Cns Pharmaceuticals, Inc. · NASDAQ:CNSP
Cns Pharmaceuticals — Investor Day (December 2024)
44 slides from Cns Pharmaceuticals's investor day presentation, Dec 11, 2024
Slide 1: Virtual Analyst & Investor Day
1 NASDAQ: CNSP December 11, 2024 Virtual Analyst & Investor Day
Slide 2: Forward Looking Statements
2 Forward Looking Statements This presentation incorporates information from materials filed with the SEC and contains forward - looking statements. All statements contained herein other than statements of historical fact, including statements regarding our future results of operations and financial
Slide 3: Presenting management team
3 John M. Climaco, Esq President & Chief Executive Officer Sandra L. Silberman, MD, PhD Chief Medical Officer Zena Muzyczenko Vice President, Clinical Operations
Slide 4: Agenda
4 Agenda • Opening and Introductions • CNS Pharma Overview • Glioblastoma Multiforme: The Patient Journey and Unmet Need • The Blood Brain Barrier and Overcoming This Significant Challenge • Berubicin: A Novel Anthracycline Designed to Cross the Blood Brain Barrier • Berubicin: Clinical Data and Pat
Slide 5: Prof. Michael Weller, MD
5 Prof. Michael Weller, MD Director of Department of Neurology, Universitatsspital, Zurich, Switzerland since 2008 Member of Swiss Neurological Society, German Society for Neurology, German Cancer Society, European Association for Neuro - Oncology, Society for Neuro - Oncology, European Society
Slide 6: Samuel Goldlust, MD
Samuel Goldlust, MD 6 B oard - certified and fellowship - trained neuro oncologist with Saint Luke’s Cancer Specialists Specialized training in neuro - oncology and extensive experience treating patients with primary and metastatic brain tumors as well as the neurological complications of cancer
Slide 7: Erin Dunbar, MD
7 Erin Dunbar, MD Founding physician of the Brain Tumor Center and Director of Neuro - Oncology at Piedmont Atlanta Hospital, Piedmont Atlanta, GA She specializes in the comprehensive care of brain and spine tumor patients who are battling both primary and metastatic tumors Completed fellowshi
Slide 8: CNS Pharma Overview
8 CNS Pharma Overview John M. Climaco, Esq President & Chief Executive Officer
Slide 9: Overview
Overview Lead Program: Berubicin, a Novel Anthracycline • First drug of its class to appear to cross the blood - brain barrier • A clinical trial designed to be pivotal now f u l ly enrolled • The primary analysis of data in the 1 st half of 2025 • No evidence of cardiotoxicity in hundreds of patien
Slide 10: A Focused and Targeted CNS Oncology Pipeline
A Focused and Targeted CNS Oncology Pipeline 10 Highlights Phase 3 Phase 2 Phase 1 Preclinical Indication Program • Study fully enrolled • Primary analysis data expected H1 2025 Glioblastoma Multiforme (GBM) Berubicin • Recently in - licensed • Plan to engage with regulators to design potential regi
Slide 11: Proven Execution and Milestones
11 Proven Execution and Milestones June 2020 Dec 2020 May 2021 June 2021 Sept 2021 April 2022 April 2022 April 2022 Sept 2022 Sept 2023 Oct 2023 Dec 2023 Jan 2024 Berubicin Development Program Orphan Drug Designation IND Accepted for Berubicin Initiated Potentially Pivotal Global Berubicin Trial for
Slide 12: Glioblastoma Multiforme: The Patient Journey
12 Glioblastoma Multiforme: The Patient Journey Prof. Michael Weller, MD Samuel Goldlust, MD Erin Dunbar, MD
Slide 13: Glioblastoma Multiforme (GBM)
Glioblastoma Multiforme (GBM) 13 1: https://braintumor.org/take - action/about - gbm/ 2: 8 Major Markets includes USA, France, Germany, Italy, Spain, UK, Japan and urban China 3: Global Data, “Glioblastoma Multiforme (GBM): Opportunity Analysis and Forecasts to 2027” (2017) One of the most aggressiv
Slide 14: GBM: Treatment
GBM: Treatment 14 Treatment depends upon the stage of disease, which may include: Craniotomy - a surgical procedure that involves removing a section of the skull to access the brain. The bone is replaced after surgery Resection of brain tumor or retrieve a sample of the brain tumor Temozolomide PCV
Slide 15: GBM Current Standard of Care
GBM Current Standard of Care 15 *Europe does not usually administer any drugs after the primary diagnosis in patients with an unmethylated MGMT promotor; the US usually provides Temozolomide to all patients Comments Europe US Standard of Care First Line X X Resection and Radiotherapy First Line* X X
Slide 16: The Blood Brain Barrier and Overcoming This Significant Challenge
16 The Blood Brain Barrier and Overcoming This Significant Challenge Sandra L. Silberman, MD, PhD Chief Medical Officer
Slide 17: The Blood Brain Barrier (BBB)
17 The Blood Brain Barrier (BBB) Key Functions • Protection: • Blocks toxins, pathogens and potentially harmful molecules from entering the brain by transporters that bind to these substances and deliver them back to the bloodstream • Selective Permeability : • Allows essential nutrients like glucos
Slide 18: Strategies for Therapies to Gain Access through the BBB
18 Strategies for Therapies to Gain Access through the BBB Liposomal Formulations • Resemble the lipid bilayer of the endothelial cell membrane • Can circumvent mechanisms that transport drugs out of the brain and that confer resistance to tumor cells Intratumoral/Intracerebral or Intrathecal Admini
Slide 19: Berubicin: A Novel Anthracycline Designed to Cross the Blood Brain Barrier
Berubicin: A Novel Anthracycline Designed to Cross the Blood Brain Barrier 19 Molecule derived from the structure of other anthracyclines Structure • Discovered due to molecular modeling and structure - based rational design through computational chemistry • Chosen based on its ability to circumvent
Slide 20: Berubicin Increases Survival in Mouse Glioma Model
20 Berubicin Increases Survival in Mouse Glioma Model • N ude mice ( nu/nu ) were seeded with U - 87 MG cells by direct intracerebral injection • Implanted mice were treated with two doses of Berubicin by intraperitoneal (i.p.) injection at 10 mg/kg on Day 1 and 5 mg/kg on Day 10 • U - 87 MG tumor -
Slide 21: Berubicin: First-In-Human Trial
21 Berubicin: First - In - Human Trial Sandra L. Silberman, MD, PhD Chief Medical Officer
Slide 22: Berubicin: First-In-Human Trial Design
Berubicin: First - In - Human Trial Design 22 with recurrent or refractory glioblastoma multiforme (GBM) or other primary brain cancers 29 were GBM, 4 AO and 2 AA 35 Subjects DOSE Intravenous berubicin over 2 hours for 3 consecutive days (one course) every 21 days Doses were escalated using an accel
Slide 23: Berubicin: Results of Phase 1 Dose-Finding and PK Study
23 Berubicin: Results of Phase 1 Dose - Finding and PK Study 44% of subjects demonstrated “stable disease or better” Two responses with up to 80% tumor shrinkage Extremely well tolerated with a good safety profile (no off - target toxicities) DURABLE COMPLETE RESPONSE (CR) - One subject remains canc
Slide 24: Berubicin: Clinical Data Generated to Date
24 Berubicin: Clinical Data Generated to Date Prof. Michael Weller, MD Samuel Goldlust, MD Erin Dunbar, MD
Slide 25: Established Clinical Global Infrastructure and Network
25 Established Clinical Global Infrastructure and Network Zena Muzyczenko Vice President, Clinical Operations
Slide 26: Clinical Development Infrastructure Optimized for Brain Cancer Drug Development
Clinical Development Infrastructure Optimized for Brain Cancer Drug Development 26 • Deliberate establishment of a global, CNS focused network • Commitment to work in this disease • Deep understanding of the landscape of clinical trials in GBM • Seamless transition to our next asset • Built to last
Slide 27: Berubicin: Designed-to-Be-Pivotal Clinical Trial and Path to Potential Approval
27 Berubicin: Designed - to - Be - Pivotal Clinical Trial and Path to Potential Approval Sandra L. Silberman, MD, PhD Chief Medical Officer
Slide 28: Primary Analysis from Trial Expected 1st Half 2025
Primary Analysis from Trial Expected 1 st Half 2025 28 Announced Independent Data Safety Monitoring Board (DSMB) Recommendation on 12/18/23 Continuation of Clinical Trial of Berubicin Without Modification x 45 centers in 5 countries x 252 patients randomized x Pivotal endpoint 6 - 12 months Berubici
Slide 29: Interim Analysis and Rationale
29 Interim Analysis and Rationale • Independent Data Safety Monitoring Board (DSMB) • Subject matter experts (oncologists, statistician) • Independently oversaw and monitored the clinical trial • Ensured the safety of the patients and the integrity of the data collected • DSMB made informed decision
Slide 30: Berubicin: Next Major Milestone
Berubicin: Next Major Milestone 30 Primary Analysis Planned H1 2025 x Study enrollment c omplete Patients on treatment and in survival follow - up continue unchanged Meeting with FDA to discuss the program based on the primary analysis Potential approval
Slide 31: Berubicin: Potentially Pivotal Clinical Trial
31 Berubicin: Potentially Pivotal Clinical Trial Prof. Michael Weller, MD Samuel Goldlust, MD Erin Dunbar, MD
Slide 32: TPI 287: Novel Blood Brain Barrier Permeable Abeotaxane
32 TPI 287 Late Stage, Novel Blood Brain Barrier Permeable Abeotaxane for Treatment of Brain Malignancies Sandra L. Silberman, MD, PhD Chief Medical Officer
Slide 33: TPI 287: A Novel Taxane Derivative
33 TPI 287: A Novel Taxane Derivative TPI 287 Paclitaxel • Taxanes • A class of chemotherapy that binds to microtubules and prevents them from breaking down normally, which stops cancer cells from dividing • A substrate for P - glycoprotein (Pgp), which is upregulated in cells that become taxane - r
Slide 34: Readily Penetrates the Blood Brain Barrier in Animal Models
34 Readily Penetrates the Blood Brain Barrier in Animal Models
Slide 35: Clinical Trials with TPI 287
Clinical Trials with TPI 287 35 Evaluated in multiple Phase 1 and Phase 2 studies Engaging with regulators to advance into registrational study Fast Track Designation Orphan Drug Designation with 7 years market exclusivity in the U.S. ✓ ✓ ✓ ✓
Slide 36: TPI 287 Phase 2 trial with bevacizumab in recurrent glioblastoma
TPI 287 36 Samuel Goldlust, MD Phase 1 trial of TPI 287, a microtubule stabilizing agent, in combination with bevacizumab in adults with recurrent glioblastoma
Slide 37: TPI 287 in Combination with Bevacizumab for Recurrent Glioblastoma
TPI 287 in Combination with Bevacizumab for the Treatment of Recurrent Glioblastoma 37 Progression - Free Survival Overall Survival Median OS: 13.4 mo [95% C.I. 10.9, 17.9] 12 - month OS: 64% Median PFS: 5.5 mo [95% C.I. 4.1, 8.2] 6 - month PFS: 40%
Slide 38: Improved GBM Survival in Combination with Bevacizumab
38 * Graphs represent aggregate data from multiple studies Improved GBM Survival in Combination with Bevacizumab
Slide 39: TPI 287: Late Stage, Novel Blood Brain Barrier Permeable Abeotaxane
39 TPI 287: Late Stage, Novel Blood Brain Barrier Permeable Abeotaxane for Treatment of Brain Malignancies Prof. Michael Weller, MD Samuel Goldlust, MD Erin Dunbar, MD
Slide 40: CNS Pharma Positioned for a Transformational 2025
40 CNS Pharma Positioned for a Transformational 2025 John M. Climaco, Esq President & Chief Executive Officer
Slide 41: Commercial Planning
Commercial Planning 41 Key Appointment of Amy Mahery to Board of Directors Commercial Strategy Under Development Focus on Hiring the Right People Medical Affairs/Medical Liaisons Product Launch & Commercialization Specialist
Slide 42: 2025 Milestones
2025 Milestones 42 Berubicin Primary Analysis H1 2025 Pediatric Pre - Clinical Studies with Berubicin Pre - Clinical Studies with Berubicin Meetings with FDA to Review Strategies for Berubicin and TPI 287 Contract with Key Vendors to Launch TPI 287 Study Transfer of TPI 287 INDs TPI 287 Site Identif
Slide 43: Q&A
Q&A
Slide 44: Investor & Media Relations
44 NASDAQ: CNSP Investor & Media Relations: JTC Team, LLC Jenene Thomas 908.824.0775 CNSP@JTCIR.com