MoonLake Immunotherapeutics announced that continued treatment with Nanobody sonelokimab led to significant improvements across all key outcomes at 24-week data from the ARGO trial in psoriatic arthritis (PsA) and other important R&D updates. These updates will be presented and discussed in detail at the Company?s R&D Day to be held on March 10, 2024. Positive 24-week data from the ARGO trial in PsA: The ARGO trial, which involved 207 patients with active PsA, demonstrated that the primary endpoint, the American College of Rheumatology (ACR) 50, continued to improve from week 12 and exceeded 60% by week 24.

The more rigorous ACR70 outcome was achieved by approximately 40% of patients by week 24. In addition, by week 24, over 80% and 60% of patients treated with sonelokimab achieved Psoriasis Area Severity Index (PASI) 90 and 100, respectively. Both doses of sonelokimab yielded similar results.

The responses surpassed those for adalimumab, the active reference arm in the study, and were also higher when indirectly compared to competitors using the same active reference arm as a standard. Treatment with sonelokimab resulted in unprecedented improvements in composite scores that reflect responses in different domains simultaneously. ACR50+PASI90 up to 59%, ACR 50+PASI 100 up to 52%, ACR 70+PASI 100 up to 48% and MDA up to 61% response.

In all composite scores, sonelokimab showed 16-29 percentage point differences to the reference adalimumab arm, comparatively higher to competitors using the same reference arm. While the 60mg dose was found to be sufficient to reach high levels of response in the general trial population, the 120mg dose was found to improve responses further in specific patient sub-groups, which suggests two doses being carried over to Phase 3. The safety profile of sonelokimab was consistent with previous trials with no new safety signals detected. The discontinuation rate of the second part of ARGO remained low at 5%, in line with other sonelokimab trials.

Overall, sonelokimab continues to show a favorable safety profile. Across the sonelokimab clinical program to date, the company has not seen any signal of suicide ideation/behavior (SI/B) or liver enzyme elevations related to sonelokimab treatment. The 24-week results build upon the 12-week results announced in November 2023.

Full results from the ARGO trial will be submitted for publication in a peer-reviewed medical journal. Sonelokimab is not yet approved for use in any indication. HS positive regulatory status and market opportunity: MoonLake has recently announced the successful outcome of its end-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA) the E.U. European Medicines Agency (EMA), with both regulatory bodies supporting MoonLake?s proposed approach for advancing its Phase 3 program of the Nanobody sonelokimab in hidradenitis suppurativa (HS).

During the R&D Day, the Company will provide further details on trial design, expectations for the single 120mg dose being tested and timelines for this program, named VELA which is set to enroll 800 patients. Furthermore, the Company will share findings from a recent analysis of US real-world data pertaining to the HS market.i It revealed that between 2016 and 2023, two million unique patients were diagnosed and treated for HS, with an average of 240,000 new patients each year as per claims. This corresponds to a approximately 1% prevalence of diagnosed and treated patients, aligning well with estimates that over 2% of the population, including those undiagnosed and untreated, have HS. These real-world data also substantiate a potential market size exceeding $10 billion by 2035.

Notably there is a low penetration of current biologics (around 3%) and a high dropout rate from treatment with current biologics within the first year (median of 11 months). Moreover, claims show that HS patients are lost in their treatment journey (e.g., more than 50-60% of patients are on long term on antibiotics and many of them are also on steroids /opioids, and 15% of patients receive surgery in year 1) representing a bleak prognosis for patients, physicians, and healthcare systems. This real-world perspective substantiates the company?s market size estimates and highlights the need for more effective therapies. MoonLake further announces that it will imminently commence four additional development programs, across dermatology and rheumatology where IL-17A and IL17-F inhibition in deep tissues has the opportunity to lead among all therapies.

In dermatology, Phase 2 work is expected to be initiated in palmo-plantar pustulosis (PPP), a debilitating disease affecting a significant number of patients (estimated 0.3% prevalence) and for which there are no currently approved therapies. This new indication will strengthen MoonLake?s standing within the dermatology community. Furthermore, MoonLake expects to initiate a Phase 3 trial in juvenile HS a disease that typically begins at this early stage of a patient?s life, and also the period in which irreversible damage and inflammatory remission is most critical.

It is anticipated that this trial will run concurrently with MoonLake?s adult Phase 3 program, marking the first time clinical trial evidence is generated specifically for this demographic. In rheumatology, MoonLake will also extend its development work in seronegative spondyloarthritis. Phase 2 work in radiographic and non-radiographic axial spondyloarthritis (axSpA) is expected to start this year, with trials featuring an innovative design complementing traditional clinical outcomes with modern imaging techniques, adding two new indications to the pipeline.

The Company plans to also run an additional trial in PsA, to link the impact of sonelokimab in traditional clinical outcomes (e.g., ACR50) with objective imaging measurements in different domains. The new axSpA and PsA studies are designed to employ cutting-edge MRI-PET imaging.