Adarx Pharmaceuticals, Inc. · NASDAQ:ADRX
Adarx Pharmaceuticals — IPO Roadshow (September 2026)
35 slides from Adarx Pharmaceuticals's IPO roadshow presentation, September 2026
Slide 1: Focused on Developing Next-Generation siRNA Therapeutics
Focused on Developing Next- Generation siRNA Therapeutics Designed to Treat Broad Spectrum of Diseases September 2026
Slide 2: Disclaimers
Disclaimers ADARx Pharmaceuticals, Inc. (the “Company,” “ADARx,” “we,” “us,” or “our”) has filed a registration statement (including a preliminary prospectus) on Form S-1 (File No. 333-298782) with the Securities and Exchange Commission (the “SEC”) for the offering to which this presentation and the
Slide 3: ADARx Pharmaceuticals
ADARx Pharmaceuticals Developing next-generation siRNA therapeutics designed to treat a broad spectrum of diseases BROAD siRNA PIPELINE ADVANCING THE POTENTIAL OF siRNA • 5 wholly-owned, internally generated clinical • Hepatic: designed to increase depth and + advanced preclinical programs for chron
Slide 4: Shared Vision of Building Next-Generation siRNA Therapeutics Company
Shared Vision of Building Next-Generation siRNA Therapeutics Company Management team with deep experience in RNAi medicine Board of Directors Zhen Li, PhD Ying Huang, PhD President & CEO Former CEO, Legend Biotech Erez Chimovits, MBA Judith J. Li, MBA Partner, OrbiMed Partner, Lilly Asia Ventures Ti
Slide 5: ADARx Pipeline of Next-Generation siRNA Programs
ADARx Pipeline of Next-Generation siRNA Programs IND- PROGRAM TARGET INDICATION DISCOVERY PHASE 1 PHASE 2 PHASE 3 NEXT ANTICIPATED MILESTONE ENABLING Renal Mid-27: IgAN 9m UPCR (lgAN, C3G, IC-MPGN) 2H27: C3G 9m UPCR; IgAN 1-yr eGFR Agazisiran CFB PNH 2H27: PNH Ph2, 6m Hb HEPATIC GA Ongoing: GA Ph2,
Slide 6: ADARx Differentiation: Technology and Strategy
ADARx Differentiation: Technology and Strategy Core proprietary technologies developed to optimize our siRNA sequences and delivery to both hepatic & extrahepatic tissues MST designed with the goal of 01 consistently achieving highest Sequence possible inherent potency and Engineering durability of
Slide 7: Reproducibility Demonstrated Across Multiple Clinical Programs
Reproducibility Demonstrated Across Multiple Clinical Programs Observed quality, reproducibility and speed potentially enables scalable & efficient pipeline expansion Onvuzosiran Agazisiran ADX-626 PKK CFB FXI CLINICAL DATA Quality Clinical Data Observed Observed Reproducibility Capital-Efficiency &
Slide 8: Unlocking Extrahepatic Tissue Access for siRNA May Drive Future Pipeline Expansion
Unlocking Extrahepatic Tissue Access for siRNA May Drive Future Pipeline Expansion ADARx technology has Shown the ability to selectively target six ADARx strategic focus developed to overcome siRNA extrahepatic tissues limitations: Neuron • Extrahepatic-targeted: Designed to overcome key barriers Ey
Slide 9: Agazisiran
Agazisiran A potent long-acting siRNA silencing complement factor B (CFB) mRNA for the treatment of multiple complement- mediated diseases 9
Slide 10: Agazisiran Has Pipeline-in-a-Product Potential
• We believe FDA’s approval of the complement factor B (CFB) inhibitor FABHALTA® validates CFB as a target in the complement pathway, which is Encouraging implicated in the pathophysiology of a broad range of complement- Agazisiran Has clinical data mediated diseases • Pipeline-in-a- • Designed to s
Slide 11: Agazisiran Ph1 Data Supports Potential for Both Duration & Depth of AP Activity Inhibition
Agazisiran Ph1 Data Supports Potential for Both Duration & Depth of AP Activity Inhibition Clinical Phase 1 data • At 6.0 mg/kg, we observed near-complete inhibition of AP after a single SC injection of agazisiran – A mean inhibition of 99.9% of the complement AP activity was achieved at nadir – Sup
Slide 12: Agazisiran Clinical Development Program: Multiple Phase 2 Trials Ongoing
Agazisiran Clinical Development Program: Multiple Phase 2 Trials Ongoing with Cadence of Data Anticipated in 2027 and 2028 Program Trial Design Protocol Summary Endpoints IgAN: Agazisiran 300 mg Q3M N=15 Renal Diseases • Global, multi-center, open-label multiple dose • Primary: safety and tolerabili
Slide 13: Onvuzosiran
Onvuzosiran A potent long-acting siRNA silencing PKK mRNA for the prevention of HAE attacks 13
Slide 14: Onvuzosiran Targeting >75% Attack-free Rate
• Onvuzosiran is designed to selectively target and degrade PKK mRNA Encouraging in the liver and prevent protein synthesis, thereby reducing PKK protein levels and preventing activation of bradykinin Onvuzosiran clinical data • In Phase 1 at 6 mg/kg, we observed that a single SC injection reduced T
Slide 15: Onvuzosiran Observed Deep and Durable Target Knockdown
Onvuzosiran Observed Deep and Durable Target Knockdown Phase 1 clinical data in healthy volunteers at select dose cohorts • At the 6 mg/kg dose level, we observed that a Change From Baseline(%) single SC injection reduced circulating PKK/KK Percent PKK-KK Protein levels by a mean of 94% at nadir and
Slide 16: STOP-HAE: Phase 3 Study of Onvuzosiran in Hereditary Angioedema
STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo- Controlled Study of Onvuzosiran Targeting Prekallikrein in Subjects With Hereditary Angioedema • Phase 3 global study ongoing • 300 mg Q6M Cohort: – Phase 1 support: 94% mean (88-97%) reduction at nadir, 84% mean (78-92%) reduction at 6mo at 30
Slide 17: Onvuzosiran: Interim Phase 2a Results
Onvuzosiran: Interim Phase 2a Results 3-Month attack-free 6-Month attack-free 6-Month attack-free • Phase 2a: Dosed at Q6M, all were doses either significantly lower than in Phase 3 or less frequent (Phase 3 dose cohorts: 300 mg Q6M or 240 mg Q3M) • All three patients (3/3) were attack-free for 3 mo
Slide 18: ADX-626
ADX-626 A potent long-acting siRNA silencing coagulation Factor XI (FXI) for secondary stroke prevention with additional clinical potential for stroke prevention in atrial fibrillation 18
Slide 19: ADX-626 is a Potent & Long-acting siRNA
• FXI is a key component of the intrinsic coagulation pathway Genetically & clinical • Plays an important role in thrombus propagation/growth ADX-626 is a validated target • Less critical for the initial clot formation that largely depends on the extrinsic pathway and platelet function Potent & Long
Slide 20: ADX-626 NHP Data: Deep and Sustained FXI Protein Suppression
ADX-626 NHP Data: Observed that a Single SC Injection Demonstrated Deep and Sustained FXI Protein Suppression in NHP Study % Factor XI Protein Level Reduction NHP ADX-626, 3 mg/kg (n=2) and 5 mg/kg (n=2) Serum Protein Reduction % 0 • Single SC dose achieved near-complete, sustained 20 reduction of F
Slide 21: ADX-626: Deep and Durable Target Reduction Observed in Ongoing Phase 1 Study
ADX-626: Deep and Durable Target Reduction Observed in Ongoing Phase 1 Study1 • Deep target reduction: We observed that a single SC dose at 400 mg dose reduced the mean of 97.4% and 95.4% at nadir in FXI protein and FXI activity, respectively (Four of the six participants were below LLOQ for FXI act
Slide 22: ADX-077
ADX-077 siRNA preclinical therapeutic candidate for obesity via a novel undisclosed target in adipose tissue 22
Slide 23: ADX-077 Designed to Selectively Inhibit a Novel Target in Adipose Tissue
• ADX-077 was designed to selectively inhibit an undisclosed target Novel approach (“OBE1”) in adipose tissue ADX-077 targeting weight loss • OBE1 reduces adiposity through mechanism distinct from appetite suppression associated with GLP-1 receptor agonism with preservation of Designed to lean mass
Slide 24: Observed Deep, Durable Fat Loss With Lean Muscle Preserved in DIO Mice
Observed Deep, Durable Fat Loss With Lean Muscle Preserved in DIO Mice Deep target Meaningful durable Robust fat reduction, observed without mRNA knockdown weight loss (Q6W SC) lean mass loss OBE1 mRNA Knockdown in Adipose Percent Body Weight White Adipose and Skeletal Muscle Body Weight change (%)
Slide 25: ADX-077: Encouraging NHP Data
ADX-077: Encouraging NHP Data Meaningful Effect on Body Weight and Fat Reduction Observed in Spontaneous Obese NHPs OBE1 mRNA Knockdown in Adipose Body Weight • ADX-077 − Steady weight loss and meaningful fat mass reduction observed in obese NHPs Starting NHP BMI=42-53 − Preserved lean mass Body Fat
Slide 26: ADX-199
ADX-199 siRNA preclinical therapeutic candidate for selectively silencing and degrading APP mRNA in neurons for neurodegenerative diseases 26
Slide 27: ADX-199 is Designed to Selectively Silence and Degrade APP mRNA
Extrahepatic • ADX-199 is an siRNA therapeutic candidate engineered to selectively reduce APP mRNA in neurons ADX-199 is delivery into neurons Designed to Selectively Validated • Human genetic evidence indicates that elevated levels of APP protein Silence and target or its cleavage products, includi
Slide 28: ADX-199: Engineered to Selectively Reduce APP mRNA in Neurons
ADX-199: Engineered to Selectively Reduce APP mRNA in Neurons for Neurodegenerative Diseases Observed Deep and Durable Target Suppression in NHPs ADX-199 in NHP Study • Targeting a well validated neurodegenerative disease driver (APP) • Achieved potent and durable reduction of target APP protein and
Slide 29: Preferential APP mRNA Knockdown Observed in NHP
Preferential APP mRNA Knockdown Observed in NHP ADX-199 NHP study: IT lumbar Puncture, Single 60 mg dose, 3 months post dose , n=2 150 % APP mRNA Expression Normalized to Vehicle 125 100 We observed deep APP 75 Control mRNA reduction across 50 ADX-199 various brain regions at 25 3 months, including
Slide 30: ADARx Summary
ADARx Summary 30
Slide 31: Internally Generated, Wholly Owned Programs Designed to Efficiently Deliver siRNA
Internally Generated, Wholly Owned Programs Designed to Efficiently Deliver siRNA to Both Hepatic & Extrahepatic Tissues Agazisiran (CFB, Ph2) Onvuzosiran (PKK, Ph3) ADX-626 (FXI, Ph1) A potent long-acting siRNA silencing CFB A potent long-acting siRNA silencing PKK A long-acting siRNA silencing coa
Slide 32: Meaningful Cadence of Data Milestones Anticipated Across Our Portfolio
Meaningful Cadence of Data Milestones Anticipated Across Our Portfolio Program 2027 2028 Mid-27: IgAN Ph2, 9m UPCR reduction data Agazisiran 2H27: PNH Ph2, 6m Hb readout 1H28: IgAN China combo, 9m UPCR reduction CFB 2H27: IgAN Ph2, 1-yr eGFR stabilization 2H27: C3G Ph2, 9m UPCR reduction Onvuzosiran
Slide 33: Thank you!
Thank you! Innovative. Efficient. Focused. 33
Slide 34: Appendix
Appendix 34
Slide 35: Explorative Tox Study: ADX-077 Was Well Tolerated in NHP at 50 mg/kg
Explorative Tox Study: ADX-077 Was Well Tolerated in NHP at 50 mg/kg ClinicalChemistry Clinical Chemistry Body Weight Body Weight Alanine Aminotransferase Aspartate Aminotransferase Alkaline Phosphatase Gamma Glutamyl Transferase 4.0 Control - 0 mg/kg Body Weight (kg) (Mean ± SEM) 150 100 Ref. Range
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