Nuformix plc (LON:NFX) Executive Director Dan Gooding caught up with DirectorsTalk discuss what the NXP002 programme is & its purpose, key findings from the results, and what’s next for the company and the programme.
Q1: You’ve provided an update regarding the Company’s NXP002 programme, could you just remind us of what that programme is and its purpose?
A1: Before answering the question, I just want to step back a bit and give people that maybe haven’t heard of Nuformix story a little introduction to what the business is about exactly.
We are involved in drug development and that means that we develop various programmes up towards a proof of concept, demonstrating proof of concept, before looking to out-licence our programmes to a development partner. Also, you mentioned the keyword that differentiates us, we’re reprofiling, that means that we’re reusing old drugs and developing them in new ways.
So, that whole piece; licencing, reprofiling, M&A etc, is an area in which the team have proven quantity. What enables us to do this in a unique way is the IP that we have. We find ways of solving historic problems with drugs that have prevented their development or certainty caused problems in their development.
We’re able to finance solutions, patent those solutions and then look to out-licence the results of those improved drug forms indications for which we’re involved. The two indications which we’re are focussed is oncology and then also fibrosis, specifically the idiopathic lung fibrosis, and that’s the news that we’ve released with the updated results that we have. So, that’s the background to the business.
In terms of the work, it’s probably also useful just to describe a little bit about the molecule we’re developing, a little bit about the disease area as well so that the actual impact of the results that we announced could be understood.
Going back to the concept of drug reprofiling, we have a molecule known as ‘tranilast’, we have some improved proprietary forms of that molecule, this molecule is approved across Asia for oral use in Asthma and scar prevention.
More recently, it’s shown immense promise in fibrotic disease which have been validated during COVID so you had this kind of COVID-induced fibrosis. One of the limitations with this molecule is it can’t be developed orally for chronic diseases, for long term use.
However, what we have done is we have intellectual property that unlocks the ability to deliver tranilast to the lung, and it’s direct delivery to the lung and avoids the oral delivery problems. So, we’re actually progressing our NXP002 programme using tranilast as an inhaled treatment for IPF.
IPF, Idiopathic Pulmonary Fibrosis is a progressive lung disease with a really quite high mortality, you have lung tissue that becomes scarred and you have this progressive scarring effect, resulting in a loss of function. This means an ability to take on oxygen. So, if diagnosed with this disease, you have something like 2.5-5 years left in terms of median survival and those are quite unpleasant years as well.
It’s a rare disease but saying that there are two existing treatments that are approved, they don’t change the trajectory of a disease progression much. They are blockbusters in terms of sales, in spite of some quite severe side effects, even so severe that quite a few patients actually decline treatment. So, in summary, the disease area is commercially attractive and that’s a great unmet need.
Typically, patients are older males, anything from late 50’s to early 60’s, it’s a complex disease and it’s initiated by probably a lung injury event which I guess then incubates for something like a 10-20 year period.
The reason I’m mentioning this is because this complexity means it’s very difficult to reduce the disease down to a model that one can understand well and develop drugs for before moving into patients. The existing models don’t really reflect the disease very well and so, this is what today’s announcement is about. We’ve been able to access a model that uses human tissue and patients who are at really end-stage of the disease.
One of the few treatment options that’s actually successful in prolonging life in lung transplantation so patients that are at that end-stage of the disease who are lucky enough to be eligible for a transplant, we’ve actually found a way of getting access to the tissue once it’s removed from the patient.
We’ve been able to then, thanks to developments in the model, test NXP002 against human disease tissue and see how well we’re able to stop that progressive fibrotic process.
So, the results we’re announcing today, use a new iteration of that model that really decrease the variability in the results. This means we’re able to take a really high confidence view on what NXP002 can achieve alone but also in combination with standard care in stopping the progression of fibrosis in IPF.
Q2: Like you say, you’ve received results from the studies, could you give us a summary of those findings?
A2: One of the reasons that we’ve approached the study in the way that we have is that the last 6 months have seen data emerge for existing treatments, also standards of care, which mean that all patients diagnosed with IPF, unless they decline standard of care, will actually be receiving one of two drugs that are approved for use for IPF.
Given some of the side effect profiles that these drugs have, this is quite unexpected but caused this shift in how regulators and potentially development partners are considering new therapies.
So, we had to adapt really to that new thinking in line with standard of care and how new potential therapies in IPF will perform alongside those standards of care.
The objectives of this study were to use this new improved iteration of the human tissue model that allows us to test the much greater number of samples. It allows us to look at NXP002 alone, and in combination with standard of care to better understand the extent to which NXP002 can improve disease treatment.
What we saw were three key things:
- Firstly, we saw no evidence of any toxic events, this is important because we’re taking a drug that’s been delivered orally in the past and now delivering it straight to the lung, we need to understand if there’s going to be any tolerability issues. All the data to that end was great.
- We saw that NXP002 alone produced a really strong antifibrotic effect so this is looking at halting the progression of fibrosis in IPF.
- Really, quite importantly, what we saw was that NXP002 when it was combined with standard of care, after 4 days of treatment of these tissue samples, only tissue samples treated with drug combinations of NXP002 and standard of care had lower levels of fibrosis biomarkers than when the study started.
So, really, this is absolutely the best case scenario that we could’ve wished for when thinking about the objectives of the study.
Q3: Just following the success achieved, what will be next for Nuformix and NXP002?
A3: As we are building out towards being able to initiate discussions with potential development partners and licencing partners, what we’re talking about is a switch of an oral drug into an inhaled treatment for a new disease. There’s a few things that we need to demonstrate as part of that process.
It’s really well accepted, I think now, that the particular drug in question here, tranilast, at the heart of NXP002 is an important molecule for treating fibrosis, I think that aspect is really well validated.
What we also demonstrated really well is that our IP allows inhaled delivery of this particular molecule, and that when we can deliver that molecule in an inhalation setting, that the molecule can leverage its pharmacology following inhalation. These are all boxes that we’ve nicely ticked as we move forward to initiating out-licencing discussions.
So, now what we’re looking at doing is really strongly validating this additive effect of NXP002 and standard of care. So, we’ve done this first study in one tissue donor, in order for licensees and potential partners to have confidence in that data, we need to demonstrate that in up to three donors of tissue. So, that’s one thing we’ll be looking at doing.
The other thing that we’ll be doing is taking existing samples from the study that we’ve just done. We’ve only looked at antifibrotic biomarkers in the study to date, now we’ve seen the data, we can have confidence and go and look at those existing samples in terms of anti-inflammation biomarkers, which is another really important component of treatment. So, that’ll be a second that we’ll do.
The last box, if you like, that we need to tick as well, is to demonstrate that following inhalation, or certainly that this molecule has a long duration of action in terms of the time for which it exerts its pharmacology. We’re actually using a healthy human lung tissue model to that and that’s work that’s just started so we’ll be looking at completing those studies as well in the coming weeks and months.
Depending on those results, we ‘re also looking to get to a point where we can demonstrate in three donors of healthy lung tissue, that we can show a really strong duration of action.
So, once we have this combined data set, duration of action shown, additive effect in terms of what NXP002 offers alongside standard of care and reducing fibrosis, we’ll be in a really strong position for which to initiate discussions. That is what the company will start to work on once we get to that point.
