ALZT-OP1 is a combined oral-inhaled therapy being developed by Boston-based AZTherapies to slow the progression of early Alzheimer’s disease.

How ALZT-OP1 works

In Alzheimer’s, inflammation of the brain, or neuroinflammation, can destroy brain cells. This is caused by long-term activation of brain immune cells, such as astrocytes and microglia, that release excessive amounts of inflammation-causing chemicals known as cytokines, which can damage nerve cells. Scientists believe the accumulation of Aβ plaques in the brains of Alzheimer’s patients triggers the neuroinflammatory process.

ALZT-OP1 is a two-part therapy that consists of a dry-powder inhaler containing cromolyn (designated ALZT-OP1a) and an oral pill containing ibuprofen (designated ALZT-OP1b).

Cromolyn, a mast-cell stabilizer used to treat asthma, has been shown to stop Aβ plaque formation in the brain as well as reduce the release of inflammation-causing cytokines. Ibuprofen, a non-steroidal, anti-inflammatory medication commonly used as a painkiller, also acts to slow the inflammatory process in Alzheimer’s.

By targeting different parts of the immune system, ALZT-OP1 can stop the destruction caused by neuroinflammation, potentially slowing down or even stopping progression of Alzheimer’s while the disease is in its early stages.

ALZT-OP1 in clinical trials

A Phase 1 clinical trial (NCT02482324) that tested the pharmacokinetics of ALZT-OP1 in 26 healthy volunteers found that the combination therapy resulted in adequate blood and brain concentrations needed to slow neural damage and disease progression. The trial, which began and ended in 2015, took place at the Panax Clinical Research in Miami, Florida.

COGNITE, a Phase 3 clinical trial (NCT02547818) for ALZT-OP1 now underway, will assess the safety, tolerability and efficacy of the ALZT-OP1 combo therapy in 600 participants with early-stage Alzheimer’s, ages 55 to 79. Researchers also seek to determine the effect of ALZT-OP1 therapy on the severity of dementia symptoms.

Participants are divided into four treatment groups. Group 1 receives the ALZT-OP1a inhalational therapy plus an oral placebo pill; group 2 receives ALZT-OP1a inhalational therapy plus ALZT-OP1b oral therapy; group 3 receives ALZT-OP1b oral therapy plus inhaled placebo, and group 4 receives both inhaled and oral placebos. Patients receive these treatments over the course of one and a half years.

The trial began in September 2015 across U.S., Australian and Eastern European sites, and will conclude in September 2019.

Further details

In 2014, AZTherapies obtained a patent on the combined cromolyn-ibuprofen therapy used to treat Alzheimer’s and related disorders. The company is also investigating ALZT-OP1a in a Phase 2 clinical trial (NCT03202147) as an adjuvant treatment of post-stroke cognitive impairment.

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