Financial Highlights for Neurodegenerative Disease Drug Pipeline
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The document summarizes a biotech startup company developing new drug candidates to treat transthyretin-related diseases like familial amyloid polyneuropathy (FAP). Their lead products target FAP, familial amyloid cardiomyopathy (FAC), and senile systemic amyloidosis (SSA). The company takes a computational screening approach to identify drug candidates with higher specificity and efficacy but lower toxicity than existing options. Their business model involves licensing and partnership agreements with pharmaceutical companies. They are seeking $2.8 million in investment and expect a net present value of $6.9 million with a payback period of 2.1 years.
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Financial Highlights for Neurodegenerative Disease Drug Pipeline
- 1. Financial Highlights Promotor team Catarina Maia, MBA Target Market A suite of computational screening workflows Partners Center for Neuroscience and Cell Biology University of Coimbra Maria João Saraiva, PhD University of Porto Ernesto Freire, PhD Johns Hopkins University Sheena Radford, PhD University of Leeds Our ApproachThe Problem - Drug R&D: high costs, long times - Dry out of the drug discovery pipelines - Ageing of World population - Increasing incidence of neurodegenerative diseases Our Solutions Products — First in pipeline New drug candidates against transthyretin-related diseases: - Familial Amyloid Polyneuropathy (FAP) - Familial Amyloid Cardiomyopathy (FAC) - Senile Systemic Amyloidosis (SSA) Our Advantages - Higher specificity / efficacy - Lower toxicity and side-effects - Lower risks and costs Surfing chemical space to hit biological targets Anti-FAP products up to US$1.2 billion/year* Estimated costs and revenues in thousands of US Dollars Our Business Model Licensing and joint-venture agreements with strategic partners in Pharma and Biotech industries Secured and required funds in thousands of US Dollars (2012-2016) Competition *Based on the number of FAP patients and the asking price for VYNDAQEL® Investment sought: US$2.8M NPV: US$6.9M Payback: 2.1 years Compound Company Discovery in vitro PoC in vivo PoC Preclinical Phase I Phase II Phase III Market VYNDAQEL® Small molecule Pfizer SOM0226 Repositioned SOM Biotech mds84 Small molecule Pentraxin Therapeutics ISIS-TTRRX Antisense ISIS Pharmaceuticals ALN-TTR02 siRNA Alnylam Pharmaceuticals asking price of US$150/patient/year + Milestones AT-06,09,12 Small molecule BSIM2 Carlos J. V. Simões, PharmD Rui M. M. Brito, PhD