There is a considerable potential for our drug in treating dementia in DS and beyond. Overall, 13.3% of DS individuals have dementia, giving 139,650 eligible cases for treatment.  In the US, the number of individuals eligible for treatment would therefore be 33,250, which should qualify for orphan drug designation. The introduction of Aduhelm (aducanumab) to the market has generated increased interest in the dementia field, demonstrating that a drug affecting a biomarker may receive an accelerated registration.  It has also set a precedent regarding pricing in this field; one year’s treatment being priced at USD 56 000. Bottom-up market analysis show that Pharmasum´s drug candidate PST-674 has a large commercial potential depending on annual price and market penetration.

Using dementia in DS as a stepping-stone, other dementia indications will be explored and may provide significant additional commercial upside. Approximately 2m patients live with Parkinson’s in the US and 2.4m in EU. Dementia occurs in approximately 50% of patients with Parkinson’s disease during their lifetime and is an additional indication for PST-674. Should our drug be successfully developed for the treatment of this additional indication, sales would be achievable above 1bn USD per year. For Alzheimer’s disease, which contributes to 60-70% of the dementia cases in the general population, the market potential would be dramatically and several bn USD per year. As a reference, the cognitive enhancer drug Aricept had annual sales of 3.3bn USD in 2010 before the patent expired (Al-Shamahi & Jago 2012, A Thompson Reuters Report, Spotlight on Alzheimer´s disease) and the predicted peak-year sales of the dementia drug Aduhelm have been forecast to reach up to 10bn USD.

Pharmasum Therapeutics aims to develop novel, differentiated drugs and partner these for late-stage development. The company may reserve commercial rights in certain territories.