Calliditas Therapeutics AB announced additional data analyses from the 2-year Phase 3 NeflgArd trial evaluating Nefecon (TARPEYO (budesonide) delayed-release capsules/Kinpeygo) in patients with IgA nephropathy (IgAN), were presented at the ISN World Congress of Nephrology in Buenos Aires, Argentina on April 13-16, 2024. Poster Title: "Nefecon treatment provides kidney benefits for patients with IgAN that extend to those with low levels of UPCR: A sub-analysis of the phase III NefIgArd trial". An extended analysis of patients with baseline UPCR levels above and below 0.8 g/g was performed to further explore the potential benefits of Nefecon.

In the full analysis involving 364 patients regardless of baseline UPCR, Nefecon treatment consistently improved the estimated glomerular filtrate rate (eGFR) over the 2-year study period compared to placebo. 72 patients with a baseline UPCR <0.8 g/g experienced sustained eGFR improvement (p=0.0026), which persisted for up to 18 months after treatment initiation, even after the treatment cessation at month 9. Those patients also achieved an eGFR slope of -0.25 mL/min/1.73 m2 per year, indicating that Nefecon treatment may support them in reaching the RaDaR treatment target of an eGFR decline of <1 mL/min/1.73 m2 per year. This objective is pivotal in mitigating the risk of kidney failure in their lifetime.

Poster Title: "eGFR decline in patients with IgAN treated with Nefecon or placebo: Results from the 2-year NefIgArd Phase 3 trial". During the 9-month treatment period, Nefecon showed a 30% reduction in UPCR compared to the placebo, sustained for 2 years. The percentage of patients with a confirmed 30% reduction in eGFR or kidney failure was lower in the Nefecon arm compared to placebo, and the time to such events was significantly delayed with Nefecon (hazard ratio [HR] 0.45; 95% confidence interval 0.26, 0.75]; p=0.0014 [1-sided]).

Supplementary analysis with rescue medication yielded similar results, irrespective of the handling of rescue medication: Rescue medication counted as an event: HR 0.51 (95% CI 0.33, 0.79), Regardless of rescue medication: HR 0.44 (95% CI 0.27, 0.71). The treatment effect of Nefecon on the risk of kidney function decline was consistent regardless of baseline UPCR. These findings strongly suggest preserved kidney function and provide support for Nefecon as a disease-modifying therapy in patients with IgAN.

Poster Title: Nefecon effect on quality of life in patients with IgAN: SF-36 results from the Phase 3 NefIgArd trial". The 2-year results of quality of life (QoL) analyses based on 36-Item Short Form Survey (SF-36) assessments at 9 and 24 months revealed no meaningful differences in any QoL domain between Nefecon and placebo groups after 9 months of treatment. These SF-36 scores remained consistent after 15 months of off-drug observational follow-up further supporting the benefit/risk profile of Nefecon.

Poster Title: "Nefecon treatment response in Asian and White patient populations with immunoglobulin A nephropathy: A 2-year analysis of the Phase 3 NefIgArd trial". The responses to Nefecon treatment from the full 2-year NefIgArd trial were assessed in patients identifying as Asian (n=83) or White (n=275). Regardless of race and ethnicity, Nefecon showed a favorable change in eGFR compared to placebo of 5.5 mL/min/1.73 m2 in Asian patients and 4.8 mL/min/1.73 m2 in White patients.

Nefecon also demonstrated greater reductions in UPCR at 9 and 24 months with notable delays in kidney function decline events. These effects were consistent across races and ethnicities. Additionally, Nefecon significantly reduced the rate of microhematuria in both Asian and White patients.

Overall, these findings highlight Nefecon's efficacy and tolerability across different racial and ethnic groups. Indication: TARPEYO is indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. Important Safety Information: Contraindications: TARPEYO is contraindicated in patients with hypersensitivity to budesonide or any of the ingredients of TARPEYO.

Serious hypersensitivity reactions, including anaphylaxis, have occurred with other budesonide formulations. Warnings and Precautions: Hypercorticism and adrenal axis suppression: When corticosteroids are used chronically, systemic effects such as hypercorticism and adrenal suppression may occur. Corticosteroids can reduce the response of the hypothalamus-pituitary-adrenal (HPA) axis to stress.

In situations where patients are subject to surgery or other stress situations, supplementation with a systemic corticosteroid is recommended. When discontinuing therapy or switching between corticosteroids, monitor for signs of adrenal axis suppression.