Musings on what Scarlett will bring to the FDA

Forum rules
- Comments must be civil and on topic
- Back up claims with evidence/reasoning/sources (posting links is allowed)
- No commercials/harassment/spam
Post Reply
Posts: 235
Joined: Fri Jul 20, 2018 5:13 pm

Musings on what Scarlett will bring to the FDA

Post by biopearl123 » Wed Dec 04, 2019 7:12 pm

I thought it might be fun to compile a list of what might go before the FDA at the end of PII meeting presumptively scheduled for March 2020. I invite board members to add or subtract and to help rank order in terms of importance:

1. The MF study that demonstrates significant MOS improvement. That data could include OS survival as well since the study probably will have no survivors by the time of presentation. (Conclusion set for April on clintrials site, I am guessing that is when OS data i.e. no survivors, will be available.). Pro's: impressive survival improvement Con: RWD rather than conventional control arm. Discontinuation rates high. Discussion: lots of support for RWD as new standard but FDA has not finalized criterion and is going case by case. Support: Ned Sharpless, although he has left FDA head is back to position at NCI and probably "gets it."And has a background in understanding the importance of the telomere and telomerase in cancer pathology. Indeterminate: we do not know the fate of all of the patients. Were AML progression rates affected? What was the MOS of the TN patients etc.

2. KOL's have spoken very positively and publicly.

3. Dr. Rizo will probably make the presentation.

4. Preclinical supportive data re selective targeting of malignant clone will likely be presented in both ET and MF.

5. I doubt the MDS data from part I will play any role.

6. The Tefferi study showed CRs and PRs but we really don't know much about the fate of these patients. This "tantamount to a cure" thing I believe helped to severe the relationship with Geron since it just wasn't true and those who hold on to this platitude should ask where the cures are. Ain't none.

7. ASH poster showing potential for combinations. Doubt this will play any prominent role with end of part II MF study presentation since it wasn't part of it to begin with (but of course is very exciting).

As an aside, I have taken some heat suggesting that there will not be much of an update to the Part I MDS study presented as part of the poster presentation. I continue to believe all the focus will be on the Part II protocol and getting the word out to enroll new patients. Even though we and patients deserve to know what the durability of the the 8 and 24 week TI's are to date, I just don't think the nebulous Scarlett has it in his plans since it does not really change how Part II is regarded beyond the already presented data. I do hope the Part II data is spectacular since much more experience with dosing should keep more patients in the study than Part I.

All thoughts welcome. Best Holiday Wishes to all. bp

Post Reply