Pila Pharma

Pila Pharma AB (“Pila Pharma” or “the Company”) announced on 19 December that the Company has initiated preclinical studies of its drug candidate XEN-D0501 in obesity. The studies are conducted in collaboration with Gubra, a specialist in preclinical contract research services and drug development within metabolic diseases.

Analyst Group’s view on the initiation of the studies

The newly initiated preclinical obesity studies, conducted in collaboration with Gubra, are expected to be carried out in two established rat models of obesity (DIO and Zucker rats) with the objective of achieving preclinical proof-of-concept in obesity. The studies are financed through an oversubscribed rights issue completed during the summer of 2025. The DIO rat is a well-established animal model for obesity, in which normal rats are fed a high-fat diet to induce weight gain. The Zucker rat represents another established and relevant obesity model, in which a genetic mutation leads to hyperphagia, causing the animals to spontaneously develop obesity despite a standardized diet, due to chronic caloric surplus.

The primary objective of the study is to demonstrate preclinical proof-of-concept for the Company’s candidate XEN-D0501 in obese rats by evaluating effects on body weight, while additional endpoints related to various aspects of obesity and inflammation will also be assessed. The study will investigate the effects of four weeks of treatment with Pila Pharma’s TRPV1 antagonist XEN-D0501, which is expected to enable the Company to present results in early 2026, ahead of the subscription period for the Company’s TO2 warrants, which commences on 5 February.

Initially, the intention was to use the same oral formulation that had previously been successfully applied in a 13-week toxicology study in healthy rats conducted by Pila Pharma. However, it has been decided instead to use a formulation that is well established at Gubra, to ensure optimal suitability for dosing in obese rats. The decision to change formulation is expected to introduce some uncertainty regarding anticipated drug exposure, as the selected formulation has not previously been used in combination with XEN-D0501. Nevertheless, the choice has been made in line with Gubra’s recommendations and is considered the most appropriate solution for dosing in obese rats. The selected formulation may potentially result in lower bioavailability, which has been considered in the study design by administering relatively high dose levels of XEN-D0501. In addition, sampling will be conducted at the end of the studies to verify actual systemic exposure and thereby reduce pharmacokinetic uncertainty.

Following completion of preclinical studies, the ambition is to pursue two parallel development tracks in overweight patients with and without type 2 diabetes, with the aim of creating a comprehensive and commercially attractive data package. Regardless of the outcomes of the preclinical studies, Analyst Group considers it feasible for Pila Pharma to advance development within obesity into the clinical phase. Analyst Group assesses that clinical studies in obesity could receive regulatory approval, given the extensive safety data available for the candidate from previous studies, in which XEN-D0501 has been tested in more than 300 individuals historically, demonstrating a favorable safety profile.

The planned clinical studies in obesity and type 2 diabetes would comprise Phase Ib/IIa trials in individuals with obesity as well as in individuals with obesity and type 2 diabetes, with a treatment duration of three months. The studies are intended to establish the maximum tolerated dose, confirm the safety profile, and generate preliminary data on weight reduction, which may form the basis for a potential sale or licensing agreement with a larger pharmaceutical company, and position XEN-D0501 as a potential best-in-class oral treatment for obesity and obesity-related comorbidities.

In summary, Analyst Group views positively that Pila Pharma has initiated preclinical studies of XEN-D0501 in obesity, as this marks an important step toward broadening the candidate’s scope of application to a large and commercially attractive indication. The studies, conducted in well-established obesity models together with the experienced partner Gubra, aim to achieve preclinical proof-of-concept, with results expected in early 2026.

Analyst Group will return with an equity research report on Pila Pharma in January.