Percheron Therapeutics Limited · ASX:PER
Percheron Therapeutics — Investor Presentation (November 2021)
42 slides from Percheron Therapeutics's investor presentation, Nov 1, 2021
Slide 1: Investor Presentation - October 2021
(ASX: ANP) Investor Presentation - October 2021 Not for release to US wire services or distribution in the United States
Slide 2: Disclaimer
Antisense Therapeutics Limited Investor Presentation - Disclaimer The following notice and disclaimer applies to this investor presentation (Presentation or document) and you are therefore advised to read this carefully before reading or Australian Accounting Standards. Prospective investors are cau
Slide 3: Investment Highlights
Investment Highlights Clinical unmet need DMD market $10bn by 2030 1 in 3,500 boys suffer from DMD with limited treatment options From Kamet Research Lead Program ATL1102 in DMD Positive Phase II trial results In licensed from Ionis Pharmaceuticals Across multiple measures of muscle strength and fun
Slide 4: Summary
Summary Duchenne Muscular Dystrophy (DMD) is a rare and fatal genetic muscle wasting disease that affects boys. Therapeutic options for DMD patients are very limited particularly for more advanced (non-ambulant) patients where ATL1102’s safety and efficacy was assessed in a successful Phase II c
Slide 5: What is DMD?
What is DMD? DMD is a rapidly progressing genetic disease resulting in low QOL and 100% mortality into patients’ 30’s • Duchenne Muscular Dystrophy (DMD) is a devastating genetic muscular disease caused by loss of dystrophin with progressive muscle wasting & associated muscle injury leading to infla
Slide 6: DMD Prevalence and Segmentation
DMD Prevalence and Segmentation DMD is a rapidly progressing genetic disease resulting in low QOL and 100% mortality into patients 30’s Affects 1 in 3,5001 - 5,0005 Male Births Worldwide Up to Approximately Up to Approximately 18,000 1,3 26,000 2,3 patients in the US patients in the EU 75% 50% Appro
Slide 7: ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense
ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense additional information Appendix B: Key risks Appendix C: Offering jurisdictions 7
Slide 8: Lead program: ATL1102 in DMD - Profile Summary
Lead program: ATL1102 in DMD - Profile Summary ATL1102 well positioned for global success within high-growth multi-billion-dollar DMD market • Novel and Differentiated MOA: ATL1102 is the only therapy in development for DMD targeting CD49d to slow disease progression by reducing inflammation and res
Slide 9: Clinical Development Plan
Clinical Development Plan European regulatory authorities have advised ATL1102 Phase IIb/III trial to be a potential pivotal study EU Phase IIb/III Clinical Trial • ANP will conduct a multi-centre, randomised, double-blind placebo-controlled Phase IIb/III study of ATL1102 in non-ambulant patients do
Slide 10: EU Phase IIb/III Study Program
EU Phase IIb/III Study Program A multicentre, randomised, double-blind, placebo-controlled study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 to be conducted in 108 (114 randomised) DMD non-ambulant participants in ~9 countries across >30 trial sites. Pla
Slide 11: EU Phase IIb/III Study Program (Continued)
EU Phase IIb/III Study Program (Continued….) Design: • A multicentre, randomised, double-blind, placebo-controlled study to determine the efficacy, safety, and pharmacokinetic profile of ATL1102 (25 mg and 50 mg) administered once weekly by subcutaneous (sc) injection for 52 weeks in non-ambulatory
Slide 12: EU Phase IIb/III Study Program (Continued)
EU Phase IIb/III Study Program (Continued….) Primary objective: To evaluate the effect of ATL1102 on upper limb muscle function in non-ambulant participants with DMD as assessed by change in the Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) score compared to placebo Secondary objectives: To
Slide 13: EU Phase IIb/III Study Program (Continued)
EU Phase IIb/III Study Program (Continued….) ATL1102 Open Label Extension Study: • All participants completing the Treatment Period of the Phase IIb/III study will be offered to the enter an open label extension (OLE) study of ATL1102 based on the assumption of acceptable safety. The OLE study will
Slide 14: Anticipated EU Phase IIb/III Study Timeline
Anticipated EU Phase IIb/III Study Timeline Defined Terms: CTA: Clinical Trial Application CSR: Clinical Study Report LOI: Letter of Intent EUR Com Decision: European Commission Decision 14 Note: The above timeline is indicative and illustrative only and is not a forecast or projection or any assura
Slide 15: Major Achievements and Upcoming Key Activities
Major Achievements and Upcoming Key Activities Regular progress and key market updates during the Phase IIb/III trial – multiple value creation opportunities ATL1102 Phase ATL1102 IIb / III Futility ATL1102 Phase IIb/III Analysis Report Phase IIb / III ATL1102 Phase Open Label Recruitment ATL1102 Ne
Slide 16: Significant Market Opportunity
Significant Market Opportunity ATL1102 - anti-inflammatory and immune modulating agent with potential for multiple clinical applications ANTI-INFLAMATORY DMD THERAPIES CORTICOSTEROIDS The market^ size is expected global The global DMD drug market The global steroid market is anti-inflammatory reach
Slide 17: ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense
ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense additional information Appendix B: Key risks Appendix C: Offering jurisdictions 17
Slide 18: Details of the Capital Raising
Details of the Capital Raising Antisense is raising approximately $20.0 million in a Placement to institutional and sophisticated investors (“Placement”). The placement will be conducted under the company’s existing placement capacity under ASX Listing Rule 7.1. Antisense also intends to underta
Slide 19: Detailed Use of Funds
Detailed Use of Funds • Funds raised via the Placement ($20.0M) and the Entitlement Offer ($16.8M), if fully subscribed, are intended to be used to fund the Phase IIb/III clinical trial through to futility analysis expected to be completed mid-CY2023. Should the Entitlement Offer not be fully subscr
Slide 20: Capital Raising Timetable
Capital Raising Timetable Event Date Placement Bookbuild Friday, 29 October Announcement of Placement and Entitlement Offer Monday, 1 November Ordinary Shares Recommence Trading Settlement of Placement Thursday, 4 November Allotment & Trading of Shares Issued Under the Placement Friday, 5 November L
Slide 21: ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense
ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense additional information Appendix B: Key risks Appendix C: Offering jurisdictions 21
Slide 22: Antisense Corporate Overview
Antisense Corporate Overview Share Price Performance Company Details $0.30 Prior Capital Raisings • Market Capitalisation (27 Oct 2021) @ $0.295 = $169M $0.25 • Ordinary fully paid shares on issue = 574M • Cash Balance as at 30 June 2021 = $6M • Largest shareholder – Platinum Asset Management $0.20
Slide 23: Antisense - ATL1102
Antisense - ATL1102 Improved therapies are needed to ameliorate DMD severity & delay disease progression ATL1102 • ATL1102 is a 2’MOE gapmer antisense oligonucleotide drug to integrin a4 RNA (CD49d alpha subunit of VLA-4), an adhesion molecule expressed on most human leukocytes • ATL1102 is designed
Slide 24: ATL1102 – IP Protection
ATL1102 – IP Protection Antisense has developed a substantial portfolio of intellectual property across multiple high-value markets Patent Portfolio Orphan Drug Designation (ODD) Granted ATL1102 Methods of reducing circulating leukocytes • Protected by Commercial Exclusivity (data Australia 20113017
Slide 25: ATL1102 – Phase II Study Results
ATL1102 – Phase II Study Results “Positive effects across multiple measures of muscle structure, function & strength”1 Open label Phase II trial in nine non-ambulant (wheelchair bound) boys 10-18 years of age with DMD conducted over 24 weeks of dosing: • Primary endpoint met with confirmation of dru
Slide 26: ATL1102 Phase II Study Data (Continued)
ATL1102 Phase II Study Data (Continued…) Efficacy Parameters – Muscle Strength (MyoGrip & MyoPinch) Comparison of Phase II Study Data with published literature showing statistically significant improvements. (Ricotti et. al. 2016) When ATL1102 Phase II study grip and pinch strength data is compared
Slide 27: ATL1102 Phase II Study Data (Continued)
ATL1102 Phase II Study Data (Continued…) Efficacy Parameters – Muscle Structure (MRI) Comparison of ATL1102 Phase II study data with data in published literature (Ricotti et. al. 2016) Mean Change (95% CI) Screening/Baseline to Week 24/ 6 Months “Based on the MRI data from the study, the observed st
Slide 28: ATL1102 Phase II Study Data (Continued)
ATL1102 Phase II Study Data (Continued…) Efficacy Parameters – Performance of Upper Limb Function (PUL.2.0) • ATL1102 data presented at the 25th International Annual Congress of the World Muscle Society in 2020 • ATL1102 treated patients demonstrated a statistically significant improvement in the me
Slide 29: ATL1102 New Indications
ATL1102 New Indications R&D collaboration with the Murdoch Children’s Research Institute (MCRI • ATL1102 modulates CD49d+ immune cells and disease processes in DMD and Multiple Sclerosis • Several inflammatory muscle disease indications (like DMD) have been identified which may benefit from similar
Slide 30: ATL1102 New Indications (Continued)
ATL1102 New Indications (Continued…) Statistically significant modulation in two DMD disease modifier proteins supports potential of ATL1102 in ambulant DMD and fibrotic conditions ATL1102 data presented at the 26th International Annual Congress of the World Muscle Society in 2021 • Statistically si
Slide 31: The DMD Market
The DMD Market The Global DMD drug market is expected to reach over US$4 Billion by 2023 and US$10 Billion by 20301 DMD Market by therapies in 20202 2020 DMD Market Share 2020 DMD Market Revenue 1 kametresearch.com/dmd-duchenne-muscular-dystrophy-market-analysis/ 31 2 DelveInsight - Duchenne Muscula
Slide 32: DMD Development Landscape
DMD Development Landscape Limited emerging competition in non-ambulant space ATL1102 is a well-positioned differentiator from other products in development for treatment of DMD • ATL1102 has a novel MOA to reduce inflammation in DMD INTERVENTION MECHANISMS ASSET SPONSORS patients ATL1102 o Anti-infl
Slide 33: DMD Market – Sarepta Comparison
DMD Market – Sarepta Comparison Sarpeta Therapeutics Inc – Exondys 51, Vyondys 53 & Amondys 45 • First FDA approved treatment for DMD (2016) • Exondys 51 only applicable for 13% of boys with the exon 51 mutation • Vyondys 53, approved in 2019 for approx. 8% boys with exon 53 mutation • Amondys 45, a
Slide 34: The Board of Directors
The Board of Directors Highly experienced Board and Management with prior success in drug development and commercialisation DR. CHARMAINE GITTLESON (Chairperson) MR. MARK DIAMOND (Managing Director & CEO) Dr Gittleson is a senior executive with international experience as a pharmaceutical Mark Diamo
Slide 35: The Management Team
The Management Team MR. MARK DIAMOND (Managing Director & CEO) MS. NUKET DESEM (Director of Clinical & Regulatory Affairs) Mark Diamond has over 30 years experience in the pharmaceutical and biotechnology Ms Desem has over 25 years’ experience in global regulatory affairs, clinical development indus
Slide 36: ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense
ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense additional information Appendix B: Key risks Appendix C: Offering jurisdictions 36
Slide 37: Key risks
Key risks INVESTMENT RISKS Partnering and Licensing This section highlights some of the risks that potential investors should consider prior to entering into the investment opportunity referredDue to the significant costs in drug discovery and development it is common for biotechnology companies to
Slide 38: Key risks (Continued)
Key risks (Continued…) Environmental Regulation and Performance Litigation, Claims and Disputes The Company is involved in pharmaceutical research and development, much of which is contracted out to third parties, and it is the The Company may be subject to litigation and other claims and disputes i
Slide 39: ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense
ATL1102 Phase IIb/III clinical development plan Details of the Capital Raising Appendix A: Antisense additional information Appendix B: Key risks Appendix C: Offering jurisdictions 39
Slide 40: International Offer Restrictions
International Offer Restrictions International Offer Restrictions This document does not constitute an offer of new ordinary shares ("New Shares") of the Company in any jurisdiction in which it would be unlawful. In particular, this document may not be distributed to any person, and the New Shares m
Slide 41: International Offer Restrictions (Continued)
International Offer Restrictions (Continued…) Singapore This document and any other materials relating to the New Shares have not been, and will not be, lodged or registered as a prospectus in Singapore with the Monetary Authority of Singapore. Accordingly, this document and any other document or ma
Slide 42: For more information
For more information: Investment enquiries: Mark Diamond Gennadi Koutchin Managing Director XEC Partners +61 (0)3 9827 8999 +61 (0)423 500 233 www.antisense.com.au gkoutchin@xecpartners.com.au
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