Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (March 2020)
28 slides from Ovid Therapeutics's investor presentation, Mar 13, 2020
Slide 1: March 2020 Corporate Presentation
March 2020 Corporate Presentation (NASDAQ: OVID) 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," "believe," "expect," "plan," "anticipate" and similar expressions (as well as other words or expre
Slide 3: The Ovid Opportunity
The Ovid Opportunity Experienced, Proven Leadership Team Focused on Execution Cash of ~$77M(1); fully funded through clinical data events (1) As of December 31, 2019 Pivotal Phase 3 trial for OV101 in Angelman syndrome ongoing; expect data in mid-2020 NEPTUNE OV935 program in Phase 2 studies for rar
Slide 4: Multiple Clinical Data Readouts Expected in 2020
Multiple Clinical Data Readouts Expected in 2020 PRODUCT CANDIDATE RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 INDICATION Angelman Syndrome Fragile X NEPTUNE – Ongoing ELARA OLE – Ongoing ROCKET – Ongoing SKYROCKET - Ongoing CDKL5 Deficiency Disorder / Dup15q Syndrome Dravet / LGS ARCADE(1) – Ongoi
Slide 5: Addressing Unmet Needs in Significant Populations
Addressing Unmet Needs in Significant Populations 1 Based on estimated prevalence. Epilepsy estimate includes only four types currently represented in Ovid clinical studies: Dravet, LGS, Dup15q, CDD., estimate patients in the US only $100K/Pt/Year $50K/Pt/Year Illustrative Example of Gross Sales 2 2
Slide 6: NEPTUNE, if positive, will be part of a broad data set to support registrational filings
NEPTUNE, if positive, will be part of a broad data set to support registrational filings for OV101 in the U.S. and rest of world Angelman Syndrome STARS study OV101 QD provides foundation for Phase 3 program given statistically significant, and clinically meaningful effect in CGI-I versus placebo Fa
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 19651 Treatment options are symptomatic and include anti-seizure therapies Affects 1:12,000 to 20,000 people2,3,4 Affects males and females in equal numbers3 Disease r
Slide 8: Background on Angelman Syndrome and OV101
UBE3A, ubiquitin protein ligase E3A; GABA, ɣ-aminobutyric acid. Background on Angelman Syndrome and OV101 Restores impaired tonic inhibition Highly selective extrasynaptic GABAA receptor (GABAAR) agonist, which binds to the δ-subunit of GABAAR as an orthosteric agonist Differentiated from all other
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: 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 11: 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. Effect over multiple domains in this heterogeneous population is best captured by CGI-
Slide 12: STARS Data Supports Once a Day Dosing for the Phase 3 NEPTUNE Trial
STARS Data Supports Once a Day Dosing for the Phase 3 NEPTUNE Trial AS=Angelman syndrome; BID=twice a day; CGI-I=Clinical Global Impressions–Improvement; QD=every day. Bird LM et al. Poster presented at: AACAP 2018. Subjects in Each CGI-I Score Category (%) 22.2 66.6 42.8 Effect Increases Over Time
Slide 13: 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 74% of responders experienced clinical meaningful improvement in multiple domains QD dosing arm had 3X number of responders vs. placebo (66% vs. 22%) 1 Effect of
Slide 14: 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 15: Clear Regulatory Path Forward to Registration for OV101 for AS
Clear Regulatory Path Forward to Registration for OV101 for AS* Strategic Positioning First in disease, first-in-class, first-line treatment for individuals with Angelman syndrome* No approved therapies for Angelman syndrome Orphan drug designation in US and EU US Registration Package to Support Glo
Slide 16: Fragile X Syndrome
Fragile X Syndrome OV101 normalizes anxiety, irritability, repetitive behaviors and hyperactivity in animal models of Fragile X ~70K patients in the US No approved therapies ROCKET Phase 2 signal-finding study to assess: Three different daytime dose regimens Safety and tolerability (primary endpoint
Slide 17: 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 18: OV101: Upcoming Data Readouts
OV101: Upcoming Data Readouts NEPTUNE Phase 3 study in Angelman syndrome Topline readout anticipated mid-2020 ROCKET & SKYROCKET Phase 2 studies in Fragile X syndrome Data anticipated early 2Q 2020
Slide 19: Robust clinical program for developmental and epileptic encephalopathies (DEE) patients
Robust clinical program for developmental and epileptic encephalopathies (DEE) patients In adult Phase 1b/2a study Good safety profile and generally well tolerated 61% reduction in median seizure frequency ENDYMION (open label extension) evaluating long-term safety and efficacy In a cohort from our
Slide 20: 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 21: 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 22: Phase 1b/2a Placebo-Controlled 12-week Adult DEE Trial Results
Phase 1b/2a Placebo-Controlled 12-week 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
Slide 23: ENDYMION: An OLE Study to Evaluate Long-Term Safety and Efficacy of Soticlestat
ENDYMION: 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 titratio
Slide 24: ENDYMION: Median Seizure Frequency Reduction Increased Over Time
ENDYMION: Median Seizure Frequency Reduction Increased Over Time Chart shows data from the 6 patients in adult DEE trial who continued in ENDYMION after the 2001 Study Break between end of Adult DEE trial and ENDYMION (range 6-52 weeks) Baseline seizure frequency of median 11.5 seizures per 28 days
Slide 25: 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 SAEs reported Consistent with prior observations in 12-week Phase 1b/2a adul
Slide 26: Soticlestat: Upcoming Data Readouts
Soticlestat: Upcoming Data Readouts ARCADE (Ultra Rare DEEs) All completed 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 (LGS and Dravet) Interest in trial has been
Slide 27: 2020: A Transformative Year for Ovid
2020: A Transformative Year for Ovid We anticipate significant value-creating events over the coming year 1 As of December 31, 2019. 2 As of March 4, 2020. Financial highlights ~$76.7 million in cash, cash equivalents and short term investments1 ~54.7 million shares of common stock outstanding2 OV10
Slide 28: Ovid Therapeutics: Our Inspiration to Succeed
Ovid Therapeutics: Our Inspiration to Succeed
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