3rd Edition Track Finals: BSim2 poster

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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 Approach The 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-TTR RX Antisense ISIS Pharmaceuticals ALN-TTR 02 siRNA Alnylam Pharmaceuticals asking price of US$150/patient/year + Milestones AT-06,09,12 Small molecule BSIM 2 Carlos J. V. Simões, PharmD Rui M. M. Brito, PhD

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Transcript of 3rd Edition Track Finals: BSim2 poster

Page 1: 3rd Edition Track Finals: BSim2 poster

Financial Highlights

Promotor team

Catarina Maia, MBA

Target Market

A suite of computationalscreening workflows

Partners

Center for Neuroscience andCell Biology

University of Coimbra

Maria João Saraiva, PhDUniversity of Porto

Ernesto Freire, PhDJohns Hopkins University

Sheena Radford, PhDUniversity 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 SolutionsProducts — First in pipeline

New drug candidates againsttransthyretin-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 revenuesin thousands of US Dollars

Our Business ModelLicensing and joint-venture agreementswith strategic partners in Pharma and Biotech industries

Secured and required fundsin 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 vitroPoC

in vivoPoC

Preclinical PhaseI

PhaseII

PhaseIII

Market

VYNDAQEL®

Small moleculePfizer

SOM0226Repositioned SOM Biotech

mds84Small molecule

PentraxinTherapeutics

ISIS-TTRRXAntisense

ISISPharmaceuticals

ALN-TTR02

siRNAAlnylam

Pharmaceuticals

asking price of US$150/patient/year

+ Milestones

AT-06,09,12Small molecule

BSIM2

Carlos J. V. Simões, PharmD

Rui M. M. Brito, PhD