Small molecule precision medicines
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Maze Therapeutics to Participate in Two Upcoming Investor Conferences
Globenewswire· 2025-07-10 20:05
Group 1 - Maze Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for patients with renal, metabolic, and cardiovascular diseases [2] - The company will participate in two upcoming investor conferences in July, with live webcasts available on their website [1] - Maze Therapeutics utilizes its Compass platform to link genetic variants with biological pathways in disease, advancing a pipeline that includes two wholly owned programs, MZE829 and MZE782 [2] Group 2 - The H.C. Wainwright 4th Annual Kidney Virtual Conference is scheduled for July 14, 2025, at 2:30 p.m. ET [3] - The BTIG Virtual Biotech Conference will take place on July 30, 2025, at 9:20 a.m. ET [3]
Maze Therapeutics Reports First Quarter 2025 Financial Results and Reiterates Upcoming Milestones
GlobeNewswire News Room· 2025-05-14 20:01
Core Insights - Maze Therapeutics is advancing two clinical programs, MZE829 and MZE782, which are expected to drive growth and innovation in the treatment of chronic kidney disease (CKD) and phenylketonuria (PKU) [2][10] Clinical Programs - MZE829 is an oral small molecule APOL1 inhibitor targeting APOL1 kidney disease (AKD), a subset of CKD affecting over one million people in the U.S. The Phase 2 HORIZON trial is actively enrolling patients and initial data is expected in Q1 2026 [3][7] - MZE782 is an oral small molecule targeting the solute transporter SLC6A19, with potential as a first-in-class treatment for approximately five million U.S. patients with CKD who do not adequately respond to existing therapies, and as a best-in-class therapy for PKU. Initial data from the Phase 1 trial is expected in Q3 2025 [4][8] Financial Performance - As of March 31, 2025, Maze Therapeutics reported cash and cash equivalents of $294.4 million, an increase from $196.8 million as of December 31, 2024. This cash position is expected to fund operations into the second half of 2027 [5][14] - Research and development expenses for Q1 2025 were $27.6 million, up from $21.9 million in Q1 2024, primarily due to increased clinical trial costs for MZE829 and MZE782 [6] - General and administrative expenses rose to $7.8 million in Q1 2025 from $6.1 million in Q1 2024, reflecting higher personnel-related costs [9] Company Overview - Maze Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for renal, cardiovascular, and metabolic diseases. The company utilizes its Compass platform to link genetic variants with disease pathways [10]
Maze Therapeutics to Participate in the 3rd Annual H.C. Wainwright BioConnect Investor Conference
GlobeNewswire News Room· 2025-05-13 11:30
Company Overview - Maze Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecule precision medicines for patients with renal, metabolic, and cardiovascular diseases [3] - The company utilizes its Compass platform to gain insights into genetic variants associated with diseases and their biological pathways [3] Upcoming Event - Jason Coloma, Ph.D., CEO of Maze Therapeutics, will present a company overview and engage in one-on-one meetings at the 3rd Annual H.C. Wainwright BioConnect Investor Conference on May 20, 2025 [1][2] - The presentation is scheduled for 11 a.m. ET and will be accessible on the company's website, with a replay available for 60 days [2] Pipeline and Programs - Maze Therapeutics is advancing a pipeline led by two wholly owned programs, MZE829 and MZE782, which represent novel precision medicine approaches for patients [3]
Rapport Therapeutics to Present Data at the 2025 American Academy of Neurology Annual Meeting
Newsfilter· 2025-03-21 11:00
Core Insights - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing small molecule precision medicines for neurological and psychiatric disorders [4] - The company will present data on its investigational drug RAP-219 at the 2025 American Academy of Neurology Annual Meeting, highlighting its potential in treating refractory focal epilepsy [1][2] Group 1: Company Overview - Rapport Therapeutics specializes in discovering and developing precision medicines targeting neurological and psychiatric disorders, leveraging its RAP technology platform [4] - The company's lead product, RAP-219, is designed to selectively target TARPγ8, a receptor associated protein, to achieve neuroanatomical specificity [3][4] - The company is also exploring RAP-219 for other conditions, including bipolar disorder and peripheral neuropathic pain, indicating a broad therapeutic potential [3][4] Group 2: Research and Development - RAP-219 is currently in Phase 2 development for refractory focal epilepsy, showcasing its role as an AMPA receptor negative allosteric modulator [2][3] - The upcoming presentations will include findings on the optimal cut point for seizure frequency reduction and the antiseizure effects of RAP-219 in preclinical models [6] - Data from the presentations suggest a 30% reduction in long episode frequency correlates with a clinically meaningful reduction in seizures, and RAP-219 demonstrated potent, dose-dependent antiseizure effects in preclinical models [6]
Rapport Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update
Globenewswire· 2025-03-11 11:00
Core Viewpoint - Rapport Therapeutics is advancing its lead program RAP-219, targeting neurological and psychiatric disorders, with significant clinical milestones expected in the near future [2][4]. Business Highlights - The Phase 2a trial of RAP-219 for refractory focal epilepsy is progressing well, with topline results anticipated in Q3 2025 [2][4]. - A Phase 2a trial for bipolar mania is expected to start in Q3 2025, with results projected in the first half of 2027 [6]. - The company appointed Dr. Jeffrey Sevigny as Chief Medical Officer, bringing extensive experience in drug development [8]. - As of December 31, 2024, the company reported $305.3 million in cash and equivalents, sufficient to fund operations through the end of 2026 [4][12]. Clinical Development - Recent PET and MAD-2 trial results support RAP-219's potential for treating CNS disorders, demonstrating neuroanatomical specificity and favorable tolerability [5][10]. - The company is finalizing plans for a Phase 2a trial in diabetic peripheral neuropathic pain after receiving feedback from the FDA [7]. Financial Results - For Q4 2024, the net loss was $20.0 million, compared to $13.5 million in the same period of the previous year [11]. - Research and Development expenses increased to $17.2 million in Q4 2024 from $11.8 million in Q4 2023, reflecting higher operational costs [11]. - General and Administrative expenses rose to $6.3 million in Q4 2024 from $3.0 million in Q4 2023, driven by business growth and public company requirements [11]. Cash Position - The company ended Q4 2024 with $305.3 million in cash, down from $320.7 million at the end of Q3 2024, primarily due to operating cash outflows [11][12].