Integrated Clinical Development Planning for Iron Overload Candidate

An exciting biotechnology company that develops gene therapeutic technology based on RNA wanted to develop a baseline, integrated development plan for a lead iron overload (IO) candidate. The client was keen to take a data led approach to understand and document the following:

  • Competitor activity, dosing and development status, including relevant input for strategic business development activities and interactions with the regulatory agency
  • Lead target indication(s)
  • Leading countries, KOLs and trialists
  • Protocol design, including baseline patient characteristics, standard of care, outcome measures and inclusion/ exclusion criteria

Within three months Phesi successfully delivered to the Board a baseline, fully integrated development plan document covering:

  • Product positioning: TPP and commercial potential, the potential candidate value increasing 5-fold as a result of the analysis
  • Clinical development timelines and implementation planning
  • Regulatory strategy
  • NCD strategy
  • Program timelines, resourcing and budget
  • Leading authorities from a KOL and trialist stand point

Phesi identified opportunities to secure orphan drug status in 2 lead indications, increasing the potential peak sales by $2.3bn, leading to investment by a third party and a dramatic rise in stock value.