Anti-inflammatory drug cromolyn successfully delayed disease onset and was neuroprotective in a mouse model of ALS
Company expects to advance its re-engineered inhaled cromolyn candidate AZT-101 into clinical development for patients with ALS in 1H 2020

BOSTON, Mass., December 3, 2019 AZTherapies, Inc., a biopharmaceutical company developing therapeutics to extend brain health, today announced the publication of preclinical data in Scientific Reports demonstrating the anti-inflammatory properties of cromolyn in a mouse model of amyotrophic lateral sclerosis (ALS). The study was conducted by investigators at Massachusetts General Hospital in collaboration with AZTherapies.

The publication, titled “Cromolyn Sodium Delays Disease Onset and is Neuroprotective in the SOD1G93A Mouse Model of Amyotrophic Lateral Sclerosis,” supports the potential of targeting neuroinflammation with cromolyn to treat ALS, as evidenced by injected cromolyn’s ability to delay the onset of neurological symptoms, reduce neuroinflammation, decrease the expression of pro-inflammatory cytokines and chemokines, increase spinal cord motor neuron survival, and decrease denervation of muscle in a mouse model of ALS.

“These results further support the hypothesis that inflammation plays a significant role in the progression of ALS,” said lead author Ghazaleh Sadri-Vakili, Ph.D., Director of the NeuroEpigenetics Laboratory at the Sean M. Healey & AMG Center for ALS and the MassGeneral Institute for Neurodegenerative Disease. “We therefore believe that exploring anti-inflammatory treatments may be of great value in developing an effective treatment for the disease.”

“AZTherapies is advancing several clinical candidates that use our re-engineered version of inhaled cromolyn to target the neuroinflammation we believe to be a root cause of a number of serious neurodegenerative diseases, including Alzheimer’s disease and ALS,” said David R. Elmaleh, Ph.D., Founder, CEO, and Chairman of AZTherapies and an author of the publication. “While the exact cause of ALS is not fully understood, accumulating evidence suggests that during both ALS and Alzheimer’s disease progression, cromolyn can shift activated microglia from the aggressive, pro-inflammatory state to a neuroprotective state. These new published data strengthen earlier results and further support the scientific rationale behind our clinical pipeline.”

Dr. Elmaleh continued: “Importantly, unlike commercially available versions of cromolyn, our development programs use a proprietary formulation designed to cross the blood brain barrier and reach critical areas of inflammation. We are currently nearing completion of the Phase 3 clinical trial of our lead program, ALZT-OP1, for the treatment of early Alzheimer’s disease, and we anticipate initiation of a Phase 2a study of our AZT-101 candidate in the treatment of ALS during the first half of 2020.”

About AZTherapies

AZTherapies is an advanced clinical-stage biopharmaceutical company developing novel therapies that aim to fundamentally change neurodegenerative disease progression, extending normal cognition and function and improving quality of life in the aging population. Advancing a growing pipeline of candidates to treat patients with few therapeutic options, our lead candidate, ALZT-OP1, is built on a multi-modal approach that recognizes neuroinflammation as a root cause of serious neurodegeneration and seeks to stop or slow the progression of disease. Our Phase 3 COGNITE trial in early Alzheimer’s disease is fully enrolled and we are expecting the first clinical results in late 2020. Following our lead program, we are advancing candidates for the treatment of post-ischemic stroke cognitive impairment, amyotrophic lateral sclerosis (ALS), and other indications. AZTherapies is a private company headquartered in Boston, Massachusetts. To learn more, please visit


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