Dr. Tefferi presented new data pending publication of new prognostic data: significance of ASXL1 mutation in survival and discussion of ability to target the ASXL1+ mutant diseases as target for new interventions. Dr. Tefferi is not discussing Telomerase inhibitors. Dr. Goel will discuss that topic and Jakafi inhibitors (last presentation). "We are going to brag about Imetelstat later when Dr. Goel presents (in response to direct question about legal actions by MPN skeptic.
Dr. Goel presented all the current clinical trials for MF and other myeloid disorders. These consists of 5 JAK2 inhibitors, HSP90 inhibitors, 5 combination therapies all involving ruxolintinib (Jakafi) looking for other drugs to offset the side effects or toxicity (e.g. anemia). All were in Phase II or Phase III with significant side effects and only clinical symptom reduction. Imetelstat was also presented with no new data. Even with existing data, it was clearly superior (even to lay patients) to the alternatives. The purpose of this exercise was to ask the physicians in attendance what was the best alternative. The majority response was Imetelstat "if not on hold." Two stated after larger population studied that reported at ASH. In response to a question, Dr. Tefferi stated (and 2 other times), "If I had MF, I would want Imetelstat." He also stated, "I want a drug that does more than reduce anemia and spleen size."
Dr. Tefferi statements: "The that patients want this drug!." "I know the molecular signature that predicts CR." "My Phase II trials are ready to go." (This in response to when the hold would be lifted) " There were Grade 1 and Grade 2 LFT and FDA concerned for ET patients who 'live forever'. FDA wants us to show them the full picture so they can be comfortable." "Never cleared the marrow of fibrosis before." There was no new data reported. There was much personal enthusiasm from Dr. Tefferi.
Irish, if you read this, here are some questions you might want to ask::
1. How many (or what %) of patients remain on Imetelstat out of the original 60 that were enrolled?
2. Is the MC seeing a continuation of the 20% rate of remissions in the broader patient population beyond what was already reported at ASH.
3. Do the Imetelstat patients that respond have long term benefits that are durable, or are some of the patients regressing at some time point?
4. Are Imetelstat liver toxicities forcing discontinuance of the drug for any of the MC patients?
5. How and when is the MC planning to respond to the FDA hold?
end - Dr. Tefferi was extremely enthusiastic and encouraging and "promising" about the future of Imetelstat. However, he was not ready/able to publicly confirm any of the data you seek (yet). I'm sorry. However, I think it is coming soon IMO. He wants to help the patients as soon as possible there is no doubt. No discontinuations to best of my knowledge. I had the impression FDA response is underway.
He's not excited about Imetelstat, is he?
As I opined yesterday, I don't think that anything will be said about the liver data while Mayo and GERN are preparing the FDA response.
Of course, IMO.
Hey Opined - see this
The title of the last presentation is:
2-2:30 pm Swati Goel: “Review of Current Clinical Trials in Myeloproliferative Neoplasms: What’s The Professors’ Take On It?”