Maze Therapeutics
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- Employees
- 121
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- Introduction
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.
Therini Bio Secures $39M in Series A Extension to Advance Novel Fibrin-Targeting Therapy for Neurodegenerative Diseases
• Therini Bio has raised $39 million in Series A extension financing, bringing total Series A funding to $75 million with support from new investors Angelini Ventures and Apollo Health Ventures.
• The funding will advance Phase 1b trials of THN391, a first-in-class monoclonal antibody targeting fibrin-mediated neuroinflammation in Alzheimer's Disease and Diabetic Macular Edema.
• THN391 demonstrated promising safety and pharmacokinetic profiles in Phase 1a trials, with no adverse hematological effects and a half-life supporting monthly dosing regimens.
Ascentage Pharma's IPO Funds Global Expansion of Cancer Therapies
• Ascentage Pharma raised $126.4 million in a U.S. IPO to support late-stage clinical development of olverembatinib and lisaftoclax, aimed at treating CML and CLL, respectively.
• Olverembatinib, already approved in China for CML, is undergoing Phase 3 trials in the U.S., Canada, Australia, and China, with plans for an FDA submission in 2026.
• Lisaftoclax, a Bcl-2 inhibitor, is under regulatory review in China for advanced CLL/SLL, with potential to compete with AbbVie and Genentech's Venclexta.
• Takeda Pharmaceuticals holds an option to license olverembatinib outside of Greater China and Russia, potentially bringing Ascentage up to $1.2 billion in milestone payments.
Aardvark Therapeutics Files for $100M IPO to Advance Novel Hyperphagia Treatment
• Aardvark Therapeutics has filed for a $100 million IPO on NASDAQ, seeking to advance its Phase III drug candidate ARD-101 for treating hyperphagia in Prader-Willi syndrome.
• The company's lead candidate ARD-101 is an oral gut-restricted small molecule that works by inhibiting bitter taste receptors, with Phase III trial results expected in early 2026.
• Aardvark plans to expand ARD-101's clinical development to include Phase II trials for hyperphagia associated with acquired hypothalamic obesity from craniopharyngioma treatment.
Sionna Therapeutics Launches $191M IPO to Challenge Vertex in Cystic Fibrosis
• Sionna Therapeutics has launched a $191 million IPO to fund its cystic fibrosis drug development program, aiming to compete with Vertex Pharmaceuticals.
• Sionna's approach focuses on stabilizing a difficult-to-drug region of the CFTR protein, potentially restoring function more effectively than existing therapies.
• The company plans to advance a two-drug combination into late-stage testing and evaluate one of its stabilizers alongside Vertex's Trikafta.
• Sionna has secured substantial venture funding and is among several biotechs pursuing IPOs, despite recent market challenges for newly public companies.
Metsera Aims for $289 Million IPO to Advance GLP-1RA Obesity Treatment
• Metsera is seeking a $289 million IPO to fund the Phase III trial of its lead candidate, MET-097i, an injectable GLP-1 receptor agonist for obesity.
• MET-097i demonstrated up to 20% weight loss in Phase II trials, showcasing a long half-life that could allow for less frequent injections.
• The company is also advancing other pipeline candidates, including MET-233i (ultra-long acting injectable) and MET-002 (oral GLP-1RA), to address the growing obesity market.
• Metsera's IPO is viewed as a positive sign for the biopharma fundraising landscape, potentially catalyzing other companies to enter the public markets.
Maze Therapeutics Initiates Phase 2 Trial of MZE829 for APOL1 Kidney Disease
• 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.
Maze Therapeutics Secures $115 Million to Advance Kidney Disease 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.
Pompe Disease Pipeline Shows Promise with Novel Therapies in Development
• 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.
Trace Neuroscience Launches with $101M to Tackle ALS Using Antisense Technology
• Trace Neuroscience launched with $101 million in funding to develop genetic medicines for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease with limited treatment options.
• The company's approach is based on recent discoveries linking RNA-processing molecules to proteins impaired in ALS, aiming to target the underlying cause of the disease.
• Trace plans to leverage learnings from Biogen's Qalsody, an antisense oligonucleotide approved for ALS, to accelerate its drug development timeline and enter human trials by early 2026.
• The startup focuses on human genetics and a well-defined mechanism of action to mitigate risks associated with ALS drug development, aiming for meaningful clinical data.
Maze Therapeutics' MZE829 Shows Promise in Phase I Trial for APOL1 Kidney Disease
• 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' MZE829 Shows Promise in Phase 1 Trial for APOL1 Kidney Disease
• 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.
Advancements in Pompe Disease Treatment: A Pipeline Review
• Pompe disease, a rare genetic disorder, is seeing advancements with over 20 therapies in the pipeline.
• Key players like Amicus Therapeutics and Spark Therapeutics are developing novel treatments, including gene therapies and enzyme replacements.
• Clinical trials are underway, evaluating the safety and efficacy of drugs like Cipaglucosidase alfa and SPK-3006.
• These emerging therapies target various mechanisms, such as alpha-glucosidase replacement and glycogen synthase kinase modulation.
Recordati Acquires Global Rights to Sanofi's CAD Treatment Enjaymo in $825M Deal
• Recordati has secured worldwide rights to Enjaymo, the first and only approved treatment for cold agglutinin disease (CAD), from Sanofi for an upfront payment of $825 million with potential milestone payments of $250 million.
• Enjaymo, a humanized monoclonal antibody targeting C1s in the classical complement pathway, has generated approximately €100M in revenue over the past year and is projected to reach peak sales of €250-300M.
• The acquisition strengthens Recordati's rare disease portfolio and addresses an unmet medical need, with Enjaymo already approved by regulatory authorities in the US, Europe, and Japan.
Maze Therapeutics Initiates Phase 1 Trial of MZE782 for Chronic Kidney Disease
• Maze Therapeutics has begun a Phase 1 clinical trial for MZE782, a novel oral small molecule targeting SLC6A19, in healthy volunteers.
• MZE782 aims to address the unmet needs of approximately five million CKD patients in the U.S. who do not respond adequately to current treatments.
• The trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of MZE782, with initial results expected in the second half of 2025.
• MZE782 represents a potential first-in-class therapy that could be used alone or in combination with existing standard-of-care treatments for CKD.
Inaxaplin Shows Promise in Addressing APOL1-Mediated Kidney Disease Disparity in Black Americans
• A clinical trial of inaxaplin, a novel drug targeting the APOL1 protein, demonstrated a significant reduction in protein leakage in the urine of patients with serious kidney disease.
• The study focused exclusively on African American participants with APOL1 gene variants (G1 and G2) linked to a higher risk of renal failure, addressing a significant racial health disparity.
• Vertex Pharmaceuticals is conducting a larger Phase 2/3 trial to investigate inaxaplin's efficacy across multiple kidney disease types in individuals with two APOL1 gene variants.
• Insights into APOL1 may also refine kidney transplant policies by using APOL1 genotype instead of race to assess donor kidney viability, potentially increasing the number of available kidneys.
Maze Therapeutics Secures $190 Million to Advance Precision Medicine Programs
• Maze Therapeutics has raised $190 million to support its nine precision medicine programs targeting genetically defined diseases.
• The funding will advance clinical trials for MZE001, a treatment for Pompe disease, expected to begin in the first half of 2022.
• Maze's Compass platform, designed to accelerate drug discovery through genetic and genomic data analysis, will also be expanded.
• The financing will also support programs for chronic kidney disease and amyotrophic lateral sclerosis (ALS).
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