Harnessing the Power of Neuro-Immunology with AZTherapies

Harnessing the Power of Neuro-Immunology with AZTherapies

Company & Pipeline Overview

Due to neurodegenerative diseases, more than 10 million people in the US suffer from a cognitive and physical impairment. AZTherapies, Inc. is on a mission to improve the lives of these 10 million people through neuroimmunology. A private clinical-stage biopharmaceutical company, AZTherpaies, is headquartered in Boston, Massachusetts. The company is developing small molecule and biologic therapies that target neuroinflammatory mechanisms with the hopes of changing the progression of neurodegenerative diseases.

Very little progress has been seen in early diagnosis and discovery of effective treatments for such neuroinflammatory diseases, resulting in post-stroke cognitive impairment. So, AZTherapies is trying to fill the gap with their therapeutic strategies and aiming to extend normal cognition and function and improve the quality of life in an aging population. AZTherapies has a world-class management team, scientific advisors, a Board of Directors, and a Business Board Advisory. Leading positions are held by:

  • Chief Executive Officer – Marc de Garidel
  • Chief Scientific Officer & Executive Chairman – David R. Elmaleh, PhD
  • Chief Financial & Accounting Officer – Brian C. Bartlett

Founders and scientists at AZTherapies have been pioneers in putting forth evidence of neuroinflammation playing a role in neuronal death. AZTherapies has discovered ways to potentially shift microglia cells’ chronic proinflammatory state (as seen in these diseases) to their neuroprotective state to tackle neuroinflammatory and neurodegenerative diseases.

AZTherapies has more than 100 patents and pending applications in its name, has been very active in conducting clinical trials, and has a robust pipeline. Recently, AZTherapies has completed their Phase 3 ALZT-OPI. It was a global, placebo-controlled, randomized 72-weeks long clinical trial conducted under Special Protocol Assessment SPA with the U.S Food and Drug Administration.

During this phase, the evaluation and efficacy of ALZT-OPI were conducted in which early-stage Alzheimer’s patients were given an investigational combination treatment. The data analysis has begun to validate their hypothesis that targeting neuroinflammation could be the key to modifying disease progression. AZTherapies will now be advancing candidates for the treatment of Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. To learn more, please visit: http://www.aztherapies.com/

In this article, we will cover the science portfolio, publications, and pipeline of the AZTherapies company.

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AZTherapies Portfolio

AZtherapies is building its name in the pharma and biotech industry through its research on finding possibilities to treat neurodegenerative disease by targeting neuroinflammation.

The scientists at AZTherapies have been successfully building clear research on the role of neuroinflammation in neuronal death in the brain and spinal. Their research indicates that within the CNS maintaining healthy microglia is the key to slowing down neurodegeneration.

Having made this observation they, are following an approach to shifting microglia into its neuroprotective state to produce drugs for the treatment of neurodegenerative diseases, some of which are:

Alzheimer’s Disease

AZTherapies had developed ALZT-OP1 for the treatment of early Alzheimer’s treatment. It comprises two approved small molecules that have been optimized to cross the blood-brain barrier and suppress the neuroinflammatory response in the brain. The approach is to halt/ decrease the formation and build-up of amyloid plaque by recruitment of Microglia.


Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease. It affects the brain and the spinal system and impairs motor function, often leading to paralysis and, eventually, death. AZTherapies is working on a pre-clinical ALZT-OP1a (SOD1) model.

Currently, evaluation of safety and efficacy is being carried out in a phase 2a clinical trial with mild to moderate stage ALS.


Tregs are regulatory T-cells that maintain the homeostasis of the immune system. Taking advantage of their regulation function, researchers at AZTherapies are engineering Tregs with chimeric antigen receptors (CAR), forming CAR-Treg to target the CNS.

Currently, this is being validated in human in vitro assays and in vivo models of neurodegenerative disease.

AZTherapies Publications

Some of the key publications that highlight the ongoing research and pipeline of AZTherapies are titled:

AZTherapies Pipeline

AZTherapies has a broad and fast-growing pipeline to cater to all types of neurodegenerative diseases and develop treatments. It has the potential to treat a broad array of serious diseases due to unchecked neuroinflammation.

The pipeline includes the following stages:

  1. Discovery
  2. Preclinical
  3. Phase 1
  4. Phase 2
  5. Phase 3

AZTherapies has candidates at different stages of the pipeline, and they are:


ALZT-OP1 is a first-in-class candidate that is based on a unique multimodal approach. Combination treatment includes an oral Ibuprofen plus an optimized formulation of cromolyn.  Both are re-engineered to suppress the brain’s neuroinflammatory response and come as a convenient and easy-to-use pulmonary and oral delivery kit.

The Phase 3 Cognite trial with early Alzheimer’s patients has been completed and qualifies for a 505(b) 2 streamlined FDA approval process.


ALZT-OP1a is for the treatment of Amyotrophic Lateral Sclerosis (ALS). A pre-clinical ALS (SOD1) model has been designed to demonstrate that cromolyn sodium can significantly delay disease onset and progress, reduce motor defects and reduce proinflammatory cytokine levels in the spinal cord and plasma.

Based on this, AZTherapies is evaluating the safety and efficacy of ALZT-OP1a  in a phase II clinical trial.


The CAR-Treg program is an advanced lead program to engineer Tregs with chimeric antigen receptors (CAR) to direct Tregs to the CNS to restore the balance of inflammatory and regulatory cells in the brain.

AZTherapies is validating CAR-Treg in various models of neurodegenerative disorders.

Next Generation

Scientists at AZTherapies are developing a novel, next-generation drug candidate for treating  a range of neurodegenerative diseases. They expect to file an investigational new drug (IND) application in 2022.

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AZTherapies is a private biopharmaceutical company conducting breakthrough research and clinical trials to introduce treatment methods for early Alzheimer’s disease by changing the neurodegeneration disease progression and improving the quality of life of the aging population. They also have a vision for the treatment of other diseases, like Parkinson’s, Stroke, and ALS, and are running a pipeline to deliver drugs for these diseases.

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