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
ALZT-OP1a | ALS – Phase 2a Ongoing
ALZT-OP1a | Ischemic Stroke – Phase 2 ready
ALZT-OP3 | Oral mast cell stabilizer – IND expected in 2021
CAR-Treg
Microbiome Exploratory Study

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
  • 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 currently evaluating the safety and efficacy of ALZT-OP1a in a Phase 2a randomized, open-label, multi-center, multi-dose clinical trial in subjects with mild to moderate-stage ALS.
  • Since cromolyn is a mast cell stabilizer with proven anti-inflammatory properties, we are developing our proprietary formulation of inhaled cromolyn sodium (as ALZT-OP1a) for the treatment of PSCI.
  • With an approved IND, we are currently poised to advance ALZT-OP1a directly into Phase 2 clinical development in patients with PSCI.
  • ALZT-OP1a 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.
  • Regulatory T cells (Tregs) maintain homeostasis in the immune system and have been shown to confer protection in models of Alzheimer’s disease, ALS, stroke, Parkinson’s disease, and MS, and also demonstrated the ability to delay the progression of ALS in a previous clinical trial.
  • We are currently advancing our program to engineer Tregs with chimeric antigen receptors (CAR) to direct Tregs to the CNS to restore a healthy balance of inflammatory and regulatory cells in the brain.
  • Further, we are leveraging a proprietary technology to generate allogeneic (non-self), off-the-shelf cell therapies that could reduce the patient burden and production hurdles associated with current therapies.
  • We are currently validating the efficacy of our CAR-Treg platform in several models of neurodegenerative diseases, with the first proof-of-concept read out expected in late 2020.

ALZT-OP3

We are developing ALZT-OP3, a novel, next-generation oral candidate to treat a range of neurodegenerative diseases. We expect to file an investigational new drug (IND) application to begin clinical development of ALZT-OP3 in 2021.

Microbiome Exploratory Study

  • We are currently exploring conducting an observational (non-interventional) study evaluating the microbiome and circulating metabolites in patients with Alzheimer’s disease.
  • The study is expected to enroll 500 patients across 50 centers in North America profiling the microbiome of stool samples and circulating metabolites in plasma of male and female patients aged 55-79 diagnosed with AD to look for any correlation.