Theranexus : Présentation JPM / Biotech showcase / Sachs Forum

INVESTOR PRESENTATION

January 2023

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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)

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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

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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

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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

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