Full Press Release Details
announces significant improvements with Nanobody sonelokimab over 24
weeks in active psoriatic arthritis (PsA) and other important updates at its R&D Day
Switzerland, March 10, 2024 - MoonLake Immunotherapeutics ("MoonLake"; Nasdaq: MLTX), a clinical-stage biotechnology
company focused on creating next-level therapies for inflammatory diseases, today announces 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 today, Sunday, March 10 (see access details below).
24-week data from the ARGO trial in PsA
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
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.
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.
Reich, MD, PhD, Founder and Chief Scientific Officer at MoonLake, commented: "These positive results from the ARGO trial
at week 24, showing that continued treatment with sonelokimab led to significant improvements across all key outcomes, reinforce the
advantages of using a smaller biologic with albumin-binding capability to effectively inhibit IL-17F in addition to IL-17A for the treatment
of deep tissue inflammation. We are particularly grateful to the patients and investigators who participated in our Phase 2 program and
look forward to initiating our Phase 3 trials in PsA and HS this year."
Joseph F. Merola, MD, MMSc, Founding President of the Psoriasis and Psoriatic Arthritis Clinics Multicenter Advancement Network Consortium
(PPACMAN), added: "There is a vital need for new treatment options for psoriatic arthritis - a chronic, inflammatory, recurrent,
and debilitating multidomain disease that has profound impact across many aspects of patients' lives. It is highly encouraging
to see that the positive high clinical responses across joint and skin endpoints and stringent composite measures, such as minimal disease
activity, observed as early as week 12 with sonelokimab were further increased through week 24."
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.
positive regulatory status and market opportunity
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.
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
$10bn 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.
Kenneth B. Gordon, Chair of Dermatology at the Medical College of Wisconsin, commented: "The positive data that is being
generated for the Nanobody sonelokimab across chronic inflammatory indications, including HS, is raising the bar on the
level of outcomes that can be achieved for patients. Patients are waiting for new treatment options with a prolonged effect, and the
start of the Phase 3 trials is bringing hope that sonelokimab could be a promising potential option to the many patients that live and
suffer with HS, a disease that has not received the attention it deserves until recently."
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.
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.
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.
Santos da Silva, PhD, Founder and Chief Executive Officer at MoonLake, said: "The robust data that we continue to amass
with our Nanobody shows that sonelokimab has the potential to be a best-in-class product in the fast-growing inflammation
field, providing us with conviction to help even more patients by expanding into further indications beyond HS and PsA. With the positive
feedback received to date from the FDA and EMA, together with our strong financials, we are now rapidly pursuing plans to commence Phase
3 trials in both HS and PsA before the end of this year and are expanding the development pipeline with the aim of elevating care with
our next-level therapies via truly innovative clinical trials."
Day today, Sunday, March 10
will hold an R&D Day today, Sunday, March 10 alongside the AAD annual meeting. The event will take place from 09:00 - 11:30
PDT/12:00 - 14:30 EDT/17:00 - 19:30 CET at Hotel Westin Bayview, San Diego and will be webcast for virtual attendees.
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:
event will feature presentations from leading clinicians in dermatology and rheumatology. Professor Joseph Merola, Chair of Dermatology,
Professor of Dermatology, Medicine and Rheumatology, UT Southwestern Medical Center and Professor Kenneth B. Gordon, Chair of Dermatology
at the Medical College of Wisconsin, will share their perspectives on the potential of MoonLake's investigational Nanobody
sonelokimab in IL-17A and IL-17F driven inflammatory diseases.
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.
breaker presentation of the 24-week data from the MIRA trial in HS at the AAD
announced in March 2024, the 24-week data
from the Phase 2 MIRA trial with Nanobody sonelokimab in moderate to severe HS, will be presented by Professor Brian
Kirby MD, FRCPI on March 10 at 14:00 PDT/ 17:00 EDT / 22:00 CET during the late breaking research session 2 (S050) in room 20B at the
AAD Annual Meeting, taking place from March 8-12, in San Diego, California.
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.
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 American College of Rheumatology (ACR) 50 response. The trial also evaluates a number of secondary endpoints, including