Proqr Therapeutics N.v. · NASDAQ:PRQR

Proqr Therapeutics — Investor Day (April 2026)

28 slides from Proqr Therapeutics's investor day presentation, Apr 8, 2026

  1. Slide 1: Expanding the Axiomer RNA Editing Opportunity Beyond AX-0810

    EXPANDING THE AXIOMER RNA EDITING OPPORTUNITY BEYOND AX-0810 Nasdaq: PRQR | April 8, 2026

  2. Slide 2: Agenda and Speakers

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 Agenda Welcome & Agenda Sarah Kiely Opening Remarks Daniel A. de Boer Platform and Pipeline Programs Cristina Lopez Lopez Gerard Platenburg Agenda Corporate Outlook Dennis Hom Q&A Daniel A. de Boer Cristina Lopez Lopez Gerard Platenburg D

  3. Slide 3: Forward-looking statements

    Forward-looking statements This presentation contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "

  4. Slide 4: Strategic Overview 2026

    Presenter: Daniel A. de Boer Strategic Overview ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 4

  5. Slide 5: Multiple clinical catalysts within the runway

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 5 Multiple clinical catalysts within the runway Core focus on cholestatic development, with value creating pipeline beyond Q2 2026 AX-0810 cholestatic disease Phase 1 target engagement data in healthy volunteers YEAR END 2026 AX-0810 chol

  6. Slide 6: Pipeline Programs 2026

    Presenters: Cristina Lopez Lopez & Gerard Platenburg Pipeline Programs ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 6

  7. Slide 7: Addressing unmet need in cholestatic diseases through NTCP modulation

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 7 Addressing unmet need in cholestatic diseases through NTCP modulation NTCP, sodium taurocholate co-transporting polypeptide. References: 1Trivedi PJ, et al. Clin Gastroenterol Hepatol. 2022 Aug;20(8):1687-1700.e4; 2Schreiber RA, et al.

  8. Slide 8: AX-0810 reduces bile acid accumulation in hepatocytes by modulating NTCP activity

    AX-0810 reduces bile acid accumulation in hepatocytes by modulating NTCP activity ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 8 Halilbasic E, et al. J Hepatol. 2013 Jan;58(1):155-68; Nyholm I, et al. J Hepatol. 2025 Aug;83(2):440-452. HEALTHY LIVER Normal bile acids homeostasis in

  9. Slide 9: NTCP modulation reduces fibrosis markers and elevates circulating bile acids

    0.0 0.5 1.0 1.5 Relative mRNA expression NTCP modulation reduces fibrosis markers and elevates circulating bile acids ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 9 NTCP channel blocking increases plasma bile acids concentrations, up to 3-fold in cholestatic disease mouse model Pro-

  10. Slide 10: Assessing Safety, PK and Target Engagement of AX-0810 in First-in-Human Trial

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 10 Assessing Safety, PK and Target Engagement of AX-0810 in First-in-Human Trial CTA, Clinical Trial Application; DMC, Data Monitoring Committee; MAD, Multiple Ascending Dose; PK, Pharmacokinetics; TUDCA, Tauroursodeoxycholic acid; AX-081

  11. Slide 11: Biliary atresia is a severe pediatric disease with no approved therapies

    Biliary atresia is a severe pediatric disease with no approved therapies ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 Adike A, et al. Expert Rev Gastroenterol Hepatol. 2018 Oct;12(10):1025-1032; Verkade HJ, et al. J Hepatol. 2016 Sep;65(3):631-42; Sundaram SS, et al. Liver Transpl.

  12. Slide 12: Biliary atresia as primary indication for AX-0810

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 12 Biliary atresia as primary indication for AX-0810 SEVERITY AND UNMET NEED BIOLOGICAL RATIONALE CLINICAL DE-RISKING CENTRALIZED CARE REGULATORY PATHWAY Leading cause of pediatric liver transplantation and no approved therapies AX-0810 t

  13. Slide 13: AI-guided EON design accelerates discovery

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 13 AI-guided EON design accelerates discovery ~90% faster discovery and up to 6× improvement in EON performance Trained on 12+ years of PROPRIETARY AXIOMER DATA Trained on experimentally-validated editing outcomes of numerous EONS and tar

  14. Slide 14: AX-0811: AI-enabled next-gen RNA editing therapy targeting NTCP

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 14 AX-0811: AI-enabled next-gen RNA editing therapy targeting NTCP for cholestatic diseases ~3x higher editing efficiency • Improved efficacy and dosing convenience over AX-0810 Upcoming development milestones • AX-0811 program advancing

  15. Slide 15: Assessing Safety, PK and Target Engagement of AX-0811 in First-in-Human Trial

    Assessing Safety, PK and Target Engagement of AX-0811 in First-in-Human Trial ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 15 CTA enabling activities ongoing • CTA filing in mid 2026 • Target engagement data expected by year-end 2026 Treatment AX-0811 GalNAc conjugated editing oligo

  16. Slide 16: ProQR building a leading NTCP franchise in cholestatic disease

    NTCP FRANCHISE AX-0811 ProQR building a leading NTCP franchise in cholestatic disease ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 16 Rapid path to patients with high unmet medical need Establishes clinical validation Awareness amongst physicians and centers of excellence AI-discove

  17. Slide 17: AX-0422 RNA editing therapy to address Hurler Syndrome

    AX-0422 RNA editing therapy to address Hurler Syndrome ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 17 GAGs: glycosaminogylcans; MPS1: Mucopolysaccharidosis type I. 1Baldo G, et al, 2018, https://doi.org/10.1111/cge.13224 HURLER SYNDROME • Most severe form of MPS1 • Early onset, mul

  18. Slide 18: Increases in IDUA enzymatic activity drive meaningful clinical impact

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 18 Increases in IDUA enzymatic activity drive meaningful clinical impact A restoration of 1-15% of normal IDUA enzymatic function2 can improve phenotype Scheie Hurler-Scheie Hurler Diagnosis Teens Childhood < 18 months Life expectancy Nor

  19. Slide 19: RNA editing achieves therapeutically meaningful enzyme restoration in Idua mouse model

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 19 RNA editing achieves therapeutically meaningful enzyme restoration in Idua mouse model 1AX-0422 surrogate treatment of Idua-W392X mice, SC, 30 mg/kg, Q1W until 8 wks, data at 8 weeks, n=6, mean, SEM; 2AX-0422 surrogate treatment of Idu

  20. Slide 20: AX-0422 shows differentiated activity vs standard of care in Idua mouse model

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 20 AX-0422 shows differentiated activity vs standard of care in Idua mouse model Greater biomarker reduction and functional improvement vs ERT 1Idua-W392X mice, AX-0422 surrogate treatment: SC, 30 mg/kg, ERT (Laronidase) treatment: IV, 0.

  21. Slide 21: AX-0422 achieves robust, durable CNS editing with functional enzyme restoration

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 21 AX-0422 achieves robust, durable CNS editing with functional enzyme restoration 1AX-0422 surrogate treatment of Idua-W392X mice ICV, 250μg, single dose, n=6, 4 weeks, ddPCR, mean, SEM / western blot, mean, SEM; 2IT administration, 10.6

  22. Slide 22: AX-0422 positioned to redefine the standard of care in Hurler Syndrome

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 22 AX-0422 positioned to redefine the standard of care in Hurler Syndrome Positioned to deliver systemic and neurological benefit through a single, targeted mechanism DIFFERENTIATED APPROACH • Impact liver and neurological driven symptoms

  23. Slide 23: AX-0422 preliminary clinical development

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 23 AX-0422 preliminary clinical development A two-step approach with liver delivery followed by CNS delivery DS: dermatan sulfate; HS: heparan sulfate Subcutaneous administration for Liver • Primary objective: safety, tolerability • Secon

  24. Slide 24: AX-2911 RNA editing therapy to address MASH

    1Sandireddy R, et al. Front Cell Dev Biol. 2024 Jul 16;12:1433857; 2Tsedendorj Yumchinsuren et al., 2025; 3 Sookoian Silvia et al., 2011; 4 Souza Matheus et al., 2024; 5Chen, Yunzhi et al, 2020 ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 24 AX-2911 RNA editing therapy to address me

  25. Slide 25: Editing has functional advantage over knockdown

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 25 Editing has functional advantage over knockdown AX-2911 substantially reduces liver fat vs clinical-stage ASO2 1N=4-6, 20mg/kg, 14 doses, GalNAc conjugated-AX-2911 or 0.7mg/kg, 14 doses, AZ AZD2693 treatment, SC, readout at day D28; 2A

  26. Slide 26: AX-2911 development strategy

    ProQR Therapeutics – Investor & Analyst Event - April 8, 2026 26 AX-2911 development strategy Exploring an Investigator-Initiated Trial (IIT) in China OBJECTIVE Generate early proof-of-concept in patients De-risk the program and inform development strategy ACCELERATED APPROACH Parallel preparation f

  27. Slide 27: ProQR development pipeline and milestones

    Progress undisclosed DEVELOPMENT PIPELINE AX-0810 for Cholestatic diseases NTCP Modulate Target engagement data 1H 2026 ~100K patients AX-0811 for Cholestatic diseases NTCP Modulate Target engagement data in 2026 AX-0422 for Hurler Syndrome IDUA Correct CTA filing early 2027; Clinical biomarkers in

  28. Slide 28: \ITSIN

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