Breakthrough Clinical Results
Disc Medicine will present data from its Phase 1b trial of DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia at the 2025 American Society of Nephrology (ASN) Kidney Week. The study assessed the safety, tolerability, and pharmacokinetics of DISC-0974, as well as its pharmacodynamic activity, including changes to hepcidin, iron, and hematologic parameters. The presentation will include data from all single and multiple-dose cohorts.
Key Highlights
- Disc Medicine to present Phase 1b data of DISC-0974 at ASN Kidney Week 2025.
- The study focuses on NDD-CKD patients with anemia.
- Data includes safety, tolerability, PK, and pharmacodynamic markers.
- The abstract is available on the ASN Kidney Week conference website.
Incidence and Prevalence
Latest Global Estimates of Anemia Incidence and Prevalence
According to the World Health Organization (WHO), anemia affects more than 1.62 billion people worldwide based on 2021 data. The global burden of this condition remains significant despite some improvements over recent decades.
Global Prevalence
The 2024 data shows that the global age-standardized point prevalence of anemia in 2019 was 23,176.2 per 100,000 population (22,943.5-23,418.6), with years lived with disability (YLD) rates of 672.4 per 100,000 (447.2-981.5). Encouragingly, the global age-standardized point prevalence decreased by 13.4% (12.1-14.5%) between 1990-2019, while the YLD rate decreased by 18.8% (16.9-20.8%) during the same period.
A 2016 study reported that global anemia prevalence in 2010 was 32.9%, causing 68.36 million years lived with disability. By 2011, anemia affected approximately 496 million non-pregnant women, 32 million pregnant women, and 273 million children worldwide.
Regional Variations
The highest national point prevalences of anemia in 2019 were found in: - Zambia: 49,327.1 per 100,000 (46,838.5-51,700.1) - Mali: 46,890.1 per 100,000 (44,301.1-49,389.8) - Burkina Faso: 46,117.2 per 100,000 (43,640.7-48,319.2)
In East Africa, a 2022 study found anemia prevalence among lactating mothers was 36.5% (95% CI: 35.55%, 36.75%). A 2015 study showed that concentrations of mean hemoglobin were lowest and anemia prevalence was highest in south Asia and central and west Africa.
Age and Gender Patterns
In 2019, the global point prevalence of anemia was highest in the 15-19 age group for females and the 95+ age group for males. A 2016 study in rural China reported anemia prevalence at 51.5% in 2006 and 53.7% in 2008, with a 2-year cumulative incidence of 42.1%. Both prevalence and incidence were higher in women than in men, with incidence sharply increasing after 45 years of age in both sexes.
Causes and Trends
Iron deficiency remains the dominant cause of anemia globally. A 2017 systematic review challenged the common assumption that iron deficiency causes half of all anemia cases, finding that for pre-school children, the proportion of anemia associated with iron deficiency was 25.0% (95% CI: 18.0, 32.0) and for non-pregnant women of reproductive age, it was 37.0% (95% CI: 28.0, 46.0).
Between 1995 and 2011, global mean hemoglobin improved slightly from 125 g/L to 126 g/L in non-pregnant women, from 112 g/L to 114 g/L in pregnant women, and from 109 g/L to 111 g/L in children. During this period, anemia prevalence decreased from 33% to 29% in non-pregnant women, from 43% to 38% in pregnant women, and from 47% to 43% in children.
Developing countries account for 89% of all anemia-related disability, with the burden being lower in regions with higher socio-economic development.
Key Unmet Needs and Target Populations for Anemia
Vulnerable Populations
Pregnant women represent a critical target population, with anemia affecting 41.8% globally. Adolescent pregnant mothers face heightened risk, with 28.7% prevalence in urban Malaysian settings. In Ethiopia, anemia rates are increasing despite intervention efforts, with over 50% of cases attributed to iron deficiency in some regions.
Children under five years bear substantial burden with a reported YLD of 1,252.88 per 100,000 population. Children are more likely to be anemic if stunted or having an anemic mother. Those with inflammatory bowel disease experience anemia as one of the most common extraintestinal manifestations.
Elderly populations show high anemia prevalence (42.2% in a Nigerian study), with predictors including hypoalbuminaemia, reduced food intake, and low monthly income.
Ethnic minorities remain understudied, with a study of Karen minorities in Thailand showing 27.9% anemia prevalence, with inherited hemoglobin disorders contributing to 49.3% of cases.
Disease-Specific Populations
Chronic kidney disease patients face affordability challenges with erythropoietin treatment in developing countries. Despite new HIF-PHIs approvals, safety concerns limit their use to maintenance dialysis patients only.
Cancer patients show significant treatment gaps, with many remaining refractory to erythropoietin. Those with chemotherapy-induced anemia face serious shortcomings in treatment rates and unsatisfactory outcomes.
Myelofibrosis patients encounter limited efficacy, durability, and tolerability of current therapies, with anemia representing a major unmet need that restricts access to JAK inhibition.
COVID-19 patients show significant correlation between anemia and disease severity, suggesting anemia should be considered an independent prognostic risk factor.
Treatment Challenges
Oral iron preparations prove inefficient for chronic disease patients due to limited tolerability and restricted enteral iron uptake during inflammation.
In β-thalassemia, there is no efficacious oral medication modifying anemia, with current developmental therapies requiring parenteral administration.
For multiple myeloma related anemia, current treatment strategies remain inadequate with many patients relying on transfusions.
Erythropoiesis stimulating agents used in chronic inflammatory diseases are associated with potentially hazardous side effects and poor outcomes.
Promising Approaches
Emerging therapies targeting the TGF-β-BMP/SMAD signaling pathway (luspatercept, momelotinib, imetelstat) represent important additions to treatment options.
Hypoxia-inducible factor prolyl hydroxylase inhibitors show promise compared to traditional treatments but require further safety evaluation.
Multidisciplinary, comprehensive strategies involving healthcare providers, school health teams, and community assets are recommended to address the complex nature of anemia across vulnerable populations.
Study Design Parameters
Study Design Parameters and Endpoints in Key Anemia Trials
Study Designs
Key anemia trials employ diverse methodological approaches including: - Large clinical trials like the EPIC study (1-year duration) evaluating deferasirox treatment - Randomized controlled trials such as the CREATE trial (2001) comparing early vs. late intervention with epoetin beta - Open-label, single-centre, phase IV trials for iron deficiency in pregnancy - Open-label, partially randomized designs like the Japanese phase 3 study (NCT02988973) - Retrospective reviews of patient records (e.g., IDA study from 2018-2020) - Cross-sectional comparative designs analyzing data from multiple countries - Quasi-experimental designs testing educational interventions
Study Populations
Trial populations vary based on anemia type: - Thalassemia major (n=1114), myelodysplastic syndromes (n=336), and sickle-cell disease (n=80) in the EPIC study - Non-dialysis-dependent CKD patients in the CREATE trial (600 patients) and Japanese phase 3 study (262 patients) - Pregnant women (200) between 14-21 weeks gestation with iron deficiency - Heart failure patients with anemia (1328) in registry studies - Pediatric patients (823) aged 6 months to 18 years in the HEMO-AI study
Primary Endpoints
Common primary outcome measures include: - Hemoglobin changes: Percentage with decreases ≥0.5 g/dL; mean change from baseline; concentration at specific timepoints - Hematological improvement: Proportion achieving ≥1.5 g/dL increase for 8 weeks without transfusions - Cardiovascular outcomes: Effect on left ventricular mass index; time to first cardiovascular event - Iron parameters: Relationships between liver iron concentration (LIC), transferrin saturation, labile plasma iron, and serum ferritin
Secondary Endpoints
Trials frequently assess: - Hemoglobin targets: Percentage achieving Hb ≥12 g/dL or increase ≥2 g/dL - Quality of life measures: Using Linear Analog Scale Assessment (LASA) and Functional Assessment in Cancer Therapy-Anemia (FACT-An) - Hospital outcomes: Duration, ICU length of stay, readmission rates - Iron metabolism: Changes in hepcidin level and other iron-related parameters - Blood cell parameters: Changes in leukocytes, neutrophils, and lymphocytes
Safety Endpoints
Safety monitoring includes: - Adverse events: Neutropenia, hypophosphatemia, fatigue - Gastrointestinal effects: Diarrhea, epigastralgia, vomiting, nausea - Cardiovascular complications: Heart failure hospitalization - Infection rates and mortality
Measurement Techniques
Various assessment methods are employed: - Magnetic resonance imaging (MRI) for liver iron concentration - Smartphone camera imaging (HEMO-AI study) - Cardiopulmonary exercise testing including peak VO₂ - Red blood cell mass (RBCM) profiles - Reticulocyte production index (RPI) to evaluate reticulocyte production ability
These diverse study designs and endpoints reflect the complexity of anemia management across different patient populations and clinical contexts.