[Executive Summary](index=1&type=section&id=executive-summary) This section summarizes Rezolute, Inc.'s focus on hyperinsulinism, recent pipeline progress, CEO's strategic outlook, and key financial results for FY2025 [Introduction and Company Focus](index=1&type=section&id=introduction-company-focus) Rezolute, Inc. is a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism, reporting financial results for the fourth quarter and full fiscal year ended June 30, 2025 - Rezolute, Inc. (Nasdaq: RZLT) is a late-stage rare disease company[1](index=1&type=chunk) - The company focuses on treating hypoglycemia caused by hyperinsulinism[1](index=1&type=chunk) - Financial results are reported for the fourth quarter and full fiscal year ended June 30, 2025[1](index=1&type=chunk) [CEO's Strategic Outlook](index=1&type=section&id=ceo-strategic-outlook) CEO Nevan Charles Elam highlighted substantial progress across both congenital and tumor hyperinsulinism indications for ersodetug, noting FDA alignment on a streamlined Phase 3 trial for tumor HI and anticipating topline results from the sunRIZE trial in December 2025 - Substantial progress has been made across two indications for ersodetug: **congenital and tumor hyperinsulinism**[2](index=2&type=chunk) - FDA alignment on a streamlined Phase 3 trial in tumor hyperinsulinism is seen as recognition of ersodetug's broad applicability and transformative potential[2](index=2&type=chunk) - Topline results from the sunRIZE trial are expected in **December 2025**[2](index=2&type=chunk) [Recent Pipeline Progress and Anticipated Milestones](index=1&type=section&id=recent-pipeline-progress-anticipated-milestones) This section details the latest advancements in ersodetug's clinical development for congenital and tumor hyperinsulinism, including enrollment completion and expected topline results [Congenital Hyperinsulinism (HI) Program](index=1&type=section&id=congenital-hyperinsulinism-hi-program) Rezolute completed enrollment for the sunRIZE Phase 3 study of ersodetug for congenital HI, exceeding targets with 62 participants, and expects topline results in December 2025 - Completed enrollment in sunRIZE, a Phase 3 study for congenital HI, exceeding enrollment with **62 participants** (approximately **15% from U.S. sites**)[5](index=5&type=chunk) - Topline results from the sunRIZE trial are expected in **December 2025**[5](index=5&type=chunk) - **Preliminary Patient Demographics And Baseline Characteristics From A Phase 3 Study (sunRIZE):** | Characteristic | Value | | :------------- | :---- | | Average Age | 3.4 years (35% <2 years old) | | Hypoglycemia Events | 15 (average) per week | | Daily Percent Time in Hypoglycemia | 19% | | Taking ≥1 SOC treatments | 95% | [Tumor HI Program](index=1&type=section&id=tumor-hi-program) The Company achieved FDA alignment on a significantly streamlined clinical development path for its upLIFT Phase 3 study of ersodetug for tumor HI, reducing the study to as few as 16 participants in a single-arm open-label portion, with topline results expected in the second half of 2026 - Achieved FDA alignment on a significantly streamlined clinical development path for the ongoing Phase 3 study (upLIFT) of ersodetug for tumor HI[5](index=5&type=chunk) - The truncated upLIFT study will include as few as **16 participants** and be limited to the single-arm open-label portion, removing the need for a double-blind randomized placebo-controlled trial[5](index=5&type=chunk) - Enrollment for upLIFT is underway, and topline results are expected in the **second half of 2026**[5](index=5&type=chunk) [Corporate Updates](index=2&type=section&id=corporate-updates) This section highlights recent organizational changes, including key personnel appointments, to strengthen the company's commercialization efforts [Key Personnel Appointment](index=2&type=section&id=key-personnel-appointment) In August 2025, Dr. Sunil Karnawat was appointed as Chief Commercial Officer, bringing over 25 years of experience in global biopharmaceutical commercialization to spearhead the launch strategy for ersodetug - Dr. Sunil Karnawat was appointed as Chief Commercial Officer in **August 2025**[13](index=13&type=chunk) - Dr. Karnawat has over **25 years of experience** in global commercialization of biopharmaceuticals and medical devices[13](index=13&type=chunk) - He will spearhead launch strategy and global market readiness for ersodetug, having previously led commercial functions for launching four ultra-rare disease products at Ultragenyx[13](index=13&type=chunk) [Financial Results](index=2&type=section&id=financial-results) This section presents Rezolute's financial performance for Q4 and the full fiscal year 2025, detailing cash position, operating expenses, net loss, and key balance sheet data [Cash, Cash Equivalents and Investments](index=2&type=section&id=cash-cash-equivalents-investments) Rezolute's cash, cash equivalents, and investments in marketable securities increased to $167.9 million as of June 30, 2025, up from $127.1 million a year prior - **Cash, Cash Equivalents and Investments in Marketable Securities:** | Metric | June 30, 2025 (in millions) | June 30, 2024 (in millions) | YoY Change (in millions) | | :------------------------------------ | :-------------------------- | :-------------------------- | :----------- | | Cash, cash equivalents and investments | $167.9 | $127.1 | +$40.8 | [Operating Expenses](index=2&type=section&id=operating-expenses) Total operating expenses increased year-over-year for both Q4 and the full fiscal year 2025, driven primarily by higher R&D expenditures related to clinical trials, manufacturing, and employee costs, as well as increased G&A expenses from professional fees and headcount - **Total Operating Expenses:** | Period | FY2025 (in thousands) | FY2024 (in thousands) | YoY Change (in thousands) | | :----- | :-------------------- | :-------------------- | :--------- | | Q4 | $25,850 | $23,102 | +$2,748 | | Full Year | $79,894 | $70,423 | +$9,471 | [Research and Development (R&D) Expenses](index=2&type=section&id=research-development-expenses) R&D expenses increased in both Q4 and the full fiscal year 2025, primarily due to higher clinical trial, manufacturing, and employee-related costs - **Research and Development (R&D) Expenses:** | Period | FY2025 (in thousands) | FY2024 (in thousands) | YoY Change (in thousands) | | :----- | :-------------------- | :-------------------- | :--------- | | Q4 | $20,863 | $19,089 | +$1,774 | | Full Year | $61,527 | $55,743 | +$5,784 | - The increase in R&D expenses was primarily due to increased expenditures in clinical trial activities, manufacturing costs for ersodetug, and higher employee-related expenses (including compensation and stock-based compensation)[8](index=8&type=chunk) [General and Administrative (G&A) Expenses](index=2&type=section&id=general-administrative-expenses) G&A expenses rose in both Q4 and the full fiscal year 2025, mainly driven by increased professional fees and employee-related costs due to higher headcount - **General and Administrative (G&A) Expenses:** | Period | FY2025 (in thousands) | FY2024 (in thousands) | YoY Change (in thousands) | | :----- | :-------------------- | :-------------------- | :--------- | | Q4 | $4,987 | $4,013 | +$974 | | Full Year | $18,367 | $14,680 | +$3,687 | - The increase in G&A expenses was primarily attributable to professional fees and employee-related expenses due to increased headcount[9](index=9&type=chunk) [Net Loss and EPS](index=2&type=section&id=net-loss-eps) Rezolute reported an increased net loss for both Q4 and the full fiscal year 2025, with basic and diluted net loss per common share also increasing year-over-year - **Net Loss and Basic/Diluted Net Loss Per Common Share:** | Metric | Period | FY2025 (in thousands) | FY2024 (in thousands) | YoY Change (in thousands) | | :----- | :----- | :-------------------- | :-------------------- | :--------- | | Net Loss | Q4 | $(24,390) | $(22,976) | $(1,414) | | Net Loss | Full Year | $(74,412) | $(68,459) | $(5,953) | | Basic and diluted net loss per common share | Q4 | $(0.26) | $(0.44) | +$0.18 | | Basic and diluted net loss per common share | Full Year | $(0.98) | $(1.33) | +$0.35 | [Condensed Consolidated Financial Statements Data](index=4&type=section&id=condensed-consolidated-financial-statements-data) The condensed consolidated financial statements provide detailed data for the statements of operations and balance sheets, showing increases in total assets, working capital, and total stockholders' equity, alongside the reported losses and increased accumulated deficit - **Condensed Consolidated Statements of Operations Data (Selected):** | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Year Ended June 30, 2025 (in thousands) | Year Ended June 30, 2024 (in thousands) | | :-------------------------- | :-------------------------------------- | :-------------------------------------- | :------------------------------------ | :------------------------------------ | | Research and development | $20,863 | $19,089 | $61,527 | $55,743 | | General and administrative | $4,987 | $4,013 | $18,367 | $14,680 | | Total operating expenses | $25,850 | $23,102 | $79,894 | $70,423 | | Loss from operations | $(25,850) | $(23,102) | $(79,894) | $(70,423) | | Non-operating income (expenses), net | $1,460 | $126 | $5,482 | $1,964 | | Net loss | $(24,390) | $(22,976) | $(74,412) | $(68,459) | | Basic and diluted net loss per common share | $(0.26) | $(0.44) | $(0.98) | $(1.33) | | Shares used to compute basic and diluted net loss per common share | 94,340 | 52,235 | 75,999 | 51,466 | - **Condensed Consolidated Balance Sheets Data:** | Metric | June 30, 2025 (in thousands) | June 30, 2024 (in thousands) | | :-------------------------------- | :--------------------------- | :--------------------------- | | Cash and cash equivalents | $94,107 | $70,396 | | Investments in marketable debt securities | $73,751 | $56,741 | | Working capital | $159,233 | $119,047 | | Total assets | $175,490 | $132,737 | | Accumulated deficit | $(403,856) | $(329,444) | | Total stockholders' equity | $162,127 | $121,003 | [Product Information](index=2&type=section&id=product-information) This section provides an overview of ersodetug, a fully human monoclonal antibody designed to treat hypoglycemia caused by hyperinsulinism [About Ersodetug](index=2&type=section&id=about-ersodetug) Ersodetug is a fully human monoclonal antibody designed to treat hypoglycemia caused by hyperinsulinism by allosterically binding to the insulin receptor, thereby decreasing over-activation and offering potential universal effectiveness across all forms of HI - Ersodetug is a fully human monoclonal antibody[11](index=11&type=chunk) - It binds allosterically to the insulin receptor to decrease receptor over-activation by insulin and related substances (such as IGF-2) in hyperinsulinism (HI), thereby improving hypoglycemia[11](index=11&type=chunk) - Ersodetug has the potential to be universally effective at treating hypoglycemia due to any congenital or acquired form of HI, as it acts downstream from the pancreas[11](index=11&type=chunk) [Company Information](index=2&type=section&id=company-information) This section offers a brief profile of Rezolute, Inc., emphasizing its mission as a late-stage rare disease company developing ersodetug for hyperinsulinism [About Rezolute, Inc.](index=2&type=section&id=about-rezolute-inc) Rezolute, Inc. is a late-stage rare disease company focused on hyperinsulinism-induced hypoglycemia, developing ersodetug, an antibody therapy that has demonstrated meaningful clinical benefit for both congenital and tumor HI - Rezolute is a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism (HI)[12](index=12&type=chunk) - The Company's antibody therapy, ersodetug, is designed to treat all forms of HI[12](index=12&type=chunk) - Ersodetug has shown meaningful benefit in clinical trials and real-world use for the treatment of both congenital and tumor HI[12](index=12&type=chunk) [Legal and Investor Information](index=3&type=section&id=legal-investor-information) This section includes important disclosures regarding forward-looking statements and provides contact details for investor relations inquiries [Forward-Looking Statements](index=3&type=section&id=forward-looking-statements) This section contains standard forward-looking statements regarding prospective performance, strategies, and anticipated milestones, including trial results and ersodetug's effectiveness, and advises caution due to inherent uncertainties and risks detailed in SEC filings - The release contains forward-looking statements regarding prospective performance and strategies, covered by safe harbor provisions[14](index=14&type=chunk) - These statements include the timing of topline results from sunRIZE and upLIFT trials, ersodetug's applicability and effectiveness for hyperinsulinism, and clinical trial timelines[14](index=14&type=chunk) - Readers are cautioned that actual results may differ materially from anticipated results due to inherent uncertainties and factors discussed in SEC filings (e.g., Risk Factors in 10-K and 10-Q)[14](index=14&type=chunk) [Contacts](index=3&type=section&id=contacts) This section provides contact information for investor relations inquiries - Contact information for Rezolute, Inc. is provided for inquiries[15](index=15&type=chunk)

Core Insights - Rezolute, Inc. has made significant advancements in its treatment for hypoglycemia caused by hyperinsulinism, particularly with its drug ersodetug, and is preparing for potential commercialization [2][12] Financial Results - As of June 30, 2025, the company reported cash, cash equivalents, and investments in marketable securities totaling $167.9 million, an increase from $127.1 million as of June 30, 2024 [6] - Research and development (R&D) expenses for Q4 2025 were $20.9 million, up from $19.1 million in Q4 2024, with total R&D expenses for the fiscal year 2025 reaching $61.5 million compared to $55.7 million in 2024 [7] - General and administrative (G&A) expenses for Q4 2025 were $5.0 million, compared to $4.0 million in Q4 2024, with total G&A expenses for the fiscal year 2025 at $18.4 million, up from $14.7 million in 2024 [9] - The net loss for Q4 2025 was $24.4 million, compared to a net loss of $23.0 million in Q4 2024, with a full-year net loss of $74.4 million versus $68.5 million in 2024 [10][15] Pipeline Progress and Milestones - The company completed enrollment in the sunRIZE trial, a Phase 3 study for congenital hyperinsulinism, with 62 participants, and topline results are expected in December 2025 [8] - FDA alignment was achieved for a streamlined Phase 3 study (upLIFT) for tumor hyperinsulinism, which will include as few as 16 participants, with topline results anticipated in the second half of 2026 [8] - Dr. Sunil Karnawat was appointed as Chief Commercial Officer to lead the launch strategy for ersodetug, bringing over 25 years of experience in biopharmaceutical commercialization [8] About Ersodetug - Ersodetug is a fully human monoclonal antibody designed to treat hypoglycemia due to hyperinsulinism by decreasing receptor over-activation, showing potential effectiveness across various forms of hyperinsulinism [11][12]

Core Insights - Rezolute, Inc. is a biopharmaceutical company focused on developing treatments for rare metabolic diseases, with its lead candidate, Ersodetug, having received the FDA's Breakthrough Therapy designation, indicating significant potential for accelerated approval [2][6] - Despite a negative price-to-earnings (P/E) ratio of -7.41, Rezolute's stock is rising in 2025, driven by investor interest in Ersodetug's potential, reflected in a high price-to-sales ratio of 11,579.47 [3][6] - The company's financial metrics show challenges, including a high enterprise value to sales ratio of 11,358.42 and a negative enterprise value to operating cash flow ratio of -10.04, but a low debt-to-equity ratio of 0.0215 indicates minimal reliance on debt [4][5] Financial Stability - Rezolute's current ratio of 8.43 indicates a strong ability to cover short-term liabilities with short-term assets, suggesting financial stability despite the lack of profitability [5]

Core Viewpoint - Rezolute, Inc. is experiencing a positive trajectory in its stock price, driven by the FDA's Breakthrough Therapy designation for its lead candidate, Ersodetug, which is expected to expedite its approval process [1][2] Group 1: Drug Development and Market Potential - Ersodetug is designed to treat hard-to-treat hyperinsulinism, with initial focus on tumor-induced cases, but has potential for broader applications [2] - The company anticipates that sales could peak at over $1 billion for its initial target markets, positioning Ersodetug as a potential blockbuster product [3] - Revenue generation is expected to begin in 2026, with a projected growth trajectory over the next eight to ten years as treatment indications expand [3][4] Group 2: Financial Outlook and Analyst Sentiment - Analysts unanimously rate RZLT stock as a Buy, with a price target suggesting a 55% upside from the current price [5] - H.C. Wainwright's analyst has set a price target of $14, indicating a potential 20% upside compared to the consensus [6] - Institutional ownership exceeds 80%, with increasing bullish activity, suggesting continued upward momentum for the stock [7] Group 3: Stock Performance and Risks - The stock price has increased over 200% in 2025, with technical indicators showing bullish trends, although resistance near $8 may limit short-term gains [13] - The company is currently pre-revenue but has sufficient capital to sustain operations for approximately six more quarters, despite ongoing net losses [11][12] - There is a risk of dilution due to potential partnerships and cash injections, with share count increasing by 36% year-to-date [12]

Core Insights - The article discusses Rezolute, Inc. (NASDAQ: RZLT) and its potential as a candidate in the pharmaceutical sector, particularly focusing on the drug RZ402 [2]. Company Overview - Rezolute, Inc. is highlighted for its innovative approaches in the biotech industry, with a specific emphasis on RZ402, which may present unexpected opportunities for investors [2]. Investment Analysis - The author provides a comprehensive analysis of Rezolute, including a model portfolio of over 10 small and mid-cap stocks, aimed at assisting healthcare investors in making informed decisions [2].

Core Insights - The presentation features a discussion between Kelly McCarthy from Morgan Stanley and Nevan Elam, CEO of Rezolute, highlighting the company's narrative and strategic direction [1]. Company Overview - Rezolute is positioned within the healthcare sector, and the conference serves as a platform to elaborate on its business model and future prospects [1].

Summary of Resolute's Conference Call Company Overview - **Company**: Resolute - **Focus**: Treatment of hyperinsulinism, a rare disease affecting insulin regulation in patients, particularly in congenital and tumor settings [2][4] Core Points and Arguments - **Disease Background**: Hyperinsulinism leads to dangerous hypoglycemia due to excessive insulin production, either from congenital genetic defects or tumors [2][3] - **Therapeutic Approach**: Resolute is developing an antibody designed to modulate insulin binding rather than blocking it, allowing for a universal treatment across different forms of hyperinsulinism [3][4] - **Clinical Development**: Currently in Phase 3 trials for both congenital and tumor-related hyperinsulinism, with promising results in correcting glucose levels in patients [4][6] - **Market Size**: Approximately 3,500 individuals in the US have congenital hyperinsulinism, while the tumor market is estimated to be twice as large, indicating significant commercial potential [5][24] - **FDA Alignment**: Recent alignment with the FDA on the uplift study for tumor hyperinsulinism is seen as a significant development, allowing for a streamlined Phase 3 study [6][7] - **Data Timeline**: Expected data from the congenital hyperinsulinism program in December 2025, with a goal of demonstrating a 35% difference in treatment efficacy compared to placebo [11][13] Important Insights - **Patient Impact**: The therapy has shown substantial improvements in quality of life for patients previously facing end-of-life situations, highlighting the therapy's potential impact [7][16] - **Regulatory Path**: Breakthrough therapy designation received for both congenital and tumor hyperinsulinism, with plans to file a Biologics License Application (BLA) by mid-2026 [18][19] - **Commercial Strategy**: The company plans to independently commercialize the drug in the US, with potential partnerships evaluated for international markets [29][26] - **Pricing Strategy**: Anticipated robust pricing for the drug, especially in pediatric rare disease markets, with expectations of minimal pushback from payers [27][26] Additional Considerations - **Competitive Landscape**: Resolute does not view other therapies as direct competitors, emphasizing its unique position as the only therapy applicable across all forms of hyperinsulinism [41][42] - **Community Engagement**: There is growing excitement among key opinion leaders (KOLs) and patient advocacy groups due to the long absence of effective therapies for hyperinsulinism [22][23] - **Team Dynamics**: The company has a dedicated team with low attrition, fostering a mission-driven culture focused on making a difference in patients' lives [32][30] Conclusion - Resolute is positioned at a critical juncture with its innovative therapy for hyperinsulinism, showing promise in clinical trials and receiving regulatory support, which could lead to significant advancements in treatment options for affected patients [43][44]

Core Insights - Rezolute, Inc. has aligned with the FDA on a streamlined clinical development path for its Phase 3 study (upLIFT) of ersodetug, aimed at treating hypoglycemia caused by tumor hyperinsulinism (HI) [1][2] - The FDA has approved modifications to the study design, allowing for a single-arm open-label trial with as few as 16 participants, eliminating the need for a double-blind randomized placebo-controlled trial [2][3] - The pivotal sunRIZE trial in congenital HI is expected to report topline results in December 2025 and will serve as confirmatory evidence for the efficacy of ersodetug [2] Company Overview - Rezolute is a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism, with its antibody therapy, ersodetug, showing meaningful benefits in clinical trials and real-world applications [8] - The company has treated over 10 patients with tumor HI under its Expanded Access Program, contributing to the favorable outcomes that influenced the FDA's decision [3] Study Details - The upLIFT study is a single-arm, open-label pivotal trial involving approximately 16 participants with insulinoma or non-islet cell tumors experiencing uncontrolled hypoglycemia due to tumor HI [4] - Participants will receive ersodetug at a dosage of 9 mg/kg per week for 8 weeks, with the primary endpoint being a 50% reduction in intravenous glucose requirements [4] Disease Context - Tumor hyperinsulinism is a rare disease caused by islet cell tumors (ICTs) and non-islet cell tumors (NICTs), leading to hypoglycemia through over-activation of the insulin receptor [5] - Insulinomas are the most common ICTs, while various NICTs, particularly hepatocellular carcinoma, can also cause hypoglycemia by secreting insulin-like substances [5] Mechanism of Action - Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor, reducing receptor over-activation by insulin and related substances, thereby improving hypoglycemia [7]

Core Insights - Rezolute, Inc. has appointed Sunil Karnawat as Chief Commercial Officer to lead the launch strategy and global market readiness for its therapy, ersodetug, which is currently in Phase 3 studies for treating hyperinsulinism [1][2] Company Overview - Rezolute is a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism (HI) with its antibody therapy, ersodetug, which has shown meaningful benefits in clinical trials [4] Leadership Appointment - Sunil Karnawat brings over 25 years of experience in global commercialization of biopharmaceuticals and medical devices, having successfully launched products in various therapeutic areas, including ultra-rare diseases [2] - Karnawat's previous roles include Vice President at Cytokinetics and Ultragenyx, where he led the launch of four ultra-rare disease products [2] - His educational background includes an MBA from the Wharton School and a Ph.D. in Engineering [2] Inducement Grant - In connection with Karnawat's appointment, the Board of Directors approved an inducement stock option of 275,000 shares at an exercise price of $6.55 per share, with a vesting schedule tied to his continued employment [3]

Core Viewpoint - Rezolute, Inc. is actively participating in the BTIG Virtual Biotechnology Conference, indicating its commitment to engaging with investors and stakeholders in the biotechnology sector [1][2]. Company Overview - Rezolute, Inc. is a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism (HI) [3]. - The company's antibody therapy, ersodetug, aims to treat all forms of HI and has demonstrated significant benefits in both clinical trials and real-world applications for congenital HI and tumor HI [3].