(Información remitida por la empresa firmante)
STOCKHOLM, Aug. 15, 2023 /PRNewswire/ — Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) (“Calliditas”) today announcedpublication in The Lancet of the full data from the Phase 3 NefIgArd Study with Nefecon® (TARPEYO® (budesonide) delayed release capsules/Kinpeygo®) in adults with Primary IgA nephropathy (IgAN). The Phase 3 trial met the primary endpoint, estimated glomerular filtration rate (eGFR), with Nefecon demonstrating significant kidney protective effect over placebo.
“IgAN is a severe debilitating disease leading to end-stage kidney disease in more than 50% of the patients. The full results from NefIgArd study demonstrate the ability of Nefecon to slow kidney function deterioration and as such to slow down the disease progression and delay the need for dialysis and kidney transplantation,” said Jonathan M. Barratt, M.D., Mayer Professor of Renal Medicine, University of Leicester. “These results also support the key role of the gut immune system in the pathogenesis of IgAN and the differentiated effect of Nefecon treating the disease at its origin.”
The analysis published in The Lancet shows that Nefecon demonstrated a highly statistically significant and clinically relevant benefit compared to placebo in eGFR over the two-year period of 9-months of treatment with Nefecon and 15-months of follow-up off drug. After the two-year period, there was a 6.11 mL/min/1.73 m2 decline in eGFR in the Nefecon arm compared with a 12.0 mL/min/1.73 m2 decline in the placebo arm, corresponding to a difference in two-year eGFR total slope of 2.95 mL/min/1.73m2 per year (p0·0001).
The reduction in UPCR observed with Nefecon treatment was durable, reflecting a long-lasting treatment effect during the 15-month follow-up period off treatment. Patients treated with Nefecon maintained a greater than 30% proteinuria reduction from the end of the 9-month treatment through the entire follow-up period, with a reduction in UPCR of over 50% observed at 12 months.
Nefecon was generally well tolerated, with the majority of treatment-emergent adverse events (TEAE) being mild or moderate, and with TEAEs leading to discontinuation of study drug in 10% of patients. Objective measures of mean weight and blood pressure showed non-clinically relevant changes.
“The data demonstrated supportive 2-year total slope analyses that were not only statistically significant but also clinically meaningful, showcasing a sustained treatment benefit. The eGFR benefit was observed across the entire study population, irrespective of baseline urine protein-to-creatinine ratio (UPCR),” said Richard Lafayette, M.D., F.A.C.P., Stanford Healthcare and lead author of the publication. “The sustained reduction of proteinuria and the protective effect on kidney function support the disease-modifying effect of Nefecon. These robust results provide new hope for patients and reinforce Nefecon’s potential to make a meaningful difference in the lives of those affected by this challenging disease.”
“We are thrilled to see the NefIgArd Phase 3 data published in The Lancet, highlighting these important results for the IgAN patient community,” said Renée Aguiar-Lucander, Chief Executive Officer at Calliditas. “The established long-term eGFR benefit reflects Nefecon’s ability to slow kidney function decline by targeting the origin of the disease and providing a differentiated and disease-modifying treatment alternative.”
Nefecon is currently approved under accelerated approval to reduce proteinuria in adults with Primary IgAN at risk of rapid disease progression, generally a UPCR =1.5 g/g. In June 2023, Calliditas submitted a supplemental new drug application (sNDA) to the United States Federal Drug Administration (FDA) seeking full approval based on the full NefIgArd study data. Calliditas is supporting its partner STADA Arzneimittel AG with the filing for full approval with the European Commission and the UK MHRA in 2H of 2023.
The peer-reviewed article in The Lancet can be viewed here.
For further information, please contact:
Åsa Hillsten, Head of Investor Relations, Calliditas
Tel.: +46 76 403 35 43, email: asa.hillsten@calliditas.com
The information was sent for publication, through the agency of the contact persons set out above, on August 15, 2023 at 08:00 a.m. CET.
Indication
TARPEYO® (budesonide) delayed release capsules is a corticosteroid indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) =1.5 g/g.
This indication is approved under accelerated approval based on a reduction in proteinuria. It has not been established whether TARPEYO slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory clinical trial.
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 [see Dosing and Administration] or switching between corticosteroids, monitor for signs of adrenal axis suppression.
Patients with moderate to severe hepatic impairment (Child-Pugh Class B and C, respectively) could be at an increased risk of hypercorticism and adrenal axis suppression due to an increased systemic exposure to oral budesonide. Avoid use in patients with severe hepatic impairment (Child-Pugh Class C). Monitor for increased signs and/or symptoms of hypercorticism in patients with moderate hepatic impairment (Child-Pugh Class B).
Risks of immunosuppression: Patients who are on drugs that suppress the immune system are more susceptible to infection than healthy individuals. Chicken pox and measles, for example, can have a more serious or even fatal course in susceptible patients or patients on immunosuppressive doses of corticosteroids. Avoid corticosteroid therapy in patients with active or quiescent tuberculosis infection; untreated fungal, bacterial, systemic viral, or parasitic infections; or ocular herpes simplex. Avoid exposure to active, easily transmitted infections (eg., chicken pox, measles). Corticosteroid therapy may decrease the immune response to some vaccines.
Other corticosteroid effects: TARPEYO is a systemically available corticosteroid and is expected to cause related adverse reactions. Monitor patients with hypertension, prediabetes, diabetes mellitus, osteoporosis, peptic ulcer, glaucoma, cataracts, a family history of diabetes or glaucoma, or with any other condition in which corticosteroids may have unwanted effects.
Adverse reactions: In clinical studies, the most common adverse reactions with TARPEYO (occurring in =5% of TARPEYO patients and =2% higher than placebo) were hypertension (16%), peripheral edema (14%), muscle spasms (13%), acne (11%), dermatitis (7%), weight increase (7%), dyspnea (6%), face edema (6%), dyspepsia (5%), fatigue (5%), and hirsutism (5%).
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