REG - Nuformix PLC - Confirmation of European Orphan Drug Designation

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RNS Number : 7159G  Nuformix PLC  30 April 2025

30 April 2025

 

Nuformix plc

 

("Nuformix" or the "Company")

 

Confirmation of European Orphan Drug Designation for NXP002 Programme in IPF

 

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."

 

Enquiries:

 

 Nuformix plc
 Dr Dan Gooding, Executive Director  Via IFC Advisory

 CMC Markets
 Douglas Crippen                     +44 (0) 20 3003 8632

 IFC Advisory Limited
 Tim Metcalfe                        +44 (0) 20 3934 6630

 Zach Cohen                          nuformix@investor-focus.co.uk

 

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 preclinical assets with potential
for significant value and early licensing opportunities.

 

About IPF

 

IPF is a chronic lung disease characterised by progressive tissue scarring
that prevents proper lung function. It is a progressive, fatal, age-associated
lung disease affecting approximately three out of every one hundred thousand
people in Europe. IPF typically presents in adults 65 or older and is usually
fatal within two to five years after diagnosis.

 

 

 

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