Nuformix plc (LON:NFX) Executive Director Dr Dan Gooding caught up with DirectorsTalk to discuss the company’s NXP002 programme for idiopathic pulmonary fibrosis, the significance of FDA Orphan Drug Designation, and the purpose of the recent fundraise.
Q1: You recently announced both the securing of an orphan drug designation for your NXP002 programme in IPF and a fundraise. For those who are new to Nuformix, can you just tell us about the company, what IPF is and what the NXP002 programme is?
A1: Nuformix is a drug development company. We are unique in that we develop new forms of known drugs for new uses so this is a slightly de-risked R&D strategy, as you might usually see for typical biotech that are developing new therapies from scratch. Our business model is to develop those therapies up to a proof of concept and then license on the associated IP and data to drug companies that take the programmes forward and onto market, as is typical for our industry.
So, we have a lead programme, which is an inhaled treatment of a fibrotic lung disease known as idiopathic pulmonary fibrosis, or as we abbreviate it to, IPF. IPF itself is a rare disease, which is why it’s recognised as an orphan condition, we’ll come onto that. It’s a high mortality condition, it impacts about 4 in 10,000 people in the US, which is the news that we’re talking about today with respect to the US orphan drug designation. So, it’s below the threshold for orphan drug consideration in that territory.
Those diagnosed with this condition have a median life expectancy of about four years from diagnosis, which is a pretty bleak outlook. It’s very poorly treated, which is why there’s a real push for new therapies. There are approved therapies on the market, but they don’t, however, really halt lung function decline. They also don’t extend life or improve lung function. So, they are not the most efficacious therapies, and they all have severe side effects, predominantly as a cause of being oral therapies. However, a couple of these approved products, the first two approved products, did achieve blockbuster status. So, promising therapies are therefore highly sought after both by patients, because there’s a lot of unmet need, but also by drug companies, because there’s a commercial validation in terms of the sorts of sales revenue they can generate.
In terms of NXP002, this is our lead programme. As I mentioned, it’s an inhaled treatment for IPF and related to fibrotic lung conditions. The inhalation piece is what’s unique, and it’s enabled by our technology and our intellectual property. This inhalation route of delivery gives us the opportunity for reduced side effects and increased efficacy in a lung condition. As I mentioned, our approach is based on an approved drug, and this approved drug, tranilast, has shown promise in a variety of related fibrotic conditions following oral delivery, which means that when we’re thinking about using it in the lung for IPF, we can have confidence in its clinical utility, safety, and potential for efficacy. So, we have been able to look at existing data.
What’s really exciting about this particular molecule is it seems to have this potential that over a period of treatment, it can actually potentially resolve fibrosis and restore organ function. It’s done quite well in a series of smaller clinical studies following oral delivery. We believe that this sort of differentiated profile that it has with respect to resolution of fibrosis and organ function restoration is really what makes it different from other therapies in development.
So, we’re keen to take this programme forward.
Q2: Moving specifically then to the orphan drug designation. What are the benefits of this and why is it particularly relevant to NXP002?
A2: For those that don’t know, orphan drug designation is a regulatory status. It’s granted to companies developing medicines that are considered to treat rare diseases. Depending on the territory that you’re in and the regulatory body that you’re seeking orphan drug designation from, they will have different thresholds in terms of the rarity of that particular disease.
In general, this status is designed to incentivise companies to develop treatments for smaller patient populations that might in certain circumstances be commercially unattractive due to the investment costs required to actually achieve approval on the number of sort of treatable patients. So, it’s really to incentivise the development of therapies for rare diseases.
There are some really quite attractive benefits. So, if you’re able to secure orphan drug designation for a particular therapy and a particular disease, what this means is that competitors can’t market the drug that you’re developing for that indication during what’s termed an exclusivity period. Now, the exclusivity period depends on what territory you’re in. In the US, it’s seven years after drug approval, you get market exclusivity. In Europe, it’s 10 years. So, that’s the top level commercial benefits.
There’s also some other important benefits too. There’s regulatory support. Usually your discussions with regulators, the EMA, the FDA can be quite expensive, they can be quite time consuming but actually, in the orphan situation, you get advice around your clinical protocol design. You can also get scientific advice from advisors and regulators, plus faster development pathways. This also all comes with fee reductions and also occasionally tax credits as well for the money that’s been spent on R&D.
So, why this is particularly relevant news for NXP002, certainly the US orphan drug designation side of it, is that the global market for IPF is really dominated by sales, firstly to the US and then to European patients. In the US, you see the highest patient prevalence there. Also, drug prices are highest there, meaning that the US accounts, as by some reports, for as much as 80% of the global IPF market itself. So, to secure market exclusivity is really attractive for 80%, effectively, of the market.
When companies receive this orphan drug designation, it’s actually transferable to future licensing and development partners. So, securing orphan drug designation for the US market and also for Europe, as we have already, is considered highly attractive to potential partners. This kind of protection also supplements our existing IP. We obviously have patent-protected products that are long lasting, but this piece on top for the orphan drug designation really enhances that.
Lastly, what this means is in terms of getting the approval from both the EMA and the US, this is really adding a lot of plausibility to the position that we’ve got this differentiated product profile versus other therapies, be they approved therapies or those in development. So, it’s a really good stamp of approval from the two leading agencies globally.
Q3: Why fundraise now?
A3: I think as we’ve stated, our objective is to find a licensing development partner from this moment. The orphan drug designation should help us pursue that objective towards completion. To do so effectively, we need to negotiate from a position of financial strength and also progress the programme. We need to address technical questions as they come up during the due diligence process, which, as some people know, has been going on for some time.
So, with the raise, we’re now able to address a small number of points that have arisen from that process to date and by addressing those exact points that have arisen, we’re adding value to the programme each time we’re able to do that. We know exactly what these studies look like in order to address the points that have arisen during the due diligence process. So, that’s the reason for the raise.
Q4: Just moving forward, what’s next for Nuformix?
A4: We’re investing, going back to all of the parties with whom we’re in contact and have had discussions and shared data, etc. We’re giving them the news that we’ve been successful in securing the orphan drug designation piece. That, for those that have certain questions, that they know that we’re now able to give them answers to those questions, which is really exciting.
So, that’s what’s coming next. Conducting those studies, even being able to communicate that we’ve initiated that work is going to be something really important to talk to them about, as well as the orphan drug designation itself. Then it’s really seeking to close a licensing or collaborative development agreement with a partner from this moment onwards, really. It might not be necessary for us to complete that complete package of work that we envisage in order to address the questions that have arisen in the process. That remains to be seen.
What’s next for us is going back to our partners, re-engaging with them on this news and addressing the questions that have arisen in the due diligence to date and progressing from there.
