NI Research (NIR) has released the December issue of NeuroPerspective, which features a comprehensive review of two orphan disorders; Rett Syndrome and Frontotemporal Dementia. These disorders impact populations at opposite ends of the developmental timeline, but with devastating effect.
1) Rett Syndrome’s inclusion in the Autistic Spectrum category links it to areas of intensifying interest for the pharma industry, both in terms of its monogenic roots and the commercial opportunity afforded by the pricing flexibility attendant to rare disorders. It offers an accessible entry-point into the area of rare pediatric neurodevelopmental disorders, and a patient population that goes considerably beyond the micro-scale of ultra-rare diseases. There are 20,000 Rett Syndrome patients in the US: After six to eighteen months of seemingly normal development, communication capabilities and some motor skills reach a premature plateau, often then regressing until permanently lost. To this point, there is but a limited dataset of clinical trial results in Rett Syndrome. Neuren does have encouraging data from its recent NNZ-2566 trial. The trials of MS immunomodulators (from Tevaand Novartis) are longshots. The respiratory problems seen with Rett is to be addressed by Newron‘s sarizotan program, Neurolixis is on-deck as they work towards an IND.
2) Frontotemporal Dementia is often but erroneously lumped in with Alzheimer’s, it has some pathophysiological links to ALS. The ill-defined upper limit of estimated prevalence for FTD is near 50,000 patients. FTD in all of its variants exacts a devastating toll on patients and families, given its relatively younger age of onset and the rapid descent into severe impairment.
The heterogeneity of FTD has been a deterrent to larger pharmaceutical companies who find rare disorders appealing partly because of the hope that their clinical trial populations will be relatively homogenous. But any tau or TDP-43 targeting drug that achieves success in a larger-scale population will be then evaluated in the appropriate FTD population, given the pathophysiological common ground. Among small companies who have programs whose mechanisms are in theory applicable to FTD, only TauRx and Forum Pharmaceuticalshave chosen to emphasize FTD. If 2016 sees any clinical-stage FTD program show a Hint of Concept in its first Phase II trial, this will accelerate interest dramatically.Read More