27th February 2024: N4 Pharma PLC (AIM: N4P), the pre-clinical stage specialist pharmaceutical company, recently acquired a controlling interest in Nanogenics Limited. Nanogenics has developed a lipid and peptide-based delivery system, LipTide®, which it is using in the formulation of a novel siRNA product – ECP105 – targeting an unmet clinical need in the ophthalmology market.

2024年2月27日：临床前期专业制药公司N4 Pharma PLC（AIM:N4P）最近收购了Nanogenics Limited的控股权。Nanogenics开发了一种基于脂质和肽的递送系统LipTide®，它正在用于配制新型siRNA产品ECP105，以满足眼科市场尚未满足的临床需求。

Alongside its ongoing pre-clinical research into Nuvec® - N4 Pharma’s unique novel silica nanoparticle delivery system for cancer treatments, gene therapy and vaccines - Nanogenics 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.

除了对Nuvec®-N4 Pharma独特的新型二氧化硅纳米粒子递送系统进行临床前研究，用于癌症治疗，基因治疗和疫苗之外，Nanogenics最近签署了一份合同，开始配方和序列选择工作，以制备ECP105，用于伦敦国王学院的临床前研究测试。

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. Glaucoma patients who have failed to respond to medication need surgery to lower intraocular pressure which causes fibrosis at the surgery site.

ECP105是为青光眼手术后治疗的恢复而开发的，它包含专有的siRNA序列，可沉默纤维化基因MRTF-B，MRTF-B也负责肝脏和肺部的纤维化。药物治疗无效的青光眼患者需要手术降低眼压，从而导致手术部位纤维化。

This fibrosis often leads to failure of the surgery and patients may have to undergo further surgery. ECP105 is intended for injection into the ocular cavity to switch off the gene responsible for the 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.

这种纤维化通常会导致手术失败，患者可能不得不接受进一步的手术。ECP105旨在注射到眼腔中，以关闭导致纤维化的基因。与目前使用丝裂霉素C等有毒化疗药物进行的标签外治疗不同，ECP105旨在重复使用，以防止需要进一步手术。

Nanogenics has completed 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. 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.

Nanogenics已经完成了一项初步的体内概念验证研究，该研究表明，单剂量的ECP105可以与丝裂霉素C的抗纤维化作用相匹配，而没有任何细胞毒性副作用。重要的是，这意味着ECP105可以在必要时重复给药。该公司现在将与FDA和MHRA进行讨论，以批准GLP毒性研究，并首次进行人体临床试验。

Pre-clinical work on Nuvec has demonstrated its potential to enable more effective cancer treatments by reducing the ability for tumour escape. Recent studies have shown that Nuvec can bind not only single, butmultiple siRNAs aimed at simultaneously targeting identified pathways responsible for cancer progression after initial treatments.

Nuvec的临床前研究表明，它有可能通过降低肿瘤逃逸的能力来实现更有效的癌症治疗。最近的研究表明，Nuvec不仅可以结合单个siRNA，还可以结合多个siRNA，旨在同时靶向初始治疗后负责癌症进展的已鉴定途径。

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.

与单载siRNA相比，用两种siRNA（EGFR和PLK1）测试这些siRNA在双载siRNA 48小时后显示出积极的累加效应。

Through its research program with the University of Queensland N4 Pharma is also evaluating 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 two separate doses.

通过其与昆士兰大学的研究计划，N4制药公司也在评估Nuvec作为包括DNA和RNA在内的寡核苷酸口服递送系统的潜力。实验现已证实，在体内成功口服Nuvec，该Nuvec载有卵清蛋白的DNA质粒，并在两次单独剂量后表现出良好的卵清蛋白表达。

N4 Pharma is continuing its pre-clinical research programme into Nuvec, with further updates on the key areas of siRNA, oral delivery and AAV viral vectors expected in 2024.

N4 Pharma正在继续其针对Nuvec的临床前研究计划，预计2024年将进一步更新siRNA，口服递送和AAV病毒载体的关键领域。

Nigel Theobald, CEO of N4 Pharma commented: “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. 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.”

N4 Pharma首席执行官奈杰尔·西奥博尔德（Nigel Theobald）评论道：“在令人兴奋的基因治疗和肿瘤学领域，非病毒，非脂质递送系统的需求量很高，我们现在有两个这样的递送系统。Nuvec继续展示其作为新型递送系统的多功能性，我们正在开发我们的第一个商业产品，我们的目标是将其纳入第一阶段临床试验。”

About N4 Pharma

关于N4 Pharma

N4 Pharma is a specialist pharmaceutical company developing novel delivery systems for oncology, gene therapy and vaccines. N4 Pharma’s business model is to partner with companies developing novel antigens in these fields to use Nuvec as the delivery vehicle and to develop its own product for post glaucoma surgery.

N4 Pharma是一家专业制药公司，开发用于肿瘤学，基因治疗和疫苗的新型递送系统。N4 Pharma的商业模式是与在这些领域开发新型抗原的公司合作，以使用Nuvec作为递送载体，并开发自己的青光眼后手术产品。

As these products progress through preclinical and clinical programmes, N4 Pharma will seek to receive upfront payments and ultimately royalty payments once products reach the market.

随着这些产品通过临床前和临床计划的进展，N4 Pharma将寻求收到预付款，并在产品上市后最终获得特许权使用费。