AML is a type of heterogeneous hematological malignancy that originates from immature white blood cells (blasts) in the bone marrow, which may be derived from either a hematopoietic stem cell or a lineage-specific progenitor cell. "Acute" means that the leukemia may progress rapidly - AML generally spreads quickly to the bloodstream and can then spread to other parts of the body including the lymph nodes, spleen, central nervous system, and testicles.
Latest Key Takeaways
Relapsed/refractory AML remains an area of unmet need, and new therapies are being developed. Not including FLT3 or IDH inhibitors, there are currently five late-stage therapies being evaluated for this setting (devimistat, DFP-10917, flotetuzumab, Iomab-B, and uproleselan).
Key recent events include the approval of Onureg and presentation of numerical data from Venclexta's VIALE-A trial. There were Phase III failures for Helsinn's pracinostat combined with azacitidine in the newly diagnosed setting and for Roche's idasanutlin and Otsuka's guadecitabine in the relapsed/refractory setting. Other Phase III failures include trials evaluting Xospata combined with azacitidine and Daurismo combined with intensive chemotherapy in front-line AML, and an Idhifa monotherapy trial in the third- and fourth-line setting.
Key upcoming catalysts for 2021 include topline results from Phase III trials for devimistat, Iomab-B, uproleselan, and Zeltherva, and a possible NDA submission for FT-2102.
The overall likelihood of approval of a Phase I AML asset is 6.3%, and the average probability a drug advances from Phase III is 50%. AML drugs, on average, take 10.8 years from Phase I to approval, compared to 9.6 years in the overall oncology space.
Key Topics Covered:
OVERVIEW
Latest key takeaways
DISEASE BACKGROUND
Definition
Patient segmentation
Other risk factors
Symptoms
Diagnosis
TREATMENT
First-line treatment of AML consists of induction and consolidation
EPIDEMIOLOGY
Incidence methodology
MARKETED DRUGS
PIPELINE DRUGS
KEY REGULATORY EVENTS
Oncology, Rare Disease Drugs Dominate New China Approvals
Agios Withdraws MAA for Tibsovo
Onureg Offers New Dose Form, New Therapy Setting For Azacitidine
Xospata Reimbursement Marks A First For AML In England
Keeping Track: Targeted Oncologics Take Center Stage, Led By Retevmo And Trabecta Approvals
CHMP Adopts a Positive Opinion for Daurismo
NICE Draft Guidance: Rejects Keytruda In Head & Neck Cancer, Xospata In AML
PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
Agios Sells Cancer Portfolio To Servier To Focus On Genetic Diseases
Ireland's ONK Expands To US, Options Three NK Candidates
Syros Obtains Arsenic-Based Leukemia Candidate From Orsenix
Vor Hopes NCI's CAR-T Candidate Is Synergistic With Lead AML Therapy
Kite Will Partner With HiFiBiO On AML Discovery Efforts
Agios announces that it will receive $255m from Royalty Pharma
Gilead Calls Forty Seven Buyout Complementary To Kite, Other IO Efforts
Apollomics Gains China-Plus Rights To GlycoMimetics' E-Selectin Antagonists
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Recent events
DRUG ASSESSMENT MODEL
MARKET DYNAMICS
FUTURE TRENDS
Venclexta is set to become a blockbuster in AML
Immunomodulators are being developed for AML
New drug launches, including therapies for relapsed/refractory disease, will drive growth in the AML market over the
forecast period
FLT3 inhibitors are expected to move into the first-line maintenance setting
IDH inhibitor combinations are expected to move into earlier stages of AML
Oral hypomethylating agents are under development in AML
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