Azafaros enters clinical stage with AZ-3102, an oral small molecule being developed for rare neurogenetic disorderss

  • Phase 1 clinical study investigates safety and tolerability, pharmacokinetics and pharmacodynamic effects of first-in-class azasugar AZ-3102 in healthy subjects
  • AZ-3102 is being initially developed for the treatment of patients suffering from GM1 and GM2 gangliosidoses, two rare neurogenetic lysosomal storage disorders
  • Company announces receipt of an additional funding of EUR 2.75 M from lead investor Forbion in an extension of the Series A financing round


Leiden, The Netherlands, April 6, 2021 – Azafaros B.V. announced today that the first cohort of healthy subjects has been dosed in a two-part Phase 1 clinical study with clinical candidate AZ-3102, a first-inclass small molecule compound being developed for the treatment of rare neurogenetic disorders.

This double-blind, placebo-controlled, Phase 1 study is evaluating the safety and tolerability of ascending single- (SAD) and multiple-doses (MAD) of AZ-3102 administered orally to healthy subjects. The pharmacokinetic properties of AZ-3102 and its pharmacodynamic effects on specific glycosphingolipid biomarkers will also be assessed. The results of this study will support establishing the dose levels and dosage regimen suitable for administration to patients in future clinical studies.

Azafaros’ initial objective with AZ-3102 is to develop a potential disease-modifying therapy for GM1 and GM2 gangliosidoses, two rare life-threatening neurogenetic lysosomal storage disorders affecting infants, adolescents, and adults, for which there is only palliative care. Both conditions lead to the harmful accumulation of GM1 or GM2 gangliosidesin multiple organs, in particular in the brain causing progressive neurological deterioration. AZ-3102 is an orally available azasugar designed to reach the central nervous system and to interfere with the metabolism of glycosphingolipids through a unique and selective dual mode of action with equally potent inhibition of glucosylceramide synthase (GCS) and non-lysosomal glucosylceramidase (GbA2). With this new paradigm, AZ-3102 has the potential to reduce metabolite accumulation and to ameliorate the function of the impaired lysosome

Beside the clinical trial initiation, Azafaros announced that it has raised an additional EUR 2.75 million from the Company’s lead investor Forbion, which exercised an option to increase its investment level, thus bringing the total funds raised in Azafaros’ Series A to EUR 28.75 million.

“Reaching clinical stage with AZ-3102 is a major turning point for Azafaros and is the concrete result of a tireless team effort,” said Olivier Morand, PhD, Azafaros’ Chief Executive Officer. “Azafaros has a solid financial foundation to progress the development of our clinical candidate and advance further our pipeline of disease-modifying therapies for people living with rare metabolic disorders.”

“Currently, GM1 and GM2 gangliosidoses can only be treated symptomatically and carry the inevitable risk of severe neurological impairments and early death, especially for infants and children,” said Carlo Incerti, MD, Azafaros’s Chairman of the Board. “With its novel dual mode of action and ease of oral 2 administration, AZ-3102 holds the potential of a convenient life-long therapy which could mitigate the underlying pathophysiology of these diseases and favorably alter the patients’ clinical trajectory.”