NEWARK, Calif.--(BUSINESS WIRE)--AceLink Therapeutics, Inc. (AceLink), invested and incubated by Viva BIoInnovator (VBI), is an innovative biopharmaceutical company developing transformative therapies for genetic diseases. Recently they presented positive data from a Phase 1 trial of AL01211 in healthy volunteers and preclinical data on the development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor.

AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase inhibitor (GCSi) being developed for the treatment of Fabry disease. The Phase 1 trial is a randomized, double-blind, placebo-controlled, dose escalation study of AL01211 in 69 healthy adult participants.

Here are some highlights Data:

  • AL01211 was safe and well-tolerated, showing no significant or serious adverse events in healthy volunteers.
  • Treatment with AL01211 resulted in dose-dependent PK and PD (reduction of plasma GL1 and GL3) properties.
  • In preclinical studies, AL01211 was widely distributed in peripheral organs and lacked blood-brain barrier penetration. This makes AL01211 an excellent choice for treating diseases that primarily affect peripheral tissues.
  • The increased potency and low brain penetration make AL01211 a potentially safer and more efficacious molecule for treating Fabry disease patients, especially in younger patients seeking a convenient and lifelong treatment option.

“We are encouraged by the results of our first-in-human trial. AL01211 achieved a clean safety profile withnno significant or serious adverse events,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink. “In addition, AL01211 treatment led to dose-dependent reduction of plasma GL1 and GL3, which is the pharmacodynamic biomarker of GCS inhibition. AL01211 has the potential to be a transformative therapy for Fabry patients who desperately need more convenient and more effective alternatives to enzyme replacement therapy. We look forward to commencing our Phase 2 program in patients with Fabry disease later this year.”

AL00804, a highly brain penetrant GCS inhibitor, efficiently reduced GL1 accumulation in the brain of preclinical models . In a head-to-head comparison in a neuronopathic model for Gaucher disease, AL00804 exhibited higher potency and greater brain penetration compared to other GCS inhibitors currently in development. AL00804 is IND-ready and aims to be the best-in-class treatment of neuronopathic Gaucher disease, GM2 and GM1 gangliosidosis.

Data Highlights:

  • Pharmacokinetics studies support once daily, oral administration of AL00804.
  • AL00804 is highly brain penetrant that efficiently reduced brain GL1 and lyso-GL1 accumulation in preclinical disease models.
  • AL00804 reduced CNS disease symptoms, significantly delayed motor function decline and extended survival.
  • When tested head-to-head in a neuronopathic model of Gaucher disease, AL000804 performed better than Venglustat.
  • The increased potency and superior brain penetration compared to Venglustat make AL00804 a potentially safer and more efficacious molecule for treating neuronopathic GSL storage disorders.

“We are pleased with the efficacy of AL00804 in reducing symptoms of CNS disease in several preclinical models,” said Jerry Shen, PhD, Chief Executive Officer and Founder of AceLink. “AL00804 was selected based on its unique properties, including high potency and ability to cross the blood-brain barrier, to more effectively treat patients with neuronopathic diseases. We have completed IND-enabling work in support of AL00804 and are excited for its further development in clinical settings.”

About AceLink Therapeutics, Inc.


Founded in 2018, AceLink Therapeutics is an innovative biopharma startup focusing on developing safe and effective medicines to address genetic diseases with high unmet needs. The company’s initial focus is to develop novel therapeutics for Fabry disease. For more information, please visit www.acelinktherapeutics.com.