G.P.284 - The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development

D Wells,E Heslop,C Csimma,V Straub,J Mccall,K Nagaraju,K Wagner,D Caizergues,R Korinthenberg,K Flanigan,J Mendell,M Kelly,P Kaufmann,E Mcneil,A Robertson,L Johnston,K Bushby

doi:10.1016/j.nmd.2015.06.308

Abstract

G.P.284 - The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development

Highlights

Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials
Despite hundreds of publications on potential therapies for these diseases based on work in cell and animal models [14], only a handful were moving forward into clinical trials, and there was no systematic way for such potential therapies to be evaluated for their development potential
While patient organisations and other funders were being asked to fund therapeutic development programmes, not all of them have established objective advisory system to appraise the clinical potential for candidates, independent from their existing internal scientific advisory boards who were responsible for making funding decisions

Summary

Discussion

Utilisation and outputs of TACT 2010–2014 To date TACT has held 10 review meetings, 6 in Europe and 4 in the US and reviewed 29 program applications from both academic investigators and industry in several diseases: Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Becker Muscular Dystrophy, Congenital Muscular Dystrophy, Inclusion Body Myositis and X-Linked Myotubular Myopathy. Of the 29 programs reviewed, (Table 1) 19 (66%) were from industry and 10 (34%) were from academia; 15 (52%) were for novel compounds and (48%) were for repurposed drugs; (58%) were small molecules and 11 (42%) were biologics; (48%) were preclinical stage applications and (52%) were clinical stage applications. Preclinical: The need to use gold standard operating procedures for animal model experiments, for example, utilising SOPs available via TREAT-NMD for models of three neuromuscular diseases: (www.treat-nmd.eu/sopdmd) (www.treat-nmd.eu/ sopsma) and (www.treat-nmd.eu/sopcmd). In a number of cases TACT encouraged the applicants to ensure their results were reproducible through independent validation [16]

Formulation

Repurposing

Findings

Clinical Trial design

Manufacturing