Boston, USA - Novotech, the global full-service clinical Contract Research Organization (CRO) that partners with biotech companies to accelerate the development of advanced and novel therapeutics at every phase, has released an expert report, Acute Myeloid Leukaemia - Global Clinical Trial Landscape, offering critical data-backed analysis of the latest developments in Acute Myeloid Leukemia (AML) research.

The report presents a comprehensive look at the AML funding landscape, trial density, patient recruitment data across geographies, standard of care, emerging therapies, recent USFDA approvals, and regulatory trends. Importantly it also includes an in-depth SWOT analysis to guide strategic decision-making, prioritize research areas, and identify challenges for clinical stage biotechs.

AML is a malignant condition affecting blood-forming stem cells, marked by disrupted differentiation and uncontrolled growth of myeloid lineages, and is the second most prevalent leukemia in both adults and children.

The Acute Myeloid Leukaemia (AML) - Global Clinical Trial Landscape report notes that

since 2019, the global biotech and biopharmaceutical industry initiated over 1,000 clinical trials for AML.

The clinical trial data shows:

  • North America leads at 40%
  • Asia-Pacific is second at 33%
  • Europe stands third at 22%
  • ROW contributes a moderate share of 5%.

“In North America, the United States holds a majority trial share at 86%, while in Europe, Spain, France, Germany, and Italy lead AML trials, showing the region's contribution to advancing research. Within the Asia-Pacific region, China and Australia collectively represent the highest trial share (around 65%). Among the ROW countries, Israel takes the lead with 55% of AML trials,” the report found.

The report includes an examination of the various AML risk factors, including age, gender, chemical exposure, smoking, previous cancer treatments, and genetic predisposition.

It also highlights that chemotherapy is typically used in conjunction with targeted drug therapy to treat AML, however it finds that the treatment paradigm is shifting “to personalized approaches guided by molecular and genetic profiles and incorporating targeted therapies. The goal is to advance precision medicine for AML by understanding its evolving molecular complexities”.

The report states, “Clinical trial initiatives in precision oncology for AML are revolutionizing treatment approaches, driving transformative advancements, and enhancing patient outcomes”.

The incidence data shows key disparities across regions, with Asia claiming almost half of worldwide diagnoses at 46.6% (Europe 22.1%, North America 14.7% and ROW 16.6%). As such, measures have been taken amongst medical professionals in Asia to aid in early detection and support the increasing burden in the region. 

  • Asia reported over 68,000 AML cases, with Mainland China, India, and Japan contributing approximately 30% to the global incidence in the Asia-Pacific region.
  • Mainland China alone accounted for over 35% of the reported AML cases in Asia.
  • Europe documented over 32,000 AML cases, constituting over 20% of the global incidence.
  • North American countries specifically the United States and Canada, together accounted for around 15% of the global incidence, with over 21,000 AML cases.

Additional key data covered in the report includes:

  • The United States leads with venture capital funding ($1786.7m), followed by China ($1218.5m), with a large gap between countries following this.
  • Among the leading companies that received funding for AML were Opna Bio SA, Shorla Oncology, CytosinLab Therapeutics Co. Ltd., and OncoVerity Inc.
  • The emergence of molecular studies has revolutionized the diagnostic and therapeutic landscape of AML.
  • FDA-approved molecular targeted drugs, including FLT3, IDH1, and IDH2 inhibitors, alongside mutation-independent agents like venetoclax and gemtuzumab ozogamicin, highlight the precision focus in AML management.
  • AML drug development data shows 105 drugs in preclinical stages, 113 in Phase I, 104 in Phase II, and 22 in Phase III. 
  • Marketed drugs for AML display a diverse array of molecule types. Small molecules constitute the majority (>65%) of these drugs, while Proteins and Antibody-Drug Conjugates make up the remaining portion.
  • Key opportunities identified were:
  • Ongoing research focuses on developing new agents and immunotherapies with improved efficacy and reduced toxicity profiles.
  • Identifying reliable biomarkers can further personalise treatment strategies and predict response to specific agents.
  • Research on early detection methods could improve prognosis and treatment outcomes.

FDA drug approvals received for AML over the last two years include ivosidenib particularly for its use in combination with azacitidine to treat newly diagnosed AML with a susceptible IDH1 mutation. Olutasidenib capsules received approval specifically for relapsed or refractory AML cases with a susceptible IDH1 mutation. Approval for quizartinib was received for the treatment of newly diagnosed FLT3 ITD-positive AML in adults. Additionally, the LeukoStrat CDx FLT3 Mutation Assay also gained approval as a companion diagnostic for quizartinib.

The Novotech research analyst team provides these expert reports on a monthly basis, completely free of charge. These reports offer current insights into global clinical trial activity, revealing which regions experience the highest trial volumes and the unique factors behind these trends. They tackle the potential and real hurdles faced by biotech firms in specific therapeutic areas in the hopes to positively impact and inform clinical trial decision making, eventually improving rates of success with new treatments.

Download the report here

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About Novotech 

Founded in 1997, Novotech is a global full-service clinical Contract Research Organization (CRO) focused on partnering with biotech companies to accelerate the development of advanced and novel therapeutics at every phase. 

Recognized for its industry-leading contributions, Novotech has received numerous prestigious awards, including the CRO Leadership Award 2023, the Asia Pacific Cell & Gene Therapy Clinical Trials Excellence 2023, the Asia-Pacific Contract Research Organization Company of the Year Award since 2006. 

The Company offers a comprehensive suite of services including laboratories, Phase I facilities, drug development consulting, regulatory expertise, and has experience with over 5,000 clinical projects, including Phase I to Phase IV clinical trials and bioequivalence studies. With a presence in 34 office locations and a dedicated team of 3,000+ professionals worldwide, Novotech is a trusted end-to-end strategic partner of choice. 

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