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 […]

10% of CF patients have a nonsense mutation. Ataluren has been trialled with nonsense mutation CF.

Previous phase 3 trial results (no TOBI):
+ 5.7% relative improvement in FEV1 compared to placebo
41% reduction in pulmonary exacerbations

New phase 3 trial is planned to start in the first half of 2014. Data is expected mid 2016.

Phase 3 confirmatory trial planned to start early 2014. Conditional EMA application also planned for early 2014.

New graphs show stability in the previous phase 3 trial with those not on inhaled antibiotics. After 48 weeks those taking Ataluren (no antibiotics) dropped 0.2%.

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There are multiple approaches that may help nonsense mutations.

It is thought that Ataluren helps ‘read through,’ allowing the ribosome to ignore the stop codon and produce the normal protein.

A new approach may increase this protein function further, when used in conjunction with Ataluren.

Summary of the first PTC investor conference, held August 12, 2013:

1. Second phase 3 study planned to start in 2014 with nonsense mutations.
2. Plan to apply to the EMA this year.
3. Potential for combination with Vertex’s Kalydeco discussed, PTC looking at ways to evaluate this concept.

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About 10% of people with CF have a class 1 nonsense mutation.

Ataluren has been in trials with these mutations. The phase 3 trial showed a 5.7% relative improvement in lung function compared to placebo, with those not on inhaled Tobramycin.

There is some debate about how Ataluren helps nonsense mutations.

CFTR Modulators currently help multiple organs by increasing the function of the CFTR protein.

Gene Therapy helps the lungs with a less frequent medication, but is further behind in terms of trial development.

In the future could gene therapy be used in conjunction with CFTR modulators?