Sure, Neuren has 4 areas but the main value drivers of this company, even though concussion and tbi are "topical", are the opportunities in orphan drug with Rett Syndrome and Fragile X which currently have no alternative treatments. Orphan drugs are for rare diseases with less than 200k patients in the USA or 250k in Europe. The opportunity is bigger in fxs and retts than tbi/concussion because the average orphan drug sells for US$100,000 up to -US$400,000 per treatment per year so even if you have small patient markets, the revenue opportunities are multiple billions and that is why 50% of M&A's in 2015 involved Orphan drugs via takeovers or via partnering deals with smaller companies. Make no mistake tbi is a "nice-to-have" but it is not the main attraction. By way of background, after we failed to break 20 cents from retts people said "I'm thinking that if the Fragile X results are positive we will be attractive to big pharma". Now at least a couple of people are suggesting we need to wait for tbi (another binary event) to attract big pharma interest. Big pharma look at the end market revenue opportunities and pay multiples of that in t/o's and via partnering deals. In the 2 core products that ALREADY have successful Phase II trials alone before you talk about concussion, traumatic brain injury or other things we have conservatively US$5billion-9billion of revenue in end markets compared to a US$150m market cap. MAKE NO MISTAKE NEU is already attractive to Big Pharma (management hinted this with the appointment of Leerink and the Chairman has signaled increased interest following the results) but it isn't the kind of interest we'll be able to measure about until a deal is signed, sealed,etc. (remember, neuro is a key focus area at present and 50% of deals are Orphan drugs so NEU is well placed). So ex tbi and concussion Neurens end market numbers are MASSIVE (US$ billions) for any big pharma. As a refresher, Shire and Baxalta are targeting US$20 billion rare disease revenue by 2020 and NEU could contribute 25-50% to that (or perhaps 100%+ and perhaps if it is proven suitable for MS, Epilepsy, Neuropathic Pain, Parkinsons, Alzheimers, Mood Disorders (Bi Polar and depression), Shizophrenia and inflammation attributed to aging i.e. aspirin for the brain). Regardless, PURELY based on the FXS/Retts results (ignoring TBI/Concussion) we not only have significantly derisked $9 billion end market from FXS and Retts, but also the entire Autism Spectrum (1% of population=much more valuable than topical TBI and concussion).
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