Ataluren Update- Phase 3 trial planned for 2014

[GenH]

PTC had their first investor conference yesterday, here is a brief summary:

- Second phase 3 Ataluren study planned to start in the first half of 2014 with CF nonsense mutations
- Plan to apply to the EMA (Europe) this year
- Potential for combination with Vertex's Kalydeco discussed during investor conference, PTC looking at ways to evaluate this concept

More info here: http://sixtyfiverosesblog.wordpress.com/2013/08/13/august-ataluren-update/

 

[lcdbot27]

Interesting. I have a residual function mutation that Kalydeco works on and one nonsense that theoretically should work with Ataluren. I wonder how much (if any?) benefit I would get from Ataluren combined...Kalydeco is already very effective by itself.

 

[GenH]

If Ataluren and Kalydeco are safe and effective together, then theoretically your overall level of CFTR function would be likely to further improve. Hard to say how much further benefit until we hear more.