With Few Good Treatment Choices, Which Emerging Therapies Hold the Greatest Potential for Managing AML and What Types of Therapies Will Receive Reimbursement?

Despite the introduction and use of hypomethylating agents for acute myeloid leukemia (AML), treatment of this disease has changed little over the past several decades. Withdrawal of the only FDA-approved monoclonal antibody for AML—Pfizer’s Mylotarg (gemtuzumab ozogamicin)—from the U.S. market in 2010 was not only met with disappointment and controversy, but it left clinicians with fewer treatment choices. Given patients’ short survival times, the level of unmet need remains high, particularly for elderly patients, who may not be eligible for intensive induction chemotherapy. Even though regulatory incentives such as orphan drug designation and breakthrough therapy designation are available to developers of therapies for AML, which is a rare cancer, these incentives have not been enough to attract substantial levels of clinical development activity in AML. Although several therapies being investigated for AML will offer new treatment options for elderly AML patients with newly diagnosed disease, opportunity remains high for therapies that extend survival and slow disease progression.

Questions Answered in This Report:

  • Longer overall survival (OS), complete remission (CR), and progression-free survival (PFS) are key goals in the treatment of AML. What are the key primary and secondary clinical trial end points with which new therapies are evaluated? How do U.S. and European hematological oncologists weight specific efficacy end points and other drug attributes in their prescribing decisions for newly diagnosed, elderly AML patients?

  • A regimen of cytarabine (Pfizer’s Aracytine, Nippon Shinyaku’s Cylocide, generics) + daunorubicin (Pfizer’s Daunoblastin, generics) is the 2012 major-market sales leader for AML (newly diagnosed, elderly). What weaknesses exist in its profile that would allow emerging therapies to gain a foothold in the market? Have emerging therapies demonstrated potential on the attributes that surveyed hematological oncologists indicate are the most important in their prescribing decisions? Which emerging therapies will offer the clinical improvements over currently available therapies that surveyed MCO PDs seek from new therapies?

  • OS and price are key drivers of physicians’ prescribing decisions and/or are the focus of drug development for new AML therapies. What trade-offs across these and other clinical attributes are U.S. hematological oncologists willing to make when considering the use of emerging therapies for the treatment of AML, particularly for newly diagnosed, elderly patients? Based on the trade-offs in price and performance across key drug attributes that U.S. hematological oncologists are willing to make, how do physician preference and prescribing likelihood vary across different target product profiles for AML?

  • Quizartinib (Ambit Biosciences) + chemotherapy could emerge as the future gold-standard therapy for newly diagnosed elderly AML patients according to our Drug Comparator Model because of its superior clinical profile over the key current therapies we evaluated. On what clinical attributes is quizartinib/chemotherapy most differentiated from its competitors? Which current therapies are at greatest risk of being replaced by quizartinib/chemotherapy?


Attributes included in conjoint analysis based assessment of target product profiles for acute myeloid leukemia (newly diagnosed patients, aged ≥ 60 years):

- Overall survival rate at two years.

- Disease-free survival rate at two years.

- Event-free survival rate at two years.

- Complete remission rate.

- Hematological toxicity: rate of grade 3/4 leukopenia/thrombocytopenia/neutropenia.

- Drug formulation.

- Price of therapy.

Attributes included in assessment of U.S. payers’ receptivity to new therapies for acute myeloid leukemia (newly diagnosed patients, aged ≥ 60 years):

- Effect on two-year overall survival rate.

- Effect on two-year event-free survival rate.

- Effect on completion remission rate.

- Incidence of hematological toxicities: thrombocytopenia, leukopenia, and neutropenia.

Physicians surveyed: 63 U.S. and 30 European hematological oncologists.

Payers surveyed: 20 U.S. MCO PDs.

Comprehensive List of Therapies Included in Our Research and Modeling:

Current Therapies

- Azacitidine (Celgene’s Vidaza, generics)

- Cytarabine (Pfizer’s Aracytine, Nippon Shinyaku’s Cylocide, generics)

- Daunorubicin (Pfizer’s Daunoblastin, generics)

- Decitabine (Eisai/Johnson & Johnson/Astex Pharmaceuticals’ Dacogen, generics)

- Gemtuzumab ozogamicin (Pfizer’s Mylotarg)

- Idarubicin (Pfizer’s Idamycin/Zavedos, generics)

Emerging Therapies

- CPX-351 (Celator Pharmaceuticals)

- Quizartinib (Ambit Biosciences)

- Sapacitabine (Cyclacel Pharmaceuticals)

- SGI-110 (Astex Pharmaceuticals [a subsidiary of Otsuka Pharmaceutical])

- Volasertib (Boehringer Ingelheim)

- Vosaroxin (Sunesis Pharmaceuticals)

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