Highlights from the conference call 3 Nov 2016

[Fishermangents]

Some highlights from the conference call:

'Janssen has been sponsoring numerous preclinical studies to explore the effects of imetelstat in other hematologic myeloid malignancies.'
That's where the CML and AML are coming from. Obviously there is much more going on than we can see.

'University of California and San Diego and Janssen R&D will present data from non-clinical models of chronic myeloid leukemia and blast crisis'
CML is a new kid on the block and could be the next indication to be tested for. Janssen is co-presenter, which indicates that they are publicly associating themselves with imetelstat.

'median overall survivals regularly is reduced once patients with MF fail or discontinue ruxolitinib the only approved drug for MF today'
Supports the unmet need and the role for imetelstat to play to address that need.

IMbark: 'the safety profile at both doses was consistent with prior clinical studies in imetelstat in hematologic malignancies and did not identify any new safety signals.'
Saftey of imetelstat seems to be okay.

IMbark: ' the higher starting dose of 9.4 milligrams per kilogram showed encouraging trends in efficacy among the first 20 patients and warranted further investigation'
Imetelstat seems to work.

IMerge: 'IMerge safety and efficacy appears to be consistent with the data reported from the MDS patients in the Mayo Clinic pilot study. We are encouraged by these preliminary data in the trial currently continues unmodified.'
Everything goes according to expectations and plan.

IMerge: 'We expect Janssen to assess the benefit risk profile imetelstat in Part 1 as well as data from imetelstat program as a whole. And regulatory review before deciding whether to move forward Part 2 of IMerge.'
At the end of Q2 2017 JnJ wants to get the full picture of imetelstat and share that with the FDA. A Continuations decision seems obvious by that time.

'We and Janssen continue to be committed to advancing the development of the story novel drug.'
The partnership goes well. This validates JnJ's coprorate presentations showing imetelstat as top-ten 1B+ drug and JnJ's filing ambitions as well.

From the Q&A;

About the R/R MF population of the trial: 'So this is quite a severely ill population and I think that it represents a more ill population than any that we've studied previously and that as far as I know we've been studying by other drugs in the space in the field.'
This confirms that the IMbark patients are more difficult to treat than 'normal' MF patients and could explain why it takes longer before responses occur. Any success here will beat BAT.

'And then they also calculated a mean survival in patients who had actually discontinued treatment with drugs. I mean we don't know exactly why that presumably because they were no longer responding and it was very substantial difference.'
The database study showed that the R/R MF patients have a substantial worse life expectancy than non R/R patients. That favors the risk/benefit balance in the approval process. It is also the rationale for a fast track program.

'I will say that one of the benefits potentially or at least one of the rationale for studying imetelstat in hematologic myeloid malignancies are that many of these if not all of them of these types of malignancies appear to be associated with highly proliferative neoplastic clonal populations.'
The ET abstract (https://ash.confex.com/ash/2016/webprog ... 91467.html) proves that imetelsat is targeting these neoplastic clones. That supports the assumption that imetelstat will be able to target 'many if not all' types of hem malignancies in the future.

AML: 'So we did see anti-leukemic activity and we've also stated before that it would be necessary in our and I believe also Janssen’s view to actually treat patients with combination therapy. ../.. I don't really have anything to announce or give you any specifics around AML, but I can tell you it continues to remain very interested.'
So any trial on AML will probably be a combination therapy, which seems likely to be in the making.

[biopearl]

Kudos, Fish and thank you. Scarlett said he did not plan a post ASH conference as in the past but that does not rule out that Janssen will and we might get some further info then. Regards, bp