According to a press release from California-based biotechnology company Zogenix, the Company recently completed a multi-million dollar acquisition of Modis Therapeutics. Before its acquisition, Modis focused on the development of investigational therapies for genetic diseases that have often have no currently approved treatments.
As another result of the acquisition, Zogenix will now lead the development of MT1621 – previously Modis’ lead product candidate. MT1621 is an investigational treatment for thymidine kinase 2 (TK2) deficiency, a highly rare mitochondrial depletion syndrome.
About TK2-Related mDNA Depletion Syndrome
TK2-related mDNA depletion syndrome is a genetically-inherited disorder characterized by mitochondrial dysfunction, leading to progressive muscle weakness and, eventually, loss of both voluntary and automatic muscle control. Mitochondrial DNA (mDNA) depletion syndromes are a collection of conditions characterized by the ineffective copying and replication of mDNA, the short but unique chains of genetic material localized completely within mitochondria that contain vital instructions for the organelle’s function.
The condition is caused by mutations to a gene called TK2. In otherwise healthy individuals, TK2 is responsible for coding the production of an enzyme called thymidine kinase 2 (TK2). TK2 is found in mitochondria and plays a vital role in the synthesis of mitochondrial DNA. When TK2 is deficient, mDNA can’t be adequately replicated, meaning the body can’t repair or replace the vital energy-providing organelles. As a result, mDNA depletion syndromes are frequently and quickly fatal.
MT1621 Now in The Zogenix Pipeline
MT1621 is an experimental deoxynucleoside substrate enhancement therapy. This potentially new form of therapy seeks to treat TK2-related mDNA depletion syndromes by providing the deficient substances normally produced by TK2, while inhibiting the oxidative effects of certain by-products of the DNA synthesis process. One study in mice with simulated TK2 deficiency suggested that administration of TK2’s products was associated with a two-to-three fold increase in life expectancy.
It’s important to note that these treatments are still highly experimental. Recently, a phase 2 study of M1621 in 38 patients suggested significant benefit in human patients who had been receiving the drug for up to seven years. Data was compared to an earlier case study of 68 individuals with TK2 deficiency disorders. Findings suggest that M1621 brought about significant improvements in patients, such as improved functional abilities and statistically significant increases to life expectancy.
Despite encouraging results, however, further testing must be conducted. Zogenix intends to conduct additional clinical studies in the next few years to progress towards global availability. Modis, now a subsidiary of Zogenix, is entitled to royalty payments on each future sale of MT1621 – and is eligible for a lump-sum payment from its proprietor upon approval of the drug from the US Food and Drug Administration and the European Medicines Agency.
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