Percheron Therapeutics Limited · ASX:PER
Percheron Therapeutics — Investor Presentation (September 2024)
26 slides from Percheron Therapeutics's investor presentation, Sep 4, 2024
Slide 1: ASX Announcement: Percheron Therapeutics Corporate Presentation
ASX Announcement PERCHERON THERAPEUTICS CORPORATE PRESENTATION Melbourne, Australia – 4 September 2024: Percheron Therapeutics Limited (ASX: PER or “the Company”), an international biotechnology company focused on the development of novel therapies for rare diseases, is pleased to provide an updated
Slide 2: About Percheron Therapeutics Limited
About Percheron Therapeutics Limited Percheron Therapeutics Limited [ASX: PER | US OTC: ATHJY | FSE: AWY] is a publicly listed biotechnology company focused on the development and commercialisation of novel therapies for rare diseases. The company’s lead program is ATL1102, an antisense oligonucleot
Slide 3: Developing High Impact Therapies for Orphan Diseases
For personal use only Developing High Impact Therapies for Orphan Diseases Corporate Overview September 2024 www.PercheronTx.com ASX: PER | US OTC: ATHJY
Slide 4: 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 5: 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 avicursen (ATL1102), an antisense oligonucleotide (ASO) treatment for For personal use only Duchenne muscular dystrophy (DMD) and oth
Slide 6: Percheron's pipeline of potential first-in-class orphan disease assets
Percheron’s pipeline comprises potential first-in-class assets for orphan diseases with high unmet clinical need Avicursen (ATL1102) Antisense oligonucleotide targeting CD49d For personal use only PRECLIN. PHASE 1 PHASE 2 PHASE 3 MARKET Muscular Dystrophies Duchenne Muscular Dystrophy lead Professor
Slide 7: FY2024 progress advancing the pipeline
In FY2024, Percheron continued to make good progress with advancing its pipeline, against a backdrop of favourable market dynamics For personal use only January 2024 May 2024 May 2024 May 2024 May 2024 September 2024 Publication of full data WHO selects ‘avicursen’ Initial data from 9-month US PTO a
Slide 8: Avicursen (ATL1102) in Duchenne Muscular Dystrophy: Phase IIb Clinical Program
For personal use only Avicursen (ATL1102) in Duchenne Muscular Dystrophy Phase IIb Clinical Program 5
Slide 9: Duchenne muscular dystrophy is an incurable genetic condition
Duchenne muscular dystrophy 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 normal 2
Slide 10: 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 11: Avicursen's mechanism of action is well validated
Avicursen’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 a Ordinarily, the genetic code in a patient’s DNA is transcribed to RNA, which is used to direct synthesis of prote
Slide 12: Preclinical investigation of avicursen in DMD
Preclinical investigation of avicursen (ATL1102) in DMD has shown it to be effective at reducing CD49d+ T-cells and improving muscle function ATL1102 reduces CD49d in skeletal …leading to an increase in muscle Key Takeaways For personal use only muscle… function • Preclinical investigation of avicur
Slide 13: Clinical efficacy in phase IIa pilot study of 9 non-ambulant boys
Avicursen 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 Pe
Slide 14: 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 avicursen 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≈16) confi
Slide 15: Six-month phase IIb data will define the path to market
Six-month data from the ongoing phase IIb trial of avicursen 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 regard
Slide 16: Commercial opportunity in DMD of ~US$4 billion
PRELIMINARY The commercial opportunity in DMD is substantial, with a potential market size of ~US$4 billion, reflecting favourable pricing dynamics Comparator Revenues (2022-23) Comparator Pricing Avicursen Addressable Market For personal use only Company Product 2022 2023 Company Product Annual Cos
Slide 17: Competitive landscape in DMD is not crowded
The competitive landscape in DMD is not crowded, and most companies have focused on dystrophin-restoration therapies rather than anti-inflammatory approaches Early Development Late Development Approved Exon-Skipping Therapies For personal use only SRP-5051 ESTs are only suitable for patients with sp
Slide 18: Avicursen potentially competitive with existing therapies
PRELIMINARY Avicursen appears potentially competitive with existing therapies, which have generally shown limited evidence of functional benefit For personal use only Exon Skipping Therapies • ESTs each only applicable to 8-12% of patients Emerging Avicursen Profile • ESTs require weekly iv infusion
Slide 19: Data in other indications validates avicursen's mechanism
Data in a broad range of other indications validates avicursen’s mechanism and points to potential future expansion opportunities Multiple Sclerosis (clinical) Autoimmune Epilepsy (preclinical) Asthma (preclinical) For personal use only Cumulative Number of New Active Lesions Number of Seizures per
Slide 20: Outreach program to cultivate potential partners
The company has initiated a broad outreach program to identify and cultivate potential future partners for avicursen Illustrative Composition of Typical Pharma Partnering Benchmarks for Phase II Rare Disease Partnering Transactions For personal use only Transactions (2016 – 2023) (n=47) • A share of
Slide 21: Partnering opportunity for avicursen is substantial
Partnering opportunity for avicursen is substantial, with benchmark transactions suggesting opportunity for significant value realisation Licensing Transactions Licensee Licensor Asset Indication Stage Date Deal Value (US$) For personal use only CAP-1002 (United States) Duchenne muscular dystrophy P
Slide 22: Corporate Profile
19 For personal use only Corporate Profile
Slide 23: New Percheron team brings 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 24: Strong financial position with phase IIb well funded
AS AT 30 JUN 2024 Percheron enjoys a strong financial position, with the ongoing phase IIb study of avicursen well funded Operating Cashflows (AU$ ,000) For personal use only 3,000 Corporate Fundamentals Market Capitalisation: ~AU$ 77M 2,000 Primary Listing: ASX: PER 1,000 Secondary Listings: FSE: A
Slide 25: Near-term news flow with multiple value-driving catalysts
PRELIMINARY 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 avicursen in Duchenne muscular dystrophy 1H CY2024 ✓ Operational compl
Slide 26: For personal use only
For personal use only info@PercheronTx.com www.PercheronTx.com 23
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