- Imsidolimab has successfully completed two global Phase 3
studies in Generalized Pustular Psoriasis
- Vanda expects to immediately begin preparing BLA and MAA
applications for the US and EU
- Anaptys to receive $15 million
from Vanda, comprised of a $10
million upfront payment and $5
million for existing drug supply
- Anaptys to receive a 10% royalty on global net sales of
imsidolimab
WASHINGTON and SAN
DIEGO, Feb. 3, 2025 /PRNewswire/ -- Vanda
Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) and AnaptysBio, Inc.
(Anaptys) (Nasdaq: ANAB) today announced an exclusive, global
license agreement for the development and commercialization of
imsidolimab (IL-36R antagonist mAb), which has successfully
completed two registration-enabling global Phase 3 trials, GEMINI-1
and GEMINI-2, evaluating the safety and efficacy of imsidolimab in
patients with Generalized Pustular Psoriasis (GPP).
GPP is a rare skin disorder often caused by mutations in the
IL36RN gene that codes for a regulatory protein that balances the
activity of the proinflammatory IL-36 family of cytokines.
Dysregulation of this balance in IL-36 signaling leads to severe
chronic skin inflammation with pustules and systemic symptoms
which carry significant morbidity and mortality often associated
with sepsis and multi-organ failure.1
Imsidolimab inhibits the function of the IL-36R, compensating
for the deficiency of the endogenous IL-36 regulator in patients
with GPP. Imsidolimab has successfully concluded its development
program in GPP, including the GEMINI-1 and GEMINI-2 global Phase 3
studies.
In 2025, Vanda intends to initiate and complete the technology
transfer activities and will immediately begin preparing the BLA
and MAA applications for the US and EU and making preparations for
commercialization.
"We are excited to add imsidolimab to Vanda's product portfolio
for rare orphan disorders, as well as explore the potential of this
IL-36 signal regulator in the treatment of additional inflammatory
conditions where the IL-36 homeostatic balance is dysregulated,"
said Mihael H. Polymeropoulos, M.D.,
Vanda's President, CEO and Chairman of the Board. "Imsidolimab has
great synergy with our commercial portfolio, leveraging both our
rare disease expertise in the US and EU as well as the
anti-inflammatory portfolio that includes Ponvory® for
multiple sclerosis, psoriasis and ulcerative colitis."
GPP prevalence estimates in the general population vary
considerably, between 1.76 and 124 patients per million persons
worldwide.2 The majority of GPP cases are caused by
genetic variants in IL36RN.3,4 Loss-of-function
mutations in IL36RN are mostly missense mutations in a
recessive pattern and result in unrestricted IL-36
activity.5,6
"GPP is a severely debilitating, life-threatening skin disease
in need of novel therapeutic approaches," said Johann Gudjonsson, M.D., Ph.D., Arthur C. Curtis
Professor of Molecular Skin Immunology and Scholar of the Taubman
Medical Research Institute, University of
Michigan. "The positive Phase 3 data, demonstrating GPP
patients achieved rapid disease clearance through Week 4 after a
single dose of infused imsidolimab, and maintained clear to almost
clear skin for at least 24 weeks, with no clinically meaningful
safety signals, represents a promising new option for patients
living with this disease. I'm excited imsidolimab is progressing
toward a regulatory filing this year."
"Vanda is an ideal partner for imsidolimab due to their strong
regulatory and commercial capabilities in the US and Europe, evidenced by successful recent
launches in specialty and rare diseases, and their commitment to
invest in label expansion across their therapeutic portfolio,
including their growing presence in inflammatory disease," said
Daniel Faga, president and chief
executive officer of Anaptys. "Following our productive pre-BLA
meeting with FDA in 2024, we look forward to Vanda's BLA and MAA
submissions later in 2025, with the hope that this potentially
differentiated therapeutic option will be made available for
patients living with GPP, a burdensome, and sometimes
life-threatening skin disease."
Under the terms of the agreement, Vanda will make to Anaptys an
upfront payment of $10 million and a
$5 million payment for existing drug
supply. Anaptys is also eligible to receive up to $35 million for future regulatory approval and
sales milestones in addition to a 10% royalty on net sales. Vanda
will receive an exclusive global license to develop, manufacture
and commercialize imsidolimab.
Guggenheim Securities acted as financial advisor and Fenwick
& West LLP served as legal counsel to Anaptys on this
transaction. Cantor Fitzgerald & Co. acted as financial advisor
and Orrick, Herrington & Sutcliffe LLP served as legal counsel
to Vanda.
About GEMINI-1 and GEMINI-2 Studies
In the 45-patient GEMINI-1 Phase 3 trial, patients were
randomized 1:1:1 to receive a single infusion of 750mg intravenous
(IV) imsidolimab, 300mg IV imsidolimab or placebo at Day 0. Of the
patients who received a single dose of 750mg IV imsidolimab, 53%
achieved a GPP Physician Global Assessment (GPPPGA) score of 0/1
(clear or almost clear skin) at Week 4 (primary endpoint), compared
to 13% of the patients on placebo (p=0.0131). Of the patients who
received a single dose of 300mg IV imsidolimab, 53% achieved GPPPGA
0/1 at Week 4.
Sixteen GPPPGA 0/1 responder patients from GEMINI-1 were
subsequently re-randomized to monthly maintenance dosing of either
200mg subcutaneous (SC) imsidolimab or placebo in the GEMINI-2
Phase 3 trial. Patients were followed for at least 24 weeks and up
to a maximum of 92 weeks. Of the eight responding patients from
GEMINI-1 who were re-randomized to monthly 200mg SC imsidolimab
maintenance therapy, 100% maintained a GPPPGA score of 0/1 and none
of them experienced a flare. Of the remaining eight responding
patients from GEMINI-1 who were re-randomized to placebo, 25%
maintained a GPPPGA score of 0/1 and 63% experienced a flare.
Data from both trials demonstrated a consistent, favorable
safety and tolerability profile with no treatment-related serious
adverse events (SAEs) or SAEs leading to discontinuation reported
in imsidolimab-treated patients.
About Imsidolimab and GPP
Imsidolimab is a fully humanized IgG4 antibody that inhibits the
function of the interleukin-36-receptor, or IL-36R, that is being
developed for the treatment of GPP. Regulatory and patent
exclusivity is expected to extend into the late 2030s.
GPP is a rare, chronic, systemic autoinflammatory disease that
is potentially life-threatening, if left untreated.
During a GPP flare, individuals experience the sudden eruption
of painful pustules. These pustules appear over large areas of the
skin, accompanied by redness, severe itchiness, and dry, cracked,
or scaly skin. People with GPP may also experience more general
symptoms such as fever, headache, extreme tiredness, or a burning
sensation on the skin.
About Vanda Pharmaceuticals, Inc.
Vanda is a leading global biopharmaceutical company focused on
the development and commercialization of innovative therapies to
address high unmet medical needs and improve the lives of patients.
For more on Vanda Pharmaceuticals Inc., please visit
www.vandapharma.com and follow us on X @vandapharma.
About Anaptys
Anaptys is a clinical-stage biotechnology company focused on
delivering innovative immunology therapeutics for autoimmune and
inflammatory diseases. Its lead program, rosnilimab, a depleter and
agonist targeting PD-1+ T cells, is in a Phase 2b trial for the treatment of rheumatoid
arthritis and in a Phase 2 trial for the treatment of ulcerative
colitis. Other antibodies in its portfolio include ANB033, an
anti-CD122 antagonist, in a Phase 1 trial and ANB101, a BDCA2
modulator, soon to enter clinical development. Anaptys has also
discovered multiple therapeutic antibodies licensed to GSK in a
financial collaboration for immuno-oncology, including an anti-PD-1
antagonist (Jemperli (dostarlimab-gxly)) and an anti-TIM-3
antagonist (cobolimab, GSK4069889). To learn more, visit
www.AnaptysBio.com or follow us on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the "safe harbor" provisions of the Private
Securities Litigation Reform Act of 1995, including, but not
limited to: the anticipated timing of the preparation and
submission of regulatory filings in the US and EU and the
initiation and completion of technology transfer activities;
Vanda's plans with respect to its commercial launch activities; the
estimated prevalence of GPP; the commercial availability of
imsidolimab to treat patients with GPP; the potential for Anaptys
to receive any royalties or milestone payments from the Vanda
license agreement; and the potential for any patent life extension
for imsidolimab. Statements including words such as "plan,"
"intend," "continue," "expect," or "ongoing" and statements in the
future tense are forward-looking statements. These forward-looking
statements involve risks and uncertainties, as well as assumptions,
which, if they do not fully materialize or prove incorrect, could
cause its results to differ materially from those expressed or
implied by such forward-looking statements. Forward-looking
statements are subject to risks and uncertainties that may cause
each company's actual activities or results to differ significantly
from those expressed in any forward-looking statement, including
risks and uncertainties related to each company's ability to
advance its product candidates, obtain regulatory approval of and
ultimately commercialize each of its product candidates, the timing
and results of preclinical and clinical trials, each company's
ability to fund development activities and achieve development
goals, each company's ability to protect intellectual property, and
the accuracy of the of the estimates of the number of patients with
GPP worldwide, and other risks and uncertainties described under
the heading "Risk Factors" in documents each of the companies files
from time to time with the Securities and Exchange Commission.
These forward-looking statements speak only as of the date of this
press release, and each of the companies undertakes no obligation
to revise or update any forward-looking statements to reflect
events or circumstances after the date hereof.
Vanda Pharmaceuticals Contact:
Kevin Moran
Senior Vice President, Chief Financial Officer and Treasurer
202.734.3400
pr@vandapharma.com
Jim Golden / Jack Kelleher / Dan
Moore
Collected
Strategies
VANDA-CS@collectedstrategies.com
Anaptys Contact:
Nick
Montemarano
Executive Director, Investor Relations
858.732.0178
investors@anaptysbio.com
References:
- Armstrong, A. W. et al.
Generalized pustular psoriasis: A consensus statement from the
National Psoriasis Foundation. J Am Acad Dermatol 90, 727–730
(2024).
- Prinz, J. C. et al. Prevalence, comorbidities and mortality of
generalized pustular psoriasis: A literature review. Journal of the
European Academy of Dermatology and Venereology 37, 256–273
(2022).
- Marrakchi, S. et al. Interleukin-36–Receptor Antagonist
Deficiency and Generalized Pustular Psoriasis. New England Journal
of Medicine 365, 620–628 (2011).
- Sugiura, K. et al. The Majority of Generalized Pustular
Psoriasis without Psoriasis Vulgaris Is Caused by Deficiency of
Interleukin-36 Receptor Antagonist. Journal of Investigative
Dermatology 133, 2514–2521 (2013).
- Lee, C.-C., Huang, Y.-H., Chi, C.-C., Chung, W.-H. &
Chen, C.-B. Generalized pustular psoriasis: immunological
mechanisms, genetics, and emerging therapeutics. Trends Immunol 46,
74–89 (2025).
- Sachen, K. L., Arnold Greving,
C. N. & Towne, J. E. Role of IL-36 cytokines in psoriasis and
other inflammatory skin conditions. Cytokine 156, 155897
(2022).
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SOURCE Vanda Pharmaceuticals Inc.