Breakthrough Clinical Results
Coya Therapeutics announced that the FDA has accepted its Investigational New Drug (IND) application for COYA 302, a biologic combination therapy for amyotrophic lateral sclerosis (ALS). This acceptance triggers a $4.2 million milestone payment from Dr. Reddy's Laboratories, Coya's partner. COYA 302 is designed to enhance regulatory T cell function and suppress inflammation. The FDA acceptance allows Coya to initiate a Phase 2 multicenter, double-blind, placebo-controlled clinical trial to evaluate COYA 302's efficacy and safety in ALS patients. This represents a significant step forward in the development of a potential new treatment for ALS.
Key Highlights
- FDA accepts IND application for COYA 302 for ALS treatment.
- Phase 2 clinical trial to begin evaluating COYA 302's efficacy and safety.
- $4.2 million milestone payment received from Dr. Reddy's Laboratories.
- COYA 302 is a dual immunomodulatory biologic combination therapy.
Incidence and Prevalence
Global Incidence and Prevalence of Amyotrophic Lateral Sclerosis (ALS)
The worldwide prevalence of ALS is estimated at 4-6 per 100,000 population, with a global incidence of 1.5-2.7 per 100,000 per year that may be increasing. More recent data indicates the incidence is approximately 1-2.6 cases per 100,000 persons annually, with prevalence approximately 6 cases per 100,000.
The number of ALS cases globally is projected to increase from 222,801 in 2015 to 376,674 in 2040, representing a 69% increase predominantly due to aging populations, particularly in developing nations.
Regional variations in ALS epidemiology are significant:
In Europe, a study analyzing data from five population registers (Ireland, Netherlands, Italy, Scotland, and England) identified 6,274 ALS cases from a catchment population of about 34 million people. In Norway, the prevalence remained stable at 7.6 per 100,000, with a male:female ratio of 1.8. Central Italy reported a mean incidence of 3.5 per 100,000 person-years with a male-to-female ratio of 1.2. Italian prevalence ratios ranged from 6 per 100,000 for a 3-year period to 12.1 per 100,000 for a 21-year period.
In Asia, Japan reported an annual crude prevalence of 9.9 per 100,000 and an incidence of 2.2 per 100,000 person-years, with the 70-79 years age group showing the highest prevalence. A Beijing study reported an average yearly incidence of 0.8/100,000 persons, with a male-female ratio of 1.63:1.
In the United States, the National ALS Registry identified age-adjusted incidence rates of 1.7 (2014), 1.5 (2015), and 1.5 (2016) per 100,000 population, with an estimated 12,187 prevalent cases diagnosed with definite ALS between October 2010 and December 2011.
Ethnic variations are notable, with data suggesting a lower frequency in Hispanic and African groups compared to those of European descent. In South Africa, substantial differences in age- and sex-adjusted incidence rates were found: highest in European ancestry (2.62), lowest in African ancestry (0.56), and intermediate in mixed ancestry (1.09). A London study found adjusted incidence in people of African ancestry was 1.35 per 100,000 person-years and in those of European ancestry 1.97 per 100,000 person-years.
In Latin America, Uruguay had a crude prevalence of 2.19 per 100,000 persons, Chile reported 1.39 per 100,000, and Cuba showed 0.55 per 100,000, confirming lower incidence and prevalence in countries with admixed populations.
The COVID-19 pandemic may have affected reporting, with an Italian study showing a mean decrease in incidence of 5.8% during 2020-2021, possibly due to delayed diagnosis.
Genetic features of ALS in the Chinese population differ significantly from those in Caucasian populations, indicating an association between genetic susceptibility and population origin. Sporadic ALS accounts for 90-95% of cases, while the remaining 5-10% are hereditary (familial ALS).
Economic Burden
Economic Burden of Treating ALS in USA and Europe
United States
The most recent estimate (2023) shows that total annual costs per patient with ALS in the USA were estimated at US$ 69,475 (standardized to 2015 US$). The national economic burden of ALS in the USA was estimated at US$ 279-472 million.
A more recent US-based study (2023) estimated that the total national costs for ALS in the United States spanned approximately $212 million to $1.4 billion USD per year, consisting of direct costs associated with healthcare resource utilization and indirect costs. The national cost of approximately $1.02 billion USD per year (estimated using a prevalence of 16,055 cases) best aligns with prevalence estimates found in the targeted literature review.
A 2024 US study using insurance claims data found that annualized costs increased significantly as ALS progressed: early-stage ($31,411), middle-stage ($51,481), and late-stage ($121,903). The increase in costs was primarily driven by higher frequency of and cost per hospital admission.
A 2015 case study reported total disease-duration costs of $1,433,992 (85% paid by insurance, 9% paid by family, 6% paid by charities). The highest costs were for in-home caregivers ($669,150), ventilation ($212,430) and hospital care ($114,558).
Europe
In Germany (2023), total annual costs per patient with ALS were estimated at 83,060€. The cost-utility for ALS in Germany was estimated at 138,960€/QALY.
In Spain (2009), the mean annual cost per patient with ALS was 36,194€. The most important categories of costs were informal care, early retirement, medications, and orthopaedic devices.
In Denmark (2013), the annual mean excess health-related cost was 18,918€ for each ALS patient.
In Greece (2015), the total annual cost per ALS patient was 7,450€ (direct cost: 4,305€ or 57.8%, indirect cost: 3,145€ or 42.2%).
Common Findings
The main cost drivers across European countries were informal care and disease-modifying treatments. Disease progression and loss of individual autonomy were the main predictors of high costs.
Costs associated with ALS were greater than other neurological diseases. The economic burden increases with disease severity, as demonstrated by the US study showing costs increasing substantially with each disability milestone (non-invasive ventilation: $58,973 vs. hospice: $76,179).
Therapeutic and supportive measures to maintain patient autonomy may contribute to reducing high personal burden and long-term costs. The findings highlight the potential value of delaying progression into a more resource-intensive stage by diagnosing and adequately treating people living with ALS earlier in the disease course.
COYA 302 Clinical Trials Beyond ALS
Based on a comprehensive review of available information, there are currently no documented clinical trials of COYA 302 (a combination therapy of low-dose interleukin-2 and CTLA-4 Ig) for indications other than Amyotrophic Lateral Sclerosis (ALS).
The therapeutic combination of low-dose IL-2 with CTLA-4 Ig represents a novel approach that appears to be in early stages of clinical investigation, with current research efforts focused specifically on ALS.
While this combination therapy may have theoretical applications in other autoimmune disorders, neurodegenerative conditions, or inflammatory diseases due to the immunomodulatory properties of both components, no formal clinical trials exploring these potential applications have been registered or reported in the available literature.
The absence of non-ALS clinical trials for COYA 302 means there are no established intervention models, dosing regimens, or administration protocols for this combination therapy outside the ALS setting.
Researchers and clinicians interested in the potential applications of COYA 302 beyond ALS will need to monitor future clinical trial registrations and published research to determine if this therapeutic approach expands to address other medical conditions.