3 New Trials, all starting First Half 2022

[Ryan]

3 New Trials, eh? Last I checked they were making sure they could pay to finish MDS Phase III and most of the MF Phase II. Now they are starting up 3 new trials, all within the next 7 1/2 months.

Fantastic developments. Finally AML is on the table.
Good to hear the experts confirming from their own mouths the “remarkable” data from Imetelstat to date.

Anyways, something else has got to give if they are really going to launch those 3 trials in a few short months. (Although obviously these early stage trials will be much smaller in scope). As they say, “stay tuned”.

[Hoosier Investor]

Two of them are ISTs….which means a substantial portion of the trial costs are being covered by the Third Party. This is similar to what they did with Mayo a few years back.

[Hoosier Investor]

They’re basically doing what a few of us asked/suggested they do a few years ago….utilize creative (non-dilutive) IST funding to initiate studies involving promising treatment opportunities. They’ll likely have to pay a future royalty to the sponsor upon commercialization, but that’s a good trade-off at this point.

CKTC can explain it better, but there’s reason to expect a synergistic effect when combining telomerase inhibitors with BCL-2 inhibitors. Thus, the IMET plus Venetoclax IST study will be of particular interest.

Geron will self-fund the RUX plus IMET study, but it’s a small trial that can be absorbed without too much difficulty.

Geron is anticipating they’ll be in a financial position (post-commercialization in LR-MDS) to self-fund any registration trials that are informed & justified by these small studies.

I approve of their IMET plans & path moving forward. They’re likely holding back on a Ex-US partner for now as we’ll be able to get better terms after top line MDS results are known (assuming good results).

As for IMET2.0, it appears it will not play a significant role in near-term studies. I’m not sure why they remained silent on the topic and excluded it from the upcoming pre-clinical study.

[bucbeard]

MD Anderson the likely sponsor?!

[Ryan]

I'm looking forward to seeing this:

In regards to the Investigator Sponsored Trial, it was my understanding that "sponsor" could mean a host of involvement, from fully funding down to just supplying free supply of the drug.(?)
Definitely, or I should say from my ear, was not specified if/who is funding the trial yesterday.

CKTC can explain it better, but there’s reason to expect a synergistic effect when combining telomerase inhibitors with BCL-2 inhibitors. Thus, the IMET plus Venetoclax IST study will be of particular interest.

[Hoosier Investor]

While I can't provide a medical explanation behind the synergistic effect (1+1=3) of combining a Telomerase Inhibitor with a BCL-2 inhibitor, the potential effect & benefits are depicted in Slide #50 of Geron's presentation from yesterday. Have a close look at the middle graph. Also, note the "40% of mice" comment shown on the right-hand side graph reflects the study results associated with combining the Telomerase Inhibitor (Imet) with a BCL-2 inhibitor (ABT-199). Note: ABT-199 is the Venetoclax compound.

Mt. Sinai will be the "Investigator" for this open-label study of IMET + Venetoclax. I say this because the "Mt. Sinai" logo is shown on the top of slide 51 which illustrates the enrollment plans for the study. Dr. Mascarenhas appears to be the Principal Investigator which is excellent news for us.

One nice aspect about the IST's (besides the subsidized cost benefits) is the Principal Investigator can make enrollment and/or dosage modifications along the way. Dr. Tefferi jacked around with his dosage regimen so much that he undermined the usefulness of the data. I believe the Mayo IST was deemed as a "Phase 2", so there was a time when we hoped his data could be used to enable a New Drug Application (NDA). Geron never publicly stated why the Ph.2 had to be repeated, but I believe Dr. Tefferi's data set was 1) thin on enrollment numbers, and 2) confounded by all of the dosage modifications he made along the way. The upcoming Mt. Sinai effort appears to be a Ph. 1/2 based on the safety and dose-finding objectives. I suspect Dr. Mascarenhas will likely operate in a more controlled manner than Dr. Tefferi with regard to dosing, but he could still elect to enroll additional patients outside of the core study group....which would be very useful information.

It's clear from yesterday's information that MD Anderson is going to do the pre-clinical study involving Lymphoid Malignancies. My mom died from B-Cell Lymphoma a few years back, so it would be nice if Imetelstat shows promise in this area.

Based on the presentation (slide 49), it appears EMSCO will be the "Investigator" for the single agent study involving HR-MDS and AML. Dr. Platzbecker will be the Primary Investigator. FYI.....EMSCO stands for European Myelodysplastic Syndromes Cooperative Group.