COMPANY SUMMARY BIO Boston 1x1 meeting LYSOGENE ___________ NAME - - PDF document

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COMPANY SUMMARY BIO Boston 1x1 meeting LYSOGENE ___________ NAME - - PDF document

COMPANY SUMMARY BIO Boston 1x1 meeting LYSOGENE ___________ NAME OF THE CEO MISSION Karen AIACH Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a


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COMPANY SUMMARY BIO Boston 1x1 meeting

LYSOGENE ___________ NAME OF THE CEO Karen AIACH ADRESS 18/20 rue Jacques Dulud, 92200 Neuilly-sur-Seine _____________________ EMAIL karen.aiach@lysogene.com www.lysogene.com MANAGEMENT TEAM CEO: Karen Aiach COO: Philippe Mendels-Flandre CSO: Ralph Laufer CTO: Mark Plavsic CMO: Sophie Olivier VP of Regulation and Quality Affairs: Sean O’Bryan TARGETED MARKET ____________ Orphan CNS disease (MPS IIIA, GM1 Gangliosidosis and Fragile X) and looking for expanding the portfolio. LISTED COMPANY CREATION DATE 2009

MISSION

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and

  • ther genetic disorders of the CNS. A pivotal clinical trial in MPS IIIA is expected to

start in 2018, a phase 1-2 clinical trial in GM1 Gangliosidosis in 2019, while we are currently collaborating to define the clinical development path for the treatment of Fragile X syndrome.

TECHNOLOGY

Lysogene is a gene therapy company using AAV vectors and addresses monogenic diseases.

COMPETITION

Lysogene is the most advanced company in discovering gene therapies for MPS III A, GM1 ganliosidosis and Fragile X; competition in MPS IIIA is including ABEONA (US) and ESTEVE (Private Spain). There is no identified competition other than publicly -funded research for GM1-Gangliosidosis and gene therapy in Fragile X.

ALLIANCES/PARTNERSHIPS

University of Massachusetts Medical School Auburn University SAHMRI (South Australian Health and Medical Research Institute) University of Strasbourg, SATT Conectus

UPCOMING CATALYSTS

  • Start of pivotal trial in MPS IIIA : H2 2018
  • Start of phase 1/2 for LYS-GM101: 2019
  • Phase 3 results for MPS IIIA : 2020
  • Proof of concept for Fragile X : early 2020

MARKET FIGURES (listed companies)

Revenues 2017: 0 Date of IPO: February 2017 Ticker: EPA.LYS (ISIN: FR0013233475) Exchange: Euronext Currency: Euro Market cap: 25 millions € Price: 2,08 52-weeks-high: 5,94 € 52-weeks-low: 1,98 € Average daily volume: 4536

KEY FIGURES

€ m 2016 2017 Growth (%) Sales EBIT

  • Net Income

(7,5) (17,8)

  • Cash Position

6,3 14,1

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COMPANY SUMMARY BIO Boston 1x1 meeting ANALYST COVERAGE

Broker Date Reco. (buy/neutral/h

  • ld)

Target Price Potential (%) Société Générale 24/06/2017 BUY 7 336.54% Gilbert Dupont 13/04/2018 BUY 6 288.46% Average:

  • 6,5

312.5%

SHAREHOLDERS (percentage)

Sofinnova Partners: 29.41% BPI France: 20.62% Novo: 17.45% KGA: 10.37% Others: 22.15%

PIPELINE

LYSOGENE ___________ NAME OF THE CEO Karen AIACH ADRESS 18/20 rue Jacques Dulud, 92200 Neuilly-sur-Seine _____________________ EMAIL karen.aiach@lysogene.com www.lysogene.com MANAGEMENT TEAM CEO: Karen Aiach COO: Philippe Mendels-Flandre CSO: Ralph Laufer CTO: Mark Plavsic CMO: Sophie Olivier VP of Regulation and Quality Affairs: Sean O’Bryan TARGETED MARKET ____________ Rare CNS disease (MPS IIIA, GM1 Gangliosidosis and Fragile X) and looking for expanding the portfolio. LISTED COMPANY CREATION DATE 2009