- Ipsen obtains exclusive options to Spherical Nucleic Acids
(SNAs™) currently under discovery evaluation for Huntington’s
disease and Angelman syndrome
- Exicure will be responsible for discovery and certain
pre-clinical development activities. In the event Ipsen exercises
its option to the two programs, Ipsen will be responsible for
further development and worldwide commercialization
- Exicure will receive a $20m upfront payment and is eligible to
receive up to $1B in option exercise fees and milestone payments
should Ipsen opt into both programs, as well as tiered
royalties
Regulatory News:
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Ipsen (Euronext: IPN; ADR: IPSEY) and Exicure Inc. (NASDAQ:
XCUR) have signed an exclusive collaboration agreement to research,
develop, and commercialize novel Spherical Nucleic Acids (SNAs) as
potential investigational treatments for Huntington’s disease and
Angelman syndrome.
Oligonucleotides are synthetic structures of nucleic acids that
can be used to modulate gene expression via a range of processes,
including gene activation, inhibition, and splice-modulation. These
molecules have demonstrated potential in many different therapeutic
areas.1 Achieving efficient oligonucleotide delivery to target
organs and tissues, including the brain, remains a major limitation
to their use.1,2 Exicure’s SNAs provide distinct chemical and
biochemical properties to oligonucleotides. In preclinical models,
SNAs have been shown to enhance the cell penetration,
biodistribution and organ persistence properties of
oligonucleotides,3,4 which may potentially enhance drug delivery to
previously inaccessible target tissues, including deep brain
regions.5,6
Philippe Lopes-Fernandes, Chief Business Officer at Ipsen, said
“Neuroscience is deeply rooted within Ipsen as a key strategic
driver for our business. We are pleased to partner with Exicure to
progress development of investigational treatment options for
Huntington’s disease and Angelman syndrome, two areas of
significant unmet need. This collaboration marks an important step
in maximizing the potential of this novel technology, bringing
together the expertise of Exicure and the robust heritage of Ipsen
in neuroscience. With this new collaboration we will deepen our
commitment to people living with neurological conditions around the
world.”
“We are thrilled to partner with Ipsen, a leading global company
with significant expertise and commitment to developing treatments
for patients with rare neurological diseases,” said David
Giljohann, Ph.D., Chief Executive Officer, Exicure, Inc. “In
collaboration with Ipsen, we have the opportunity to apply our
technology to Huntington's disease and Angelman syndrome, both
indications requiring deep brain penetration and technological
advances to reach previously hard-to-drug targets. We believe our
platform technology with its deep penetration and persistence of
medicinal effect will allow Exicure and Ipsen to overcome
challenges from first-generation oligonucleotides and bring new
medicines to patients in need."
Under the agreement, Ipsen will receive exclusive options to
license SNA-based therapeutics arising from two collaboration
programs for Huntington’s disease and Angelman syndrome. Ipsen will
pay Exicure a cash upfront payment of $20m upon closing and Exicure
will be responsible for discovery and certain pre-clinical
development activities. In the event Ipsen exercises its option,
Ipsen will be responsible for further development and
commercialization of the licensed products. Exicure will receive a
$20m upfront payment and is eligible to receive up to $1B in option
exercise fees and milestone payments should Ipsen opt into both
programs, as well as tiered royalties.
ENDS
Huntington’s disease
Huntington’s disease (HD) is a progressive, fatal
neurodegenerative disorder and the most common monogenic
neurological disorder in the developed world, affecting about
40,000 individuals in the US.7 HD is caused by an expanded CAG
trinucleotide repetition in the huntingtin (HTT) gene in chromosome
4. HD is characterized by involuntary movements, psychiatric
disorders, cognitive deterioration, and early mortality, with death
often occurring within 10 to 20 years after motor symptoms appear.
Mean age of onset of motor symptoms is around 40 years of age, with
longer CAG repeats causing earlier disease onset. 8 There is
currently no approved therapy to address the underlying molecular
cause of HD to slow or stop disease progression.9
Angelman syndrome
Angelman syndrome (AS) is a severe neurodevelopmental disorder.
The prevalence of Angelman syndrome is estimated to be 1 in
12,000-20,000 people in the general population.10 The disorder is
characterized by severe intellectual deficit, speech impairment,
epilepsy, ataxic movements and behavioral abnormalities. AS results
from loss of function of the maternally inherited copy of the
ubiquitin-protein ligase E3A (UBE3A) gene on chromosome 15.11
Disruption of UBE3A function in neurons prevents synapse formation
and remodeling, leading to significant neurodevelopmental
disability. There is currently no approved disease-modifying
therapy for AS and standard-of-care treatment is supportive, such
as medications for seizures and behavioral abnormalities.12
Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused
on transformative medicines in Oncology, Rare Disease and
Neuroscience; it also has a well-established Consumer Healthcare
business. With Total Sales of over €2.5bn in FY 2020, Ipsen sells
more than 20 medicines in over 115 countries, with a direct
commercial presence in more than 30 countries. The Company’s
research and development efforts are focused on its innovative and
differentiated technological platforms located in the heart of
leading biotechnological and life-science hubs: Paris-Saclay,
France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has
c.5,700 colleagues worldwide and is listed in Paris (Euronext: IPN)
and in the U.S. through a Sponsored Level I American Depositary
Receipt program (ADR: IPSEY). For more information, visit
ipsen.com.
Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company
developing therapeutics for neurology, immuno-oncology,
inflammatory diseases, and other genetic disorders based on its
proprietary Spherical Nucleic Acid, or SNA technology. Exicure
believes that its proprietary SNA architecture has distinct
chemical and biological properties that may provide advantages over
other nucleic acid therapeutics and may have therapeutic potential
to target diseases not typically addressed with other nucleic acid
therapeutics. Exicure is in preclinical development of XCUR-FXN a
lipid-nanoparticle SNA–based therapeutic candidate, for the
intrathecal treatment of Friedreich’s ataxia (FA). Exicure’s
therapeutic candidate cavrotolimod (AST-008) is in a Phase 1b/2
clinical trial in patients with advanced solid tumors. Exicure is
based in Chicago, IL and in Cambridge, MA.
Ipsen’s forward-looking statements
The forward-looking statements, objectives and targets contained
herein are based on Ipsen’s management strategy, current views and
assumptions. Such statements involve known and unknown risks and
uncertainties that may cause actual results, performance or events
to differ materially from those anticipated herein. All of the
above risks could affect Ipsen’s future ability to achieve its
financial targets, which were set assuming reasonable macroeconomic
conditions based on the information available today. Use of the
words ‘believes’, ‘anticipates’ and ‘expects’ and similar
expressions are intended to identify forward-looking statements,
including Ipsen’s expectations regarding future events, including
regulatory filings and determinations. Moreover, the targets
described in this document were prepared without taking into
account external growth assumptions and potential future
acquisitions, which may alter these parameters. These objectives
are based on data and assumptions regarded as reasonable by Ipsen.
These targets depend on conditions or facts likely to happen in the
future, and not exclusively on historical data. Actual results may
depart significantly from these targets given the occurrence of
certain risks and uncertainties, notably the fact that a promising
product in early development phase or clinical trial may end up
never being launched on the market or reaching its commercial
targets, notably for regulatory or competition reasons. Ipsen must
face or might face competition from generic products that might
translate into a loss of market share. Furthermore, the Research
and Development process involves several stages each of which
involves the substantial risk that Ipsen may fail to achieve its
objectives and be forced to abandon its efforts with regards to a
product in which it has invested significant sums. Therefore, Ipsen
cannot be certain that favorable results obtained during
pre-clinical trials will be confirmed subsequently during clinical
trials, or that the results of clinical trials will be sufficient
to demonstrate the safe and effective nature of the product
concerned. There can be no guarantees a product will receive the
necessary regulatory approvals or that the product will prove to be
commercially successful. If underlying assumptions prove inaccurate
or risks or uncertainties materialize, actual results may differ
materially from those set forth in the forward-looking statements.
Other risks and uncertainties include but are not limited to,
general industry conditions and competition; general economic
factors, including interest rate and currency exchange rate
fluctuations; the impact of pharmaceutical industry regulation and
health care legislation; global trends toward health care cost
containment; technological advances, new products and patents
attained by competitors; challenges inherent in new product
development, including obtaining regulatory approval; Ipsen's
ability to accurately predict future market conditions;
manufacturing difficulties or delays; financial instability of
international economies and sovereign risk; dependence on the
effectiveness of Ipsen’s patents and other protections for
innovative products; and the exposure to litigation, including
patent litigation, and/or regulatory actions. Ipsen also depends on
third parties to develop and market some of its products which
could potentially generate substantial royalties; these partners
could behave in such ways which could cause damage to Ipsen’s
activities and financial results. Ipsen cannot be certain that its
partners will fulfil their obligations. It might be unable to
obtain any benefit from those agreements. A default by any of
Ipsen’s partners could generate lower revenues than expected. Such
situations could have a negative impact on Ipsen’s business,
financial position or performance. Ipsen expressly disclaims any
obligation or undertaking to update or revise any forward-looking
statements, targets or estimates contained in this press release to
reflect any change in events, conditions, assumptions or
circumstances on which any such statements are based, unless so
required by applicable law. Ipsen’s business is subject to the risk
factors outlined in its registration documents filed with the
French Autorité des Marchés Financiers. The risks and uncertainties
set out are not exhaustive and the reader is advised to refer to
Ipsen’s 2020 Registration Document, available on ipsen.com.
Exicure’s forward-looking statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements in this press release other than statements of
historical fact could be deemed forward looking including, but not
limited to, statements regarding the company’s exclusive
collaboration with Ipsen; the ability of SNAs to potentially
enhance drug delivery to previously inaccessible target tissues and
other benefits of SNAs including as potential treatment options for
Huntington’s disease and Angelman syndrome; the ability of the
company’s technology to overcome challenges from first-generation
oligonucleotides and bring new drugs to patients in need; the
ability of the company to realize contingent milestone payments and
royalties under the collaboration agreement with Ipsen; and the
advancement, timing and success of the company’s preclinical and
clinical programs. The forward-looking statements in this press
release speak only as of the date of this press release, and the
company undertakes no obligation to update these forward-looking
statements. Forward-looking statements are based on management’s
current beliefs and assumptions that are subject to risks and
uncertainties and are not guarantees of future performance. Actual
results could differ materially from those contained in any
forward-looking statement as a result of various factors,
including, without limitation: the risks that the ongoing COVID-19
pandemic may disrupt the company’s business and/or the global
healthcare system more severely than it has to date or more
severely than anticipated; unexpected costs, charges or expenses
that reduce the company’s capital resources; the company’s
preclinical or clinical programs do not advance or result in
approved products on a timely or cost effective basis or at all;
the results of early clinical trials are not always being
predictive of future results; the cost, timing and results of
clinical trials; that many drug candidates do not become approved
drugs on a timely or cost effective basis or at all; the ability to
enroll patients in clinical trials; possible safety and efficacy
concerns; the ability of the company to collaborate successfully
with strategic partners; regulatory developments; exposure to
litigation, including patent litigation, and/or regulatory actions;
and the ability of the company to protect its intellectual property
rights. For a discussion of other risks and uncertainties, and
other important factors, any of which could cause the company’s
actual results to differ from those contained in the
forward-looking statements, see the section titled “Risk Factors”
in the company’s Annual Report on Form 10-K for the year ended
December 31, 2020, as updated by the company’s subsequent filings
with the Securities and Exchange Commission. All information in
this press release is as of the date of the release, and the
company undertakes no duty to update this information, except as
required by law.
References
1 Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide
drug delivery. Nature Reviews Drug Discovery 2020;19:673–694.
Available here: https://www.nature.com/articles/s41573-020-0075-7 2
Crawford L, Rosch J, Putnam D. Concepts, technologies, and
practices for drug delivery past the blood–brain barrier to the
central nervous system. Journal of Controlled Release. 2016;240:
251-266. Available here:
https://www.sciencedirect.com/science/article/pii/S0168365915302923?via%3Dihub#bb0020
3
https://investors.exicuretx.com/news/news-details/2019/Exicure-Announces-Preclinical-Data-Supporting-Development-of-SNA-Technology-in-the-Central-Nervous-System/default.aspx
4
https://s1.q4cdn.com/907903764/files/doc_news/archive/b984683d-76f4-4759-9add-d2c65150ebb6.pdf
5 Rosi NL, Giljohann DA, Thaxton S, et al. Oligonucleotide-modified
gold nanoparticles for intracellular gene regulation. Science
2006;312(5776):1027–1030. Available here:
https://science.sciencemag.org/content/312/5776/1027?ijkey=8fbfd37b2763498a2658cc0dfaa5dc15df0e81a0&keytype2=tf_ipsecsha
6
https://www.news-medical.net/life-sciences/What-is-an-Oligonucleotide.aspx
7 Yohrling et al., Huntington Study Group 2019 Annual Meeting 8
Bates et al., Nature Reviews Disease Primers, 2015 9 Tabrizi et
al., Nature Reviews Neurology, 2020 Available here:
https://www.nature.com/articles/s41582-020-0389-4?proof=t 10 NORD.
https://rarediseases.org/rare-diseases/angelman-syndrome/ 11
Buiting et al., Nature Reviews Neurology, 2016; US Census Data 12
NIH, National Institute of Neurological Disorders and Stroke,
accessed on July 27, 2021 Available here:
https://www.ninds.nih.gov/Disorders/All-Disorders/Angelman-Syndrome-Information-Page
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Ipsen Investors
Craig Marks Vice President, Investor Relations +44 7584 349
193
Adrien Dupin de Saint-Cyr Investor Relations Manager +33
6 64 26 17 49
Media Jess Smith
Senior Director, Global Communications, R&D and Business
Development +44 7557 267 634
Exicure contact Karen Sharma +1 781-235-3060
ksharma@macbiocom.com
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