Replies to post #154204 on Biotech Values

[DonShimoda]

Were you aware of these cases before FDA label?

Yes, I was aware of the hepatotoxicity issue before yesterday. I believe this issue was discussed during the initial presentation of the PACE data at last years ASH meeting. If I'm not mistaken, all 3 deaths occurred in blast phase patients. Clearly, this isn't going to be an issue in a 1/2 line setting. As far as I'm concerned, it will have little impact on the long-term commercial success of the drug.

[iandy]

I haven't found any explanation on 3 liver failure cases. Were you aware of these cases before FDA label? I don't think at this point you can exlude the possibility that ponatinib better efficacy but with higher hepatotoxicity. We have to wait and see once it is used in wider patient population.

Discontinuation due to liver toxicity was not a feature of the PACE trial.
All 3 serious events were in patients at the extreme of the patient population.
They believe liver toxicity will be inconsequential in the use of the drug.

Pona has been granted the European version of accelerated approval.
There will be no black box.

We will see if they give the drug the broad label granted by the FDA.
While the European agencies have been generally tougher on approvals and reimbursements than here, I think they are keenly aware of the superior efficacy of this drug.

I want to thank you for your input.
I am always reevaluating my investments.

[JJM760]

I think by the time you get your engraved in stone data as to the cause of the SAE's, Ponatinib (Iclusig) will be well on it's way to gaining momentum as a leading CML drug. By that time, the PPS sale you are seeing now (and it may go lower) will have been a distant memory and another lost opportunity to invest in this company at a substantial discount to what it will be trading at in a few years.

FWIW, I am definitely not the smartest or most qualified man in this room, but I get the feeling that sometimes it's more important to some here to be right than to make money investing in biotech companies.

All possible respect to you JQ. I read your posts/tweets religiously. However, you have never been a big fan of Ariad and that includes when it was trading at $5.

[DonShimoda]

it won't likely get reimbursement for 2nd line until there are data available for that setting

I agree that additional data will be needed (See "D" of my earlier post msg-82507924), however, I don't think head-head trials with sprycel and tasigna will be necessary to obtain 2nd-line re-imbursement. The EPIC trial should be sufficient as it will allow direct comparison with the ENESTnd and DASISION results.

Gleevec going generic will certainly impact front-line pricing, however, it has always been my contention that the 2nd-line is the real opportunity for Iclusig. There is a direct correlation between long-term patient outcome and how quickly/deeply a patient achieves molecular response. As I've pointed out, gleevec, tasigna and sprycel aren't even close to achieving the numbers Ariad just released at ASH. Over time, i suspect the new NCCN guidelines will lead to earlier testing which, in turn, will lead to earlier/more frequent switching which, in turn, will benefit Iclusig at the expense of both sprycel and tasigna. If one of the current therapies fails to produce an MMR in the front-line, which drug are you going to want to take in 2nd-line: iclusig, which is able to achieve a 51% MMR in 3/4-line at 30months, or tasigna with a 28% MMR at 24 months in a 2nd-line setting?

One last point, salvage therapy alone is a $500mm market which means that at todays pps ZERO value for the first/second line opportunity is being priced into the stock let alone the ALK/EGFR inhibitor.