Replies to post #145686 on Biotech Values

[biomaven0]

What is your opinion of Ponatinib and Ariad's prospects?

Well obviously the stock has had a huge run-up, but long-term I am still quite bullish and it remains my biggest single position by far.

Pona is by a good margin the best CML drug out there. It's efficacy is clearly better than the other drugs and its tolerability is also excellent. Basically it's producing outcomes in 3rd line patients that are consistent with those Gleevec produces in 1st line patients. These are patients where the other drugs would simply fail completely - the more different drugs a patient has been on the harder they are to treat - indeed some of this population might not even have a normal clone left and so might be inherently untreatable by a TKI.

If all patients were treated by KOL's in CML, and absent insurance issues, I believe pona would pretty much immediately take the market for new patients and patients that fail an existing drug (patients that are doing fine on their existing treatment will obviously stay there). In practice it's going to be much slower, as many docs will wait for the outcome of the front line trial. Gleevec will also go generic in a few years, and so some insurers will require step therapy. That's not necessarily bad for pona, as people fail Gleevec more quickly and more frequently than they do the 2nd line agents.

Pona also is likely to have significant activity outside CML - FLT3+ AML, GIST, some breast, RET+ in NSCLC. It also likely synergises with some chemo drugs via inhibition of the resistance-induced efflux mechanisms according to a recent publication. Absent a partner, these secondary indications will be slow to develop, however.

The second leg of the ARIA story is AP26113. We'll only get results from the Phase I in September, but there are already some good patient anecdotes out there. The competitive environment is much easier than in CML, and the market size in ALK+ is similar to that of CML. EGFR+ NSCLC is a huge wild card - if it pans out, then this will be a huge drug, much bigger than pona.

Peter