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

ASPIRE: A Trial to Evaluate Efficacy and Safety of Buloxibutid in People With Idiopathic Pulmonary Fibrosis. (clinicaltrials.gov) - P2 | N=360 | Recruiting | Sponsor: Vicore Pharma AB | Trial completion date: Mar 2027 ➔ Jun 2027

Trial completion date • 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=360 | Recruiting | Sponsor: Vicore Pharma AB | N=270 ➔ 360

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

ASPIRE is on track to complete enrollment in the first half of 2026… (ACCESS Newswire)

Trial status • Idiopathic Pulmonary Fibrosis

Buloxibutid Receives Orphan Drug Designation in Japan for the Treatment of Idiopathic Pulmonary Fibrosis (ACCESS Newswire) - "Buloxibutid is being evaluated in the global Phase 2b ASPIRE trial for the treatment of IPF."

Orphan drug • Idiopathic Pulmonary Fibrosis

Vicore will present new findings from the 36-week Phase 2a AIR trial of buloxibutid in idiopathic pulmonary fibrosis (IPF), including a synthetic control arm (SCA) analysi (ACCESS Newswire) - "Among patients with comparable baseline characteristics, buloxibutid showed statistically significant improvement in forced vital capacity (FVC) at 36 weeks relative to both baseline and synthetic control, confirming the disease-modifying signal observed in the AIR trial."

P2a data • Idiopathic Pulmonary Fibrosis

Late Breaking Abstract - Synthetic Arm Generation Utilizing Real-World Patient Data Demonstrates Treatment Effect in the Phase 2a AIR trial of Buloxibutid in IPF (ERS 2025) - "We have demonstrated a statistically significant treatment effect for 100mg BID of buloxibutid at 36 weeks, utilizing MCCV-generated synthetic control arms with matched clinical variables. This is the first use of synthetically generated control arms for efficacy testing in IPF."

Clinical • Late-breaking abstract • P2a data • Real-world • Real-world evidence • Idiopathic Pulmonary Fibrosis • Immunology

AT2R agonists buloxibutid (Compound 21) and NAc inhibit fibrogenesis in human precision cut lung slices ex vivo (ERS 2025) - "Buloxibutid, also known as Compound 21 (C21), is an angiotensin type 2 receptor AT 2 R agonist in early clinical trials for IPF, having shown antifibrotic efficacy in a bleomycin mouse model (PMID 29636695; 2018)...To compare C21 with an experimental AT 2 R agonist NAc and the IPF drug pirfenidone (PFD), using human precision cut lung slices (hPCLS)...C21 and NAc, at 50-fold lower concentrations than PFD, significantly reduced secretion of both fibrogenic and inflammatory markers. Establishing reversal of fibrosis by AT 2 R agonists in hPCLS from IPF lung would address a major limitation of current therapy and further support clinical translation of this novel drug class for IPF."

Preclinical • Fibrosis • Immunology • Inflammation • CXCL8 • IL6 • TGFB1 • TNFA

The novel angiotensin II type 2 receptor agonist buloxibutid improves lung function in IPF compared to real-world external IPF control arms (ERS 2025) - "Background: Buloxibutid (C21) is an oral, selective angiotensin II type 2 receptor agonist, which drives upstream repair pathways in idiopathic pulmonary fibrosis (IPF) that improve alveolar epithelial type 2 cell survival and surfactant production, reduces fibrosis, and resolves vascular remodeling... FVC improvement during treatment with buloxibutid differed from the real-world treatment-naive FVC decline in severity matched patients. This is consistent with the proposed mechanism of action of buloxibutid, indicating its potential for IPF disease modification."

Clinical • Real-world • Real-world evidence • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology

Vicore's Phase 2b ASPIRE trial remains on track and is expected to complete enrollment in the first half of next year. (ACCESS Newswire)

Enrollment status • Idiopathic Pulmonary Fibrosis

Anti-Fibrotic Profile of a Next-Generation AT2 Receptor Agonist in Human Fibrotic Kidney Tissue (ERA 2025) - "Previous studies with AT2R agonists, such as buloxibutid (also referred to as C21), have demonstrated efficacy in preclinical models of CKD by reducing fibrosis, inflammation, and improving renal function... The superior anti-fibrotic profile of the novel highly potent and selective AT2R agonist C112 in human fibrotic kidney slices highlights the potential for AT2R-targeted interventions in CKD. By reducing profibrotic mediators (COL1A1 and TGF-β1) and increasing the fibrolytic protease MMP-1, AT2R agonists may offer a valuable new therapeutic approach for CKD management. Studies of C112 in vivo are ongoing to confirm these promising translational ex vivo findings."

Chronic Kidney Disease • Fibrosis • Inflammation • Nephrology • Renal Disease • COL1A1 • KIM1 • MMP1 • TGFB1

Buloxibutid Potently Inhibits Fibrosis Biomarkers in the Scar-in-a-Jar Primary Human Lung Fibroblast Assay (ATS 2025) - "This abstract is funded by: Vicore Pharma RATIONALE In a recent open-label Phase 2a clinical trial, the selective, orally available angiotensin II type 2 receptor (AT2R) agonist buloxibutid (also known as C21) improved lung function in patients with idiopathic pulmonary fibrosis (IPF) over a 36-week period. CONCLUSION The findings demonstrate that buloxibutid potently inhibits collagen synthesis in activated human lung fibroblasts, demonstrating its potential as an effective antifibrotic IPF therapy. Buloxibutid was more potent than nintedanib and nerandomilast."

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

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) - P2 | "Buloxibutid (C21) is an oral, selective angiotensin II type 2 receptor agonist, driving an upstream repair pathway that improves alveolar epithelial type 2 cell function, triggers anti-fibrotic pathways, and resolves disease-associated vascular remodeling...The trial will include 270 participants with IPF confirmed by central read of high-resolution computed tomography (HRCT), FVC ≥50% predicted and diffusing capacity of carbon monoxide ≥35% predicted, who are on a stable dose of nintedanib or currently not treated... The ASPIRE trial is designed to provide robust evidence of efficacy and safety of the novel angiotensin II type 2 receptor agonist buloxibutid in IPF, while using patient and caregiver experiences to enable easy participation. Collecting direct participant feedback on trial experience is a novel approach to provide insights that may support patients' wishes to enroll and remain in clinical trials."

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

The AIR Phase 2 Trial of the Angiotensin II Type 2 Receptor Agonist, Buloxibutid, in Individuals With Idiopathic Pulmonary Fibrosis: A Responder Analysis (ACCESSWIRE) - P2a | N=52 | NCT04533022 | Sponsor: Vicore Pharma AB | "A key finding in the Phase 2a AIR trial was that a substantial proportion of participants demonstrated improvement in lung function. An analysis comparing the proportion of patients experiencing improvement in lung function at 0, 5 and 10 ppFVC in the Phase 2a AIR trial with the Phase 3 studies leading to the approval of the current standard of care compared favorably with those for the approved antifibrotic agents. In addition to the responder analysis, Dr. Maher presented a SCA drawn from a pool of over 10,000 real world IPF patients on the Qureight platform and tightly matched to the Phase 2a AIR population using a Monte Carlo cross validation analysis. Comparison of forced vital capacity (FVC) in these SCAs to the impact of buloxibutid on FVC in the Phase 2a AIR trial revealed a robust treatment effect with a high degree of statistical significance (p=.0025)."

P2a data • Real-world • Idiopathic Pulmonary Fibrosis

ASPIRE: A Phase 2b trial of Buloxibutid in IPF (ATS 2025) - "The ASPIRE IPF trial of buloxibutid is being designed in close collaboration with world leading clinical experts and patient advocacy organizations to create a patient and site friendly experience, while ensuring high quality standards are maintained. Key features of ASPIRE IPF: - A randomized, double-blind, placebo-controlled, parallel group, multicentre trial of two doses of buloxibutid - 52 weeks treatment duration - N=270 participants (90/treatment arm) - People with IPF on stable nintedanib or not on SoC - Primary endpoint – change from baseline in FVC"

P2b data • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology

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) - P2 | "In the pivotal trials of nintedanib and pirfenidone, forced vital capacity percent predicted (FVCpp) increased by ≥5% in 6-11% of patients and by ≥10% in 0-3% of patients after 48-52 weeks of treatment1,2. Buloxibutid (also known as C21) is an orally available, selective angiotensin II type 2 receptor (AT2R) agonist... The selective AT2R agonist buloxibutid was associated with a high proportion of FVC responders. Importantly, the percentage of patients experiencing more than 5% or 10% increase in FVCpp compared favorably with reported rates for licensed antifibrotic therapies. Buloxibutid's efficacy and safety as a treatment for IPF is now being further evaluated in ASPIRE, a global, 52-week, phase-2b, randomized, double-blind, placebo-controlled trial."

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

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

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