MoonLake announces significant improvements with Nanobody® sonelokimab over 24 weeks in active psoriatic arthritis (PsA) and other important updates at its R&D Day
- Positive 24-week data from the ARGO trial of sonelokimab in PsA:
- Significant improvements observed across all key outcomes, including approximately 60% of patients treated with sonelokimab achieving an ACR50 response at week 24
- Unprecedented multi-domain responses across joints, skin and other domains, including up to 52% of patients achieving ACR50+PASI100 and up to 61% of patients achieving Minimal Disease Activity (MDA), supporting potential best-in-class profile of sonelokimab
- Monthly maintenance with 60mg or 120mg doses showed leading responses above TNF reference arm across all key outcomes including in higher treatment goals (ACR70, PASI100, composites) – 120mg added benefit for specific patient subgroups
- Low discontinuation rate around 5% and safety profile of sonelokimab consistent with previously reported studies with no new safety signals
- Update on sonelokimab in hidradenitis suppurativa (HS):
- Following interactions with the FDA and EMA, MoonLake intends to commence Phase 3 trials in HS in Q2 2024, under the VELA program; the program is expected to enroll 800 patients and reflect a similar protocol design to that used in the MIRA Phase 2 trial, with top-line primary endpoint data expected as early as mid-2025
- Real-world data indicates that at least 2 million Americans have been diagnosed with HS as of 2023, highlighting a significant unmet need and impact on healthcare systems, and a market opportunity exceeding
$10bn by 2035
- MoonLake further announces that it will imminently commence four additional clinical trials of sonelokimab across dermatology, and rheumatology, including innovative trials in palmo-plantar pustulosis, juvenile HS and seronegative spondyloarthritis
- The Company is hosting an R&D Day on
Sunday, March 10 at09:00 PDT /12:00 EDT /17:00 CET via webcast (registration link below), alongside theAmerican Academy of Dermatology (AAD) annual meeting
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
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.
Professor
The 24-week results build upon the 12-week results announced in
HS positive regulatory status and market opportunity
MoonLake has recently announced the successful outcome of its end-of-Phase 2 interactions with the
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 ~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
Professor
New indications
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.
R&D Day today,
MoonLake will hold an R&D Day today,
The R&D Day will highlight the 24-week ARGO data, discuss regulatory interactions and paths to Phase 3, and other important business updates from MoonLake’s executive team including:
- Analysis of the HS and PSA market opportunities and leadership potential for sonelokimab.
- Pipeline updates and details of additional catalysts for 2024 and 2025, including new indications to be pursued.
- Summary and financials.
The event will feature presentations from leading clinicians in dermatology and rheumatology. Professor
A live Q&A session involving all presenters will follow the event. Register to attend either the in-person event or webcast here. A recording and additional details will be available on the Events & Presentations section of the Company’s website at www.ir.moonlaketx.com.
Late breaker presentation of the 24-week data from the MIRA trial in HS at the AAD
As announced in
- Ends -
About Psoriatic Arthritis
Psoriatic arthritis (PsA) is a chronic and progressive inflammatory arthritis associated with psoriasis primarily affecting the peripheral joints. The clinical features of PsA are diverse, involving pain, swelling, and stiffness of the joints, which can result in restricted mobility and fatigue. PsA occurs in up to 30% of patients with psoriasis, most commonly those aged between 30 and 60 years. The symptom burden of PsA can have a substantial negative impact on patient quality of life. Although the exact mechanism of disease is not fully understood, evidence suggests that activation of the IL-17 pathway plays an important role in the disease pathophysiology.
About the ARGO trial
The ARGO trial (M1095-PSA-201) is a global, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of the Nanobody® sonelokimab, administered subcutaneously, in the treatment of adult patients with active PsA. The trial is designed to evaluate different doses of sonelokimab, with placebo control and adalimumab as an active reference arm. The primary endpoint of the trial is the percentage of participants achieving ≥50% improvement in signs and symptoms of disease from baseline, compared to placebo, as measured by the
About Hidradenitis Suppurativa
Hidradenitis suppurativa (HS) is a severely debilitating chronic skin condition resulting in irreversible tissue destruction. HS manifests as painful inflammatory skin lesions, typically around the armpits, groin, and buttocks. Over time, uncontrolled and inadequately treated inflammation can result in irreversible tissue destruction and scarring. The disease affects 0.05–4.1% of the global population, with three times more females affected than males. Onset typically occurs in early adulthood and HS has a profound negative impact on quality of life, with a higher morbidity than other dermatologic conditions. There is increasing scientific evidence to support IL-17A- and IL-17F-mediated inflammation as a key driver of the pathogenesis of HS, with other identified risk factors including genetics, cigarette smoking, and obesity.
About the MIRA trial
The MIRA trial (M1095-HS-201) is a global, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of the Nanobody® sonelokimab, administered subcutaneously, in the treatment of adult patients with active moderate-to-severe hidradenitis suppurativa. The trial recruited 234 patients, with the aim to evaluate two different doses of sonelokimab (120mg and 240mg) with placebo control and adalimumab as an active reference arm. The primary endpoint of the trial is the percentage of participants achieving Hidradenitis Suppurativa Clinical Response 75 (HiSCR75), defined as a ≥75% reduction in total abscess and inflammatory nodule (AN) count with no increase in abscess or draining tunnel count relative to baseline. The trial also evaluated a number of secondary endpoints, including the proportion of patients achieving HiSCR50, the change from baseline in International Hidradenitis Suppurativa Severity Score System (IHS4), the proportion of patients achieving a Dermatology Life Quality Index (DLQI) total score of ≤5, and the proportion of patients achieving at least 30% reduction from baseline in Numerical Rating Scale (NRS30) in the Patient’s Global Assessment of
About Sonelokimab
Sonelokimab (M1095) is an investigational ~40 kDa humanized Nanobody® consisting of three VHH domains covalently linked by flexible glycine-serine spacers. With two domains, sonelokimab selectively binds with high affinity to IL-17A and IL-17F, thereby inhibiting the IL-17A/A, IL-17A/F, and IL-17F/F dimers. A third central domain binds to human albumin, facilitating further enrichment of sonelokimab at sites of inflammatory edema.
Sonelokimab is being assessed in two trials, the Phase 2 ARGO trial in PsA and the Phase 2 MIRA trial in HS. In
Sonelokimab has also been assessed in a randomized, placebo-controlled Phase 2b trial (NCT03384745) in 313 patients with moderate-to-severe plaque-type psoriasis. High threshold clinical responses (Investigator’s Global Assessment Score 0 or 1, and Psoriasis Area and Severity Index 90/100) were observed in patients with moderate-to-severe plaque-type psoriasis. Sonelokimab was generally well tolerated, with a safety profile similar to the active control, secukinumab (Papp KA, et al.
In an earlier Phase 1 trial in patients with moderate-to-severe plaque-type psoriasis, sonelokimab has been shown to decrease (to normal skin levels) the cutaneous gene expression of pro-inflammatory cytokines and chemokines (Svecova D. J Am Acad Dermatol. 2019;81:196–203).
About Nanobodies®
Nanobodies® represent a new generation of antibody-derived targeted therapies. They consist of one or more domains based on the small antigen-binding variable regions of heavy-chain-only antibodies (VHH). Nanobodies® have a number of potential advantages over traditional antibodies, including their small size, enhanced tissue penetration, resistance to temperature changes, ease of manufacturing, and their ability to be designed into multivalent therapeutic molecules with bespoke target combinations.
The terms Nanobody® and Nanobodies® are trademarks of Ablynx, a Sanofi company.
About MoonLake Immunotherapeutics
Cautionary Statement Regarding Forward Looking Statements
This press release contains certain “forward-looking statements” within the meaning of the
Forward-looking statements are based on current expectations and assumptions that, while considered reasonable by MoonLake and its management, as the case may be, are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with MoonLake’s business in general and limited operating history, difficulty enrolling patients in clinical trials, and reliance on third parties to conduct and support its clinical trials, and the other risks described in or incorporated by reference into MoonLake’s Annual Report on Form 10-K for the year ended
Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements in this press release, which speak only as of the date they are made and are qualified in their entirety by reference to the cautionary statements herein. MoonLake does not undertake or accept any duty to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or in the events, conditions or circumstances on which any such statement is based.
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