Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (February 2019)
36 slides from Ovid Therapeutics's investor presentation, Feb 19, 2019
Slide 1: Ovid Therapeutics Corporate Presentation February 2019
Ovid Therapeutics Corporate Presentation February 2019 Exhibit 99.1
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 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 OV101 is entering a Phase 3 pivotal study in 2H 2019 Angelman Syndrome is a rare neurodevelopmental disorder for which no treatments exist End of Phase 2 meetin
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 The company is considering a rationalization of its resources to focus on its clinical stage assets, OV101 and OV935, which may include elimination or consolidation of functions, including the elimination or reduction of early stage research
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: OV101: First-in-Class GABAA Receptor Agonist with Potential to Restore Tonic Inhibition
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 Angelman syndrome and Fragile X syndrome result in reduced GABA levels,
Slide 9: STARS is First Industry-Sponsored Clinical Trial in Angelman Syndrome
STARS is 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=
Slide 10: STARS Trial: OV101 Achieved Primary Endpoint of Safety and Tolerability
Four discontinued due to AEs (placebo, n=1 (irritability), QD, no discontinuations, BID, n=3 (myoclonus, seizure, irritability/anxiety/sleep disorder) At least one treatment-emergent AE: placebo (25/29 – 86.2%), 15 mg QD (27/29 --93.1%) and BID (25/29 – 86.2%) There were no deaths across OV101 and p
Slide 11: CGI-I is a Common Outcome Measure to Assess Overall Clinical Symptomatology
Clinical Global Impressions – Improvement (CGI-I) is a Common Outcome Measure used to Assess Overall Clinical Symptomatology * Scores are assessed from baseline, defined as initiation of treatment. AS = Angelman syndrome ** a framework will be developed for study investigators to ensure uniform use
Slide 12: STARS Data Suggest Changes in Sleep and Motor Contributed to CGI-I Improvement
STARS Data Suggest Changes in Sleep and Motor Contributed to the Statistically Significant Improvement in CGI-I Global Improvement CGI-I Sleep Actigraphy Clinical Impression -sleep Motor Bayley PEDI-CAT CHAQ Zeno™ Walkway Exploratory Endpoints: Observed Changes CGI-I symptoms overall (MMRM1) = (QD)
Slide 13: STARS Trial: OV101 Reached Statistical Significance in CGI-I at Week 12
Response Based on CGI-I Symptoms Overall and Clinical Impression of 9 Domains at Week 12* 100 90 80 70 60 50 40 30 20 10 0 Proportion of subjects (%) Placebo (n=28) Combined OV101 (n=57) STARS Trial: OV101 Reached Statistical Significance in CGI-I at Week 12 27.4% difference p=0.0206 66.7% 39.3% Bas
Slide 14: STARS Trial: CGI-I Symptoms Overall Scores Compared with Placebo
Data provide supportive rationale for OV101 15 mg QD as an efficacious dose in AS STARS Trial: CGI-I Symptoms Overall Scores Compared with Placebo Based on Mixed Model Repeated Measures (MMRM) analysis including fixed effects for visit, treatment, age (adult vs adolescent) and visit by treatment int
Slide 15: STARS Phase 2 Trial: Summary and Conclusions
STARS Phase 2 Trial : Summary and Conclusions Source: Ovid press release dated December 6, 2018 STARS achieved primary endpoint of safety and tolerability OV101 was well-tolerated with an overall favorable safety profile Global function Statistically significant improvement in CGI-I in the OV101 QD
Slide 16: OV101's Development in Angelman Syndrome – Next Steps
OV101’s Development in Angelman Syndrome – Next Steps Pivotal Phase 3 NEPTUNE trial Design based on End-of Phase 2 Meeting with the FDA Single 12-week trial, two-arm, randomized, double-blind, placebo-controlled trial Once-daily dose Approximately 50-60 patients aged 4 to 12 years Primary endpoint o
Slide 17: 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 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: Novel Mechanisms of Action are 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 20: Robust Preclinical Data on Potential Anti-Epileptogenesis Mechanism of OV935
Robust Preclinical Data Across Several Well-Established Models on Potential Anti-Epileptogenesis Mechanism of OV935 Survival benefit in two transgenic epilepsy models Anti-inflammatory and glial modulatory activity contributing to disease modification in animal models Neuroprotection from a certain
Slide 21: 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 22: OV935: First in Class Inhibitor of CH24H
l. J May address underlying mechanisms behind refractory seizures in epilepsy May provide benefit through pathways that are not targets of conventional anti-epileptic drugs (AEDs) Orphan Drug designations for Dravet and Lennox- Gastaut syndromes Composition-of-matter patents through 2032, without re
Slide 23: OV935: Summary Results from Four Phase 1 Trials in Healthy Volunteers
Single doses up to 1,350mg well tolerated; no SAEs reported In 14-day MAD* trial, doses of 100mg, 300mg and 400mg QD were well tolerated All TEAEs resolved with continued dosing through day 15 OV935 led to a dose dependent decrease in 24HC levels Correlation between plasma 24HC and enzyme occupancy
Slide 24: 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: Randomized, double-blind, placebo-controlled, titrati
Slide 25: Phase 1b/2a Trial: Patient Disposition and Demographics
Part 2 (60 days) Baseline/screening Completed (n=14) Part 1 Withdrawal† † (n=2) Screened (n=26) Screen failure* (n=8) Placebo (n=4) OV935 (n=14) Withdrawal† (n=2) OV935 (n=16) (30 days) Safety and PK assessments Safety, PK, and exploratory efficacy assessments (28-42 Days) Did not meet entrance crit
Slide 26: 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 27: Phase 1b/2a Trial Results: OV935 Generally Well Tolerated
*There were no deaths across OV935 and placebo treatment arms †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. † † Safety set:
Slide 28: OV935 Reduced Plasma 24HC vs Placebo – Potential Biomarker of Target Engagement
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 29: 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 30: 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 31: OV935 Phase 1b/2a Trial in Adults with DEE - Summary
Primary endpoints: Safety and Tolerability OV935 Phase 1b/2a Trial in Adults with DEE - Summary OV935 had a favorable safety profile and was generally well tolerated; most AEs were mild PK OV935 plasma levels were dose proportional Seizure frequency 61% reduction in median seizure frequency was obse
Slide 32: Focus of Broad OV935 Development Program: Severe DEEs
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 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: Next Steps for OV935 Clinical Development
ELEKTRA and ARCADE Trials in Pediatric Patient Populations Continue to initiate sites and enroll eligible patients in the ENDYMION open-label extension trial Continue dose and titration schedule being used in the Phase 2 ELEKTRA & ARCADE trials Amend ELEKTRA/ARCADE protocols to: Specify perampanel u
Slide 35: Financial Highlights
Financial Highlights As of December 31, 2018: $41.5 million in cash, cash equivalents and short-term investments ~24.6 million shares of common stock outstanding
Slide 36: 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 2018 2019 Fragile X Syndrome OV101 DEE & Rare Epilepsies OV935* Program Phase 2 STARS top-line d
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