Praxis Precision Medicines, Inc. · NASDAQ:PRAX
Praxis Precision Medicines — Q2 2025 Earnings Presentation
23 slides from Praxis Precision Medicines's earnings presentation, Aug 4, 2025
Slide 1: RADIANT topline
RADIANT topline August 4, 2025
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 the cu
Slide 3: Positioned to bring innovation to patients with CNS disorders
Positioned to bring innovation to patients with CNS disorders into 4 5 2 2028 Assets in late Clinical readouts Discovery Cash stage in next 4 quarters platforms to runway optimize drug development 3
Slide 4: Praxis pipeline and upcoming catalysts
Praxis pipeline and upcoming catalysts Program Pre clin Ph 1 Ph 2 Ph 3 Upcoming Catalyst Vormatrigine Focal Onset Seizures & Generalized Epilepsy EMPOWER observational study Ongoing RADIANT Phase 2 Topline results by mid-2025 POWER1 Phase 2/3 Finalize study in Q4 2025 POWER2 Phase 2/3 Initiate in Q3
Slide 5: Focal epilepsy is a serious medical condition with inadequate therapeutic options
Focal epilepsy is a serious medical condition with inadequate therapeutic options impacting approximately 3M patients in the US Epilepsy is a chronic neurological disorder of patients require that affects all age groups, causing life- 63% multiple ASMs1 threatening seizures Patients need a new thera
Slide 6: Vormatrigine is poised to quickly transform the epilepsy landscape
Vormatrigine is poised to quickly transform the epilepsy landscape Superior Ease of Ideal Tolerability Efficacy Administration and Limited DDIs Best-in-disease efficacy in Once daily dose, fast acting No expected restrictions the RADIANT study with co-administration with No need to be taken with foo
Slide 7: RADIANT Best-in-class execution in epilepsy
RADIANT Best-in-class execution in epilepsy Full dataset for focal 37 patients and generalized 99 Patients 61 patients dosed to date** included in today’s expected by Q4 Screened read-out ~75 patients* Foundation of the safety database and Proven recruitment engine -> scalable across the Best-in-dis
Slide 8: RADIANT patients represent the real-world refractory group
RADIANT patients represent the real-world refractory group Observation 30 mg vormatrigine QD Safety Follow-up baseline 8 weeks N=37 Age (Years) Mean (SD) 43.1 (12.78) Sex M,F 14,23 # background ASMs Mean (SD) 2.2 (0.81) Sodium Channel Blocker 81% SV2A 65% Concomitant ASM GABA modulators 30% Others 5
Slide 9: Vormatrigine effect in RADIANT: Best-in-Disease Efficacy
Vormatrigine effect in RADIANT: Best-in-Disease Efficacy MEDIAN % SEIZURE REDUCTION BY WEEK PATIENTS WITH ≥50% REDUCTION 56.3% OVERALL MEDIAN SEIZURE REDUCTION Wilcoxon sign test p<0.05 in all weeks and overall; LOESS: Locally weighted plot smoothing 9
Slide 10: Rapid, durable and expanding response from beginning to end of study
Rapid, durable and expanding response from beginning to end of study PROPORTION OF PATIENTS ACHIEVING ≥50% REDUCTION PATIENTS WITH 100% REDUCTION 10
Slide 11: Significant response independent of baseline seizure burden
Significant response independent of baseline seizure burden PROPORTION OF PATIENTS ACHIEVING ≥ 50% REDUCTION Seizure cut defined by baseline median 11
Slide 12: Most challenging ASM background to demonstrate effect in any focal epilepsy study
Most challenging ASM background to demonstrate effect in any focal epilepsy study ASM CATEGORY BY % OF UNIQUE PATIENTS 30% of patients on cenobamate SV2a: Synthetic vesicle protein 2A 12
Slide 13: Response rate maintained over best current treatment
Response rate maintained over best current treatment PROPORTION OF PATIENTS ACHIEVING ≥ 50% REDUCTION ON TOP OF MAJOR BACKGROUND ASM 13
Slide 14: Vormatrigine was generally well tolerated, with no new safety signals
Vormatrigine was generally well tolerated, with no new safety signals SAFETY POPULATION VORMATRIGINE 30 MG • Lowest rate of TEAEs and CNS N=61 AEs with modern ASMs** Patients with ≥1 TEAEs 36 (59.0%) • Most AEs were mild to moderate and transient Patients with severe AEs 3 (4.9%) Serious AEs 3 (4.9%
Slide 15: Learnings from RADIANT to inform POWER2
Learnings from RADIANT to inform POWER2 • Preliminary dose response modeling supports potential for greater effect with 40 mg vormatrigine • Reducing background ASMs is an effective strategy to manage AEs without adversely affecting seizure reduction • Including depression/mood endpoint based on rep
Slide 16: POWER2 staged to initiate this quarter, complete enrollment in 2H 2026
POWER2 staged to initiate this quarter, complete enrollment in 2H 2026 POWER2, n= ~400 12 weeks, QD 20 mg Safety Follow-up 30 mg Observation period or 40 mg Open-label Extension Placebo 16
Slide 17: Majority of focal epilepsy patients quickly progress to multiple ASM use
Majority of focal epilepsy patients quickly progress to multiple ASM use by trial and error AVG. DAYS ON FOS DX - PATIENTS BY LINE OF THERAPY THERAPY, BY LINE 295 Days 168 Days Improvised layering 155 Days of drugs does not result in better patient outcome 129 Days 114 Days Simpler and more rational
Slide 18: POWER3 designed to demonstrate the potential of vormatrigine as a stand-alone agent
POWER3 designed to demonstrate the potential of vormatrigine as a stand-alone agent Safety Follow-up • Screening Monotherapy Vormatrigine or • Observation conversion monotherapy Open-label Extension Key study aspects: • Refractory epilepsy with 1-2 current ASMs • Initiate vormatrigine while titratin
Slide 19: Vormatrigine leads the way to be the best-in-disease ASM
Vormatrigine leads the way to be the best-in-disease ASM • Rapid and compelling seizure reduction overall and in subgroup analyses • Favorable safety and tolerability profile compared to ASMs currently in the market or in development* • Ideal dosing profile: once-daily, no titration, taken with or w
Slide 20: Praxis is revolutionizing how epilepsy is treated
Praxis is revolutionizing how epilepsy is treated SPECTRUM OF EPILEPSY Common (3M+ patients) DEE-driven rare (200k+ patients) Vormatrigine Relutrigine - Best in class potential ASM for common - BTD in SCN2A and 8A DEEs epilepsy patients - EMBOLD study enrolling SCN2A and 8A DEEs - Pursuing standalon
Slide 21: DARE GOR MORE
DARE GOR MORE
Slide 22: Vormatrigine development program to demonstrate efficacy in focal and generalized epilepsy
Vormatrigine development program to demonstrate efficacy and bring an improved therapy to focal and generalized epilepsy patients OBJECTIVE 1H 2024 2H 2024 1H 2025 2H 2025 1H 2026 POC in epilepsy patients PPR Registry to help epilepsy patients track their seizures EMPOWER Evaluate efficacy, safety a
Slide 23: Vormatrigine safety profile positioned to be best-in-disease ASM
Vormatrigine safety profile positioned to be best-in-disease ASM Vormatrigine 30 mg Cenobamate 400 mg XEN1101 25 mg (N = 61) (N = 111) (N = 114) Study RADIANT Study C0171 X-TOLE2 Discontinuation 14 (23 %) 30 (27%) 26 (23,%) Patients with ≥ 1 TEAE 36 (59 %) 100 (90 %) 97 (85 %) Patients with severe A
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