CAN201 / CANbridge Pharma, AstraZeneca - LARVOL DELTA

Azeliragon, a RAGE inhibitor, in combination with temozolomide and radiotherapy in patients with newly diagnosed glioblastoma: Preliminary results of phase Ib/II CAN-201 NDG trial. (ASCO 2025) - P1/2 | "Azeliragon in combination with standard RT and TMZ is safe, with no dose-limiting toxicities reported so far at the initial three dose levels. To further explore the safety and efficacy profile of azeliragon, we are now expanding the study to include two additional dose levels of 30 mg/day (L4) and 50 mg/day (L5). Enrollment is currently open for level L4."

Clinical • Combination therapy • P1/2 data • Alzheimer's Disease • Brain Cancer • CNS Disorders • CNS Tumor • Epilepsy • Glioblastoma • Oncology • Solid Tumor • MGMT

Azeliragon, a RAGE inhibitor, in combination with temozolomide and radiotherapy in patients with newly diagnosed glioblastoma: Phase Ib/II CAN-201 NDG trial design. (ASCO 2024) - P1/2 | "The study received regulatory approval and accrual started in September 2023. As of January 2024, recruitment in dose level 1 was completed with 6 patients and 3 patients were already included in dose level 2."

Clinical • Combination therapy • P1/2 data • Alzheimer's Disease • Brain Cancer • CNS Disorders • CNS Tumor • Fatigue • Glioblastoma • Infectious Disease • Oncology • Solid Tumor • HMGB1 • MGMT • S100A9

Preclinical evaluation of CAN201 containing a human GLA transgene under the control of a liver‐specific promoter for Fabry disease (ESGCT 2023) - "Target tissues (liver, kidney, heart, and spleen) also demonstrated dose-dependent increases in α-GAL activity (up to ∼2 to 3-fold).In conclusion, the DJ-CAN201 surrogate vector demonstrated efficacy and safety in Fabry mice, while in PXB mice, the CAN201 clinical candidate produced high circulating levels of α-GAL that were taken up by peripheral tissues. These preclinical studies support the further clinical development of CAN201 for Fabry disease."

Preclinical • Cardiovascular • Fabry Disease • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Myocardial Infarction • Rare Diseases • Renal Disease