Balancing Profitability and Mission

LEO Pharma has a strong focus on orphan drug development. What sets your approach apart from other companies in this space, and how do you balance the need for profitability with the mission of providing treatments for rare diseases?

I mean, in our case, it's actually a renewed focus. It's again something that LEO attempted historically, and now we're kind of going back and looking at orphan drugs, and specifically now we've just recently completed the acquisition of a company called timber pharmaceuticals, which was is developing a treatment for a really rare and debilitating disease tape called Congenital ichthyosis, and the two subtypes of ichthyosis that we treat effectively, you have patients that have large surfaces of their body covered with what appear to be like fish scales. So, imagine, again, having a child born with effectively fish scales, and the amount of time that they spend descaling themselves, the amount of time they spend trying to find, you know, different kind of ways of finding comfort when there is no treatment for this disease.

Now, it is very rare. I mean, I'll talk to a lot of dermatologists, will say I've seen one patient with this condition in my entire career, or there's someone who I maybe saw during medical school. That's not something that you know is very prevalent. Which brings, comes back to your question, you know, does this make sense from a sustainability standpoint? Is it profitable for us to move into this disease state? I think in this particular instance, this is a great example of how LEO Pharma is going to be the best owner for this type of asset, because it's taking an old treatment that is very well known in Accutane or isotretinoin, reformulating it as a topical in a way that has very low systemic absorption and therefore can be used on large body surfaces. Now, is that as interesting and innovative as a gene therapy, or, you know, some of the other areas that other companies are going into in rare disease, I can tell you, for someone living with congenital ichthyosis, it is a massive innovation. And for the provider that is treating them again, it's providing them with a solution when previously they didn't have a solution.

I think the other thing that's really important when you get into rare disease is to understand that while rare diseases typically get treated at a few academic centers across the nation. Patients living with these diseases don't necessarily live within the footprint of an academic center, so having a company that does have a field force that is calling on dermatologists across the country, making sure that those dermatologists are aware that now there's a treatment for this condition, helping them to understand what to do if they see a patient that presents themselves for these treatments, whether it's a referral to that academic center or maybe, in this case, an opportunity for them to treat themselves. Is why I think that, again, this is a really good area for us to get into. It's finding that kind of white space in the market, and then, quite frankly, what I feel great. About is that there are patients that wouldn't otherwise have a treatment if it wasn't for LEO pharma, acquiring this company, continuing the clinical development program, while that company was kind of struggling to fundraise. And, you know, knock on wood that the data comes strong and we're able to file and get this innovation to the marketplace.