Company Description
Maze Therapeutics, Inc. (Nasdaq: MAZE) is a clinical-stage biopharmaceutical company that focuses on developing small molecule precision medicines for patients with kidney and metabolic diseases, including obesity. According to the company’s public disclosures, Maze harnesses the power of human genetics to guide drug discovery and development, aiming to translate genetic insights into targeted therapies for clearly defined patient groups.
Maze is headquartered in South San Francisco, California and operates in the biotechnology industry within the healthcare sector. The company’s common stock is listed on The Nasdaq Stock Market under the ticker symbol MAZE, as noted in its SEC filings. Maze describes itself as a clinical-stage organization, meaning its lead drug candidates are in human clinical trials rather than commercial use.
Genetics-Driven Drug Discovery and the Compass Platform
Maze reports that its research and development engine is built around its proprietary Compass™ platform. The platform is designed to identify and characterize genetic variants associated with disease and then link those variants to the biological pathways that drive disease in specific patient groups. In its filings and press releases, Maze refers to this process as variant functionalization.
By integrating variant discovery with functional studies, Maze seeks to prioritize genetically validated targets for small molecule drug development. The company states that Compass is intended to inform multiple phases of the drug development process, including target selection and clinical trial design. Maze emphasizes that its goal is to use this genetics-based approach to discover and advance programs with first- or best-in-class potential in kidney and metabolic diseases.
Therapeutic Focus: Kidney and Metabolic Diseases
Across its public communications, Maze consistently describes its focus on kidney and metabolic diseases, including obesity and inherited metabolic disorders. The company highlights chronic kidney disease (CKD) and APOL1-mediated kidney disease (AMKD) as key areas of interest within nephrology, as well as phenylketonuria (PKU) within metabolic medicine.
Maze notes that AMKD is a subset of CKD that is estimated to affect over one million people in the United States alone. The company also references a broader CKD population that includes patients who inadequately respond to currently available therapies. In metabolic disease, Maze focuses on PKU, an inherited disorder characterized by elevated phenylalanine levels, and indicates that its approach is intended to be independent of specific genotypes or phenylalanine hydroxylase activity.
Lead Program: MZE829 for APOL1-Mediated Kidney Disease
Maze’s most advanced program, as described in its S-1 registration statement and press releases, is MZE829, an oral, small molecule inhibitor of apolipoprotein L1 (APOL1). MZE829 is being developed for patients with APOL1-mediated kidney disease (AMKD), which the company identifies as a genetically defined subset of CKD.
The company characterizes MZE829 as a dual-mechanism APOL1 inhibitor and notes that it is in Phase 2 clinical development. Maze is enrolling patients in the Phase 2 HORIZON trial, described as an open-label basket design study. According to Maze, this trial includes AMKD patients who carry two high-risk APOL1 alleles (G1 or G2) and covers a broad range of clinical phenotypes, including:
- Patients with diabetic kidney disease
- Patients with non-diabetic kidney disease, such as hypertensive nephropathy
- Patients with severe focal segmental glomerulosclerosis (FSGS) and nephrotic-level proteinuria
Maze states that the HORIZON trial is intended to generate proof-of-concept data in this genetically defined population. Earlier Phase 1 data, as summarized in the S-1, indicated that MZE829 was evaluated in healthy volunteers across a range of doses, with Maze reporting that treatment was well tolerated at the doses tested and that pharmacokinetics were dose-proportional with relatively low variability.
Second Lead Program: MZE782 for PKU and Chronic Kidney Disease
The company’s second lead program is MZE782, described as an investigational, potent, oral inhibitor of SLC6A19, a sodium-dependent neutral amino acid transporter expressed in the small intestine and proximal tubule of the kidney. Maze is advancing MZE782 for two indications:
- Phenylketonuria (PKU), an inherited metabolic disorder
- Chronic kidney disease (CKD), including patients who may not respond adequately to existing CKD therapies
According to Maze, SLC6A19 plays a key role in the absorption and reabsorption of neutral amino acids, including phenylalanine (Phe). In PKU, SLC6A19 enables Phe uptake from the gut and reabsorption in the kidney, both of which contribute to elevated plasma Phe levels in patients with deficient phenylalanine hydroxylase activity. Maze explains that inhibiting SLC6A19 with MZE782 offers a genotype- and PAH-agnostic oral approach to lowering plasma Phe by limiting its entry into circulation.
In CKD, Maze states that SLC6A19-mediated reabsorption may contribute to metabolic overload in the kidney’s proximal tubule. By blocking this transporter, MZE782 may reduce the burden of amino acids and toxins, which Maze suggests could potentially slow disease progression. The company notes that this mechanism is complementary to, and distinct from, SGLT2 inhibition.
MZE782 has completed a Phase 1 randomized, double-blind, placebo-controlled study in healthy adult volunteers, as detailed in Maze’s press releases. Maze reports that MZE782 was well tolerated across all tested doses, with no serious adverse events observed. The company also reports dose-dependent increases in urinary excretion of phenylalanine and glutamine, which it interprets as evidence of target engagement and SLC6A19 inhibition. In addition, Maze notes that a dose-dependent initial dip in estimated glomerular filtration rate (eGFR) was observed, similar in magnitude to changes seen with certain renoprotective therapies, and that this effect reversed after dosing stopped.
Based on these findings, Maze has stated its intention to advance MZE782 into Phase 2 proof-of-concept trials in both PKU and CKD, with trial designs focusing on plasma Phe reduction in PKU and proteinuria reduction in CKD.
Broader Pipeline and Partnerships
Beyond its two lead programs, Maze references additional research and discovery efforts that also rely on its Compass platform. Public financial disclosures note prior license agreements, including an exclusive license with Shionogi & Co., Ltd. for rights to MZE001, an investigational oral glycogen synthase 1 (GYS1) inhibitor aimed at addressing Pompe disease by limiting disease-causing glycogen buildup. Maze has also disclosed an exclusive license agreement with Trace Neuroscience, Inc. for a discovery research program targeting UNC13A for the potential treatment of amyotrophic lateral sclerosis.
These collaborations illustrate that Maze’s genetics-guided approach has been applied to multiple therapeutic areas beyond its wholly owned kidney and metabolic programs, although the company’s current clinical focus, as described in its more recent communications, centers on MZE829 and MZE782.
Corporate and Capital Markets Profile
Maze Therapeutics is incorporated in Delaware, as indicated in its S-1 filing. The company identifies itself as an emerging growth company and a smaller reporting company under U.S. securities laws. Its common stock trades on The Nasdaq Global Market under the symbol MAZE.
In September 2025, Maze entered into a securities purchase agreement for an oversubscribed private placement of common stock and pre-funded warrants. The company’s press release and related Form 8-K state that the private placement was expected to generate gross proceeds of approximately $150 million, before fees and expenses. Maze has indicated that it intends to use these proceeds, together with existing cash, to advance MZE829 in AMKD, initiate Phase 2 trials of MZE782 in PKU and CKD, continue research programs, and further develop the Compass platform.
Maze subsequently filed a registration statement on Form S-1 to register the resale of shares issued in the private placement and shares underlying the pre-funded warrants. The S-1 describes the company’s business, risk factors, and clinical programs, and confirms that Maze’s common stock is listed on Nasdaq under the MAZE ticker.
Position Within Biotechnology and Therapeutic Areas
Within the biotechnology sector, Maze positions itself as a clinical-stage precision genetics company focused on kidney and metabolic diseases. Its public materials emphasize:
- Use of human genetics to select and validate drug targets
- Development of oral small molecule therapies rather than biologics
- Concentration on renal indications such as AMKD and CKD
- Expansion into metabolic and inherited disorders such as PKU and obesity-related conditions
Maze’s approach, as described in its filings and press releases, is to align therapeutic development with genetic evidence, aiming to match specific mechanisms—such as APOL1 inhibition or SLC6A19 inhibition—to well-defined patient populations.
FAQs about Maze Therapeutics, Inc. (MAZE)
- What does Maze Therapeutics, Inc. do?
Maze Therapeutics is a clinical-stage biopharmaceutical company that uses human genetics to develop small molecule precision medicines for patients with kidney and metabolic diseases, including obesity. Its work centers on genetically validated targets and oral small molecule drug candidates. - What are Maze’s lead drug candidates?
Maze’s public disclosures identify two lead programs: MZE829, an oral APOL1 inhibitor in Phase 2 development for APOL1-mediated kidney disease, and MZE782, an oral SLC6A19 inhibitor advancing to Phase 2 with potential applications in phenylketonuria and chronic kidney disease. - What is APOL1-mediated kidney disease (AMKD) in Maze’s pipeline?
AMKD is described by Maze as a subset of chronic kidney disease associated with high-risk APOL1 genetic variants. The company estimates that AMKD affects over one million people in the United States and is developing MZE829 as a potential treatment for this genetically defined population. - How is MZE782 intended to address PKU and CKD?
According to Maze, MZE782 inhibits SLC6A19, a transporter involved in neutral amino acid absorption and reabsorption. In PKU, this mechanism is intended to lower plasma phenylalanine levels by reducing its entry into circulation. In CKD, Maze believes that reducing amino acid reabsorption may lessen metabolic stress in the kidney. - What is the Compass platform?
Maze describes its Compass platform as a genetics-based discovery and development system that identifies disease-associated genetic variants, links them to biological pathways, and supports variant functionalization. The platform is intended to guide target selection, program design, and clinical trial strategy. - Which diseases does Maze focus on?
Maze’s public materials highlight kidney and metabolic diseases, including chronic kidney disease, APOL1-mediated kidney disease, phenylketonuria, and obesity-related metabolic conditions. The company also references earlier work in Pompe disease and amyotrophic lateral sclerosis through licensing arrangements. - Where is Maze Therapeutics headquartered?
Maze states that it is headquartered in South San Francisco, California, a hub for biotechnology and life sciences companies. - On which exchange is MAZE stock listed?
Maze’s common stock is registered under Section 12(b) of the Securities Exchange Act and is listed on The Nasdaq Stock Market, specifically The Nasdaq Global Market, under the ticker symbol MAZE. - What is Maze’s corporate status under U.S. securities regulations?
In its S-1 registration statement, Maze identifies itself as an emerging growth company and a smaller reporting company, which affects certain reporting and disclosure requirements under federal securities laws. - How does Maze use proceeds from its financings?
In connection with its 2025 private placement, Maze disclosed that it intends to use the proceeds, together with existing cash and investments, to advance MZE829 in APOL1-mediated kidney disease, initiate Phase 2 trials of MZE782 in PKU and CKD, support research and discovery programs, and further develop its Compass platform, as well as for working capital and general corporate purposes.