Praxis Precision Medicines : May 2025 Corporate Presentation

Published: 2025-05-02 10:31:49 ET PRAX

Published on 05/02/2025 at 10:31

®

Positioned to bring innovation to patients with CNS disorders

4

6

2

into

2028

Four pillars guide how we develop medicines

GENETICS

Focus on therapeutic targets identified through human genetics

Translational tools validate potential of target and product candidate and can provide early proof of biology

EFFICIENT & RIGOROUS

Efficient, rigorous clinical development paths to proof-of-concept in humans applying an agile way of working

PfiTIENT-GUIDED

Patient-guided development strategies to deliver on what

patients actually need

Two platforms to generate optimized therapies

CEREBRUM

SMALL MOLECULE PLATFORM

Molecule

ulixacaltamide

Indication

Essential Tremor

Mechanism

T-type calcium channel modulator

vormatrigine

Cerebrum utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies

relutrigine*

Focal Onset Seizures & Generalized Epilepsy

DEE Epilepsies

PRAX-020^

KCNT1 Epilepsy

Sodium channel functional state modulator for broad use

Sodium channel functional state modulator for pediatric use

KCNT1 specific inhibitor

PRAX-050

Not disclosed

Not disclosed

SOLIDUS

ANTISENSE OLIGONUCLEOTIDE (ASO) PLATFORM

Solidus is an efficient, targeted precision medicine discovery and development engine for ASOs anchored on proprietary, computational methodology

Molecule

elsunersen**

Indication

SCN2A GoF

Mechanism

Gapmer ASO

PRAX-080

PCDH19 Mosaic expression

Gapmer ASO

PRAX-090

SYNGAP1 LoF

Splice switching ASO

PRAX-100

SCN2A LoF

Undisclosed mechanism ASO

^PRAX-020 (KCNT1) has been iicensed to UCB

* Relutrigine has received ODD and RPD from the FDA, and ODD from the European Medicines Agency (EMA) for the treatment of SCN2A-DEE and SCN8A-DEE and RPD for Dravet Syndrome 5

** Elsunersen has received ODD and RPD from the FDA, and ODD and PRIME designations from the EMA for the treatment of SCN2A-DEE

Disclaimer

Praxis Precision Medicines Inc. published this content on May 02, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 02, 2025 at 14:21 UTC.