Pila Pharma

Pila Pharma AB (”Pila Pharma” or ”the Company”) announced on August 18, 2026, that it has received regulatory approval to conduct PP-CT04, a two-part, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics, and efficacy of XEN-D0501 in people living with obesity over 12 weeks. The study comprises a total of 46 participants and applies an individual dose escalation up to a maximum of 16 mg twice daily. Part 1 constitutes an initial safety cohort of eight participants with more intensive monitoring, while Part 2 comprises the remaining 38 participants and may commence following a favorable recommendation regarding the safety observed in Part 1. The Company states that Part 1 can be conducted without additional capital and has, in connection with the approval, entered into an agreement with Clinical Trial Consultants AB regarding the conduct of this part.

• Clinical development in obesity can now be initiated: the approval represents the gate that had to be passed before value-creating clinical data can be generated, and development thereby shifts from preparation to execution.
• The study tests what has so far been missing: higher doses and a longer treatment duration than XEN-D0501 has previously been evaluated at, in a setting where dose and exposure can be controlled.
• The dose-finding component is assessed to have relevance beyond the obesity track: an established tolerable dose over three months of treatment is expected to facilitate continued development in type 2 diabetes and erythromelalgia as well as in further indications.
• The capital requirement remains for the efficacy part: Part 1 is covered by existing funds, while Part 2 and the parallel clinical studies are assessed to require additional external financing.

Analyst Group’s View on the Announcement

With an approval in place, Pila Pharma moves from a period characterized by preparation to actual execution, which Analyst Group views as the principal implication of the announcement. Since the summer of 2025, when the Company financed its obesity initiative through a substantially oversubscribed rights issue, development has essentially consisted of preclinical work, selection of contract research organizations, and regulatory preparation, activities that are necessary but that do not in themselves generate clinical data. The approval is the gate that had to be passed for continued value creation to be driven by actual study results, and it simultaneously confirms Analyst Group’s earlier assessment that the extensive safety documentation for XEN-D0501 would enable a clinical study. Analyst Group therefore assesses that the announcement moves the Company from a phase in which risk has primarily been regulatory and preparatory to one in which upcoming value drivers are clinical.

The Study Tests Higher Doses and a Longer Treatment Duration Than Previously

The analytically most material aspect of the study design is that it addresses the question that has previously remained unanswered throughout the development program. XEN-D0501 has to date been evaluated at 4 mg twice daily for up to one month, whereas PP-CT04 titrates up to 16 mg twice daily over a total of twelve weeks. The Company has for several years emphasized that both higher doses and a longer treatment duration are assessed to be necessary in order to achieve a meaningful effect on body weight, and Analyst Group therefore views the newly approved study as the step that genuinely tests that hypothesis. At the same time, it should be noted that each participant continues at their highest tolerated dose, meaning that the exposure achieved is determined by tolerability during the escalation period. The outcome of Part 1 thus becomes not only a safety checkpoint but the data point that determines the dose level at which the subsequent efficacy evaluation can be conducted.

The Dose-Finding Component is Assessed to Have Relevance Beyond the Obesity Track

Analyst Group assesses that the value of the dose-finding part extends beyond the indication it formally concerns. The approval obtained relates to obesity, and the previously planned Phase IIa study in obesity and type 2 diabetes constitutes a separate trial requiring its own approval. The dose, safety, and pharmacokinetic information generated is, however, largely tied to the compound rather than to the indication, and a favorable outcome in Part 1 is therefore expected to facilitate both that application and the planned study in erythromelalgia, where the Company holds orphan drug designation from the FDA. Analyst Group notes at the same time that tolerability may differ between patient populations, meaning that an established dose level cannot automatically be transferred between indications. Even with that reservation, Part 1 is viewed as a value driver for the development portfolio as a whole, which strengthens the conditions for the broader data package underpinning future partnering discussions.

Financing and the Pace of Continued Development

Part 1 corresponds to the study the Company has previously communicated that existing funds are sufficient to finance, while Part 2 and the parallel clinical studies depend on additional financing. This is consistent with the view Analyst Group has conveyed since the outcome of the series TO2 warrants, where proceeds of approximately SEK 5.4m were assessed to enable an initial clinical study in obesity but not a broader clinical acceleration. Analyst Group expects Part 1 to be completed around the turn of the year 2026/2027, after which the efficacy part and the parallel studies can be initiated as financing is secured. Conducting the initial part beforehand is at the same time assessed to be advantageous, as operational risk is reduced ahead of a future capital requirement. In terms of Analyst Group’s estimates, the approval is in line with the pace of development that has been assumed, and the announcement is therefore assessed primarily to affect the risk level of the development plan rather than its timing.

In summary, the approval represents an important step for Pila Pharma, as clinical development in obesity can now be initiated and upcoming value drivers become to a greater extent results-driven. The study addresses the central question of higher doses and a longer treatment duration, while the dose-finding component is expected to create the conditions for continued development across more indications than obesity.