Danish company closes in on treatment for devastating degenerative muscle disease

New method halts disease progression

’Sporadic inclusion body myositis’ (sIBM) is a rare degenerative muscle disease; associated with progressive limb muscle atrophy leading to walking difficulty, frequent falls and the need for mobility aids. sIBM can progress to cause severe disability, as patients lose function of arms, legs or ultimately the respiratory passage. Today, no effective treatment exists. This is what the company Orphazyme is about to change, by delivering the first available disease-modifying drug on the market.

A large phase 2 clinical trial conducted by University of Kansas and the University College London is scheduled to establish final verification. But to reach market readiness, Orphazyme must not only document safety and efficacy of the drug. The clinical trials must be documented down to every detail, to verify the results. For this reason, this project aims to build this bridge between research and market. The result will ensure patient access to this new innovative treatment.

A helping hand to scope commercialisation

Orphazyme has succeeded in raising funding for pre-clinical and clinical trials, which has helped emphasise the huge potential of the solution. The company approached us with the need to secure funding for the commercial preparation. Together, we scoped a project highlighting the challenges of market approval in relations to the planned clinical trial. The proposal demonstrated the strong commercial prospective, and in the end convinced the Eurostars evaluators of its investment potential.

Danish company closes in on treatment for devastating degenerative muscle disease
Grant: Eurostars
Budget: 0.7M €
Funding: 0.3M €
Partners: University College London
Institute of Neurology