Abionyx Pharma Sa · EURONEXT:ABNX
Abionyx Pharma Sa — Shareholder Meeting (June 2022)
29 slides from Abionyx Pharma Sa's shareholder meeting presentation, Jun 28, 2022
Slide 1: General Meeting of Shareholders
General Meeting of Shareholders 28 June 2022 J U N E 2 0 2 2 | N O N C O N F I D E N T I A L
Slide 2: Important notice
Important notice You must read the following before continuing. In accessing this document, you agree to be bound by the following terms and conditions. References herein to this presentation (this “Presentation”) shall mean and include this document, the oral presentation accompanying this document
Slide 3: Agenda
1. Abionyx Agenda 2. CER-001 3. COVID-19 4. LCAT Deficiency 5. Sepsis induced Acute Kidney Injury 6. Uveitis 3
Slide 4: 1. Abionyx
1. Abionyx J U N E 2 0 2 2 | N O N C O N F I D E N T I A L
Slide 5: A seasoned management team
Cyrille Tupin Connie Keyserling Ronald Barbaras, CEO Peyrottes, M.S. Ph.D. Senior VP Clinical Senior VP R&D Development & A seasoned Operations management team specialized in Margit Holzer, Ph.D. Pierre-Paul Elena, Ph.D. Senior VP Bio- Scientific Director HDL and production Ophthalmology Yann Quentr
Slide 6: Abionyx Vision and Mission
Covid Sepsis Abionyx Vision and Re-inventing Mission the bio-HDL Statement for the benefit of patients Ophthalmology Renal failure with rare or severe diseases 6
Slide 7: Indications under investigation
Indications under investigation Product Early Stage Preclinical Phase 1 Phase 2 Phase 3 Early Access Market size LCAT CER-001 $50M - $100M CER-001 SEPSIS $800M - $1,000M ABNX-1001 $500M - $700M ABNX-1010 $300M - $500M ABNX-1520 $200M - $300M ABNX-2501 $2,000M - $3,000M ABNX-2100 $2,000M - $3,000M AB
Slide 8: A strategy focused on rapid access to market
A strategy focused on rapid access to market Diversify the medical franchises Pillar 1 Pillar 2 Pillar 3 FRANCHISE RENAL OPHTALMOLOGY CRO • World leadership in PK and • ODD in a rare disease (LCAT • ODD in a rare disease (LCAT preclinical ophthalmology renal) ophtha/Fish-eye disease) • Unique expert
Slide 9: 2. CER-001, A unique recombinant bio-HDL
2. CER-001, A unique recombinant bio-HDL
Slide 10: CER-001: one of the most advanced therapeutic bioproducts
CER-001 : one of the most advanced therapeutic ı A bioengineered complex ı A safety profile allowing ı Commercially viable bioproduct comprising natural human HDL protein and administration at very high doses and frequencies manufacturing process with stability of 5 years apolipoprotein A-I (apoA-I)
Slide 11: Bio-HDL: a safe bioproduct with more than 900 patients treated
Bio-HDL: a safe bioproduct with more than 900 patients treated intravenously for up to 18 months Study Number of subjects Indication / doses Conclusion Single ı Healthy Volunteers 32 ı Safety and pharmacokinetic established Ascending Dose 0.25 to 45 mg/kg SAMBA ı Familial Primary Hypoalphalipoprotei
Slide 12: Bio-HDL: major efficacy impacts for Ophthalmology
Endothelial Systemic anti- Bio-HDL : 3 major Elimination of lipid protection : inflammatory disorders : efficacy impacts “Scavenger” “Vascular protection” for Ophthalmology • Non-cellular interactions – Removal of toxic lipids • Stabilize intercellular junctions • Macrophage-induced (LPS) and lipid
Slide 13: No direct competition: CER-001 is the only recombinant HDL in development
No direct competition: CER-001 is the only recombinant HDL in development Code Product Company Status Patients Completion CSL-112 ı Human derived ı CSL ı Phase 3 ı 17’000 ACS ı June 2023 HDL (purified ongoing patients endogenous from plasma) ETC-216 ı ApoA-I Milano ı The Medicine ı Terminated ı 126
Slide 14: 3. COVID-19
3. COVID-19
Slide 15: Compassionate use program in COVID-19
Compassionate use program in COVID-19 COVID-19 is associated with respiratory symptoms characterized by acute lung injury, Compassionate rapidly progressing to acute respiratory distress syndrome Access Authorization for CER-001 in The pulmonary dysfunction is rapidly accompanied by a major "cytokin
Slide 16: 4. LCAT Deficiency
ABIONYX 4. LCAT Deficiency
Slide 17: Emblematic ultra-rare disease for nephrologists: LCAT deficiency
Emblematic ultra-rare disease for nephrologists: LCAT deficiency A severe systemic disease affecting mainly the kidneys but also the cornea Corneal opacity Kidney biopsy | Diminished plasma HDL | Corneal opacities | Hypertriglyceridemia | Proteinuria Corneal opacity in patient with LCAT Kidney biops
Slide 18: Clinical Proof-of-Concept in ophthalmology
Positive clinical results in a renal indication that is also ophthalmologic for a bio-HDL Clinical Proof-of- Concept in ı ATUn at the Toulouse-Rangueil Hospital to treat LCAT deficiency, an ultra-rare disease ophthalmology with both renal and ophthalmic manifestations (Fish-Eye Disease) ı A patient
Slide 19: Treatment of CER-001 in severe FLD patient improved kidney function
Treatment of ı Double Orphan Drug Designation received from FDA and EMA ı Named compassionate treatment with CER-001 of an Italian patient with LCAT deficiency was CER-001 in authorized by the Friuli Venezia Giulia Regional Ethical Committee, (Opinion CEUR-2020-EAP-012- ASUFC) in February 2020. seve
Slide 20: 5. Sepsis-Induced Acute Kidney Injury
5. Sepsis-Induced Acute Kidney Injury
Slide 21: Ongoing phase 2a study: RACERS
Ongoing phase 2a study: RACERS ı Clinical study in partnership with the University of Bari and the CBVF consortium and Initiation of a Phase 2a study fully funded ı Randomized, open-label, placebo-controlled, parallel-group ı ATUn showed promising study evaluating the safety and efficacy of CER-001
Slide 22: RACERS Study treatment protocol
RACERS Study Treatment protocol - 5 patients per group Standard of Care Only Randomization CER-001 5 mg/kg BID + Standard of Care Screening CER-001 10 mg/kg BID + Standard of Care CER-001 20 mg/kg BID + Standard of Care Dosing Day -1 Day 1 Day 2 Day 3 Day 6 Day 9 Day 30 Evaluations 22
Slide 23: Positive Interim Results from Phase 2a Clinical Trial
Positive Interim Results Rapid reversal of cytokine storm in septic patients from Phase 2a Clinical Trial Evaluating Enhanced resolution of biomarkers for inflammation CER-001 in the including leukocytosis compared to standard of care Treatment of Septic Patients at High Risk of Developing No treatm
Slide 24: 6. Uveitis
ABIONYX 6. Uveitis
Slide 25: Uveitis is a systemic inflammatory disease affecting the uvea
Uveitis is a systemic inflammatory disease affecting the uvea ı Uveitis affects approximately 200 person per 100,000 in the western population ı More than half of all patients with uveitis develop complications related to their disease, and up to 35% of patients suffer severe visual impairment. Uvei
Slide 26: ABNX-1101: Effect on the modified McDonald-Shadduck score in the rabbit EIU model
24 hours after the induction of inflammation and the treatment with CER-001: ı Graph A reports the protein concentrations in the aqueous humor. In the intravitreal injection route, CER-001 strongly reduces the protein concentration and thus has an ABNX-1101: effect in LPS-induced uveitis. Effect on
Slide 27: The support of prestigious KOL in ophthalmology
The support of prestigious KOL in ophthalmology • Prof. Christophe Baudouin, Professor of Ophthalmology in Paris, Head of the Ophthalmology Department at the Centre Hospitalier National d'Ophtalmologie de l'Hôpital des Quinze-Vingts (Paris), Director of the "S12" research team at the Institut de la
Slide 28: Why invest in ABIONYX
One of the most advanced bioproduct with renewed IP (+20 yrs) including ophthalmology and biomanufacturing Several phase 2 trials and 1 MA by 2025 Why invest in ABIONYX A large portfolio of medical indications and bio/CRO activities that diversify the overall risks Committed teams, significant share
Slide 29: ABIONYX Pharma contact details
ABIONYX Pharma 33-43 av. Georges Pompidou Bât. D2 31130 Balma - France Tel : +33 5 62 24 97 06