Calliditas Therapeutics Shares Data at 61st European Renal Association Congress

Calliditas Therapeutics AB recently presented new findings regarding their treatment Nefecon (marketed as TARPEYO®) and a real-world analysis of systemic glucocorticoid (SGC) use in patients with IgA nephropathy (IgAN) at ERA 2024. The event, held both virtually and in Stockholm from May 23-26, 2024, provided a platform for these significant revelations.

The efficacy analysis demonstrated that a nine-month course of Nefecon resulted in a better estimated glomerular filtration rate (eGFR) outcome compared to continuous use of sparsentan over two years. This comparison was established through a matching-adjusted indirect comparison (MAIC), which is recognized as a valid method for assessing treatment effects when direct head-to-head trial data is unavailable. The analysis showed that Nefecon resulted in a mean eGFR improvement of 5.68 mL/min/1.73 m² after nine months, highlighting the drug’s rapid and sustained efficacy.

Additionally, the real-world analysis of SGCs like Prednisone and Prednisolone indicated significant drawbacks, including severe side effects and high healthcare costs for IgAN patients. This analysis underscores the need for careful consideration of SGCs due to their associated risks, such as severe infections and frequent hospital visits.

Richard Philipson, Chief Medical Officer of Calliditas, emphasized the importance of these findings in advancing treatment for IgAN, noting that TARPEYO, as the only approved therapy targeting the production of Gd-IgA1, could become a foundational treatment for this condition.

The study details were shared in two presentations:

1. “Matching-adjusted indirect comparison of eGFR in patients with immunoglobulin A nephropathy treated with Nefecon (TRF budesonide) or sparsentan”: This presentation detailed the MAIC methodology used to compare Nefecon with sparsentan. It highlighted the superior effects of Nefecon on eGFR over various time points, demonstrating its potential benefits in managing IgAN.

2. “Real-world challenges associated with the use of systemic glucocorticoids in a US IgAN cohort”: This analysis followed KDIGO guidelines, revealing the significant side effects and healthcare resource utilization associated with SGC use in high-risk IgAN patients. The findings pointed out the need to weigh the benefits of SGCs against their potential toxicity and costs carefully.

Nefecon's indication is to reduce kidney function loss in adults with primary IgAN at risk for disease progression. However, its use comes with several safety considerations:

- Contraindications: It should not be used in patients with hypersensitivity to budesonide or any of its ingredients, as serious allergic reactions can occur.
- Warnings and Precautions: Long-term corticosteroid use can lead to systemic effects like hypercorticism and adrenal suppression, necessitating additional corticosteroid supplementation during stress situations. Patients with moderate to severe hepatic impairment should be monitored closely due to increased risks.
- Risks of Immunosuppression: Patients on immunosuppressive therapies are more susceptible to infections. TARPEYO should be avoided in patients with active infections or those exposed to easily transmitted diseases.
- Other Corticosteroid Effects: Regular monitoring is essential for patients with conditions like hypertension, diabetes, osteoporosis, and glaucoma, as corticosteroids can exacerbate these conditions.
- Adverse Reactions: Common side effects include peripheral edema, hypertension, muscle spasms, acne, headaches, and upper respiratory tract infections.
- Drug Interactions: Budesonide is metabolized by CYP3A4, and its use should be avoided with potent CYP3A4 inhibitors. Grapefruit juice, which inhibits CYP3A4, should also be avoided.

TARPEYO is formulated as an oral 4mg delayed-release capsule designed to deliver budesonide directly to the ileum, targeting the mucosal B-cells responsible for Gd-IgA1 production in IgAN.

IgA nephropathy is a chronic autoimmune disease leading to kidney damage and potentially end-stage renal disease, often beginning in late adolescence to early adulthood. This condition arises when autoantibodies recognize galactose-deficient IgA1, forming immune complexes that deposit in kidney tissue.

These findings highlight the potential of TARPEYO in transforming the treatment landscape for IgAN, offering a more effective and targeted approach compared to traditional systemic therapies.

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