Praxis Precision Medicines, Inc. · NASDAQ:PRAX
Praxis Precision Medicines — Technical Presentation (April 2024)
15 slides from Praxis Precision Medicines's technical presentation, April 2024
Slide 1: Assessing Clinically Meaningful Improvement Associated with Ulixacaltamide Treatment in Essential Tremor on Propranolol
Assessing Clinically Meaningful Improvement Associated with Ulixacaltamide Treatment in Adults with Essential Tremor on Concomitant Propranolol: Findings from Essential1 Richard Able, PhD VP, Global Medical Affairs Lead, Movement Disorders
Slide 2: Disclosure Information
Disclosure Information Richard Able PhD is a current employee of Praxis Precision Medicines and is a Praxis stockholder. CONFIDENTIAL | 2
Slide 3: Essential Tremor (ET) is the Most Common Movement Disorder
Essential Tremor (ET) is the Most Common Movement Disorder Up to 7 million people in the United States Action tremors significantly disrupt daily may have ET1 living for people with ET Almost all ET patients suffer from at least one Hallmark feature is action tremor that primarily comorbid condition
Slide 4: ET Hidden Burden: High Treatment Discontinuation
ET Hidden Burden: High Treatment Discontinuation Propranolol is the only medication approved for ET ~40 years ago and is not widely used 1 million Propranolol Use Over Time patients with ET remain untreated 100% • ~1 million people are diagnosed with ET and % Continuations on treatment, while anothe
Slide 5: Ulixacaltamide is a Selective Small Molecule T-type Calcium Channel Modulator Addressing the Driver of ET
Ulixacaltamide is a Selective Small Molecule T-type Calcium Channel Modulator Addressing the Driver of ET Formulated as a once- Designed to block daily oral medicine that Well-tolerated in clinical T-type calcium channels can be taken studies to date any time of day, with or without food Source:
Slide 6: Our Journey to Develop a New ET Therapy
Our Journey to Develop a New ET Therapy CLINICAL STUDIES Phase 1 Phase 2 Phase 3 Approval • COMPLETE: Essential1 study Now enrolling! • 14-week results available: Essential1 open- label extension study 6 Ulixacaltamide is currently being studied in ET and is not approved by the U.S. Food & Drug Admi
Slide 7: Essential1 Phase 2b Study Evaluating the Efficacy and Safety of Ulixacaltamide for Essential Tremor
Essential1 Phase 2b Study Evaluating the Efficacy and Safety of Ulixacaltamide for Essential Tremor Adults = 18 years or older with moderate to severe ET* • Had ET symptoms for at least 3 years • Willing to maintain stable doses of certain ET 132 participants assigned to ulixacaltamide or placebo me
Slide 8: Essential1 Study Results Snapshot
Essential1 Study Results Snapshot Primary study: Sub-study: Sub-study: Essential1 Open-label extension Randomized withdrawal • Ulixacaltamide showed • Participants taking • Participants saw ADL clinically meaningful ulixacaltamide continued to scores worsen when they improvement benefit after 14 wee
Slide 9: Pursuing Clinically Meaningful Benefit in ET
Pursuing Clinically Meaningful Benefit in ET A modified index of activities of daily living, based on the Tremor Research Group Essential Tremor Rating Assessment Scale (TETRAS), a clinical rating scale for measuring tremor impact.1 Essential1 provided confidence in a modified index of activities of
Slide 10: mADL11: A Modified Activities of Daily Living Scale
Pursuing Clinically mADL11: A Modified Activities of Daily Living Scale Meaningful Benefit in ET 11 Items from the TETRAS-ADL Speaking Dressing Using Keys Hygiene Pouring Working Writing Drinking from a glass Feeding with a spoon Carrying food trays, plates or similar items Overall disability with m
Slide 11: Essential1 Propranolol Post-hoc Analysis Approach
Essential1 Propranolol Post-hoc Analysis We sought to assess clinically meaningful change related to ulixacaltamide treatment in adults with ET able to maintain a stable dose of propranolol for the duration of the study. Approach • mADL11 as a robust measure of clinically meaningful benefit in ET
Slide 12: Adding Ulixacaltamide Benefited More Patients on Propranolol
Adding Ulixacaltamide Benefited More Patients on Propranolol Responder Analysis: % of patients achieving >3-point improvement in mADL11 Week 8 80% 70% Among patients who could 60% tolerate propranolol, adding ulixacaltamide led to ~2-fold 50% increase in the proportion 40% 48% achieving clinically m
Slide 13: Rapid Improvement at Week 14 in Patients Transitioning from Placebo
Rapid Improvement at Week 14 in Patients Transitioning from Placebo Responder Analysis: % of patients achieving >3-point improvement in mADL11 Week 14 80% 70% Patients on propranolol 60% 67% transitioning from placebo to +19% ulixacaltamide have a rapid 50% efficacy response, consistent 50% 48% 40%
Slide 14: Essential1 Phase 2b Set Foundation for Essential3 Phase 3 Program
Essential1 Phase 2b Set Foundation for Essential3 Phase 3 Program Building on findings from Essential1, and focusing on measures determined to be most meaningful to patients with ET, these results highlight the potential for ulixacaltamide to revolutionize the treatment of ET • Strong efficacy signa
Slide 15: Acknowledgements
Acknowledgements • This work is presented on behalf of The Essential1 Study Team • We thank the patients of the Essential1 trial, and our collaborators, clinical sites and investigators Sign up for news and updates: http://eepurl.com/ituodA Connect with us Email us: Booth #1834 clinicaltrials@praxis
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