buloxibutid (C21) / Vicore Pharma, Nippon Shinyaku - LARVOL DELTA

Vicore Announces Presentations at the 2025 American Thoracic Society International Conference (ACCESSWIRE) - "Vicore Pharma Holding AB...announced that the Company will present multiple oral presentations and posters at the 2025 American Thoracic Society (ATS) International Conference, taking place in San Francisco, California from May 16 - May 21, 2025. These posters and presentations will highlight new translational data demonstrating buloxibutid's unique upstream mechanism of action for the treatment of idiopathic pulmonary fibrosis (IPF), a further analysis of buloxibutid's Phase 2a data in IPF patients reflecting disease-modifying potential, and the patient-centric approaches that Vicore has taken in both the ongoing Phase 2b ASPIRE study in IPF patients and in digital health innovation."

Clinical data • Idiopathic Pulmonary Fibrosis

The AIR Phase 2 Trial of the Angiotensin II Type 2 Receptor Agonist, Buloxibutid, in Individuals With Idiopathic Pulmonary Fibrosis: A Responder Analysis (ATS 2025) - No abstract available

Clinical • P2 data • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Buloxibutid Potently Inhibits Fibrosis Biomarkers in the Scar-in-a-Jar Primary Human Lung Fibroblast Assay (ATS 2025) - No abstract available

Fibrosis • Immunology

ASPIRE Trial in Idiopathic Pulmonary Fibrosis: A Patient Experience-focused Phase 2b Randomized, Double-blind, Placebo-controlled, Multicenter Trial of the Novel Angiotensin II Type 2 Receptor Agonist Buloxibutid (ATS 2025) - No abstract available

Clinical • P2b data • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Buloxibutid: Expiry of patents in US/EU related to formulation and method-of-use in 2042 (Q32 Bio) - Corporate Presentation

Patent • Inflammatory Bowel Disease

The FDA Grants Vicore’s Buloxibutid Fast Track Designation for Idiopathic Pulmonary Fibrosis (ACCESSWIRE) - "Vicore Pharma Holding AB...announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation to its lead candidate buloxibutid, recognizing its disease-modifying potential for the treatment of idiopathic pulmonary fibrosis (IPF)....This designation underscores the potential of buloxibutid to offer a significant improvement over existing treatments, as demonstrated by available clinical and non-clinical data."

Fast track • Idiopathic Pulmonary Fibrosis

ASPIRE: A Trial to Evaluate Efficacy and Safety of Buloxibutid in People With Idiopathic Pulmonary Fibrosis. (clinicaltrials.gov) - P2 | N=270 | Recruiting | Sponsor: Vicore Pharma AB | Not yet recruiting ➔ Recruiting

Enrollment open • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

ASPIRE: A Trial to Evaluate Efficacy and Safety of Buloxibutid in People With Idiopathic Pulmonary Fibrosis. (clinicaltrials.gov) - P2 | N=270 | Not yet recruiting | Sponsor: Vicore Pharma AB

New P2 trial • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Effects of the angiotensin II type 2 receptor agonist buloxibutid on human lung myofibroblasts and IPF lung tissue (ERS 2024) - "Rationale In a recent interim analysis of an open label phase 2a clinical trial, the selective orally available angiotensin II type 2 receptor (AT2R) agonist buloxibutid (C21) improved lung function in IPF patients over a 36-week period...In addition, buloxibutid stimulated the release of surfactant protein B and C by 70% and 120%, respectively.Conclusions In human tissues and cells relevant to IPF pathology, buloxibutid inhibited TGFβ1 expression and ECM-producing mesenchymal cells, and stimulated surfactant protein release. These findings are consistent with the observed clinical efficacy of buloxibutid in IPF."

Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • CDH2 • TGFB1

AIR – an open-label, 36-week, clinical trial of buloxibutid – investigating the disease modifying potential of a novel angiotensin II type 2 receptor agonist in IPF (ERS 2024) - "Buloxibutid (C21) is an oral, selective angiotensin II type 2 (AT2) receptor agonist with disease modifying potential...Ten individuals reported reversible, mild to moderate hair thinning. The efficacy and safety of buloxibutid in IPF will be confirmed in the global, 52-week, phase 2b ASPIRE trial."

Clinical • Alopecia • Fibrosis • Gastrointestinal Disorder • Idiopathic Pulmonary Fibrosis • Immunology • MMP13 • TGFB1

Patients and caregivers' core aspirations to maximize the attractiveness of ASPIRE - a phase 2b trial to investigate the disease modifying potential of buloxibutid in IPF (ERS 2024) - "To increase the probability of success of ASPIRE, a 52-week, phase 2b trial of buloxibutid (C21), an oral angiotensin II type 2 (AT2) receptor agonist with disease modifying potential, Vicore established an advisory panel with six IPF patients and two caregivers from the United States and United Kingdom...The ASPIRE trial aims to maximise trial attractiveness through continuous feedback from patients and caregivers. The three core aspirations continue to guide the conduct and reporting of ASPIRE through empowered trial participants."

Clinical • P2b data • Idiopathic Pulmonary Fibrosis • Immunology

Buloxibutid: "Buloxibutid is an upstream intervention with a novel mechanism of action expected to drive tissue repair in IPF"; Idiopathic pulmonary fibrosis (Vicore Pharma) - ERS 2024: FVC stabilization and improvement, across IPF HRCT patterns, combined with reduction of plasma TGF-β1and increase of MMP-13, are consistent with the proposed mechanism of action of buloxibutid"

P2a data • Idiopathic Pulmonary Fibrosis

Phase 2a Safety Trial Shows Improvement in IPF Treatment (Managed Healthcare Executive) - P2a | "'If the improvement in FVC could be replicated in a randomized placebo controlled trial the buloxibutid would transform our current expectations for the treatment of IPF,' Maher said. 'At present, we hope to slow disease decline. To genuinely improve lung function would be transformational.'"

Media quote • P2a data • Idiopathic Pulmonary Fibrosis • Pulmonary Disease

Buloxibutid, A Novel Angiotensin II Type 2 Receptor Agonist, Stabilized and Improved Lung Function in Individuals With Idiopathic Pulmonary Fibrosis in the 36-week Phase 2 AIR Trial (ATS 2024) - P2 | "Buloxibutid (C21) is an oral, selective angiotensin II type 2 (AT2) receptor agonist, driving an upstream pathway that improves alveolar epithelial type 2 cell function, triggers a cascade of anti-fibrotic activity, and resolves disease-associated vascular remodeling... The selective AT2 receptor agonist buloxibutid stabilized FVC at 24 weeks and improved lung function at 36 weeks with reduced plasma TGF-β1 and increased MMP-13, consistent with buloxibutid's efficacy and proposed mechanism of action. The safety and tolerability profile was favorable. Buloxibutid's safety and efficacy as a treatment for IPF will be evaluated further in the global, 52-week, phase 2b, randomized, double-blind, placebo-controlled trial, ASPIRE."

Clinical • Late-breaking abstract • P2 data • Alopecia • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases • MMP13 • TGFB1

Crafting a Patient-focused Phase 2b Trial (ASPIRE) to Evaluate Efficacy and Safety of Buloxibutid in Individuals With Idiopathic Pulmonary Fibrosis (IPF) (ATS 2024) - "Methods In ASPIRE, a 52-week, randomized, double-blind, placebo-controlled, phase 2b trial of buloxibutid (C21), an oral angiotensin type 2 receptor agonist (ATRAG) with documented effects on fibrosis and vascular remodeling, early patient involvement is secured by the establishment of an advisory panel consisting of six patients and two caregivers from the US and UK...1183/23120541. 00602-2022)."

Clinical • P2b data • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Deciphering the Clinical Efficacy Mechanisms of Buloxibutid in Idiopathic Pulmonary Fibrosis (ATS 2024) - "Rationale Buloxibutid (C21) is an oral high-affinity angiotensin II type 2 (AT2) receptor agonist in clinical development for treatment of idiopathic pulmonary fibrosis (IPF)...Conclusions Buloxibutid has shown notable clinical efficacy in the ongoing, open-label 36-week phase 2a IPF AIR trial. Non-clinical and translational studies suggest that buloxibutid's efficacy may be driven by inhibition of TGFβ1 expression, inhibition of ECM-producing mesenchymal cells, and upregulation of surfactants to prevent or reverse alveolar collapse."

Clinical • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases • CDH2 • TGFB1

Vicore Announces Positive Final Results from the Phase 2a AIR Trial Demonstrating Buloxibutid Improves Lung Function Over 36 Weeks in Patients with Idiopathic Pulmonary Fibrosis (Vicore Pharma Press Release) - P2a | N=52 | AIR (NCT04533022) | Sponsor: Vicore Pharma AB | "Vicore plans to progress buloxibutid to Phase 2b development in the first half of this year....Vicore Pharma Holding AB...announced positive final results from its Phase 2a AIR trial evaluating buloxibutid (C21) in idiopathic pulmonary fibrosis (IPF)....The trial was positive for both its primary and secondary endpoints, demonstrating excellent safety, tolerability, and efficacy. Over 36 weeks of treatment, buloxibutid improved lung function, measured by forced vital capacity (FVC), with a significant effect over expected decline in untreated patients. In untreated patients, a decline corresponding to approximately 180 mL over 36 weeks has been reported. In patients enrolled in the AIR trial, FVC increased by an average of 216 mL from baseline to week 36, nearly 400 mL over the untreated trajectory (n=28, p<0.001)."

New P2b trial • P2a data • Idiopathic Pulmonary Fibrosis • Immunology • Respiratory Diseases

Buloxibutid: Final data from P2a trial (NCT04533022) for IPF in H1 2024 (Vicore Pharma) - Q1 2024 Results

P2a data • Idiopathic Pulmonary Fibrosis

Safety, Efficacy and Pharmacokinetics of C21 in Subjects With IPF (clinicaltrials.gov) - P2 | N=52 | Completed | Sponsor: Vicore Pharma AB | Active, not recruiting ➔ Completed

Trial completion • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

RENOPROTECTIVE EFFECTS OF THE ANGIOTENSIN II TYPE 2 RECEPTOR (AT2R) AGONIST, C21, AGAINST ACUTE ISCHEMIC KIDNEY INJURY IN FEMALE RATS (ISN-WCN 2024) - "In agreement with other studies, we found that IR damage in female rats was less severe than in males. uKIM was elevated before changes in GFR and plasma creatinine, representing a sensitive biomarker to evidence minor tissue damage in female rats. We previously demonstrated that a dose of 1 mg/kg/day of C21 have renoprotective effects against the severe damage induced by IR in male rats."

Preclinical • Acute Kidney Injury • Cardiovascular • Fibrosis • Nephrology • Renal Disease • Reperfusion Injury • KIM1

ANGIOTENSIN II TYPE 2 RECEPTOR (AT2R) AGONIST C21 ELICITS A NEPHROPROTECTIVE EFFECT DURING RENAL ISCHEMIA/REPERFUSION BY STABILIZING THE PRIMARY CILIA IN TUBULAR CELLS (ISN-WCN 2024) - "C21, HDAC6-inhibitor (Tubastatin A 1 μM, TubA) or MEK1/2 inhibitors (PD98059 5μM or U0126 10μM) were added to the media 24h before IR. AT2R nephroprotective effect during renal IR is associated with prevention of tubular cell deciliation in the rat. The cellular mechanism underlying this effect involves AT2R mediated inhibition of ERK1/2 kinases, and concomitant increase in primary cilia stability. Our results support a model where AT2R induced inhibition of ERK1/2 activation leads to cilia stabilization by prevention of HDAC6 activation."

Acute Kidney Injury • Cardiovascular • Nephrology • Renal Disease • Reperfusion Injury

Utilizing venous occlusion plethysmography to assess vascular effects: A study with buloxibutid, an angiotensin II type 2 receptor agonist. (PubMed, Clin Transl Sci) - "Buloxibutid (also known as C21) is a potent and selective angiotensin II type 2 receptor (AT2R) agonist, in development for oral treatment of fibrotic lung disease...Infusions of sodium nitroprusside (SNP) solution in doses of 0.8-3.2 μg/min were administered as a positive control...Intra-arterial infusion of buloxibutid in low, ascending doses increased FBF, indicating that buloxibutid may be effective in conditions associated with endothelial dysfunction. Venous occlusion plethysmography was found to be a useful method to explore pharmacodynamic vascular effects of novel AT2R agonists, while avoiding systemic adverse effects."

Journal • Fibrosis • Pulmonary Disease • Respiratory Diseases

Vicore and Nippon Shinyaku Enter into an Exclusive License Agreement to Develop and Commercialize C21 in Japan (ACCESSWIRE) - "Vicore Pharma Holding AB...announced it has entered into an exclusive licensing agreement with Nippon Shinyaku Co. Ltd...to develop and commercialize Vicore's drug candidate C21 in Japan. Under the terms of the agreement, Vicore will receive an upfront payment of USD 10 million and is entitled to potential development and commercial milestone payments up to a total of USD 275 million. Vicore is eligible to receive tiered royalties extending into the low 20s based on annual net sales of C21 in Japan. Nippon Shinyaku holds the exclusive right to develop and commercialize C21 in Japan focusing initially on the treatment of idiopathic pulmonary fibrosis (IPF)."

Licensing / partnership • Idiopathic Pulmonary Fibrosis • Immunology • Respiratory Diseases

Vicore Confirms IPF Development Program on Track and Provides Early-Stage Pipeline Updates (Vicore Pharma Press Release) - "Vicore Pharma Holding AB...announced pipeline updates as the company continues to focus on development of C21 for idiopathic pulmonary fibrosis (IPF). The updates announced today include confirmation that development of the lead program, C21 for IPF is on track, with final Phase 2a AIR data in the first half of next year. In addition, start-up activities for the global, double-blind, placebo-controlled, 52-week, Phase 2b ASPIRE study of C21 for IPF are progressing with initiation expected in the first half of 2024. Development of the Almee digital therapy advances as well, with topline results of the COMPANION pivotal study for anxiety associated with pulmonary fibrosis expected to be reported in January 2024....Vicore will discontinue development of the preclinical IMiD program, inhaled thalidomide for IPF cough."

Clinical data • Discontinued • New P2b trial • P2a data • Idiopathic Pulmonary Fibrosis • Immunology

Safety, Efficacy and Pharmacokinetics of C21 in Subjects With IPF (clinicaltrials.gov) - P2 | N=60 | Active, not recruiting | Sponsor: Vicore Pharma AB

Trial completion date • Trial primary completion date • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases