Moleculin Biotech, Inc. is a pre-clinical and clinical-stage pharmaceutical company focused on the development of game-changing anti-cancer drug candidates, many of which are based on discoveries made at M.D. Anderson Cancer Center. Each of our projects represents a breakthrough discovery and a highly disruptive technology.

Annamycin

annamycinAnnamycin is an anthracycline intended for the treatment of relapsed or refractory AML. The therapy of combining two chemotherapeutic drugs, which always includes an anthracycline, in inducing a remission of leukemic cells (called “induction therapy”) has not improved since it was first used in the 1970s and we estimate that this induction therapy has the same cure rate of about 20% as at that time. Currently, the only viable long term option for acute leukemia patients is a bone marrow transplant, which is successful in a significant number of patients. However, in order to qualify for a bone marrow transplant, patients must first undergo induction therapy.

One of the leading anthracyclines used for induction therapy in acute leukemia patients is doxorubicin, which has reported over $700 million in annual revenues. Despite the importance and success of approved anthracyclines like doxorubicin, they are all unfortunately cardiotoxic, which can result in damage to the heart and limit the dosage amount that may be administered to patients. Additionally, the tumor cells being treated often have or develop resistance to the first line anthracycline, often through what is called “multidrug resistance” making them capable of purging themselves of the current anthracyclines and limiting the effectiveness of the therapy. Consequently, there remains no effective therapy for these patients and most will succumb quickly to their leukemia. This is where we believe Annamycin can be a complete game-changer.

Annamycin is a unique liposome formulated anthracycline (also referred to in literature as “L-Annamycin”) that has been designed to eliminate cardiotoxicity and avoid the multidrug resistance mechanisms that often defeat current anthracyclines. In animal models designed to test for cardiotoxicity, Annamycin as shown to be non-cardiotoxic and in human clinical trials focused on leukemia, it showed fewer dose-limiting toxicities than are normally experienced with doxorubicin (one of the leading first-line anthracyclines used for induction therapy).

Annamycin demonstrated efficacy 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 acute lymphocytic leukemia demonstrated a similar efficacy profile, with 3 of 8 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.

We believe Annamycin is better than the currently approved induction therapy drugs in four key ways: (i) it has demonstrated clinical activity in a patient population for whom there are currently no effective therapies, (ii) it appears to be capable of avoiding the “multi-drug resistance” mechanisms that often limit the effectiveness of currently approved anthracyclines; (iii) it has been shown to be non-cardiotoxic in animal models, when compared with doxorubicin; and (iv) in laboratory studies using AML cell lines, it has been shown to be more potent than the leading approved drug.

Based on initial conversations with the FDA, and because of this serious unmet medical need, we believe Annamycin may qualify for for accelerated approval. We also believe Annamycin may qualify for Orphan Drug status, which could entitle us to market exclusivity of up to 7 and 10 years from the date of approval of a New Drug Application (NDA) and Marketing Authorization (MA), in the US and the European Union (EU), respectively.

 

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