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Wednesday, 12/07/2016 3:18:06 AM

Wednesday, December 07, 2016 3:18:06 AM

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Market Size. Based on the patient populations, we constructed an estimate for potential sales across the NPC markets in the US and EU. Figure 8 highlights the sales potential for Trappsol Cyclo depending on the rate of market penetration. We assume pricing of $250,000 per patient annually based on our analysis of the market, which is above Company guidance of $140,000. Expected pricing in this range is reasonable considering the extremely low incidence of NPC and the market acceptance of high prices for drugs that treat similar orphan diseases. Our estimates are based on market penetration of 30%, 60%, and 90%, respectively. The low and mid sales penetrance estimates may be conservative in light of the severity and lack of treatment options for NPC, but account for risks that this analysis otherwise does not incorporate such as difficulty of diagnosis. In the event of greater physician and patient awareness of NPC combined with higher rates of diagnosis, the market opportunity for Trappsol Cyclo may be closer to 90% of the overall patient population.

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