go to link Zywie LLC is a therapeutics company focused on the treatment of metabolic and neurodegenerative disorders known as Lysosomal Storage Diseases (LSDs), a multi-billion dollar market. Many of these disorders are characterized by protein misfolding, instability and impaired trafficking of proteins within cells. One approach to treatment is the application of pharmacological chaperones (PCs), small molecules that enter cells and serve as a molecular scaffolding in order to cause misfolded mutant proteins to fold and route correctly within the cell.
see Zywie’s lead candidate, known as “Ziva”, is a proprietary oral suspension of a pharmaceutical chaperone that targets the molecules that enter cells and serve as a molecular scaffolding in order to cause misfolded mutant proteins to fold and route correctly within the cell. Zywie is:
- Developing Ziva as the first approved treatment of Type 3 (T3) Gaucher Disease (GD)
- Evaluating the potential to pursue approval for other indications.
Mechanism of Action
As a pharmaceutical chaperone, Ziva. Initially, Zywie is targeting T3 GD, an ultra-rare LSD, with “neuronopathic” neurological symptoms. Enzyme Replacement Therapies (ERTs) are able to treat many of the symptoms of T1 GD (e.g., spleen function) but are not able to penetrate the Blood-Brain-Barrier, which would be required to treat the neuronopathic form of Gaucher Disease (such as T3 GD).
Intellectual Property (“IP”) & Other Protection
Ziva’s active ingredient has completed a Phase 1 clinical trial and has been used in a multi-year compassionate use study in Japan that supports therapeutic efficacy in T3 GD patients. Ziva is protected by:
- A portfolio of issued use patents licensed from Toronto’s Hospital for Sick Children;
- New patents that have been filed for a proprietary formulation and oral dispenser for treatment of the target diseases; and
- Orphan drug designation for treatment of Gaucher Disease, which offers 7 years’ exclusivity in the US and 10 years in the EU.