INVESTOR PRESENTATION
January 2023
1
Speakers
Julien | Mathieu | Thierry |
VEYS | CHARVERIAT | LAMBERT |
Chief Business | Co-founder, CSO | Chief Financial Officer |
Development Officer | & Deputy CEO |
Graduate in Science from the | Ecole Polytechnique and |
University of Aix-Marseille | Mines Paris-Tech |
MBA (Master of Business | Doctor in Neuroscience and |
Administration) from HEC | Cell Biology from the Pierre |
Paris | and Marie Curie Institute. |
20 years of experience in the | HEC Challenge + program |
healthcare industry |
Business Administration at the University of Birmingham
MBA (Master of Business
Administration) from INSEAD
Chartered Accountant and Certified Public Accountant ICAEW (Institute of Chartered Accountants in England and Wales)
2
Profile and pipeline
THERANEXUS IS A CLINICAL STAGE COMPANY DEVELOPING FIRST-IN-CLASS OR FIRST TO MARKET INNOVATIONS TO TACKLE RARE NEUROLOGICAL DISORDERS
2013 | 2017 | 2019 | 2022 |
Spin off from | Partnership with | Launch of P1/2 | |
Entered the | trial of Batten-1 | ||
Beyond Batten | |||
an academic | Euronext stock | in young adults | |
Disease | |||
lab in Paris | market | with Batten | |
Foundation | |||
area | disease | ||
21 | Paris area |
14 | Lyon |
Batten-1
Juvenile Batten disease (CLN3) P1-2 fully recruited & ongoing
TFEB platform
Lysosomal dysfunction platform
Neuronal hyperexcitability platform
Screening | Lead | Nonclinical | IND | Phase I | Phase II |
selection | PoC |
New candidate in H2 2023
Phase III | NDA |
Entering P3
in H2 2023
3
Juvenile Batten disease (CLN3) in brief
700-1,000 patients in the US1 | Autosomal recessive | No treatment registered |
800 - 1,000 patients in the EU2 | Founding effect localised in the Nordic countries |
Diagnosis in | Loss of visual | Cognitive decline | Motor symptoms |
children | |||
acuity leading to | and epileptic | ||
before the age of | |||
aged 4 to 8 years | to blindness | seizures | 20 |
6-10 years | 12-20 years | Patients die in | |
their 20s |
The absence of CLN3 leads to the accumulation of toxic deposits of glycosphingolipids
and results in neuronal death
Our objective : to market the first treatment for Batten disease by 2026:
- Targeting glycosphingolipids synthesis to change the course of the disease
- with a formulation adapted to the patient population
1: based on US insurance claims (Decision Resources Group)
2: based on Orphanet data
4
Batten-1 in CLN3: a strong molecular rationale
Gangliosides | ||||||||
Gb3Cer | ||||||||
(e.g. GM1) | ||||||||
iGb3Cer | ||||||||
Globo series | ||||||||
↗ | Lysosomal | |||||||
(e.g. Gb3, Gb4) | load | |||||||
Ceramides | GlcCer | LacCer | ||||||
GCS | Lacto & neo | |||||||
lacto series | ||||||||
(e.g. Lc3, Lc4) | ||||||||
Accumulation of | ||||||||
Batten-1 | glycosphingolipids in CLN3 |
Batten-1 (miglustat) is an inhibitor of glucosylceramide synthase (GCS), a key upstream enzyme in the synthesis of most glycosphingolipids
Batten-1, through its direct effect on glycosphingolipid synthesis, blocks neuroinflammation and prevents cell death in Batten disease
NEURONAL APOPTOSIS
AND
NEUROINFLAMMATORY
PROCESS
Inhibition arrow
Lactose
Glucose
5
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Theranexus SA published this content on 05 January 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 05 January 2023 17:37:02 UTC.