On December 15, 2022 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported an update on its clinical programs evaluating Annamycin for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases (Press release, Moleculin, DEC 15, 2022, View Source [SID1234625316]). These updates were recently presented during a reception for potential investigators held outside of the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in New Orleans.
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Key updates made during the presentation were as follows:
Announced 80% overall response rate (ORR) in final cohort (n=5) of the European trial of Annamycin as a single agent for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML) with one CRi (complete response with incomplete recovery of peripheral blood count) and three PRs (Partial Response).
Of a total of 42 subjects in three of the Company’s Annamycin clinical trials, 100% demonstrated no signs of cardiotoxicity as confirmed by independent expert. All expert reviews included analysis of ejection fraction, echo strain and certain troponin levels intended to assess the potential for both acute and chronic heart damage.
In two of the Company’s Annamycin clinical trials, 32 subjects have been, as allowed by the trial protocol, safely treated above the current FDA lifetime maximum anthracycline dose (550 mg/m2) with up to 1800 mg/m2 of Annamycin treatment with no evidence of cardiotoxicity as confirmed by an independent expert.
Data so far have resulted from treatment with Annamycin as a single agent, however, based on additional preclinical animal data from sponsored research, Annamycin in combination with Cytarabine demonstrated a 68% improvement in the median overall survival (OS) compared to Annamycin as a single agent and a 241% increase in OS compared to Cytarabine alone. These data were recently presented at the 62nd Annual Meeting & Exposition of the American Society for Hematology (ASH) (Free ASH Whitepaper) under the title: "High Efficacy of Liposomal Annamycin (L-ANN or Annamycin) in Combination with Cytarabine in Syngeneic p53-null AML Mouse Model."1
Annamycin has been in two single agent Phase 1 clinical trials treating R/R/AML, one in the US and one in Europe. Annamycin is currently in two Phase 1b/2 clinical trials for the treatment of soft tissue sarcoma metastasized to the lungs, again one in the US and one in Europe. Furthermore, based on the preclinical and clinical data discussed above, the Company has begun screening in Poland for a Phase 1b/2 clinical trial using Annamycin in combination with Cytarabine for the treatment of R/R AML. The Company is looking to expand this trial into other countries in Europe to potentially improve recruitment rates.
"Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic. We have continued to make significant progress across our clinical trial programs evaluating Annamycin in STS lung mets and AML. Based on the encouraging data seen to date, we are optimistic in Annamycin’s potential to treat a number of indications and remain committed to furthering its development," commented Walter Klemp, Chairman and CEO of Moleculin. "Additionally, we continue to receive encouraging feedback from our clinical staff and recently had the opportunity to meet with our current and potential investigators during the ASH (Free ASH Whitepaper) Annual Meeting. We intend to expand our Phase 1b/2 clinical trial using Annamycin in combination with Cytarabine for the treatment of R/R AML beyond the borders of Poland. We have gained valuable insight in all of our ongoing development programs, and I believe we are well-positioned to successfully execute on our clinical development initiatives moving forward."
Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of STS lung metastases and the treatment of relapsed or refractory AML.