− Late-breaking data presented from a Phase 2a trial at UEG
Week 2019, Barcelona, Spain, highlights potential of
investigational medicine to prevent gluten induced immune
activation in patients with celiac disease
− Takeda acquires exclusive global license to investigational
medicine CNP-101/TAK-101, an immune modifying nanoparticle designed
to induce tolerance to gluten in patients with celiac
disease
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK)
(“Takeda”) and COUR Pharmaceutical Development Company, Inc.
(“COUR”) today announced that Takeda has acquired an exclusive
global license to develop and commercialize the investigational
medicine CNP-101/TAK-101, an immune modifying nanoparticle
containing gliadin proteins. Based on COUR’s antigen specific
immune tolerance platform, TAK-101 is a potential first-in-class
treatment targeting the aberrant immune response in celiac disease,
a serious autoimmune disease where the ingestion of gluten leads to
inflammation and damage in the small intestine.
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Results of a randomized, double-blind, placebo-controlled
clinical trial to assess the markers of potential efficacy and
safety of the investigational medicine in 34 adults with proven
celiac disease was presented today as a late-breaking abstract at
UEG Week 2019, Barcelona, Spain. At inclusion, patients had
well-controlled biopsy proven celiac disease. After inclusion, they
underwent an oral gluten challenge. Based on the study, Takeda
exercised its option to acquire the exclusive global license to
TAK-101.
“While many people living with celiac disease can manage their
symptoms by following a gluten free diet, there are currently no
treatment options for those who continue to have symptoms,” said
Asit Parikh M.D., Ph.D., Head, Gastroenterology Therapeutic Area
Unit at Takeda. “Our collaboration with COUR has shown, for the
first time, that it is possible to induce specific immune tolerance
to a foreign antigen in autoimmune diseases such as celiac disease.
With our expertise in inflammatory diseases, Takeda is well
positioned to further develop TAK-101 in pursuit of providing the
first approved treatment option for patients with celiac
disease.”
In the trial, treatments were administered intravenously on day
1 and day 8. The gluten challenge began seven days after the second
treatment administration and included 12 grams of gluten per day
for three days followed by 6 grams of gluten per day for 11 days.
The primary endpoint was change from baseline in interferon-gamma
(IFN-γ) spot forming units (SFUs) at day 6 after gluten challenge
using a gliadin-specific enzyme-linked immunospot (ELISpot) assay.
This test is a direct measure of gluten-specific systemic T cell
activation in celiac disease, and blocking this response suggests
individuals with celiac disease could be protected from the effects
of gluten exposure. 34 patients were randomized and treated, 6
discontinued due to gluten related symptoms, and 28 completed the
14-day gluten challenge per protocol.
The primary endpoint of the trial was achieved with a mean
change from baseline in IFN-γ ELISpotSFUs of 2.10 and 17.57 with
TAK-101 and placebo, respectively (p=0.0056). Also seen was a trend
in protection from small intestinal mucosal damage with
deterioration of 0.18 with TAK-101 compared with 0.63 with placebo
(p=0.079). The most frequent adverse events in patients receiving
TAK-101 that exceeded the frequency seen in placebo treated
patients were nausea, headache, abdominal pain, and back pain. No
patient had clinically significant changes in vital signs, routine
clinical labs, or serum cytokines/chemokines, gliadin-specific T
cell proliferation and cytokine secretion.
Takeda intends to initiate a dose-ranging study to further
explore the potential of TAK-101 in the treatment of patients with
celiac disease on a gluten free diet to inform future
registrational trials. COUR is eligible to receive up to $420
million in future payments, and royalties on sales of any
commercialized products resulting from the license.
“We are encouraged by the data from this first human proof of
concept study of our proprietary nanoparticle platform designed to
reprogram the immune system,” said John J. Puisis, CEO of COUR
Pharmaceuticals. “As Takeda assumes responsibility for the celiac
disease program, COUR will focus on advancing our pipeline of
therapies for a variety of other immune disorders ranging from
multiple sclerosis to peanut allergy.”
COUR’s proprietary immune modifying nanoparticles bind
inflammatory cells to initiate tolerogenic immune reprogramming.
The interior core can be loaded with disease specific antigen – in
this case, gliadin proteins – to induce tolerance in autoimmune
conditions like celiac disease.
About Celiac Disease Celiac disease is a genetically
driven chronic immune-mediated disorder where abnormal immune
responses to gluten peptides lead to small intestinal mucosal
damage.1,2,3 Recent population-based studies in the U.S. indicate
that the prevalence of celiac disease is around 1%4 and
approximately 0.5% globally.5 The threshold of daily gluten that
will cause mucosal injury in both adults and children is 10 to 50mg
per day – or about 1/100th of a slice of bread.6,7 Celiac disease
can cause symptoms including abdominal pain, diarrhea, nausea, and
vomiting. Long-term complications of celiac disease may include
malnutrition, accelerated osteoporosis, nervous system problems and
problems related to reproduction. Currently the only available
treatment for patients with celiac disease is maintaining a
gluten-free diet, which involves strict, lifelong avoidance of
exposure to gluten proteins from wheat, barley, and rye, which is
not always effective.8
Takeda’s Commitment to Gastroenterology Gastrointestinal
(GI) diseases can be complex, debilitating, and life-changing.
Recognizing this unmet need, Takeda and our collaboration partners
have focused on improving the lives of patients through the
delivery of innovative medicines and dedicated patient disease
support programs for over 25 years. Takeda aspires to advance how
patients manage their disease. Additionally, Takeda is leading in
areas of gastroenterology associated with high unmet need, such as
inflammatory bowel disease, acid-related diseases, and motility
disorders. Our GI Research & Development team is also exploring
solutions in celiac disease and liver diseases, as well as
scientific advancements through microbiome therapies.
About COUR Pharmaceuticals COUR Pharmaceuticals is developing
first-in-class therapies designed to reprogram the immune system to
achieve antigen-specific tolerance for immune-mediated disease.
COUR’s platform of immune-modifying nanoparticles treats the root
cause of immune disease unlike traditional approaches, which only
minimize symptoms using toxic immune suppression. COUR’s lead
product for celiac disease, partnered with Takeda, is the first
demonstration of induction of antigen-specific immune tolerance in
any autoimmune disease. Data from clinical and preclinical settings
demonstrate the opportunity for the COUR nanoparticle platform to
address a wide range of immune and inflammatory conditions. The
underlying technology was acquired from Northwestern University and
draws from more than 30 years of research by the laboratory of
Stephen D. Miller, Ph.D., the Judy E. Guggenheim Research Professor
of Microbiology-Immunology. For more information, visit
www.courpharma.com.
About Takeda Pharmaceutical Company Limited Takeda
Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global,
values-based, R&D-driven biopharmaceutical leader headquartered
in Japan, committed to bringing Better Health and a Brighter Future
to patients by translating science into highly-innovative
medicines. Takeda focuses its R&D efforts on four therapeutic
areas: Oncology, Gastroenterology (GI), Rare Diseases and
Neuroscience. We also make targeted R&D investments in
Plasma-Derived Therapies and Vaccines. We are focusing on
developing highly innovative medicines that contribute to making a
difference in people's lives by advancing the frontier of new
treatment options and leveraging our enhanced collaborative R&D
engine and capabilities to create a robust, modality-diverse
pipeline. Our employees are committed to improving quality of life
for patients and to working with our partners in health care in
approximately 80 countries and regions.
For more information, visit https://www.takeda.com
Forward-Looking Statements This press release and any
materials distributed in connection with this press release may
contain forward-looking statements, beliefs or opinions regarding
Takeda’s future business, future position and results of
operations, including estimates, forecasts, targets and plans for
Takeda. Without limitation, forward-looking statements often
include words such as “targets”, “plans”, “believes”, “hopes”,
“continues”, “expects”, “aims”, “intends”, “ensures”, “will”,
“may”, “should”, “would”, “could” “anticipates”, “estimates”,
“projects” or similar expressions or the negative thereof.
Forward-looking statements in this document are based on Takeda’s
estimates and assumptions only as of the date hereof. Such
forward-looking statements do not represent any guarantee by Takeda
or its management of future performance and involve known and
unknown risks, uncertainties and other factors, including but not
limited to: the economic circumstances surrounding Takeda’s global
business, including general economic conditions in Japan and the
United States; competitive pressures and developments; changes to
applicable laws and regulations; the success of or failure of
product development programs; decisions of regulatory authorities
and the timing thereof; fluctuations in interest and currency
exchange rates; claims or concerns regarding the safety or efficacy
of marketed products or product candidates; the timing and impact
of post-merger integration efforts with acquired companies; and the
ability to divest assets that are not core to Takeda’s operations
and the timing of any such divestment(s), any of which may cause
Takeda’s actual results, performance, achievements or financial
position to be materially different from any future results,
performance, achievements or financial position expressed or
implied by such forward-looking statements. For more information on
these and other factors which may affect Takeda’s results,
performance, achievements, or financial position, see “Item 3. Key
Information—D. Risk Factors” in Takeda’s most recent Annual Report
on Form 20-F and Takeda’s other reports filed with the U.S.
Securities and Exchange Commission, available on Takeda’s website
at: https://www.takeda.com/investors/reports/sec-filings/ or at
www.sec.gov. Future results, performance, achievements or financial
position of Takeda could differ materially from those expressed in
or implied by the forward-looking statements. Persons receiving
this press release should not rely unduly on any forward-looking
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forward-looking statements contained in this press release or any
other forward-looking statements it may make, except as required by
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1 Jabri B, Sollid LM. T Cells in Celiac Disease. J Immunol
2017;198:3005-3014. 2 Molberg O, McAdam S, Lundin KE, et al. T
cells from celiac disease lesions recognize gliadin epitopes
deamidated in situ by endogenous tissue transglutaminase. Eur J
Immunol 2001;31:1317-23. 3 Dieterich W, Ehnis T, Bauer M, et al.
Identification of tissue transglutaminase as the autoantigen of
celiac disease. Nat Med 1997;3:797-801. 4 Leonard MM, Sapone A,
Catassi C, et al. Celiac Disease and Nonceliac Gluten Sensitivity:
A Review. JAMA 2017;318:647-656. 5 Lionetti E, Gatti S, Pulvirenti
A, et al. Celiac disease from a global perspective. Best Pract Res
Clin Gastroenterol 2015;29:365-79. 6 Might gluten traces in wheat
substitutes pose a risk in patients with celiac disease? A
population-based probabilistic approach to risk estimation. Am J
Clin Nutr 2013;97:109-16. 7 Catassi C, Fabiani E, Iacono G, et al.
A prospective, double-blind, placebo-controlled trial to establish
a safe gluten threshold for patients with celiac disease. Am J Clin
Nutr 2007;85:160-6. 8
https://www.niddk.nih.gov/health-information/digestive-diseases/celiac-disease/symptoms-causes
(accessed October 9, 2019)
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Takeda Pharmaceuticals Media in Japan Kazumi Kobayashi
+81 (0) 3-3278-2095 kazumi.kobayashi@takeda.com U.S Media Chris
Stamm +1 (617) 374-7726 chris.stamm@takeda.com European Media Luke
Willats +41 44-555-1145 luke.willats@takeda.com COUR
Pharmaceuticals Canale Communications Mari Purpura +1 (619)
849-5384 mari@canalecomm.com
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