Praxis Precision Medicines, Inc. · NASDAQ:PRAX
Praxis Precision Medicines — Investor Presentation (August 2026)
30 slides from Praxis Precision Medicines's investor presentation, Aug 6, 2026
Slide 1: Corporate Overview August 2026
Corporate Overview August 2026
Slide 2: Forward-looking statements
2 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 current beliefs, expectatio
Slide 3: Praxis' Mission
3 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. PRAXIS’ MISSION
Slide 4: Pipeline Snapshot
4 FILINGPH 3PH 2PH 1Pre ClinicalPROGRAM Ulixacaltamide Cerebrum SMALL MOLECULE PLATFORM Essential Tremor1 Relutrigine SCN2A- and SCN8A-DEE2 Broad DEEs Vormatrigine Adjunctive FOS Monotherapy FOS PRAX-020 KCNT1 Elsunersen Solidus ASO PLATFORM Early Onset SCN2A3 PRAX-080 PCDH19 PRAX-090 SYNGAP1 PRAX-1
Slide 5: CNS Portfolio with >$20B in Peak Sales Potential
5 CNS Portfolio with >$20B in Peak Sales Potential Four late-stage assets. Three BTDs. Two upcoming PDUFA dates. Cash runway into 2028 ulixacaltamide Essential Tremor PDUFA January 2027 relutrigine SCN2A/8A: PDUFA December 2026, Broad DEEs vormatrigine FOS & Generalized Epilepsyelsunersen Early Onse
Slide 6: Pricing for Recent Approvals Reflects Significant Value to Patients
6 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 SKYCLARYS® omaveloxolone EVRYSDI® risdiplam SEPH
Slide 7: Movement Disorders: Ulixacaltamide for Essential Tremor
7 MOVEMENT DISORDERS: Ulixacaltamide for Essential Tremor
Slide 8: Essential Tremor: A Major Unmet Need
8 • 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) • Significant proportion receive no or inadequate treatment Essential Tremor: A
Slide 9: Patients Participating in ESSENTIAL3 had Significant Impact to Daily Activities at Baseline
9 Patients Participating in ESSENTIAL3 had Significant Impact to Daily Activities at Baseline Farmer et al. AAN 2026 Plenary Presentation TETRAS-ADL: The Essential Tremor Rating Assessment Scale – Activities of Daily Living subscale Baseline TETRAS-ADL Disability Profile (Safety Population, n=698) •
Slide 10: ESSENTIAL3: Two Positive Phase 3 Studies
10 PRIMARY ENDPOINT % MAINTAIN RESPONSE 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 -4.3 -1.7 p= <0.0001 ULIXACALTAMIDE (n=199) PLACEBO (n=233) 55% 33% p=0.037 ULIXACAL
Slide 11: Rapid, Durable, and Consistent Effects Observed in Both Study 1 and Study 2
11 Rapid, Durable, and Consistent Effects Observed in Both Study 1 and Study 2 Across Multiple Endpoints Intention-to-treat population *LS Means; (+/- 95% CI) Shtilbans et al. AAN 2026 Poster Presentation PGI-C: Patient Global Impression of Change; CGI-S: Clinical Global Impression of Severity 0 -2
Slide 12: Benefit Maintained Across Increasingly Stringent Response Thresholds
12 Benefit Maintained Across Increasingly Stringent Response Thresholds in Studies 1 and 2 *Treatment difference: p-value < 0.01 at all responder thresholds (logistic regression) Intention-to-treat population for the combined Study 1 and 2 populations for ulixacaltamide patients Shtilbans et al. AAN
Slide 13: Building Commercial Capabilities for Ulixacaltamide Launch in Early 2027
13 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 (ANN) conference Building medical community awareness about the profile of ulixacalta
Slide 14: Physicians Validate Ulixacaltamide's Profile Across Key Dimensions
14 Physicians Validate Ulixacaltamide’s Profile Across Key Dimensions Findings from HCP Observational Study Conducted in March 2026 Results reinforce peak potential of >$10B in the US for ulixacaltamide surveyed >2,300 US Physicians representing >43,000 ET Patients* Meaningfulness of endpoint • Phys
Slide 15: Patient Survey Overview – Everyday Moments Most Impacted by Essential Tremor
15 65% Had seen a Physician in the last 12 Months 1,294 Total Patient Responses Matching the Needs of Patients with Ulixacaltamide Patient Survey Overview – Everyday Moments Most Impacted by Essential Tremor 210 (16%) 289 (22%) 416 (32%) 481 (37%) 601 (46%) 792 (61%) 946 (73%) 988 (76%) 1114 (86%) 0
Slide 16: Developmental & Epileptic Encephalopathies (DEEs): Relutrigine, Elsunersen
16 DEVELOPMENTAL & EPILEPTIC ENCEPHALOPATHIES (DEEs): Relutrigine Elsunersen
Slide 17: Relutrigine: Potential for Class Leading Efficacy and Tolerability
17 Relutrigine: Potential for Class Leading Efficacy and Tolerability AE: adverse event, DEE: developmental & epileptic encephalopathy, NaV: voltage-gated sodium channel, SAE: serious adverse event Relutrigine Small molecule functional state modulator No titration required Once daily dosing Liquid f
Slide 18: EMBOLD Study: Disease-Modifying Results in SCN2A/8A DEEs
18 OLE: open label extension Kamireddy et al AES 2025 EMBOLD Study: Disease-Modifying Results in SCN2A/8A DEEs Study stopped early at interim analysis; NDA accepted with PDUFA December 27, 2026 • >80% of patients were on stable doses of sodium channel blockers at baseline • AEs were mostly mild to m
Slide 19: Relutrigine Sodium Channel MOA Targets Phenotypic DEEs
19 • Seizure-activity in DEEs, independent of etiology, requires participation of sodium channels • Relutrigine’s mechanism of action targets hyperactive NaV channels addressing the neuronal hyperexcitability driving seizures • By targeting a common pathway implicated in DEE symptomology, relutrigin
Slide 20: EMERALD Study Targets Phenotypic DEEs, Regardless of Etiology
20 EMERALD Study Targets Phenotypic DEEs, Regardless of Etiology Topline readout expected Q4 2026 Key Inclusion Criteria • Ages ≥2 and ≤65 years • Has a documented diagnosis of a developmental and epileptic encephalopathy in childhood • Has 4 or more countable motor seizures during the 28-day observ
Slide 21: Elsunersen: First-in-Class ASO for Early Onset SCN2A DEE
21 Mechanistic Precision: • Selective targeting of SCN2A gain-of-function mutations, a key driver of early onset, severe seizure activity • ASO-mediated degradation of SCN2A mRNA reduces NaV1.2 hyperactivity, normalizing neuronal excitability Clinical Profile: • Significant reduction in seizures ach
Slide 22: EMBRAVE Part A Topline Results Show Marked Seizure Reduction
22 EMBRAVE Part A Topline Results Show Marked Seizure Reduction, with Disease-Modifying Benefit ClinicalTrials.gov Identifier: NCT05737784 Praxis data on file Elsunersen 3:1 Randomization OPEN LABEL EXTENSIONElsunersen 1 mg IT every 4 weeks for 24 weeks Sham procedure every 4 weeks for 24 weeks AGED
Slide 23: The Pivotal EMBRAVE3 Trial is Currently Enrolling
23 The Pivotal EMBRAVE3 Trial is Currently Enrolling ClinicalTrials.gov Identifier: NCT07019922 EMBRAVE3 - STUDY DESIGN Key Inclusion Criteria • Documented early onset SCN2A variant with seizures prior to 3 months of age • Between the ages of 0 to ≤18 years at Screening (ages 2-18 go to Cohort 1, 1-
Slide 24: Common Epilepsy: Vormatrigine
24 COMMON EPILEPSY: Vormatrigine
Slide 25: Vormatrigine: Best-in-Disease Sodium Channel Modulator
25 Kahlig et al AAN 2023; Patel et al AAN 2024; Hansen et al IEC 2025; Hansen et al AES 2025 1Praxis Claims Analysis on File 2024. FOS patient cohort (n = 440k); Gazdag et al AES 2026 ASM: Anti-seizure medication • An estimated 3 million patients live with epilepsy • ~35% of patients change medicati
Slide 26: RADIANT Phase Study Showed Disease-leading Efficacy
26 MEDIAN % REDUCTION IN FOS (Weeks 1-16) RADIANT Phase 2 Study Showed Disease-leading Efficacy Hansen et al AES 2025 RADIANT RESULTS DISEASE IMPACTING CRITERIA • Rapid response after only 1 week of dosing • Median reduction maintained at 100% after 10 weeks • Generalized epilepsy patients had simil
Slide 27: Vormatrigine ENERGY Program: Developing for Broad, Foundational Use
27 Vormatrigine ENERGY Program: Developing for Broad, Foundational Use POWER2 — Ph3 Reinitiating in 4Q26 • Testing vormatrigine at multiple strengths POWER3 — Ph3 Finalizing Design to initiate in 4Q26 • Single-agent, first-line use ADDRESSABLE FOS PATIENTSPATH TO ADJUNCTIVE, 3rd LINE+ PATIENTS PATH
Slide 28: Long, Multi-layered and Strong IP Position Across the Clinical Portfolio
28 Long, Multi-layered and Strong IP Position Across the Clinical Portfolio 2039 2042 2046 relutrigine ulixacaltamide vormatrigine US Current Exclusivity1 US Potential Exclusivity2 ulixacaltamide relutrigine vormatrigine elsunersen elsunersen 1. Based on US Patent Nos. 11,649,207; 11,427,540; 12,077
Slide 29: Two Platforms Enabling Repeatable CNS Innovation
29 Two Platforms Enabling Repeatable CNS Innovation 1 Ulixacaltamide has received Breakthrough Therapy Designation (BTD) 2 Relutrigine has received BTD, Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation from the FDA, and ODD from the European Medicines Agency (EMA) for the t
Slide 30: Dare or More
DARE -oR MORE
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