May 22, 2014

May Ataluren Update

Cystic Fibrosis & Ataluren
About 10% of CF patients have a nonsense mutation
Current treatments do not address the underlying cause of nonsense mutation CF

Previous phase 3 trial results
Patients on Ataluren were stable after a year, this stability continued in the extension study (see graphs below)
These previous results were recently published in a Lancet Journal article

 Upcoming confirmatory phase 3 trial
– Planned to start by June 2014, 48 week trial
– Design likely to be similar to the previous trial, with lung function as the primary endpoint and pulmonary exacerbations as a secondary endpoint
– The major change from the prior study is that patients taking inhaled tobramycin are excluded from enrolling
– Tobramycin binds to the ribosome, making it harder for Ataluren to be effective

Further information about the upcoming trials
Patients need to have at least one nonsense mutation, be aged 6 or older, and between 40-90% FEV1
Upcoming phase 3 trial (new patients)
– Estimated enrollment 208 patients
Upcoming phase 3 trial (for patients who were in the previous trial)
– Estimated enrollment 80 patients

Images from the May Investor Presentations

May Slide 8

Previous Phase 3 Results

May Slide 29

May Slide 47

May Slide 30

Sources: May 6 Investor Presentation & May Presentation


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1. Class 1 Nonsense Mutations, 3. PTC - Ataluren