You make a very good point. The Children's Hospital certainly has a pool of patients with the condition and likely would be soon ready to go with treatment after the initial assessment and baseline parameters are compiled. If we are only talking a couple dozen patients and that is likely the case, and its indeed open label without a placebo group, and they look at the data in a 15 week time frame as with the AZ trial...which would make sense, then you could indeed be looking at results in the 1st half of 2019, maybe even earlier than alzheimer's trial results come in.
Couple comments on that. I do hope its placebo controlled but considering those doctors are already working with and familiar with this population, you have eliminated any "false positives" already. It's not like the 20% of the alzheimer's population that is understood for misdiagnosis. These patients without question have the disease. You are also eliminating the moral dilemma of picking and choosing between patients to offer a novel approach that could be life altering for children. Most likely this study is "exploratory" in nature and used to create a P2/3 trial for the future as the PR states. I'm very satisfied to see the lack of FDA concern for safety in this young population that others have had trouble navigating. JMHO
Secondly, can you imagine the validation of the synaptic MOA these results could produce if revealed before the confirmatory alzheimer's trial? I suppose it could be argued that if the Fragile X results weren't spectacular, that it was due to lack of mature synapses from which to regenerate as opposed to mature but damaged synapses in the elderly. If the results are promising it would almost without question be due to synaptic growth factors or as the PR stated "transformation of immature dendritic spines to mature synapses". I can only imagine how sweet that phrase is to a parent of a fragile X child. If promising and delivered pre-top line alzheimer's results next summer, you could be talking well over a half billion market cap and some amazing press.
I think InTheTrenches gave a straight forward answer as to orphan drug status. It's a big plus. I think what could really push this thing forward is the fragile X Society. Incredibly well organized group of motivated parents who will have no problem pushing the FDA to expedite any potential cures. They can gain a lot of media coverage. Even though results are likely a ways off, I'm very excited to get this kicked off. I posted well over a year ago that I felt Fragile X might be an easier route to approval.
AI
