Zogenix Presents New Data at the World Muscle Society Conference 2020

EMERYVILLE, Calif., Oct. 02, 2020 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ:ZGNX), a global biopharmaceutical company developing and commercializing rare disease therapies, and its subsidiary Modis Therapeutics, shared new data and information about MT1621, the company’s investigational therapy for Thymidine kinase 2 deficiency (TK2d), at this week’s International Congress of the World Muscle Society (WMS 2020). TK2d is an inherited mitochondrial DNA depletion syndrome that primarily affects infants and children and is often fatal.

“These new analyses highlight the unmet need in TK2 deficiency and underscore the important progress we are making in our MT1621 development program,” said Joanne Quan, M.D., Chief Medical Officer for Modis Therapeutics. “We are excited to be collaborating with researchers and clinicians internationally to understand and develop a treatment for this devastating disease.”

The company’s WMS 2020 posters, now available on the Zogenix Newsroom, include:

  1. MOA: MT1621 for Thymidine kinase 2 deficiency (TK2d) Mechanism of Action is via Mitochondrial DNA Incorporation
    (Seves, Kenny, Blazquez Bermejo et al)
  2. Modis Untreated Dataset: Analysis of Morbidity and Mortality in Untreated Patients with Thymidine Kinase 2 Deficiency
    (Domínguez-González, Garone, D’Souza et al)
  3. Collaborative Model for Diagnosis and Treatment of Very Rare Diseases: Experience in Spain with Thymidine Kinase 2 Deficiency
    (Paradas, Domínguez-González, Madruga-Garrido et al)
  4. Study 104 Protocol: Design of a Prospective Clinical Study for the Treatment of Thymidine Kinase 2 Deficiency (TK2d)
    (Hirano; Ali, Dominguez-Gonzalez, et al)

About TK2 Deficiency

Thymidine kinase 2 deficiency (TK2d) is a genetic disorder that primarily affects infants and young children and results in mitochondrial dysfunction, leading to inadequate energy production in cells. The disease presents as progressive and severe muscle weakness that profoundly impairs movement, breathing, eating, and other normal functions, and is often fatal. There are currently no approved therapies for this disease. To learn more, please visit www.tk2d.com.

About Zogenix

Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases. The company’s first rare disease therapy, FINTEPLA® (fenfluramine) oral solution, C-IV has been approved by the U.S. FDA, is under review in Europe, and is in development in Japan for the treatment of seizures associated with Dravet syndrome, a rare, severe childhood onset epilepsy. FINTEPLA is also in development for the treatment of seizures associated with Lennox-Gastaut syndrome, another rare childhood-onset epilepsy.

Through its subsidiary Modis Therapeutics, Zogenix is also developing MT1621, an investigational novel deoxynucleoside substrate enhancement therapy for the treatment of TK2 deficiency, a rare genetic disorder. Deoxynucleoside combination therapy has been shown to improve cell function and prolong life in preclinical models of TK2d. Data from initial clinical studies suggest that this therapy may meaningfully alter the course of disease in patients with TK2d. The company plans to conduct a Phase 1 pharmacokinetic study in renal impairment and is compiling additional data required to submit a new drug application (NDA) for MT1621, which is expected to be submitted in the first half of 2022.

Forward Looking Statements

Zogenix cautions you that statements included in this press release and the poster presentations that are not a description of historical facts are forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “indicates,” “will,” “intends,” “potential,” “suggests,” “assuming,” “designed,” and similar expressions are intended to identify forward-looking statements. These statements include Zogenix’s development plan for MT1621, including the timing of the submission of an NDA to the U.S. FDA. These statements are based on Zogenix’s current beliefs and expectations. The inclusion of forward-looking statements should not be regarded as a representation by Zogenix that any of its plans will be achieved. Actual results may differ from those set forth in this release due to the risks and uncertainties inherent in Zogenix’s business, including, without limitation: the timing of enrollment or results of Study 104 or the planned Phase 1 pharmacokinetic study in renal impairment may be delayed; the COVID-19 pandemic may disrupt Zogenix’s business operations, impairing the ability to complete the planned studies of MT1621; unexpected adverse side effects or inadequate therapeutic efficacy of MT1621 that could limit development or commercialization, or that could result in recalls or product liability claims; and other risks described in Zogenix’s prior press releases as well as in public periodic filings with the U.S. Securities & Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Zogenix undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.


Melinda Baker
Senior Director, Corporate Communications
+1 (510) 788-8732
[email protected]

Brian Ritchie
Managing Director, LifeSci Advisors LLC
+1 (212) 915-2578
[email protected]

Stefanie Tuck
Vice President, Porter Novelli
+1 (978) 390-1394
[email protected]


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