In phase 1/2

• Approved by the FDA for treatment of epileptic seizures
• Restricts Calcium ion inflow into cells

In phase 1/2

• It is the main treatment for Parkinson’s disease and might have an effect in ALS
• In Parkinson’s disease patients, Carbidopa levodopa markedly improves patients’ rigidity

In phase 1/2

• Antiretroviral therapy for Human Endogenous Retrovirus-K (HERV-K) Suppression
• Some ALS patients have a high serum level of HERV-K; reason is unknown
• Pilot study: drug was well tolerated

Enrolling in Phase 1 trial

• AP-101 is a human monoclonal antibody that reduces misfolded superoxide dismutase-1 (SOD1)

In phase 1

• A type of white blood cells that prevent unnecessary inflammation once an infection has been cleared
• ALS patients have lower levels of Tregs
• Rate of disease progression is faster in aggressive ALS
• Increasing the number of Tregs in the blood slowed disease progression in ALS mice

In phase 1

• CNS10-NPC-GDNF are stem cells called human neural progenitor cell (hNPC)
• Genetically engineered to produce GDNF
• Rat studies showed
  • The transplanted cells produced human GDNF and the rat neurons were taking up the GDNF released by the hNPCs
  • vhNPCs also differentiated into astrocytes
  • After seven and a half months, the transplanted hNPCs were still intact in the spinal cord

Completed Phase 1/2 trial

• VM202 delivers a gene that produces the HGF protein
• HGF triggers blood vessel formation and nerve growth
• In ALS, HGF acts directly on motor neuron cells & regenerates muscles
• Safe and well-tolerated

In phase 1

• APB-102 uses viral vector to silence SOD-1 gene
• Faulty SOD-1 production is reduced

PHASE 1– Trametinib, Tamoxifen, EPO, Perampanel, Sinemet, TAF, AP-101, Tregs, CNS10-NPC-GDNF, VM202, APB-102