Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Conference Presentation (June 2023)
23 slides from Zevra Therapeutics's conference presentation, June 2023
Slide 1: A Clinical Study with KP1077 in Adults with Idiopathic Hypersomnia
A Clinical Study with KP1077 in Adults with Idiopathic Hypersomnia Sponsored by 1
Slide 2: Speakers
Speakers Rene Braeckman, PhD Senior Vice President, Clinical Development Zevra Therapeutics Christopher Drake, PhD, FAASM, DBSM Director of Sleep Research, Henry Ford Health, Detroit, MI Principal Investigator, Zevra Study KP1077.D01 2
Slide 3: Presentation Outline
Presentation Outline • Zevra Company Overview Dr Rene Braeckman • Idiopathic Hypersomnia (IH) Disease Background Dr Chris Drake • New Treatment Option for IH (KP1077) Dr Rene Braeckman • Zevra’s Phase 2 Study in IH Dr Chris Drake 3
Slide 4: Zevra's Mission
Zevra’s Mission • Zevra is a rare disease therapeutics company driven by science, data and patients’ unmet need to create transformational therapies for diseases with limited or no treatment options. • Zevra pushes boundaries beyond what is possible to advance new therapies that meaningfully improve
Slide 5: IH Disease Background
SZEVRA THERAPEUTICS IH Disease Background
Slide 6: Still Sleepy After All These Years
Still Sleepy After All These Years A New Approach to the Treatment of Idiopathic Hypersomnia 6
Slide 7: Sleepiness is Common
Sleepiness is Common N = 3283 10% mean = 8.0 ± 4.6 9% 20% Sleepiness 8% 7% % Population 6% 5% 4% 3% 2% 1% 0% 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 Epworth Sleepiness Scale Score (0-24) Drake CL, et al. Sleep 2010; 33:745–752. 7
Slide 8: Excessive Sleepiness is a Medical Symptom
Excessive Sleepiness is a Medical Symptom N = 3283 10% mean = 8.0 ± 4.6 9% 8% 7% % Population 6% 5% 4% Idiopathic Hypersomnia 3% 2% 1% 0% 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 Epworth Sleepiness Scale Score (0-24) Drake CL, et al. Sleep 2010; 33:745–752. 8
Slide 9: What is Idiopathic Hypersomnia
What is Idiopathic Hypersomnia • Excessive Daytime Sleepiness (EDS) Irrepressible need to sleep or daytime lapses into sleep • “Sleep Drunkenness” / Sleep Inertia Prolonged difficulty waking, with frequent reentries into sleep, confusion, automatic behavior, and irritability • Unrefreshing Nighttime
Slide 10: Drowsy Driving Is a Serious Health and Safety Risk
Drowsy Driving Is a Serious Health and Safety Risk • The National Highway Traffic Safety Administration Estimated 1.35 million US drivers involved in a estimates that between 2005 drowsy driving related crash and 2009 drowsy driving in five-year period ending in was responsible for an 2002 annual av
Slide 11: Treatment of Excessive Sleepiness
Treatment of Excessive Sleepiness 11
Slide 12: New Treatment Option for IH
New Treatment Option for IH Zevra’s KP1077 Product 12
Slide 13: Prodrug Technology
Prodrug Technology Approved Drug with Ligand Prodrug with Improved Cleaved Drug through Human Sub-Optimal Properties Attributes Metabolic Process Following ingestion, normal human metabolic processes cleave the ligand and release the active drug 13
Slide 14: Serdexmethylphenidate (SDX)
Serdexmethylphenidate (SDX) Approved Drug with Ligand Prodrug with Improved Cleaved Drug through Human Sub-Optimal Properties Attributes Metabolic Process Prodrug: Serdexmethylphenidate (SDX) Active Drug: d-methylphenidate Ligand: Carboxymethylene linker + niacin + L-serine 14
Slide 15: SDX – Improved Attributes
SDX – Improved Attributes • Less abuse potential • Unique Pharmacokinetic Profile Pharmacokinetics is the study of a drug’s journey through a person’s body, including examining the amount of drug in a person’s plasma. The total amount of drug, the peak level of the drug, and the time it takes to rea
Slide 16: Pharmacokinetics (PK)
Pharmacokinetics (PK) Peak Occurs Around Waking DOSE 16
Slide 17: Pharmacokinetics (PK)
Pharmacokinetics (PK) Exposure Remains Until Bedtime DOSE DOSE 17
Slide 18: Zevra's Ongoing Phase 2 Trial in IH
Zevra’s Ongoing Phase 2 Trial in IH Study KP1077.D01 18
Slide 19: Study Design
Study Design Open-Label Double-Blind Screening Period Titration Period Withdrawal ≤35 days 5 weeks Period 2 weeks KP1077 KP1077 Placebo V1 V1A V2 V3 V4 V5 V6 V7 V8 FU Screening Visit LD ↑ ↑↓ ↑↓ ↑↓ 1 week Titration Window V = Visit; FU = Follow-Up Phone Call; LD = Low Dose (Starting Dose) 19
Slide 20: Eligibility Criteria for Study Participation
Eligibility Criteria for Study Participation Adults (18 years of age or older) who meet the following criteria may be eligible to participate: • Previously diagnosed with IH or not yet diagnosed with IH. • Patients with symptoms of IH but not yet diagnosed will be diagnosed in the screening period o
Slide 21: Study Participation
Study Participation If you have Idiopathic Hypersomnia and are interested in participating in the study: Visit the Zevra booth at the conference to get a list of clinical sites in the US, with location and contact information of a site near you. 21
Slide 22: Assistance to Participants in the Study
Assistance to Participants in the Study • No cost for patients to participate in the study • Payment for sleep & nap studies in a sleep lab, needed for new IH diagnosis • Reimbursement of travel expenses to/from study site For more information about study design and qualification criteria, visit www
Slide 23: Questions?
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