Ariad Prepares for Early Launch of CML Drug Ponatinib The Pink Sheet Daily. 2012 Aug 6, E Hayes

says it will be commercially ready in the U.S. by the end of October and hopes to launch in the first quarter of 2013.
“We want to be ready to go ahead of schedule if the FDA gives us an earlier approval, which we can’t [count] on but certainly we have to be ready for,” CEO Harvey Berger said during an Aug. 2 earnings call.

Ponatinib targets the Bcr-Abl mutation as well as isoforms of the mutation – such as T315I – that cause treatment resistance in CML. Ariad’s development partner MolecularMD Corp. simultaneously submitted a companion diagnostic for the T315I mutation to FDA.
At the agency’s request, Ariad filed a rolling submission in resistant or intolerant CML and Philadelphia chromosome-positive acute lymphoblastic leukemia, the company announced July 30. To complete the application, Ariad plans to submit routine chemistry/manufacturing/control data in the third quarter.

According to the company, 99.9% of the application is complete and the agency will begin a “comprehensive review of the entire file immediately.”
Ariad has requested a six-month priority review for the drug and is requesting accelerated approval based on a single-arm Phase II trial.

The firm does not expect an advisory panel review. “If I had to guess I would say we’re not going to have a panel,” Berger said during the call.
“The data are pretty clear. FDA tends to use panels when there are major questions outstanding.”
And while a user fee date won’t be assigned until the application is complete, Berger stressed during the call that this would not slow down the review.............

The company is planning to launch in Europe in the second half of 2013. Ariad will then need to negotiate coverage and reimbursement in individual countries. Ariad will be starting trials in Japan shortly.

From Relapse To Front-line

In the U.S. and Europe, Ariad’s plan is to launch first in intolerant or resistant patients, but move rapidly into front-line CML, following the lead of Sprycel and Tasigna................

EPIC
The EPIC study, which has started recruiting, the company announced July 27, tests ponatinib against Gleevec in 500 patients with chronic phase CML. Ariad believes that Gleevec is the appropriate comparator because it is the dominant front-line therapy and because Sprycel and Tasigna are not yet fully approved in this setting, as they received accelerated approval contingent upon results from longer-term studies.
Ariad is also aiming for accelerated approval of ponatinib in this population.

Patient enrollment in EPIC will wrap up by the end up 2013 and results are due in 2014. The trial is 90% powered to detect a 15% absolute improvement in the primary endpoint of MMR (major molecular response) at 12 months over Gleevec. Secondary endpoints include MMR at five years, molecular response at three months, complete cytogenic response at 12 months, progression-free survival and overall survival. Patients will be followed for eight years from the time of randomization of the last patient.
“We’ve powered the trial appropriately. We didn’t try to skimp and come up with the smallest trial possible,” Berger said.

Ponatinib achieved a 30% MMR in the highly resistant or intolerant population in PACE study, as a secondary endpoint. “The 30% rate is pretty remarkable in a highly resistant/intolerant population and we do know that MMR rates are significantly higher in newly diagnosed, earlier-stage patients than in end-stage or more advanced patients,” Berger said.

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