Confirmation of European Orphan Drug Designation

Nuformix plc (LSE:NFX), a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing, is pleased to announce, further to the Company's announcement on 28 January 2025, that it has received notification from the European Medicines Agency's ("EMA") Committee for Orphan Medicinal Products ("COMP") of its positive opinion regarding Orphan Drug Designation ("ODD") in Idiopathic Pulmonary Fibrosis ("IPF") for tranilast, the active drug substance enabled for inhaled delivery in Nuformix's NXP002 lead programme.

EMA ODD is granted to drugs intended for the treatment, diagnosis, or prevention of life-threatening or chronically debilitating conditions affecting no more than five in 10,000 individuals in the European Union ("EU"). In its communication, the COMP confirmed that NXP002 satisfies the criteria for orphan designation and that the Company has established that NXP002 has the potential to be of significant benefit to those affected by IPF. The COMP also concluded that NXP002's additive effect in combination with anti-fibrotic agents, as well its potential to be used in patients intolerant to these medicines constituted a clinically relevant advantage.

The Company now awaits the European Commission's final ratification of the EMA opinion, which is expected to be received within a 30-day period.

Dr Dan Gooding, Executive Director, Nuformix, said: "We are delighted to receive news of the COMPs positive opinion regarding NXP002's eligibility for Orphan Drug Designation in IPF, a high-mortality rare disease, in urgent need of new treatments.

"The EMA's procedure for awarding ODD status involves considerable scientific scrutiny. Therefore, the opinion serves as powerful independent third-party validation of NXP002's underlying scientific rationale and existing data supporting its potential efficacy in treating fibrotic lung diseases such as IPF. In addition to this validation, there are numerous developmental and commercial incentives to securing ODD status for NXP002, including 10 years marketing exclusivity, all of which would be transferable to future licensing partners. The COMP's opinion also underscores the significant unmet need for patients, in spite of the fact that treatments have been approved in the EU. We will now progress to applying for US FDA orphan drug designation. Given the EMA's positive opinion, we are hopeful that the US FDA would conclude similarly. We have shared the EMA's opinion with potential future licensing partners and will provide further updates in due course as appropriate."

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