Newcelx Ltd. · NASDAQ:NCEL
Newcelx — NLS Pharmaceutics — Investor Presentation (April 2022)
30 slides from Newcelx's investor presentation, Apr 6, 2022
Slide 1: Investor Presentation | April 2022
Investor Presentation | April 2022 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 undergoing Phase 2 development for narcolepsy treatment: $3.9B (2021) annual market ** ⌾ Active ingredient in Quilience ® , mazindol, was used extensively in compassionate use off - label ⌾ Or
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 to get “deals done” . Served as Chief Operating Officer at Vifor
Slide 5: 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 6: 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 $3.9 billion in 2021, and are projected to reach $6.6 billion by 2025, a CAGR of 11.2%.** Significant valuations are being assigned to public compa
Slide 7: 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 8: Quilience key attributes
□ - 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 □ Phase 2a trial ongoing, very encouraging inter
Slide 9: Mazindol: Unique Dual Mechanism of Action (MOA)
Mazindol: Unique Dual Mechanism of Action (MOA) Quilience ® (mazindol ER) 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 10: 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 11: Quilience: Phase 2a Clinical Trial Overview
Quilience ® : Phase 2a Clinical Trial Overview 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 (ESS). Key observat
Slide 12: Polaris: Mazindol ER development program in Narcolepsy
Polaris: Mazindol ER development program in Narcolepsy 12 Polaris program currently represents two U.S. clinical trials both approved by FDA/WIRB □ NLS - 1021 Phase IIa A four - week double - blind, placebo controlled, randomized, U.S. multi - center study of Mazindol ER 3 mg once daily vs. placebo
Slide 13: NLS-1021 Study Design - Efficacy Assessments
NLS - 1021 Study Design - Efficacy Assessments 13 Efficacy Assessments: □ Primary endpoint: □ Epworth Sleepiness Scale (ESS) □ Secondary and exploratory endpoints: □ Cataplexy Diary (reduction in weekly cataplexy attacks) □ Clinician Global Impression of Severity (CGIs) □ Patient Global Impression o
Slide 14: NLS-1021 Phase IIa – Demographics
NLS - 1021 Phase IIa – Demographics Demographics NLS - 1021 Narcolepsy Type 1 34% Narcolepsy Type 2 66% Male 21% Female 79% Age 34,6 (19;52) Rolled over into OLE (NLS - 1022) 82% 14
Slide 15: NLS-1021 Phase IIa – Prior Medication before Wash-out
NLS - 1021 Phase IIa – Prior Medication before Wash - out Previous wake promoting medication alone or in combination before inclusion (number of patients) Amphetamines 12 Modafinil 9 Methylphenidate 5 Armodafinil 4 Sodium Oxybate 4 Solriamfetol 2 Pitolisant 2 None 3 15
Slide 16: NLS-1021 Phase IIa – Interim Analysis (FDA consent)
NLS - 1021 Phase IIa – Interim Analysis (FDA consent) 16 □ An interim analysis (IA) was conducted at ~50% of the total planned sample size that completed the Week 4 visit or discontinue from the study □ Purpose of the IA: to providing top line information on the clinical development. □ Actions: No a
Slide 17: Summary Interim Top-Line EFFICACY Data
Summary Interim Top - Line EFFICACY Data (World Sleep Congress 2022) □ Mean ESS 7.3 point reduction from baseline of 18.6 (best in class) □ Clinically meaningful improvement in ESS score at 4 weeks □ Rapid onset of action and clinical effect (within 1 week) even at 2mg dose □ Sustained EDS improveme
Slide 18: Primary Endpoint: Change from Baseline on Epworth Sleepiness Scale
Primary Endpoint: Change from Baseline to Last Visit on Epworth Sleepiness Scale (ESS) (ITT Population) Mean (+/ - Standard Error) Change from Baseline in Epworth Sleepiness Scale (ESS) Total Score by Visit Baseline Mazindol ER (N=13) Week 1 (Day 7) Week 2 (Day 14) Week 3 (Day 21) Week 4 (Day 28) Pl
Slide 19: Mazindol ER vs. Placebo Weekly Total ESS Score
Mazindol ER vs. Placebo Weekly Total ESS Score Visit Placebo, N=14 Mazindol ER, N=13 Baseline ESS Score 18.0 (2.72) 18.6 (2.81) Week 1 Day 7 2mg ESS score 15.9 (3.25) 14.6 (5.16) Difference from Baseline to W1 Mean - 2.1 (2.80) - 4.0 (3.87) Week 2 Day 14 3mg ESS score 15.6 (3.76) 13.7 (4.89) Differe
Slide 20: ESS Scores for Approved Narcolepsy Treatments and Mazindol ER
ESS Scores for Approved Narcolepsy Treatments and Mazindol ER Drug, Sponsor Dose Mean baseline ESS scores Mean ESS decrease vs baseline Treatment period Narcolepsy trial & No. of patients Solriamfetol (Sunosi; Jazz) ✶ 75 mg/day 17.3 - 3.8 12 weeks Pivotal trial, n=172 150 mg/day 17.0 - 5.4 12 weeks
Slide 21: NLS-1021 Phase IIa: Interim Analysis – Safety and Tolerability
NLS - 1021 Phase IIa : Interim Analysis – Safety and Tolerability Number of Adverse Events (AEs) per Patient (including both drug and placebo) Total Adverse events out of 27 patients 19 Patients with AE 12 Patients with 1 AE 10 Patients with 2 AEs 1 Patients with 3 AEs 1 Adverse Events Severity: Mil
Slide 22: Summary of Interim Top-Line SAFETY/TOLERABILITY Results
Summary of Interim Top - Line SAFETY/TOLERABILITY Results □ No patient discontinued treatment to due to adverse reactions □ All AEs resolved spontaneously, no intervention was required □ No serious adverse events or unexpected AEs □ Well - tolerated with the most commonly known adverse reactions occ
Slide 23: Quilience: Anticipated Development Timeline
2021 2022 2023 Q2 2021 Opened IND Sept. 2021 Initiated Phase IIa trial Q1 2022 Reported interim topline results at World Sleep 2022 3Q 2024 Results: Phase IIb /III Pivotal Trials 3Q - 2022 Data Readout for Phase IIa trial 1Q - 2023 Initiate Phase IIb/III Pivotal Trials 23 Q2 2022 Initiate Early Acce
Slide 24: 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 25: Positive Phase 2 Results for Nolazol 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 • Studies suggest ADHD and narcolepsy are genetically related with possible common unde
Slide 26: Phase 2 Trial Primary Endpoint Results – ADHD
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 27: 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 Pharmacokinetics of a Contro
Slide 28: Overview of Pipeline & Earlier-Stage Projects
Overview of Pipeline & Earlier - Stage Projects 28
Slide 29: Anticipated Milestones and Corporate Priorities
Anticipated Milestones and Corporate Priorities Final topline results of Phase 2a trial for Quilience ® in narcolepsy anticipated 3Q 2022 Potential for accelerated regulatory pathway designations (fast - track, break - through, PRIME) Open compassionate use program in Europe with potential for non -
Slide 30: Thank you.
System Therapies Thank you. 30
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