Rezolute, Inc. · NASDAQ:RZLT
Rezolute — Investor Presentation (May 2025)
27 slides from Rezolute's investor presentation, May 2025
Slide 1: A Late-stage Rare Disease Company Treating Hyperinsulinism
A Late-stage Rare Disease Company Treating Hyperinsulinism Corporate Presentation NASDAQ: RZLT
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation, like many written and oral communications presented by Rezolute and our authorized officers, may contain certain forward-looking statements regarding our prospective performance and strategies within the meaning of Section 27A of the Securities Act and S
Slide 3: A Rare Disease Company Treating Hyperinsulinism
A Rare Disease Company Treating Hyperinsulinism RZ358 (ersodetug) is Two rare disease Compelling real- Total $1B+ global Seasoned an antibody Phase 3 programs world evidence of market opportunity management team designed to treat evaluating patient benefit with additional with demonstrated hypoglyce
Slide 4: Management Team
Management Team Nevan Charles Elam Brian Roberts Daron Evans Founder & Chief Executive Officer Chief Medical Officer Chief Financial Officer Susan Stewart Michael Deperro Chief Regulatory Officer SVP, Corporate Development | 4
Slide 5: Two Phase 3 Indications Targeting Hyperinsulinism
Two Phase 3 Indications Targeting Hyperinsulinism IND- Next Milestone Program Target Phase 1 Phase 2 Phase 3 Enabling Milestone Expected RZ358 Congenital December Topline data (ersodetug) Hyperinsulinism 2025 Patient RZ358 Tumor enrollment in 1H 2025 (ersodetug) Hyperinsulinism Phase 3 study | 5
Slide 6: Ersodetug RZ358
RZ358 Ersodetug Treatment for Hyperinsulinism (HI)
Slide 7: Hypoglycemia as a Result of HI
Hypoglycemia as a Result of HI Hypoglycemia o Severe, persistent, life-threatening complication of over activation of the insulin receptor o Consequence of multiple forms of HI o Lack of effective treatment options congenital HI tumor HI Rare pediatric genetic disease Rare disease caused by tumors t
Slide 8: Antibody Designed to Treat All Forms of HI
Antibody Designed to Treat All Forms of HI o Fully human monoclonal antibody with a INSULIN or IGF-2 GLUCOSE novel mechanism acting downstream from production source (e.g. pancreas) Ersodetug Increases o Allosterically binds to the insulin receptor Available Blood to counteract excess signaling by i
Slide 9: Congenital HI
Congenital HI Congenital HI
Slide 10: Disease Background
Disease Background o 1 in 28,000 live births in the US1, translating to approximately 130 new patients per year o Often presents within first month of life o Most common cause of persistent hypoglycemia in infants and children o Requires constant monitoring as serious hypoglycemic lows are often mis
Slide 11: Inadequate Standard of Care
Inadequate Standard of Care o Diazoxide (DZ) is first line treatment and the only approved medication for hypoglycemia caused by HI • 60% of patients do not respond to DZ • May experience frequent and serious adverse reactions including volume overload, heart failure, and pulmonary hypertension • Pa
Slide 12: Therapies in Development
Therapies in Development Ersodetug is a fully human monoclonal antibody designed to treat all forms of HI and has shown substantial benefit in clinical trials and real-world use Asset Mechanism Stage Dosing HI Indication Clinical Barriers Insulin receptor IV1, once Congenital, Ersodetug Phase 3 N/A
Slide 13: Phase 3: The sunRIZE Study
Phase 3: The sunRIZE Study o Global, multi-center, double-blind, randomized, controlled, safety and efficacy registrational study o Patient population (n=56) • Ages 3 months + who do not have adequate glycemic control with standard of care medical management o Primary endpoint: change in average hyp
Slide 14: Phase 2b RIZE Study Results
Phase 2b RIZE Study Results o 23 participants • Average age ~6.5 (16 participants were between 2-6 years of age) • Diverse group across gender and genetics o ~20% average daily time in hypoglycemia and 13 hypoglycemia events per week at baseline • Participants were on standard of care o Predictable
Slide 15: Substantial Improvement in All Hypoglycemia Metrics
Substantial Improvement in All Hypoglycemia Metrics Pooled 6 and 9 mg/kg dose levels representative of Phase 3 population and dosing Hypoglycemia (<70 mg/dL) Severe Hypoglycemia (<50 mg/dL) Event Rate by BGM (N=15∧) Time by CGM [N=15∧] Event Rate by BGM (N=15∧) Time by CGM [N=15∧] p < 0.0005 p< 0.05
Slide 16: Compelling Patient Responses
Compelling Patient Responses Average Daily Time in Hypoglycemia ~40% On Treatment RZ358 6 mg/kg 2-Year-Old on SSA 93% Improvement Average Daily Time in Hypoglycemia ~45% On Treatment RZ358 9 mg/kg 50 40 6-Year-Old; Hypoglycemia 30 % Time Failed meds, pancreatectomy 20 10 0 Baseline End of Treatment
Slide 17: Addressable U.S. Market Driven by Both Diazoxide-Responsive and Unresponsive Patients
Addressable U.S. Market Driven by Both Diazoxide-Responsive and Diazoxide-Unresponsive Patients DZ Tolerated Responsive to Diazoxide ~50 pts/yr Side Effects w/ DZ ~130 cHI Patients Unresponsive Current SoC: Addressable Diagnosed in US per to Diazoxide Pancreatectomy population of ~1,500 Year1 ~60 pt
Slide 18: Tumor HI
REZOLUTE @} Tumor HI
Slide 19: Disease Background
Disease Background o Hypoglycemia caused by two distinct tumor types: • Islet Cell Tumors (ICT) • Excessive secretion of insulin • Malignant insulinomas are the most common ICTs that cause hypoglycemia • Non-Islet Cell Tumors (NICT) • Produce and secrete insulin-like substances such as IGF-2 that
Slide 20: Treatment Options and Unmet Need
Treatment Options and Unmet Need o Tumor-directed therapies do not directly treat hypoglycemia • Adequate hypoglycemia management is required prior to initiation of tumor-targeted therapies o Therapies to treat malignant insulinoma are often ineffective or poorly tolerated • Diazoxide (DZ) is the on
Slide 21: Real-world Patient Benefit in Expanded Access Program of Ersodetug
Real-world Patient Benefit in Expanded Access Program of Ersodetug o Multiple ICT patients with severe refractory hypoglycemia • Hospitalized and in life-threatening or hospice-bound condition • Required continuous high volume/concentration intravenous dextrose or nutritional infusion • Tumor-direct
Slide 22: Phase 3 Study Overview
Phase 3 Study Overview o Multi-center, double-blind, randomized, controlled, safety and efficacy registrational study o Patient population (n= up to 48) • Adult ICT and NICT patients with HI who have not achieved adequate hypoglycemia control with SOC therapies • 24 participants in double-blind, pla
Slide 23: Immediately Addressable U.S. ICT Market
Immediately Addressable U.S. ICT Market Malignant Insulinoma Hypoglycemia (Hypo) Diagnosis and Treatment Pathway1 ~25% Further surgery or ~40% No Further HI other de-bulking Treatments ~60% Refractory Hypo, HI Treatment Post- >500 Immediately Surgery 5-Year Prevalence ~55% ~225 pts Addressable Patie
Slide 24: Immediately Addressable U.S. NICT Market
Immediately Addressable U.S. NICT Market Hepatocellular Carcinoma + Hypoglycemia (Hypo) Diagnosis and Treatment Pathway1 ~40% ~50% Further surgery or No Further HI other de-bulking Treatments ~50% Refractory Hypo, HI Treatment Post- >1,000 Immediately Surgery 5-Year Prevalence ~20% ~800 pts Addressa
Slide 25: Commercial Opportunity
Commercial Opportunity o Potential to Address Two Rare Disease Markets • ~1,500 addressable cHI patients in US; equivalent patient population in Europe • >500 islet cell tumor patients and >1,000 non-islet cell tumor patients addressable in the US o Highly Concentrated Physician Base for cHI • 60% o
Slide 26: A Rare Disease Company Treating Hyperinsulinism
A Rare Disease Company Treating Hyperinsulinism Mission-driven to RZ358 (ersodetug) is Compelling real- Total $1B+ global improve outcomes an antibody world evidence of market opportunity for individuals with designed to treat patient benefit with additional severe hypoglycemia hypoglycemia under th
Slide 27: REZOLUTE @}
REZOLUTE @}