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AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor. Currently, it has completed the ongoing Phase II clinical trial studies for the treatment of GM2 Gangliosidosis and Type C Niemann-Pick Disease.
Lead Product(s): Nizubaglustat
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 12, 2024
Details:
AZ-3102 (nizubaglustat) is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
Lead Product(s): Nizubaglustat
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 11, 2023
Details:
AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 08, 2023
Details:
AZ-3102 is a, novel small molecule, candidate developed for Lysosomal Storage Disorders with neurological involvement. AZ-3102 is an oral, brain penetrant azasugar, having dual mode of action by inhibiting two key enzymes which modulate the metabolism of glycosphingolipids.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: IND EnablingProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 05, 2023
Details:
AZ‑3102, an orally available, small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism, is designed to selectively inhibit two enzymes involved in glycolipid metabolism aims to reduce toxic glycolipid accumulation.
Lead Product(s): AZ-3102
Therapeutic Area: Rare Diseases and Disorders Product Name: AZ-3102
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 24, 2022
Details:
Positive safety, tolerability and pharmacodynamics data from multiple dose levels in healthy volunteers support evaluation of AZ-3102, a small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism in Gangliosidoses.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 14, 2022
Details:
The ODD for AZ-3102, selectively inhibit two enzymes involved in glycolipid metabolism GCS and GbA2 for the treatment ofGM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 01, 2022
Details:
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.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: AZ-3102
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 06, 2021
Details:
The financing helps Azafaros to build its organization and expand its executive team with passionate and experienced people and to advance its rare metabolic disorders pipeline.
Lead Product(s): AZ-3102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: UndisclosedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Forbion
Deal Size: $28.0 million Upfront Cash: Undisclosed
Deal Type: Series A Financing February 06, 2020
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