Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001, a Novel Therapeutic Candidate for the Treatment of Pompe Disease
May 10 2024 - 2:00AM
Business Wire
- Agreement Adds to Shionogi’s Growing Pipeline in Rare
Disease
- MZE001 has the Potential to be the First Oral Therapy for Pompe
Disease
Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief
Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) and
Maze Therapeutics, Inc. (Head Office: South San Francisco,
California, USA; Chief Executive Officer: Jason Coloma, Ph.D.;
hereafter “Maze”) announced the companies have completed an
exclusive worldwide license agreement for the rights to MZE001, an
investigational oral glycogen synthase 1 (GYS1) inhibitor that aims
to address Pompe disease by limiting disease-causing glycogen
buildup.
Pompe disease is a rare, inherited disorder caused by mutations
in the gene coding for acid alpha-glucosidase (GAA), which can lead
to the buildup of glycogen in skeletal muscle, respiratory muscle
and cardiac muscle tissues resulting in progressive weakness and
respiratory compromise.
Under the terms of the agreement, Shionogi has acquired
exclusive worldwide rights for MZE001 as well as related programs
and intellectual property. Shionogi will pay an upfront fee of $150
million, and Maze will be eligible for milestone payments based on
development, regulatory and commercial achievements plus tiered
royalties based upon future net sales. The required 30-day waiting
period outlined in United States Hart-Scott-Rodino (HSR) Act has
expired, and the transaction is completed.
“This agreement is a strong strategic fit for Shionogi. It will
help meaningfully advance our commitment to develop innovative
medicines that address unmet medical needs and complement
Shionogi's rapidly expanding pipeline in the focus areas designated
in our Medium-Term Business Plan STS2030 Revision,” said Isao
Teshirogi, Ph.D., CEO of Shionogi. “The science behind MZE001 is
differentiated and promising, and we look forward to developing the
compound as both mono and add-on therapy to enzyme replacement
therapies.”
MZE001 is a small molecule and specific inhibitor of GYS1, an
enzyme involved in glycogen synthesis. It reduces the glycogen
concentration in muscles by inhibiting this enzyme, and the results
from the Phase 1 study of MZE001 suggest that it has the potential
to be the first oral therapy for the treatment of Pompe disease.
MZE001 has the potential to be used both as a monotherapy option
and as an add-on therapy with enzyme replacement, the current
standard of care, to enhance the treatment of patients with Pompe
disease.
“Shionogi is committed to advancing and commercializing MZE001
because they understand the potential this therapy has for patients
and the unmet medical needs it could address,” said Jason Coloma,
Ph.D., CEO of Maze. “Shionogi has a track record of developing and
delivering innovative medicines to patients worldwide, and we’re
confident they are the right partner to continue to advance MZE001
through clinical trials so that it may reach patients with this
life-threatening condition as soon as possible.”
In 2022, the U.S. Food and Drug Administration (FDA) granted
Orphan Drug Designation to MZE001. The FDA grants Orphan Drug
designation to prevent, diagnose or treat a rare disease or
condition. Under the FDA's Orphan Drug Act, orphan drug status
provides incentives, including tax credits, grants and waiver of
certain administrative fees for clinical trials, and seven years of
market exclusivity following drug approval.
About Pompe Disease Pompe Disease is a rare congenital
metabolic disorder in which there is a deficiency of the enzyme
necessary for the breakdown of glycogen within cells, resulting in
the accumulation of glycogen in cells throughout the body,
especially in muscle cells. This leads to various symptoms such as
muscle weakness and delayed growth. In Japan, it has been
designated as a "specified rare disease" and a "specific pediatric
disease," making it eligible for medical expense assistance
programs and other forms of support.
About Maze Maze Therapeutics is a biopharmaceutical
company that is harnessing the power of human genetics to transform
the lives of patients, with a focus on genetically informed
therapies for common diseases such as chronic kidney disease. Maze
applies variant functionalization in tandem with advanced data
science methods and a robust suite of research and development
capabilities to advance a pipeline of novel precision medicines.
Maze has developed the Maze Compass Platform TM, a proprietary,
purpose-built platform to understand and integrate the critical
step of variant functionalization into each stage of drug
development. Utilizing the Maze Compass PlatformTM, Maze is
building a broad portfolio of wholly owned and partnered programs.
Maze is based in South San Francisco. For more information, please
visit mazetx.com, or follow us on LinkedIn and Twitter.
About Shionogi Shionogi & Co., Ltd. is a leading
global research-driven pharmaceutical company dedicated to bringing
benefits to patients based on its corporate philosophy of
"supplying the best possible medicine to protect the health and
well-being of the patients we serve." Shionogi has discovered and
developed novel antibiotics, medicines for HIV and influenza and
currently markets medicines for infectious diseases and central
nervous system disorders. Shionogi’s global pipeline includes
research programs in infectious disease, pain/CNS, metabolic
disorders, rare disease, oncology and stroke. For more information,
visit https://www.shionogi.com/global/en/.
Forward-Looking Statements This announcement contains
forward-looking statements. These statements are based on
expectations in light of the information currently available,
assumptions that are subject to risks and uncertainties which could
cause actual results to differ materially from these statements.
Risks and uncertainties include general domestic and international
economic conditions such as general industry and market conditions,
and changes of interest rate and currency exchange rate. These
risks and uncertainties particularly apply with respect to
product-related forward-looking statements. Product risks and
uncertainties include, but are not limited to, completion and
discontinuation of clinical trials; obtaining regulatory approvals;
claims and concerns about product safety and efficacy;
technological advances; adverse outcome of important litigation;
domestic and foreign healthcare reforms and changes of laws and
regulations. Also for existing products, there are manufacturing
and marketing risks, which include, but are not limited to,
inability to build production capacity to meet demand, lack of
availability of raw materials and entry of competitive products.
The company disclaims any intention or obligation to update or
revise any forward-looking statements whether as a result of new
information, future events or otherwise.
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For Further Information, Contact: SHIONOGI Website
Inquiry Form: https://www.shionogi.com/global/en/contact.html
U.S. Media: ShionogiCommunications@shionogi.com
SEU Press Office: pressoffice@shionogi.eu
Maze Therapeutics: Jillian Connell
jconnell@mazetx.com
Dan Budwick, 1AB dan@1abmedia.com