Recombinant Biglycan

Recombinant Biglycan for Treatment of Duchenne and Becker Muscular Dystrophy

Dr. Joel Braunstein
Tivorsan Pharmaceuticals

22nd June 2012

Tivorsan Pharmaceuticals presented a detailed and well described development plan with specific milestones to advance a recombinant form of human biglycan for treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). They propose to perform a Phase Ia clinical trial with single ascending dose in healthy volunteers to assess safety and pharmacokinetics (PK), followed by a Phase Ib clinical trial with multiple ascending doses in ambulatory patients with DMD. This therapeutic approach is based on published work from Dr. Justin Fallon that shows beneficial effects of recombinant biglycan in young mdx mice treated for up to 3 months. Importantly this work has been replicated in independent laboratories. TACT suggested some additional pre-clinical studies be performed prior to taking this therapy to the clinic, largely based on the requirement to obtain a further understanding of the dose-response relationship. TACT also advised on the selection of outcome measures linking biopsy results to functional changes and suggested a 6-month clinical study for the main outcome measures instead of the proposed 3 months of dosing in order to have greater potential to see functional changes. Consistent with Tivorsan’s plans, TACT further advised that any trials in BMD be sequential to and distinct from studies in DMD.

12 Apr 2017