The client is a large, multinational biotechnology firm that is developing three novel therapies for acute myeloid leukemia (AML). AML is a rare, extremely aggressive form of blood cancer that is characterized by multiple molecular and cytogenetic mutations that are heterogeneous across patients. Patients are typically elderly (>60 years), although it sometimes develops in younger patients as well. Currently, a typical AML treatment takes the form of intensive chemotherapy treatments, or administration of hypomethylating agents, depending on the age and health/fitness of the patient. Survival rates for patients are very low, and a significant portion of sufferers relapse following initial treatment. Our client has three very early-stage agents: two oral pills each inhibiting a distinct disease-related biomarker, and one IV infusion that is an immuno-oncology agent that engages T cells against any cell expressing a specific antigen, which is expressed broadly on leukemic cells, but also some healthy cells as well.
The client wants an understanding of the addressable patient population for their products, specifically the incident population across all ages in the U.S. How many patients could be candidates for the client’s three agents in a given year? The client has shared a few data points they have collected on their own: incidence for individuals 60: ~10/100,000. What is the annual incidence then for AML? (click for answer)
Treatment of AML can really be divided into four stages: the first is called induction, where patients are administered short bouts of high-intensity chemotherapy to de-bulk their leukemic cells. Following induction is consolidation, where additional chemotherapy is administered in order to further push a patient into remission. Next, the patient enters the maintenance phase, or a longer-term therapy meant to prevent re-emergence of leukemic cells. If a patient relapses or is refractory to initial treatments, they enter the salvage stage, where further intensive chemotherapy treatments are applied. The client has three agents: two small molecule inhibitors and the one immuno-oncology (IO) product. The client believes IO has large potential in AML, but wants a recommendation. Based on the drug’s mechanism of action, which of the treatment settings may be best to target, and why? (click for answer)
The client is developing three assets for one indication – what are some pros and cons of having multiple agents in development for a single disease? (click for answer)