AL01211 / AceLink Therap - LARVOL DELTA

AceLink Therapeutics Presents Interim Results from a Phase 2 Trial of the GCS Inhibitor AL01211 in Treatment-Naïve, Classic Male Fabry Disease Patients at the WORLD Symposium 2025 (Businesswire) - P2 | N=16 | NCT06114329 | Sponsor: AceLink Therapeutics, Inc. | "These findings were highlighted in a late-breaking oral platform presentation at the 2025 WORLD Symposium....AceLink completed enrollment of 18 patients across six sites in China in December 2024. Topline data from this Phase 2 trial is expected in Q3 2025....Interim results indicate that AL01211 is generally safe and well tolerated in classic male Fabry patients. Treatment with 30 mg once daily reduced GL3 levels by 50%, while a higher daily dose of 60 mg resulted in a faster and greater reduction of GL3 substrate levels. Preliminary clinical data suggest that AL01211 stabilizes Fabry disease symptoms, including eGFR and proteinuria levels, and shows positive trends in pain reduction, quality of life, and global disease symptom assessment."

Enrollment closed • Late-breaking abstract • P2 data • Fabry Disease

Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral AL01211 in Healthy Chinese Volunteers. (PubMed, Clin Drug Investig) - "AL01211 showed a favorable pharmacokinetic, pharmacodynamics, safety, and tolerability profile in healthy Chinese volunteers. These data support the further clinical development of AL01211 as a therapy for GSL storage diseases."

Clinical • Journal • PK/PD data • Fabry Disease • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Phase 1 Healthy Volunteer Study of AL01211, an Oral, Non-brain Penetrant Glucosylceramide Synthase Inhibitor, to Treat Fabry Disease and Type 1 Gaucher Disease. (PubMed, Clin Pharmacol Drug Dev) - "Increasing exposure inversely correlated with a decrease in GSL with plasma glucosylceramide and globotriacylceramide reduction from baseline levels, reaching 78% and 52% by day 14, respectively. AL01211 was generally well-tolerated with no AL01211 associated serious adverse events, thus supporting its further clinical development."

Journal • P1 data • Fabry Disease • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease (clinicaltrials.gov) - P2 | N=16 | Recruiting | Sponsor: AceLink Therapeutics, Inc.

New P2 trial • Fabry Disease • Genetic Disorders

To Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AL01211 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=69 | Completed | Sponsor: AceLink Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease