Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”), today announced seven abstract presentations, including a late-breaking poster presentation, highlighting additional analyses of the Phase 3 NefIgArd study at the upcoming American Society of Nephrology (ASN) Kidney Week 2023 in Philadelphia, PA, November 1-5, 2023.
Investigators will be presenting additional analyses of the Phase 3 NefIgArd study’s 2-year data with Nefecon (TARPEYO® (budesonide) delayed-release capsules/Kinpeygo®) for the treatment of Primary IgA nephropathy (IgAN). Presentations will highlight the statistically significant and clinically meaningful treatment benefit of Nefecon, including an oral presentation on the preservation of eGFR in patients with IgAN treated with Nefecon, as well as the Company’s late-breaking poster presentation that will showcase the long-term renal benefit with Nefecon in Chinese patients with primary IgAN. Additionally, pre-clinical data on the treatment of Alport syndrome with setanaxib will be presented following the Company’s recent orphan drug designations granted in the US and receiving a favourable opinion regarding orphan drug classification in Europe.
“We take great pride in the robust clinical evidence we have gathered and look forward to presenting additional findings at ASN from our Phase 3 NefIgArd trial that further establish the beneficial effect of Nefecon on eGFR,” said Richard Philipson, Chief Medical Officer at Calliditas. “These analyses, in addition to a presentation highlighting our new clinical program in the renal space targeting Alport syndrome, reinforce our dedication to expanding treatment options and establishing novel standards of care in rare kidney diseases.”
Poster presentation details are below and will be available on the Presentation and Publication page on the Calliditas’ corporate website following the meeting.
Oral Presentation Details:
Title: eGFR decline in patients with IgAN treated with Nefecon or placebo: Results from the 2-year NefIgArd Phase 3 trial
Presenter: Richard Lafayette, M.D., F.A.C.P., Stanford Healthcare
Date and Time: Thursday, November 2, 5:15 p.m. – 5:24 p.m. ET
Session: Glomerular Diseases: Clinical and Translational Studies [OR1402]
Presentation No.: TH-OR26 (Abstract 3938831)
Presentation Venue: Room 103 (Pennsylvania Convention Center)
Late-Breaking Poster Presentation Details:
Title: Long-term renal benefit with Nefecon in Chinese patients with primary immunoglobulin A nephropathy: 2-year NefIgArd trial results
Presenter: Hong Zhang
Date and Time: Thursday, November 2, 10:00 a.m. – 12:00 p.m. ET
Session: Late-Breaking Posters [LB-PO]
Presentation No.: TH-PO1123 (Abstract 3969868)
Presentation Venue: Exhibit Halls B–D (Pennsylvania Convention Center)
Poster Presentation Details:
Title: The NOX inhibitor setanaxib combined with ramipril reduces glomerular function decline and fibrosis in a mouse model of Alport syndrome
Presenter: Thierry Christophe
Date and Time: Thursday, November 2, 10:00 a.m. – 12:00 p.m. ET
Session: Genetic Diseases: Glomerulopathies – I [PO1202-1]
Presentation No.: TH-PO481 (Abstract 3940209)
Presentation Venue: Exhibit Halls B–D (Pennsylvania Convention Center)
Title: Analysis of the NefIgArd Part A study confirms Nefecon modulated proteins involved in the intestinal immune network for IgA production
Presenter: Roisin Thomas
Date and Time: Saturday, November 4, 10:00 a.m. – 12:00 p.m. ET
Session: Glomerular Diseases: Therapeutics [PO1402-1]
Presentation No.: SA-PO893 (Abstract 3941804)
Presentation Venue: Exhibit Halls B–D (Pennsylvania Convention Center)
Title: Analysis of the NefIgArd Part A study population confirms Nefecon reduces
levels of dietary antigen-specific IgA in patients with IgA nephropathy
Presenter: Victoria Cotton
Date and Time: Saturday, November 4, 10:00 a.m. – 12:00 p.m. ET
Session: Glomerular Diseases: Therapeutics [PO1402-1]
Presentation No.: SA-PO892 (Abstract 3941892)
Presentation Venue: Exhibit Halls B–D (Pennsylvania Convention Center)
Title: Modeling based on NefIgArd 2-year eGFR total slope predicts long-term clinical benefit of Nefecon in a real-world IgAN population
Presenter: Jonathan Barratt, M.B.Ch.B., Ph.D., University of Leicester
Date and Time: Saturday, November 4, 10:00 a.m. – 12:00 p.m. ET
Session: Glomerular Diseases: Therapeutics [PO1402-1]
Presentation No.: SA-PO886 (Abstract 3943328)
Presentation Venue: Exhibit Halls B–D (Pennsylvania Convention Center)
Title: TRF-budesonide (Nefecon) reduces serum biomarkers of lymphocyte
activation in IgA nephropathy, which correlate with changes in serum BAFF levels
Presenter: Nadia Nawaz, University of Leicester
Date and Time: Saturday, November 4, 10:00 a.m. – 12:00 p.m. ET
Session: Glomerular Diseases: Therapeutics [PO1402-1]
Presentation No.: SA-PO890 (Abstract 3941858)
Presentation Venue: Exhibit Halls B–D (Pennsylvania Convention Center)
For more information, visit the American Society of Nephrology (ASN) website here.
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%).
Drug interactions: Budesonide is a substrate for CYP3A4. Avoid use with potent CYP3A4 inhibitors, such as ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO. Intake of grapefruit juice, which inhibits CYP3A4 activity, can increase the systemic exposure to budesonide.
Use in specific populations
Pregnancy: The available data from published case series, epidemiological studies, and reviews with oral budesonide use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with IgAN. Infants exposed to in utero corticosteroids, including budesonide, are at risk for hypoadrenalism.
Please see Full Prescribing Information.
About TARPEYO1
Calliditas has introduced TARPEYO, the first FDA-approved therapy for the treatment of the autoimmune renal disease primary IgA Nephropathy, or IgAN, to reduce proteinuria in adults with primary IgAN who are at risk of rapid disease progression, generally a UPCR≥1.5g/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 benefit in a confirmatory clinical trial.
TARPEYO is an oral, delayed release formulation of budesonide, a corticosteroid with potent glucocorticoid activity and weak mineralocorticoid activity that undergoes substantial first pass metabolism. TARPEYO is as a 4 mg delayed release capsule and is enteric coated and designed to remain intact until it reaches the ileum. Each capsule contains coated beads of budesonide that target mucosal B-cells present in the ileum, including the Peyer’s patches, which are responsible for the production of galactose-deficient IgA1 antibodies (Gd-Ag1) causing IgA nephropathy. It is unclear to what extent TARPEYO’s efficacy is mediated via local effects in the ileum vs systemic effects.
About the NeflgArd Study
The global clinical trial NefIgArd is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of TARPEYO 16 mg once daily vs placebo in adult patients with primary IgAN (N=364), as an addition to optimized RAS inhibitor therapy. Part A of the study included a 9-month blinded treatment period and a 3-month follow-up period. The primary endpoint was UPCR, and eGFR was a secondary endpoint. Part B included a 12-month observational period off drug and assessed eGFR over the entire 2-year period for patients who were treated with the TARPEYO or placebo regimen in Part A. The full NefIgArd trial met its primary endpoint. Topline data from the full NefIgArd study were reported on March 12, 2023.
About Primary Immunoglobulin A Nephropathy
Primary immunoglobulin A nephropathy (IgA nephropathy or IgAN or Berger’s Disease) is a rare, progressive, chronic autoimmune disease that attacks the kidneys and occurs when galactose-deficient IgA1 is recognized by autoantibodies, creating IgA1 immune complexes that become deposited in the glomerular mesangium of the kidney.2,3 This deposition in the kidney can lead to progressive kidney damage and potentially a clinical course resulting in end- stage renal disease. IgAN most often develops between late teens and late 30s.3,4
For further information, please contact:
Åsa Hillsten, Head of IR, Calliditas
Tel.: +46 76 403 35 43, E-Mail: asa.hillsten@calliditas.com
The information was sent for publication, through the agency of the contact persons set out above, on October 19 at 8.00 a.m. CET.