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.

New hope for acute leukemia patients…

New hope for acute leukemia patients…

In a Phase II clinical trial, Annamycin was given to patients who had failed an average of five previous induction therapy attempts, and 30% of those patients cleared enough of their leukemic cells to qualify for a bone marrow transplant…

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Tackling the most difficult cancers…

Tackling the most difficult cancers…

By reducing the activity of certain tumor supporting transcription factors, tumor cells lose their ability to survive and reproduce. By increasing the activity of other transcription factors, WP1066 actually calls the patient’s natural immune system into…

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Breaking through to brain tumors…

Breaking through to brain tumors…

Brain tumors are among the most difficult cancers to treat, largely because of something called the “blood brain barrier”. WP1122 was specifically designed to slip past the blood brain barrier, using the same chemical trickery that converts…

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  • About Us
  • About Childhood Leukemia
  • About CTCL

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of oncology drug candidates, some of which are based on discoveries made at M.D. Anderson Cancer Center. Our clinical stage drugs are Annamycin, an anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity being studied for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML, and WP1066, an immuno-stimulating STAT3 inhibitor targeting brain tumors, pancreatic cancer and AML. We are also engaged in preclinical development of additional drug candidates, including additional STAT3 inhibitors and compounds targeting the metabolism of tumors.

Our lead product candidate is Annamycin, a Phase II clinical stage anthracycline for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML.

Unlike current therapies that risk cardiotoxicity and can have their effectiveness limited due to multidrug resistance, Annamycin appears capable of avoiding both of these problems and has already demonstrated the ability to save lives in clinic.  We are now preparing to seek accelerated approval for this game-changing drug.

child-cancerPROBLEM: Cardiotoxicity and multidrug resistance severely limit pediatric oncologists’ ability to effectively treat children suffering from acute leukemia.

SOLUTION: We believe Annamycin’s ability to avoid both cardiotoxicity and multidrug resistance has the potential to eliminate this critical problem.

One of the most exciting scientific discoveries here at Moleculin, which we believe will have a profound impact on the treatment of children suffering from Acute Leukemia, is our lead drug Annamycin’s ability to be non-cardiotoxic.

We believe that this characteristic alone has the ability to change the way pediatric oncologists’ treat children suffering from acute leukemia for the foreseeable future.

Cardiotoxicity can be a killer, causing severe, permanent and sometimes fatal damage to the heart of both adults and children and cardiotoxicity is highly present in the currently approved drugs used to treat all acute leukemia patients. The drugs that are used to treat acute leukemia patients are called “anthracyclines.”

Oncologists (and especially pediatric oncologists) are limited in how they can treat their patients, due to the harmful effects of cardiotoxicity.


Here are some quotes from other publications:

“The effectiveness of anthracyclines and related compounds (doxorubicin, daunorubicin, idarubicin, epirubicin, and the anthraquinone mitoxantrone) is hindered by their cardiotoxicity.” – Cardiotoxicity of anthracycline-like chemotherapy agents

“Anthracyclines remain an important class of drugs in the treatment of cancer, but also remains a problematic chemotherapeutic agent given their cardiotoxic effects.” – Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment

To better understand how cardiotoxicity impacts children suffering from acute leukemia, consider that acute leukemia is the most common form of cancer in children, comprising approximately 30 percent of all childhood malignancies. Of the acute leukemias, acute lymphoblastic leukemia (ALL) occurs more commonly than acute myeloid leukemia (AML), however the treatment of both typically involves the same induction therapy (the preparation for a bone marrow transplant) used in adults to reduce the presence of leukemic cells in the patient’s bone marrow and bloodstream in preparation for a curative bone marrow transplant.

Most unfortunately for children, however, the standard drug used in such induction therapies is significantly cardiotoxic.

This often means that even though the chances are often good that current treatments can cure a child’s leukemia, it is very likely that the child’s lifespan will be shortened and their quality of life diminished as a result of permanent heart damage caused by current therapies.

This is why we believe Annamycin’s lack of cardiotoxicity has the ability to change the way children are treated for the foreseeable future.

Annamycin has been shown to be non-cardiotoxic in animal models, when compared with one of the leading anthracyclines (doxorubicin).

To make matters worse, the leading induction therapy drugs lose efficacy due to something called “multidrug resistance” requiring more and more drug to try and prepare patients for a successful bone marrow transplant, but because of the harmful effects of cardiotoxicity, patients simply cannot tolerate an increase in dosage.  At the same time that Annamycin avoids cardiotoxicity, it also avoids multidrug resistance, and as a result, Annamycin has demonstrated in early clinical trials the ability to deliver efficacy in patients who have failed repeated attempts with the existing induction therapy drugs.

Oncologists, and especially Pediatric Oncologists, have been clear that there is a tremendous unmet need here and we believe Annamycin has the potential to fill that need and change forever how children with leukemia are treated.  The severity of this unmet need may allow us to position Annamycin for accelerated approval under FDA guidelines and we will be working hard this year to secure an accelerated approval pathway. We look forward to moving quickly to translate this important breakthrough into new hope for those children who have run out of options.

Here are some interesting articles on the effects of cardiotoxicity:

Risk of heart damage follows some childhood cancer survivors

Woman battling breast cancer diagnosed with heart failure

Cutaneous T-cell lymphoma (CTCL), a form of non-Hodgkin’s lymphoma, is a rare cancer that occurs when T-cells in the skin grow uncontrollably. It is estimated that about 16,000 to 20,000 people in the U.S. have CTCL with a higher incidence in men between the ages of 50 and 60 years old.

CTCL is often mistaken for eczema, psoriasis or contact dermatitis, since it initially presents as patches or plaques. Over time, the patient may develop tumors or generalized erythroderma (abnormal redness of the skin over widespread areas of the body).

The disease is categorized as Stages I-IV, each of which represents an increased risk for metastasis, or the spread of cancer to distant parts of the body. About three-quarters of the patient population fall into Stages IA, IB and IIA, the lower risk group. Once the disease becomes systemic, however, the prognosis—or outlook—is significantly worse. Beginning with Stage IIB, CTCL is considered progressive (or advanced) and median survival drops to 4 years.

“I have been in drug development for close to 35 years and led the development of carboplatin and cisplatin, which have been blockbuster drugs for years. In all my experience, Annamycin is one of the best drug development opportunities I’ve seen.”

Donald Picker, PhD Donald Picker, PhD

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