Replies to post #201606 on Biotech Values

[DewDiligence]

IONS—In short, today’s news could signify a fundamental issue with the company’s platform.

[iwfal]

IONS

Reading between the lines it would seems that a patient died and I'd venture platelet issues go beyond the two programs by the deliberate wording of "at these levels" remarks.

Agree it seems likely that someone died - since he refused to give a direct answer. Further I would speculate it was with APOC3 FCS trial just because his tone when asked a specific question about the APOC3 events was very down. Also, if someone had died in the older TTR trial we probably would have heard about it already.

The other interesting things on the CC were:

a) His odd denial that there was a class effect despite being seen in multiple drugs now. Clearly this is a classic Stan semantics issue.

b) He points out that there have so far been no events in US sites and that there have been no events in the long trials at this dose in the various ph2s and OLEs (and I'd acknowledge this is odd given that this is not a small number of patients on the drugs for the 4 months it takes to see the problem).

c) The timeline (it shows up at about 4 months) is odd (e.g. Drisapersen also has a thrombo issue - but it shows up later)

d) He was asked explicitly about whether he was certain these events have happened in the treatment arms and he said that they are still blinded, but he thinks it very unlikely they are in the placebo arm.

e) The items they are exploring for potential cause are higher dose, sequence related, disease interaction (the latter seems unlikely given the appearance of an event in APOC3 trial - but I am sure they haven't reset their sights yet with the new APOC3 data).