MoonLake Immunotherapeutics (MLTX) R&D Day 2025 summary

Pipeline overview and program updates

• VELA-1 and VELA-2 phase 3 trials evaluated sonelokimab in adults with moderate to severe hidradenitis suppurativa (HS), enrolling over 800 patients with identical designs and endpoints, and over 90% completion at week 16.

• Sonelokimab demonstrated a differentiated profile with strong efficacy, favorable safety, and convenient dosing.

• All patients receive sonelokimab from week 16 onward in the VELA trials.

• The program includes additional ongoing studies in PPP, PsA, axSpA, and adolescent HS, with key readouts expected from late 2025 through 2026.

Clinical trial data and development milestones

• VELA-1 met all primary and secondary endpoints, while VELA-2's primary endpoint was not met under the composite strategy due to a high placebo response, but was significant under the treatment policy strategy.

• HiSCR75 response rates at week 16 were 35% (VELA-1) and 36% (VELA-2) for sonelokimab, with a 17% delta to placebo and robust statistical significance.

• Statistically significant improvements were observed in all key secondary endpoints, including HiSCR50, IHS4-55, pain reduction, HiSQOL, and DLQI, with benefits seen as early as week 4.

• Sonelokimab's safety profile remained favorable, with no new safety signals and low rates of serious adverse events.

• Efficacy continued to build beyond week 16, with strong cross-over responses and a week 52 readout planned.

R&D strategy and innovation priorities

• Two pre-specified statistical strategies (composite and treatment policy) were used in accordance with regulatory advice to ensure robustness.

• The development strategy leverages a robust pipeline in multiple inflammatory indications, with innovative trial designs and biomarker integration.

• Nanobody® technology is highlighted for its potential advantages in tissue penetration, stability, and multivalent design.