Rezolute, Inc. · NASDAQ:RZLT
Rezolute — Investor Day (November 2025)
40 slides from Rezolute's investor day presentation, Nov 12, 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 0 2 © 2025 Rezolute, Inc. Proprietary and Confidential. 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 wit
Slide 3: Today's Speakers
Today’s Speakers 03 © 2025 Rezolute, Inc. Proprietary and Confidential. Mansa Krishnamurthy, M.D. Pediatric Endocrinologist, Cincinnati Children’s Hospital Medical Center Assistant Professor, Department of Pediatrics, University of Cincinnati Nevan Charles Elam, J.D. Chief Executive Officer & Founde
Slide 4: Today's Agenda
Today's Agenda 04 © 2025 Rezolute, Inc. Proprietary and Confidential. 01 Welcome & Opening Remarks Nevan Charles Elam, J.D. 02 Congenital Hyperinsulinism (HI): Background, Patient Journey, & Current Treatment Landscape Mansa Krishnamurthy, M.D. 03 Tumor HI: Background, Patient Journey, & Current Tre
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 • Estimated incidence varies geographically • 1 in 22,000* to 1 in 2,500 live births in consanguineous populations • Two forms: transient and persistent • Persistent: chronic, >1 year • Average of 15 years particularly in patients with KATP
Slide 7: Significant Unmet Clinical Need
Significant Unmet Clinical Need • 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% of patients • Brain is highly dependent on glucose as its prim
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 warnin
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 wi
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) • Both of which may cause hypoglycemia due to over - activation of the insulin receptor • Surgery or other de - bulking therapies are commonly utilized, but chroni c medical management with hypoglycemia - directed therapies is necessary for multi - focal or malignant tumor
Slide 12: Pancreatic Neuroendocrine/Islet Cell Tumors
Pancreatic Neuroendocrine/Islet Cell Tumors • 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 correction of hypoglycemia is the primary management goal • 85% -
Slide 13: Insulinoma Patient Journey
Insulinoma Patient Journey • 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 made by: • 48 - 72 hour fast with confirmation of Whipp
Slide 14: Insulinoma Treatment Landscape
Insulinoma Treatment Landscape • 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 ability to receive such therapies • Chronic medical management with hypoglycemia - dire
Slide 15: NICTH
NICTH • 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 occurrence in 15+ large tumor types, underdiagnosis, and lack of physician and patie
Slide 16: NICTH Patient Journey
NICTH Patient Journey • 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 confirm NICTH (e.g., typically low insulin in the setting of hypoglycemia) • Tumor s ize impacts t
Slide 17: NICTH Treatment Landscape
NICTH Treatment Landscape • 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 of the tumor • Limited hypoglycemia - directed treatments available f
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 •
Slide 20: sunRIZE Patient Demographics
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
• 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 control • Primary endpoint: number of participants achieving ≥50% reduction from baseline IV glucose requirements (
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 •
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
© 2025 Rezolute, Inc. Proprietary and Confidential. 24 18% 23% 33% 16% 10% 0% 10% 20% 30% 40% 0 to 2 3 to 5 6 to 10 11 to 17 18+ Patients Age Distribution* Congenital HI: Market Insights • Updated Incidence • 1:22,000* versus literature - based 1:28,000 + births • Revised incidence of 165 vs.130 bir
Slide 25: Initially Addressable Pediatric Patients in U.S.
© 2025 Rezolute, Inc. Proprietary and Confidential. 25 Verified in the claims database 1 • Hypoglycemia + SOC congenital HI medical therapies* 1 Claims database: Forian and Veeva Compass. *SOC = Standard of Care which includes Diazoxide (DZ), Somatostatin Analogs (SSAs), and NTP. There is no s pe ci
Slide 26: Vast Majority of Patients Managed at Major Children's Hospitals
© 2025 Rezolute, Inc. Proprietary and Confidential. 26 Lowest * Based on Forian and Veeva Compass claims analysis. Vast Majority of Patients Managed at Major Children’s Hospitals (MCHs) ~25% ~30% ~25% ~20% 2 centers of excellence ~35 large academic hospitals ~50 small academic hospitals ~250 communi
Slide 27: Patient Preferences Influence Infusion Journey
© 2025 Rezolute, Inc. Proprietary and Confidential. 27 Patient Preferences Influence Infusion Journey I NFANTS C HILDREN A DOLESCENTS AND A DULTS Children may be suitable for infusion therapy in less intensive and more convenient settings ( i .e., at home ) Patients aged 10 and older may benefit fro
Slide 28: Payers Acknowledge Ersodetug's Compelling Efficacy
© 2025 Rezolute, Inc. Proprietary and Confidential. 28 Payers Acknowledge Ersodetug’s Compelling Efficacy • Payers Believe: • High unmet need in congenital HI • Ersodetug has demonstrated meaningful efficacy • Payers Anticipate: • Ultra - rare pediatric disease pricing • DZ trial before use of ersod
Slide 29: Tumor HI
Tumor HI a
Slide 30: Malignant Insulinoma: Market Insights
© 2025 Rezolute, Inc. Proprietary and Confidential. 30 Malignant Insulinoma: Market Insights • 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 primarily tre
Slide 31: Initially Addressable Malignant Insulinoma Patients
© 2025 Rezolute, Inc. Proprietary and Confidential. 31 Malignant insulinoma patients identified in claims (includes two or more C25.4 or E31.21 + ) ~ 40% patients refractory to surgery and medical management including DZ Initial commercial effort: refractory patient population at nationally recogniz
Slide 32: NICTH: Market Insights
© 2025 Rezolute, Inc. Proprietary and Confidential. 32 NICTH: Market Insights • 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: Initially Addressable NICTH Patients in U.S.
© 2025 Rezolute, Inc. Proprietary and Confidential. 33 * Analysis identified patients in the Forian and Veeva Compass claims database that matched phase 3 tumor HI clinical inclusion/exclusion criteria. ~1,500 Initially Addressable NICTH Patients in U.S. Severe NICTH patients identified in claims* (
Slide 34: Majority of Tumor HI Patients Managed at Academic Centers
© 2025 Rezolute, Inc. Proprietary and Confidential. 34 © 2025 Rezolute, Inc. Proprietary and Confidential. +There are 73 National Cancer Institutes (NCI); however, 7 of them are 'Basic Laboratory Cancer Centers' focused on research and not clinical care (e.g., Salk Institute, Cold Spring Harbor), so
Slide 35: Combined Commercial Opportunity
Combined Commercial Opportunity
Slide 36: Weight-based Dosing Applies to Both Indications
© 2025 Rezolute, Inc. Proprietary and Confidential. 36 • 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 patient assuming infusion every four weeks • Tumor HI maintenance dose
Slide 37: Initial Combined U.S. Market Opportunity
© 2025 Rezolute, Inc. Proprietary and Confidential. 37 • 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
• 13 patients with tumor HI treated in our Expanded Access Program (EAP) to date • Hospitalized and in life - threatening or hospice - bound condition • Discontinued t umor - directed therapies (e.g., embolization, radiotherapy, chemotherapy) • U niversal hypoglycemia improvement enabling hospital d
Slide 40: Q&A
Q&A