Compounds under development for the treatment of mucopolysaccharidoses

Ongoing development of the DO-10 drug candidate

Dorphan is developing DO-10, a repositioned molecule, for the treatment of GM1-gangliosidosis and mucopolysaccharidosis IVB, two orphan indications in which the beta-galactosidase enzyme is mutated.

Although DO-10 does not interact directly with beta-galactosidase in patient cells, this molecule very significantly reduces in at least 2/3 of the mutations tested, the concentration of substrates whose accumulation is harmful. DO-10 has essential pharmacological properties for the targeted indications, in particular:

  • Oral bioavailability
  • Brain penetration

DO-10 is a molecule that was developed several years ago for another indication and has been tested in humans up to phase 2. DO-10 has been extensively tested especially in pre-clinical (acute, subacute and chronic toxicology), mutagenicity, teratogenicity, and pharmacokinetics). In addition, this product has been tested for its safety in humans in two phase 1 studies where it has demonstrated good tolerability and good safety. The compound is currently in the development phase to enter phase 1b in patients in GM1-gangliosidosis and mucopolysaccharidosis IVB in the US as soon as possible.