One thing to note is that ISIS-SMNRx Phase 1 clinical trial on children was only initiated in December 2011. This compound is in the same stage of development as ATL1103 for acromegaly.
SMA is due to defective SMN1 genes. ISIS-SMNRx promotes the increased SMN2 gene, thus creates a normal level of healthy SMN genes to compensate for the defective ones.
There are about 35000 SMA patients in USA,Europe and Japan. Even though 1 in 50 ppl in US carries this defective gene.
If ATL1103 is offered $30m upfront payment, $80m milestone payment and royalties of up to $200m for ATL1103 and cost to carrynthe trials to Phase 2/3, most likely It would be the 'too hard to refuse' offer.
Let's hope.
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