N4 Pharma PLC - N4 101 In Vivo Study Results
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Released
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RNS Number : 1308I
N4 Pharma PLC
12 May 2025
12 May 2025
N4 Pharma plc
("N4 Pharma" or the "Company")
N4 101 In Vivo Study Results
Positive results from first in vivo study of dual-loaded Nuvec® for IBD
N4 Pharma plc (AIM: N4P), the UK biotech developing Nuvec®, its proprietary gene delivery
system to enable advanced therapies for cancer and other diseases, is pleased to announce
positive results from its first in vivo study evaluating the use of its Nuvec® proof-of-concept
programme, N4 101, an orally delivered anti-inflammatory treatment for Inflammatory Bowel
Disease (IBD).
Following the successful completion of in vitro profiling in December 2024, N4 Pharma has now
completed its first in vivo study using an industry-standard mouse model of IBD. The study
explored the therapeutic potential of orally administeredNuvec® particles loaded with siRNA
alone and combined with mRNA.
Over a nine-day dosing period, mice with chemically induced acute IBD received single (siRNA) or
dual (siRNA and mRNA)Nuvec®-loaded formulations, and versions of these therapies specifically
targeted at cells involved in gut inflammation using modified Nuvec® particles. Samples were
collected for analysis on day 15.
Key highlights from the study include:
•
•
•
•
Reduction in inflammation: both single anddual-loadedNuvec® particles
demonstrated marked improvements compared with controls across all key
indicators of colitis, including Disease Activity Index (DAI), colon length, and body
weight loss.
Marked increase in efficacy achieved with targeting:both the single and dual
loaded Nuvec® combined with a targeting agent performed better and showed an
even greater reduction in the inflammatory marker TNF alpha than the untargeted
therapies.
Sustained therapeutic effect: six days after the final administration, both single
and dual-loaded targeted Nuvec® particles showed a near complete reduction in
TNF alpha levels in intestinal tissues.
Effective oral delivery:the study also provides clear in vivo evidence that Nuvec®
particles successfully deliver therapeutic nucleic acid cargos (siRNA and mRNA) to
the gut via oral administration, resulting in the sustained anti-inflammatory effects
observed.
Nigel Theobald, Chief Executive Officer of N4 Pharma, commented:
"We are delighted with these very promisingin vivodata, which demonstrate a sustained antiinflammatory effect in a validated IBD model - a major milestone for our N4 101
programme.Demonstrating a clear increase in performance through targeting the treatment to
specific cells is the holy grail of nucleic acid therapeutics, and these results place Nuvec® at the
leading edge of nucleic acid delivery technologies.
"The study also provides good evidence that Nuvec® can successfully deliver nucleic acid
therapies to the gut via oral administration, opening up exciting new possibilities for orally
delivered therapies for gastrointestinal diseases such as IBD and some cancers.
"This work is at an early stage, butas we move into optimisation studies, these findings will
continue to add to our data set along with the extensive studies supported by the recent
fundraise, to validate Nuvec® as a novel delivery platform for advanced therapies, strengthening
our discussions with commercial partners.
"We look forward to advancing this programme into the next phase of development and
providing further updates on thisand our broader platform work plans for the coming months in
due course."
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Ends -
For more information, please contact:
N4 Pharma plc
Nigel Theobald, CEO
Luke Cairns, Executive Director
Via N4 Pharma Investor
Hub:investors.n4pharma.com
Submit your questions directly to the
management team via the N4 Pharma
Investor Hub
https://investors.n4pharma.com/link/8r6zor
SP Angel CorporateFinance LLP
Nominated Adviser and Joint Broker
Matthew Johnson/Jen Clarke (Corporate
Finance)
Vadim Alexandre/Abigail Wayne/Rob
Rees (Corporate Broking)
Tel: +44 (0)20 3470 0470
Turner Pope Investments (TPI)
Limited
Joint Broker
Andy Thacker
James Pope
Tel: +44 (0)20 3657 0050
Northstar Communications Limited
Investor Relations
Sarah Hollins
Tel: +44 (0)113 730 3896
About N4 Pharma
N4 Pharma is a pre-clinical biotech company developing Nuvec®,its proprietary gene delivery
system, to enable advanced therapies for cancer and other diseases.
RNA therapeutics are set to impact the treatment of a wide range of diseases and Nuvec® has
several key advantages for RNA gene delivery including the ability to deliver multiple RNA
therapies in a single particle, ease of manufacturing,protection of the RNA payload to allow for
oral delivery, no unwanted immune response and excellent stability and storage.
N4 Pharma is building out its preclinical data set and working towards first-in-human clinical data
to support significant licensing deals for its Nuvec® platform with gene therapy partners.
N4 Pharma's lead programme, N4 101, is an oral anti-inflammatory product for IBD which serves
as a proof-of-concept programme showcasing all the benefits of the Nuvec® platform.
For
further
information
on
the
Company
visitwww.n4pharma.comor
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athttps://investors.n4pharma.com/auth/signup.
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