Novel Acute Myeloid Leukemia Therapies to Offer Clinical Benefit in Small Patient Cohorts

LONDON, June 3, 2015 /PRNewswire/ --

The treatment pipeline for Acute Myeloid Leukemia (AML) addresses clinical gaps in the market, says GBI Research.

Analysis from business intelligence provider GBI Research - Acute Myeloid Leukemia Therapeutics Market to 2020 - forecasts the value of the global AML therapeutics market to increase moderately from $632.6 million in 2013 to $878.6 million by 2020, representing a Compound Annual Growth Rate (CAGR) of 4.8%.

Several drugs to treat AML are expected to be approved during the forecast period, including CPX-351, quizartinib, StemEx, Treosulfan, and Midostaurin. However, the approval of these treatments will be confined to small patient cohorts, reflecting their limited success in clinical trials.

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Treatment and prognosis in AML is strongly influenced by a patient's age and cytogenetic profile. The majority of diagnosed patients are aged 65 or over, with only 50% eligible for intensive chemotherapy, typically with cytarabine and daunorubicin. 

Treatment options are very limited for relapsed patients and elderly patients too frail for intensive induction therapy. However, the AML therapeutics pipeline addresses these gaps in the market, along with the significant lack of targeted therapies.

Senior Analyst Katie Noon: "The current developmental pipeline for AML has a strong number of drug candidates in both the late and early stages, especially for an orphan indication.

"While AML treatment is dominated by generic chemotherapeutic agents, which have been the treatment of choice for the past four decades, the pipeline dynamics now suggest a strong focus on targeted therapies. These are predominately small molecule inhibitors of serine threonine proteins kinases, immunological agents against tumor-associated antigens/genes, and antagonists against cell-surface receptors."

Key Findings Include:

• Five of the eight late-stage pipeline products are being developed as non-intensive therapies for the elderly
• Variation in molecule type has shifted away from small molecules, the dominance of which has decreased from 95% across marketed products to 65% across the pipeline
• There has been a significant shift away from cytotoxic agents, with key mechanisms of action including targeted therapies against CD33, CD123 and WT1Ac

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SOURCE GBI Research