PILA PHARMA AB (publ) (FN STO: PILA), a clinical stage biotech company pioneering development of an oral TRPV1-antagonist as a potential first-in-class treatment for obesity and diabetes, announces it has received regulatory approval from the European Medicines Agency, to conduct PP-CT04, a two-part, randomised, double-blind, placebo-controlled, parallel-group, 12‑week trial investigating the safety, tolerability, pharmacokinetics and efficacy of XEN-D0501 in obese participants.
Malmö, Sweden, 18 August 2026
The trial is designed to explore a within-subject dose titration approach to reach anticipated therapeutic plasma concentrations of XEN-D0501 not previously investigated.
Dose escalation will be carried out individually for each participant, beginning at 4 mg BID and increasing stepwise up to a maximum of 16 mg BID. Each dose level will be administered for 1 week, resulting in a maximum dose escalation period of 4 weeks. Once the highest tolerable dose has been reached and confirmed to be tolerable, the participant will continue treatment at that dose for an 8-week maintenance period.
The trial is divided into two parts:
Part 1 will function as a pilot safety cohort with 8 participants randomised 3:1 to XEN-D0501 (6 participants) or placebo (2 participants).
Part 2 will comprise the remaining 38 participants randomised 1:1 to XEN-D0501 (19 participants) or placebo (19 participants).
Part 2 may start once all participants in Part 1 have been dosed for at least 5 weeks and once a favourable recommendation has been given by the internal safety review committee.
The overall design of both parts is the same, but Part 1 entails more intense safety monitoring.
Part 1 will be carried out without the need for additional capital. After the successful outcome of the clinical trial application, a follow-on agreement with CTC Clinical Trial Consultants AB, Sweden for the conduct of Part 1 of PP-CT04 has been entered into today.
Chief Executive Officer Gustav H. Gram comments:
“It’s really exciting to now have regulatory approval to continue our clinical development. It’s a grand achievement of Founder/CSO Dorte X. Gram with her large virtual R&D team. They’ve worked tremendously dedicated to achieve this important milestone. As we mentioned before, higher doses and longer treatment durations are considered necessary to achieve good efficacy. Determining tolerability levels over three months in obese individuals, will therefore be an important part of our drug candidate XEN-D0501 and its continued clinical development.”