Nuformix Regulatory News (NFX)

23 September 2022

Nuformix plc

("Nuformix" or the "Company" or the "Group")

NXP002 Update

Nuformix plc (LSE: NFX), a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing, is pleased to announce the following update regarding the Company's NXP002 programme, a proprietary new form of tranilast, being developed as a novel inhaled treatment for Idiopathic Pulmonary Fibrosis ("IPF").

2022 European Respiratory Society Congress ("ERS")

The Company recently presented NXP002 for the first time to the global IPF medical and pharmaceutical specialist community at the ERS Congress in Barcelona.  The Company's NXP002 poster sessions were well-attended by, amongst others, potential licensing partners, while the conference confirmed that inhalation is now widely accepted as a future IPF treatment modality.

In spite of their limited efficacy and poor side effect profile, existing standard-of-care therapies (OFEV®, Esbriet®) are not expected to be displaced in the near future as emerging data relating to prevention of exacerbations, now linked with mortality, are supportive of their continued use. Therefore, NXP002's clinical route to patients will be additive to current standards-of-care.

As in cancer and other severe respiratory diseases, combination therapies are likely to become the first-line treatment regimen in IPF. NXP002 standard-of-care combinations will therefore now become the focus of the Company's future NXP002 development activities, initially using 3D human tissue models of IPF, to better align development with the growing preference of licensing partners and regulators.

NXP002 remains a significant potential value driver for the Company given no new therapies have been approved in IPF, the potential impact of COVID on fibrotic lung disease, the progress of the NXP002 programme in discharging key development risks and commercial intelligence obtained at ERS. Furthermore, the multiple clinical research publications emerging during 2022 studying the acute use of oral tranilast in treating severe COVID patients' fibrosis are highly supportive of NXP002's potential in treating IPF chronically via inhalation.

Pre-Clinical Update

The pre-clinical inhalation strategy, initiated by the Company's previous management, has significantly progressed the overall NXP002 programme:

o  tranilast, as NXP002, can be delivered in-vivo by a range of nebulisers at the optimum particle size;

o  very high doses appear to be well-tolerated; and

o  an in-vivo dose response was observed for inflammatory and fibrotic biomarkers following inhalation that is consistent with previous ex-vivo human IPF tissue studies.

However, the pre-clinical inhalation disease model chosen has proven to be complex, and unlike previous studies in human IPF tissue, it continues to be challenging to achieve consistent and reproducible results, which will likely increase in the study of combination therapies, which are now clearly required given the clinical trends intelligence gathered at the ERS. As a consequence, the Company is stopping its current study and will initiate studies in 3D human IPF lung tissue using a disease and species relevant model that is now available commercially via a CRO. Work under this new strategy will start immediately and focus on NXP002 in combination with current standards-of-care, with the ultimate aim of reducing their negative side effects whilst enhancing their efficacy.

NXP002 combinations have already shown great promise in human diseased IPF tissue, showing a pleasing synergistic efficacy effect with low doses of standards-of-care, resulting in the Group filing a new combination patent application earlier in 2022. The change in pre-clinical strategy supported by intelligence gathered at ERS is therefore aligned with and further enhances the Company's overall patent position as it continues to build assets for eventual out-licensing.

In addition to new work in human diseased IPF tissue, the Company will evaluate NXP002 ex-vivo in healthy human lung tissue and human peripheral blood mononuclear cells. These studies will be funded from the Company's existing cash position and alongside further human IPF lung tissue studies, will allow the Company to rapidly and cost-effectively investigate NXP002's duration of action. With the exception of Investigational New Drug Application enabling studies, these studies are expected to be the last non-clinical studies conducted as the Company seeks to move NXP002 to the next stage of development.

Results will continue to be generated throughout H2 2022 with further updates announced in due course, as appropriate.

Commenting, Dr Dan Gooding, Executive Director of Nuformix, said: "Our first attendance at ERS has introduced our lead programme to major respiratory players, whilst generating valuable intelligence, allowing us to maintain and develop our current market-orientated development strategy. As a virtual company, Nuformix can rapidly adapt its development strategy to align with emerging trends. Fortunately, we are in a moment where those trends are converging with our programme, our enhanced IP position and our access to a new, close-to-patient, disease relevant model. The prospect of increased efficacy with improved quality of life for IPF patients is a realistic possibility for NXP002 combination therapies based on our existing pilot human IPF tissue data. I am confident that our revised pre-clinical strategy will maximise the value of our IP estate and will generate further compelling data to support both progression and partnering of NXP002. I look forward to providing further updates throughout the remainder of the year."

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About Nuformix

Nuformix is a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing. The Company aims to use its expertise in discovering, developing and patenting novel drug forms, with improved physical properties, to develop new products in new indications that are, importantly, differentiated from the original (by way of dosage, delivery route or presentation), thus creating new and attractive commercial opportunities. Nuformix has a pipeline of pre-clinical assets with potential for significant value and early licensing opportunities.

About Fibrosis

Fibrotic disease is typically associated with high patient mortality, increasing prevalence and a lack of safe and effective treatments. Whilst fibrosis treatments are in their infancy the emerging lung fibrosis market demonstrates their blockbuster potential. Idiopathic Pulmonary Fibrosis is classified as a rare disease and presents a global commercial market that is forecast to grow to $5bn by 2025. Sales of standard-of-care therapies OFEV® and Esbriet® achieved $2.58bn and $1.04bn respectively in 2021.