Slide 1: RZ358 CHI: Program Updates
RZ358 CHI: Program Updates 22 Sep 2019 Copyright 2019. 1
Slide 2: Background
Background Copyright 2019. 2
Slide 3: Rezolute: A Metabolic and Orphan Disease Company With a Diversified Pipeline
Rezolute: A Metabolic and Orphan Disease Company With a Diversified Pipeline Program Description Preclinical Phase 1 Phase 2 RZ358 Antibody for CHI Phase 2b dosing anticipated 2H’19 IND anticipated mid- RZ402 Oral PKI for DME year ’20 AB101 Weekly insulin Top-line results anticipated 2H’19 Copyright
Slide 4: Observational Study Demonstrates Unmet Need
Observational Study Demonstrates Unmet Need Conducted in partnership Continuous Glucose Monitoring (CGM) for Two Weeks: Summary of Results • Blood glucose <70 mg/dL is hypoglycemia • On average, patients had ~3 hours / day (~180 min) of hypoglycemia, even on standard of care (SOC) medications • Youn
Slide 5: Unique Mechanism Attenuates Insulin Effects
Unique Mechanism Attenuates Insulin Effects Circulation Beta Cells Orthosteric Target Cells Site Pancreas Insulin Liver, Fat, Muscle • 11 known distinct gene defects Insulin Recept or • Failure to suppress insulin • Reduced signaling secretion in the setting of hypoglycemia Allosteric Site RZ358 • H
Slide 6: RZ358 Works Like a Freeway Metering Light
RZ358 Works Like a Freeway Metering Light Copyright 2019. 6
Slide 7: Potential to Address Limitations of Standard of Care
Potential to Address Limitations of Standard of Care Glucos e Target Organs Pancreas Insulin RZ358 (Broad Focus) Standard of Care (Narrow Focus) Development Tailored for CHI Not developed for CHI Insulin receptor/signal on insulin-dependent target Targeting Beta cell only tissues Relevancy Potential
Slide 8: Phase 2a – Completed Proof of Concept
Phase 2a – Completed Proof of Concept Blood Glucose (BG) Design Time in Target Range (70-180 mg/dL) • Single IV doses of 1 to 9 mg/kg in patients with CHI 100 • Ages ≥ 12 in Europe and ≥ 18 in the US 80 Results Percent Time 60 • PK comparable with healthy volunteers 100 Improvement in Hypoglycemia w
Slide 9: RZ358-606: Phase 2b Study Protocol
RZ358-606: Phase 2b Study Protocol Copyright 2019. 9
Slide 10: RZ358-606: Phase 2b Study Overview
RZ358-606: Phase 2b Study Overview • Design: Open-label, repeat-dose study in 4 sequential ascending dosing cohorts (6-8 patients per cohort) • Population: CHI ≥ 2 years old with baseline hypoglycemia by specified CGM thresholds • Duration of individual participation: ~27 weeks • Principal assessmen
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Slide 12: What CHI does to the body…
What CHI does to the body… If you live with CHI, you live with the uncontrolled release of insulin from the pancreas which floods your body, leaving you with dangerously low blood sugar levels. 12
Slide 13: What is RZ358?
What is RZ358? RZ358 is an antibody that guards against the chaos of uncontrolled insulin release by binding to cells throughout the body and limiting RZ358 the ability of insulin to read and signal . 13
Slide 14: What are some things that will be done during the study?
What are some things that will be done dur ing the study? Fasting will occur and the patient may be asleep for part of it. RZ358 Blood is an antibody samples may administered be taken. by IV over 30 minutes. 14
Slide 15: What are some things the doctor will ask during study?
What ar e some things the doctor will ask dur ing study? Check blood with a Glucometer. Fill out a da ily elect ronic dia ry. Wear a CGM Device on Today I felt… your belly. 15
Slide 16: RZ358 has been tested in previous studies in patients with CHI and healthy volunteers
RZ358 has been tested in previous studies in patients with chi and healthy volunteer s. 16
Slide 17: Road Map to Study Start
Road Map to Study Start 1 . Health Authority Submissions • Country Level • Local Level 2 . Site Identification and Start - up Activities • Site Needs • Patient/Caregiver Needs • Training 3 . Site Activations in Q4 2019 • Screening • Enrollment 17
Slide 18: Clinical Trial Participation – Your rights!
Clinical Trial Participation – Your rights! • To receive adequate information (written and verbal) about: • Aim of the study • What will happen during the study and what is expected of you: procedures, medications, diaries, etc. • Which possible benefits AND possible risks or side effects you can ex
Slide 19: Clinical Trial Participation – Your responsibilities
Clinical Trial Participation – Your responsibilities • To follow instructions with regard to: • Study drug intake • Visit schedule • Maintaining diary • Other study procedures (in this case: CGM-device) • Report to the site’s study team: • Any observation/untoward event (possible side effect) • Any
Slide 20: Questions?
Questions? Copyright 2019. 20