ProgramResearchLead OptimizationPreclinicalIND EnablingINDPhase 1Phase 2
Neuroinflammation: SLC22A8 inhibitor
       
NPT520-34: Parkinson's Disease
 
 
   
               
NPT520-34: Amyotrophic Lateral Sclerosis (Orphan Drug Designation by FDA)
 
 
   
               
Neuroinflammation: TLR2 Antagonists (multiple chemical series)
     
NPT1220-312: Amyotrophic Lateral Sclerosis
 
 
 
       
               
NPT1220-312: Parkinson's Disease
 
 
 
       
               
Backups & 2nd Generation
 
 
 
       
               
Autophagy
             
NPT520-337: PI3K inhibitor (MA)
 
 
 
 
       
               
Cathepsin A inhibitors (CMA)
 
 
 
 
         
               
New Targets (MA/CMA/MIT)
 
 
 
 
         
               
New Targets   
 
 
 
 
         
               
α-synuclein Oligomerization Inhibitor
         
UCB0599: Parkinson's Disease
ucb logo
 
 
 
 
 
               

MA = macroautophagy;
CMA = chaperone-mediated autophagy;
MIT = mitophagy

Following up upon the success of its first clinical candidate (UCB-0599) under collaborative development with UCB Pharma for Parkinson’s disease Neuropore Therapies is currently advancing two exciting novel therapeutic candidates (NPT520-34 and NPT1220-312) as disease-modifying therapeutics for the treatment of neurodegenerative disorders.

NPT520-34 is clinical stage orally bioavailable small molecule that reduces astrocytic and microglial markers of neuroinflammation with robust beneficial effects on neuropathology and motor function in animal models of Parkinson’s disease. NPT520-34 also reduces the expression of markers of neuroinflammation and neuropathology in animal models of amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease. NPT520-34 is currently being evaluated for safety, pharmacokinetics and target engagement in Phase 1 clinical studies in healthy volunteers. Biomarker-based proof-of-mechanism studies in patients with neurodegenerative disorders are scheduled to start in 2020.

NPT1220-312 is a potent and selective Toll-like receptor 2 (TLR2) antagonist that reduces markers of inflammation and neurotoxic protein burden in multiple cell systems including Parkinson’s disease patient-derived iPS neurons. With promising preclinical safety and pharmacokinetic profiles and potent target engagement in vivo already demonstrated, it is anticipated that NPT1220-312 will enter clinical development in 2020. Initial safety studies in healthy volunteers will include blood markers of target engagement, while subsequent studies in patients will incorporate brain imaging and perhaps peripheral biochemical markers of inflammation. Importantly, NPT1220-312 is just the first of multiple structurally diverse and promising small molecule TLR2 antagonists being developed for applications in a range of central neurodegenerative and peripheral inflammatory disorders. 

In addition to its anti-aggregation and neuroinflammatory therapeutic franchises, Neuropore Therapies has developed an integrated autophagy platform comprised of high throughput screening assays of different forms of autophagy (macroautophagy, chaperone-mediated autophagy and mitophagy). The NPT Autophagy Platform also includes biochemical assays to conduct hit confirmation as well as animal models of target engagement and efficacy.  Compounded with Neuropore’s agile medicinal chemistry capabilities, the NPT Autophagy Platform is fine-tuned to efficiently identify novel autophagy targets and chemistries for small molecule drug-discovery.