Corporate Presentation
A Metabolic and Orphan Disease Company
NASDAQ: RZLT
Forward Looking Statements
The information provided herein contain "forward-looking statements" within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These Forward-Looking statements include, but are not limited to, statements regarding the sunRIZE clinical study, the DME RZ402 study, the RIZE study, the ability of RZ358 and RZ402 to become effective treatments, the effectiveness or future effectiveness of RZ358 and RZ402 as treatments, and statements regarding clinical trial timelines for either treatment. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on Rezolute's current beliefs, expectations and assumptions regarding the future of its businesses, results of and timing of clinical trials, financial condition and results of operations, future plans and strategies, projections, anticipated events and trends, the economy and other future conditions. Because forward- looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of Rezolute's control. Rezolute's actual results including the timing and results of clinical trials may differ materially from those indicated in the forward- looking statements. Therefore, you should not place undue reliance on any of these forward-looking statements. Important factors that could cause Rezolute's actual results including the timing and results of clinical trials to differ materially from those indicated in the forward-looking statements are discussed or identified in Rezolute's filings made with the U.S. Securities and Exchange Commission. Any forward-looking statements made by Rezolute in this information are based only on information currently available to Rezolute and speak only as of the date on which the statement is made. Rezolute undertakes no obligation to update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise except as required by applicable law.
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Creating Long-term Value By Advancing Transformative Therapies
RZ358: Monoclonal antibody addressing all forms of hyperinsulinism (HI)
- Congenital HI: Global Phase 3 trial (sunRIZE); topline results expected mid-2025
- Tumor HI: success in multiple cases under our Expanded Access Program (EAP)
RZ402: Once daily oral therapy in Phase 2 for diabetic macular edema (DME)
- Potential to change treatment paradigm dominated by eye injections
• Topline results expected Q2 2024
Seasoned management team
• Demonstrated success from early development through commercialization
Cash runway through Q3 2025
• Past topline results for both programs
Strong investor base focused on long-term value
Each program has potential >$1B+ market opportunity
- Potential upside with expanded indications
EAP: expanded access program. | | | 3 |
Pipeline
Program | Target | Status | Preclinical | Phase 1 | Phase 2 | Phase 3 |
Congenital Hyperinsulinism (HI) | Currently | Mid- |
rarepediatric disease | enrolling | 2025 |
RZ358
Tumor Hyperinsulinism (HI) | EAP Enabled | Alignment with FDA for potential |
raredisease | Emergency Use | late-stage clinical study |
Authorization |
RZ402 | Oral PKI for Diabetic Macular | Fully enrolled | 2Q 2024 |
Edema (DME) | |||
EAP: expanded access program | | 4 |
RZ358
Antibody for Hyperinsulinism (HI)
An Antibody Created to Address all Forms of HI
- RZ358 allosterically binds to the insulin receptor to modulate the signaling effect of ligands such as insulin and other substances to maintain glucose values in a healthy range
- Novel mechanism of action by operating downstream from pancreatic insulin over- secretion (usual SOC target)
- Administered by IV infusion every 2 to 4 weeks
LIGANDS | GLUCOSE |
IncreasesGLUT4
Available Blood
Glucose
RZ358
Reduces
Ligand
Signaling
Reduces
Glucose
Uptake
HI: hyperinsulinism. SOC: standard of care. GLUT4: glucose transporter type 4. | | | 6 |
Congenital HI
Pediatric rare genetic disease characterized by dysregulated and excessive insulin production
Congenital HI Disease Background
- 1 in 28,000 live births in the US
- 25 years of treatment required on average
- ~3500 cases in the US
- Often presents within first month of life
- Most common cause of persistent hypoglycemia in infants and children
- Symptoms often not recognized until life-threateningo Risk of coma, death, and other serious complications o 50% of children have neurological deficiencies
- No therapy has been developed and approved for this indication
HI: hyperinsulinism. SOC: standard of care. 1. Banerjee I, et al. Orphanet J Rare Dis. 2022;17(1):61.
Psychosocial and | Carbohydrate |
Financial Burden | Supplementation |
"Everything I see just | Dependence on |
drives home the fact we | nutritional support |
will never lead a normal | limits the ability to |
life. So many of my hopes | have normal lives, |
and dreams for our | especially in social |
family and for our little | situations1 |
one are shattered."1 |
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Available Treatment Options are Suboptimal
Ineffective in 50% of patients (with KATP mutations) FDA black box for pulmonary hypertension Fluid retention affecting heart/lungs Facial changes and excessive hair growth
Current formulations short-acting Temporary measure for emergent glucose correction Repeated use may deplete liver glucose stores Long-term efficacy is modest
Marginal efficacy, effects wane with repeat dosing Gastrointestinal side effects Risk of NEC, particularly in newborns
Potential interaction with pituitary hormones (growth and thyroid)
Diazoxide Carbohydrate
[Approved] Supplementation
Glucagon Pancreatectomy
[Off label]
Somatostatin Analogues
[Off label]
Frequent, glucose-enriched oral feeds or continuous enteral feeds via NG/GT Multifactorial feeding aversions affect a large proportion of children with cHI
Negative impact on normal daily activities and social interactions
Resection for diffuse disease
Invasive procedure, not done globally
Hypoglycemia may persist for years in up to half of patients
Eventually insulin therapy required
Requires adjuvant medical management, and/or repeat surgery
Resection for focal disease
Limited number of overall cases
Only done at specialized centers
Can be curative for patients
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Congenital HI Treatment Landscape
Diagnosis and Treatment Pathway
~3,500 congenital HI
patients in the US
Diazoxide Trial
Period
(up to 15 mg/kg/day)
HI: hyperinsulinism. DZ: diazoxide.
After 5-Day Treatment:
40%
Responsive
to DZ
Unresponsive
to DZ 60%
~70%
Tolerated
Unacceptable
Side Effects
~30% or 420 pts
~60% or 2100 pts Diffuse
Focal
~40%
RZ358
Addressable
Patient
Population
~2,500
Surgical
Candidates
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Rezolute Inc. published this content on 06 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 06 March 2024 12:40:24 UTC.