Slide 1: Soticlestat (OV935) ENDYMION Update
Soticlestat (OV935) ENDYMION Update September 23, 2019 Exhibit 99.1
Slide 2: Disclaimers and Forward-Looking Statements
Disclaimers and Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate" and similar expressions (as well as other words or expressions refe
Slide 3: Initial Positive Data from Ongoing ENDYMION Open-Label Extension Trial
Initial Positive Data from Ongoing ENDYMION Open-Label Extension Trial Continue to Support Development of Soticlestat in Rare Epilepsies While a small number of patients, open-label ENDYMION data reinforce the seizure reduction demonstrated in our previous 12-week Phase 1b/2a adult DEE (developmenta
Slide 4: Soticlestat is Being Evaluated in a Robust and Comprehensive Clinical Development Program in DEE
Soticlestat is Being Evaluated in a Robust and Comprehensive Clinical Development Program in DEE Chromosome 15q Duplication Syndrome CDKL5 Deficiency Disorder Lennox-Gastaut syndrome Dravet syndrome ARCADE: Phase 2 Open-label pilot trial ~30 patients ELEKTRA: Phase 2 Double-blind, placebo-controlled
Slide 5: Soticlestat is a Highly Selective, First-in-Class CH24H Inhibitor
Epileptogenic insult ↑ Modulation of NMDA Receptor ↑ Modulation of Glial Function Epilepsy ↑ 24HC Cholesterol ↑ Glutamate Release ↑ Inflammation ↑ Tissue Excitability CH24H Mechanism of soticlestat1,2 Soticlestat is a Highly Selective, First-in-Class CH24H Inhibitor in Development to Reduce Excessiv
Slide 6: DEE Encompass Diverse Etiologies with Few or No Approved Treatments
DEE Encompass Diverse Etiologies with Few or No Approved Treatments CDKL5 Deficiency Disorder (CDD) Chromosome 15q Duplication Syndrome (Dup15q) Dravet Syndrome (DS) Lennox-Gastaut Syndrome (LGS) Etiology Mutations in CDKL5 gene on X chromosome Duplication in chromosome 15q11.2-q13.1 region 80% have
Slide 7: Previous Phase 1b/2a Adult DEE Trial Results
Previous Phase 1b/2a Adult DEE Trial Results Soticlestat Was Well Tolerated with Reduced Seizure Frequency Over Time Soticlestat achieved primary endpoint of safety and tolerability as measured by incidence of adverse events (AEs)* The majority of AEs were mild Seizure Frequency 61% reduction in med
Slide 8: Previous Phase 1b/2a Adult DEE Trial: Key Takeaways and Learnings
Previous Phase 1b/2a Adult DEE Trial: Key Takeaways and Learnings Safety Soticlestat showed a favorable safety profile and was generally well tolerated Efficacy Soticlestat progressively reduced seizure frequency over time Two of 11 patients became seizure-free (to Day 92) Soticlestat appears to be
Slide 9: ENDYMION Design
ENDYMION DESIGN An OLE Study to Evaluate Long-Term Safety and Efficacy of Soticlestat Trial Design Prospective, interventional, open-label, multi-site, extension trial Endpoints Aged ≥2 and ≤65 years Has participated in a previous soticlestat trial Soticlestat Treatment (104 weeks) (Up to 2 weeks ti
Slide 10: Today's Data Cut from ENDYMION Consists of Patients Who Completed the 12-Week Phase 1b/2a
Today’s Data Cut from ENDYMION Consists of Patients Who Completed the 12-Week Phase 1b/2a Adult DEE Trial Patients who completed Adult DEE trial All patients experienced an interruption in treatment with soticlestat from end of adult trial to start of ENDYMION ranging 6 weeks – 52 weeks, due to timi
Slide 11: ENDYMION Patient Baseline Demographics
Most patients were on multiple anti-epileptic medications Most common AED (taken by at least 2 patients) were lamotrigine, oxcarbazepine, rufinamide, topiramate, valproate. ENDYMION Patient Baseline Demographics Patient population was not enriched for any specific condition Patients were each diagno
Slide 12: ENDYMION Safety and Tolerability
ENDYMION Safety and Tolerability Majority of AEs Were Mild and Consistent with Phase 1b/2a Adult DEE Study Most common TEAEs, any group; number (%) Patients (n=6) Abdominal pain, upper 1 (16.7) Nausea 1 (16.7) Pyrexia 1 (16.7) Bronchial wall thickening 1 (16.7) Rales 1 (16.7) Number of AEs (%) Patie
Slide 13: ENDYMION Efficacy: Median Seizure Frequency Reduction Increased Over Time
ENDYMION Efficacy Median Seizure Frequency Reduction Increased Over Time Chart shows data from the 6 patients in adult DEE trial who continued in ENDYMION Break between end of Adult DEE trial and ENDYMION (range 6-52 weeks) Baseline seizure frequency of median 11.5 seizures per 28 days in ENDYMION L
Slide 14: Individual Patients' Seizure Frequency in Adult and ENDYMION Studies
Individual Patients’ Seizure Frequency in Adult (last 28 days) and ENDYMION Studies Denotes seizure frequency reduction at last 28-day period of adult DEE study Baseline=2 Baseline=71 Baseline=4 Baseline=3 Baseline=20 Baseline=19
Slide 15: ENDYMION Summary of Findings and Interpretations
ENDYMION Summary of Findings and Interpretations Safety Soticlestat continues to show a favorable safety and tolerability profile No new safety signals were identified with longer term exposure Majority of AEs are mild; no serious adverse events (SAEs) reported Consistent with prior observations in
Slide 16: Soticlestat: Next Steps
Soticlestat: Next Steps ARCADE Five patients have rolled over into ENDYMION ARCADE trial initial data anticipated for Q1 2020 If positive, will engage global regulatory authorities to discuss registration path ELEKTRA Interest in trial has been strong and 9 patients have rolled over into ENDYMION En
Slide 17: Ovid: Building a Leading Company in Neurology
Ovid: Building a Leading Company in Neurology PRODUCT CANDIDATE RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 INDICATION Angelman Syndrome Fragile X NEPTUNE – Ongoing ELARA OLE – Ongoing ROCKET – Ongoing CDKL5 Deficiency Disorder / Dup15q Syndrome Dravet / LGS ARCADE – Ongoing ENDYMION OLE – Ongoing
Slide 18: Ovid Therapeutics would like to thank the patients, families, caregivers, clinicians, and trial staff
Ovid Therapeutics would like to thank the patients, families, caregivers, clinicians, and trial staff for their support and active involvement in the soticlestat clinical development program