Biotechnology company Pila Pharma's XEN-D0501 is being developed primarily as a treatment for type 2 diabetes. The drug candidate targets the protein TRPV1, which has been shown to play a role in the body's insulin secretion - making it a natural parameter in the disease. However, TRPV1 is actually best known for its role in the body's ability to perceive pain and promote local inflammation, so it was perhaps not so surprising when Pila Pharma announced plans to also enter a pain disease with the candidate.
Expanded portfolio the big news in Q2
The big news in the report for the second quarter of 2022 was the broadening of the portfolio to include erythromelalgia in addition to diabetes. During the quarter, Pila Pharma applied for, and then received approval from, FDA, orphan drug status, so-called Orphan Drug Designation, for XEN-D0501 for the treatment of erythromelalgia. In connection with this, Dr. Hans Qviding has been hired as project manager to lead the work. Previous clinical results with the candidate make it possible to go straight into clinical phase II/III studies in the new indication.
In an upcoming article, we will take a closer look at what erythromelalgia is and what entering the indication means for the company as a whole.
Toxicological studies continue during the fall
Within the diabetes project, the production partner could warehouse completed production of study drug with good results. This meant that during the quarter, the three-month preclinical toxicology study could also be initiated, which is the last major step before phase IIb studies with XEN-D0501 begin.
An agreement has also been signed with British contract manufacturer SGS – Quay Pharmaceuticals Limited to develop a suitable formulation of XEN-D0501 for use in the preclinical studies. Agreements have also been signed with the British contract research company DDS Drug Development Solutions (formerly called LGC) to develop an analytical method for measuring XEN-D0501 in samples from the preclinical studies.
According to Pila Pharma, work on the toxicological study will continue during the autumn and the aim is to have results ready sometime by the end of the year.
Pila Pharma prepares for capital raising
Looking at the financial side of the company, the result for the second quarter was -6,5 MSEK, which can be compared with the same period last year when the result was -4,7 MSEK. Cash at the end of the quarter amounted to approximately 10,8 MSEK. In the report, the company writes that it has begun preparations to raise more capital.
Comments from the CEO
BioStock contacted Pila Pharma's CEO Dorte X. Gram to get her thoughts on the past quarter and to hear what she sees ahead.
First of all, how would you describe the second quarter?
– It has been a positive experience and since the stock market listing last year, the train has started rolling properly. We have grown as a company and have now added a couple of extra cars and brought a lot of expertise into the company. I feel that during the second quarter we have really got our organization in place. We now have a large project organization that has matured and works more independently.
– During the quarter, we were also able to tick off the first major milestone as a listed company, by completing the production of the API that we will use in our studies.
– We are now entering the next phase and warming up for the clinical studies. Here we are working on the final design of the phase IIb study while evaluating which clinical CRO we will work with in the study.
– In other words, we are proceeding completely according to plan.
You are currently conducting a three-month toxicology study with XEN-D0501. When do you expect your results from it and what needs to be done before your planned Phase II study can be initiated?
– The toxicological study will continue throughout the autumn until the end of the year. The study itself will run for three months, but then there is also a whole host of different things that need to be done afterwards before we have a result.
– As for the clinical part, it is, as I mentioned earlier, about finalizing the study plan and reaching an agreement with a contract research company to carry out the study itself.
Given your new focus on erythromelalgia, how do you view the financial situation in the company?
– We have sufficient funding to cover the current activities with the preclinical study. In order to prepare the application for and then conduct the phase IIb study and to enter erythromelalgia, we will need to raise more money. Conditions have not been the best on the stock market this spring, but things have started to brighten a bit recently. The board plans to arrange funding in time to enable the next step in the development plan.
You can read more about Pila Pharma here.