Year: 2013-14

Company: Sarepta Therapeutics

Liaison(s): Bo Cumbo, Emily Naughton

Sarepta Therapeutics is focused on developing firstin- class RNA-based therapeutics to improve and save the lives of people affected by life-threatening rare and infectious diseases. The company’s long-term vision is to become a leading, independent biotech company capable of sustainably developing a pipeline of breakthrough therapeutics and bringing them to the patients who need them. To accomplish this vision, Sarepta has a unique phosphorodiamidate morpholino oligomer (PMO) technology at its disposal that is uniquely versatile, allowing for the development of a range of therapeutic candidates that target different types of RNA. Sarepta’s pipeline currently includes its lead clinical candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy as well as potential treatments for influenza and Marburg hemorrhagic fever virus. Sarepta is interested in adding candidates to its pipeline that are based on its platform PMO technology. However, Sarepta has devoted its resources to gaining approval for its current pipeline and hasn’t formally evaluated which disease state the company should pursue next. The Sarepta TMP team was tasked with evaluating the risk/benefit profiles of 7 disease states to determine which indications would provide the highest return on investment (ROI) while limiting risk. To accomplish this task, an IP landscape and competitor analysis was conducted to look for areas with freedom to operate. Next, the feasibility of conducting and designing successful clinical trials that would support a regulatory pathway with limited clinical endpoints was analyzed. Finally, a financial analysis that compared the potential market opportunity to the costs of development of a therapeutic for each disease was completed to ascertain ROI. In support of the original objective, a report which included a high level comparative analysis of the risks and benefits of developing a therapeutic was performed for each of the variables being examined to form the prioritization strategy for Sarepta.