“Best-in-Class Science and Research, Leading to Exciting New Products”
A novel program, shaping the future of Alzheimer’s treatment
Our current programs for drug development and commercialization have the following foci: to neutralize the amyloid triggers of Alzheimer’s disease (ALZT-OP1), to facilitate drug regimen dosing and compliance in persons that already experience some memory loss (ALZT-Patch), and to lessen the impact of potential damage already done by Alzheimer’s disease (ALZT-QoL).
ALZT-OP1 treatment for Alzheimer’s disease. Our lead product candidate ALZT-OP1 combines two easily administered, small molecule drugs:
- ALZT-OP1a, delivered by inhalation, has been shown to inhibit the formation of amyloid-beta protein aggregates, or Aβ plaques, that intoxicate neurons and block their signaling; and inhibits production of pro-inflammatory cytokines.
- ALZT-OP1b, delivered orally, treats the early invisible neuroinflammation response in the brain that triggers nerve death and progressive brain damage.
When administered together, the combination treatment is intended to attenuate the multiple triggers leading to the neurodegeneration and neuronal death associated with AD. We believe that the decline in cognitive performance and function seen in the disease can be reversed, due to preserved or improved neuronal plasticity and neurogenesis in the hippocampus, if disease progression is arrested at a very early stage.
Our proprietary use of drug combination, drug formulation, dosing, and method of delivery achieves blood and brain concentrations necessary to slow neural damage and disease progression. This is supported by the data from our two Phase I pharmacokinetic studies of ALZT-OP1 in normal and AD subjects which concluded in 2016.
Our current Phase III trial, a randomized, placebo-controlled pivotal study with 600 subjects in 80 sites in Europe, Canada and the U.S., is designed to evaluate the safety and tolerability of ALZT-OP1 and to assess the impact of the ALZT-OP1 combination therapy on cognitive and functional declines in patients with mild cognitive impairment, a precursor of early Alzheimer’s disease. Our Phase III trial the Cognite Trial is being conducted under a special protocol assessment agreement with the FDA and the 505(b)(2) accelerated pathway for approval.
Based on the 72 required weeks of required treatment and the estimated trial enrollment, we expect to complete the study in 2018.
ALZT-OP1a treatment for Post Stroke Cognitive Impairment (PSCI). We believe that ALZT-OP1a, one of the two drugs used in our ALZT-OP1 combination therapy, also has promise as an adjuvant treatment for PSCI. We believe that ALZT-OP1a has the potential, by inhibiting mast cell migration and glial activation, to attenuate multiple triggers leading to neurodegeneration and neuronal death and to thereby slow down or possibly prevent post-ischemic stroke cognitive decline, especially when given as early as possible post-ischemic stroke. Our current Phase II trial for ALZT-OP1a for PSCI is a randomized, placebo controlled study with 500 subjects in 60 sites in North America and Europe. We expect to complete this study in the first quarter of 2017.
ALZT-Patch. We are in the process of developing a method of delivering an amyloid-beta plaque inhibitor via a transdermal patch, as a component of a combination therapy similar to our ALZT-OP1 product candidate. We refer to this product under development as our ALZT-Patch. We believe that transdermal delivery may enable more consistent dosing and better drug compliance among early AD patients than delivery by inhalation. The drug to be delivered by the ALZT-Patch will be a new, proprietary formulation of an amyloid-beta plaque inhibitor chosen from a series of pro-drug compounds, derived from the chemical structure of ALZT-OP1a and having better skin penetration. We expect to file an Investigational New Drug Application, or IND, for the ALZT-Patch with the FDA by mid 2017.
ALZT-QoL. Our fourth lead product candidate, ALZT-QoL, is intended to increase neuronal activity to improve cognitive function, mood, and social behavior, leading to enhanced quality of life in those already demonstrating symptoms of Alzheimer’s disease. ALZT-QoL works via the utilization of M1-type muscarinic receptor agonists. ALZT-QoL is progressing in preclinical acute and chronic toxicity studies before an IND filing that we expect will be followed by a Phase I toxicity study for a new molecule. An initial preliminary Phase I clinical trial plan is currently in development with two cohorts planned and approximately 20 subjects with AD. We expect to file an IND with the FDA by late 2017 and to initiate a Phase I trial of ALZT-QoL in mid-2018.
AZHALER-D. We have also developed a novel, single-use, disposable inhaler, the AZHALER-D, that we believe is a significant improvement over current technologies. It is easy to use, requires no maintenance, is inexpensive to produce, and can used for a variety of drugs delivered by inhalation. The main advantages of disposable inhalers were realized by device companies for use in treating Chronic Obstructive Pulmonary Disease, however, technical solutions did not alleviate problems of operation, human experience, and compliance. AZHALER-D overcomes these shortcomings.