Palvella Therapeutics, Inc. · NASDAQ:PVLA

Palvella Therapeutics — Technical Presentation (June 2019)

11 slides from Palvella Therapeutics's technical presentation, Jun 13, 2019

  1. Slide 1: Phase 2a Study of Hepcidin Antagonist PRS-080 in Anemic CKD Patients on Hemodialysis

    Lutz Renders Department of Nephrology Klinikum rechts der Isar Technical University of Munich A PHASE 2A STUDY TO EVALUATE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF REPEATED ADMINISTRATIONS OF THE HEPCIDIN ANTAGONIST PRS-080 OVER 4 WEEKS IN ANEMIC CHRONIC KIDNEY DISEASE PATIENTS UNDERGOI

  2. Slide 2: Antagonizing Elevated Hepcidin Levels in Anemias of Chronic Disease

    Antagonizing Elevated Hepcidin Levels in Anemias of Chronic Disease  Hepcidin is elevated in multiple chronic inflammatory conditions associated with anemia – Infections, cancer, rheumatoid arthritis (RA), chronic kidney disease (CKD)  Iron metabolism is regulated by hepcidin/ferroportin – Hepcidi

  3. Slide 3: PRS-080: Phase 1b & Phase 2a Outline

    PRS-080: Phase 1b & Phase 2a Outline Patient Population: ESRD/CKD patients on dialysis  Ferritin ≥ 300 ng/ml  Hepcidin 5-75 nM Phase 1b: Single Administration Phase 2a: Repeated Administration  Hb 9 – 12 g/dL  Hb < 10.5 g/dL  TSAT < 40%  TSAT < 30% Dose Level 2: 8mg/kg Dose Level 3: 8mg/kg 4 +

  4. Slide 4: PRS-080: Phase 1b & Phase 2a (P2a) Outline

    PRS-080: Phase 1b & Phase 2a (P2a) Outline Patient Population: ESRD/CKD patients on dialysis  Ferritin ≥ 300 ng/ml  Hepcidin 5-75 nM Phase 1b: Single Administration Phase 2a: Repeated Administration  Hb 9 – 12 g/dL  Hb < 10.5 g/dL  TSAT < 40%  TSAT < 30% Dose Level 2: 8mg/kg Dose Level 3: 8mg/

  5. Slide 5: Mean Iron Values of 4 mg/kg Patient Group

    P2a: Mean Iron Values of 4 mg/kg Patient Group  Iron response and mobilization in serum iron after each dose of PRS-080 in drug-treated patients  No iron response in placebo-treated patients 5

  6. Slide 6: Mean TSAT% Values of 4 mg/kg Patient Group

    P2a: Mean TSAT% Values of 4 mg/kg Patient Group  Mobilization of iron in TSAT after each dose of PRS-080 in drug- treated patients  No iron response in placebo-treated patients 6

  7. Slide 7: Mean Iron and TSAT% Values of 8 mg/kg Patient Group

    P2a: Mean Iron and TSAT% Values of 8 mg/kg Patient Group  Iron response and mobilization in both serum iron and TSAT after each dose of PRS-080 in drug- treated patients  Slightly higher peak iron response in the 8 mg/kg treatment group vs 4 mg/kg group No iron response in placebo-treated patient

  8. Slide 8: At 8mg/kg, Preliminary Evidence of an Increase in Hb With PRS-080 Treatment

    P2a: At 8mg/kg, Preliminary Evidence of an Increase in Hb With PRS-080 Treatment Compared to Placebo Group  Both Placebo and PRS-080 groups with no iron administration during study  Modest increase in Hb in the treated patient group  Decline in the placebo group, possibly related to discontinuati

  9. Slide 9: Hepcidin Analysis – Dose-Dependent Rebound

    P2a: Hepcidin Analysis – Dose-Dependent Rebound  Hepcidin rebound does not reduce TSAT and iron response 9

  10. Slide 10: PRS-080 half-life in CKD patients

    P2a: PRS-080 half-life in CKD patients PRS-080 concentration-time profiles (4 mg/kg) PRS-080 concentration-time profiles (8 mg/kg) 1000 1000 PRS-080 plasma concentration, µg/mL PRS-080 plasma concentration, µg/mL 100 100 10 10 1 0.1 1 12 13 15 16 22 23 24 26 0.01 0 168 336 504 672 840 1008 1176 1344

  11. Slide 11: Phase 2a Multidose Study of PRS-080: Summary

    Phase 2a Multidose Study of the Hepcidin Inhibitor PRS-080 in Anemic Chronic Kidney Disease Patients Undergoing Hemodialysis: Summary  PRS-080 was safe and well tolerated at both 4 mg/kg and 8 mg/kg treatment dose levels (data not shown)  No treatment-related adverse events (AEs) or serious advers

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