N4 Pharma announces success in positive in vivo results from first Nuvec study for IBD dual-loaded with siRNA and mRNA

N4 Pharma Plc has successfully completed the first in vivo study of orally delivered Nuvec^® using its lead programme N4 101, an orally administered anti-inflammatory treatment for inflammatory bowel disease (IBD). In the study, both single (siRNA) and dual-loaded (siRNA and mRNA) Nuvec^® formulations were tested to explore whether the combination enhances overall therapeutic effect, and both demonstrated marked improvements of the key signs of IBD, including reduced gut inflammation, less weight loss and healthier colon tissue.

IBD affects millions of people globally. Most current treatments rely on immune suppression and injections, often with limited long-term efficacy. The ability to deliver gene therapies orally, precisely to the gut, opens a new avenue for treating not only IBD, but a wide range of gastrointestinal and systemic diseases including cancer.

Key findings from the study included:

• Reduced inflammation: both single and dual-loaded Nuvec^® particles demonstrated marked improvements - compared with controls - across all key indicators of colitis, including Disease Activity Index (DAI), colon length, and body weight loss.
• Increased efficacy 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 promising in vivo data. 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. While early-stage, these findings significantly strengthen our dataset as we move into optimisation studies and continue broader development efforts across our platform. With the support of our recent fundraise, we look forward to progressing discussions with potential commercial partners and updating the market as we advance this exciting programme.”

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