Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (September 2019)
38 slides from Ovid Therapeutics's investor presentation, Sep 9, 2019
Slide 1: September 2019 Corporate Presentation
September 2019 Corporate Presentation 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: Ovid Highlights: Building a Leading Company in Neurology
Our vision is to transform the lives of people with rare neurological disorders Robust pipeline of first-in-class therapeutics with near-term value inflection clinical data points Four clinical read-outs over the next 12 months with two late stage programs Initiated NEPTUNE, our pivotal Phase 3 tria
Slide 4: Four Major Data Readouts Expected within the Next Months
Four Major Data Readouts Expected within the Next 12 Months PRODUCT CANDIDATE RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 INDICATION Angelman Syndrome Fragile X STARS – Completed NEPTUNE – Initiated ELARA OLE – Ongoing ROCKET – Ongoing CDKL5 Deficiency Disorder / Dup15q Syndrome Dravet / LGS ARCADE
Slide 5: We Are Focused on Addressing Unmet Need in Significant Populations
We Are Focused on Addressing Unmet Need in Significant Populations 1 Based on estimated prevalence. Epilepsy estimate includes only four types currently represented in Ovid clinical studies: Dravet, LGS, Dup15q, CDD. $100K/Pt/Year $50K/Pt/Year Illustrative Example of Gross Sales 2 2 Based on illustr
Slide 6: Both FDA and BfArM agree that a single pivotal study could support NDA/MAA
Both FDA and BfArM agree that a single pivotal study could support NDA/MAA Angelman Syndrome STARS study OV101 QD provides foundation for Phase 3 program given statistically significant effect in CGI-I versus placebo Favorable safety and tolerability profile Phase 3 NEPTUNE Alignment with FDA and Bf
Slide 7: Angelman Syndrome: Need for effective treatment options could not be greater
Angelman Syndrome Need for effective treatment options could not be greater No approved therapies since condition first described in 1965.1 Treatment options are symptomatic and include anti-seizure therapies. Affects 1:12,000 to 20,000 people2,3 Affects males and females in equal numbers3 Character
Slide 8: Background on Angelman Syndrome and OV101
UBE3A, ubiquitin protein ligase E3A; GABA, ɣ-aminobutyric acid. Background on Angelman Syndrome and OV101 Multi-symptomatic genetic condition caused by impaired tonic inhibition Loss of function in UBE3A gene on maternal chromosome 15 disrupts GABA signaling and results in impaired tonic inhibition
Slide 9: STARS: A Phase 2 Clinical Trial in Angelman Syndrome
Assessment Points (weeks) Placebo (placebo morning, placebo night) OV101 BID (10 mg morning, 15 mg evening) OV101 QD (placebo morning, 15 mg evening) -2 0 6 12 Screening Baseline & Randomize (1:1:1) STARS: A Phase 2 Clinical Trial in Angelman Syndrome * Excluding, poorly controlled seizure activity,
Slide 10: STARS: OV101 Achieved the Primary Endpoint of Safety and Tolerability
STARS: OV101 Achieved the Primary Endpoint of Safety and Tolerability The most frequent AEs across OV101 and placebo arms were vomiting, somnolence, irritability, aggression, and fever There were no deaths across OV101 and placebo arms * Descriptive data. † Drug-related TEAEs (n=4 subjects): Placebo
Slide 11: Measuring Efficacy of Therapeutics in Angelman Syndrome
Measuring Efficacy of Therapeutics in Angelman Syndrome Angelman syndrome effects all cells in the brain Patient presentations are highly heterogeneous Individual patients with AS have varying degrees of impairment with sleep, communication, behavior, motor, or seizures Ovid in consultation with reg
Slide 12: STARS Demonstrated Meaningful Improvement in Overall AS Symptomology
STARS Demonstrated Meaningful Improvement in Overall AS Symptomology Changes in global function as measured by CGI-I provided support for primary endpoint selection for the NEPTUNE trial Global Function: Statistically significant improvement on CGI-I scale Sleep: Reduction in latency to sleep onset
Slide 13: CGI-I Captures the Improvement in the Totality of a Patient's Symptoms
CGI-I Captures the Improvement in the Totality of a Patient’s Symptoms Validated measure used in neurology and psychiatry trials Co-primary or key secondary endpoint in several FDA-approved and ongoing clinical trial programs including: Nuplazid (Psychosis), Intuniv (ADHD) Clinician assessed Capture
Slide 14: STARS Data Supports Once a Day Dosing for the Phase 3 NEPTUNE Trial
*Least-square mean difference [Drug-Placebo] (95% CI) was -0.78 (-1.22, -0.35) with OV101 QD and -0.21 (-0.64, 0.22) with OV101 BID. BID=twice a day; CGI-I=Clinical Global Impressions–Improvement; QD=every day. Bird LM et al. Poster presented at: AACAP 2018. STARS Data Supports Once a Day Dosing for
Slide 15: Improvements Increase in Younger Ages and Over Time
Improvements Increase in Younger Ages and Over Time Age Group
Slide 16: 74% of STARS Study Responders Experienced Clinical Improvement in Multiple Domains
74% of STARS Study Responders Experienced Clinical Improvement in Multiple Domains 1 3 Domains Improvement Driver(s): Domains: 2 Domains 1 Domain (1) Responder analysis from the QD patient arm of STARS study n=27.
Slide 17: Consistent Positive Results from the Phase 2 STARS Study
Consistent Positive Results from the Phase 2 STARS Study Strong safety and tolerability profile Statistically significant results in CGI-I Effect of OV101 increases over time Effect is strongest in younger patients 74% of responders experienced clinical improvement in multiple domains QD dosing arm
Slide 18: CGI-I-AS to serve as the sole primary endpoint
CGI-I-AS to serve as the sole primary endpoint to measure clinical benefit in the Angelman syndrome population Same CGI-I scale utilized in STARS Developed anchors and clinician training materials specific to the core signs and symptoms of Angelman syndrome, including the domains that showed the mos
Slide 19: NEPTUNE – Pivotal Phase 3 Study in Angelman Syndrome Initiated
NEPTUNE – Pivotal Phase 3 Study in Angelman Syndrome Initiated; Anticipate Data by Mid-2020 Placebo (at bedtime) OV101 QD (weight based dosing, at bedtime) -2 0 6 12 Assessment Points (weeks) Screening Baseline & Randomize (1:1) Trial design Objectives Primary endpoint: CGI-I-AS at Week 12 of OV101
Slide 20: Clear Regulatory Path Forward to Registration for OV101 for AS
Clear Regulatory Path Forward to Registration for OV101 for AS Expedited Registration Supported by Streamlined Global Development Plan: Planned filing package = Phase 3 NEPTUNE study (single pivotal trial) + Phase 2 STARS study (supportive evidence) Extensive safety database US: Fast Track and Orpha
Slide 21: Fragile X Syndrome
Fragile X Syndrome OV101 normalizes anxiety, irritability, repetitive behaviors and hyperactivity in animal models of Fragile X ROCKET Phase 2 signal-finding study to assess: Three different daytime dose regimens Safety and tolerability (primary endpoint) Changes in behavioral symptoms Data expected
Slide 22: Significant Unmet Medical Need: Fragile X Syndrome
Significant Unmet Medical Need No approved therapies Current treatment options limited to physical, behavior, communication, and symptomatic therapy Most common inherited form of intellectual disability Prevalence ~ 1:4,000 males,~ 1:8,000 females* Diagnosed around 3 years; life-long condition Chara
Slide 23: FMR1 Gene Mutations Disrupt GABA Signaling and Result in Impaired Tonic Inhibition
FMR1 Gene Mutations Disrupt GABA Signaling and Result in Impaired Tonic Inhibition FMRP=fragile X mental retardation protein; FXS=fragile X syndrome; GABA=γ-aminobutyric acid; 1. Egawa K et al. Sci Transl Med. 2012;4:163ra157. doi:10.1126/scitranslmed.3004655. 2. Olmos-Serrano JL et al. Dev Neurosci
Slide 24: OV101 Normalizes Anxiety, Irritability, Repetitive Behaviors and Hyperactivity in FXS Mice
OV101 Normalizes Anxiety, Irritability, Repetitive Behaviors and Hyperactivity in FXS Mice Anxiety-like Behaviors Irritability / Aggressive Behaviors Repetitive Behaviors Hyperactive Behavior Fmr1 y/- mice (KO2) Treated with vehicle or OV101 30 min prior to behavioral testing
Slide 25: ROCKET: Phase 2 Signal Finding in Fragile X Syndrome
ROCKET: Phase 2 Signal Finding in Fragile X Syndrome to Assess Safety, Tolerability, Efficacy, and Optimal Dose OV101 5 mg QD OV101 5 mg TID OV101 5 mg BID Screening Baseline & Randomize (1:1:1) Key Inclusion Criteria Endpoints Primary Endpoint: Safety and tolerability of OV101 over 12 weeks across
Slide 26: Robust clinical program for developmental and epileptic encephalopathies (DEE) patients
Robust clinical program for developmental and epileptic encephalopathies (DEE) patients In adult study (-2001) Drug with good safety profile and generally well tolerated Reduced seizure over time in DEE ENDYMION (open label extension) to evaluate long term safety and efficacy Interim data Q3:19 Open
Slide 27: DEE Encompass Diverse Etiologies and Have Limited Approved Treatment Options
DEE Encompass Diverse Etiologies and Have Limited Approved Treatment Options 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
Slide 28: CH24H is a Novel Target That Potentiates Glutamatergic Signaling
1. Schmidt D, Schachter SC. BMJ. 2014. doi:10.1136/bmj.g254. 2. Russell DW et al. Annu Rev Biochem. 2009;78:1017–1040. doi:10.1146/annurev.biochem.78.072407.103859. 3. Rogawski et al. Cold Spring Harb Perspect Med. 2010. 4. Salamone A. Epilepsia. 2018. 5. Barker-Haliski M, White HS. Cold Spring Harb
Slide 29: OV935 (soticlestat): 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 OV9351,2 OV935 (soticlestat): A Highly-selective First-in-Class CH24H Inhibitor in Development to Reduce Glutamate
Slide 30: OV935 Was Evaluated in a 12-Week Phase 1b/2a Study in Adult Patients With DEE
OV935 (soticlestat) Was Evaluated in a 12-Week Phase 1b/2a Study in Adult Patients With DEE Two-Part Trial Design Part 1 Double-blind Tx (30 days) Part 2 Open-label Tx (60 days) Follow-up (30 days) Safety and PK assessments Safety, PK, and exploratory assessments OV935 BID End of trial Screening/bas
Slide 31: In Phase 1b/2a Study, OV935 Was Generally Safe and Well Tolerated
In Phase 1b/2a Study, OV935 (soticlestat) Was Generally Safe and Well Tolerated †Four patients discontinued due to AEs in OV935 treatment arms; Part 1: weakness (n=1); difficulty with walking/worsening lethargy (n=1); Part 2: seizure cluster (n=2) † † All SAEs were seizure clusters in three patients
Slide 32: OV935 Demonstrated 61% Reduction in Median Seizure Frequency at Day 92
Median Reduction from Baseline in Total Seizures* per 28 Days (FSA, Part 2, excluding patients on perampanel) *Data represent per protocol analyses; ITT group (n=18) demonstrated median reduction in total seizures of 36% at Day 85. Two patients withdrew in Part 1 (weakness (n=1); difficulty with wal
Slide 33: OV935 is Being Evaluated in a Robust and Comprehensive Clinical Development Program
OV935 is Being Evaluated in a Robust and Comprehensive Clinical Development Program Chromosome 15q Duplication Syndrome CDKL5 Deficiency Disorder Lennox-Gastaut syndrome Dravet syndrome ARCADE: Phase 2 Open-label pilot trial ~30 pediatric patients ELEKTRA: Phase 2 Double-blind, placebo-controlled tr
Slide 34: ENDYMION: An OLE Study to Evaluate Long-Term Safety and Efficacy of OV935
Trial Design Endpoints Key Inclusion Criteria ENDYMION: An OLE Study to Evaluate Long-Term Safety and Efficacy of OV935 *Patients who rollover from an open-label study will continue on their current dose for 104 weeks without titration. Clinicaltrials.gov/NCT03635073. Accessed January 29, 2019. Safe
Slide 35: ARCADE is a Phase 2 Open Label Study in Patients With Two Rare Epilepsy Disorders
ARCADE is a Phase 2 Open Label Study in Patients With Two Rare Epilepsy Disorders 8 weeks Follow-up Visit 15q Duplication (n~15) Baseline ~ 4-6 weeks Screening CDKL5 Deficiency Disorder (n~15) Dose Optimization Period Maintenance Period 12 weeks Key Inclusion Criteria Endpoints Primary efficacy: % C
Slide 36: ELEKTRA is a Phase 2 Double Blinded, Placebo Controlled Study in LGS and Dravet
ELEKTRA is a Phase 2 Double Blinded, Placebo Controlled Study in Patients With LGS and Dravet 8 weeks Follow-up Visit Dravet syndrome (n=40) Baseline ~ 4-6 weeks Screening Lennox Gastaut syndrome (n=86) Titration & Dose Optimization Period Maintenance Period 12 weeks Placebo Placebo Key Inclusion Cr
Slide 37: Transforming Lives of People with Rare Neurological Disorders
Transforming Lives of People with Rare Neurological Disorders NEPTUNE is a pivotal Phase 3 program for patients with Angelman syndrome CGI-I-AS is the sole primary endpoint Topline data by mid-2020 Clear route to registration No other programs for AS in clinic We anticipate significant value-creatin
Slide 38: Ovid Therapeutics: Our Inspiration to Succeed
Ovid Therapeutics: Our Inspiration to Succeed
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