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Ionis/Roche's Huntington's Drug 'Next Spinraza'?

 
• By Alexandra Shimmings

Further details of a Phase I/II study results are raising hopes that it could be the first disease-modifying treatment for the disease, but it is not yet known whether the reduction in Huntintin seen will translate into a clinical benefit.

DNA & RNA
• Source: Shutterstock

New top-line data from a Phase I/II study of Ionis Pharmaceuticals Inc.'s investigational Huntington's disease antisense product IONIS-HTTRx (RG6042) suggest it can significantly reduce levels of the disease-causing mutant huntingtin protein, making it the first product to show disease-modifying potential in the disease.

Partner Roche is now speeding the product, which has US and EU orphan drug status, into a pivotal study. 

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