Truseltiq (infigratinib) / Novartis, BridgeBio, Pfizer, LianBio, Xediton Pharma, Juniper Bio, Kyowa Kirin - LARVOL DELTA

Fibroblast Growth Factor 2 Enhances Contraction of Arterioles in the Medulla Oblongata and the Basilar Artery in Mice With Chronic Kidney Disease. (PubMed, Acta Physiol (Oxf)) - "CKD enhances cerebrovascular contractile responses to vasoconstrictors, and this effect is mediated by activation of the FGF2 pathway. Targeting the FGF2 pathway may provide treatment of cerebrovascular dysfunction in CKD."

Journal • Preclinical • Chronic Kidney Disease • Nephrology • Renal Disease • EDN1 • FGF2 • FGFR

Comparative Efficacy and Safety of Biomarker-Guided Targeted Therapies Versus Conventional Treatment in Advanced Biliary Tract Cancer: A Systematic Review and Network Meta-Analysis (ACG 2026) - "14 studies involving 3,842 patients were included, comprising 2,116 males (55.1%) and 1,726 females (44.9%), with median age ranging from 58â69 years. Evaluated therapies included FGFR inhibitors (pemigatinib, futibatinib, infigratinib), the IDH1 inhibitor ivosidenib, HER2-directed regimens (trastuzumab-based therapies and zanidatamab), dabrafenib plus trametinib, zenocutuzumab, and conventional chemotherapy/immunotherapy controls. Most patients had metastatic disease (81.6%), ECOG 0â1 status (87.3%), and prior systemic therapy exposure."

Biomarker • IO biomarker • Metastases • Retrospective data • Review • Biliary Cancer • Biliary Tract Cancer • Oncology • Solid Tumor • FGFR2 • IDH1 • NRG1

Infigratinib Versus Placebo for Patients with High-risk Resected Urothelial Cancer Bearing an FGFR3 Genomic Alteration: Results from the PROOF302 Phase 3 Trial. (PubMed, Eur Urol) - No abstract available

Journal • P3 data • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR3

Longer-term efficacy and safety results of infigratinib in children with achondroplasia (ESPE 2026) - No abstract available

Clinical • Late-breaking abstract • Genetic Disorders

A randomized controlled trial of oral infigratinib in children with achondroplasia: Results from the PROPEL 3 study (ESPE 2026) - No abstract available

Clinical • Late-breaking abstract • Genetic Disorders

Infigratinib in Achondroplasia - A Potentially Giant Step Forward. (PubMed, N Engl J Med) - No abstract available

Journal • Genetic Disorders

Fibroblast growth factor receptor 3 (FGFR3) alterations in PROOF 302: A phase III trial of infigratinib (BGJ398) as adjuvant therapy in patients (pts) with invasive urothelial carcinoma (UC). (ASCO 2023) - P3 | "Background: Radical surgery with or without (neo)adjuvant cisplatin-based chemotherapy [(N)AC] is standard in fit pts with muscle invasive UC of bladder (UBC) or upper tract (UTUC). FGFR3 alterations were seen in only 19% of all pts screened for PROOF-302, a trial that was enriched for UTUC: 71/237 (30%) of UTUC and 48/369 (13%) of UBC. The trial was stopped early by the sponsor, but the genomic analysis provides insights into the prevalence of FGFR3 alterations; assessment of correlations with the primary/secondary endpoints is ongoing. The nature and frequency of co-occurring alterations in tissue samples is being investigated to help inform combination therapy strategies and putative resistance mechanisms."

Clinical • P3 data • Oncology • Solid Tumor • Urothelial Cancer • CORIN • FGFR3

PROPEL Infant and Toddler: Study design and ongoing enrollment of a phase 2/2b study of infigratinib in children under 3 years old with achondroplasia (ESPE 2026) - No abstract available

Clinical • P2 data • Genetic Disorders

Defective Memory is restored and Epileptic seizures are rescued in a Mouse Model of Hypochondroplasia by the FGFR Inhibitor Infigratinib (ASBMR 2026) - No abstract available

Late-breaking abstract • Preclinical • CNS Disorders • Epilepsy

Infigratinib versus placebo in patients with resected urothelial cancer (UC) bearing FGFR3 mutation or fusion: Primary DFS analysis from the phase 3, randomized PROOF302 study. (ASCO-GU 2024) - P3 | "Our failure to accrue sufficient patients to the current trial precludes any definitive conclusions around the role of infigratinib as adjuvant therapy for FGFR3-altered UC. In the process of study conduct, however, we garnered substantial insights that may aid in the development of future precision oncology trials for adjuvant UC. Clinical trial information: NCT04197986."

Clinical • P3 data • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR3

Lack of Peripheral FGFR1 Inhibition at Clinically Relevant Infigratinib Exposures Supports the Safety Profile Observed in Children with Achondroplasia (ASBMR 2026) - No abstract available

Clinical • Late-breaking abstract • Genetic Disorders

A BMSC Transplantation Model to study Fgfr1-Fgf23-Phosphate dysregulation and evaluate Infigratinib in Osteoglophonic Dysplasia (ASBMR 2026) - No abstract available

Transplantation • FGF23 • FGFR1

Clinical response to FGFR inhibitors in patients with FGFR2-amplified advanced gastric cancer: two case reports. (PubMed, Front Oncol) - "Both patients experienced predictable and manageable FGFR inhibitor-related adverse events (e.g., keratitis, onychotoxicity, hyperphosphatemia). This case series offers hypothesis-generating observations suggesting potential activity of FGFR inhibitors (such as pemigatinib and infigratinib) in patients with FGFR2-amplified advanced gastric cancer who have limited conventional therapeutic options or are intolerant to chemotherapy, highlighting the importance of next-generation sequencing-guided precision therapy."

IO biomarker • Journal • Gastric Cancer • Hematological Disorders • Keratitis • Metabolic Disorders • Nephrology • Ocular Inflammation • Oncology • Ophthalmology • Pain • Renal Disease • Solid Tumor • FGFR2 • HER-2

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) (clinicaltrials.gov) - P2 | N=77 | Recruiting | Sponsor: QED Therapeutics, a BridgeBio company | Trial primary completion date: Mar 2032 ➔ Mar 2030

Trial primary completion date • Genetic Disorders

Genetic Bone Diseases: A Scoping Review of Pathology, Symptoms, Diagnosis, Treatment, and New Horizons. (PubMed, Adv Genet (Hoboken)) - "These treatments include: fresolimumab for osteogenesis imperfecta, small interfering ribonucleic acid (RNA) therapy for Osteopetrosis, denosumab for Paget's disease of bone, vosoritide/recifercept/infigratinib for achondroplasia, mesenchymal stem cell therapy for craniosynostosis, and combination losartan and atenolol therapy for Marfan syndrome. These treatments are generally more recently acknowledged in literature and are either actively undergoing research or require further research to determine their efficacy."

Journal • Review • Genetic Disorders • Orthopedics • Rare Diseases

Sensitisation pathways revealed by CRISPR-based combinatorial dependency analysis in paediatric high-grade glioma (ISPNO 2026) - "Each cell line was transduced with a genome-wide knockout library and subsequently exposed to 11 different compounds, including FDA-approved agents (Trametinib, ONC201, Temozolomide, Ribociclib, Infigratinib, Avapritinib, Erlotinib, and Alpelisib) and additional pathway inhibitors (TP0184, ACT001 and Paxalisib). Finally, KEGG analysis of pan-therapy dependency genes identified significant enrichment of the ubiquitin system, spliceosome, kinetochore dephosphorylation, and mitotic checkpoint complex pathways, suggesting the presence of conserved, lineage-independent vulnerabilities across pHGGs. Together, these finding highlight the presence of a robust pan-dependency signature across drugs and points to conserved vulnerabilities that may offer complementary therapeutic opportunities."

Brain Cancer • Glioma • High Grade Glioma • Pediatrics • Solid Tumor • Targeted Protein Degradation • PI3K

Central lactate uptake during exercise contributes to hypothalamic fibroblast growth factor 21 induction and systemic fat mobilization. (PubMed, Mol Cell Endocrinol) - "Male Wistar rats received intracerebroventricular (ICV) administration of FGF21 or lactate, or performed acute endurance exercise under three conditions: standard exercise, exercise with inhibited brain lactate transport using α-cyano-4-hydroxycinnamate (4-CIN), or exercise with reduced systemic lactate production via dichloroacetate (DCA)...ICV FGF21 increased catecholamines, adipose cAMP, and HSL phosphorylation, effects blocked by the FGF21 receptor antagonist BGJ-398. Inhibiting brain lactate uptake before exercise attenuated hypothalamic FGF21 induction, sympathetic activation, and fat mobilization, whereas systemic lactate reduction had a weaker effect, suggesting that brain lactate uptake contributes importantly to exercise-induced hypothalamic FGF21 regulation and fat mobilization. Taken together, these findings support a role for central lactate uptake in the activation of hypothalamic GPR81-p38-MAPK-FGF21 signaling and suggest that this pathway contributes to..."

Journal • Metabolic Disorders • FGF21

Integrative multi-omics and single-cell analysis identifies EGFR pathway activation and metabolic reprogramming as potential synthetic lethal vulnerabilities in resistance to the FGFR inhibitor AZD4547. (PubMed, J Transl Med) - "This study establishes a comprehensive multi-omics atlas of resistance to the FGFR inhibitor AZD4547, delineating convergent mechanisms of metabolic reprogramming and EGFR-mediated bypass signaling. Our findings characterize the resistance as a dynamic network rewiring and nominate rational combination strategies to overcome this therapeutic bottleneck. While FGFR-EGFR co-inhibition is experimentally supported, metabolic co-targeting remains a computationally derived, hypothesis-generating strategy."

Journal • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • Fascin • FSCN1 • PPFIBP2

Comparative Real-World Safety Profiles of Fibroblast Growth Factor Receptor Inhibitors: A Pharmacovigilance Study. (PubMed, Asia Pac J Clin Oncol) - "FGFR inhibitors demonstrated differences in adverse event reporting patterns rather than a uniform class effect, although pharmacovigilance data do not allow estimation of true incidence, risk, or causal relationships."

Adverse events • Journal • Real-world evidence • Dry Eye Disease • Metabolic Disorders • Nephrology • Ophthalmology • Renal Disease • FGFR

Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia. (PubMed, N Engl J Med) - P3 | "In children with achondroplasia, treatment with once-daily oral infigratinib for 52 weeks resulted in a significantly greater increase from baseline in the annualized height velocity than placebo. (Funded by BridgeBio Pharma; PROPEL 3 ClinicalTrials.gov number, NCT06164951; EudraCT number, 2023-506130-67.)."

Journal • P3 data • Genetic Disorders • FGFR1 • FGFR3

BridgeBio to Present Primary Results from Phase 3 PROPEL 3 Trial of Oral Infigratinib for Children Living with Achondroplasia at ICCBH 2026 (The Manila Times) - "BridgeBio will also share an oral presentation and four posters at the meeting highlighting quality of life, early intervention research, observational study findings, and educational resources through MyAchonJourney for individuals with achondroplasia and related skeletal dysplasias. Additionally, the Company will share an autosomal dominant hypocalcemia type 1 (ADH1) poster on findings from CLARIFY, its disease monitoring study of autosomal dominant hypocalcemia (ADH) type 1 and type 2."

Clinical data • Genetic Disorders

Molecular Glues Recruiting RNF213 As an E3 Ligase for Targeted Protein Degradation: A Minimal Dibromoacetamide Warhead As a Recruitment Ligand. (PubMed, J Am Chem Soc) - "We developed CYB-5067 by equipping the pan-FGFR inhibitor Infigratinib with a minimal dibromoacetamide covalent warhead...Our work identifies RNF213 as an exploitable ligase for TPD and establishes covalent molecular glues as a modular platform. This strategy expands the scope of degrader design beyond conventional E3 ligases, offering an avenue for developing potent and selective therapeutics."

Journal • Oncology • Targeted Protein Degradation • Transplantation • CDK12 • CRBN • FGFR1 • FGFR2

Inhibition of FGFR signaling attenuates phosphaturia and early kidney injury in sickle cell disease mice. (PubMed, Biochem Biophys Rep) - "In summary impaired FGFR downstream signaling mediated phosphaturia in SCD that could be modulated by BGJ398. We further suggest that osteopontin and aberrant integrin signaling contributes to kidney inflammation that can be partially rescued by BGJ398."

Journal • Preclinical • Genetic Disorders • Glomerulonephritis • Hematological Disorders • Inflammation • Oncology • Renal Disease • Sickle Cell Disease • FGF23 • FGFR • IL6 • KIM1 • SPP1 • TNFA

Efficacy and safety of infigratinib in patients with refractory advanced gastric or gastroesophageal junction adenocarcinoma harboring FGFR2 gene amplification: a single-arm, multicenter phase 2 trial. (PubMed, Br J Cancer) - P2a | "The findings support continued investigation of FGFR-targeted strategies in FGFR2-amplified GC/GEJ adenocarcinoma, while underscoring the need for larger studies, refined biomarker selection, and deeper characterization of resistance mechanisms."

Journal • P2 data • Gastric Adenocarcinoma • Gastric Cancer • Gastroesophageal Junction Adenocarcinoma • Hematological Disorders • Neutropenia • Oncology • Solid Tumor • FGFR1 • FGFR2

Converting FGFR inhibitors into selective covalent molecular glue degraders via transposable gluing handles. (PubMed, Eur J Med Chem) - "In this study, by incorporating diverse molecular glue handles and amino acid-based degrons into the solvent-exposed exit vectors of infigratinib, we synthesized a library of 30 candidate MGDs...Mechanistic investigations revealed that the covalent engagement of the gluing handle is a critical determinant for successful proteasomal degradation of FGFR2. This work provides a framework for the rational transformation of kinase inhibitors into covalent molecular glue degraders, underscoring the potential of FGFR degradation as a next-generation precision medicine strategy for FGFR2-driven malignancies."

Journal • Gastric Cancer • Oncology • Solid Tumor • Targeted Protein Degradation • FGFR2