Percheron Therapeutics Limited · ASX:PER
Percheron Therapeutics — Investor Presentation (February 2024)
21 slides from Percheron Therapeutics's investor presentation, Feb 26, 2024
Slide 1: Percheron Therapeutics Corporate Presentation (ASX announcement)
PERCHERON THERAPEUTICS CORPORATE PRESENTATION Melbourne, Australia – 26 February 2024: Percheron Therapeutics Limited, an international biotechnology company focused on the development of novel therapies for rare diseases, is pleased to provide an updated non-confidential corporate presentation for t
Slide 2: Developing High Impact Therapies for Orphan Diseases
For personal use only Developing High Impact Therapies for Orphan Diseases Corporate Overview February 2024 www.PercheronTx.com ASX: PER | FSE: AWY | US OTC: ATHJY
Slide 3: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements do not relate strictly to historical or current facts and may be accompanied by words such as ‘c
Slide 4: Percheron is a late-clinical stage biotech focused on orphan diseases
Percheron Therapeutics (ASX: PER) is a late-clinical stage biotech company, focused on development of novel high-value therapies for orphan diseases • Lead program is ATL1102, an antisense oligonucleotide treatment for Duchenne muscular For personal use only dystrophy (DMD) and other diseases • Inte
Slide 5: Good progress continues to be made on ATL1102
Good progress continues to be made on ATL1102, with positive developments in recent months Deliver Phase IIb Clinical Trial in DMD • All five countries open to recruitment Next Milestones • Recruitment on track for ~1Q completion Complete recruitment: 1Q CY2024 For personal use only Provide high-qua
Slide 6: Pipeline of potential first-in-class assets for genetic diseases
Percheron’s pipeline comprises potential first-in-class assets for genetic diseases with high unmet clinical need ATL1102 Antisense oligonucleotide targeting CD49d For personal use only PRECLIN. PHASE 1 PHASE 2 PHASE 3 MARKET Muscular Dystrophies Duchenne Muscular Dystrophy (monotherapy) lead Profes
Slide 7: Duchenne muscular dystrophy affects approximately 300,000 children and young adults worldwide
Duchenne muscular dystrophy (DMD) is an incurable genetic condition that affects approximately 300,000 children and young adults worldwide For personal use only 1 Inherited genetic Duchenne Incidence is DMD also abnormality renders patient muscular approximately associated with unable to produce nor
Slide 8: Two fundamental approaches to pharmacological treatment of DMD
There are two fundamental approaches to the pharmacological treatment of DMD: (1) target the underlying genetic abnormality, and (2) target its effects 1 • Most therapies are only applicable to For personal use only Target the underlying genetic a small proportion of patients abnormality to restore
Slide 9: ATL1102 mechanism of action is well validated
ATL1102’s mechanism of action is well validated and clearly understood, with nine FDA-approved antisense oligonucleotide therapies already available to patients For personal use only 1 Ordinarily, the genetic code in a patient’s DNA is transcribed to RNA, which is used to direct synthesis of protein
Slide 10: ATL1102 phase IIa pilot study efficacy results
ATL1102 has shown compelling evidence of clinical efficacy across multiple validated endpoints in a phase IIa pilot study of 9 non-ambulant boys Key Study Parameters Study Results (Efficacy) [at 6 months] For personal use only Population Endpoint Description ATL1102 Result Historical Comparator Perf
Slide 11: Ongoing double-blind phase IIb clinical study design
An ongoing, double-blind phase IIb clinical study has been designed to provide definitive evidence of efficacy for ATL1102 in non-ambulant boys with DMD For personal use only Patient Population Blinded Treatment Open-Label Treatment Follow-Up Period Placebo Period Period • Genetically (n=15) confirm
Slide 12: Phase IIb data will define the likely path to market
Six-month data from the ongoing phase IIb trial of ATL1102 will define the likely path to market for the drug For personal use only Pediatric Priority Review Voucher (pPRV) Discussion with FDA and other regulatory • PRV system designed to incentivise private sector to Positive Data agencies regardin
Slide 13: The commercial opportunity in DMD is substantial
PRELIMINARY The commercial opportunity in DMD is substantial, with a potential market size of ~US$ 4 billion, reflecting favourable pricing dynamics Comparator Revenues (2021-22) Comparator Pricing ATL1102 Commercial Opportunity For personal use only Company Product 2022 2021 Company Product Annual
Slide 14: The competitive landscape in DMD is not crowded
The competitive landscape in DMD is not crowded, and most companies have focused on dystrophin-restoration therapies rather than anti-inflammatory approaches Early Development Late Development Approved Exon-Skipping Therapies For personal use only SRP-5051 ESTs are only suitable for patients with sp
Slide 15: Opportunity to expand ATL1102 beyond non-ambulant DMD
ILLUSTRATIVE Although focus is on non-ambulant DMD patients for now, there is rich opportunity to expand the use of ATL1102 beyond this patient population For personal use only HIGH Combo with Non- ESTs ambulant DMD Bubble size = market opportunity Close adjacencies in muscular Ambul- ant dystrophy
Slide 16: Broad outreach program to identify potential partners
The company has initiated a broad outreach program to identify and cultivate potential future partners for ATL1102 Illustrative Composition of Typical Pharma Partnering Benchmarks for Phase II Rare Disease Partnering Transactions For personal use only Transactions (2016 – 2023) (n=47) • A share of n
Slide 17: Partnering opportunity for ATL1102 is substantial
Partnering opportunity for ATL1102 is substantial, with benchmark transactions suggesting opportunity for significant value realisation Licensing Transactions Licensee Licensor Asset Indication Stage Date Deal Value (US$) For personal use only CAP-1002 (United States) Duchenne muscular dystrophy Pha
Slide 18: New Percheron team with extensive international experience
New Percheron team brings extensive international experience in drug development, partnering, and commercialisation For personal use only Dr Charmaine Gittelson Dr Gil Price Dr James Garner Board Chair Non-Executive Director CEO & Managing Director 25 years of experience, Experienced biotech includi
Slide 19: Strong financial position, phase IIb study well funded
AS AT 31 JAN 2024 Percheron enjoys a strong financial position, with the ongoing phase IIb study of ATL1102 well funded Operating Cashflows (AU$ ,000) For personal use only 2,000 Corporate Fundamentals Market Capitalisation: ~AU$ 52M 1,000 Primary Listing: ASX: PER Secondary Listings: FSE: AWY; OTC:
Slide 20: Near-term news flow with multiple value-driving catalysts
Percheron is rich in near-term news flow, with the potential for multiple value-driving catalysts over the next 18 months ✓ - completed CY2024 For personal use only Full recruitment to international phase IIb study of ATL1102 in Duchenne muscular dystrophy 1Q CY2024 Operational completion of 9-month
Slide 21: For personal use only
For personal use only info@PercheronTx.com www.PercheronTx.com 19
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