BIGLYCAN - A Unique Utrophin Upregulator

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • This research is pre-clinical, meaning it has not advanced to clinical trials involving people yet.

 

Where is this research being done and who is funding this research?

  • This research is taking place at Tivorsan and in the laboratory of Dr. Justin Fallon at Brown University. This work was funded by PPMD’s End Duchenne GAP program. Current funding is coming from private investors in Tivorsan, previously awarded PPMD and MDA foundation grants to Tivorsan, and an NIH grant (“UO1 mechanism”) to Dr. Fallon.

 

What is the goal or purpose of this research?

  • The goal of this research is to use a Glossary Link protein called TVN-102 (recombinant human biglycan) to increase utrophin and neuronal NOS (nNOS) at the muscle cell membrane, resulting in reduced muscle damage and improved muscle function. Utrophin is a Glossary Link molecule that is related to dystrophin in structure and form and can “stand in” for dystrophin when present in larger than normal quantities.

 

What is the current state of this research?

  • Independent laboratories have reproduced the beneficial effect of TVN-102 in mice that lack dystrophin. A reliable method to manufacture the protein has been established and a scalable production process is being optimized. Additional pre-clinical studies to support dosing in the clinic are currently in process.
  • An optimized version of rhBGN has been developed and this molecule, called TVN-102, has been designated the lead clinical candidate.
  • Tivorsan has held discussions with FDA to define its IND-enabling preclinical studies and early clinical development plan.

 

What steps need to be completed before moving into a clinical trial?

  • Manufacturing of TVN-102 needs to be scaled-up to produce quantities and purity necessary for use in humans.
  • Safety testing must be completed and the pharmacology properties of TVN-102 must be determined.

 

What is your best estimate for the length of time it will take to move this research into clinical trials?

  • 12 months

 

Where would a clinical trial take place?

  • It is too early to know where a clinical trial for this research would be located. Many complex factors go into determining the right location(s) for a clinical trial.

 

Who would be eligible to participate in a clinical trial?

  • Again, it is too early to know what the inclusion criteria would be for a future clinical trial. However, TVN-102 therapy is applicable to all forms of Duchenne, regardless of the underlying Glossary Link mutation.

 

Where can I learn more about this research?

(Updated 4/11/2017)