- New trials in
three new indications have been initiated with the Nanobody®
sonelokimab: Phase 3 VELA-TEEN trial in adolescent hidradenitis
suppurativa (HS), Phase 2 LEDA trial in palmoplantar pustulosis
(PPP) and Phase 2 S-OLARIS trial in axial spondyloarthritis
(axSpA)
- MoonLake now
independently running seven trials in 2025 across large dermatology
and rheumatology indications: adult HS, adolescent HS, psoriatic
arthritis (PsA), PPP and axSpA
- Phase 2 and
Phase 3 data read-outs across all target indications expected in
2025 (adult HS, and PPP) and 2026 (adolescent HS, PsA and
axSpA)
- Data from the Phase 3 VELA-TEEN
trial may be combined with the Phase 3 VELA program in adults with
moderate-to-severe HS to support the first Biologics License
Application (BLA) for the Nanobody® sonelokimab
Zug, Switzerland, January 8,
2025 – MoonLake Immunotherapeutics (MoonLake; Nasdaq: MLTX), a
clinical-stage biotechnology company focused on creating next-level
therapies for inflammatory diseases, today announced that patients
have been screened in three new trials across three new indications
to evaluate sonelokimab, an investigational Nanobody® designed to
treat inflammatory disease. The clinical program for sonelokimab
now includes patients with adolescent hidradenitis suppurativa
(HS), palmoplantar pustulosis (PPP) and axial spondyloarthritis
(axSpA), in addition to adult patients with HS and active psoriatic
arthritis (PsA). This increases the number of clinical trials led
by MoonLake to ten and aligns with the plan to enroll over 3,000
patients in clinical trials with sonelokimab since the Company was
founded in 2021.
Evidence indicates that activation of IL-17A and
IL-17F plays a crucial role in the pathophysiology of these
inflammatory diseases. Sonelokimab is designed to directly target
sites of inflammation by inhibiting all relevant IL-17 dimers,
namely the IL-17A/A, IL-17A/F, and IL-17F/F dimers, and to
penetrate difficult-to-reach inflamed tissues. The initiation of
the Phase 3 VELA-TEEN trial in HS, the Phase 2 LEDA trial in PPP
and the Phase 2 S-OLARIS trial in axSpA expands the portfolio of
sonelokimab to large dermatology and rheumatology indications,
where significant unmet needs remain.
Dr. Jorge Santos da Silva, Founder and
Chief Executive Officer of MoonLake Immunotherapeutics,
said: “The start of three new trials across three new
dermatology and rheumatology indications underscores MoonLake’s
remarkable and rapid progress in independently realizing our
ambitious plans. 2024 has been a year of execution, and 2025 is
anticipated to mark a transformational and data-rich year as we
look forward to notably reporting key Phase 3 data for HS, a market
projected to reach $15bn by 2035. We are progressing towards
registration and commercialization with a robust cash position. A
huge thanks is due to our talented team for expanding
possibilities, and of course, to all the patients and healthcare
professionals for their participation in our trials.”
Prof. Kristian Reich, Founder and Chief
Scientific Officer at MoonLake commented: “Building upon
our ongoing Phase 3 studies in HS and PsA, we are further
leveraging the molecular advantages of our Nanobody®, sonelokimab,
in adolescent HS, PPP, and axSpA, all diseases characterized by
activation of the IL-17 and IL-17F pathway in difficult-to-reach
tissues. We are pioneering a paradigm shift in innovative trial
design, which has given us the agility to rapidly execute these new
clinical trials.”
Building upon the Phase 2 MIRA trial and ongoing
Phase 3 VELA trials in adult HS, the Phase 2 ARGO trial and the
ongoing Phase 3 IZAR trials in active PsA, the three new trials
are:
Phase 3 VELA-TEEN: the first
dedicated clinical trial in adolescent patients with
moderate-to-severe hidradenitis suppurativa (HS)
- An open-label, single-arm trial
with the primary endpoint to evaluate the pharmacokinetics, safety,
and tolerability of the 120mg subcutaneous (SC) dose of sonelokimab
over 24 weeks in 30-40 adolescents, aged 12-17, with
moderate-to-severe HS, from U.S. sites with experience in pediatric
dermatology.
- HS is a chronic, debilitating skin
condition affecting an estimated 2% of the population. It often
first manifests during adolescence, and adolescent patients are
particularly physically and emotionally affected by the disease.
Early intervention is therefore crucial to prevent disease
progression and reduce the long-term physical and emotional impact.
- Data from the VELA-TEEN trial may
be combined with data from the Phase 3 VELA program in adults with
moderate-to-severe HS to support a single Biologics License
Application (BLA).
- Topline data for the primary and
secondary endpoints, including the higher clinical response level
of HiSCR75, are anticipated in 2026.
Amy S. Paller, M.D, Walter J. Hamlin
Professor and Chair of Dermatology and Professor of Pediatrics at
Northwestern University Feinberg School of Medicine, and Principal
Investigator of the trial commented: “Adolescents with HS
face substantial unmet medical needs and are a critically
underserved patient population. The VELA-TEEN trial marks a
significant advancement in HS treatment. By prioritizing early
intervention, we hope to alter the disease course, reduce tissue
destruction, and prevent permanent damage.”
Phase 2 LEDA: the first
clinical trial in palmoplantar pustulosis (PPP) for an IL-17A and
IL-17F inhibitor
- The trial aims to enroll
approximately 30 patients across multiple sites to evaluate the
120mg SC dose of sonelokimab. The primary endpoint will be percent
change from baseline in Palmoplantar Psoriasis Area and Severity
Index (ppPASI) with important secondary endpoints including
ppPASI75 (at least 75% improvement in the ppPASI).
- A biomarker-controlled clinical
trial.
- The topline primary endpoint
readout is expected during 2025.
Mark Lebwohl, MD, Dean for Clinical
Therapeutics, Icahn School of Medicine at Mount Sinai, and Chairman
Emeritus, Kimberly and Eric J. Waldman Department of Dermatology
commented: “Palmoplantar pustulosis is a chronic,
recurrent inflammatory condition affecting the palms and soles,
marked by aggressive pustule eruptions, skin destruction, and
severe pain. This ongoing skin damage is debilitating,
significantly impacting patients’ daily lives and activities.
Despite the severity, advanced therapies remain scarce. It is
exciting to see studies with innovative technologies like
nanobodies to address this urgent medical need.”
Phase 2 S-OLARIS: the first
trial for an IL-17A and IL-17F inhibitor using an innovative design
combining traditional clinical outcomes for axial spondyloarthritis
(axSpA) with PET/MRI imaging
- An open-label trial to evaluate a
60mg SC dose of sonelokimab in approximately 25 patients with
active axSpA. The primary endpoint is the change from baseline
(CfB) at week 12 in the uptake of 18F-NaF in the sacroiliac joints
and spine using PET in combination with MRI imaging. By combining
established clinical measures with innovative PET imaging
techniques and a biomarker program, the trial will for the first
time measure sonelokimab's impact on deep tissue inflammation in
unprecedented detail.
- axSpA is a chronic inflammatory
condition that primarily affects the spine and sacroiliac joints,
with a global prevalence ranging between 0.5% and 1.5%. Up to 40%
of patients either do not respond to or cannot tolerate currently
available treatments.
- The topline primary endpoint
readout is expected in early 2026.
Professor Xenofon Baraliakos,
Rheumazentrum Ruhrgebiet Herne, Ruhr-University Bochum, Germany
commented: “Despite advancements in axial
spondyloarthritis (axSpA) treatment over the past two decades,
innovation remains crucial. Many patients experience inadequate
responses to current therapies, and we need to continue to push the
boundaries and explore novel approaches that can address not only
inflammation across several domains but potentially modify the
underlying disease processes. Integrating cutting-edge imaging
techniques like MRI-PET with clinical outcomes, as being used in
the S-OLARIS trial, offers a promising path forward. I am excited
to see such a promising innovation like nanobodies, and
specifically sonelokimab, being developed for axSpA. Our goal must
be to develop more effective treatments that can improve long-term
outcomes, reduce disease progression, and enhance the quality of
life for all axSpA patients."
The Company also plans to initiate a Phase 2
trial, P-OLARIS, in patients with active PsA using an innovative
design combining traditional clinical outcomes with PET/MRI
imaging.
- Ends -
About the VELA-TEEN trialThe
Phase 3 VELA-TEEN trial is an open-label, single-arm trial designed
to evaluate sonelokimab 120mg administered subcutaneously once
every two weeks (Q2W) until week six and once every four weeks
(Q4W) from week eight onwards. The trial aims to enroll 30-40
adolescents, aged 12-17, with moderate-to-severe hidradenitis
suppurativa (HS), from U.S. sites experienced in clinical trials
and pediatric dermatology. The primary trial phase will be 24 weeks
with a primary endpoint evaluating the pharmacokinetics, safety,
and tolerability of sonelokimab. VELA-TEEN will also evaluate
several secondary endpoints, including the proportion of patients
achieving the higher clinical response measure of the Hidradenitis
Suppurativa Clinical Response Score (HiSCR) 75, in addition to
HiSCR50. Other outcomes are the change from baseline in the
International Hidradenitis Suppurativa Severity Score System
(IHS4), which includes the quantitative measure of draining
tunnels, and the proportion of patients achieving a meaningful
reduction of the Children’s Dermatology Life Quality Index (CDLQI)
and the Patients Global Assessment of Skin Pain (PGA Skin
Pain).
About Hidradenitis
SuppurativaHS 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 an estimated 2% of
the population, with three times more females affected than males.
Real-world data in the US indicates that at least 2 million unique
patients have been diagnosed with and treated for HS between 2016
and 2023 alone, highlighting a significant unmet need and impact on
healthcare systems, and a market opportunity projected to reach
$15bn by 2035. 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 S-OLARIS
trialS-OLARIS is an open-label Phase 2 proof-of-concept
trial aiming to investigate sonelokimab 60mg administered
subcutaneously in approximately 25 patients with active axial
spondylarthritis (axSpA). The primary endpoint is the change from
baseline (CfB) at week 12 in the uptake of 18F-NaF in the
sacroiliac joints and spine using PET in combination with MRI
imaging. Throughout the trial, several other endpoints will be
assessed including established clinical disease activity outcomes
(e.g., ASAS), scores related to physical function, spinal mobility,
and enthesitis as well as patient reported outcomes. The trial also
includes an exploratory peripheral blood and tissue biomarker
program.
About active axial
spondyloarthritisAxSpA typically impacts young people,
with diagnosis based on chronic inflammatory back pain lasting more
than three months with onset under 45 years of age. Advanced
disease can lead to progressive and pathologic bone formation and
joint fusion, severely limiting spinal mobility. Global reported
prevalence of axSpA ranges from 0.5% to 1.5%. AxSpA can be
categorized by disease progression into two subtypes:
non-radiographic axSpA and ankylosing spondylitis (AS), also known
as radiographic axSpA, which is diagnosed based on radiographic
evidence of structural changes to the sacroiliac joints. Patients
with axSpA experience fatigue, persistent morning stiffness, and
pain that worsens at night and can disrupt sleep. Many patients
also face the burden of comorbidities such as psoriatic arthritis
and psoriasis. Studies have found elevated IL-17 levels in the
blood and synovial fluid of patients with axSpA, and IL-17A and
IL-17F are both thought to be key contributors to pathogenesis
across the spondyloarthropathies.
About the LEDA TrialThe LEDA
trial is a Phase 2 trial designed to evaluate the efficacy and
safety of sonelokimab 120mg administered subcutaneously in adult
patients with palmoplantar pustulosis (PPP). The primary endpoint
of the trial is percent change from baseline in Palmoplantar
Psoriasis Area and Severity Index (ppPASI) with important secondary
endpoints including ppPASI75 (at least 75% improvement in the
ppPASI). The LEDA trial features an innovative translational
research program using peripheral blood and tissue biomarkers as
trial controls.
The trial design has been informed by previous
successful studies of sonelokimab, including the landmark Phase 2
MIRA trial in hidradenitis suppurativa, which identified the
optimal dosing and demonstrated the potential of sonelokimab to
target deep tissue inflammation effectively.
About Palmoplantar
PustulosisPPP is characterized by the development of
blister-like pustules within erythematous, scaly plaques on the
palms and the soles of the feet. PPP typically develops in
adulthood, more frequently impacts females. Patients frequently
experience significant pain, burning, and itching sensations on the
palms and soles of the feet which can be debilitating and impair
their ability to work, sleep, or perform other activities of daily
living. Currently, the treatment of PPP is challenging with a
significant unmet need for novel therapies to reduce the symptom
burden for patients. Evidence suggests that activation of the IL-17
pathway has an important role in disease pathophysiology.
About SonelokimabSonelokimab
(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 lead
indications, HS and psoriatic arthritis (PsA), and the Company is
pursuing other indications in dermatology and rheumatology,
including adolescent HS, PPP and axSpA.
For adults with HS, sonelokimab is being
assessed in the Phase 3 trials, VELA-1 and VELA-2, following the
successful outcome of MoonLake’s end-of-Phase 2 interactions with
the FDA and as well as positive feedback from its interactions with
the EMA announced in February 2024. In June 2023, topline results
of the MIRA trial (NCT05322473) at 12 weeks showed that the trial
met its primary endpoint, the Hidradenitis Suppurativa Clinical
Response (HiSCR)75, which is a higher measure of clinical response
versus the HiSCR50 measure used in other clinical trials, setting a
landmark milestone. In October 2023, the full dataset from the MIRA
trial at 24 weeks showed that maintenance treatment with
sonelokimab led to further improvements in HiSCR75 response rates
and other high threshold clinical and patient relevant outcomes.
The safety profile of sonelokimab in the MIRA trial was consistent
with previous trials with no new safety signals detected.
A Phase 3 trial, VELA-TEEN, is also ongoing in
adolescent HS, a disease that typically begins at this early stage
of a patient’s life, and is also the period in which irreversible
damage and inflammatory remission are most critical.
For PsA, sonelokimab is being assessed in the
Phase 3 trials, IZAR-1 and IZAR-2, following the announcement in
March 2024 of the full dataset from the global Phase 2 ARGO trial
(M1095-PSA-201) evaluating the efficacy and safety of the Nanobody®
sonelokimab over 24 weeks in patients with active PsA. Significant
improvements were observed across all key outcomes, including
approximately 60% of patients treated with sonelokimab achieving an
American College of Rheumatology (ACR) 50 response and Minimal
Disease Activity (MDA) at week 24. This followed the positive
top-line results in November 2023, where the trial met its primary
endpoint with a statistically significant greater proportion of
patients treated with either sonelokimab 60mg or 120mg (with
induction) achieving an ACR50 response compared to those on placebo
at week 12. All key secondary endpoints in the trial were met for
the 60mg and 120mg doses with induction. The safety profile of
sonelokimab in the ARGO trial was consistent with previous trials
with no new safety signals detected.
Sonelokimab is also being assessed in the Phase
2 LEDA trial, which is ongoing for palmo-plantar pustulosis (PPP),
a debilitating inflammatory skin condition affecting a significant
number of patients.
Additionally, Sonelokimab is being assessed in
the ongoing Phase 2 S-OLARIS trial, for active axSpA. The trial
features an innovative design complementing traditional clinical
outcomes with cellular imaging techniques.
Sonelokimab has also been assessed in a
randomized, placebo-controlled third-party 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. Lancet. 2021; 397:1564-1575).
In an earlier third-party 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
ImmunotherapeuticsMoonLake Immunotherapeutics is a
clinical-stage biopharmaceutical company unlocking the potential of
sonelokimab, a novel investigational Nanobody® for the treatment of
inflammatory disease, to revolutionize outcomes for patients.
Sonelokimab inhibits IL-17A and IL-17F by inhibiting the IL-17A/A,
IL-17A/F, and IL-17F/F dimers that drive inflammation. The
company’s focus is on inflammatory diseases with a major unmet
need, including hidradenitis suppurativa and psoriatic arthritis –
conditions affecting millions of people worldwide with a large need
for improved treatment options. MoonLake was founded in 2021 and is
headquartered in Zug, Switzerland. Further information is available
at www.moonlaketx.com.
Cautionary Statement Regarding Forward
Looking StatementsThis press release contains certain
“forward-looking statements” within the meaning of the U.S. Private
Securities Litigation Reform Act of 1995. Forward-looking
statements include, but are not limited to, statements regarding
MoonLake’s expectations, hopes, beliefs, intentions or strategies
regarding the future including, without limitation, statements
regarding: plans for and timing of clinical trials, including
timing of topline results of the Phase 3 VELA-TEEN clinical trial
of sonelokimab in adolescent patients with HS, Phase 2 LEDA
clinical trial of sonelokimab in patients with PPP, Phase 2
S-OLARIS trial in patients with axSpA, Phase 3 VELA trials in adult
HS and Phase 3 IZAR trials in active PsA and initiation of Phase 2
P-OLARIS trial in patients with active PsA, the efficacy and safety
of sonelokimab for the treatment of adult HS, adolescent HS, PPP,
PsA and axSpA, including in comparison to existing standards or
care or other competing therapies, clinical trials and research and
development programs, including the combination of data from the
VELA-TEEN trial in adolescents with data from the Phase 3 VELA
program in adults with moderate to severe HS to support a single
BLA, the anticipated timing of the results from those studies and
trials and potential market opportunities for sonelokimab and
MoonLake’s anticipated cash position. In addition, any statements
that refer to projections, forecasts, or other characterizations of
future events or circumstances, including any underlying
assumptions, are forward looking statements. The words
“anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,”
“intend,” “may,” “might,” “plan,” “possible,” “potential,”
“predict,” “project,” “should,” “would” and similar expressions may
identify forward-looking statements, but the absence of these words
does not mean that statement is not forward looking.
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, state and federal healthcare reform
measures that could result in reduced demand for MoonLake’s product
candidates and reliance on third parties to conduct and support its
preclinical studies and 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 December 31, 2023 and
subsequent filings with the Securities and Exchange Commission.
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.
MoonLake Immunotherapeutics Media &
Investors RelationsCarla Bretes, Director IR &
External Communicationsir@moonlaketx.com
ICR Healthcare Mary-Jane
Elliott, Namrata Taak, Ashley TappTel: +44 (0) 20 3709
5700MoonLake@ICRHealthcare.com
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