ECP105 is a breakthrough siRNA therapy intended for injection into the ocular cavity and contains a proprietary siRNA sequence to silence the fibrotic gene responsible for fibrosis (MRTF-β), or scarring, which occurs post-surgery. Unlike current off-label treatment using toxic chemotherapy drugs like Mitomycin C (MMC), ECP105 is intended for repeat use to prevent the need for further surgery.  

An unmet need

Glaucoma patients who have failed to respond to medication need surgery (trabeculectomy) to lower intraocular pressure. However, this causes fibrosis at the surgery site which often leads to failure of the procedure and patients may have to undergo further surgery as a result. Even with adjunctive anti-fibrotic therapy, five-year failure rates can be as high as 50%. Current treatments to prevent fibrosis, for example MMC, are unlicensed and carry the risk of severe side effects.

Market opportunity

Glaucoma currently affects 80 million people worldwide and this is estimated to rise to nearly 112 million by 2040[i]. 

ECP105 will replace the use of off-label MMC to achieve the same efficacy but without the toxic side effects and can be used again if repeat surgery is needed.

ECP105 proof of concept

Following a single subconjunctival administration of LipTide™, N4 Pharma’s peptide/lipid delivery system, containing an MRTF-β siRNA in an animal model, the results were:

  • MRTF mRNA expression reduced by 30%
  • ICH revealed less scarring
  • Bleb survival increased from 11 days to 22 days
  • A single dose of LipTide™ with 25ug MRTF siRNA had the same effect in this in vivo model of glaucoma fibrosis as the current standard treatment mitomycin C
  • No local or systemic toxicity was observed with LipTide™

[i] https://www.futuremarketinsights.com/reports/glaucoma-treatment-market 

LipTide™

LipTide™ is a patented lipid and peptide-based delivery system for nucleic acids that mimics a natural virus for targeted delivery of RNA into cells. The peptide binds payload and targets specific cells while the lipid allows for efficient endosomal release into the cell. The proprietary delivery technology was developed by Nanogenics which was recently acquired by N4 Pharma.

With appealing clinical properties, the capability to deliver genetic material to almost any cell type, low toxicity, and repeat dosing, LipTide™ represents new promise across several therapeutic areas where there is unmet need.

siRNA therapeutics

As a non-viral, non-lipid delivery system, LipTide™ is supporting the development of a novel siRNA product for the recovery of post-surgical treatment of glaucoma and as a proprietary siRNA sequence for reducing fibrosis.

Proprietary cell targeting platforms available to third parties for own development, for more information contact us here.