Pipeline

ADVANCING A BROAD PIPELINE TARGETING NEURODEGENERATIVE DISEASE

Our robust product pipeline leverages our scientific insights, and, we believe, has the potential to treat a broad array of serious diseases resulting from unchecked neuroinflammation.

  • DISCOVERY
  • PRECLINICAL
  • PHASE 1
  • PHASE 2
  • PHASE 3
DISCOVERY
PRECLINICAL
PHASE 1
PHASE 2
PHASE 3
ALZT-OP1 | Early Alzheimer’s Disease – Phase 3 fully enrolled
AZT-101 | Ischemic Stroke – Phase 2 ready
AZT-101 | ALS – IND expected in 1H 2020
AZT-211 | Oral mast cell stabilizer – IND expected in 2020
CAR-Treg
AZT-301 | Oral Parkinson’s pro-drug
AZHALER Device | Broad potential use

ALZT-OP1

A first-in-class candidate to slow the progression of Alzheimer’s disease

  • Unique multi-modal approach that includes attacking the neuroinflammation that leads to neuronal death by shifting the brain’s microglial immune cells to their neuroprotective state.
  • Proprietary, combination treatment consisting of an optimized formulation of cromolyn for inhalation plus oral ibuprofen, both re-engineered to provide a daily dose to potentially suppress the brain’s neuroinflammatory response.
  • Designed to be a convenient and easy-to-use pulmonary and oral delivery kit for once-daily home use.
  • Phase 3, global, randomized, placebo-controlled clinical trial (COGNITE) in patients with early Alzheimer’s disease:
    • Now fully enrolled with more than 600 patients; completion expected in late 2020
    • Unique trial design with narrow patient age spread and homogeneity of stage of patient disease (as measured by the Clinical Dementia Rating (CDR) scale and a key biomarker level)
  • Clear regulatory pathway using validated rating scales of dementia:
    • Conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA)
    • Qualifies for a 505(b)2 streamlined FDA approval process

AZT-101 FOR PSCI

For post-ischemic stroke cognitive impairment (PSCI)

  • Since cromolyn is a mast cell stabilizer with proven anti-inflammatory properties, we are developing our proprietary formulation of inhaled cromolyn sodium (as AZT-101) for the treatment of PSCI.
  • With an approved IND, we are currently poised to advance AZT-101 directly into Phase 2 clinical development in patients with PSCI.
  • AZT-101 is designed to slow or arrest cognitive impairment by:
    • Inhibiting mast cell migration across the blood brain barrier following stroke,
    • Inhibiting cytokine production, and
    • Effectively interrupting the ischemic inflammatory cascade.

AZT-101 FOR ALS

For amyotrophic lateral sclerosis (ALS)

  • Evidence suggests that neuroinflammatory processes are implicated in the initiation and progression of ALS.
  • In a pre-clinical ALS (SOD1) model, we have demonstrated that our proprietary formulation of cromolyn sodium:
    • Delayed disease onset and progress,
    • Reduced motor deficits, and
    • Significantly spared lumbar spinal cord motor neurons and reduced pro-inflammatory cytokine/chemokine levels in the spinal cord and plasma.
  • We are developing AZT-101 for the treatment of ALS and are currently planning to file for approval to begin clinical development.

OTHER PROGRAMS

We are also developing a number of novel and next-generation oral candidates to treat a range of neurodegenerative diseases, as well as a unique inhalation device resulting from our work on our current clinical candidates.

In addition, through our acquisition of Smith Therapeutics, we have added a pre-clinical program exploring the use of T regulatory cells engineered with Chimeric Antigen Receptors (CARs) to restore a healthy balance of inflammatory and regulatory cells in the brain. We believe these CAR-Tregs could have broad therapeutic application across common and rare neurodegenerative diseases.