Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Cowen and Company 39th Annual Health Care Conference (March 2019)
36 slides from Ovid Therapeutics's conference presentation, Mar 11, 2019
Slide 1: Ovid Therapeutics, Cowen 39th Annual Health Care Conference
Ovid Therapeutics Cowen and Company 39th Annual Health Care Conference March 11, 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: Ovid Highlights: Building a Leading Company in Neurology
Ovid Highlights: Building a Leading Company in Neurology Ovid is focused on developing novel medicines for patients with rare CNS disorders Phase 2 and Phase 3 clinical development stage programs OV101: Phase 3 pivotal study of OV101 in Angelman Syndrome (NEPTUNE study, start 2H’19) OV101: Phase 2 o
Slide 4: Focused Product Pipeline in Neurodevelopment and Epilepsies
Focused Product Pipeline in Neurodevelopment and Epilepsies * Also known as TAK-935. Co-development program with Takeda Pharmaceutical Company Limited pursuant to a license and collaboration agreement. RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 δ-selective GABAA receptor agonist Angelman Syndrome
Slide 5: Focus on Rare Neurology with Ongoing Programs in Neurodevelopment Disorders and Epilepsy Types
Focus on Rare Neurology with Ongoing Programs in Neurodevelopment Disorders and Epilepsy Types Glutamatergic Pathways (Excitatory) GABAergic Pathways (Inhibitory) Rare Neurology Angelman syndrome Fragile X syndrome Epilepsies Dravet syndrome CDKL5 Deficiency Disorder Neurodevelopment Lennox-Gastaut
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, a First-in-Class Extrasynaptic δ-Selective GABAA Receptor Agonist
OV101, a First-in-Class Extrasynaptic δ-Selective GABAA Receptor Agonist OV101 is differentiated from other GABAA modulators Chemical structure conformationally rigid leading to greater specificity at binding pocket Active when endogenous GABA levels are low or absent Selectively targets extrasynapt
Slide 8: Tonic Inhibition is Central to Angelman & Fragile X Syndrome Pathophysiology
Tonic Inhibition is Central to Angelman & Fragile X Syndrome Pathophysiology Loss of tonic inhibition leads to dysregulation of neuronal circuit excitability Synaptic GABAA receptors mediate phasic inhibition Extrasynaptic δ-subunit-containing GABAA receptors mediate tonic inhibition Mouse models of
Slide 9: Data Suggest a Prolonged Pharmacodynamic Effect of OV101
Data Suggest a Prolonged Pharmacodynamic Effect of OV101, Despite its Similar Pharmacokinetic Profile to Other GABAA Agonists Statistical analysis of planar gradiometer configured *magnetoencephalographic (MEG) data OV101 (n=10) Source: Neuropharmacology 88 (2015), D. Nutt et al 60mins 160mins Zolpi
Slide 10: OV101: Scientific Rationale in Two Serious Rare Disorders
OV101: Scientific Rationale in Two Serious Rare Disorders Well Defined Genetic Disorders High unmet medical need No approved therapies in US, EU, or ROW; treatment is limited to supportive care Lifelong disorders that present early in childhood Fragile X Syndrome Caused by CGG expansion in FMR1 gene
Slide 11: Angelman Syndrome: Clinical Characteristics
Angelman Syndrome: Clinical Characteristics Average age of diagnosis is 21 Months Requires life-long care Life span typically normal Source: Angelman Syndrome Foundation website Physical / Motor Wide-based, unstable gait Protruding tongue Suck / swallow disorders Frequent drooling Other fine and gro
Slide 12: STARS was First Industry-Sponsored Clinical Trial in Angelman Syndrome
STARS was First Industry-Sponsored Clinical Trial in Angelman Syndrome * Excluding, poorly controlled seizure activity, concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, or cannabinoid derivatives or any other investigational agent, device, and/or procedure. BID=t
Slide 13: STARS Trial: OV101 Achieved Primary Endpoint of Safety and Tolerability
STARS Trial: OV101 Achieved Primary Endpoint of Safety and Tolerability and Demonstrated Statistical Significance in Exploratory Endpoints Primary Endpoint (Safety/Tolerability): OV101 was well-tolerated with a favorable risk profile Majority of AEs were mild Exploratory Endpoints (Efficacy): Global
Slide 14: CGI-I the Right Endpoint for Studying Patients with Angelman Syndrome
CGI-I (Clinical Global Impression-Improvement) the Right Endpoint for Studying Patients with Angelman Syndrome Patients Rated on a Likert 7-Point Scale* 1 = very much improved 2 = much improved 3 = minimally improved 4 = no change 5 = minimally worse 6 = much worse 7 = very much worse Validated meas
Slide 15: Data support rationale for OV101 mg QD as an apparent efficacious dose in AS
Data support rationale for OV101 15 mg QD as an apparent efficacious dose in AS Based on Patients’ Scores at Week 12, Two-Thirds Experienced Clinically-Meaningful Improvement and Nearly One-Third Had Much Improvement *Mean CGI-I Symptoms Overall Score at Week 12 (MMRM) *LS mean difference [Drug-Plac
Slide 16: STARS Phase Trial in Angelman Syndrome: Summary and Conclusions
STARS Phase 2 Trial in Angelman Syndrome: Summary and Conclusions Source: Ovid press release dated December 6, 2018 STARS achieved primary endpoint of safety and tolerability Exploratory efficacy with improvements in global function and relevant symptom domains in QD dosing arm vs placebo Global: St
Slide 17: OV101's Development in Angelman Syndrome – Pivotal Phase and Next Steps
OV101’s Development in Angelman Syndrome – Pivotal Phase 3 and Next Steps Pivotal Phase 3 NEPTUNE trial Design based on End-of Phase 2 Meeting with the FDA Single 12-week, two-arm, randomized, double-blind, placebo-controlled trial Once-daily (weight-based) dose Approximately 60 patients aged 4 to 1
Slide 18: OV101: Scientific Rationale in Two Serious Rare Disorders
OV101: Scientific Rationale in Two Serious Rare Disorders Well Defined Genetic Disorders High unmet medical need No approved therapies in US, EU, or ROW; treatment is limited to supportive care Lifelong disorders that present early in childhood Fragile X Syndrome Caused by CGG expansion in FMR1 gene
Slide 19: Fragile X Syndrome: Clinical Characteristics
Fragile X Syndrome: Clinical Characteristics Average age of diagnosis Males: 35 – 37 months Females: 41.6 months 29% of FXS families have 2 or more children with FXS Life span typically normal Williams et al., 2006 Physical Large prominent ears, prominent jaw & forehead Hyperflexible joints Classic
Slide 20: OV101 in Fragile X Syndrome: Phase 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 3 daily dosing regimens Patient Population (n=30): Adult and adolescent males with diagnosis of FXS Ages:
Slide 21: Phase ROCKET Trial in Patients with Fragile X Syndrome
Phase 2 ROCKET Trial in Patients with Fragile X Syndrome 1 OV101 Treatment Arm (5mg QD) OV101 Treatment Arm (5mg TID) OV101 Treatment Arm (5mg BID) Screening Baseline & Randomize (1:1:1) Week 12 Timeline Key Endpoints Trial Initiated: Q3:18 Topline Data Expected: 2H: 19 Primary Endpoint: Incidence o
Slide 22: OV935 New Approach to Potentially Treat Rare Developmental and Epileptic Encephalopathies
OV935 New Approach to Potentially Treat Rare Developmental and Epileptic Encephalopathies
Slide 23: Refractory seizures do not respond to medicines with same target or MOA as marketed AEDs
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 24: Focus of Broad OV935 Development Program: Severe Developmental and Epileptic Encephalopathies
Focus of Broad OV935 Development Program: Severe Developmental and Epileptic Encephalopathies Dravet 80% have mutation in SCN1A gene US prevalence: ~1;15,000 – 1:21,000 Few treatment options Lennox- Gastaut Some patients have SCN2A mutations US prevalence: ~1:10,000 <10% controlled by existing medic
Slide 25: CH24H Pathway is Unique to the Brain – 24HC is a Measurable Marker in Plasma
CH24H is a brain-specific enzyme for cholesterol catabolism 24HC is a clinically monitorable plasma marker that appears to correlate with active target engagement OV935: Mechanism of Action OV935 T In Plasma In Brain *CH24H, cholesterol 24‑hydroxylase ; 24HC, 24S-hydroxycholesterol CH24H Pathway is
Slide 26: OV935: Highly potent, orally administered inhibitor of cholesterol 24-hydroxylase
Highly potent, orally administered, inhibitor of brain specific cholesterol 24-hydroxylase Composition-of-matter patents through 2032, without regulatory extensions and Orphan Drug designations for Dravet and Lennox-Gastaut syndromes Novel mechanism of action and acts through pathways that are not t
Slide 27: OV935 Phase 1b/2a Clinical Trial Design
Key endpoints Primary endpoint Safety and tolerability Secondary and exploratory endpoints Pharmacokinetics (PK) parameters 24S-Hydroxycholesterol (24HC) plasma levels Change in seizure frequency from baseline Trial design Two-part trial* Part 1: Titration Titration (20 days): OV935 100/200 mg BID T
Slide 28: Phase 1b/2a Trial Results: Heterogeneous Patient Population
* Diazepam excluded; generally taken as needed and not as a scheduled medication † All patients prescribed concomitant AEDs Diagnoses Total (N=18) Lennox-Gastaut Syndrome 6 Epileptic Encephalopathy†† 6 Partial Seizures with Secondarily Generalization 1 Frontal Lobe Epilepsy 1 Tuberous Sclerosis Comp
Slide 29: Phase 1b/2a Trial Results: OV935 Generally Well Tolerated
Primary endpoints: Safety and Tolerability OV935 had a favorable safety profile and generally well tolerated; most AEs were mild PK OV935 plasma levels were dose proportional Seizure Frequency 61% reduction in median seizure frequency observed at Day 92 (end of Treatment Period, excluding patients o
Slide 30: Phase 1b/2a Trial Results: OV935 Reduced Plasma 24HC vs Placebo
Plasma 24HC concentration assessed at baseline, pre-dose at Days 11-85, and Day 121 (~30 days following the end of the Treatment) Plasma 24HC (ng/mL) On OV935 in both Part 1 and Part 2 On Placebo and who cross-over to OV935 after Day 31 4 13 4 12 4 7 4 OV935 plasma levels confirm PK/PD modeling and
Slide 31: Phase 1b/2a Exploratory Analysis: OV935 Reduced Seizure Frequency Over Time in Part 2
24% 28% 45% 0% 20% 40% 60% 80% 100% Reduction Median Reduction from Baseline in Total Seizures* per 28 Days (FSA, Part 2, including patients on perampanel) 69 85 Time (Study Days) 92 n=16 n=14 n=14 37% 41% 61% 0% 20% 40% 60% 80% 100% Reduction Time (Study Days) Median Reduction from Baseline in Tota
Slide 32: Change in Seizure Frequency vs. Plasma 24HC
Percent change in seizure frequency vs plasma 24HC in Part 2 treatment period Correlation Coefficient (p-value) Day Pearson Spearman Day 85 (n=6) 0.7747 (0.0704) 0.6957 (0.1248) Day 92 (n=6) 0.7962 (0.0581) 0.9856 (0.0003) Time 24 HC Seizure Day 11 Day 11 Day 2 - 11 Day 31 Day 31 Day 22 - 31 Day 85
Slide 33: Current OV935 Clinical Development Program for DEE
Dup15q syndrome CDKL5 syndrome Lennox-Gastaut syndrome Dravet syndrome ARCADE: Phase 2 Open-label pilot trial ~30 pediatric patients ELEKTRA: Phase 2 Double-blind, placebo-controlled trial 126 pediatric patients Primary endpoint: Safety and tolerability Phase 1b/2a Adult patients Primary endpoint: P
Slide 34: Financial Highlights
Financial Highlights As of December 31, 2018 (1) $72.5 million in cash, cash equivalents and short-term investments ~40.1 million shares of common stock outstanding (1) Pro forma for the net proceeds, before expenses, from an equity financing in February 2019.
Slide 35: 2019 Business Priorities and Milestones
*Also known as TAK-935. Co-development program with Takeda Pharmaceutical Company Limited pursuant to a license and collaboration agreement 2019 Business Priorities and Milestones Angelman Syndrome OV101 Fragile X Syndrome OV101 DEE & Rare Epilepsies OV935* Finalize Phase 3 NEPTUNE protocol and subm
Slide 36: Ovid Therapeutics: Our Inspiration to Succeed
Ovid Therapeutics: Our Inspiration to Succeed
Ovid Therapeutics — Cowen and Company 39th Annual Health Care Conference (March 2019) on 6ix