Maze Therapeutics, Inc. is a clinical-stage biopharmaceutical company, which engages in harnessing the power of human genetics to develop novel, small molecule precision medicines for patients living with renal, cardiovascular, and related metabolic, or CVRM diseases. Its pipeline includes Compass platform, which allows to identify and characterize genetic variants in disease and then link those variants to the biological pathways that drive disease in specific patient groups through a process to refer a variant functionalization. The company was founded by Mark Daly, Aaron D. Gitler, Stephen Elledge, Sekar Kathiresan, and Jonathan S. Weissman on August 28, 2019 and is headquartered in San Francisco, CA.
Clinical Trials
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33.3%
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- Maze Therapeutics appointed Paula Johnson, M.D., M.P.H., and Sophie Kornowski, Pharm.D., M.B.A., to its Board of Directors, adding deep clinical and industry experience. - The appointments come as Maze advances toward late-stage development, with additional MZE829 data in broad AMKD patients expected in late 2026 or early 2027. - Dr. Johnson brings over 30 years of cardiology and women's health expertise, while Dr. Kornowski contributes more than 35 years of biopharmaceutical leadership. - Maze's pipeline is led by MZE829, a dual-mechanism APOL1 inhibitor, and MZE782, a SLC6A19 inhibitor, both in Phase 2 development.
- Shionogi has enrolled the first patients in Esprit, a global Phase 2 clinical trial evaluating S-606001 as the first potential oral substrate reduction therapy for late-onset Pompe disease. - The 52-week multicenter study will assess S-606001 as an add-on treatment to standard enzyme replacement therapy in adults across the U.S., European Union, and United Kingdom. - S-606001 works by inhibiting glycogen synthase to limit glycogen buildup, targeting the opposite mechanism from current enzyme replacement therapies that break down accumulated glycogen. - Late-onset Pompe disease affects approximately one in 22,000 people worldwide and causes severe muscle weakness and respiratory issues due to glycogen accumulation from enzyme deficiency.
- Maze Therapeutics reported positive Phase 1 results for MZE782, an oral SLC6A19 inhibitor, showing up to 42-fold increase in urinary phenylalanine excretion in healthy volunteers. - The drug demonstrated dose-dependent eGFR changes similar to SGLT2 inhibitors, suggesting potential kidney protective effects for chronic kidney disease patients. - MZE782 showed excellent safety profile with no serious adverse events across all dose levels, supporting advancement to Phase 2 trials in both PKU and CKD in 2026. - The compound targets SLC6A19 transporter and represents potential best-in-class therapy for phenylketonuria and first-in-class treatment for chronic kidney disease.
- Frazier Healthcare Partners has successfully closed a $1.3 billion venture capital fund specifically targeting early-stage biotechnology companies and startup creation. - The firm was one of the most active biotech investors in 2024, participating in 17 deals and leading nearly one-third of those transactions. - Recent successful exits include Scorpion Therapeutics' $2.5 billion drug sale to Eli Lilly and major acquisitions like Novartis' $3.5 billion purchase of Chinook Therapeutics. - The new fund launch comes amid declining biotech venture funding, which dropped from $7 billion to $4.8 billion in Q2 2025 according to HSBC Innovation Banking.
• Maze Therapeutics has dosed the first patient in its Phase 2 HORIZON study, evaluating MZE829 for APOL1 kidney disease (AKD). • The HORIZON trial is an open-label, basket design study including AKD patients with varying degrees of proteinuria and diabetic AKD. • The primary endpoint is a 30% or greater reduction in proteinuria, measured by urinary albumin-to-creatinine ratio (uACR) at week 12. • Interim proof-of-concept data from the HORIZON study is expected in the first quarter of 2026, according to Maze Therapeutics.
• Atlas Venture closed its fourteenth fund, securing $450 million to invest in and cultivate new biotech startups focused on developing innovative medicines. • Nuvig Therapeutics completed a $161 million Series B financing to advance its lead drug, NVG-2089, into Phase 2 trials for autoimmune diseases like chronic inflammatory demyelinating polyneuropathy (CIDP). • Maze Therapeutics raised $115 million in a Series D round to progress clinical development of its oral APOL1 inhibitor MZE829 for APOL1 kidney disease and SCL6A19 inhibitor MZE782 for chronic kidney disease. • Antag Therapeutics secured €80 million in Series A funding to develop AT-7687, a GIPR antagonist, as a next-generation weight-loss therapy, potentially used alongside incretin therapies.
• Maze Therapeutics has completed a $115 million Series D financing to support the development of precision medicines for renal, cardiovascular, and metabolic diseases. • The funding will advance MZE829, an oral APOL1 inhibitor, into a Phase 2 trial for APOL1 kidney disease (AKD), including focal segmental glomerulosclerosis (FSGS), expected in Q1 2025. • MZE782, an oral SCL6A19 inhibitor, is currently in a Phase 1 trial and is being considered as a treatment for chronic kidney disease (CKD) and phenylketonuria (PKU), with data expected in H2 2025.
• The Pompe disease pipeline is robust, featuring over 20 drugs in development across 15+ companies, targeting novel treatment approaches. • GeneCradle Therapeutics' GC301, a gene therapy, is in Phase I/II trials, showing potential for enzyme replacement therapy discontinuation and motor ability improvement. • Maze Therapeutics' MZE001, an oral glycogen synthase inhibitor, is in Phase I, aiming to reduce glycogen buildup in Pompe disease patients. • Key players like Amicus Therapeutics and Spark Therapeutics are advancing therapies such as Cipaglucosidase alfa and SPK-3006 through clinical trials.
• Maze Therapeutics' MZE829 demonstrated favorable safety and tolerability in a Phase I trial involving healthy volunteers. • Pharmacokinetic analysis suggests MZE829 is suitable for once-daily oral administration, simplifying dosing for patients. • The trial results support the advancement of MZE829 into a Phase II trial for APOL1 kidney disease (AKD), planned for early 2025. • MZE829, an APOL1 inhibitor, holds potential as a novel therapy for AKD, particularly benefiting individuals of West African descent.
• Maze Therapeutics announced positive Phase 1 results for MZE829, an oral APOL1 inhibitor, in healthy volunteers, showing it was well-tolerated with dose-proportional pharmacokinetics. • The trial supports once-daily dosing of MZE829 and its potential co-administration with standard-of-care medications for APOL1 kidney disease (AKD). • A Phase 2 open-label basket trial is planned for Q1 2025 to assess MZE829's efficacy in AKD patients with varying clinical characteristics and disease severity. • MZE829 targets APOL1, a genetically validated driver of kidney disease, offering a precision medicine approach for individuals of West African ancestry at high risk.
