On August 9, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center, reported on several recent FDA approvals for new drugs for the treatment of acute myeloid leukemia (AML) (Press release, Moleculin, AUG 9, 2017, View Source [SID1234520119]).

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Walter Klemp, CEO of Moleculin commented: "the recent approvals of three new drugs (Rydapt, Vyxeos and Idhifa) for the treatment of AML are exciting, since they provide additional options for treatments in defined subpopulations, and because they help underscore the magnitude of the potential opportunity for Annamycin, which we will be studying for relapsed or refractory AML. With regard to AML, Rydapt is approved only for patients with a specific gene mutation, and for use in combination with the standard of care chemotherapy. Vyxeos is approved as an option to the standard of care, but only for specific AML patients, namely those with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Jazz Pharmaceuticals purchased this drug in their $1.5 billion acquisition of Celator Pharmaceuticals.

Mr. Klemp continued: "although FDA approval of both of those drugs was based on overall survival comparisons with a standard of care, Idhifa was approved based on an accelerated clinical trial design that showed a 19% response rate in patients with relapsed or refractory AML and IDH2 mutation. What’s interesting is that Idhifa was approved with a single Phase 1/2 clinical trial based on response rate, not overall survival, and a relatively low response rate at that. Also, the patient population for which it is approved represents only 13% of all AML patients. We look forward to working with FDA on a similar approach for Annamycin — reliance on response rate in an accelerated path — but for a larger population of AML patients."

"While these new drugs make valuable incremental improvements in AML therapy," concluded Mr. Klemp, "most AML patients will still fail to respond to (or relapse from) initial therapy; therefore, our initial clinical development plan will attempt to address the significant unmet need of patients who relapse from, or are refractory to, initial therapy. We also believe that, if Annamycin can demonstrate superior efficacy and safety to the current standard of care, the drug may be able to fill major areas for first-line AML treatment. In the meantime, these transactions serve to remind us of the opportunity for our company if Annamycin shows significant activity in our planned clinical trials."