Newron Pharmaceuticals S.p.a. · SIX:NWRN

Newron Pharmaceuticals — German Spring Conference 2024

24 slides from Newron Pharmaceuticals's conference presentation, May 13, 2024

  1. Slide 1: Committed to CNS Drug Development

    COMMITTED TO CNS DRUG DEVELOPMENT INNOVATIVE TREATMENTS TO IMPROVE QUALITY OF LIFE Stefan Weber, CEO German Spring Conference May 13, 2024

  2. Slide 2: Disclaimer

    DISCLAIMER RESTRICTED SCOPE; EXCLUSION OF LIABILITY; CONFIDENTIALITY This document has been prepared by Newron Pharmaceuticals S.p.A. ("Newron") solely for your information. The information contained herein has not been independently verified. No representation or warranty, express or implied, is ma

  3. Slide 3: Company Highlights

    COMPANY HIGHLIGHTS Unique portfolio of innovative Management team with extensive CNS product candidates experience and proven track record  Xadago® for Parkinson’s disease – Global in drug development and approvals validate Newron’s development commercialization capabilities from research to market

  4. Slide 4: Evenamide – Changing the Treatment Paradigm in Schizophrenia

    EVENAMIDE – CHANGING THE TREATMENT PARADIGM IN SCHIZOPHRENIA  Large market opportunity  TRS patients: Positive results from 1-year pilot  Differentiated MoA and positioning study 014/015 in 161 TRS patients − First add-on drug  NON-TRS patients: Positive results from pivotal − Changes a non-resp

  5. Slide 5: High Medical Need for 20 Million Patients Worldwide

    HIGH MEDICAL NEED FOR 20 MILLION PATIENTS WORLDWIDE Over 30 antipsychotics available, but all provide short-term and LARGE MARKET insufficient relief of some of the symptoms OPPORTUNITY (anti-psychotics Most patients with schizophrenia demonstrate reduced control of positive market >$23bn) symptoms

  6. Slide 6: Evenamide's Differentiated MoA Demonstrated

    EVENAMIDE’S DIFFERENTIATED MOA DEMONSTRATED Selectively blocks native sodium channels, showing no off-target effect on >130 other CNS receptors, enzymes, transporters, etc. Modulates sustained repetitive firing Selectively blocks VGSCs in a Inhibits without inducing impairment of voltage-and use-dep

  7. Slide 7: Evenamide – Differentiation and Commercial Opportunity in Schizophrenia

    EVENAMIDE – DIFFERENTIATION AND COMMERCIAL OPPORTUNITY IN SCHIZOPHRENIA Large market opportunity NO direct competition as evenamide can be added to all antipsychotics Seeking to change treatment paradigm in schizophrenia First add-on antipsychotic to be First drug for treatment of Only option as add

  8. Slide 8: Evenamide First Potential Breakthrough for TRS Patients in Over 30 Years

    EVENAMIDE  Differentiated – structure, pharmacology, benign side effect profile, indication (Add-on to any antipsychotic for TRS patients), NO competitor identified FIRST POTENTIAL  Evenamide treatment of 161 TRS patients prevented ANY psychotic relapse BREAKTHROUGH or hospitalization FOR TRS PATI

  9. Slide 9: Pilot Study 014/015: Design and Key Characteristics

    PILOT STUDY 014/015: DESIGN AND KEY CHARACTERISTICS Study design: Study Population: A pilot, randomized, open-label, rater-blinded, parallel-group, 6 weeks, multi-center study followed by an extension up to  Treatment-Resistance with documented non-response to at 1 year of treatment with evenamide

  10. Slide 10: Study 014/015 – Patient Disposition by Study and Duration

    STUDY 014/015 – PATIENT DISPOSITION BY STUDY AND DURATION Randomized Completed Discontinued Completed Completed Total Discontinued 31 N = 161 N = 153 N=8 N = 132 N = 121 Withdrawal of consent 23 Lost to follow-up 5 Entered Did not enter Discontinued Discontinued N = 12 N = 11 Adverse event 2 Extensi

  11. Slide 11: Study 015 – Positive and Negative Syndrome Scale (PANSS)

    STUDY 015 – POSITIVE AND NEGATIVE SYNDROME SCALE (PANSS) MEAN CHANGE FROM BASELINE (SD) – mITT % Change from baseline 79.5 (5.04) * 70.0 * -9.5 (7.13) 66.5 * -11.9% -12.8 (7.65) 63.9 -16.1% -15.5 (10.18) -19.4% Baseline Week-6 6-Month 1-Year N=156 N=152 N=131 N=127 * p-value vs baseline < 0.001, pai

  12. Slide 12: Study 015 – PANSS Responder Analysis

    STUDY 015 – POSITIVE AND NEGATIVE SYNDROME SCALE (PANSS) PANSS RESPONDER ANALYSIS (%) – mITT PANSS Total ≥20% Improvement from baseline 84,6 Responder Non Responder 66,0 58,2 41,8 34,0 15,4 Week-6 6-Month 1-Year 12

  13. Slide 13: Patients No Longer Meeting Severity Criteria for TRS

    PATIENTS NO LONGER MEETING SEVERITY CRITERIA FOR TRS (mITT; LOCF/OC) SEVERITY CRITERIA VISIT WEEK 6 6-MONTH 1-YEAR STAT LOCF OC LOCF OC LOCF OC N 156 152 156 131 156 120 1. PANSS <70 n (%) 72 (46.1) 72 (47.3) 93 (59.6) 84 (64.1) 99 (63.5) 84 (70.0) 2. Core items* <20 n (%) 60 (38.4) 60 (39.4) 83 (53

  14. Slide 14: Study 014/015 – Proportion of Patients Who Meet Proposed Remission Criteria

    STUDY 014/015 – PROPORTION OF PATIENTS WHO MEET PROPOSED REMISSION CRITERIA N=156 Maintenance Method Criteria n (%) of patients meeting requirement remission criteria P1, P2, P3, P6, G5 ≤ 3 Lieberman et al, 1993 CGI-S «mildly ill»; 8 weeks 43 (27.6%) CGI-C «much improved» Andreasen et al, 2005 P1, P

  15. Slide 15: Evenamide: First Evidence of Benefit of Add-on Therapy in Inadequate Responders

    EVENAMIDE FIRST EVIDENCE OF EVENAMIDE ADD-ON TREATMENT IN PATIENTS WITH CHRONIC BENEFIT OF ADD- SCHIZOPHRENIA NOT RESPONDING ADEQUATELY TO THEIR ON THERAPY IN CURRENT ANTIPSYCHOTIC INADEQUATE RESPONDERS AN INTERNATIONAL, RANDOMIZED, DOUBLE-BLIND, PLACEBO- CONTROLLED TRIAL FIRST SELECTIVE PIVOTAL STU

  16. Slide 16: Design and Key Features

    DESIGN AND KEY FEATURES A potentially pivotal, phase II/III, 4-week, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, of evenamide 30 mg bid vs placebo in patients who are inadequate responders to SGAs Sample size: 291 patients randomized in a 1:1 ra

  17. Slide 17: Design and Key Features

    DESIGN AND KEY FEATURES Safety Day 0/1 Day 29 Follow up 21-day Screening Period Randomization Day 8 Day 15 Day 22 Endpoint (+ 30 days) Evenamide (30 mg bid) Antipsychotic plasma levels to confirm adherence Eligibility Process Placebo Patients receiving second–generation antipsychotics (APs) at thera

  18. Slide 18: Patient Disposition

    PATIENT DISPOSITION Evenamide 30 mg bid Placebo Overall n (%) n (%) n (%) Screened - - 428 Randomized 132 159 291 ITT/Safety population 132 (100) 159 (100) 291 (100) mITT population 131 (99.2) 156 (98.1) 287 (98.6) Discontinued 6 (4.5) 5 (3.1) 11 (3.8) Withdrawal of consent 4 (3.0) 4 (2.5) 8 (2.7) A

  19. Slide 19: Safety: Overall Summary of TEAEs

    SAFETY: OVERALL SUMMARY OF TEAEs Evenamide 30 mg bid Placebo Overall Subject N=132; n (%) N=159; n (%) N=291; n (%) With at least one TEAE 33 (25.0) 41 (25.8) 74 (25.4) Mild 22 (16.7) 30 (18.9) 52 (17.9) Moderate 11 (8.3) 10 (6.3) 21 (7.2) Severe 0 (0.0) 1 (0.6) 1 (0.3) With adverse event leading to

  20. Slide 20: Primary, Key Secondary Efficacy Endpoint – ITT Population

    PRIMARY, KEY SECONDARY EFFICACY ENDPOINT – ITT POPULATION PRIMARY ESTIMAND – TREATMENT POLICY, MEAN CHANGE FROM BL – DAY 29 Evenamide 30 mg bid Placebo Scale Visit N=132 N=159 Baseline – mean (SD) 78.4 (4.1) 78.7 (4.0) Day 29 – LS mean (SE) -10.2 (0.7) -7.6 (0.7) PANSS total score LS mean difference

  21. Slide 21: Key Findings from Study 008A

    KEY FINDINGS FROM STUDY 008A Study 008A results indicate that the primary (PANSS) and the key secondary (CGI-S) endpoints required by regulators for a pivotal study were met and showed significant superiority compared with the benefits observed in patients on placebo Benefit with evenamide appears t

  22. Slide 22: Phase III TRS Study – Design

    PHASE III TRS STUDY – DESIGN* Baseline Day 0 ICF Eligibility Randomization 12-week 26-week 52-week Screening – 42 days Double-blind treatment of 52 weeks Open-Label Extension ELIGIBILITY PIVOTAL STUDY MAINTENANCE LONG-TERM AP PLASMA LEVELS ENDPOINT ENDPOINT EFFICACY Primary Efficacy Second (long-ter

  23. Slide 23: Xadago in Parkinson's Disease – Marketed Compound

    XADAGO IN PARKINSON’S DISEASE MARKETED COMPOUND First approved NCE in >10 yrs (U.S., EU) Globally licensed Double digit/single digit % royalties >€80 million milestones/royalties collected 23

  24. Slide 24: Contact Details

    CONTACT DETAILS NEWRON Stefan Weber – CEO +39 02 6103 46 26 pr@newron.com UK/Europe Germany/Europe Simon Conway, Ciara Martin Anne Hennecke/Caroline Bergmann, FTI Consulting MC Services +44 20 3727 1000 +49 211 52925220 SCnewron@fticonsulting.com newron@mc-services.eu Switzerland USA Valentin Handsc

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