Business model & Strategy

This section is from the Year End Report for 2024 published on February 27th 2024, and thus changes to the information can have occurred since.

PILA PHARMA aspires to develop its lead candidate, XEN-D0501 as a first-in-class oral TRPV1 antagonist drug for treatment of obesity and related disorders.

The Company’s next short-term goal is to assess the safety of XEN-D0501 in people living with obesity and related disorders.

The Company aspires to clinically develop XEN-D0501 until proof-of-concept in obesity, whereafter it’s a priority to secure partnership or acquisition by a larger pharmaceutical company.

The Company is currently focused on consolidating the uniquely good safety profile of this TRPV1 antagonist, and in parallel consistently adding more evidence for a clinically meaningful effect in obesity and its associated disorders. An example of that is the preliminary very positive cardiovascular effect (reduction of ANP in man and prevention of abdominal aorta aneurysm in mice).

XEN-D0501 is currently formulated as a simple and small 4 mg tablet with a very long shelf life of up to 5 years at 25ºC. The possibility of developing new formulations is something the company is evaluating. The fact it is a small molecule allows for high optionality. This can be useful for new indications and/or in order to diversify and differentiate the portfolio with upcoming drug formulations for different diseases. Combinations with other assets is also an option.

Organisationally, the strategy has been to retain a slim organisation for maximum financial flexibility. The company works with experienced consulting specialists to secure the best clinical development process and outcome.

(Fig)
Pila Pharma currently has a pipeline with 3 projects, each evaluating the effect of XEN-D0501 in various indications.

*            Obesity / diabetes / heart failure is our primary project. A phase 2a trial is our next step to identify maximum tolerable dose and assess efficacy in body-weight and ANP is a phase 2a study.

* *         Erythromelalgia / pain (rare disease) is our secondary project where the intention is to conduct a phase 2a testing XEN-D0501 for its effect on pain in persons with erythromelalgi.

* * *       Abdominal aorta aneurism / cardiovascular disease is a project in early preclinical phase cenfilly studying  XEN-D0501 was shown to reduce on abdominal aorta aneurism growth in mice.