Pila Pharma

Pila Pharma AB (”Pila Pharma” or ”the Company”) announced on September 29, 2026, that the clinical trial site conducting the safety part (Part 1) of PP-CT04 has been initiated and activated, whereby recruitment of participants now commences. The initiation is the final step before participants can be enrolled and ensures that investigators and trial staff have been trained in the study protocol, that regulatory and ethics documentation is in place, and that the site’s procedures and facilities meet GCP requirements.

• The study moves from approval to execution: the start of recruitment means the obesity initiative has reached the clinical phase, whereby upcoming value drivers become increasingly results-driven.
• The study tests what has so far been missing: PP-CT04 evaluates up to four times the dose and three times the treatment duration of the Company’s latest clinical study, addressing tolerability and exposure, while Part 2 may also evaluate efficacy.
• Dose finding has relevance for the entire pipeline: an established tolerable dose is expected to facilitate continued development in type 2 diabetes, erythromelalgia and potentially other diseases, and could provide a basis for evaluating combination treatments in partnership discussions.
• The timeline is assessed to be intact: the study start is in line with our assumption of a clinical start in obesity in H2-26.

Analyst Group’s View on the Announcement

With the start of recruitment, the transition from preparation to execution enabled by the regulatory approval in August has now been realized, which is viewed as the principal implication of the announcement. Since the rights issue in summer 2025, through which the Company financed part of its obesity initiative, development has essentially consisted of opportunistic preclinical work that yielded limited results, selection of a clinical contract research organization, and regulatory preparation. These activities are necessary but do not in themselves generate clinical data. With the study now about to get underway, value creation in the case shifts to clinical results, while Pila Pharma realizes a study of the kind that has been part of the Company’s development plan since its listing in 2021.

PP-CT04 is a two-part, randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and efficacy of XEN-D0501 over twelve weeks in a total of 46 people living with obesity. Each participant is individually titrated from 4 mg up to a maximum of 16 mg twice daily, with one week of treatment per dose level. The participant then continues at their highest tolerated dose for an eight-week maintenance period. By comparison, XEN-D0501 was dosed at 4 mg twice daily for four weeks in PP-CT02, conducted in people with obesity and type 2 diabetes. That study showed a favorable safety profile as well as effects on glucose tolerance and insulin response. The Company has for several years emphasized that higher doses and a longer treatment duration are necessary for a meaningful effect on body weight, which PP-CT04 intends to test. Preclinical proof-of-concept in obesity was not obtained, as the absence of weight loss was explained by insufficient drug exposure, and the study thereby becomes the first setting where the exposure question can be answered under controlled conditions in humans. As each participant continues at their highest tolerated dose, the exposure achieved is determined by tolerability during the escalation. Part 1 is therefore not only a safety checkpoint, but the data point that determines the dose level at which the efficacy evaluation can be conducted.

Part 1 comprises eight participants and is conducted with more intensive safety monitoring. Part 2 comprises the remaining 38 participants with the same overall design and constitutes the efficacy part of the study. It may commence once all participants in Part 1 have been dosed for at least five weeks and the Company’s internal safety review committee has issued a favorable recommendation. In our latest equity research report, we assumed a clinical study start in obesity in H2-26, completion of Part 1 around the turn of the year 2026/2027, and completion of Part 2 around summer 2027. The start of recruitment is broadly in line with these assumptions, and recruitment in obesity is assessed to be less complex than in type 2 diabetes, as participants can generally be handled as healthy volunteers in a study context.

The value of the dose-finding component is further assessed to extend beyond the indication the study formally concerns. The dose, safety, and pharmacokinetic information generated is largely tied to the compound rather than to the indication. A favorable outcome in Part 1 is therefore expected to facilitate the planned Phase IIa study in type 2 diabetes, PP-CT03, as well as development in erythromelalgia, where the Company holds orphan drug designation from the FDA. Tolerability may differ between patient populations, meaning an established dose level cannot automatically be transferred between indications. Even with that reservation, Part 1 is a value driver for the development portfolio as a whole and for the data package underpinning the partnership agreement totaling USD 310m that Analyst Group estimates to be concluded in 2028.

In summary, the start of recruitment means that Pila Pharma has moved from regulatory approval to clinical execution in obesity. PP-CT04 tests XEN-D0501 for the first time at substantially higher doses and at longer and clinically relevant treatment durations, thereby addressing the tolerability question left unanswered in PP-CT02, the prior four-week clinical trial, which tested XEN-D0501 at 4 mg twice daily in people with obesity and type 2 diabetes. At the same time, the dose-finding component of Part 1 is expected to lay the foundation for continued clinical development in type 2 diabetes and erythromelalgia, and potentially additional indications.