Rezolute, Inc. · NASDAQ:RZLT
Rezolute — Investor Day (November 2025)
40 slides from Rezolute's investor day presentation, Nov 10, 2025
Slide 1: Rezolute Investor Event: Ersodetug Development Program
Rezolute Investor Event: Ersodetug Development Program November 10, 2025
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
Slide 3: Today's Speakers
Today’s Speakers Nevan Charles Elam, J.D. Mansa Krishnamurthy, M.D. Azeez Farooki, M.D. Chief Executive Officer & Founder, Rezolute Pediatric Endocrinologist, Cincinnati Children’s Attending Physician and Clinical Member, Endocrinology Hospital Medical Center Service, Memorial Sloan Kettering Cancer
Slide 4: Today's Agenda
Today's Agenda Welcome & Opening Remarks 01 Nevan Charles Elam, J.D. Congenital Hyperinsulinism (HI): Background, Patient Journey, & Current Treatment Landscape 02 Mansa Krishnamurthy, M.D. Tumor HI: Background, Patient Journey, & Current Treatment Landscape 03 Azeez Farooki, M.D. Overview of Rezolu
Slide 5: Mansa Krishnamurthy, M.D.
Mansa Krishnamurthy, M.D. Pediatric Endocrinologist, Cincinnati Children’s Hospital Medical Center Assistant Professor, Department of Pediatrics, University of Cincinnati
Slide 6: Congenital Hyperinsulinism (HI): Etiology and Epidemiology
Congenital Hyperinsulinism (HI): Etiology and Epidemiology Dysregulated insulin secretion from the pancreatic beta cell causes persistent hypoglycemia • Estimated incidence varies geographically • 1 in 22,000* to 1 in 2,500 live births in consanguineous populations • Two forms: transient and persist
Slide 7: Significant Unmet Clinical Need
Significant Unmet Clinical Need Severe persistent hypoglycemia is the key clinical presentation • Often presents within first month of life; most common cause of hypoglycemia in infants and children • Risk of seizure, coma, and death • Neurologic damage is a concern in all patients and occurs in ~50
Slide 8: Available Therapies Have Sub-Optimal Efficacy and Safety Limitations
Available Therapies Have Sub-Optimal Efficacy and Safety Limitations • First line: Diazoxide (DZ) is the only approved therapy for HI (and is useful in some patients) • Ineffective in the ~60% of patients with KATP mutations (“DZ-unresponsive”) • Serious safety risks including a black box warning fo
Slide 9: A Call to Action
A Call to Action • Diagnosis • Standardized protocols to reduce diagnostic delay • Referral networks/improved access to specialized centers with experience managing patients with HI (multidisciplinary model of care) • Therapies • Better tolerated long-term medical therapies for infants/children with
Slide 10: Azeez Farooki, M.D.
Azeez Farooki, M.D. Attending Physician and Clinical Member, Endocrinology Service, Memorial Sloan Kettering Cancer Center Associate Clinical Professor, Weill Cornell Medical College
Slide 11: Tumor Hyperinsulinism (HI)
Tumor Hyperinsulinism (HI) Caused by pancreatic neuroendocrine tumors (i.e., islet cell tumors/insulinomas) and non-islet cell tumors (NICTs) • Both of which may cause hypoglycemia due to over-activation of the insulin receptor • Surgery or other de-bulking therapies are commonly utilized, but chron
Slide 12: Pancreatic Neuroendocrine/Islet Cell Tumors
Pancreatic Neuroendocrine/Islet Cell Tumors Insulinomas are the most common type of functional islet-cell tumor • Hypoglycemia results from over-activation of the insulin receptor caused by overproduction of insulin • Hypoglycemia is the presenting and characteristic feature of the condition, and co
Slide 13: Insulinoma Patient Journey
Insulinoma Patient Journey Hypoglycemia is the presenting and characteristic feature • Symptoms may include confusion, shakiness, neurological or mental health conditions • Diagnosis may be delayed due to non-specific symptoms • Evaluation and diagnosis typically done by Endocrinologists • Diagnosis
Slide 14: Insulinoma Treatment Landscape
Insulinoma Treatment Landscape Multi-focal or malignant patients require chronic medical management of hypoglycemia • Surgery is considered SOC for benign/solitary tumors • Surgery or de-bulking therapies do not directly treat hypoglycemia • The presence of hypoglycemia can delay or prevent the abil
Slide 15: NICTH
NICTH Tumors may secrete insulin-like substances (e.g., IGF-2 variants) causing hypoglycemia • These growth factors bind to and over-activate the insulin receptor • NICTs are large and generally diagnosed before the onset of hypoglycemia • Incidence of NICTH may be higher than insulinomas due to its
Slide 16: NICTH Patient Journey
NICTH Patient Journey The presentation with and diagnosis of hypoglycemia may occur before or after cancer diagnosis • Due to the severe nature of the hypoglycemia, patients often present in the ER due to loss of consciousness or seizures, where an endocrinologist diagnoses them • Lab profile to con
Slide 17: NICTH Treatment Landscape
NICTH Treatment Landscape Patients require ongoing pharmacological treatment to manage hypoglycemia • Surgery or other de-bulking tumor-directed treatments are considered SOC and offer some hypoglycemia control • ~50% of patients are not candidates for resection or debulking given the advanced stage
Slide 18: Brian Roberts, M.D.
Brian Roberts, M.D. Chief Medical Officer, Rezolute
Slide 19: Ongoing Phase 3: sunRIZE Study in Congenital HI
Ongoing Phase 3: sunRIZE Study in Congenital HI • Global, multi-center, double-blind, randomized, controlled, safety and efficacy registrational study • Patient population (n=56) • Ages 3 months + with inadequate glycemic control on standard of care medical management • Pivotal treatment arms • ~48
Slide 20: sunRIZE Patient Demographics Comparable to Phase 2 Study
sunRIZE Patient Demographics Comparable to Phase 2 Study • Overall enrollment of 63 participants exceeded target of 56 • Average age of 3.4 years (range: 3m – 15y) • Average of 15 hypoglycemia events/week by SMBG • Average daily percent time in hypoglycemia on CGM of 19% • Baseline standard of care
Slide 21: Ongoing Phase 3: upLIFT Study in Tumor HI
Ongoing Phase 3: upLIFT Study in Tumor HI Achieved alignment with FDA on streamlined development path in September 2025 • Global, multi-center, single-arm, open-label registrational study • Patient population (n=~16) • Adult ICT and NICT patients with HI who have not achieved adequate hypoglycemia c
Slide 22: Significant Accomplishments in 2025
Significant Accomplishments in 2025 • Regulatory Accomplishments: • Breakthrough Therapy Designation from FDA for congenital HI • FDA confirmation of the supporting non-clinical and clinical package for initial BLA filing in congenital HI • Breakthrough Therapy Designation from FDA for tumor HI • Al
Slide 23: Sunil Karnawat, Ph.D., M.B.A.
Sunil Karnawat, Ph.D., M.B.A. Chief Commercial Officer, Rezolute
Slide 24: Congenital HI: Market Insights
Congenital HI: Market Insights • Updated Incidence • 1:22,000* versus literature-based 1:28,000+ births • Revised incidence of 165 vs.130 births per year Patients Age Distribution* • Treatment Insights 18+ 10% • >80% of the patients are treated at ~90 Major Children’s Hospitals 11 to 17 16% • ~400+
Slide 25: ~1,500 Initially Addressable Pediatric Patients in U.S.
~1,500 Initially Addressable Pediatric Patients in U.S. Verified in the claims database1 • Hypoglycemia + SOC congenital HI medical therapies* 3,000 Initial market after removing ~40% DZ responsive patients 1,800 Initial market after removing ~5% patients who have 1,650 had surgery over the previous
Slide 26: Vast Majority of Patients Managed at Major Children's Hospitals (MCHs)
Vast Majority of Patients Managed at Major Children’s Hospitals (MCHs) ~80% of congenital HI patients are treated at ~90 MCHs* Lowest Tier 1 MCH Tier 2 MCH Tier 3 MCH Tier 4 2 centers of excellence ~35 large academic hospitals ~50 small academic hospitals ~250 community clinics ~25% ~30% ~25% ~20% *
Slide 27: Patient Preferences Influence Infusion Journey
Patient Preferences Influence Infusion Journey ADOLESCENTS INFANTS CHILDREN AND ADULTS Infants require specialized Children may be suitable for Patients aged 10 and older staff, close monitoring, and infusion therapy in less may benefit from flexible dedicated vein-finding intensive and more infusio
Slide 28: Payers Acknowledge Ersodetug's Compelling Efficacy
Payers Acknowledge Ersodetug’s Compelling Efficacy • Payers Believe: Payer mix estimate • High unmet need in congenital HI 2% • Ersodetug has demonstrated meaningful efficacy 8% • Payers Anticipate: 17% • Ultra-rare pediatric disease pricing • DZ trial before use of ersodetug 61% 12% • Payer Mix: •
Slide 29: Tumor HI
Tumor HI
Slide 30: Malignant Insulinoma: Market Insights
Malignant Insulinoma: Market Insights Persistent uncontrolled hypoglycemia is the characteristic feature • Insulinoma is a clearly defined market with high unmet need and an established prescriber base • Generally managed at ~300 academic centers/national cancer institutes • Adult endocrinologists p
Slide 31: ~1,500 Initially Addressable Malignant Insulinoma Patients in U.S.
~1,500 Initially Addressable Malignant Insulinoma Patients in U.S. Malignant insulinoma patients identified in claims 3,000 (includes two or more C25.4 or E31.21+) ~40% patients refractory to surgery and medical management 1,200 including DZ Initial commercial effort: refractory patient population a
Slide 32: NICTH: Market Insights
NICTH: Market Insights Severe hypoglycemia typically presents after tumor diagnosis • Primary tumor produces excessive insulin-like substances (e.g., IGF-2 variants) that over stimulate the insulin receptor • Current management of NICTH • Tumor-directed therapies used to remove or debulk the tumo
Slide 33: ~1,500 Initially Addressable NICTH Patients in U.S.
~1,500 Initially Addressable NICTH Patients in U.S. Severe NICTH patients identified in claims* 6,000 (Tumor Diagnosis + Hypoglycemia + Steroids + Hospitalization) ~40% patients refractory to SOC (tumor-directed and/or steroids) and requiring hospital stays + IV glucose* 2,400 Initial commercial eff
Slide 34: Majority of Tumor HI Patients Managed at Academic Centers
Majority of Tumor HI Patients Managed at Academic Centers ~60% of tumor HI patients are treated at ~300 academic centers Lowest Tier 1 Centers Tier 2 Centers Tier 3 Centers 66 NCI-designated academic centers+ ~220 non-NCI academic centers ~1,100 community centers ~30% ~30% ~40% +There are 73 Nationa
Slide 35: Combined Commercial Opportunity
Combined Commercial Opportunity
Slide 36: Weight-based Dosing Applies to Both Indications
Weight-based Dosing Applies to Both Indications Tumor HI patients require ~3X more vials compared to congenital HI patients • Each vial is 80 mg/mL • Congenital HI maintenance dose: 10 mg/kg • Pediatric patient average weight: ~24 kg • Patients will use 3 vials per infusion • 39 vials per year per p
Slide 37: Initial Combined U.S. Market Opportunity: 4,500 Patients
Initial Combined U.S. Market Opportunity: 4,500 Patients • Congenital HI Market • Pediatric ultra-rare disease pricing • Lead indication establishes clinical effectiveness and payer access pathway for ersodetug in HI • Addressable market of ~1,500 pediatric patients • Tumor HI Market • Malignant Ins
Slide 38: Christen Baglaneas
Christen Baglaneas Head of Corporate Affairs, Rezolute
Slide 39: Real-world Patient Benefit of Ersodetug
Real-world Patient Benefit of Ersodetug Significantly improving quality of life for patients and their families • 13 patients with tumor HI treated in our Expanded Access Program (EAP) to date • Hospitalized and in life-threatening or hospice-bound condition • Discontinued tumor-directed therapies (
Slide 40: Q&A
Q&A