# Nuformix continues to advance and exploit current assets within the portfolio

- Link: https://www.directorstalkinterviews.com/nuformix-continues-to-advance-and-exploit-current-assets-within-the-portfolio/4121160631
- Published: 2024-06-18T07:15:06+01:00
- Author: Amilia Stone

**Nuformix plc** ([LON:NFX](https://www.directorstalkinterviews.com/company/nuformix-plc/nfx.l)),
a pharmaceutical development company targeting unmet medical needs in fibrosis and
oncology via drug repurposing, has announced its unaudited results for the six months
ended 31 March 2024.

**Operational highlights (including post-period end)**

·    The Board continues to be encouraged by the positive data generated to date
on NXP002 in context to the evolving treatment landscape and understanding of future
patient needs in the treatment of fibrosing interstitial lung diseases. Preliminary
discussions have been initiated with potential partners and are on-going.

·    Notice of Allowance was received for the Japanese National Phase Patent Application
No. 2020-555115 for NXP002 entitled “CRYSTALLINE TRANILAST SALTS AND THEIR PHARMACEUTICAL
USE” which has now formally granted. Notice of Allowance has also been received 
from the European Patent Office for the European National Phase Application, with
formal granting complete. This patent describes proprietary new forms of tranilast
being progressed by the Company as NXP002 as a potential novel idiopathic pulmonary
fibrosis (“IPF”) treatment. These proprietary drug forms uniquely enable drug delivery
via an inhaled nebulised formulation.

·    Just prior to the beginning of the period, on 18 September 2023, the Company
announced that Oxilio had acquired ownership of its NXP001 patent estate for which
Nuformix received new immediate and near-term undisclosed milestone payments, whilst
retaining further development milestones and royalties capped at £2 million per 
year.

·    During the period the Company received proceeds from the Company’s subscription
and associated sharing arrangements with Lanstead Capital Investors L.P. (“Lanstead”).
The sharing agreements ended in October 2023, concluding this arrangement and the
Company is due no further funds from Lanstead.

·    A subscription for 75,000,000 new ordinary shares at a price of 0.20 pence 
per share raised gross proceeds of £150,000, representing approximately 9.2 per 
cent. of the Company’s enlarged issued share capital.

 **Financial Highlights**

·    Loss before tax of £242,529 (31 March 2023: loss of £337,622)

·    Loss on ordinary activities (after tax credit) of £242,529 (31 March 2023: 
loss of £337,622)

·    Loss per share 0.03p (31 March 2023: 0.05p)

·    Net assets of £4,102,051 (31 March 2023: £4,228,204) including £183,523 of 
cash and cash equivalents at 31 March 2024 (31 March 2023: £259,259)

> **Dr Dan Gooding, Executive Director** **of Nuformix, said:** _“Our research efforts
> and external discussions to date demonstrate that inhaled treatment of IPF and
> related fibrotic lung diseases via NXP002 is a viable and attractive concept. 
> We remain focused on generating data and initiating, and further developing, discussions
> with potential partners that will support our efforts to secure out-license deals
> on NXP002 and NXP004._ _Progression of these opportunities is being achieved using
> existing funds thanks to a lean operational model, which will continue to operate
> until our R&D and targeted partnering activities require further resource. I am
> excited by our prospects for the remainder of the year and look forward to sharing
> results as they emerge.”_

**Chairman’s statement**

**Operational review**

**NXP002 (novel proprietary forms of tranilast): Interstitial Lung Diseases (“ILDs”)
including Idiopathic Pulmonary Fibrosis (“IPF”) and Progressive Pulmonary Fibrosis(“
PPF”)**

NXP002 is the Group’s preclinical lead asset and a potential novel inhaled treatment
for IPF and PPF and possibly other fibrosing ILDs. NXP002 is a proprietary, new 
form of the drug tranilast. NXP002’s enhanced physical property profile uniquely
delivery to the lung in an inhaled, smart nebuliser formulation.

There are more than 200 types of interstitial lung diseases (“ILD”), which are characterised
by varied amounts of inflammation, scarring, or both, that damage the lung’s ability
to absorb oxygen. IPF is the most well-known form of ILD, affecting approximately
100,000 patients per year in the US. Progressive Pulmonary Fibrosis (PPF), previously
referred to as Progressive Fibrosing ILD (PF-ILD), is a larger and even more poorly
served segment of the ILD market, affecting more than 200k patients per year in 
the US.

IPF and PPF are devastating lung diseases associated with a higher mortality rate
than many cancers with median survival of 3-5 years. Thus, IPF and PPF represent
a high unmet medical need such that the requirement for improved treatment options
represents a significant commercial opportunity. IPF is classified as a rare disease
and presents a global commercial market that is forecast to grow to US$8.8bn by 
2027. Sales of standard-of-care (“SoC”) therapies OFEV and Esbriet (now off patent)
achieved US$3.5bn and US$0.8bn respectively in 2022.

Tranilast has a long history of safe use as an oral drug for asthma, keloids and
hypertrophic scarring, but while there is growing evidence that supports its potential
use in other fibrotic conditions, including IPF, a combination of poor physicochemical
properties, variable pharmacokinetics and challenging pharmacodynamics following
oral delivery limit its potential use in ILDs. NXP002 is differentiated as it is
a patent protected, novel form of tranilast that has been optimised for formulation
and delivery direct to the lungs by inhalation, potentially overcoming the issues
using tranilast orally as a chronic treatment for ILDs.

NXP002 as a potential treatment for IPF, is a likely candidate for Orphan Drug Designation,
which could provide additional product protection against potential future competitors
in addition to product development advantages.

The inhalation route is a well-known strategy for the treatment of lung diseases
to yield greater efficacy and reduce systemic, off-target side-effects compared 
to oral treatment. Discontinuation of treatment in IPF and PPF patients is currently
an issue in the treatment of these diseases with discontinuation rates for current
SoCs up to 80% in certain patient groups due to their debilitating systemic side-
effects. Effective inhalation therapies offer the potential to overcome these limitations
of oral therapies.

The positioning of NXP002 as an inhaled treatment for IPF and PPF could be either
as added to SoC treatments or administered as a monotherapy for patients non-responsive
to SoCs and those declining these therapies due to side effects which impact quality
of life.

The Group’s pre-clinical inhalation development strategy has significantly progressed
NXP002 towards validation of its Target Product Profile (“TPP”) demonstrating:

• NXP002 can be delivered in-vivo by a range of nebulisers at the optimum particle
size for delivery to the deep lung;

• high doses appear to be well-tolerated; and

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

The Group conducted studies in a new iteration of a 3D human IPF lung tissue using
a disease and species relevant model that has been advanced to significantly reduce
output variability. The results from these studies of NXP002 alone and in combination
with current SoCs, can be summarised as follows:

• NXP002 is well tolerated in ex-vivo human lung tissue with no signs of toxicity
events;

• NXP002 alone delivers a strong, consistent anti-fibrotic and anti-inflammatory
effect as demonstrated by modulation of the release of multiple biomarkers of fibrosis
and inflammation;

• both high and low concentrations of NXP002 show an additive anti-fibrotic and 
anti-inflammatory effect to SoC;

• in particular, the higher concentrations of NXP002 with SoC’s deliver a near complete
ablation of fibrosis biomarker release, yet at lower concentrations than have been
seen in other preclinical models to date; and

• the clear, pronounced additive benefit of NXP002 on top of SoCs observed suggests
that NXP002 may provide additional efficacy, even in patients responding to SoC 
therapy.

This raises the possibility that NXP002 targets additional disease pathways to SoC’s
when increasing the combined anti-fibrotic and anti-inflammatory response. Following
success in suppressing biomarkers of fibrotic disease progression in human IPF lung
tissue, the same samples were analysed to assess additional mechanistic and anti-
inflammatory benefits on top of SoC’s and the results are summarised as follows:

• NXP002 alone delivers a strong, consistent anti-inflammatory effect as demonstrated
by suppression of the release of inflammatory cytokines by over 90% for all cytokines
studied; and

• the results further suggest that NXP002 may provide additional efficacy in combination
with SoC’s, even in patients not responding to SoC therapy alone.

Nuformix’s TPP for NXP002 seeks twice daily inhalation administration. To assess
NXP002’s duration of action, the Group initiated work in an exploratory model in
healthy human lung tissue. The model also bridges the Group’s successful preclinical
work across a variety of LPS-challenge studies. The results are summarised as follows:

• NXP002 suppresses the release of inflammatory cytokines by healthy human lung 
tissue following LPS challenge; and

• an anti-inflammatory effect remains at 12 hours post drug dosing demonstrated 
by continued suppression of the release of inflammatory cytokines following LPS 
challenge, confirming NXP002 has a duration of action that may support twice daily
dosing.

Overall, the Board is encouraged by the progress of the studies and the positive
data generated to date and is considering next steps, including potential further
R&D studies to add further value to the programme and support on-going licensing
activities.

**NXP004 (novel forms of olaparib) – Oncology**

The Group discovered novel forms of olaparib, a drug currently marketed by AstraZeneca,
as Lynparza^(®). Lynparza^(®) was first approved in December 2014 for the treatment
of adults with advanced ovarian cancer and deleterious or suspected deleterious 
germline BRCA mutation. Since then, it has secured similar approvals in breast, 
pancreatic and prostate cancers with further trials on-going. These approvals have
propelled Lynparza^(®) sales to US$2.6bn in 2022 with industry analysts forecasting
annual sales of US$9.7bn by 2028.

Subsequently, further preformulation and in-vitro studies allowed Nuformix to identify
lead cocrystals to be progressed for further development. Results from in vitro 
dissolution studies demonstrated that the two lead NXP004 cocrystals out-performed
Lynparza^(®), both in terms of rate and extent of dissolution and release of olaparib.

Enhancement of dissolution in the currently marketed formulation of Lynparza^(®)
resulted in improved bioavailability versus the initial marketed product. Therefore,
NXP004 may offer potential to further increase olaparib’s bioavailability. In addition,
the potential simplicity of NXP004-based formulations may offer improvements in 
product cost-of-goods versus the currently marketed product, which requires complex
manufacturing methods.

These attributes position NXP004 for applications in line-extensions for the currently
marketed product, or for possible development in future first-to-generic product
opportunities. This work will direct and support future out-licensing discussions
for NXP004.

**_NXP001 (new form of aprepitant) – Oncology_**

NXP001 is a proprietary new form of the drug aprepitant that is currently marketed
as a product in the oncology supportive care setting (chemotherapy induced nausea
and vomiting) initially exclusively licensed to Oxilio Limited (“Oxilio”) for oncology
indications. Oxilio has now acquired ownership of Nuformix’s NXP001 patent estate.
Nuformix retained rights to receive further development milestones and royalties
capped at £2 million per year under the terms of the acquisition.

**Outlook**

The Company continues to advance and exploit the current assets within the portfolio
through the R&D and business development activities as set out above.

The strategy of the Group is to continue to increase the value of its existing assets
while maintaining tight control of costs, including conducting business development/
licensing activities using a structured and data-driven approach, with the goal 
of seeking global licensing deals.

**Financial Review**

In the first half of the financial year, the Board has continued to focus expenditure
on R&D activities that add value to the current assets while optimising the operation
to minimise administrative expenditure and the operational cost-base.

**Dr Julian Gilbert**

**Non-Executive Chairman, Nuformix** **plc**

**18 June 2024**

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