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The licensing agreement provides Sanofi with Maze’s glycogen synthase 1 (GYS1) program, including clinical candidate MZE001, an oral GYS1 inhibitor, which is currently in development for the treatment of Pompe disease and other potential indications.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Sanofi
Deal Size: $750.0 million Upfront Cash: $150.0 million
Deal Type: Licensing Agreement May 01, 2023
Details:
MZE001 is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. It is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
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 March 22, 2023
Details:
MZE001 is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. It is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
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 27, 2023
Details:
MZE001 is an oral GYS1 inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. It is currently being evaluated for the potential oral treatment of patients with Pompe disease and potentially other glycogen storage disorders.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
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 16, 2023
Details:
MZ-301 Is a Small Molecule Inhibitor of APOL1 Pore Function That Attenuates Albuminuria in a Mouse Model of APOL1-Mediated Kidney Disease,Potently blocked APOL1 and prevented APOL1-mediated killing of trypanosomes in a parasite viability assay.
Lead Product(s): MZ-301
Therapeutic Area: Nephrology Product Name: MZ-301
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 04, 2022
Details:
In preclinical disease models, treatment with MZE001 demonstrated potent and selective inhibition of GYS1, leading to reduced accumulation of glycogen through a substrate reduction approach.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
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 August 31, 2022
Details:
In several in vitro and in vivo analyses with MZE001, GYS1 inhibitor presented and highlighted below, data demonstrated potent inhibition of GYS1, leading to reduced accumulation of glycogen through a substrate reduction approach.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 10, 2022
Details:
Maze’s lead candidate is an oral therapy that aims to reduce the activity of muscle glycogen synthase, the protein that makes glycogen in muscle cells. By blocking this protein, MZE001, act as a substrate reduction therapy, lowering toxic levels of glycogen in cells.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 31, 2022
Details:
Proceeds from the financing will be used to support advancement of the company’s nine precision medicines programs for both rare and common genetically defined diseases with high unmet need, including its three most advanced programs, MZE001 for the treatment of Pompe disease.
Lead Product(s): MZE001
Therapeutic Area: Genetic Disease Product Name: MZE001
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Matrix Capital Management
Deal Size: $190.0 million Upfront Cash: Undisclosed
Deal Type: Financing January 10, 2022
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