N4 Pharma moving novel delivery systems towards commercialization

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N4 Pharma PLC recently acquired a controlling interest in Nanogenics Limited, which has developed a lipid and peptide-based delivery system, LipTide.

N4 Pharma is using LipTide in the formulation of a novel siRNA product – ECP105 – targeting an unmet clinical need in the ophthalmology market.

According to N4 Pharma, alongside its ongoing pre-clinical research into Nuvec - N4 Pharma’s novel silica nanoparticle delivery system for cancer treatments, gene therapy and vaccines - it has recently signed a contract to start the formulation and sequence selection work to prepare ECP105 for testing in pre-clinical studies at Kings College, London.

The company noted in a news release its ECP105 has been developed for the recovery of post-surgical treatment of glaucoma and it contains a proprietary siRNA sequence to silence the fibrotic gene MRTF-B, which is also responsible for fibrosis of the liver and the lung.¹

“Non-viral, non-lipid delivery systems are in high demand in the exciting gene therapy and oncology spaces and we now have two such delivery systems,” Nigel Theobald, CEO of N4 Pharma, said in the company’s news release. “Nuvec continues to show its versatility as a novel delivery system and with LipTide we are developing our first commercial product which we are aiming to take into phase 1 clinical trials.”

Glaucoma patients who have failed to respond to medication need surgery to lower intraocular pressure which causes fibrosis at the surgery site, the company noted in its news release. This fibrosis often leads to failure of the surgery and patients may have to undergo further surgery.

Moreover, the company noted that ECP105 is intended for injection into the ocular cavity to switch off the gene responsible for fibrosis. Unlike current off-label treatment using toxic chemotherapy drugs like Mitomycin C, ECP105 is intended for repeat use to prevent the need for further surgery.

Nanogenics recently finished an initial proof of concept in-vivo study which has demonstrated that a single dose of ECP105 can match the anti-fibrotic effect of Mitomycin C without any cytotoxic side effects.¹ Importantly, this means ECP105 can be dosed repeatedly where necessary. The company will now engage in discussions with the FDA and MHRA to approve a GLP toxicity study and first in human clinical trials.

The company noted that the pre-clinical efforts on Nuvec have demonstrated its potential to enable more effective cancer treatments by reducing the ability for tumor escape. Recent studies have shown that Nuvec can bind not only single, but multiple siRNAs aimed at simultaneously targeting identified pathways responsible for cancer progression after initial treatments. Testing of these with two siRNA (EGFR and PLK1) has shown a positive additive effect after 48 hours of the dual-loaded siRNA compared to the single-loaded siRNA.

N4 Pharma, through its research program with the University of Queensland, continues to research the potential of Nuvec to act as an oral delivery system for oligonucleotides including DNA and RNA. Experiments have now confirmed, in vivo, the successful oral administration of Nuvec loaded with a DNA plasmid for ovalbumin and demonstrated good ovalbumin expression after 2 separate doses.

The company concluded by noting it will continue its pre-clinical research program into Nuvec, with further updates on the key areas of siRNA, oral delivery and AAV viral vectors expected in 2024.

N4 Pharma picked up a controlling interest in Nanogenics in September 2023, adding the siRNA product for the ophthalmology product to its lineup.¹

The company noted in a news release at the time, glaucoma impacted more than 75 million people worldwide in 2020, with a market forth more than $5.5 billion in 2021.

“The RNA sector is an exciting one with a lot of investor and commercial interest,” Theobald said at the time. “The RNA sector is an exciting one with a lot of investor and commercial interest.”

References:

1. N4 Pharma picks up controlling interest in Nanogenics. Sharecast. Accessed February 27, 2024. https://www.sharecast.com/news/news-and-announcements/n4-pharma-picks-up-controlling-interest-in-nanogenics--14816727.html