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Expert Interview

Slingshot members are talking to an expert! The topic is:

Hot on the heels of the Celator success, Moleculin IPOs with a liposomal Annamycin treatment for AML


Who's the expert?

Name: Dr Gabriel Ghiaur - MD

Institution: Johns Hopkins University

  • Medical oncologist affiliated with the Sidney Kimmel Comprehensive Cancer Center and an assistant professor at Johns Hopkins University.
  • Currently manages 80-100 patients per year with AML; sees 50 new patients a year out of 300 in his group.
  • Physician-scientist whose primary interest is normal and malignant hematopoiesis; research has translational potential in stem cell therapeutics, as well as acute leukemia.

Interview Questions

Please describe your background in AML and the number of patients that you treat.

Added By: joe_mccann

What's your impression of the drug being developed by Moleculin? Does a liposomal Annamycin sound attractive to you?

Added By: joe_mccann

In the previous trials conducted by a company named Callisto Pharmaceuticals. The company had these findings from trials of Annamycin:

  • Annamycin demonstrated significant activity in 8 of 16 patients in a Phase I study in adult relapsed or refractory AML patients, with 6 of 14 patients completely clearing leukemic blasts. A 30 patient dose-ranging Phase I/II study in ALL demonstrated a similar level of activity, with 3 of 10 patients treated with the maximum tolerable dose clearing their leukemic blasts to a level sufficient to qualify for a bone marrow transplant. One of these patients went on to receive a successful curative bone marrow transplant. The other two of these three patients died of tumor lysis syndrome, a condition resulting from the overloading of their system with the debris from leukemic blast cells destroyed by the induction therapy. 
What are your impressions of the Callisto studies?

Added By: joe_mccann

What's your impression of this study from 2013 on the drug: ?

Added By: joe_mccann

How likely do you think it is Moleculin could attain Orphan Drug Status or Special Protocol Assessment for accelerated approval?

Added By: c_admin

Are there other major factors aside from cardiotoxicity and multidrug resistance keeping the survival rate of AML patients undergoing induction therapy low?

Added By: c_admin

What type of pricing strategy do most anthracycline companies employ and does it place a major burden on most of your patients?

Added By: c_admin

Are there any plausible alternative treatments on the market or the horizon of which you're aware?

Added By: c_admin

How do you see the success of VYXEOS impacting Annamycin?

Added By: c_admin

Does it surprise you to see VYXEOS and Annamycin reappearing after both were shelved for some time?

Added By: c_admin

This was the Phase 1 trial conducted by Callisto on Liposomal Annamycin in refractory AML:

From the Abstract:
"Thirty patients were enrolled on the study. The MTD was determined to be 150 mg/m2/day for 3 days. Eight of the patients completed 1 cycle of the 3 days of treatment. Of these 8 subjects, 5 (62%) had an efficacy signal. All 5 completely cleared circulating blasts. Three subjects also cleared bone marrow blasts. Of these 3, 1 went on to a successful bone marrow transplant."

What do you make of this performance?

Added By: joe_mccann

From the same Phase 1 study:
"The other 2 had tumor lysis syndrome and unfortunately expired. Other than the tumor lysis syndromes, there was only 1 SAE definitely related to the study drug which was a grade 3 mucositis but there were also 3 other SAEs of grade 3 or 4 mucositis probably related to the study drug which comprised the MTD determination. There were no reports of cardiac toxicity"

How concerned are you that 2 patients expired from tumor lysis syndrome?

Added By: joe_mccann

Are You Interested In These Questions?

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