies. These data have shown
that one course of treatment with EG110A can potentially lead to
long-lasting efficacy without affecting the bladder function. We
are excited to move this promising product into the clinic.”
“This IND clearance for our first product is a
major milestone for EG 427, opening the way for clinical
development of EG110A across a series of medically important but
neglected neuro-urology pathologies. It is a step forward in
bringing the potential benefits of gene therapy to large disease
populations in neurology. Non-replicative HSV-1 vectors are truly
unique in their ability to deliver therapeutic solutions in a
potentially safe, redosable and cost-effective way,” said Philippe
Chambon, MD, PhD, Chief Executive Officer at EG 427.
Neurological diseases affect some 3 billion
people, or about 1 in 3 worldwide, and their medical needs are
underserved, a study by The Lancet Neurology3 found. EG 427 is
building a pipeline of products to address these diseases, based on
its proprietary non-replicative HSV vector platform, uniquely
suited to target neural cells in a safe and long-lasting
manner.
About EG 427EG
427 is the second company to bring a non-replicating HSV-1
(nrHSV-1) vector into clinical development, with an Investigational
New Drug (IND) cleared by the US Food and Drug Administration (FDA)
in June 2024. It will be the first human trial of such a vector,
targeting sensory neuron-based diseases. The product, EG110A,
addresses multiple severe bladder diseases, such as neurogenic
bladder (NDO) and overactive bladder (OAB), and has the potential
to be a major improvement over existing therapies, resulting in
better care for patients and lower costs for healthcare
systems.
The company’s unique platform delivers pinpoint
neurotherapeutics to treat prevalent diseases of the peripheral and
central nervous system. Its vectors can achieve focal transduction
in specific regions and then selective expression of transgenes in
targeted subsets of neurons thanks to the control of sophisticated
regulatory elements. With demonstrated clinical safety and possible
repeated dosing, the large payload capacity of nrHSV-1 vectors
allows either for long-term gene therapy, or all-in-one gene
editing approaches.
For more information: check our website at
www.eg427.com
follows us on
at www.linkedin.com/company/eg427/
Contacts:
EG427Philippe Chambon, M.D., Ph.D.Founder and
CEOpchambon@eg427.com |
|
Global Media Relations
Sophie
BaumontCohesion Bureausophie.baumont@cohesionbureau.com+33 6 27 74
74 49 |
1 Hamid, R., Averbeck, M.A., Chiang, H. et al. Epidemiology and
pathophysiology of neurogenic bladder after spinal cord injury.
World J Urol 36, 1517–1527 (2018).
https://doi.org/10.1007/s00345-018-2301-z2
https://uroweb.org/press-releases/incontinence-costs-european-society-over-40-billion-euros-per-year3
Global, regional, and national burden of disorders affecting the
nervous system, 1990–2021: a systematic analysis for the Global
Burden of Disease Study 2021.
https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(24)00038-3/fulltext#seccestitle210
- 20240624_EG 427_PR_IND_EN