Nuformix Plc (LON:NFX) Executive Director Dr Dan Gooding caught up with DirectorsTalk to discuss the positive EMA opinion on orphan drug designation for NXP002 in treating idiopathic pulmonary fibrosis (IPF).
Q1: First off, congratulations on the announcement this morning. How does the positive opinion from the EMA’s Committee for Orphan Medicinal Products validate Nuformix NXP002 in treating idiopathic pulmonary fibrosis?
A1: It validates it in a number of ways. First of all, the reason why we have orphan drug designation in the first place, and this is something which we see in multiple territories, in our case today, the European orphan drug designation, but also similar processes in the US, Japan. It’s a recognition that there is a rare disease, a small patient population, and that in situations where investment is made in developing therapies for small patient populations, incentives are granted to make the opportunity for getting reimbursement for some current ECOS back from development of those therapies.
So, the first reason it’s important is it’s saying, okay, we’ve been able to demonstrate that IPF is a rare disease. In the case of the European classification for that, we need to show that there’s a threshold of 5 or below for every 10,000 people that get affected with this condition. So, that’s the first thing, validating this rare disease status.
The second thing is then looking at what the current standard of care is, and this is a situation where there are treatments in existence for the disease in question here, idiopathic pulmonary fibrosis or IPF and it’s saying that even though those treatments exist, there’s still a really significant unmet need. There is a really significant unmet need in this condition, because for those that are not aware, this is a really high mortality condition, where once someone is diagnosed with this disease, one has something like between two to five years left of life. At the moment, there are therapies out there, but they’re not impacting really that trajectory in terms of mortality. So, they’re the first two pieces.
The third, I think, and probably the most important piece is that the reason that we went for European designation first is that it represents probably the highest scientific hurdle to clear. As part of the process, there’s a third-party scientific validation of the data and the rationale as to why we’re presenting Tranilast as a therapy for IPF and why we believe that this drug can be efficacious based on the data that we’ve got. So, the European process evaluates that data and then forms an opinion. It’s that third-party independent validation of really the entire data package that we’ve generated to date that’s really under scrutiny. So, the really positive news is that that has been looked at by an independent third party and found to be very positive.
I think that the comments that the EMA have made are really supportive, particularly recognising that with the approach that we’re taking with inhalation, using this drug in this way, we’ve got the potential to deliver meaningful clinical benefits.
So, that’s the summary, really, of the key points from today.
Q2: What are the potential benefits for orphan drug designation in the EU for the group?
A2: Let’s start with the science. So, as I just mentioned, the scientific side of the benefit is this independent sort of third-party validation. Really looking at the data and saying, yes, we agree with the conclusion from the data generated that our product, NXP002, should therefore have the ability to deliver this kind of meaningful clinical benefit in this patient group.
From the commercial side, there are a range of incentives that are now applied to the programme and there are incentives that can relate to development. For example, we will be eligible for more favourable fees or lack of fees entirely in getting the EMA’s approval for future protocols and such like. And advice in terms of how we structure documents and write documents to support the onward development of the molecule.
There’s also a 10-year market exclusivity benefit, which is now attached to the development of this product. That means that from the moment that this molecule becomes approved for the treatment of IPF, it would have 10 years exclusivity in the market before competitors were allowed into the market. That’s on top of all of the IP protection and enablement that Nuformix has towards this molecule for inhalation purposes as well, which is fantastic.
Now, those benefits stay with the programme so if we were to partner or license the programme to a third party, then those benefits would stay with the programme, so it becomes an asset effectively attributed to the programme itself.
Q3: How might EMA’s opinion accelerate Nuformix’s progress in securing partnerships and licensing deals for NXP002?
A3: I think it does it in exactly the two ways that we’ve discussed, really. It’s that third party validation of the data and of the potential of the molecule to meet the unmet need in the disease based on the molecule’s activity, etc. And then the data that we’ve generated today, and particularly the data we generated in patients, IPF patient lung tissue, which is really quite compelling.
The second piece is knowing, therefore, that as a developer of this molecule for this condition, they will be the beneficiary of all of these incentives, accelerated pass-through development, reimbursement of costs and advice in the development process. Also, when getting to market, they will also enjoy this 10-year period of market exclusivity in Europe. So, that’s how it will benefit those discussions.
It also benefits the discussions in a way that having presented the data in the way that we presented it, we will now be able to take that same information to the US FDA, which also have an orphan drug designation process, and also can be quite hopeful being able to secure that designation for that territory as well, which is arguably the largest market for this molecule in the world. If successful there, as one would expect to be, having been successful with the European process, we would enjoy another seven years market exclusivity in the US as well.
So, these are the kinds of benefits that one’s going to approve from this in terms of discussions with potential partners.
Q4: What can investors then expect from Nuformix in the coming months?
A4: Obviously, this now gives us a reason to go back and speak to the partners that we’ve been speaking to, to date and let them know about this success, which is really, really good news. As I say, it’s a powerful statement in terms of the potential of the molecule to go off and treat disease. So, it gives us a great reason to go back and progress those discussions further than they have been to date.
We’ll hope to report back on those discussions as they progress.
We will be present at ATS in San Francisco in May. Also speaking to companies face to face there as well and continuing those discussions. Also, as I just mentioned, be progressing with an application for drug designation with the US FDA.
So, we’ll be letting the market know about our progress with all of those activities.
