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 at 09:00 PDT/12:00 EDT/17:00
CET via webcast (registration link below), alongside the
American Academy of Dermatology (AAD) annual meeting
ZUG, 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).
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.
Kristian 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.”
Professor 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.”
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 ~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.
Professor 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.”
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.
Jorge 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.”
R&D Day today, Sunday, March
10
MoonLake 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.
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
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.
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 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.
- 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 American College of
Rheumatology (ACR) 50 response. The trial also evaluates a number
of secondary endpoints, including improvement compared to placebo
in ACR20, complete skin clearance as measured by at least a 100%
improvement in the Psoriasis Area and Severity Index (PASI),
physical function as measured by the Health Assessment
Questionnaire-Disability Index, enthesitis as measured by the Leeds
Enthesitis Index and pain as measured by the Patients Assessment of
Arthritis Pain. Further details are available on:
https://clinicaltrials.gov/ct2/show/NCT05640245
About Hidradenitis
SuppurativaHidradenitis 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 Skin Pain (PGA Skin Pain). Further details are
available at:
https://www.clinicaltrials.gov/ct2/show/NCT05322473
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 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 clinically
relevant outcomes. In November 2023, MoonLake announced positive
top-line results from its global Phase 2 ARGO trial evaluating the
efficacy and safety of the Nanobody® sonelokimab in patients with
active PsA. 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 American
College of Rheumatology (ACR) 50 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.
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. Lancet. 2021; 397:1564-1575).
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
MoonLake
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. The terms Nanobody® and Nanobodies® are
trademarks of Ablynx, a Sanofi company.
Cautionary
Statement Regarding
Forward Looking
Statements
This 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 clinical trials and
research and development programs; including the initiation of
clinical programs in new indications; anticipated support from
regulatory agencies with respect to the Company’s development
plans, anticipated size and timing of enrollment for the VELA
trial, the sufficiency of data from the VELA trial to support
regulatory filings in the US and EU, the anticipated trial design
for the VELA trial and the timing of expected readouts; our
expectations regarding the potential market size for HS; the
Company’s plans with respect to the commencement of a Phase 3 trial
in PsA; and the efficacy of our products, if approved, including in
relation to other products. 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, 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 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
InvestorsMatthias Bodenstedt, CFOinfo@moonlaketx.com
MoonLake Immunotherapeutics
MediaPatricia Sousamedia@moonlaketx.com
ICR Consilium Mary-Jane
Elliott, Namrata Taak, Ashley TappTel: +44 (0) 20 3709
5700MoonLake@consilium-comms.com
i © 2023 Komodo Health, Inc. All rights reserved. Reprinted with
permission.
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