PresentationJason ColomaCEO & Director Good morning, everyone. I'm Jason Coloma. I'm the CEO of Maze Therapeutics. And first of all, I'd like to thank the Jefferies team for inviting us to this year's conference. I'd also like to acknowledge our forward-looking statements. At Maze, our mission is simple, yet ambitious, to harness the power of human genetics to transform the lives of patients. An emphasis for us is chronic kidney disease. Kidney disease is a silent killer. Over 800 million individuals global ...

Maze Therapeutics (NasdaqGM:MAZE) 2025 Conference November 20, 2025 07:00 AM ET Company ParticipantsJason Coloma - CEOJason ColomaMorning, everyone. I'm Jason Coloma. I'm the CEO of Maze Therapeutics. First of all, I'd like to thank the Jefferies team for inviting us to this year's conference. I'd also like to acknowledge our forward-looking statements. At Maze, our mission is simple yet ambitious: to harness the power of human genetics to transform the lives of patients. An emphasis for us is chronic kidne ...

Core Insights - Maze Therapeutics has reported positive first-in-human results from the Phase 1 trial of MZE782, indicating its potential as a best-in-class therapy for phenylketonuria (PKU) and a first-in-class treatment for chronic kidney disease (CKD) [2][4][7] - The company is actively enrolling patients in the Phase 2 HORIZON trial of MZE829 for APOL1-mediated kidney disease (AMKD), with topline data expected by the end of Q1 2026 [2][6] - Maze Therapeutics has a strong financial position with $383.9 million in cash, cash equivalents, and marketable securities, providing a cash runway into 2028 [9][20] Program Progress and Anticipated Milestones - MZE829 is being developed as a treatment for AMKD, which affects over one million people in the U.S. [3] - MZE782 targets the solute transporter SLC6A19 and aims to reduce plasma phenylalanine levels in PKU and proteinuria in CKD, with Phase 2 trials planned for 2026 [4][7] Recent Corporate Highlights - In October 2025, Maze appointed Hervé Hoppenot as chairman of its board of directors [5] - The company announced an oversubscribed private placement in September 2025, raising approximately $150 million [16] Financial Performance - For Q3 2025, Maze reported a net loss of $30.1 million, compared to a net loss of $24.8 million in Q3 2024 [13] - Research and development expenses increased to $25.2 million for Q3 2025, up from $19.9 million in Q3 2024, reflecting higher clinical trial costs [11] - General and administrative expenses rose to $7.8 million for Q3 2025, compared to $6.9 million in Q3 2024 [12]

Core Insights - Maze Therapeutics is presenting seven studies at the American Society of Nephrology's Kidney Week, focusing on their lead programs MZE782 and MZE829, which target chronic kidney disease (CKD) and phenylketonuria (PKU) [1][2][3] Group 1: Company Overview - Maze Therapeutics is a clinical-stage biopharmaceutical company developing small molecule precision medicines for kidney and metabolic diseases [1][7] - The company utilizes its Compass platform to pursue genetically validated targets, integrating variant discovery and functionalization [7] Group 2: Product Pipeline - MZE782 is an oral SLC6A19 inhibitor aimed at treating PKU and CKD, showing a strong safety profile and potential to improve kidney function in Phase 1 trials [2][6] - MZE829 is an oral APOL1 inhibitor in Phase 2 development for APOL1-mediated kidney disease (AMKD), which affects over one million people in the U.S. [2][4] - The HORIZON trial for MZE829 is currently enrolling patients and aims to provide initial topline results in Q1 2026 [5] Group 3: Research Presentations - Presentations at ASN include studies on the genetic variants of APOL1 in kidney disease, the effects of small molecule inhibitors on dysfunctional signaling, and the impact of SLC6A19 variants on kidney function [3][4] - Specific presentation titles include studies on APOL1 risk variants, SLC6A19 inhibition, and pharmacokinetics of MZE782 [3]

Core Insights - Maze Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for kidney and metabolic diseases, including obesity [2] - The company will participate in four upcoming investor conferences, providing live webcasts and archived presentations [1][3] Company Overview - Maze Therapeutics utilizes human genetics to create novel small molecule precision medicines, guided by its Compass™ platform [2] - The company's leading pipeline products include MZE829, a dual-mechanism APOL1 inhibitor in Phase 2 for APOL1-mediated kidney disease, and MZE782, a SLC6A19 inhibitor advancing to Phase 2 for phenylketonuria and chronic kidney disease [2] Upcoming Events - The company will engage in the following investor conferences: - Guggenheim 2nd Annual Healthcare Innovation Conference on November 11, 2025, at 9:30 a.m. ET [3] - TD Cowen Immunology & Inflammation Summit on November 13, 2025, at 2:00 p.m. ET [3] - Jefferies Global Healthcare Conference in London on November 20, 2025, at 12:00 p.m. GMT / 7:00 a.m. ET [3] - 8th Annual Evercore Healthcare Conference on December 3, 2025, at 10:25 a.m. ET [3]

Core Insights - Maze Therapeutics has appointed Hervé Hoppenot as Chairman of the Board, succeeding Charles Homcy, M.D., who will remain on the Board [1][2] - The company is at a pivotal moment with promising first-in-human data for MZE782 and a successful $150 million private placement, positioning it for significant clinical milestones [2][3] - Maze's pipeline includes MZE829, an APOL1 inhibitor in Phase 2 for APOL1-mediated kidney disease, and MZE782, a SLC6A19 inhibitor advancing to Phase 2 for phenylketonuria (PKU) and chronic kidney disease (CKD) [4] Company Overview - Maze Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for kidney and metabolic diseases [4] - The company utilizes its Compass™ platform to pursue genetically validated targets, integrating variant discovery and functionalization [4] - The leadership of Hervé Hoppenot is expected to be instrumental in advancing the company's therapies and achieving key milestones in 2026 [3]

MZE782 Phase 1 Trial Results - MZE782 demonstrated an excellent safety profile with no serious adverse events observed[18] - MZE782 exhibited linear pharmacokinetics (PK) across single doses ranging from 30 mg to 960 mg, with a half-life of approximately 11 hours[18, 32] - The trial showed up to a 42-fold increase in urinary phenylalanine (Phe) excretion and a 68-fold increase in urinary glutamine (Gln) excretion at Day 7[18] - MZE782 induced a dose-dependent initial estimated glomerular filtration rate (eGFR) dip, similar to SGLT2 inhibitors[18] Planned Phase 2 Trials in 2026 - The company plans to initiate Phase 2 trials for both Phenylketonuria (PKU) and Chronic Kidney Disease (CKD) in 2026[18] - The PKU Phase 2 trial aims to demonstrate proof-of-concept (PoC) with plasma Phe reduction as the endpoint[56] - The CKD Phase 2 trial will focus on demonstrating PoC with proteinuria reduction as the primary endpoint[56] MZE782 Therapeutic Potential - MZE782 aims to deliver potential best-in-class plasma phenylalanine (Phe) reduction with a good safety profile for PKU patients[17] - MZE782 could be a new mechanism of action (MoA) to address CKD patients with inadequate response to current therapies, including SGLT2 inhibitors[56] Company Financial Position - The company has a strong cash position with an expected runway into 2028[12]

Core Viewpoint - Maze Therapeutics has successfully entered into an oversubscribed private placement agreement, raising approximately $150 million to advance its clinical programs and research initiatives [1][4]. Group 1: Private Placement Details - The private placement consists of 4,000,002 shares of common stock priced at $16.25 per share, along with 5,231,090 pre-funded warrants priced at $16.249 each [3]. - The transaction is expected to close on September 12, 2025, pending customary closing conditions [4]. - The placement has attracted both new and existing investors, including notable firms such as Frazier Life Sciences and Janus Henderson Investors [2]. Group 2: Use of Proceeds - Proceeds from the private placement will be utilized to further the development of MZE829 for APOL1-mediated kidney disease, initiate Phase 2 trials for MZE782 targeting phenylketonuria and chronic kidney disease, and support ongoing research and development efforts [4]. - Additional funds will be allocated for working capital and general corporate purposes [4]. Group 3: Company Overview - Maze Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for kidney and metabolic diseases [8]. - The company's lead candidates include MZE829, an APOL1 inhibitor in Phase 2 development, and MZE782, a SLC6A19 inhibitor also advancing to Phase 2 [8].

Core Insights - Maze Therapeutics has appointed Misbah Tahir as Chief Financial Officer, effective immediately, during a critical phase for the company [1][2] - The company is preparing for significant milestones, including data releases for MZE782 and MZE829 in 2025 and 2026 [2] - Mr. Tahir brings over 20 years of experience in the biopharmaceutical industry, having previously served as CFO at IGM Biosciences and held senior finance roles at other notable companies [2][3] Company Overview - Maze Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for kidney and metabolic diseases [4] - The company utilizes its Compass platform to pursue genetically validated targets, integrating variant discovery and functionalization [4] - Maze's pipeline includes MZE829, an oral APOL1 inhibitor in Phase 2 development, and MZE782, an oral SLC6A19 inhibitor in Phase 1, targeting chronic kidney disease and phenylketonuria [4] Financial Position - Maze Therapeutics has a strong balance sheet with an expected cash runway extending into the second half of 2027 [3] - The company aims to create significant value for patients and shareholders through its differentiated precision genetics platform and disciplined development approach [3]

Core Viewpoint - The National Advertising Division (NAD) is referring claims made by Maze Therapeutics regarding their drug MZE829 to regulatory authorities due to Maze's failure to respond substantively to NAD's inquiry [1][3]. Group 1: Company Overview - Vertex Pharmaceuticals and Maze Therapeutics are competitors in the development of clinical-stage treatments for APOL1-Mediated Kidney Disease (AMKD) [2]. - Maze Therapeutics has made express and implied claims about the efficacy of MZE829 compared to Vertex's drug candidate inaxaplin [2]. Group 2: Regulatory Actions - The NAD's inquiry was prompted by Vertex's request to review Maze's claims about MZE829 [2]. - Maze Therapeutics declined to participate in the NAD process, stating that it does not currently sell any product or advertise MZE829 [3]. - Due to Maze's non-participation, NAD will refer the matter to appropriate government agencies for further action [3].