Pila Pharma was introduced on Nasdaq First North Growth Market a little over a year ago, it did so with a drug candidate in early clinical phase, aimed at treating type 2 diabetes. After just over a year as a listed company, it is in a stable position with the drug candidate XEN-D0501, which is entering late clinical phase in no less than two indications.
XEN-D0501 targets the TRPV1 receptor, which plays a crucial role in the human ability to perceive pain and also promotes local inflammation. According to the company's hypothesis, excessive adipose tissue in the body leads to overstimulation of the TRPV1 receptors, which in turn leads to inhibited insulin production and insulin sensitivity. This is the very basic theme behind the main project with XEN-D0501, which is to inhibit the TRPV1 receptors in patients suffering from type 2 diabetes. Read more about Pila Pharma's clinical concept here.
The project is now preparing for Phase IIb clinical trials, where a three-month preclinical toxicology program was recently initiated.
Taking the step into pain relief
In addition to its focus on diabetes, Pila Pharma has long planned to also take the step into pain relief, which may seem natural given the drug candidate's mechanism of action. Last spring, it was announced that the time for the investment had come, and in mid-July the long-awaited announcement came – that the US Food and Drug Administration FDA gives XEN-D0501 orphan drug status, so-called Orphan Drug Designation, for the treatment of erythromelalgia.
Erythromelalgia is a rare disease that affects on average 1,3 out of 100 people and is characterized by severe redness of the skin and intense burning pain. The disease most often occurs in the feet, hands, ears and nose; but can also occur in other parts of the body in more rare cases. The affected part of the body often swells and the skin becomes very hot. There is currently no curative treatment available for patients suffering from erythromelalgia, but the treatments that are available only relieve the symptoms.
Flying start for the project
Pila Pharma's orphan drug designation represents an important step in providing erythromelalgia patients with a better alternative to the current treatment regimen. Should the company achieve market registration with the candidate, the orphan drug designation will give the company seven years of exclusivity in the US market.
Once the clinical work in the project gets underway, the company will be able to credit previous results achieved with XEN-D0501 in the diabetes project. This will allow Pila Pharma to go directly to Phase II/III studies with the candidate and is currently evaluating it to take it to patients as quickly and cost-effectively as possible.
Recruiting experienced project manager
Pila Pharma also announces the recruitment of Dr. Hans Quiding who will lead the new project. Quiding has extensive experience with over 35 years in AstraZeneca where he was involved in the development and launch of the bestsellers Cytodon and ArdinexHe thus brings valuable experience in pain management to the company that fits well into the new role, especially since he also worked at AstraZeneca on potential treatments for erythromelalgia.
Comments from the CEO
BioStock contacted Pila Pharma's CEO Dorte X. Gram to find out more about what this summer's big news means for the company.
First of all, what does it mean that you have now received orphan drug status for XEN-D0501 for the treatment of erythromelalgia?
– This means that we now have two legs to stand on. Diabetes is still our main indication and the fact that we are now entering erythromelalgia does not mean that we are shifting our focus away from diabetes. It is an opportunity for us to maximize the commercialization possibilities of our asset.
– It was not crucial for our investment in erythromelalgia, but orphan drug status means that we will be able to receive help and advice from the FDA during the course of the project. Then we will also get seven years of additional protection if we reach the market, which may also be important for our diabetes project.
Why was this disease chosen?
– We have long wanted to enter pain with XEN-D0501, which is a relatively old molecule. We wanted to enter a rare indication and had a discussion with our partner where we looked at several different possible paths. In the end, the choice fell on erythromelalgia, which was a perfect fit for us right now.
You state that you can go directly to phase II/III studies in erythromelalgia. How large a study could it be and what costs can one expect to incur to conduct one?
– Finding out is a task that lies ahead of us now. We have interest from the authorities and we will hold a dialogue there to find the best way forward. Normally, 200 – 300 patients are needed for this type of study and it is a matter of finding them, which can be difficult when dealing with rare diseases.
– Our hope is to be able to do a smaller study first and then a larger study after that, a bit like how we have worked in diabetes. The goal is to make the work as cost- and time-efficient as possible. We will work on a plan during the fall that we can then present in the spring.