Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Technical Presentation (June 2020)
41 slides from Ovid Therapeutics's technical presentation, Jun 17, 2020
Slide 1: OV935 (soticlestat) Educational Seminar
June 17, 2020 OV935 (soticlestat) Educational Seminar Exhibit 99.1
Slide 2: Welcome
Jeremy Levin, DPhil, MB, BChir Chairman & Chief Executive Officer Ovid Therapeutics Welcome
Slide 3: 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," "believe," "expect," "plan," "anticipate" and similar expressions (as well as other words or expre
Slide 4: Today's Speakers
Jeremy Levin DPhil, MB BChir Chairman, CEO Amit Rakhit MD, MBA President, CMO, Head of R&D Today’s Speakers Scott Demarest MD Assistant Professor Pediatric Neurology Children’s Hospital Colorado Jason Tardio Chief Commercial Officer Julia Tsai PhD VP, Clinical Development - Epilepsy
Slide 5: Agenda
AGENDA 1 Overview of OV935 2 3 Welcome and Introduction 4 Compelling Market Opportunity 5 Soticlestat in Rare Epilepsies 6 Wrap-up Fireside Chat and Q&A Treating a Patient with DEE Dr. Jeremy Levin Dr. Amit Rakhit Jason Tardio Dr. Scott Demarest Dr. Julia Tsai Dr. Amit Rakhit, Dr. Scott Demarest & D
Slide 6: Our Mission and Value Proposition
Our Mission and Value Proposition
Slide 7: Ovid's Rich Pipeline Focused on Rare Neurological Diseases
Ovid’s Rich Pipeline Focused on Rare Neurological Diseases PRODUCT CANDIDATE RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 INDICATION Angelman Syndrome Fragile X NEPTUNE – Ongoing ELARA OLE – Ongoing ROCKET – Positive Topline Data Announced SKYROCKET – Topline Data Announced CDKL5 Deficiency Disorder
Slide 8: Multiple Clinical Data Readouts Expected Across Late Stage Programs
Multiple Clinical Data Readouts Expected Across Late Stage Programs Phase II ELEKTRA Trial in Dravet Syndrome & Lennox-Gastaut Syndrome: Data Expected in Q3 2020 2 Open-Label Extension ENDYMION Trial in Developmental & Epileptic Encephalopathies: Data Expected in Q3 2020 4 Pivotal Phase III NEPTUNE
Slide 9: Today's Focus – OV935 (soticlestat) Results Will Define Regulatory Pathway
Phase II ELEKTRA Trial in Dravet Syndrome & Lennox-Gastaut Syndrome: Data Expected in Q3 2020 1 Open-Label Extension ENDYMION Trial in Developmental & Epileptic Encephalopathies: Data Expected in Q3 2020 3 Phase II ARCADE Open Label Trial in Dup15q Syndrome & CDKL5 Deficiency Disorder: Data Expected
Slide 10: OV935 (soticlestat) – A Comprehensive Partnership with Takeda
OV935 (soticlestat) – A Comprehensive Partnership with Takeda Partnered with Takeda, signed in 2017 Global 50 / 50 partnership to develop and commercialize OV935 Ovid is responsible for the clinical development in the initial indications (DEE) and commercialization in North America, EU and Israel De
Slide 11: Introduction: OV935 (soticlestat)
Amit Rakhit, M.D., MBA President, Chief Medical Officer & Head of R&D Ovid Therapeutics Introduction: OV935 (soticlestat)
Slide 12: Soticlestat – Unique MOA with Broad DEE Development Program
Soticlestat – Unique MOA with Broad DEE Development Program What we know Looking ahead Novel CH24H enzyme target & unique MOA Strong preclinical data support clinical development Successfully completed 12-week Phase 1b/2a study in adult DEE Longer-term data from ENDYMION (open label extension) sugge
Slide 13: Soticlestat Is a Potent, Selective, Brain-penetrating CH24H Inhibitor
Soticlestat Is a Potent, Selective, Brain-penetrating CH24H Inhibitor In Development To Help Improve Seizure Control Soticlestat has the potential to reduce seizure susceptibility and improve seizure control Reduced 24HC levels cause reduced glutamatergic signaling and modulates glial function, resu
Slide 14: Soticlestat Is Being Evaluated in a Robust and Comprehensive DEE Clinical Development Program
Chromosome 15q Duplication Syndrome CDKL5 Deficiency Disorder Lennox-Gastaut Syndrome Dravet Syndrome ARCADE: Phase 2 Open-label pilot trial 20 patients ELEKTRA: Phase 2 Double-blind, placebo-controlled trial 141 pediatric patients Safety, tolerability, efficacy- seizure frequency Safety, tolerabili
Slide 15: Compelling Market Opportunity
Jason Tardio Chief Commercial Officer Ovid Therapeutics Compelling Market Opportunity
Slide 16: DEEs are Rare Neurodevelopmental Disorders Usually Refractory to Standard AEDs
Developmental and Epileptic Encephalopathies (DEE) are Rare Neurodevelopmental Disorders Usually Refractory to Standard AEDs Lennox-Gastaut syndrome is associated with multiple types of seizures, intellectual disability and behavior problems. Dravet syndrome is a rare, lifelong form of epilepsy that
Slide 17: Disease Burden of DEE on Stakeholders is Significant
Disease Burden of Developmental and Epileptic Encephalopathies on Stakeholders is Significant Ovid Therapeutics: Internal Market Research.
Slide 18: Patient funnel for DEEs
Patient funnel for DEEs 1st line 2nd line 3rd line + Total Estimated Patients (prevalence) Total Estimated Dx and Tx patients Monotherapy Adjunct therapy Switch / Add other adjunct therapy LGS DS CDD Dup15q ~108,000 ~28,000 ~14,000 ~37,000 ~73,000 ~19,000 ~3,000 ~8,000 20% controlled on AED: ~14,600
Slide 19: The DEE Market Represents A Potentially Compelling Commercial Growth Opportunity
The DEE Market Represents A Potentially Compelling Commercial Growth Opportunity as New Treatments Emerge DelveInsight’s Dup15q Syndrome and CDKL5 Deficiency Disorder —Market Insights, Epidemiology, and Market Forecast—2030 report Dravet/LGS/CDKL5/Dup15q Market: US & EU5 (2030) 2030 Forecasted Marke
Slide 20: Treating a Patient With DEE
Scott Demarest, MD Assistant Professor Pediatric Neurology Children’s Hospital Colorado Treating a Patient With DEE
Slide 21: DEE are Rare Neurodevelopmental Disorders With Significant Long-term Impact
Generally characterized by persistent severe seizures with increased risk of sudden unexpected death and concomitant cognitive, developmental, and behavioral issues 1-3 Cause significant impact on both patients and caregivers 2 Symptoms often begin in infancy or early childhood and persist throughou
Slide 22: 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 23: Seizures Associated With DEE Remain Inadequately Controlled Despite Multiple ASMs
Seizures Associated With DEE Remain Inadequately Controlled Despite the Use of Multiple ASMs Conventional ASMs primarily act through 4 mechanisms 4: Inhibition of excitatory ionotropic glutamate receptors (eg, felbamate, perampanel) 1: Modulation of voltage-gated ion channels (eg, carbamazepine, lam
Slide 24: Patient Journey – What is This Like for Families
Patient Journey- What is This Like for Families These are young children – babies or toddlers They start to have seizures or developmental problems Their parents are terrified about what is happening Their doctors are recommending many different types of test – MRIs, EEGs, Genetic testing Their doct
Slide 25: Direct Quote From a CDD Parent
“We didn’t get her on seizure meds until she was about two months old; at which point, we were told by numerous doctors that soon as we get the seizures under control, she’ll progress normally. That was another level of grief. It was a new level of grief because, yeah, we wanted the seizures to stop
Slide 26: This is a Life-long Journey
This is a Life-long Journey New drugs are needed in highly refractory patients that have key attributes: Safety, tolerability, durability of effect in seizure, and potential benefits outside of seizure control Trial and error wit h seizure medications Physical, occupational and speech therapy Adapti
Slide 27: Soticlestat in Rare DEEs
Julia Tsai, PhD VP Clinical Development, Epilepsy Ovid Therapeutics Soticlestat in Rare DEEs
Slide 28: Soticlestat Reduced Seizure Burden and Improved Survival in a Dravet Mouse Model
Soticlestat Significantly Reduced Seizure Burden and Improved Survival an a Dravet syndrome (DS) Mouse Model Fig 1. Seizure-free Rate P<0.0001 Control (n=60) OV935 (n=100) 100 80 60 40 20 0 Percent seizure free Fig 2. Survival Rate P<0.0001 Percent survival 50 40 30 20 10 0 Age (Days) Control (n=60)
Slide 29: Soticlestat Was Evaluated in a 12-week Phase 1b/2a Study in Adult Patients With DEE
Soticlestat Was Evaluated in a 12-week Phase 1b/2a Study in Adult Patients With DEE Part A Double-Blind Tx (30 days) Part B Open-Label Tx (60 days) Follow-up (30 days) Safety and PK assessments Safety, PK, and exploratory assessments Soticlestat BID (n=16) End of Trial Screening/Baseline Randomizati
Slide 30: Phase 1b/2a Trial: Soticlestat Was Well-tolerated and Reduced Seizures
Phase 1b/2a Trial: Soticlestat Was Well-tolerated and Reduced Seizures in Developmental and Epileptic Encephalopathies Phase 1b/2a Patient Diagnosis at Trial Entry LGS (n=6), Dravet (n=1), DEE (n=6), Tuberous sclerosis (n=1), All other (n=4) Safety And Tolerability Soticlestat achieved the study's p
Slide 31: ENDYMION: OLE Study of Long-term Safety and Efficacy – Study Design
ENDYMION: An OLE Study To Evaluate Long-term Safety and Efficacy Of soticlestat — Study Design Trial Design Prospective, interventional, open-label, multi-site, extension study Key Inclusion Criteria Aged 2-65 years Participated in a previous soticlestat study and meet one of the following condition
Slide 32: ENDYMION Safety and Efficacy
ENDYMION Safety and Efficacy - Majority of AEs Were Mild and Consistent with Phase 1b/2a Adult DEE Study Treatment-emergent adverse events (TEAEs) were consistent with those reported in the adult DEE study and the majority were mild) ALL reported TEAEs were: upper abdominal pain, nausea, pyrexia, br
Slide 33: ARCADE: Signal Finding Study in CDD and Dup15q – Study Design
ARCADE: A Signal Finding Phase 2 Study In Patients With CDD and Dup15q — Study Design Follow-up (4 Weeks) End of Trial Screening/Baseline Trial Design Multicenter, open-label, pilot study Titrated: 100 mg BID, 200 mg BID, 300 mg BID (mg/kg dosing <60 kg) oral tablets Key Inclusion Criteria Aged ≥2 a
Slide 34: Summary of Initial Findings From ARCADE
Summary of Initial Findings From ARCADE (CDD & Dup15q) Demographics (n=11): Dup15q (n=6) and CDD (n=5) 7/11 female, median age: 12 yr. (range 3, 27) 7/11 on 3 or more concomitant antiseizure medications topiramate, clobazam, felbamate, rufinamide, valproate Safety: Consistent with prior observations
Slide 35: ARCADE Enrollment Complete With Data Now Expected Q3 2020
ARCADE Enrollment Complete With Data Now Expected Q3 2020 Open-label study enrolled 20 participants with CDD (n=12) and Dup15q (n=8) Current cohort sufficient to inform next steps in development for these ultra-rare (CDD) and rare (Dup15q) patient populations Data readout in Q3 2020 will inform next
Slide 36: ELEKTRA: Phase 2 Study in Pediatric Patients With DS or LGS – Study Design
ELEKTRA: A Phase 2 Study in Pediatric Patients With DS Or LGS — Study Design End of Trial Screening/Baseline Trial Design Global, multicenter, randomized, double-blind, placebo-controlled Final enrollment: n=141 Titrated: 100 mg BID, 200 mg BID, 300 mg BID (mg/kg dosing <60 kg) oral tablets Key Incl
Slide 37: ELEKTRA Efficacy Endpoints
ELEKTRA Efficacy Endpoints Primary Efficacy Endpoint Percent change from baseline in convulsive (DS) and drop (LGS) seizures frequency per 28 days OV935 vs PBO during maintenance period Secondary Efficacy Endpoint Responder rate- proportion of patients with worsening, no change, ≥25%, ≥50%, ≥75%, an
Slide 38: Data Readouts in 3Q2020 Will Determine Soticlestat Regulatory Path
Data Readouts in 3Q2020 Will Determine Soticlestat Regulatory Path ELEKTRA- Phase 2 double-blind, placebo-controlled study in DS & LGS Safety and efficacy ARCADE- Phase 2 open label study in ultra-rare CDD & Dup15q Safety and efficacy ENDYMION- long term open label extension- long term safety, toler
Slide 39: Soticlestat – Unique MOA with Broad DEE Development Program
Soticlestat – Unique MOA with Broad DEE Development Program What we know Looking ahead Novel CH24H enzyme target & unique MOA Strong preclinical data support clinical development Successfully completed 12-week Phase 1b/2a study in adult DEE Longer-term data from ENDYMION (open label extension) sugge
Slide 40: Fireside Chat and Q&A
FIRESIDE CHAT and Q&A Amit Rakhit MD, MBA President, Chief Medical Officer Head of R&D, Ovid Therapeutics Scott Demarest MD Assistant Professor Pediatric Neurology Children’s Hospital Colorado Julia Tsai PHD VP, Clinical Development – Epilepsy, Ovid Therapeutics
Slide 41: THERAPEUTICS
- THERAPEUTICS BOLDMEDICINE
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