Calliditas Therapeutics AB

An experimental medicine Otsuka Pharmaceutical acquired in a buyout six years ago has succeeded in a Phase 3 trial in a rare kidney disease, the company said Tuesday.The drug, called sibeprenlimab, met its main goal in a study in IgA nephropathy, or IgAN, a chronic condition that can lead to kidney failure. Upon a pre-specified, interim analysis after nine months of treatment, the company said sibeprenlimab therapy was associated with a statistically significant and clinically meaningful reduction, versus placebo, in the amount of protein in participants urine a marker of kidney health.Otsuka didnt provide specifics, making the magnitude of the drugs effects unclear. It also said, without details, that the drugs safety profile was consistent with previously reported data. But the company intends to discuss with the Food and Drug Administration the possibility of an accelerated approval filing while the study continues. The trial is expected to wrap up in early 2026.For Otsuka, the results are a measure of validation for its bet on Visterra, a Waltham, Massachusetts-based biotechnology startup and sibeprenlimabs developer. Visterra was founded in 2008 around a type of antibody drug discovery technology that originated at MIT. The company initially used that technology to develop drugs for infectious diseases before broadening its therapeutic scope, and tried, unsuccessfully, to go public in 2017.Otsuka stepped in a year later and acquired the company for $430 million in upfront cash. Visterra has since operated as a wholly owned subsidiary of Otsuka and has still been run by its longtime CEO, Brian Pereira. The company now has a pipeline of drugs mostly for immune diseases and led by sibeprenlimab, which, following Tuesdays study results, could become the first treatment of its kind for IgAN.Some estimates hold that upwards of 130,000 people in the U.S. have IgAN, a progressive autoimmune condition that damages the kidneys. While several therapies are available, there remains room for improvement, as patients with the disease still see their kidney function decline.The need for better therapies, along with a growing understanding of the diseases underlying biology, has made IgAN a focus of an increasing number of drug companies. Since 2023, Novartis, Biogenand Vertex Pharmaceuticals have all acquired biotechs with IgAN drugs in testing. Travere Therapeutics and Calliditas Therapeutics have recently launched IgAN medicines. Others, including Q32 Bio and Vera Therapeutics, are advancing would-be therapies as well.These emerging medicines are more commonly aimed at immune-mediated drivers of the disease, analysts at the investment bank Piper Sandler wrote in a recent report. Sibeprenlimab targets a protein dubbed APRIL thats implicated in the progression of IgAN. The drug Vertex acquired in a buyout of Alpine Immune Sciences aims at APRIL and a second protein called BAFF. Felzartamab, which Biogen acquired in its acquisition of HI-Bio, blocks the protein CD38.Otsukas trial results suggest that by targeting APRIL, we could provide a new therapeutic strategy for people living with this progressive kidney disease,“ said John Kraus, the executive vice president and chief medical officer of Otsukas pharmaceutical development and commercialization division, in a statement. '

STOCKHOLM, Sweden, Oct. 16, 2024 (GLOBE NEWSWIRE) -- Calliditas Therapeutics AB (Nasdaq Stockholm: CALTX) (“Calliditas”), today announced upcoming presence and data presentations at the American Society of Nephrology Kidney Week 2024 in San Diego, California on October 23 – 27, 2024. Presentations will highlight an open-label extension study of the efficacy and safety of Nefecon (TARPEYO® (budesonide) delayed release capsules)) in patients with primary immunoglobulin A nephropathy (IgAN), results from a real-world case series of IgAN patients, and additional insights from biomarker analysis. “We look forward to attending ASN Kidney Week and sharing additional data that reinforce Nefecon as a cornerstone treatment in IgAN,” said Richard Philipson, Chief Medical Officer at Calliditas. "This conference offers a great opportunity to exchange scientific knowledge with leading experts in nephrology and contribute to discussions around innovative treatments, including our potentially disease-modifying option." The oral presentation and poster presentation details are below. Following the meeting, they will be available on the Presentations and Publications page on Calliditas’ corporate website. Oral Presentation Details: Title: “NeflgArd Open-Label Extension: Efficacy and Safety of Nefecon in Patients with IgAN Who Completed the 2-Year Phase 3 Trial”Date and Time: October 25, 2024 from 4:40 PM to 4:50 PM PTLocation: Room 6D, Convention Center Poster Presentation Details: Title: “Results from a real-world case series of IgAN patients who received at least 9 months of Tarpeyo”Date and Time:Location: October 25, 2024 from 10:00 AM to 12:00 PM PT Title: Specificity of Nefecon in Targeting Pathogenic IgA in IgA Nephropathy While Preserving Systemic Humoral ImmunityDate and Time: October 25, 2024 from 10:00 AM to 12:00 PM PT Title: Impact of Nefecon on Complement Pathways in IgA Nephropathy: An Analysis of Lectin, Alternative, and Terminal PathwaysDate and Time: October 25, 2024 from 10:00 AM to 12:00 PM PT For more information, visit booth 1920 or visit the ASN Kidney Week 2024 website here. 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 PRECAUTIONSHypercorticism 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. 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). Immunosuppression and Increased Risk of Infection: Corticosteroids, including TARPEYO, suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Corticosteroids can: reduce resistance to new infections, exacerbate existing infections, increase the risk of disseminated infections, increase the risk of reactivation or exacerbation of latent infections, and mask some signs of infection. Corticosteroid-associated infections can sometimes be serious. Monitor for infection and consider TARPEYO withdrawal as needed. Avoid corticosteroid therapy, including TARPEYO, in patients with active or quiescent tuberculosis or hepatitis B infection; untreated fungal, bacterial, systemic viral, or parasitic infections; ocular herpes simplex; or Kaposi’s sarcoma. Avoid exposure to active, easily-transmitted infections (e.g., chickenpox, 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 or cataracts, or with a family history of diabetes or glaucoma, or with any other condition where corticosteroids may have unwanted effects. ADVERSE REACTIONS In clinical studies, the most common adverse reactions with TARPEYO (occurring in ≥5% of TARPEYO treated patients, and ≥2% higher than placebo) were peripheral edema (17%), hypertension (12%), muscle spasms (12%), acne (11%), headache (10%), upper respiratory tract infection (8%), face edema (8%), weight increased (7%), dyspepsia (7%), dermatitis (6%), arthralgia (6%), and white blood cell count increased (6%). 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 POPULATIONSPregnancy: 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 TARPEYOTARPEYO is an oral 4mg delayed release formulation of budesonide, 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. About Primary Immunoglobulin A NephropathyPrimary 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. 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. About Calliditas Calliditas Therapeutics is a biopharma company headquartered in Stockholm, Sweden, focused on identifying, developing, and commercializing novel treatments in orphan indications with significant unmet medical needs. Visit Calliditas.com for further information. For further information, please contact:Emma Santosegsantos@lifescicomms.com The information was sent for publication, through the agency of the contact persons set out above, on October 16, 2024, at 14.00 p.m. CET.

Purespring Therapeutics has reeled in £80 million ($105 million) to test two gene therapies in humans. The Sofinnova Partners-led Series B, disclosed Wednesday morning, will carry the London startup into an open-label Phase 1/2 next year for a kidney disease that has attracted a lot of attention in recent years from regulators and pharmaceutical acquirers. The startup’s lead gene therapy, PS-002, will be investigated for IgA nephropathy, or IgAN. The autoimmune and kidney disease, in which too much IgA protein stays in the kidneys, can lead to end-stage renal disease. Purespring anticipates it could snag an accelerated approval from its upcoming Phase 1/2, CEO Julian Hanak told Endpoints News . Travere , Novartis and Calliditas have received FDA nods in the past few years for their oral medicines. During that approval process, Purespring would plan to launch a confirmatory Phase 3 to get full approval, Hanak said. To fund that, Purespring would have a few options: raise a crossover round, go through an IPO and/or secure a commercialization partnership with a pharma company, the CEO said. Purespring’s experimental gene therapy goes after a particular cell type known as the podocyte, which is key to a kidney’s ability to operate. Judo Bio, a new siRNA startup that emerged earlier this week with $100 million, also plans to target podocytes to directly treat renal diseases. Purespring’s candidate “actually could work in conjunction and in complementation with some of the new therapies coming on the market,” Hanak said. “It’ll be a one-and-done therapy because it’s gene therapy and because podocytes are totally differentiated, non-dividing cells. Once the gene therapy is in them, we’ll get persistence of expression.” There’s been a wave of M&A in the IgAN field, with Novartis , Vertex and Biogen each dishing out more than $1 billion to buy clinical-stage drug developers Chinook, Alpine Immune Sciences and Human Immunology Biosciences, respectively, in the past 15 months. “I think the industry has woken up to the fact that this is a huge unmet medical need,” Hanak said. He initially joined Purespring as chief development officer in 2021, coming from Biogen, where he served as CMC leader for the gene therapy work acquired from Nightstar . Purespring could go into “any other complement-mediated kidney disease affecting podocytes, so lupus nephritis, ANCA vasculitis and so on,” Hanak said, but the initial focus is on IgAN and an undisclosed second indication that will enter the clinic before the Series B runs out in a few years. The startup could also ink partnerships over the coming two years, the CEO said. “Companies have been approaching us about access to our platform to deliver their genes to the kidney,” he added. It has an internal process development manufacturing setup, Hanak said, noting the company also works with Charles River for plasmid DNA and with Columbus, OH-based Andelyn Biosciences for vector supplies. “We have to get the cost of goods for the product down, which we’ve been able to do,” Hanak said. “Our dose is very low, 50-to-100-fold lower than a traditional, systemic gene therapy because of our platform.” Purespring derived from the work of University of Bristol professor Moin Saleem, who works in pediatric renal medicine. The company launched with a £45 million Series A from Syncona in November 2020. Syncona returned for the Series B alongside Forbion. The respective UK and Dutch investors have paired up with other gene therapy companies before, including a $170 million round for ophthalmology-focused Beacon Therapeutics in July. Other Purespring investors include Gilde Healthcare and British Patient Capital. The company has 46 employees and doesn’t plan to rapidly hire. “We have all the juice we need to deliver,” Hanak said.