PLX is an Israeli biotech specializing protein design optimization.
With an 18.7M float and 32.4M shares outstanding, its MC = ~$25M.
5/20 its partner Thiesi Global Rare Diseases submitted their Enzyme Replacement Therapy (ERT) to FDA under the Accellerated APproval Program as a treatment for Fabry's Disease.
The problem with present enzyme replacement therapies is short half- life requires treatments at least every 2 weeks. The half life of the PLX enzyme treatment is that its half-life is up to 4X longer.
The competition is significant:
FOLD has an effective treatment that can be used for 40% of Fabry patients.
Genzyme is developing a treatment that may have additive value to PLX's ERT, but will be marketed as a standalone product
IDRSF is recruiting for a Ph3 study with a competing therapy
Most importantly, SGMO, FRLN and AVRO are working on Gene therapies that could render the above approaches obsolete upon approval.
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