Latest news with #LUPUS2025
Business Wire
22-05-2025
- Business
- Business Wire
Ventus Therapeutics to Present Phase 1 Results for VENT-03, a First-in-Class cGAS Inhibitor, at LUPUS 2025
WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)-- Ventus Therapeutics, a clinical-stage biopharmaceutical company advancing two Phase 2 small-molecule programs for immunological, inflammatory, and neurological disorders, today announced the upcoming presentation of the Phase 1 safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) results of VENT-03 at the 16 th International Congress on Systemic Lupus Erythematosus (LUPUS 2025), taking place from May 21-24 in Toronto, Canada. VENT-03 is the first oral small-molecule cyclic GMP-AMP synthase (cGAS) inhibitor to successfully complete a first-in-human Phase 1 study and was discovered using Ventus' proprietary ReSOLVE ® platform. Data from the Phase 1 study, initially announced in October 2024, showed that VENT-03 was safe and well tolerated at all tested dose levels with a favorable PK profile enabling once-daily dosing. 'We are pleased to present at LUPUS 2025 the first reported Phase 1 results for a cGAS inhibitor, demonstrating that VENT-03 has the potential to become a first- and best-in-class therapy for lupus and other autoimmune disorders,' said Marcelo Bigal, M.D., Ph.D., President and CEO of Ventus. 'Ventus is eager to unlock the potential of cGAS inhibition across multiple autoimmune diseases, with the first Phase 2 trial evaluating VENT-03 in patients with lupus to commence later this year.' The Phase 1, double-blind, placebo-controlled, first-in-human trial included 72 healthy adult volunteers across single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. The study results show that VENT-03 was safe and well tolerated up to single doses of 2000 mg once daily (QD) and multiple dosing of 900 mg QD for 10 days, which are substantially higher than our anticipated Phase 2 dose. The proportion of participants with adverse events (AEs) in the MAD cohorts was similar for VENT-03 and placebo (79% vs. 75%), and the vast majority of AEs in the study were mild and considered unrelated to the study drug. The PK profile of VENT-03 supports once-daily dosing with or without food, and the PD results demonstrate robust target engagement, supporting further clinical development. 'More than five million people worldwide are estimated to have a form of lupus. These patients can experience disease progression and severe symptoms that can affect quality of life, and currently available treatment options may not be able to adequately address these symptoms for all patients,' said Mona Kotecha, M.D., Chief Medical Officer of Ventus. 'Through targeting cGAS, VENT-03 has the potential to become a safer and more effective treatment option for patients in need, addressing key unmet needs across multiple organ systems while providing the additional benefit of once-daily oral dosing. We look forward to sharing these results and to connecting with the wider lupus community at LUPUS 2025.' Details of the poster presentation are as follows: Title: Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Healthy Volunteers of VENT-03, a Novel cGAS Inhibitor for the Treatment of Systemic Lupus Erythematosus Authors: Xavier Valencia, Kelly Pike, Loraine Warner, Conrad Winters, Jeanne Stewart, Ramsay Beveridge, Patrick Cyr, Nadine Fradet, Amandine Chefson, Ofer Spiegelstein Abstract Number: Poster 286 Session Date: May 22 nd About cGAS cGAS is an intracellular pattern recognition receptor that is activated after binding to double-stranded DNA (dsDNA) in the cytoplasm. The presence of dsDNA in the cytoplasm is often the result of cellular dysfunction, which is a hallmark of many autoimmune and inflammatory diseases. Activation of cGAS leads to cGAMP formation, activation of STING, pronounced inflammation, and tissue damage. In both patients and preclinical models of disease, the cGAS pathway has been shown to be a key driver of lupus and other inflammatory diseases, such as rheumatoid arthritis, systemic sclerosis, dermatomyositis, and Sjögren's disease. About Ventus Therapeutics Ventus Therapeutics is a clinical-stage biopharmaceutical company advancing two Phase 2 small-molecule programs for immunological, inflammatory, and neurological disorders. Using its proprietary drug discovery platform, ReSOLVE ®, the company has established a robust pipeline, including two wholly-owned programs. VENT-03 is a first-in-class, oral cGAS inhibitor expected to enter Phase 2 development for lupus in 2025. VENT-02 is a best-in-class, brain-penetrant, oral NLRP3 inhibitor in Phase 2 for Parkinson's disease, and is expected to enter Phase 2 development for osteoarthritis in obese patients later in 2025. In addition, Ventus has out-licensed VENT-01, a peripherally-restricted, oral NLRP3 inhibitor in Phase 1, to Novo Nordisk A/S. For more information, please visit and engage with Ventus on LinkedIn.
Business Wire
22-05-2025
- Health
- Business Wire
New AURORA 1 Analysis: LUPKYNIS-Based Triple Immunosuppressive Therapy Yields Deep Proteinuria Reduction in Lupus Nephritis
ROCKVILLE, Md. & EDMONTON, Alberta--(BUSINESS WIRE)--Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) (Aurinia or the Company), today announced that a post-hoc analysis of the 52-week, Phase 3 AURORA 1 study showed that lupus nephritis (LN) patients who received triple immunosuppressive therapy with LUPKYNIS ® (voclosporin), mycophenolate mofetil (MMF), and low-dose glucocorticoids achieved lower proteinuria targets at substantially higher rates compared to patients in the control group who received mycophenolate mofetil (MMF) and low-dose glucocorticoids alone. The analysis assessed the achievement of urine protein creatine ratio (UPCR) targets of ≤0.4 g/g, ≤0.3 g/g, ≤0.2 g/g (classified as ultra-low UPCR), and ≤0.1 g/g in LN patients treated with LUPKYNIS-based triple immunosuppressive therapy compared to patients in the control group. Of the 357 patients in AURORA 1, 60.9% in the triple immunosuppressive therapy group (N=109) achieved a UPCR of ≤0.4 g/g at least once during the study compared to 37.1% of patients in the control group (N=66). Patients in the triple immunosuppressive therapy group also achieved higher rates of all other UPCR targets compared to patients in the control group. Adverse event rates were comparable in both groups. 'It is widely known that no level of proteinuria is safe for nephrons and that early reductions in proteinuria are predictive of better long-term kidney outcomes. Yet, UPCR endpoints have varied widely across clinical trials and in clinical practice,' said lead study author Maria Dall'Era, M.D., Professor of Medicine in the Division of Rheumatology, University of California, San Francisco. 'This analysis shows that achieving UPCR targets of ≤0.4 g/g may be a feasible goal and that a voclosporin-based triple immunosuppressive therapy regimen can reduce proteinuria to profoundly low levels in a proportion of patients.' An additional post-hoc analysis from the AURORA 1 study evaluated lipidomic profiles in LN patients based on achievement of proteinuria reductions, including ultra-low UPCR, at Week 52. The analysis found a distinct lipidomic profile in patients who achieved ultra-low UPCR. This analysis builds upon a previous analysis of AURORA 1 in which patients who received triple immunosuppressive therapy with LUPKYNIS achieved significantly greater improvements in total and low-density lipoprotein (LDL) cholesterol compared to those in the control group. While further research is needed to clarify the role of certain lipids in the biochemistry of LN patients, these preliminary findings suggest that attaining ultra-low UPCR targets may provide additional benefits to LN patients and contribute to modification of cardiovascular disease risk. An analysis of real-world baseline data from ENLIGHT-LN, a U.S.-based prospective, observational registry of adult LN patients treated with LUPKYNIS, was also presented at LUPUS 2025. 'The data presented at LUPUS 2025 highlight the critical role of LUPKYNIS in improving health outcomes for LN patients. Early reduction of proteinuria to the lowest possible levels and long-term preservation of kidney health are key goals of LN therapy. These data provide compelling evidence that LUPKYNIS-based therapy can achieve significantly lower UPCR targets, potentially reducing the risk of significant kidney damage and other comorbidities,' said Dr. Greg Keenan, Chief Medical Officer of Aurinia. Following is the complete guide to Aurinia's accepted abstracts at LUPUS 2025: Title: Achievement of Proteinuria Less Than 0.4 G/G in the Phase 3 AURORA 1 Study of Voclosporin in Lupus Nephritis Authors: Maria Dall'Era, Brad Rovin, Salem Almaani, Lily Cipolla, Vanessa Birardi, Ernie Yap Date: Thursday, May 22 Time: 2:20 PM ET Abstract Number: 232 Title: Baseline Demographics, Clinical Characteristics, and Treatment Regimens of an Initial Cohort of Patients Receiving Voclosporin for Lupus Nephritis in the Enlight-LN Registry Authors: Laura Geraldino-Pardilla, Leanna Wise, Mohammad Kamgar, Niloofar Nobakht, Lily Cipolla, Lucy Hodge, Keelin Da'Lee Poster Session: Lupus Nephritis-Clinical Date/Time: On display for duration of meeting Abstract Number: 249 Title: Attainment of Ultra-Low Levels of UPCR in the AURORA 1 Study Associated with Alterations in the Circulating Lipidome Authors: Farsad Afshinnia, Subramaniam Pennathur, Michelle Zubrycki, Linda Rehaume, Lucy Hodge Date: Thursday, May 22 Time: 12:10 – 1:10 PM ET Abstract Number: 252 About LUPKYNIS LUPKYNIS is a second generation calcineurin inhibitor with a dual mechanism of action, acting as an immunosuppressant through inhibition of T-cell activation and cytokine production and promoting podocyte stability in the kidney. The AURORA Clinical Program, comprised of the AURORA 1 pivotal trial and AURORA 2 extension trial, demonstrated the importance of triple immunosuppressive therapy with LUPKYNIS, mycophenolate mofetil, and low-dose glucocorticoids to preserve kidney health in lupus nephritis patients without reliance on chronic high-dose glucocorticoids. It is the only clinical program in lupus nephritis to include three years of triple immunosuppressive therapy. About Aurinia Aurinia Pharmaceuticals is a fully integrated biopharmaceutical company focused on delivering therapies to people living with autoimmune diseases with high unmet medical needs. In January 2021, the Company introduced LUPKYNIS ® (voclosporin), the first FDA-approved oral therapy dedicated to the treatment of adult patients with active lupus nephritis. Aurinia is also developing AUR200, a differentiated, potential best-in-class therapy for autoimmune diseases that targets both BAFF (B-cell Activating Factor) and APRIL (A Proliferation-Inducing Ligand). INDICATION AND IMPORTANT SAFETY INFORMATION INDICATION LUPKYNIS is indicated in combination with a background immunosuppressive therapy regimen for the treatment of adult patients with active lupus nephritis (LN). Limitations of Use: Safety and efficacy of LUPKYNIS have not been established in combination with cyclophosphamide. Use of LUPKYNIS is not recommended in this situation. IMPORTANT SAFETY INFORMATION Increased risk for developing malignancies and serious infections with LUPKYNIS or other immunosuppressants that may lead to hospitalization or death. CONTRAINDICATIONS: LUPKYNIS is contraindicated in patients taking strong CYP3A4 inhibitors because of the increased risk of acute and/or chronic nephrotoxicity, and in patients who have had a serious/severe hypersensitivity reaction to LUPKYNIS or its excipients. WARNINGS AND PRECAUTIONS Lymphoma and Other Malignancies: Immunosuppressants, including LUPKYNIS, increase the risk of developing lymphomas and other malignancies, particularly of the skin. The risk appears to be related to increasing doses and duration of immunosuppression rather than to the use of any specific agent. Serious Infections: Immunosuppressants, including LUPKYNIS, increase the risk of developing bacterial, viral, fungal, and protozoal infections, including opportunistic infections. These infections may lead to serious, including fatal, outcomes. Nephrotoxicity: LUPKYNIS, like other calcineurin inhibitors (CNIs), may cause acute and/or chronic nephrotoxicity. The risk is increased when CNIs are concomitantly administered with drugs associated with nephrotoxicity. Monitor eGFR regularly. Hypertension: Hypertension is a common adverse reaction of LUPKYNIS therapy and may require antihypertensive therapy. Monitor blood pressure regularly. Neurotoxicity: LUPKYNIS, like other CNIs, may cause a spectrum of neurotoxicities: severe include posterior reversible encephalopathy syndrome (PRES), delirium, seizure, and coma; others include tremor, paresthesia, headache, and changes in mental status and/or motor and sensory functions. Monitor for neurologic symptoms. Hyperkalemia: Hyperkalemia, which may be serious and require treatment, has been reported with CNIs, including LUPKYNIS. Concomitant use of agents associated with hyperkalemia may increase the risk for hyperkalemia. Monitor serum potassium levels periodically. QTc Prolongation: LUPKYNIS prolongs the QTc interval in a dose-dependent manner when dosed higher than the recommended lupus nephritis therapeutic dose. The use of LUPKYNIS in combination with other drugs that are known to prolong QTc may result in clinically significant QT prolongation. Immunizations: Avoid the use of live attenuated vaccines during treatment with LUPKYNIS. Inactivated vaccines noted to be safe for administration may not be sufficiently immunogenic during treatment with LUPKYNIS. Pure Red Cell Aplasia: Cases of pure red cell aplasia (PRCA) have been reported in patients treated with another CNI immunosuppressant. If PRCA is diagnosed, consider discontinuation of LUPKYNIS. Drug-Drug Interactions: Avoid co-administration of LUPKYNIS and strong CYP3A4 inhibitors or with strong or moderate CYP3A4 inducers. Co-administration of LUPKYNIS with strong CYP3A4 inhibitors is contraindicated. Reduce LUPKYNIS dosage when co-administered with moderate CYP3A4 inhibitors. Avoid use of LUPKYNIS with strong or moderate CYP3A4 inducers. ADVERSE REACTIONS The most common adverse reactions (≥3%) were glomerular filtration rate decreased, hypertension, diarrhea, headache, anemia, cough, urinary tract infection, abdominal pain upper, dyspepsia, alopecia, renal impairment, abdominal pain, mouth ulceration, fatigue, tremor, acute kidney injury, and decreased appetite. SPECIFIC POPULATIONS Pregnancy: Avoid use of LUPKYNIS. Lactation: Consider the mother's clinical need for LUPKYNIS and any potential adverse effects to the breastfed infant when prescribing LUPKYNIS to a lactating woman. Renal Impairment: LUPKYNIS is not recommended in patients with baseline eGFR ≤45 mL/min/1.73 m 2 unless benefit exceeds risk. If used in this population, reduce LUPKYNIS dose. Hepatic Impairment: For mild or moderate hepatic impairment, reduce LUPKYNIS dose. Avoid use with severe hepatic impairment. Please see Prescribing Information, including Boxed Warning, and Medication Guide for LUPKYNIS. References Dall'Era M. et al. Achievement of Proteinuria Less Than 0.4 G/G in the Phase 3 AURORA 1 Study of Voclosporin in Lupus Nephritis. Presented at LUPUS 2025 Congress, 2025, Toronto, CA. Geraldino-Pardilla L. et al. Baseline Demographics, Clinical Characteristics, and Treatment Regimens of an Initial Cohort of Patients Receiving Voclosporin for Lupus Nephritis in the Enlight-LN Registry. Presented at LUPUS 2025 Congress, 2025, Toronto, CA. Afshinnia F. et al. Attainment of Ultra-Low Levels of UPCR in the AURORA 1 Study Associated with Alterations in the Circulating Lipidome. Presented at LUPUS 2025 Congress, 2025, Toronto, CA. Arriens C. et al. Arthritis Care & Research. Vol. 75, No. 7, July 2023, pp 1399–1408.
