Newcelx Ltd. · NASDAQ:NCEL
Newcelx — NLS Pharmaceutics — Investor Presentation (July 2021)
29 slides from Newcelx's investor presentation, Jul 6, 2021
Slide 1: Investor Presentation | July 2021
Investor Presentation | July 2021 Alexander Zwyer, CEO & Co - Founder defining Central Nervous System Therapies
Slide 2: Forward-Looking Statements
Forward - Looking Statements 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: Business Highlights
⌾ NLS develops drug treatments for rare and complex Central Nervous System (CNS) disorders ⌾ Quilience ® is entering Phase 2 for narcolepsy treatment: $2.4B annual market** ⌾ Active ingredient in Quilience ® , mazindol, was used extensively in compassionate use off - label ⌾ Orphan Drug Designation
Slide 4: Management Team
Management Team Alex Zwyer, MBA Chief Executive Officer & Co - Founder A co - founder of the company with extensive operational, C - level pharmaceutical experience. A serial entrepreneur and strong leader with a proven track - record. Served as Chief Operating Officer at Viforpharma AG, a global sp
Slide 5: Leadership
Leadership “The heart and soul of our company is courage and innovation. Our unrelenting drive to break the status quo and develop anew where others see the impossible.” Alex Zwyer , MBA CEO & Co - Founder NLS Pharmaceutics 5
Slide 6: Focused on Rare and Complex CNS Disorders
Focused on Rare and Complex CNS Disorders Large market, high need for more effective treatments Strong validation with decades of off - label use of mazindol in narcolepsy Novel mechanism of action addressing root cause of the disease Modest development costs anticipated to achieve regulatory approv
Slide 7: Narcolepsy: Debilitating Disorder; Large and Underserved Market
Classic narcolepsy is associated with very low or undetectable levels of the hormone, orexin, in the brain.* Narcolepsy treatments generated revenues of $2.4 billion in 2018, and are projected to reach $5.4 billion by 2026, a CAGR of 10.3%.** Significant valuations are being assigned to public compa
Slide 8: Severe Rare Disease with Unmet Medical Need
Severe Rare Disease with Unmet Medical Need Source: 2018. National Know Narcolepsy Survey. https://knownarcolepsy.com/hcp/impact * Xyrem label & Safety and efficacy of long - term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice (Sleep Medicine 35 (2017) 80 - 84) Thou
Slide 9: Quilience key features and development path
- Long history of active ingredient (mazindol) used to treat narcolepsy off - label (“Compassionate Use” Programs) - Novel mechanism of action with partial Orexin - 2 receptor activation - Orphan Drug Designation (ODD) granted in the U.S. and Europe - Anticipated Phase 2 trial initiation H2 2021, in
Slide 10: Mazindol: Unique Dual Mechanism of Action (MOA)
Mazindol: Unique Dual Mechanism of Action (MOA) Quilience ® (mazindol CR) OX 2 R Downstream wake - promoting neurotransmitters Postsynaptic Neuron Confirmed robust binding to dopamine and norepinephrine transporters Enhances dopamine and norepinephrine neurotransmission Improves “top down” regulatio
Slide 11: Retrospective Benefit/Risk Ratio: Mazindol in Patients with Drug Resistant Narcolepsy
Study* funded by the French Health Ministry concluded: “Mazindol has a major effect on sleepiness, possibly greater than that of modafinil (Provigil)” - Efficacy and sustained benefits - Safety profile with long - term chronic use in adults and children Study Conclusion: “Mazindol has a long - term,
Slide 12: Quilience: Competitive Advantages
Quilience ® : C ompetitive Advantages * Discovered by scientists at NLS Pharmaceutics Well - tolerated drug substance Novel MOA* partially targeting the orexin system Validated efficacy of drug substance in chronic use off - label Schedule IV: low potential for abuse , misuse & diversion Once - dail
Slide 13: Quilience: Regulatory Exclusivity and Patent Protection
Quilience ® : Regulatory Exclusivity and Patent Protection Comprehensive Orphan Drug & IP strategy U.S. Orphan Drug Designation (ODD) secures up to 7.5 years of marketing exclusivity from launch in the U.S. EU ODD secures up to 12 years of marketing exclusivity from launch in the EU (incl. approval
Slide 14: Quilience: Planned Phase 2 Clinical Study
Quilience ® : Planned Phase 2 Clinical Study 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 Epworth Sleepiness Scale. Key secondary endpoi
Slide 15: Phase 2 Trial Design and Implications – Optimized for Success
Objective : demonstrate safety and efficacy of mazindol ER in a prospective clinical trial for treatment of narcolepsy ⌾ Trial design is patient - centric and COVID - 19 compliant with an open - label extension study to aid recruitment ⌾ Data are expected to inform a high - probability pivotal clini
Slide 16: Quilience: Anticipated Development Timeline
Quilience ® : Anticipated Development Timeline 2021 2022 2023 2024 Q2 2021 Opened IND request for expedited development designations(s) H2 2021 Initiate Phase 2 Study Q4 2021 Interim topline results from Phase 2 Study H2 2023 Results : Phase II/III Pivotal Trials Anticipated EMEA MAA and FDA NDA app
Slide 17: Expert quote: Professor 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.” Professor Dr. G.J. Lammers Professor of Neurology at Leiden University, current Chair of the Dutc
Slide 18: Quilience: Compassionate Use Strategy
Additional objectives 1: Bridge patients from the end of clinical trials to commercialization 2: Leverage real - world and patient - centric data collected (broad populations, quality of life, satisfaction) 3: Professionally address patient and physician requests (formal published policy and process
Slide 19: Mazindol CR: Treatment of Attention Deficit Hyperactivity Disorder
Mazindol CR: Treatment of Attention Deficit Hyperactivity Disorder 19
Slide 20: Nolazol (mazindol CR) Positive Phase 2 Results in ADHD
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 • Studies suggest ADHD and narcolepsy are genetically related with possible common unde
Slide 21: Phase 2 Trial Primary Endpoint Results
Least Squares Mean improvements: - 18.9 (mazindol CR) and - 5.7 (placebo) Mean difference: - 13.2 ( - 18.7, - 7.6) Mazindol CR (1 - 3 mg) Placebo ADHD - RS - DSM5 least squares mean scores * ** ** ** ** ** Phase 2 Trial Primary Endpoint Results – Greater Than or Equal to C - II Stimulant Levels Sour
Slide 22: Mazindol CR Well-Tolerated in Phase 2 Study
Mazindol CR Well - Tolerated in Phase 2 Study Source: Wigal , T. L., Newcorn , J. H., Handal , N., Wigal , S. B., Mulligan, I., Schmith , V., & Konofal , E. (2018). A Double - Blind, Placebo - Controlled, Phase II Study to Determine the Efficacy, Safety, Tolerability and Pharmacokine tics of a Contr
Slide 23: Nolazol – Highly Differentiated vs. Leading C-II Stimulants
Nolazol ® – Highly Differentiated on Major Attributes vs. Leading C - II Stimulants and Recent Launches Source: Adler LA, et al. J Clin Psychiatry. 2008;69(9):1364 - 1373.. 2 Frick G, et al. J Atten Disord . 2017.Epub ahead of print. 3. https:// www.medscape.com / viewarticle /576358#vp_2 *Based on
Slide 24: Expert quote: Professor J. Newcorn
“ We have not seen any potential treatment for ADHD that had effects as robust as we saw in the Phase 2 adult study , except for C - II stimulants . The prospect of a medication with efficacy as high as is seen with the C - II stimulants that would be a DEA Schedule IV drug would be most welcome and
Slide 25: NLS Pharmaceutical Clinical Development Pipeline
NLS Pharmaceutical Clinical Development Pipeline ADHD = Attention - Deficit/ Hyperactivity Disorder ; IH = Idiopathic Hypersomnia Product Candidate Candidate Selection Preclinical IND Enabling Studies Phase 1 Phase 2a Phase 2/3 Pivotals NDA Filing Quilience ® ( mazindol ER) Nolazol ® ( mazindol CR)
Slide 26: Overview of Pipeline & Earlier-Stage Projects
Overview of Pipeline & Earlier - Stage Projects 26
Slide 27: Anticipated Milestones and Corporate Priorities
Anticipated Milestones and Corporate Priorities Accepted 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 Interim topline
Slide 28: Thank you
System Therapies Thank you. 28
Slide 29: Contact details
Alter Postplatz 2 CH - 6370 Stans NW Switzerland +41(0) 41 618 80 00 www.nlspharma.com 29
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