Take aways from PiperJaffrey 24th Annual Healthcare Conference

[Fishermangents]

(New York November 29-30, 2016, from the webcast)
Link: http://edge.media-server.com/m/p/ezigwmnm

About MF: still the only drug today that has an effect on the malignant progenitor cells which are the sources of these diseases.

About MDS: IMerge continues; starting point is the original study with 8 transfusion dependent patients, of which 37% became transfusion independent for 8 weeks. Part 1 of IMerge is dose finding. Data read-out in Q2-2017 will be the inflection point for the MDS program. Early data are as expected and were sufficient to continue on. No new safety signals have emerged. Part 2 will be Phase 3.

MF: two arms: 4.7 and 9.4. No new safety concerns, 4.7 was not effective and stopped. 9.4 stopped enrolment, not treatment. Sufficient number of patients for next read out. Primary end points focuses on ruxolitinib, but does’t reflect imetelstat’s potential.

Breaking news about two new Jak-inhibitors: momelotinib and pacritinib had lower success rates than expected.
Momelotinib had 26% spleen response, which is less than expected. Pacritinib: Rux-experienced had 5-6% response rate for the spleen. Pacritinib has even lower response rate of 18%. So the performance of these new Jak-inhibitors is lower than expected, which leaves a whole space available for new drug. Imetelstat has different approach. Different MoA should have a significant opportunity.

MF + MDS reading out will be in Q2-2017.

AML Poster: AML pre-clinical work continuous the very good story.

ET Poster: shows that the telomere length of ET treated patients goes up, which supports that imetelstat effects the malignant progenitor clones (which is already supported by much data);

CML poster: stem cell work in CML shows that there are some interesting effects on blast populations. Pre-clinical data strongly point to the direction that imetelstat is effecting rapidly proliferating malignant progenitor clones in a multiplicity of myeloid malignancies.

Any comments are welcome.

[biopearl]

Thanks Fish, nice summary. Todays press release from INCY with pooled data to show longevity interesting and I think shows the wisdom of Janssen in targeting Jakafi failures to really highlight the uniqueness of imetelstat in targeting malignant clones. Jakafi will continue to build their brand but the data is not that impressive and Janssen is challenging it with the strategy outlined in their presentations (e.g. the natural history poster). Hope it works. Be interesting to see if J has any kind of press release at ASH. bp

[Fishermangents]

Indeed. For me Dr.S. sounded upbeat about the chances of imet beating mom and pac. He didn't say that directly, but by emphasizing the poor results of these two Jak-inhibitors he wanted to show that the window of opportunity is wide open for imetelstat.

[Fishermangents]

'Pre-clinical data strongly point to the direction that imetelstat is effecting rapidly proliferating malignant progenitor clones in a multiplicity of myeloid malignancies.'

I suppose the data of the 'numerous pre-clinical studies' that JnJ is sponsoring are supporting this statement.

[biopearl]

Fish, sure, two of which are poster presentations at ASH in CML and AML