The information below is from PTC’s first investor conference, held August 12, 2013.
In March 2013 PTC closed $65 million of private financing and in June PTC completed a $144 million initial public offering. This generated $210 million, which is planned to be invested in key areas, including Ataluren with CF.
Cystic Fibrosis & Ataluren:
Approx 10% of people with CF have class 1 nonsense mutations.
Previous phase 3 results showed clinical evidence of activity with Ataluren and provides insight for the upcoming phase 3 trial.
Previous results: 3% FEV1 difference between Ataluren and placebo at 48 weeks, however those not on inhaled Tobramycin had a 6% FEV1 difference between Ataluren and placebo. 45% of the patient study group were not on chronic inhaled antibiotics and 63% were not on inhaled Tobramycin. PTC believe that Tobramycin interferes with Ataluren’s mechanism of action, reducing the clinical benefits.
Overall there was a 23% reduction in pulmonary exacerbations, which increased to 41% (statistically significant) with patients not on inhaled Tobramycin.
PTC plan to submit a “MAA for conditional approval of Ataluren for the treatment of nonsense mutation cystic fibrosis to the EMA by the end of the year.
We are also preparing for the initiation of a confirmatory Phase 3 clinical trial in the first half of 2014. We are currently discussing the details of the trial design with the FDA and EMA. We expect the trial design for the confirmatory Phase 3 to be largely similar to the previous Phase 3 trial with FEV1 as the primary end point and exacerbation rate to be a key secondary end point.
Based on our sub-group analysis of patients not receiving inhaled tobramycin as well as the observed interference with Ataluren’s mechanism of action we are proposing that the enrollment criteria exclude patients while receiving chronic inhaled aminoglycoside antibiotics.”
A question was then asked whether Ataluren could be trialled with medications from Vertex?
This was the answer: “Yeah, sure. Obviously we are moving forward to look at Ataluren in the nonsense mutation cystic fibrosis patients. But clearly Kalydeco has some activity with being able to open up the chloride channel in other types of mutations as well as in the wild type gene. So it’s something certainly that we’re interested in evaluating and I think we will think about ways to evaluate them, that’s what we are doing right now.”