Paradigm Biopharmaceuticals Ltd. · ASX:PAR
Paradigm Biopharmaceuticals — Investor Presentation (March 2019)
38 slides from Paradigm Biopharmaceuticals's investor presentation, Mar 13, 2019
Slide 1: Investor Presentation
INVESTOR PRESENTATION Paul Rennie, CEO & MD March 2019
Slide 2: Disclaimer
DISCLAIMER This document, together with any information communicated by Paradigm Biopharmaceuticals Ltd (known as “Paradigm”, “Paradigm Biopharma” or “the Company”), in any presentation or discussion relating to this document (collectively, “Information”) is confidential, and has been prepared by th
Slide 3: Paradigm's Mission Statement
PARADIGM’S MISSION STATEMENT Paradigm’s aim is to develop and commercialise an ethical, safe and effective Pharmaceutical Agent (PPS) for the treatment of musculoskeletal disorders in humans with degenerative disease driven by injury, aging or genetic predisposition. In achieving this aim, Paradigm
Slide 4: Corporate Snapshot
CORPORATE SNAPSHOT § Paradigm Biopharmaceuticals Ltd (PAR.ASX) is an Top Shareholders Financial information Shares (m) % ASX-listed biotechnology company focused on Share price (08-March-2019) A$1.35 Paul Rennie (Managing Director) 21.6 15.4% repurposing Pentosan Polysulfate Sodium (PPS), Other Boar
Slide 5: Successful Re-purposed Drugs
SUCCESSFUL RE-PURPOSED DRUGS Re-purposed drugs have become true blockbusters BRAND ORIGINAL PHARMA PEAK ANNUAL NEW INDICATION NAME INDICATION COMPANY SALES SPRAVATO Anaesthetic (Ketamine) Treatment Resistant Depression Janssen/J&J Approved March 2019 Structural Analogue of REVLIMID Multiple Myeloma
Slide 6: Foundations for Significant Commercial Transaction
FOUNDATIONS FOR SIGNIFICANT COMMERCIAL TRANSACTION Paradigm is deal ready with all of the necessary ‘building blocks’ in place that a partnering pharmaceutical company would require to execute a transaction for OA DEAL FILE IND WITH FDA POSITIVE PHASE 2B DATA MECHANISM OF ACTION EFFICACY & SAFETY DA
Slide 7: Strong Patents & IP Position
STRONG PATENTS & IP POSITION Multi-faceted IP protection increases barriers to entry for potential competitors Valuable patent portfolio § Paradigm has patent protection because it is using PPS for new indications BMEL patent granted in EU patents granted for BMEL, respiratory diseases § Minimum lif
Slide 8: Exclusive Supply & Manufacturing
EXCLUSIVE SUPPLY & MANUFACTURING Exclusive Supply PPS Buy per/kg License Exclusivity Royalty § Paradigm has executed a 20 year exclusive supply § Agreement grants exclusive supply of only FDA agreement with bene pharmaChem Gmbh & Co. KG approved PPS for all orthopaedic (inc. alphavirus), § Bene phar
Slide 9: Market Demand – Osteoarthritis with BMEL
MARKET DEMAND – OSTEOARTHRITIS WITH BMEL Osteoarthritis - A blockbuster indication with no effective treatments § Blockbuster market - Osteoarthritis is the most common form of arthritis, affecting over 31 million people in the United States, with over 36 million outpatient visits and 750,000 hospit
Slide 10: Market Demand – OA Stages and Treatments
MARKET DEMAND – OA STAGES AND TREATMENTS There are no effective treatments for Moderate to Severe OA Effective Non-validated Ineffective Analgesics, NSAIDS, Corticosteroids Opioids *Platelet-rich Plasma, *Stem Cells, *Royal Australian College of General Practitioners hold a ‘strong against’ recommen
Slide 11: Efficacy & Safety in Humans – Positive SAS Data
EFFICACY & SAFETY IN HUMANS – POSITIVE SAS DATA TGA Special Access Scheme – Real World Evidence – 183 patients treated All 183 patients reported on (median age of 56.4 years - range 18 to 84 years) had pain and failed current standard of care - analgesics, NSAIDs or corticosteroids. At six weeks pos
Slide 12: Efficacy & Safety in Humans – Positive TGA SAS Data
EFFICACY & SAFETY IN HUMANS – POSITIVE TGA SAS DATA Pain (NRS) Before – After = 3.2 p<0.0001 Function (LKS) Before – After = 25.1 p<0.0001 51.4 % reduction in knee pain 60.8% improvement in knee function Pain (NRS 1-10) Function (LKS 1-100) 10 100 9 90 8 80 P<0.0001 7 P<0.0001 70 6 60 5 50 4 40 3 30
Slide 13: Mechanism of Action (MoA)
MECHANISM OF ACTION (MoA) New Mechanism of Action – PPS role of in reducing pain § Paradigm’s has conducted a non-clinical study to demonstrate the pain reduction MoA of PPS § The study is complete and the manuscript has been sent for peer-review and publication § The manuscript is entitled: “Human
Slide 14: Positive Phase 2b OA/BMEL Data
POSITIVE PHASE 2B OA/BMEL DATA In its Phase 2b clinical trial Paradigm demonstrated clinical efficacy five different ways § Clinical trial met the primary endpoint with a change in the Knee Injury and Osteoarthritis Outcome Score (KOOS) from baseline. Clinical efficacy was demonstrated in five ways:
Slide 15: Positive Phase 2b OA/BMEL Data
POSITIVE PHASE 2B OA/BMEL DATA Mean Change in KOOS Pain From Baseline Total population NRS 4-6 Stratum Days Days 0 11 25 39 53 81 0 11 25 39 53 81 0 0 2 2 Mean Change from Baseline Mean Change from Baseline 4 4 6 6 ß Improvement ß Improvement 8 8 10 10 12 12 14 14 16 16 18 18 20 20 § In the Total Po
Slide 16: Positive Phase 2b OA/BMEL Data
POSITIVE PHASE 2B OA/BMEL DATA Number of Subjects with a >50% Reduction from Baseline in Number of subjects with a >50% Reduction from Baseline in KOOS Pain Score at Days 11-81 – NRS: 4-6 Stratum NRS Pain Score at Days 11-81 – NRS:4-6 Stratum iPPS Placebo iPPS Placebo 100 100 90 90 80 80 Treatment P
Slide 17: Positive Phase 2b OA/BMEL Data
POSITIVE PHASE 2B OA/BMEL DATA Mean Percentage Change in NRS Pain from Baseline – NRS:4-6 Stratum Day 11 Day 25 Day 39 Day 53 Day 81 0 Mean Percentage Change from Baseline -5 -10 These data are consistent with the -15 TGA SAS Results -20 -25 -30 -35 -40 Adjusted Mean Relative Change from Baseline an
Slide 18: iPPS vs Tanezumab (Pfizer/Eli Lilly - Anti-NGF)
IPPS VS TANEZUMAB (PFIZER/ELI LILLY - ANTI-NGF) Percentage of subjects with >50% improvement in KOOS/WOMAC pain score at day 53 60.00% iPPS results are comparable to 50.00% Tanezumab 2.5mg 40.00% 30.00% 20.00% 10.00% 0.00% iPPS (NRS 4-6 Strata) Tanezumab 2.5mg (SC) Placebo (iPPS) Placebo (Tanezumab)
Slide 19: Filing of Pivotal Study with Agency (IND)
FILING OF PIVOTAL STUDY WITH AGENCY (IND) Filing of Investigative New Drug (IND) application with Regulatory Agency § Preparation of Phase 3 Clinical Trial protocols § Design Phase 3 Clinical Trial § Meet with the FDA re trial design and end-points (pre-IND meeting) § Incorporate FDA feedback (if an
Slide 20: Deals – Global Big Pharma Interest in OA
DEAL DEALS – GLOBAL BIG PHARMA INTEREST IN OA Recent transactions highlight big pharma interest in OA Safety Issues COMPANIES COMPOUND REGION UPFRONT TOTAL VALUE STATUS Anti-NGF Global US$200m US$1.8bn Phase 3 Anti-NGF Global US$250m US$1.25bn Phase 3 Corticosteroid Global Take-over* US$1.0bn* Comme
Slide 21: Rare Diseases – Deal Ready
Rare Diseases – Deal Ready March 2019 21 Investor Presentation March 2019
Slide 22: Leading MPS Treatments
LEADING MPS TREATMENTS MPS presents a significant opportunity for Paradigm COMPANY AT 2017 REVENUES YEAR APPROVED CONDITION APPROVAL (USD MILLION) BioMarin Aldurazyme™ 2003 MPS I $90.021 Pharmaceuticals Shire Elaprase® 2006 MPS II $615.7 Pharmaceuticals BioMarin Naglazyme® 2005 MPS VI $332.2 Pharmac
Slide 23: Mucopolysaccharidoses (MPS)
MUCOPOLYSACCHARIDOSES (MPS) MPS – An Orphan Indication in need of new treatments In November 2018, Paradigm in-licensed the MPS indication from the Icahn School of Medicine at Mount Sinai, New York. The License includes successful Phase 2a safety and efficacy data MPS Market Facts: 13,000+ patients
Slide 24: MPS Results – Paradigm/Hennermann et al.
MPS RESULTS – PARADIGM/HENNERMANN ET AL. Paradigm acquired highly valuable Phase 2a clinical trial data Type Phase 2a – Open Label Primary Outcome Safety Total Number of subject n=4. Number of Patients 4 GAG levels (n=1 1mg/Kg Yellow), (n=1 1mg/Kg pink),(n=1 blue Secondary Dosing Range 1mg/kg – 2mg/
Slide 25: Potential Share Price Catalysts / News Flow
POTENTIAL SHARE PRICE CATALYSTS / NEWS FLOW There is potential for significant news flow in the short term (<6 months) Ø OA Phase 2b trial results released. Partnering Discussions Commenced Ø Release Secondary End-point data front the OA Phase 2b trial – Q1/Q2 2019 Ø Potential to treat to MPS patien
Slide 26: Appendices & Additional Information
APPENDICES & ADDITIONAL INFORMATION 1. OPIOID EPIDEMIC 2. MECHANISM OF ACTION (MOA) 3. STAGES OF OA OF THE KNEE 4. MPS 5. VIRAL ARTHRITIS 6. CLINICAL PIPELINE 7. PEER COMPARISON 8. BOARD AND MANAGEMENT 26 Investor Presentation March 2019
Slide 27: 1. Market Demand – Opioid Epidemic
1. MARKET DEMAND – OPIOID EPIDEMIC What is the Opioid Epidemic? § The opioid epidemic is a crisis throughout North America that involves the widespread use of prescription painkillers and subsequent popularity of illegal opioids, resulting in unprecedented addiction and consequential overdoses, many
Slide 28: 2. Mechanism of Action (MoA)
2. MECHANISM OF ACTION (MoA) Mechanism of Action - PPS has demonstrated: § The inhibition of cartilage degrading enzymes that are released post-acute injury; § Anti-inflammatory effects, whilst blocking the effects of the pro-inflammatory cytokines TNF-alpha and IL-1; § Antithrombic and antilipadaem
Slide 29: 3. Stages of OA – Minimal, Mild, Moderate & Severe
3. STAGES OF OA – MINIMAL, MILD, MODERATE & SEVERE Healthy Knee Moderate OA Moderate OA Severe OA Source: Bioventus 29 Investor Presentation March 2019
Slide 30: 4. MPS Results – Dr Furujo
4. MPS RESULTS – DR FURUJO Paradigm’s data supported by Dr Furujo’s Findings Inflammatory Markers Type Phase 2a – Open Label Number of Patients 2 Dosing Range 0.5mg/kg – 1.5mg/kg Treatment Length 17 Weeks Primary Outcome Safety GAG & Inflammatory markers Secondary Outcomes Pain Mobility Urinary GAG
Slide 31: 4. MPS: Clinical Timeline
4. MPS: CLINICAL TIMELINE MPS - An Orphan Indication with near term commercialisation potential § Animal model data demonstrated that PPS was able to reduce the levels of Glycosaminoglycans (GAGs) (accumulation of GAGs leads to joint pain and dysfunction) and inhibit inflammatory responses due to TL
Slide 32: 4. MPS, Rare Diseases and Orphan Drugs
4. MPS, RARE DISEASES AND ORPHAN DRUGS § 7,000+ rare diseases (defined as conditions that affect fewer than 200,000 Americans) § The 1983 Orphan Drug Act in the US, and other such laws internationally incentivise pharmaceutical companies to develop medicines for rare diseases – “orphan drugs” – that
Slide 33: 5. Viral Arthritis - Alphavirus
5. VIRAL ARTHRITIS - ALPHAVIRUS No approved treatment for severely debilitating viral infection Viral Arthritis § Alphavirus infections result in the clinical symptoms of joint and muscle pain, fever and joint inflammation. § Ross River Virus (RRV) and Chikungunya (CHIKV) are mosquito-transmitted ar
Slide 34: 5. Viral Arthritis: Clinical Timeline
5. VIRAL ARTHRITIS: CLINICAL TIMELINE Potential to gain Orphan status, resulting in fast-tracked clinical development § Pre-clinical studies have been conducted by the Institute of Glycomics at Griffith University. The results suggested that: - PPS significantly alleviated the severity of disease an
Slide 35: 6. Clinical Pipeline
6. CLINICAL PIPELINE Paradigm hosts a deep clinical pipeline Indication(s) Clinical Status Market Size § Inflammatory bowel disease (“IBD”) Inflammatory Bowel Disease Safety and efficacy confirmed in IL-1RA Peptide § Cancer-related cachexia Medicines predicted to reach Phase 1/2 clinical trial (n:26
Slide 36: 7. Peer Comparison
7. PEER COMPARISON Attractive investment given low risk development and large market opportunity Ticker and Market cap Clinical stage of key Addressable market Peer exchange (A$m) Rationale product size World leader in developing and commercializing innovative biopharmaceuticals for rare diseases dr
Slide 37: 8. Board and Management
8. BOARD AND MANAGEMENT High quality Board and management, with top-tier pharmaceutical experience § Board and management are renowned leaders in the biopharmaceutical industry, having held senior management positions with top ASX- listed companies, CSL (CSL.ASX) and Mesoblast (MSB.ASX) § Extensive
Slide 38: Thank You
THANK YOU 38 Investor Presentation March 2019
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