Praxis Precision Medicines, Inc. · NASDAQ:PRAX
Praxis Precision Medicines — Investor Presentation (August 2026)
30 slides from Praxis Precision Medicines's investor presentation, August 2026
Slide 1: Corporate Overview
Corporate Overview August 2026
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation may contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to express or implied statements regarding our cu
Slide 3: Praxis' Mission
PRAXIS’ MISSION The needs of patients with CNS disorders are devastatingly urgent. Our mission is to help patients by delivering life-altering treatments faster and more effectively than has ever been done before — and to do it again and again. 3
Slide 4: Praxis: Broad and Deep CNS Pipeline
Praxis: Broad and Deep CNS Pipeline PIPELINE SNAPSHOT PROGRAM Pre Clinical PH 1 PH 2 PH 3 FILING Ulixacaltamide Essential Tremor1 Relutrigine Cerebrum SCN2A- and SCN8A-DEE2 SMALL MOLECULE Broad DEEs PLATFORM Vormatrigine Adjunctive FOS Monotherapy FOS PRAX-020 KCNT1 Elsunersen Early Onset SCN2A3 Sol
Slide 5: CNS Portfolio with >$20B in Peak Sales Potential
CNS Portfolio with >$20B in Peak Sales Potential Four late-stage assets. Three BTDs. Two upcoming PDUFA dates. Cash runway into 2028 Portfolio Peak vormatrigine >$20B elsunersen FOS & Generalized Epilepsy ulixacaltamide Early Onset SCN2A DEE Essential Tremor ~$1B >$4B PDUFA January 2027 relutrigine
Slide 6: Pricing for Recent Approvals Reflects Significant Value to Patients
Pricing for Recent Approvals Reflects Significant Value to Patients Praxis portfolio poised for similar impact • High unmet-need indications with few, if any, effective treatment modalities • Significant price potential within current market analogs Praxis Portfolio ulixacaltamide relutrigine vormat
Slide 7: Movement Disorders: Ulixacaltamide for Essential Tremor
MOVEMENT DISORDERS: Ulixacaltamide for Essential Tremor 7
Slide 8: Essential Tremor: A Major Unmet Need
Essential Tremor: A Major Unmet Need • An estimated 7 million people in the U.S. live with ET • Major functional impacts affecting writing, eating, drinking and social activities • High psychosocial burden (frustration, anxiety, embarrassment, isolation) No ET-specific • Significant proportion recei
Slide 9: Patients Participating in ESSENTIAL3 had Significant Impact to Daily Activities at Baseline
Patients Participating in ESSENTIAL3 had Significant Impact to Daily Activities at Baseline Baseline TETRAS-ADL Disability Profile (Safety Population, n=698) • ESSENTIAL3 included a broad population with high functional disability - average baseline TETRAS-ADL score of ~31 • ESSENTIAL3 patients aver
Slide 10: ESSENTIAL3: Two Positive Phase 3 Studies, PDUFA in January 2027
ESSENTIAL3: Two Positive Phase 3 Studies Supporting Breakthrough Therapy Designation, PDUFA in January 2027 The first successful Phase 3 program for a drug in Essential Tremor Study 1: 12-week Parallel-group Design Study 2: Blinded Stable Responder, Randomized (n=432) Withdrawal Design (n=80) PRIMAR
Slide 11: Rapid, Durable, and Consistent Effects Observed in Both Study 1 and Study 2
Rapid, Durable, and Consistent Effects Observed in Both Study 1 and Study 2 Across Multiple Endpoints % Responders across Patient-Reported (PGI-C) and Clinician- TETRAS-ADL Change from Baseline* Assessed (CGI-S) Outcomes Study 1 Placebo Study 1 Ulixa Study 2 Ulixa Study 1 Placebo Study 1 Ulixa Study
Slide 12: Benefit Maintained Across Increasingly Stringent Response Thresholds
Benefit Maintained Across Increasingly Stringent Response Thresholds in Studies 1 and 2 % Participants Achieving Each ADL Improvement Level at Day 56* >= 12 pts TETRAS-ADL Improvement Threshold >= 10 pts >= 8 pts >= 6 pts >= 4 pts >= 2 pts 0% 20% 40% 60% *Treatment difference: p-value < 0.01 at all
Slide 13: Building Commercial Capabilities for Ulixacaltamide Launch in Early 2027
Building Commercial Capabilities for Unprecedented Ulixacaltamide Launch in Early 2027 Building the team: Commercial leadership in place, Field force build-out on-track for launch Engaging ET physicians with "ESSENTIAL to me” disease awareness campaign launched at the American Academy of Neurology (
Slide 14: Physicians Validate Ulixacaltamide's Profile Across Key Dimensions
Physicians Validate Ulixacaltamide’s Profile Across Key Dimensions Findings from HCP Observational Study Conducted in March 2026 surveyed >2,300 US Physicians representing >43,000 ET Patients* Meaningfulness of endpoint Compelling efficacy in ESSENTIAL3 • Physicians view ADL improvement as highly me
Slide 15: Matching the Needs of Patients with Ulixacaltamide
Matching the Needs of Patients with Ulixacaltamide Patient Survey Overview – Everyday Moments Most Impacted by Essential Tremor Mean Daily Functional Impact: 5 65% 1,294 Had seen a Physician in the last 1 7 Total Patient Responses No impact Extreme impact 12 Months Most Impacted Everyday Moments Wri
Slide 16: Developmental & Epileptic Encephalopathies (DEEs): Relutrigine, Elsunersen
DEVELOPMENTAL & EPILEPTIC ENCEPHALOPATHIES (DEEs): Relutrigine Elsunersen 16
Slide 17: Relutrigine: Potential for Class Leading Efficacy and Tolerability
Relutrigine: Potential for Class Leading Efficacy and Tolerability Precision Mechanism: Superior selectivity for hyperactive NaV channels, a known driver of Relutrigine seizure activity across DEEs Clinical Profile: Small molecule functional state modulator • In EMBOLD study, demonstrated robust se
Slide 18: EMBOLD Study: Disease-Modifying Results in SCN2A/8A DEEs
EMBOLD Study: Disease-Modifying Results in SCN2A/8A DEEs Study stopped early at interim analysis; NDA accepted with PDUFA December 27, 2026 SEIZURE REDUCTION OVER TIME ON RELUTRIGINE MARKED IMPROVEMENT IN DISEASE MODIFYING COHORTS 1 and 2, COHORT 1 OLE DOMAINS • >80% of patients Months of Exposure P
Slide 19: Relutrigine Sodium Channel MOA Targets Phenotypic DEEs
Relutrigine Sodium Channel MOA Targets Phenotypic DEEs with Applicability Beyond SCN2A/8A EMBOLD Population Current US DEE market is over 200,000 patients and growing as population ages MOST SEIZURE ETIOLOGIES CONVERGE AT SODIUM • Seizure-activity in DEEs, independent of CHANNELS* etiology, requires
Slide 20: EMERALD Study Targets Phenotypic DEEs, Regardless of Etiology
EMERALD Study Targets Phenotypic DEEs, Regardless of Etiology Topline readout expected Q4 2026 DOUBLE-BLIND TREATMENT PERIOD 16 WEEKS 1:1 Primary Endpoint: Randomization Relutrigine N ~200 1 mg/kg/day Change from baseline in >50 distinct monthly motor seizure OLE frequency genetically defined pathol
Slide 21: Elsunersen: First-in-Class ASO for Early Onset SCN2A DEE
Elsunersen: First-in-Class ASO for Early Onset SCN2A DEE Awarded Breakthrough Therapy Designation in 2026 Mechanistic Precision: ELSUNERSEN • Selective targeting of SCN2A gain-of-function mutations, a key driver of early onset, severe seizure activity • ASO-mediated degradation of SCN2A mRNA reduces
Slide 22: EMBRAVE Part A Topline Results Show Marked Seizure Reduction
EMBRAVE Part A Topline Results Show Marked Seizure Reduction, with Disease-Modifying Benefit AGED 2-12 YEARS OPEN LABEL Elsunersen 1 mg IT every EXTENSION 3:1 4 weeks for 24 weeks Elsunersen Randomization Sham procedure every N=9 4 weeks for 24 weeks Starting dose of 1 mg with optional dose escalati
Slide 23: The Pivotal EMBRAVE3 Trial is Currently Enrolling
The Pivotal EMBRAVE3 Trial is Currently Enrolling EMBRAVE3 - STUDY DESIGN Observation Elsunersen (n=30) Open Label Period 1 mg Q4W for 24 weeks Extension Expected to serve as registrational trial Key Inclusion Criteria • Documented early onset SCN2A variant with seizures prior to 3 months of age for
Slide 24: Common Epilepsy: Vormatrigine
COMMON EPILEPSY: Vormatrigine 24
Slide 25: Vormatrigine: Best-in-Disease Sodium Channel Modulator
Vormatrigine: Best-in-Disease Sodium Channel Modulator Epilepsy is a chronic neurological disorder Vormatrigine poised to rapidly transform the that affects all age groups, causing life- epilepsy landscape threatening seizures Superior Efficacy • Best-in-disease efficacy in the RADIANT study • An es
Slide 26: RADIANT Phase 2 Study Showed Disease-leading Efficacy
RADIANT Phase 2 Study Showed Disease-leading Efficacy DISEASE IMPACTING MEDIAN % REDUCTION IN FOS (Weeks 1-16) RADIANT RESULTS CRITERIA • Rapid response after only 1 week of dosing Speed and • Median reduction maintained at 100% after durability of 10 weeks response • Generalized epilepsy patients h
Slide 27: Vormatrigine ENERGY Program: Developing for Broad, Foundational Use
Vormatrigine ENERGY Program: Developing for Broad, Foundational Use ADDRESSABLE PATH TO ADJUNCTIVE, 3rd LINE+ PATIENTS FOS PATIENTS POWER2 — Ph3 Reinitiating in 4Q26 • Testing vormatrigine at multiple strengths 1 million 3rd line (+) PATH TO MONOTHERAPY, 1st LINE PATIENTS POWER3 — Ph3 Finalizing Des
Slide 28: Long, Multi-layered and Strong IP Position Across the Clinical Portfolio
Long, Multi-layered and Strong IP Position Across the Clinical Portfolio ulixacaltamide relutrigine US Current Exclusivity1 vormatrigine elsunersen 2039 2042 2046 ulixacaltamide US Potential Exclusivity2 relutrigine vormatrigine elsunersen 1. Based on US Patent Nos. 11,649,207; 11,427,540; 12,077,50
Slide 29: Two Platforms Enabling Repeatable CNS Innovation
Two Platforms Enabling Repeatable CNS Innovation Cerebrum Solidus SMALL MOLECULE PLATFORM ANTISENSE OLIGONUCLEOTIDE (ASO) PLATFORM Cerebrum utilizes deep understanding of neuronal excitability and Solidus is an efficient, targeted precision medicine discovery and neuronal networks and applies a seri
Slide 30: Dare for More
SAR AXIS DARE FOR MORE
Praxis Precision Medicines — Investor Presentation (August 2026) on 6ix