MoonLake Immunotherapeutics Announces
Positive Regulatory Feedback from both FDA and EMA on Path for the
Phase 3 Program for the Nanobody®
sonelokimab in Psoriatic Arthritis
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Builds upon the positive regulatory feedback received from FDA and
EMA on Phase 3 VELA program for sonelokimab in hidradenitis
suppurativa (HS), which has started recruitment
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Clarified path for PsA Phase 3 program with study design, patient
population and endpoints agreed – program is called IZAR and two
trials are planned with one focusing on bio-naïve patients and
radiographic progression (IZAR-1) and another on TNF-IR patients
(IZAR-2)
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The main dose to be tested will be 60mg, and the 120mg dose will
also be tested, with risankizumab as a reference arm (in
IZAR-2)
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Total Phase 3 population of approximately 1,500 patients to be
complemented by Phase 2 population for registration
-
Phase 3 trial design in line with the Company’s communications and
guidance
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First patient expected to be randomized in Q4 2024; primary
endpoint readout expected end-2026
ZUG, Switzerland, June 10, 2024
– MoonLake Immunotherapeutics (NASDAQ:MLTX) (“MoonLake”), a
clinical-stage biotechnology company focused on creating next-level
therapies for inflammatory diseases, today announced the successful
outcome of its end-of-Phase 2 interactions with the U.S. Food and
Drug Administration (FDA), as well as positive feedback from its
interactions with the E.U. European Medicines Agency (EMA), with
both regulatory bodies unanimously supporting MoonLake’s proposed
approach for advancing its Phase 3 program of the Nanobody®
sonelokimab in psoriatic arthritis (PsA).
The Phase 3 program, named IZAR, is expected to
enroll around 1,500 patients and in combination with data from the
Phase 2 ARGO trial is designed to support both a Biologics License
Application (BLA) and E.U. Marketing Authorization Application. Two
global, randomized, double blind, placebo-controlled trials are
planned (IZAR-1 and IZAR-2) to evaluate the efficacy and safety of
the Nanobody® sonelokimab over one year. IZAR-1 will enroll a
biologic naïve population and include an evaluation of radiographic
progression, while IZAR-2 will enroll a TNF-IR population and will
be the first trial to include a risankizumab active reference arm.
The IZAR program will assess a 60mg sonelokimab dose as well as a
120mg sonelokimab dose. The primary endpoint (ACR50) compared to
placebo and key secondary endpoints for both trials will read out
at week 16. The readout of the primary endpoint is anticipated at
the end of 2026.
Dr. Jorge Santos da Silva, Chief
Executive Officer of MoonLake Immunotherapeutics, said:
“The recent regulatory milestones for PsA mark the second such
significant achievement for MoonLake building on the positive FDA
and EMA feedback for HS earlier this year. These regulatory
outcomes provide a clear roadmap for PsA and, by the end of 2024,
we will have independently commenced three Phase 3 programs
addressing two inflammatory indications that are under diagnosed
and underserved. This progress is a testament to our specialized
expertise and steadfast commitment to advancing the field of
inflammation and immunology.”
- 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 IZAR
IZAR-1 and IZAR-2 are planned global,
randomized, double-blind, placebo-controlled Phase 3 trials that
will be designed to evaluate the efficacy and safety of sonelokimab
compared with placebo in a total of approximately 1,500 adults with
active PsA, with a primary endpoint of superiority to placebo in
ACR 50 response at Week 16. IZAR-1 will enroll a biologic-naïve
population and include an evaluation of radiographic progression,
while IZAR-2 will enroll a TNF-experienced population — reflecting
patients commonly seen in clinical practice — and will be the first
PsA trial to include a risankizumab active reference arm. Both
trials will also include a range of secondary endpoints reflecting
the multiple disease manifestations characteristic of PsA,
including skin and nail outcomes, multidomain endpoints, and
patient-reported outcome measures such as pain and quality of life
assessments.
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-17-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 IL17F/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 IZAR program, the
trial design and patient enrollment across the IZAR-1 and IZAR-2
trials, the initiation of the Phase 3 program in PsA, 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 MediaPatricia
Sousamedia@moonlaketx.com
ICR ConsiliumMary-Jane Elliott, Namrata Taak,
Ashley TappTel: +44 (0) 20 3709
5700MoonLake@consilium-comms.com
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