MoonLake Immunotherapeutics starts Phase
3 VELA program of the Nanobody® sonelokimab in
patients with moderate-to-severe hidradenitis
suppurativa
- VELA is the first Phase 3 program
in hidradenitis suppurativa to use the higher clinical response
level of HiSCR75 as the primary endpoint
- The topline primary endpoint
readout at week 16, together with data on other endpoints, is
expected as of mid-2025
- Program will evaluate sonelokimab
for a total of 52 weeks, across VELA-1 and VELA-2, at sites in the
United States and Europe, using a design informed by the landmark
Phase 2 MIRA trial
Zug, Switzerland, May 16, 2024
– MoonLake Immunotherapeutics (MoonLake; Nasdaq: MLTX), a
clinical-stage biotechnology company focused on creating next-level
therapies for inflammatory diseases, today announced that the first
patients have been screened at a U.S. trial site in its global
Phase 3 clinical program, VELA, evaluating sonelokimab, an
investigational Nanobody® designed to treat inflammatory disease,
in patients with moderate-to-severe hidradenitis suppurativa
(HS).
HS is a severely debilitating chronic skin
condition, affecting up to 4.1% of the global population. Over
time, uncontrolled and inadequately treated inflammation can result
in irreversible tissue destruction and scarring. Sonelokimab is
designed to directly target sites of inflammation by inhibiting the
IL-17A/A, IL-17A/F, and IL-17F/F dimers and to penetrate
difficult-to-reach inflamed tissues.
Following the positive results from the landmark
Phase 2 MIRA trial, the Phase 3 VELA program is expected to enroll
800 patients across VELA-1 and VELA-2. Both trials are identical in
design comparing a single 120mg dose of sonelokimab to placebo with
the higher measure of clinical response, Hidradenitis Suppurativa
Clinical Response (HiSCR) 75, as the primary endpoint reading out
at week 16. From week 16, all patients will receive the 120mg dose
of sonelokimab through to 52 weeks, followed by an open-label
extension for up to two years. The Phase 3 program will use a
protocol design consistent with the Phase 2 MIRA trial, which
identified the optimal dose of sonelokimab for HS. The topline
primary endpoint readout (week 16) from the VELA program is
expected as of mid-2025.
Kristian Reich, Founder and Chief
Scientific Officer at MoonLake commented: “With real-world
data indicating that at least two million Americans have been
diagnosed with and treated for HS, the launch of our Phase 3 VELA
program with our Nanobody® sonelokimab, using the higher clinical
measure of HiSCR75 as the primary endpoint and a straightforward,
proven study design is a landmark moment in our efforts to develop
novel treatment options for patients suffering with this
under-diagnosed and under-treated condition. We are making
significant progress in establishing clinical trial sites to enroll
800 patients, and we eagerly anticipate reporting the week 16
primary endpoint readout around mid-2025.”
Hadar Lev-Tov, MD, MAS,
Associate Professor, Director Wound
Healing Fellowship, President Hidradenitis
Suppurativa Foundation, Dr. Phillip Frost
Department of Dermatology and Cutaneous Surgery,
University of Miami, Miller School of Medicine,
added: “HS is a chronic inflammatory skin condition with a
range of debilitating symptoms including pain, malodorous drainage,
low mood and depression. With only two FDA approved biologics,
there is still an urgent need for new treatment options that treat
all patient types and lesions, with the opportunity for
inflammatory remission. The unique characteristics and mode of
action of MoonLake’s Nanobody®, sonelokimab to effectively inhibit
IL-17F in addition to IL-17A in deep tissue inflammation has to
date shown promising outcomes, highlighting the importance of this
Phase 3 program, and placing HS at the forefront of dermatological
innovation.”
Seth B Forman, MD, Principal
Investigator at CenExel-FCR added: “It is
with great enthusiasm that I am participating as an investigator in
the Phase 3 VELA program investigating the Nanobody sonelokimab for
HS, signifying a substantial advancement in addressing the critical
unmet need for more treatment options for individuals living with
HS. As a physician, I witness first-hand the immense demand for
novel treatment options for people living with HS, particularly
those that can achieve elevated response thresholds (e.g., HiSCR75
and beyond). The findings from the Phase 2 MIRA trial offer
valuable insights into what may be possible as we work with our
patients to establish more ambitious treatment goals and alleviate
the disease burden of this debilitating condition”.
The initiation of this Phase 3 program follows
the announcement in February 2024 of the successful outcome of
MoonLake’s end-of-Phase 2 interactions with the U.S. Food and Drug
Administration (FDA), as well as positive feedback from its
interactions with the European Medicines Agency (EMA), with both
regulatory bodies unanimously supporting MoonLake’s proposed
approach for advancing its Phase 3 program in HS.
Sonelokimab is not yet approved for use in any
indication.
– Ends –
About the VELA program
The Phase 3 VELA program is expected to enroll
800 patients across VELA-1 and VELA-2. Both global, randomized,
double-blind, placebo-controlled trials are identical in design
evaluating the efficacy and safety of the
Nanobody® sonelokimab, administered subcutaneously, in adult
patients with active moderate-to-severe hidradenitis suppurativa.
Similar to the design of the landmark Phase 2 MIRA trial, the
primary endpoint 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 trials will also evaluate 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 reduction of ≥4, the
proportion of patients achieving at least 50% reduction from
baseline in Numerical Rating Scale (NRS50) in the Patient’s
Global Assessment of Skin Pain (PGA Skin Pain) and complete
resolution of Draining Tunnels (DT100). Further details are
available under NCT06411379 and NCT06411899 at
ClinicalTrials.gov.
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 lead
indications, HS and psoriatic arthritis (PSA), and the Company is
pursuing other indications in dermatology and rheumatology.
For 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.
For PsA, Phase 3 initiation is anticipated in Q4
2024 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 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.
A Phase 2 trial is expected to be initiated in
2024 for palmo-plantar pustulosis (PPP), a debilitating
inflammatory skin condition affecting a significant number of
patients. In addition, a Phase 3 trial is expected to initiate in
juvenile HS, a condition that typically manifests at this early
stage of a patient’s life, and the period in which irreversible
damage and inflammatory remission is most critical.
Sonelokimab will also be assessed in
seronegative spondyloarthritis with Phase 2 trials in radiographic
and non-radiographic axial spondyloarthritis (axSpA) and PsA
expected to start in 2024. The trials are set to incorporate
innovative designs that enhance traditional clinical outcomes with
contemporary tissue and cellular imaging techniques.
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 Hidradenitis
Suppurativa
Hidradenitis suppurativa 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. 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 exceeding $10bn 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 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.
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 and timing of clinical trials, including the
topline primary endpoint readout for the Phase 3 VELA program, the
trial design and patient enrollment across the VELA-1 and VELA-2
trials, the initiation of the Phase 3 program in PsA, commencement
of clinical trials of sonelokimab in PPP, juvenile HS and
seronegative spondyloarthritis, the efficacy and safety of
sonelokimab for the treatment of HS and PsA, including in
comparison to existing standards or care or other competing
therapies, clinical trials and research and development programs
and the anticipated timing of the results from those studies and
trials. 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
InvestorsMatthias Bodenstedt, CFOinfo@moonlaketx.com
MoonLake Immunotherapeutics
MediaPatricia Sousamedia@moonlaketx.com
ICR ConsiliumMary-Jane Elliott,
Namrata Taak, Ashley TappTel: +44 (0) 20 3709
5700MoonLake@consilium-comms.com
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