Estimated Timelines and Costs

Mouse Models – COMPLETED

• Need 3-4 mouse models with reproducibility
• 1-1.5 yr/ mouse
• $100,000/ mouse
• Characterization: $50,000/mouse

IPS CELLS – COMPLETED

• Need 3-4 cell lines with reproducibility
• Correlation to Mouse lines helpful
• $100,000 to $300,000
• 6-8 months per line

ZEBRAFISH LINE – COMPLETED

• Two models available for drug screening
• $50,000 to $100,000 per line

PROTEIN TARGET IDENTIFICATION – IN PROGRESS

• Proteomics $50,000
• Mass spectrometry $30,000
• 1-2 years

DRUG DISCOVERY, SCREENING AND DEVELOPMENT – IN PROGRESS

• $2.5 million
• 2-3 years

PHASE I (DRUG) CLINICAL TRIAL

• $250,000
• 2 years

PHASE II (PILOT) CLINICAL TRIAL – SOTICLESTAT TRIAL COMPLETE

• $1.8 million
• 1.5 years

PHASE III CLINICAL TRIAL – MARIGOLD TRIAL COMPLETE

• $8 million
• 3 years

CDKL5 CLINICAL CONSORTIUM – IN PROGRESS

• Ongoing
• NIH Clinical Trial Readiness U01 Grant in progress starting 2021
• $450,000 per year

DATABASE – IN PROGRESS

• Ongoing
• Critical for determining Therapeutic Endpoints for Trials
• $75,000