Azafaros Announces Multiple Presentations at 18th Annual WORLDSymposium™

– Presentations Include Initial First-in-Human Data on Lead Candidate AZ-3102 in Healthy Volunteers –

LEIDEN, The Netherlands–(BUSINESS WIRE)–#AZ3102–Azafaros B.V. today announced its participation at the 18th Annual WORLDSymposium™, a research conference dedicated to lysosomal storage diseases, taking place from February 7 – 11, 2022, in San Diego, CA. The conference is also offering virtual participation. Azafaros will share first-in-human data on its lead candidate AZ-3102, a novel orally available azasugar molecule with a unique dual mode of action, as well as the design for a natural history study in GM1 and GM2 gangliosidosis and preclinical in vivo data on AZ-3102 in Niemann-Pick and Sandhoff disease mouse models.

Details on the presentations are as follows:

Title: A first-in-human, randomized, double-blind, placebo-controlled, ascending single- and multiple dose study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of the L-ido azasugar AZ-3102 in healthy volunteers; Virtual Poster LB- 54.

Presenter: Cecile Paquet Luzy

Date & Time: Wednesday February 9 from 3:00 – 5:00 pm Pacific Time (PT)

Title: Prospective longitudinal study of neurological disease trajectory in children living with late infantile or juvenile onset of GM1 or GM2 gangliosidosis (PRONTO study); In Person Poster 98.

Presenter: Roberto Giugliani

Date & Time: Wednesday February 9, from 3:00 – 5:00 pm PT

Title: Characterization of AZ-3102, a novel brain-penetrant small molecule, in the Niemann-Pick disease type C mouse model; Contemporary Forum Platform Presentation, Poster 161.

Presenter: Kyle Landskroner

Date & Time: Thursday, February 10 at 2:00 pm PT

Title: AZ-3102, a novel brain-penetrant small molecule, significantly improves survival of Sandhoff disease mice; Late-Breaking Science Platform Presentation, Poster LB-70

Presenter: Jagdeep Walia

Date & Time: Friday February 11 at 11:15 am PT

The posters will be available on the Azafaros website following presentation at the WORLDSymposium™ conference.

About Azafaros

Founded in 2018 with a deep understanding of rare genetic disease mechanisms and led by a team of highly experienced industry experts, Azafaros aims to build a pipeline of disease-modifying therapeutics to offer patients and their families new treatment options. The company’s lead clinical-staged program is AZ-3102, a highly differentiated, orally available, small molecule with the potential to treat GM1 and GM2 gangliosidosis and other metabolic disorders. By applying its know-how, network, and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them.


For further information:
Azafaros B.V.


For media inquiries:
Trophic Communications

Eva Mulder and Marie-Theresa Weickert

Phone: +49 (0) 175 222 57 56

Thank you for donating to

We could not provide the Dutch News service, and keep it free of charge, without the generous support of our readers. Your donations allow us to report on issues you tell us matter, and provide you with a summary of the most important Dutch news each day.

Make a donation