RYTELO

[huntingonthebluffs]

Many of you may have known that RYTELO is the proposed trade name for Imetelstat. Anyway it was included in the FDA’s briefing document for the ODAC.

“ Executive Summary
Purpose/Objective of the AC Meeting
The Applicant is seeking approval of imetelstat (proposed trade name RYTELO) for the treatment of
transfusion-dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic
syndromes (MDS) who have failed to respond, or have lost response to, or are ineligible for
erythropoiesis stimulating agents (ESA).”

Might take some getting used to RYTELO versus Imetelstat.

[LWS]

MIAMI, FLORIDA (March 14, 2024) – Dr. Mikkael Sekeres is an expert in blood cancers at Sylvester Comprehensive Cancer Center - University of Miami Health System. He served on the Oncologic Drugs Advisory Committee (ODAC) for the trial that led to imetelstat vote of support at the FDA today. Imetelstat, also known as RYTELO, is telomerase inhibitor for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS). It will be used for the treatment of certain adult patients with low- to intermediate-1 risk MDS who have not responded to, or have lost response to, or are ineligible for erythropoietin stimulating agents (ESA) used to aid blood transfusions.

“I believe the benefits associated with the drug justify the risks. The benefits are real. When you have 40% of the patients who previously depended on red blood cell transfusions who no longer need transfusions, and this outcome lasts for an average of one year, I think that’s a significant benefit,” Sekeres said.

He added:

Some 20,000 Americans are diagnosed with MDS annually.
Anemia typically affects people 70 years or older.
No treatment options are available for anemia after ESA’s lose their effectiveness.

Dr. Sekeres is chief of the Division of Hematology at Sylvester and his specialty focuses on leukemia and myeloid disorders. He has served on the editorial boards for several medical journals and chairs scientific advisory boards for patient advocacy groups and research funding agencies. He has been the primary study investigator on dozens of phase I, II and III clinical trials, and has authored over 450 scientific articles. He is also an expert on CAR-T cell therapy causing t-cell malignancies issues, topic of ODAC’s meeting on Friday.

[LWS]

There is no medicine that does what Rytelo (imetelstat) can do as a stand alone medicine (transfusion independence, improved quality of life, longer life, disease modification, remissions, etc.). On approval (now a formality) off label uses will be suggested and tested. High on the list will be chemotherapy combinations. Other combinations are already in trials.

It now appears that Rytelo will be an important factor in the treatment of all blood cancers. It is too early to talk about cures, but some are expected.

[LWS]

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, announced that John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer, is scheduled to present at the following virtual investor conferences:

Stifel’s 2024 Virtual Targeted Oncology Days
Wednesday, April 17th at 11:30 a.m. ET (Corporate Presentation)

A webcast of each fireside chat and corporate presentation will be available through the Investor Relations section of the Company’s website under Events. An archive of the webcast will be available on the Company’s website for 30 days.

[LWS]

Stifel Oncology Forum today very positive (replay now available). #1 take away (beyond transfusion independence) --- "Dynamic Increase of Hemoglobin".