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Therapeutic Targeting of Alzheimer's Disease with a Selective Antibody
- 1. Therapeutic Targeting of Alzheimer's Disease with a Selective Antibody August 28, 2020
- 2. What is the problem? Desperate need for Alzheimer’s disease (AD) therapeutics AD Antibody – Passive Immunization Treatment for Alzheimer's Disease • AD is 100% fatal • No approved disease-modifying treatments • Current drugs only treat the symptoms of dementia – Oral regulators of acetylcholine (Aricept) or glutamate (Namenda)
- 3. What is the problem? Protein aggregation is at the core of AD AD Antibody – Passive Immunization Treatment for Alzheimer's Disease • Accumulation of aggregated amyloid-beta protein (Aβ) • Aβ senile plaques, oligomers and protofibrils Brain slice from AD patient Selkoe, Trends Cell Biol, 1998, 8, 447 Bitan et al., PNAS, 2003, 100, 330
- 4. How does product/service solve problem? Our solution is mAbSL antibody • Amyloid-β protein (Aβ) monoclonal antibody • We have obtained, cloned, sequenced, expressed, purified and characterized the monoclonal antibody – selectively targets a protofibril form of Aβ – given to early-stage AD patients – passive immunization treatment for AD Goure et al., Alzheimers Res Ther, 2014, 6, 42 mAbSL + +++ + (aducanumab) AD Antibody – Passive Immunization Treatment for Alzheimer's Disease
- 5. How does product/service solve problem? More on selectivity differences A42, ng/well 0 20 40 60 80 A450nm 0 1 2 protofibrils monomers fibrils ng A42, ng/well 0 20 40 60 80 A450nm 0 1 2 3 protofibrils monomers A B C(BAN2401) selective mAbSL113 non-selective mAbSL513 AD Antibody – Passive Immunization Treatment for Alzheimer's Disease
- 6. What is the market use? How will mAbSL be delivered? • Clinical diagnosis of early stage AD – mild cognitive impairment (MCI) and mild AD • In-patient intravenous delivery of monoclonal antibody solution • Monthly infusions • Measurement of cognitive improvement
- 7. What is the market use? Commercial potential of an AD therapy Early-stage AD (US) 10M MCI/mild cases $100M 1st year sales (10% of market) $1B after 5 years (market & case growth)
- 8. What competition exists? Drug Short-term dementia relief Stops AD progression Reverses AD effects Antibody selectivity Cholinesterase inhibitors Glutamate regulators Inflammation Tau Amyloid (Aβ) Aducanumab BAN2401 mAbSL Current trials Approved & prescribed AD Antibody – Passive Immunization Treatment for Alzheimer's Disease
- 9. What is the status of the intellectual property? • AMYLOID-BETA ANTIBODIES – University of Missouri-St. Louis – Patent application 62/889,220 (Filing date 8/20/2019) – Composition of Matter and Methods claims AD Antibody – Passive Immunization Treatment for Alzheimer's Disease
- 10. What is the stage of development? • Cloned and obtained the variable region DNA sequences for multiple selective and non-selective mAbSL antibodies • Developed methods for expression and purification of mAbSL antibodies AD Antibody – Passive Immunization Treatment for Alzheimer's Disease
- 11. Potential regulatory path for mAbSL • New Drug to pursue expedited Fast Track review • Clinical Trials, 5-7 years, early-stage AD, will seek pharma partnership – Phase 1 (safety, tolerability, immunogenicity, pharmacodynamics, pharmacokinetics), 50-100 participants – Phase 2 (clinical efficacy), 500-1000 participants – Phase 3 (efficacy, long-term safety, tolerability 1000-1500 participants • No approved comparable for AD • Average price for monoclonal antibody treatment is $96,000 (prices for oncology and hematology mAbs much higher) AD Antibody – Passive Immunization Treatment for Alzheimer's Disease
- 12. What is needed for further development? Research Milestones • Further research is needed to strengthen case that monoclonal AbSL antibody (mAbSL) is a potential AD disease-modifier 1. mAbSL production scale-up ($50K) 2. mAbSL effect in AD transgenic mice ($200K) a. diagnostics ability of mAbSL in control-treated AD mice b. prevention of pathology in mAbSL-treated AD mice (young) c. reversal of pathology in mAbSL-treated AD mice (aged) 3. humanize mAbSL antibody ($50K) AD Antibody – Passive Immunization Treatment for Alzheimer's Disease