Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Cantor Fitzgerald Global Healthcare Conference 2018
32 slides from Ovid Therapeutics's conference presentation, Oct 1, 2018
Slide 1: Corporate Presentation – Cantor Fitzgerald Global Healthcare Conference
Corporate Presentation Cantor Fitzgerald Global Healthcare Conference October 1, 2018
Slide 2: Forward-Looking Statements
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 referencing future e
Slide 3: Ovid at a Glance: Goal to Build a Leading Company in Neurology
Ovid at a Glance: Goal to Build a Leading Company in Neurology Focus on serious unmet needs: >1 million patients and families living with devastating rare neurological disorders Broad pipeline: Multiple opportunities in rare conditions; novel compounds and mechanisms; first potential medicine to tre
Slide 4: Clear Strategy That Enables Scalable Model
Late preclinical– to clinical-stage assets Clear Strategy That Enables Scalable Model
Slide 5: Robust Product Pipeline in Neurodevelopment and Epilepsies
Robust Product Pipeline in Neurodevelopment and Epilepsies *Also known as TAK-935. Co-development program with Takeda Pharmaceutical Company Limited. δ-selective GABAA receptor agonist Angelman Syndrome RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Fragile X Syndrome δ-selective GABAA receptor agonis
Slide 6: OV101 Potential First-in-Class Therapeutic for Angelman & Fragile X Syndromes
OV101 Potential First-in-Class Therapeutic for Angelman & Fragile X Syndromes
Slide 7: OV101: Significant Opportunity in Two Serious Rare Disorders
OV101: Significant Opportunity in Two Serious Rare Disorders Angelman syndrome Fragile X syndrome Well defined Genetic Disorders No approved therapies in US, EU, or ROW; treatment is limited to supportive care Lifelong disorders that present early in childhood Disruption of behavior, anxiety and cog
Slide 8: Reduced GABA Levels Lead to Loss of Tonic Inhibition
Reduced GABA Levels Lead to Loss of Tonic Inhibition in Both Angelman and Fragile X Syndrome Models FMRP Deficiency2 Reduced expression of GAD65/67 Decreased GABA synthesis Decreased GABA release UBE3A Deficiency1 GAT1 does not get ‘tagged’ Increased GAT1 Increased GABA reuptake Decreased Extrasynap
Slide 9: OV101: Potential First-in-Class GABAA Receptor Agonist
1Meera et al., J Neurophysiol. 2011; 2Belelli et al., J. Neurosci.2005; 3 Duguid et al. Journal of Neurosci. 2012 4Olmos-Serrano et al. J. Neurosci. 2010; 5Egawa et al. Sci. Trans. Med. 20126Olmos-Serrano et al. Dev. Neuro. 2011 Only δ-selective, extrasynaptic GABAA receptor agonist in clinical deve
Slide 10: OV101 in Angelman: Phase 2 Randomized, Double Blind, Placebo Controlled Trial
OV101 in Angelman: Phase 2 Randomized, Double Blind, Placebo Controlled Trial Orphan Drug and Fast Track designations First-ever double-blind, placebo-controlled clinical trial in Angelman syndrome . Source: Ovid press release August 6, 2018. STARS Topline Phase 2 Data. Primary Endpoint: Safety and
Slide 11: STARS Topline Data: Key Take-aways
STARS Topline Data: Key Take-aways as of August 6, 2018 . Source: Ovid press release August 6, 2018. STARS Topline Phase 2 Data. First positive clinical data for Angelman syndrome in >50 years Younger patients receiving single daily dose showed a trend of greater response Data consistent with hypoth
Slide 12: Presentation of STARS Phase 2 Results at AACAP
Presentation of STARS Phase 2 Results American Academy of Child & Adolescent Psychiatry (AACAP) Poster Presentation Accepted – Thursday, Oct 25, 2018: Title: Topline Results from a Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of
Slide 13: STARS Topline Data: Statistically Significant Improvement in CGI-I
Placebo OV101 QD OV101 BID Combined OV101 Week 6 (n) 27 27 27 54 Mean 3.60 3.35 3.54 3.45 Week 12 (n) 27 27 28 55 Mean 3.79 3.00 3.58 3.29 p-value Week 12 0.0006 0.3446 0.0103 *Based on Mixed Model Repeated Measures analysis adjusting for visit, treatment, age, and visit by treatment interaction Mea
Slide 14: Patients in QD Group Showed Greater Improvement in CGI-I at Week 12
Patients in each CGI-I score category (%) STARS Topline Data: Patients in QD Group Showed Greater Improvement in CGI-I at Week 12 Compared to Placebo Group . Source: Ovid press release August 6, 2018. STARS Topline Phase 2 Data. 22.2 66.6 42.8
Slide 15: OV101's Development in Angelman Syndrome: Next Steps
OV101’s Development in Angelman Syndrome Medical Conference Final results for CGI, sleep and motor at AACAP Oct 2018 Additional Trials Plan to initiate Angelman syndrome open-label extension study (ELARA) in Q4 2018 Regulatory Dialogue Expect to meet with the FDA by the end of the year; will provide
Slide 16: OV101 in Fragile X Syndrome: Phase 2 Clinical Trials Underway
OV101 in Fragile X Syndrome: Phase 2 Clinical Trials Underway Orphan Drug and Fast Track designations Primary Objective: Assess safety and tolerability of OV101 over 12 weeks of treatment across different daily dosing regimens Patient Population: Adult and adolescent males with diagnosis of FXS (n=3
Slide 17: Phase 2 ROCKET Trial in Patients with Fragile X Syndrome
1 -21 OV101 Treatment Arm (5mg QD) OV101 Treatment Arm (5mg TID) OV101 Treatment Arm (5mg BID) Screening Baseline & Randomize (1:1:1) Phase 2 ROCKET Trial in Patients with Fragile X Syndrome Week 12 Timeline Key Endpoints Primary Endpoint: Incidence of AEs Secondary Endpoint: Changes in behavior Tri
Slide 18: OV935 New Approach to Potentially Treat Rare Developmental and Epileptic Encephalopathies
OV935 New Approach to Potentially Treat Rare Developmental and Epileptic Encephalopathies
Slide 19: CH24H Pathway is Unique to the Brain - 24HC is a Measurable Marker
CH24H is a brain-specific enzyme for cholesterol catabolism 24HC is a clinically monitorable plasma marker that correlates with active target engagement CH24H Pathway is Unique to the Brain - 24HC is a Measurable Marker in Plasma OV935 T 24HC, 24S-hydroxycholesterol; CH24H, cholesterol 24‑hydroxylas
Slide 20: Why CH24H Pathway is Important in Epilepsy
Why CH24H Pathway is Important in Epilepsy 1Barker-Haliski et al.,Cold Spring Harb Perspect Med. 2015 2Mast N, et al. J Biol Chem. 2017 3Sodero AO, et al. EMBO J. 2012 4Paul SM, et al. J Neurosci. 2013 5Sun MY, et al. J Neurophysiol. 2016 6Linsenbardt AJ, et al. Neuropharmacology. The Vicious Cycle
Slide 21: OV935: Potential First-in-Class CH24H Inhibitor
OV935: Potential First-in-Class CH24H Inhibitor OV935 1Barker-Haliski et al.,Cold Spring Harb Perspect Med. 2015 2Mast N, et al. J Biol Chem. 2017 3Sodero AO, et al. EMBO J. 2012 4Paul SM, et al. J Neurosci. 2013 5Sun MY, et al. J Neurophysiol. 2016 6Linsenbardt AJ, et al. Neuropharmacology. OV935 B
Slide 22: Novel Mechanism of Actions Needed to Treat Refractory Seizures
Refractory seizures do not respond to medicines that exert anti-seizure effect through same target or MOA as marketed AEDs Approximately 100,000- 200,000 US Population with Rare Seizure Disorders* Illustrative purposes only 1 out of 3 PATIENTS Small number achieve seizure freedom with addition of 1
Slide 23: Focus of Broad OV935 Development Program: Treatment Refractory Seizures
Focus of Broad OV935 Development Program: Treatment Refractory Seizures Developmental and Epileptic Encephalopathies (DEE): Significant unmet needs with few available therapies Dravet 80% have mutation in SCN1A gene US prevalence: ~7000-9,000 Lennox-Gastaut Some patients have SCN2A mutations <10% co
Slide 24: OV935: Summary Results from Phase 1 Studies
Phase 1 Studies Design Patients Doses 1. Safety and Tolerability Randomized, double blind, placebo controlled, SAD 48 15-1, 350,mg QD 2. Safety and Tolerability Randomized, double blind, placebo controlled, MAD* 40 100-600mg QD, and 300mg BID, 14 days 3. Brain CH24H enzyme occupancy using PET Open-l
Slide 25: 24HC Levels Observed In Phase 1: Dose Dependent Reduction
24HC Levels Observed In Phase 1: Dose Dependent Reduction Dosing (days) % of Baseline plasma 24HC conc. OV935 led to a dose dependent decrease in 24HC levels Correlation between plasma 24HC and enzyme occupancy (PET) may inform dose selection
Slide 26: OV935: Phase 1b/2a Clinical Trial
OV935: Phase 1b/2a Clinical Trial Week 12 Week 4 Randomization OV935 BID Placebo BID Baseline OV935 BID Topline Data Expected Q4:18 18 patients enrolled , age 18-65 years Two-part study: Part 1: Randomized, double-blind, placebo-controlled Part 2: Open label Primary endpoint: Safety & Tolerability I
Slide 27: OV935 Phase 1b/2a Trial: Study Population
OV935 Phase 1b/2a Trial: Study Population Eligibility Criteria Treatment resistant seizures Failed at least two prior treatment regimens Formal IQ <70 Epilepsies not caused by brain trauma Currently taking 1-4 AEDS General Characteristics: All-comers Patients enrolled not well controlled on current
Slide 28: OV935: Broad Development Program in Rare DEE
OV935: Rare Developmental and Epileptic Encephalopathies (DEE) ARCADE ELEKTRA ENDYMION Multi-center, long-term open label extension (OLE) study of patients with DEE who participated in a previous OV935 clinical study Phase 2, multicenter, open-label, pilot study in approximately 30 pediatric patient
Slide 29: Financial Highlights
Financial Highlights 2017: ~ $75 million IPO 2015: ~$75 million Series B Financing 2014: ~$5 million Series A Financing Cash Position as of June 30, 2018: $62.6 million in cash, cash equivalents and short-term investments ~24.6 million shares of common stock outstanding ~$155M raised in equity finan
Slide 30: 2018 Key Pipeline Milestones: A Year of Execution and Data Readouts
*Also known as TAK-935. Co-development program with Takeda Pharmaceutical Company Limited pursuant to a license and collaboration agreement ** Preclinical data suggest potential for use of OV329 in a variety of treatment-resistant epilepsies 2018 Key Pipeline Milestones: A Year of Execution and Data
Slide 31: Ovid Therapeutics: Executing on a clear vision, strategy and plan
Ovid Therapeutics Executing on a clear vision, strategy and plan Driving development of potentially first medicine to treat Angelman syndrome Major opportunities in several rare disorders of the brain Robust clinical pipeline with two different molecules Programs based on novel mechanisms of action
Slide 32: Y The Rare Neurological Disorder
Y The Rare Neurological Disorder Company THERAPEUTICS, BOLDMEDICINE Corporate Presentation 2018
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