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 - The biotech sector is experiencing significant momentum, with many clinical-stage and commercial biopharma stocks reaching new 52-week highs due to breakthrough trial data, strategic deals, and earnings surprises [1] Company Summaries - **Metsera Inc. (MTSR)**: A clinical-stage biopharmaceutical company focused on developing hormone analog peptides for obesity and related metabolic diseases. Recently, Novo Nordisk made a $9 billion unsolicited acquisition proposal, which Metsera's board considers superior to its existing agreement with Pfizer [2][3]. Metsera announced positive Phase 2b results for its GLP-1 receptor agonist MET-097i, showing up to 14.1% weight loss after 28 weeks, supporting Phase 3 initiation in late 2025 [4]. The stock rose from $32.35 to a 52-week high of $66.10, a gain of 104.3% [5]. - **Indivior Plc (INDV)**: Develops buprenorphine-based therapies for opioid dependence. The company reported Q3 net income of $42 million, up from $22 million a year ago, with adjusted earnings of $93 million, exceeding Wall Street's expectations [6][7]. The stock increased from $20.86 to a 52-week high of $30.55, reflecting a 46.5% gain [7]. - **Insmed Inc. (INSM)**: Focused on therapies for serious and rare diseases, Insmed reported a Q3 net loss of $370 million but saw net product revenue rise to $142.3 million from $93.4 million last year [8][9]. The company raised its full-year 2025 revenue guidance for ARIKAYCE to $420 million - $430 million, indicating 15% - 18% growth year-over-year [10]. The stock surged from $76.54 to a 52-week high of $194.70, marking a 154.4% gain [10]. - **Ventyx Biosciences Inc. (VTYX)**: A clinical-stage biotech company developing therapies for autoimmune and neurodegenerative diseases. Ventyx reported positive Phase 2 results for its NLRP3 inhibitor VTX3232, showing strong safety and tolerability [11][13]. The stock rose from $3.01 to a 52-week high of $8.52, a gain of over 183% [14]. - **Inhibrx Biosciences Inc. (INBX)**: Focuses on oncology and rare diseases, announcing positive topline results from its ChonDRAgon study for ozekibart in chondrosarcoma [15]. The stock increased from $18.35 to a 52-week high of $83.78, representing a gain of 356.6% [16]. - **ABIVAX Société Anonyme (ABVX)**: Developing therapies for chronic inflammatory diseases, ABIVAX presented positive Phase 3 data for obefazimod in ulcerative colitis [17]. The stock rose from $7.83 to an all-time high of $106.73, marking a significant gain of 1263% [18]. - **Arrowhead Pharmaceuticals Inc. (ARWR)**: Developing RNAi-based therapies, Arrowhead finalized a licensing agreement with Novartis for ARO-SNCA, with financial terms including a $200 million upfront payment [19][20][21]. The stock increased from $29.70 to a 52-week high of $43.33, representing a gain of over 45% [21]. - **Kodiak Sciences Inc. (KOD)**: Focused on retinal diseases, Kodiak announced positive Phase 1b data for KSI-101, showing significant vision improvements [22][23][24]. The stock climbed from $8.98 to a 52-week high of $21.17, delivering a 135.7% gain [22]. - **Arcutis Biotherapeutics Inc. (ARQT)**: Focused on dermatological treatments, Arcutis reported Q3 net income of $7.4 million, a turnaround from a net loss last year, with revenue up 122% year-over-year [25][26]. The stock rose from $14.99 to a 52-week high of $27.08, representing a gain of over 80% [27]. - **Maze Therapeutics Inc. (MAZE)**: Developing precision therapies for various diseases, Maze announced positive Phase 1 results for MZE782 and secured $150 million in a private placement [28][29]. The stock rose from $11.21 to a new 52-week high of $34.29, returning a gain of 206% [29]. - **Supernus Pharmaceuticals Inc. (SUPN)**: Focused on CNS disorders, Supernus is expected to report Q3 earnings of $0.82 per share and revenue of $180.22 million [30][31]. The stock increased from $38.21 to a 52-week high of $57.65, representing a gain of over 50% [31].

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]

Company Highlights - Cidara Therapeutics Inc. (CDTX) reached a 52-week high of $87.19 as it advances its development plan for CD388, a non-vaccine preventive treatment for seasonal influenza, with enrollment in a Phase 3 study expected to start by September 2025 targeting 6,000 participants [1] - Maze Therapeutics Inc. (MAZE) saw its stock rise to a 52-week high of $27.09 following positive Phase 1 clinical results for MZE782, an oral therapy for Phenylketonuria and chronic kidney disease, with plans to enter Phase 2 studies in 2026 [3][4] - Medpace Holdings Inc. (MEDP) is set to report Q3 2025 financial results on October 22, 2025, and has raised its full-year guidance twice, indicating strong operational momentum, with the stock hitting a 52-week high of $506.55 [5][6] - Monopar Therapeutics Inc. (MNPR) rebounded from potential Nasdaq delisting to a 52-week high of $73.75, planning to seek approval for ALXN1840 for Wilson disease in early 2026, supported by long-term clinical data [7][8] - Prenetics Global Limited (PRE) projects 2025 revenue between $85 million and $100 million, a significant increase from $30.6 million in 2024, with its new wellness brand IM8 Health on track for $100 million in annual recurring revenue [9][10] - Protagonist Therapeutics Inc. (PTGX) is nearing a major milestone with Icotrokinra for psoriasis, having submitted a regulatory application to the FDA and EMA in July 2025, with the stock reaching a 52-week high of $66.70 [11][12] - uniQure N.V. (QURE) announced positive results from its Phase I/II study of AMT-130 for Huntington's disease, planning to submit a Biologics License Application in Q1 2026, with the stock soaring over 200% to a 52-week high of $51.21 [13][14] - Rezolute Inc. (RZLT) surged to a 52-week high of $9.02 ahead of topline results from its pivotal Phase III trial for hypoglycemia treatment, expected in December [15][16] - United Therapeutics Corp. (UTHR) reached a 52-week high of $442.01 as anticipation builds for data from the TETON-2 study of nebulized Tyvaso in idiopathic pulmonary fibrosis, with positive results reported earlier [17][18]

Core Thesis - Maze Therapeutics, Inc. is experiencing significant momentum in the small-cap biotech sector, driven by strong institutional backing, positive clinical data, and increased trading volume [2][4]. Financial Developments - The company secured a $150 million private placement at $16.25 per share, which is above the market price, indicating strong institutional confidence in its pipeline [2]. - The funding extends the company's cash runway into 2027, alleviating near-term dilution concerns and providing management with the flexibility to advance development [3]. Stock Performance - Following recent developments, MAZE stock surged 56% on September 11, rising from $16 to $25 on ten times average volume, before stabilizing in the $22–23 range [3]. - Accumulation above the $20 support zone is seen as an attractive entry point for investors, with a stop-loss at $18 to manage risk [3]. Clinical Progress - Maze's lead program, MZE782, has shown robust Phase 1 safety and compelling pharmacodynamics in a rare kidney disease, positioning it as a potential market differentiator [2][4]. Market Outlook - The setup presents a highly asymmetric risk/reward profile, with defined downside and substantial upside potential if clinical and financing catalysts continue to develop [4]. - The stock is viewed as a unique opportunity in biotech, with targets of $26, $30, and potentially $32+ if momentum persists [3][4].

Group 1: Biotechnology Investment Overview - Investing in biotechnology stocks is complex due to the underlying science and high volatility, with double-digit price movements common [1] - Biotech stocks present a compelling risk-reward proposition for long-term investors willing to endure clinical trial phases [1] Group 2: Viking Therapeutics (VKTX) - Viking Therapeutics stock surged in 2024 due to the popularity of GLP-1 weight loss drugs but dropped over 40% in 2025, including a 20% decline after an August clinical trial update [3][4] - The clinical trial showed an average weight loss of 12.2% among patients, but concerns arose over a high dropout rate of 28% and the highest dosage results compared to competitors [4] - Analysts have set a consensus price target of $87.50 for VKTX, indicating a potential upside of 270% from current levels [4] Group 3: ImmunityBio (IBRX) - ImmunityBio stock increased by approximately 8.4% in 2025, primarily due to positive pilot study results for glioblastoma, where all five patients achieved 100% disease control [9][10] - ANKTIVA, the company's drug, received FDA approval for bladder cancer and is being tested for other cancers, HIV, and Long COVID, making it a promising immunotherapy candidate [10] - Analysts have a consensus price target of $10.75 for IBRX, suggesting a potential gain of over 280, but caution is advised as the stock is trading above its 200-day SMA with an RSI of 76 [11] Group 4: Maze Therapeutics (MAZE) - Maze Therapeutics stock has risen approximately 86% since its public trading began in February 2025, with analysts optimistic about further growth [14] - The company reported positive Phase 1 results for its lead candidate MZE782, which targets phenylketonuria (PKU) and chronic kidney disease (CKD), allowing progression to Phase 2 trials [15] - Analysts project a price target of $32.67 for MAZE, with the most bullish estimate at $50, indicating potential for significant growth [16]

Takeda Pharmaceutical - Rhonda Pacheco will become the head of Takeda's U.S. business unit on September 29, succeeding Julie Kim, who will transition to CEO in June 2026 [2] Maze Therapeutics - Shares of Maze Therapeutics increased by over 50% following positive Phase 1 data for its oral medicine MZE782, which showed good tolerance and exceeded expectations for amino acid levels [2] - Maze Therapeutics raised $150 million in a private stock offering alongside the positive results [2] Intercept Pharmaceuticals - Intercept Pharmaceuticals will withdraw its liver disease drug Ocaliva from the U.S. market at the FDA's request, which has also suspended all clinical trials for the drug due to safety concerns [2] - Ocaliva has faced scrutiny since its approval in 2016, with a black box warning added in 2018 and ongoing issues with approval for other indications [2] Soleno Therapeutics - Soleno Therapeutics' stock fell nearly 20% after a patient death was reported to the FDA, although the treating physician did not believe the drug Vykat was related to the incident [2] - The company has seen its shares lose about one-third of their value since a report by Scorpion Capital raised safety concerns about Vykat, the first approved drug for Prader-Willi syndrome [2]