Pila Pharma

Pila Pharma AB (”Pila Pharma” or ”the Company”) announced on 16 April 2026 that the systemic exposure of XEN-D0501 in the obese rats in the Company’s preclinical studies was very low, and that this plausibly explains the lack of effect on body weight reported in January 2026. The Company states that at these low exposure levels it is not possible to draw any conclusions about the effect of XEN-D0501 on body weight or other parameters. Pila Pharma announces that it intends to proceed as planned towards a clinical trial in obesity using the Company’s current tablet formulation, for which good exposure has already been demonstrated in humans, and that the study is expected to evaluate higher doses and longer treatment duration than applied in the preclinical studies.

• Exposure data confirmed: the absence of weight loss effects reported in January 2026 was plausibly attributable to insufficient drug exposure resulting from the change in formulation, not to lack of efficacy. This reduces the uncertainty surrounding the development plan.
• The Company characterises the outcome as a false negative result and plans to continue clinical development in obesity, which is now assessed to proceed at lower risk than would have been the case had exposure in the obese rats been adequate, something that we argue is a positive from a valuation perspective.
• The planned clinical study in obesity is expected to evaluate higher doses and longer treatment duration using a formulation in which good human exposure has already been demonstrated, thereby reducing exposure risk.
• Pila Pharma is assessed to be funded to conduct a clinical Phase Ib/IIa study in obesity.

Analyst Group’s View on the Announcement

The confirmation that the rats in the preclinical study had low exposure to XEN-D0501 is assessed to reduce the uncertainty surrounding Pila Pharma’s obesity programme. The central question following the preliminary study results in January 2026 was whether the absence of weight loss in the obese rats was attributable to a lack of efficacy of XEN-D0501 or to insufficient drug exposure resulting from the change in formulation, with the latter now having been confirmed. This means that clinical development can proceed with a lower risk profile compared to a scenario in which the preclinical study had shown no effect despite adequate exposure, which is positive for the valuation of the Company. The planned clinical study is expected to use a tablet formulation with previously documented good human exposure through the Company’s proprietary formulation, which reduces the exposure risk in that study.

Background to the News – Previously Conducted Preclinical Studies

The background to today’s news is that Pila Pharma initiated preclinical studies in collaboration with CRO Gubra in December 2025, with the aim of achieving preclinical proof-of-concept for XEN-D0501 in obese rats. Ahead of study initiation, the formulation was changed from the one previously applied successfully in the Company’s 13-week toxicology study to a formulation well established at Gubra, as the obese rats did not tolerate the original formulation, which introduced an exposure uncertainty. In January 2026, Pila Pharma reported that the studies had been completed without any effect on body weight, while exposure data remained outstanding. The Company simultaneously announced that it had entered into an agreement with a new clinical CRO to prepare an application for a clinical trial in obesity, indicating a continued commitment to clinical development regardless of the preclinical study outcome.

The preclinical studies did not, however, yield the proof-of-concept that was the original objective of the programme, namely to demonstrate an effect on body weight in an established animal model prior to advancing to clinical phase. Given that exposure was insufficient, this preclinical proof-of-concept has not been obtained, and Pila Pharma is now advancing directly to clinical development without this underpinning. Analyst Group assesses that this represents a somewhat elevated risk profile compared to a scenario in which preclinical efficacy had been confirmed, but considers the low exposure of XEN-D0501 to be positive on balance in the current situation, in that the central uncertainty regarding efficacy potential remains open rather than having been refuted.

The Clinical Path Forward – Multiple Options Available

Regarding the clinical path forward, Analyst Group estimates the cost of a clinical Phase Ib/IIa study in obesity at approximately SEK 17m. With a combined cash position of approximately SEK 19.3m at the end of December 2025 and approximately SEK 5.4m added from the TO2 warrants in February 2026, the Company is assessed to have sufficient capital to initiate and complete a clinical study in obesity. A parallel acceleration that also encompasses a study in type 2 diabetes, which is the development plan estimated in Analyst Group’s most recent equity research report, is however assessed to require additional external capital.

Conducting a study in obesity alone is therefore expected to offer the advantage of achieving further clinical development within existing capital, whereas a parallel development of obesity and type 2 diabetes would accelerate the overall clinical programme and potentially more rapidly create a compelling data package for a prospective partner, albeit requiring additional external capital to execute. Analyst Group expects further information on clinical development priorities and capital requirements at a future presentation or capital markets day previously announced by Pila Pharma.

In summary, today’s announcement confirms that the preclinical results presented in January 2026 constituted a false negative outcome, and that the efficacy potential of XEN-D0501 in obesity has not yet been tested under adequate exposure conditions. Analyst Group assesses the news as removing the uncertainty surrounding the obesity programme and as enabling continued clinical development at lower risk than would have been the case had the outcome been the opposite. The clinical study is expected to be conducted using the existing tablet formulation for which good human exposure is already documented, with the study planned to evaluate higher doses and longer treatment duration.