Panbela Therapeutics, Inc. · OTC:PBLA
Panbela Therapeutics — Investor Presentation (May 2024)
34 slides from Panbela Therapeutics's investor presentation, May 20, 2024
Slide 1: Investor Presentation
Investor Presentation May 2024 Last modified May 20, 2024 Panbela Therapeutics
Slide 2: Disclaimers
Disclaimers Certain statements in this presentation are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and are provided under the protection of the safe harbor for forward-looking statements provided by that Act. Forward-looking statements are b
Slide 3: Company Presents A Unique Opportunity: Multi-Targeted Approach to Reset Dysregulated Biology
Company Presents A Unique Opportunity: Multi-Targeted Approach to Reset Dysregulated Biology Scientific focus on polyamines, key regulators of normal biology altered in many disease states 1 Company focus on polyamine targeted platform spanning multiple indications Polyamine targeted therapy est
Slide 4: Highly Experienced Management Team with Proven Track Record
Highly Experienced Management Team with Proven Track Record Proven orphan and oncology drug discovery, development and commercialization expertise Dr. Jennifer Simpson Sue Horvath Dr. Ashok Chavan Rachel Bragg Chief Executive Officer Chief Financial Officer VP, CMC, QA, Supply Chain VP, Clinical Dev
Slide 5: Dysregulation of the Polyamine Pathway Leads to Disease
Dysregulation of the Polyamine Pathway Leads to Disease Polyamine Dysregulation in Disease Cancer Autoimmune Diseases Pancreatic ductal adenocarcinoma Recent Onset Type 1 Diabetes Familial Adenomatous Polyposis (FAP) Colorectal Ovarian STK11 Mutant NSCLC Prostate Neuroblastoma* Initial focus on onco
Slide 6: Pipeline: Addresses Multiple Unmet Needs
Pipeline: Addresses Multiple Unmet Needs Preclinical IND Ready Phase I Phase II Phase III Milestones Phase III Enrolling; Interim PDA (First Line Metastatic) ASPIRE TRIAL Analysis Expected Q1 2025 (ivospemin) (Injection) SBP-101 PDA Neoadjuvant Phase II Ready – Open 2H 2024 Ovarian Phase I Rea
Slide 7: Potential $5B Market Opportunity for Lead Programs
Potential $5B Market Opportunity for Lead Programs Pancreatic Cancer FAP Colorectal Cancer Ovarian Cancer Incidence of Incidence of Incidence of Incidence of Pancreatic Cancer FAP Colorectal Cancer Ovarian Cancer 64K in US ~30K in US ~150K in US ~22K in US ~151K in EU ~50K in EU ~340K in EU ~79K in
Slide 8: Complementary Pharmacotherapies Targeting Dysregulation
Complementary Pharmacotherapies Targeting Dysregulation Pipeline Objective Combined pipeline pharmacotherapies hit different targets in the polyamine pathway The objective of Panbela’s pipeline is the utilization of pharmacotherapies to reduce/normalize increased disease- CPP-1X-T & CPP-1X-S associa
Slide 9: Ivospemin (SBP-101)
Ivospemin (SBP-101) Panbela Therapeutics 9
Slide 10: Preliminary Efficacy of ivospemin (SBP-101) + Gemcitabine/Nab-paclitaxel in First-Line Metastatic PDA
Preliminary Efficacy of ivospemin (SBP-101) + Gemcitabine/Nab-paclitaxel in First-Line Metastatic Pancreatic Ductal Adenocarcinoma BEST OVERALL RESPONSE Overall Disease CR PR SD PD Response Control ivospemin (0.40 mg/kg) + G/A* 14/29 24/29 1 (3%) 13 (45%) 10 (34%) 5 (17%) (Ph Ia COHORT 4 + Ph 1b) n=
Slide 11: Phase I Trial Efficacy of SBP-101 + Gemcitabine/Nab-paclitaxel
Phase I Trial Efficacy of SBP-101 +Gemcitabine/Nab-paclitaxel Progression-Free Survival Overall Survival (PFS) (OS) Median PFS=6.5 months Median OS=14.8 months • Cumulative survival rates at 6 and 12 months were 86.2% and 55.2%. with 3 subjects (10.3%) alive at 19.6, 20.1, and 22.9 months. • The cum
Slide 12: Safety Results
Ivospemin (SBP-101) + Gemcitabine/Nab-paclitaxel Demonstrated a Similar Safety Profile to Gemcitabine/Nab-paclitaxel in First-Line Metastatic Pancreatic Ductal Adenocarcinoma Safety Results SBP- Grade ≥3 AEs of Special Interest N G+A %** Grade ≥3 adverse events attributable to any study medication,
Slide 13: Phase III Pancreatic Cancer Trial in Previously Untreated Metastatic PDA
Phase III Pancreatic Cancer Trial in Subjects Previously Untreated for Metastatic Pancreatic Ductal Adenocarcinoma A Randomized, Double-Blind, Placebo Controlled Study of Nab-Paclitaxel and Gemcitabine with or Without ivospemin (SBP-101) OS-Overall Survival PFS-Progression Free Survival ORR – Overal
Slide 14: Efficacy Comparison of ivospemin + Gemcitabine/Nab-paclitaxel vs. Standard of Care
Efficacy Comparison of ivospemin (SBP-101) + Gemcitabine/Nab-paclitaxel vs. Standard of Care Treatment OS PFS ORR DCR PD CL-SBP-101-031 14.6 months 6.5 months 48.3% 82.8% 4.8% FOLFIRINOX2 11.1 months 6.4 months 31.6% 70.2% 15.2% Modified FOLFIRINOX3 10.2 months 6.1 months 35.1% 86.5% 13.5% NALIRIFOX
Slide 15: Summary of First Line Metastatic Pancreatic Cancer FDA Approvals
Summary of First Line Metastatic Pancreatic Cancer FDA Approvals MPACT Trial Gemcitabine + Abraxane vs Gemcitabine N=831 Median OS Benefit: 1.8 months (G+A 8.5 months vs Gemcitabine 6.7 months) FDA Approval 2013– Gemcitabine + Abraxane NAPOLI 3 Trial liposomal irinotecan in combination w
Slide 16: FLYNPOVI: A Combination of CPP-1X and Sulindac
FLYNPOVI: A Combination of CPP-1X and Sulindac Panbela Therapeutics 16
Slide 17: Familial Adenomatous Polyposis (FAP): Summary of Approach to Approval
Familial Adenomatous Polyposis (FAP): Summary of Approach to Approval Strong Results in the Lower GI Focus on Lower GI: statistically significant p-values and hazard ratios for clinically meaningful surgical endpoints—emphasized by regulators—in Phase III pivotal trial No surgeries in the lower
Slide 18: CPP-1X-S
CPP-1X-S Panbela Therapeutics 18
Slide 19: Targeting Type 1 Diabetes Using POLyamines (TADPOL)
Targeting Type 1 Diabetes Using POLyamines (TADPOL) A Randomized, Double-Masked, Placebo-Controlled Phase 2 Study Evaluate the Efficacy and Safety of DFMO (eflornithine) to Preserve Insulin Production in Type 1 Diabetes N=70 Multicenter, led by Indiana University School of Medicine Funded by
Slide 20: Polyamines & Immune Dysregulation
Polyamines & Immune Dysregulation Panbela Therapeutics 20
Slide 21: Role of Polyamines in Immune Dysregulation
Role of Polyamines in Immune Dysregulation Immune systems require multiple soluble and cellular components, including polyamines, for a normal immune function High levels of polyamines are present in tumor cells and in autoreactive B- and T-cells in autoimmune diseases Dysregulation of polyami
Slide 22: Potential Role of Polyamines in DLBCL CAR-T Therapy
Potential Role of Polyamines in Diffuse Large B Cell Lymphoma (DLBCL) CAR-T Therapy Polyamine metabolism upregulation through oncogenic MYC is a common metabolic irregularity in aggressive cancers, including lymphomas. MYC overexpression in relapsed/refractory DLBCL prior to CAR-T cell therapy n
Slide 23: Rationale for Eflornithine/anti-PD-1 Combination
Rationale for Eflornithine/anti-PD-1 Combination Targeting Ornithine decarboxylase (ODC) using eflornithine unmasks antitumor T cell immune response by weakening of tumor-induced immune suppression that is mediated by Myeloid- Derived Tumor Suppressor Cells (MDSCs). The addition of anti-PD-1 pre
Slide 24: Rationale for CPP-1X-S (Eflornithine Sachets) in STK11 Mutant NSCLC
Rationale for CPP-1X-S (Eflornithine Sachets) in STK11 Mutant NSCLC In preclinical tumor models, eflornithine treatment improves anti-PD-1 efficacy by: Increasing tumor-specific cytotoxic T-cell populations Increasing expression of PD-1 on tumor-associated CD8+ T cells ~10% of NSCLC cancers
Slide 25: STK11 Mutant NSCLC Investigator-Initiated Trial
STK11 Mutant NSCLC Investigator-Initiated Trial Collaborating with investigators at Moffitt Cancer Center on Phase I/II trial: “Targeting Ornithine Decarboxylase as an Immunotherapeutic Target in STK11 (LKB1) Pathway-Deficient Non-Small Cell Lung Cancer” Patient Population Phase I Phase II Stage I
Slide 26: USWorld Medicines Neuroblastoma Program
USWorld Medicines Neuroblastoma Program Panbela Therapeutics 26
Slide 27: Anticipated Milestones from USWM Neuroblastoma Program
Anticipated Milestones from USWM Neuroblastoma Program FDA granted approval to eflornithine (IWILFIN) as a treatment for adult and pediatric patients with high-risk neuroblastoma (HRNB) who have achieved a partial response or better to a previous, multiagent, multimodal treatment including an anti-G
Slide 28: Business Overview
Business Overview Panbela Therapeutics 28
Slide 29: Barriers to Entry – Flynpovi/CPP-1X
Barriers to Entry – Flynpovi/CPP-1X Orphan status (7 years exclusivity US; 10 years exclusivity EU) for CPP-1X ◦ Granted in US and EU for FAP ◦ Granted in US for Gastric cancer and Pancreatic cancer Pharmaceutical Composition Patent ◦ Fixed dose combination eflornithine + sulindac ◦ Broadly nati
Slide 30: Barriers to Entry – ivospemin (SBP-101)
Barriers to Entry – ivospemin (SBP-101) Orphan status (7 for years exclusivity US; 10 years exclusivity EU) for SPB-101 ◦ Granted in US and EU for pancreatic cancer METHODS FOR PRODUCING (6S,15S)-3,8,13,18-TETRAAZAICOSANE-6,15-DIOL ◦ PCT application filed January 29, 2019 ◦ Granted in United Sta
Slide 31: Summary of Milestones
Summary of Milestones 1H 2024 FPI NSCLC Phase 1 Trial Gastric Cancer Prevention Phase II Results 2H 2024 • Open- Neoadjuvant Pancreatic Cancer Trial • Open Phase 1 Ovarian Trial • Obtain feedback from FDA and EMA for Global Registration Program in FAP • Publication of Final Phase 1 Metastatic Pa
Slide 32: Financial Position - Cash and Debt (unaudited)
Financial Position - Cash and Debt (unaudited) Cash and Debt Cash at 3/31/2024 262 Cash received after 3/31/2024 775 Proforma net cash as of 3/31/2024 1,037 Debt at 3/31/2024 Current (Due January 31, 2025) 1,000,000 Non-current 3,194,000 Total 4,194,000 Panbela Therapeutics 32
Slide 33: Capital Stock (unaudited)
Capital Stock (unaudited) Panbela Common Stock issued and outstanding at 3/31/2024 4,854,861 Shares reserved for Options (WAE = $14,410.38) at 3/31/2024 607 Shares reserved for Warrants (WAE = $3.13) at 3/31/2024 9.095.943 Total Proforma Outstanding and Reserved 13,951,411 Panbela Therapeutics 33
Slide 34: Summary
Summary: Unique Scientific Approach: Resetting dysregulation via polyamine pathway Broad, late-stage de-risked pipeline opportunities Multiple partnerships supporting development programs Significant market potential Multiple data readouts expected Experienced management team Panbela Therapeutics 34
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