− Successful “build to buy” relationship adds second investigational therapy to Takeda’s pipeline for the potential treatment of uncontrolled celiac disease
OSAKA, Japan-Saturday 29 February 2020 [ AETOS Wire ]
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 Symptoms & Causes of Celiac Disease. National Institute of Diabetes and Digestive and Kidney Diseases. Available at: https://www.niddk.nih.gov/health-information/digestive-diseases/celiac-disease/symptoms-causes. Last updated: June 2016. Last accessed: October 2019
(BUSINESS WIRE) -- Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK)
(“Takeda”) announced that it has acquired PvP Biologics, Inc. following
the conclusion of a Phase 1 proof-of-mechanism study of investigational
medicine TAK-062 (Kuma062) for the treatment of uncontrolled celiac
disease. TAK-062 is a potential best-in-class, highly potent super
glutenase – a protein that degrades ingested gluten – that was
computationally engineered to treat celiac disease, a serious autoimmune
disease where the ingestion of gluten leads to inflammation and damage
in the small intestine. The Phase 1 study investigated TAK-062’s safety
and tolerability in both healthy volunteers and people with celiac
disease. The ability of TAK-062 to degrade ingested gluten was studied
in healthy volunteers. Takeda plans to submit data from the Phase 1
study for presentation at an upcoming medical congress.
“Many people
living with celiac disease manage their symptoms by following a
gluten-free diet, but there is no treatment for those who continue to
experience severe symptoms,” said Asit Parikh M.D., Ph.D., Head,
Gastroenterology Therapeutic Area Unit at Takeda. “PvP Biologics’ work
demonstrated that TAK-062 is a highly targeted therapy that could change
the standard of care in celiac disease. We are now applying our deep
expertise in gastrointestinal diseases to advance the clinical study of
TAK-062 and TAK-101, two programs with different modalities that have
both demonstrated clinical proof of mechanism.”
TAK-062
enzymatically digests gluten and has demonstrated enhanced catalytic
activity compared to other glutenases. It is designed to degrade the
immune-reactive parts of gluten before they exit the stomach in order to
prevent the immune response to gluten and eliminate the symptoms and
intestinal damage caused by celiac disease. Takeda is planning a Phase
2b efficacy and dose-ranging study of TAK-062 in patients with
uncontrolled disease who maintain a gluten-free diet.
Takeda
exercised its option to acquire PvP Biologics for a pre-negotiated
upfront payment as well as development and regulatory milestones
totaling up to $330 million. Takeda and PvP Biologics previously entered
into a development and option agreement, under which PvP Biologics was
responsible for conducting research and development through the Phase 1
proof-of-mechanism study of TAK-062 in exchange for funding by Takeda
related to a pre-defined development plan.
“TAK-062 was
engineered in 2015 to address the challenges seen by previous glutenases
in development for the treatment of celiac disease – their lack of
specificity for gluten and activity in the acidic conditions of the
stomach. As a result, TAK-062 demonstrated robust gluten degradation in
humans, which was anticipated based on the very potent in vitro profile
compared with other glutenases,” said Adam Simpson, president and chief
executive officer of PvP Biologics. “Takeda has been a great partner
and has the expertise, resources, and commitment to people living with
celiac disease required to lead the next stage of TAK-062 development.”
In addition to TAK-062, Takeda previously announced data from a Phase 2a study of TAK-101,
a potential first-in-class investigational therapy for the treatment of
celiac disease. In the study, TAK-101 demonstrated T-cell response
suppression, suggesting that it may induce tolerance to gluten in
patients with celiac disease by immune uptake of proprietary
nanoparticles loaded with gliadin proteins, a disease-specific antigen.
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 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
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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
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date hereof. Such forward-looking statements do not represent any
guarantee by Takeda or its management of future performance and involve
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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 statements. Takeda undertakes no
obligation to update any of the forward-looking statements contained in
this press release or any other forward-looking statements it may make,
except as required by law or stock exchange rule. Past performance is
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press release may not be indicative of, and are not an estimate,
forecast or projection of Takeda’s future results.
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 Symptoms & Causes of Celiac Disease. National Institute of Diabetes and Digestive and Kidney Diseases. Available at: https://www.niddk.nih.gov/health-information/digestive-diseases/celiac-disease/symptoms-causes. Last updated: June 2016. Last accessed: October 2019
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Contacts
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
