Newcelx Ltd. · NASDAQ:NCEL
Newcelx — NLS Pharmaceutics — Investor Presentation (April 2021)
26 slides from Newcelx's investor presentation, Apr 6, 2021
Slide 1: Redefining Central Nervous System Therapies Investor Presentation
Con f i d e n t i a l Redefining Central Nervous System Therapies Investor Presentation March 2021 Alex Zwyer, CEO Redefining Central Nervous System Therapies Investor Presentation April 2021 Alexander Zwyer, CEO
Slide 2: Forward Looking Statements
Forward Looking Statements 2 This presentation contains express or implied forward - looking statements within the Private Securities Litigation Reform Act of 1995 and other U . S . Federal securities laws . For example, we are using forward - looking statements when we discuss the expected timing o
Slide 3: NLS Pharmaceutics Overview
NLS discovers and develops drug treatments for rare and complex central nervous system (CNS) disorders Lead product candidate, Quilience ® , entering Phase 2 for narcolepsy treatment, a $ 2 . 4 billion annual market and growing** Mazindol, the active ingredient in Quilience ® was used extensively of
Slide 4: Management: Deep Expertise in Clinical Development & Drug Repurposing
Management: Deep Expertise in Clinical Development & Drug Repurposing (1) Currently engaged as consultant / strategic advisor Carlos Camozzi, MD, PhD interim Medical Director (1) Experienced Chief Medical and Corporate Governance Officer in the Biopharmaceuticals field with over 30 years of C - leve
Slide 5: Investment highlights
• Second mazindol - based product candidate with strong clinical evidence • Build on success of Nolazol ® Phase 2 clinical trial • Ability to capture income from licensing deal with Eurofarma in Latin America • Large market, need for more - effective treatments remains high • Strong validation with
Slide 6: Narcolepsy: Debilitating Disorder, Large & Underserved Market
Narcolepsy: Debilitating Disorder, Large & Underserved Market • Narcolepsy is a chronic neurologic brain disorder caused by the brain’s inability to regulate sleep - wake cycles • Characterized by excessive daytime sleepiness (EDS). Sudden loss of muscle tone (cataplexy) , sleep paralysis and halluc
Slide 7: Severe Rare Disease with Unmet Medical Need
Severe Rare Disease with Unmet Medical Need Expressed frustration with current treatment options Thought that their narcolepsy symptoms are completely or mostly under control Stated that new treatment options are needed 93% 12 % 94% • Management of narcolepsy symptoms remains a challenge for patient
Slide 8: Mazindol Controlled-Release for Excessive Daytime Sleepiness and Cataplexy in Narcolepsy
Mazindol Controlled - Release (CR) - for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy ® • Long history of active ingredient (mazindol) used to treat narcolepsy (“compassionate use” programs) • Novel mechanism of action (MOA) with partial orexin - 2 receptor activation •
Slide 9: Mazindol – Unique Dual Mechanism of Action
Mazindol – Unique Dual Mechanism of Action (“MoA”) 9 Dual – Pan – Monoamide – Reuptake – Inhibitor / Orexin - 2 Receptor Partial - Agonist
Slide 10: Long Term Benefit/Risk Ratio of Mazindol in Patients with Drug-Resistant Narcolepsy
Long Term Benefit/Risk Ratio of Mazindol in Patients with Drug - Resistant Narcolepsy • Retrospective study conducted by a third party, funded by the French Health Ministry, to provide benefit and risk measures of the long - term use of mazindol in children & adults with narcolepsy or hypersomnia •
Slide 11: Quilience – Competitive Advantages
Novel MoA* partially targeting the orexin system Once - Daily Monotherap y addressing the main narcolepsy symptoms C a t a p l ex y Validated Efficacy of drug substance in chronic use off - label Well - Tolerated drug substance Excessive Daytime S l ee p i n es s Q u ili en ce ® – C o m p et i t i v
Slide 12: Quilience Regulatory Exclusivity & Patent Protection
Quilience ® Regulatory Exclusivity & Patent Protection Quilience ® co m p r ehens i ve ODD and IP strategy FDA Orphan Drug Designation (ODD) secures up to 7.5 years of marketing exclusivity from launch in the US (incl. pediatric exclusivity) Controlled - release mazindol patent provides coverage unt
Slide 13: Quilience Planned Phase 2 Clinical Study Design
Quilience ® Planned Phase 2 Clinical Study Design Study Design ▪ 4 - week, double - blind , placebo - controlled, randomized, multicenter, titrated fixed dose (3 mg) parallel group study. Primary Endpoint ▪ Change from baseline in daytime sleepiness as measured by the Epworth Sleepiness Scale (ESS).
Slide 14: Phase 2 Trial Design & Implications
Phase 2 Trial Design & Implications Objective to demonstrate safety & efficacy of mazindol CR in a prospective clinical trial for the treatment of narcolepsy Data are expected to inform a high - probability pivotal clinical trial program following advice from FDA & EMA Potential for value inflection
Slide 15: Quilience – Anticipated Development Timeline
Quilience ® – Anticipated Development Timeline 202 4 202 3 202 2 202 1 H2 2022 Initiation of Phase II/III Pivotal Trials H2 2023 Results Phase Phase II/III Pivotal Trials 2024 EMEA MAA & FDA NDA Approval Q 2 202 1 Open i n g o f I N D R eque s t f o r e x ped i t e d development designations(s) Q 4
Slide 16: Expert quote: Prof. Dr. G.J. Lammers
PROF. Dr. G.J. LAMMERS I know some narcolepsy patients who had a very good response on mazindol, and never reached a comparable level with any other pharmacological treatment including the most recently approved substances. Prof. of Neurology at Leiden University, current Chair of the Dutch Society
Slide 17: Quilience Compassionate Use Strategy
Quilience ® Compassionate Use Strategy Named Patient Program (NPP) Individual patients Cohort Program Defined group of patients National regulations Different nomenclature Patients with severe conditions Exhausted treatment options Cannot join clinical trials First Wave Countries Austria Belgium F r
Slide 18: Mazindol CR for the Treatment of ADHD
Mazindol CR - for the Treatment of Attention Deficit Hyperactivity Disorder (ADHD) ® 18
Slide 19: Positive Phase 2 Results for Mazindol CR Demonstrated in ADHD Trial
Positive Phase 2 Results for Mazindol CR Demonstrated in ADHD Trial Nolazol ® (mazindol CR) NLS - sponsored Phase 2 trial in ADHD demonstrated efficacy comparable to first - line CII stimulants with favorable tolerability Trial validated the Company’s once - daily formulation Mazindol CR had an adva
Slide 20: Phase 2 Trial Primary Endpoint Results – Effect Size
Phase 2 Trial Primary Endpoint Results – Effect Size Greater Than or Equal to C - II Stimulant Levels Adult patients receiving Nolazol ® (mazindol CR) showed effect size greater than current ADHD best - in - class drugs Phase 2 clinical trial at seven sites in US Outpatient, multicenter, randomized,
Slide 21: Mazindol CR Well-Tolerated in Phase 2 Study
Mazindol CR Well - Tolerated in Phase 2 Study Subjects with any TEAEs 31 72.1% 21 50.0% Gastrointestinal disorders 19 44.2 7 16.7 • Constipation 3 7.0 0 0 • Dry mouth 10 23.3 2 4.8 • Nausea 5 11.6 0 0 General disorders 6 14.0 2 4.8 • Fatigue 5 11.6 1 2.4 Infections and infestations 8 18.6 7 16.7 • U
Slide 22: Competitive comparison of ADHD stimulants
Manufacturer Shire Shire Neos Neos J&J / Janssen NLS Pharmaceutics Ge n e ric N a m e Lisdexamfetamine Amphetamine Amphetamine Methylphenidate Methylphenidate Mazindol only non - amphetamine or non - methylphenidate containing product Novel MOA No No No No No YES (SDNRI and Orexin 2 partial agonist)
Slide 23: Expert quote: Prof. Dr. J. Newcorn
PROF. Dr. J. NEWCORN Associate Professor of Psychiatry and Pediatrics and Director of the Division of Child and Adolescent Psychiatry at Mount Sinai Medical Center in New York We have not seen any potential treatment for ADHD that had effects as robust as we saw in the Phase 2 adult study, except fo
Slide 24: Pipeline for Future Value Creation
Pipeline for Future Value Creation 24 ► Mazindol CR Product Candidates (Quilience ® & Nolazol ® ) Narcolepsy, Idiopathic Hypersomnia (IH), Obstructive Sleep Apnea (OSA), Substance Use Disorder (SUD), and Attention Deficit Hyperactivity Disorder (ADHD) ► Assets with Patents Issued or Patent Applicati
Slide 25: Anticipated Milestones & Corporate Priorities
File IND in U . S . to initiate Quilience ® Phase 2 trial in patients with narcolepsy ; potential for accelerated regulatory pathway designations Initiate compassionate use programs outside the U.S. with potential for revenue generation Complete Phase 2 trial for Quilience ® ; results anticipated in
Slide 26: NLS Pharmaceutics – Connecting Brains
10: NLS Pharmaceutics Connecting Brains Connecting Brains
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