[PART I: FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%3A%20FINANCIAL%20INFORMATION) [Condensed Consolidated Financial Statements](index=3&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements) The company reported a **$1.17 million** net loss in Q1 2025, a significant shift from **$12.84 million** net income in Q1 2024, primarily due to the absence of prior year's one-time licensing revenue [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet shows a slight increase in total assets to **$46.4 million** and liabilities to **$52.3 million**, resulting in a minor increase in stockholders' deficit Balance Sheet Summary (in thousands USD) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $22,675 | $26,634 | | Total current assets | $42,975 | $44,124 | | Total assets | $46,403 | $44,946 | | **Liabilities & Stockholders' Deficit** | | | | Total current liabilities | $8,371 | $6,919 | | Total liabilities | $52,283 | $50,818 | | Total stockholders' deficit | ($5,880) | ($5,872) | - Total assets increased slightly to **$46.4 million** from **$44.9 million**, while total liabilities increased to **$52.3 million** from **$50.8 million**, leading to a small increase in the total stockholders' deficit[10](index=10&type=chunk) [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) The statement of operations reflects a shift to a net loss of **$1.17 million** in Q1 2025, primarily due to the absence of prior year's **$18.0 million** licensing revenue Statement of Operations Summary (in thousands USD, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | PEDMARK product sales, net | $8,751 | $7,419 | | Licensing revenue | $0 | $17,958 | | **Total revenue** | **$8,751** | **$25,377** | | Total operating expenses | $9,559 | $11,634 | | (Loss) / income from operations | ($808) | $13,743 | | **Net (loss) / income** | **($1,165)** | **$12,837** | | **Diluted net (loss) / income per share** | **($0.04)** | **$0.41** | - Net product sales of **PEDMARK** grew **18%** YoY to **$8.75 million**, however, total revenue decreased significantly due to the absence of the **$18.0 million** in licensing revenue recognized in Q1 2024[11](index=11&type=chunk) - The company reported a net loss of **$1.17 million**, or **($0.04)** per share, in Q1 2025, compared to a net income of **$12.84 million**, or **$0.41** per diluted share, in Q1 2024[11](index=11&type=chunk) [Condensed Consolidated Statements of Stockholders' Equity/(Deficit)](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders%27%20Equity%2F%28Deficit%29) The stockholders' deficit slightly increased to **$5.880 million** due to the net loss, partially offset by stock-based compensation and option exercises - The total stockholders' deficit slightly increased from **$5.872 million** at the end of 2024 to **$5.880 million** at March 31, 2025, primarily driven by the net loss of **$1.165 million**, partially offset by stock-based compensation of **$0.798 million** and proceeds from stock option exercises[13](index=13&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash and cash equivalents decreased by **$4.0 million** in Q1 2025, primarily due to **$4.3 million** in cash used in operating activities, a reversal from the prior year's cash inflow Cash Flow Summary (in thousands USD) | Cash Flow Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash (used in) / provided by operating activities | ($4,318) | $39,047 | | Net cash provided by financing activities | $359 | ($1,132) | | **(Decrease) / increase in cash and cash equivalents** | **($3,959)** | **$37,915** | | Cash and cash equivalents - End of period | $22,675 | $51,184 | - Cash used in operating activities was **$4.3 million** in Q1 2025, a stark contrast to the **$39.0 million** provided by operations in Q1 2024, which had benefited from the upfront payment from the **Norgine** licensing deal[16](index=16&type=chunk) [Notes to the Condensed Consolidated Financial Statements](index=7&type=section&id=Notes%20to%20the%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the company's sole product **PEDMARK®**, its liquidity outlook, ongoing patent litigation, and the **Norgine** licensing agreement - The company's sole product is **PEDMARK®**, approved by the FDA to reduce the risk of ototoxicity associated with cisplatin in pediatric patients[17](index=17&type=chunk) - Management believes current funds, including the upfront payment from **Norgine**, are sufficient to fund planned activities for at least the next twelve months, despite a net loss and negative operating cash flow in Q1 2025[19](index=19&type=chunk)[30](index=30&type=chunk) - The company is engaged in ongoing patent infringement litigation against **CIPLA**, which submitted an Abbreviated New Drug Application (ANDA) for a generic version of **PEDMARK®**[95](index=95&type=chunk)[97](index=97&type=chunk)[101](index=101&type=chunk) - In March 2024, Fennec secured an exclusive licensing agreement with **Norgine** to commercialize **PEDMARQSI® (PEDMARK®)** in Europe, Australia, and New Zealand, receiving an upfront payment of approximately **$43.2 million**[29](index=29&type=chunk)[120](index=120&type=chunk)[121](index=121&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management attributes the Q1 2025 net loss to the absence of prior year's one-time licensing revenue, despite **PEDMARK®** sales growing **18%** to **$8.8 million** [Overview](index=23&type=section&id=Overview) Fennec is a commercial-stage biopharmaceutical company focused on **PEDMARK®**, its sole FDA-approved product for cisplatin-induced ototoxicity in pediatric patients - Fennec is a commercial-stage biopharmaceutical company focused on its only product, **PEDMARK®**, the first and only FDA-approved treatment to reduce the risk of cisplatin-induced ototoxicity in certain pediatric patients[133](index=133&type=chunk) - The company received European Commission Marketing Authorization for **PEDMARQSI®** (the European brand name) in June 2023 and subsequently entered an exclusive licensing agreement with **Norgine** for commercialization in Europe, Australia, and New Zealand[134](index=134&type=chunk) - **PEDMARK®** has Orphan Drug Exclusivity in the U.S. until **September 2029** and is protected by six patents listed in the FDA Orange Book, with expirations in **2039**[137](index=137&type=chunk) [Results of Operations](index=26&type=section&id=Results%20of%20Operations) Total revenue decreased significantly due to the absence of prior year's licensing revenue, despite **PEDMARK®** product sales increasing by **$1.3 million** Q1 2025 vs Q1 2024 Results (in thousands USD) | Line Item | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | PEDMARK product sales, net | $8,751 | $7,419 | $1,332 | | Licensing revenue | $0 | $17,958 | ($17,958) | | **Total revenue** | **$8,751** | **$25,377** | **($16,626)** | | Selling and marketing | $2,947 | $5,209 | ($2,262) | | General and administration | $6,145 | $5,872 | $273 | | **Total operating expense** | **$9,559** | **$11,634** | **($2,075)** | | **Net (loss) / income** | **($1,165)** | **$12,837** | **($14,002)** | - The decrease in total revenue was due to the **$18.0 million** in licensing revenue from the **Norgine** transaction recognized in Q1 2024, which did not recur in Q1 2025[153](index=153&type=chunk) - Selling and marketing expenses decreased by **$2.3 million**, largely due to the elimination of European pre-commercial activities following the **Norgine** transaction[153](index=153&type=chunk) [Liquidity and Capital Resources](index=27&type=section&id=Liquidity%20and%20Capital%20Resources) Cash and cash equivalents decreased by **$4.0 million** to **$22.7 million** in Q1 2025, primarily due to negative operating cash flow and working capital changes - Cash and cash equivalents decreased by **$4.0 million** during Q1 2025, from **$26.6 million** to **$22.7 million**, primarily due to seasonally higher operating expenses and timing of working capital collections[155](index=155&type=chunk)[158](index=158&type=chunk) - Working capital decreased from **$37.2 million** at year-end 2024 to **$34.6 million** as of March 31, 2025[155](index=155&type=chunk) - Net cash used in operating activities was **$4.3 million** in Q1 2025, compared to **$39.0 million** provided by operating activities in Q1 2024, with the difference primarily resulting from the proceeds of the **Norgine** transaction in the prior year[156](index=156&type=chunk) [Critical Accounting Policies and Estimates](index=29&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) No material changes occurred in critical accounting policies, which involve significant judgments, particularly for revenue recognition from license agreements - There have been no material changes to the company's critical accounting policies, which include revenue recognition for product sales and license agreements, credit losses, and stock-based compensation[167](index=167&type=chunk) - Revenue recognition requires significant judgments, including identifying performance obligations, estimating variable consideration, and allocating the transaction price, particularly for license agreements with milestones and royalties[170](index=170&type=chunk)[171](index=171&type=chunk) [Controls and Procedures](index=31&type=section&id=Item%203.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and internal control over financial reporting were effective as of March 31, 2025, with no material changes during the quarter - The **CEO** and **CFO** concluded that as of March 31, 2025, the company's disclosure controls and procedures were effective at a reasonable assurance level[181](index=181&type=chunk)[182](index=182&type=chunk) - Management concluded that as of March 31, 2025, the company's internal controls over financial reporting were effective[183](index=183&type=chunk) [PART II: OTHER INFORMATION](index=32&type=section&id=PART%20II%3A%20OTHER%20INFORMATION) [Legal Proceedings](index=32&type=section&id=Item%201.%20Legal%20Proceedings) The company is engaged in ongoing patent infringement litigation against **CIPLA** regarding a generic version of **PEDMARK®**, with market protection until **September 2029** due to Orphan Drug Exclusivity - Fennec is in a legal dispute with **CIPLA**, which is seeking to market a generic version of **PEDMARK®** and has challenged Fennec's patents[187](index=187&type=chunk) - The lawsuit triggers a **30-month stay** on the FDA's ability to grant final approval to **CIPLA's** generic product, additionally, **PEDMARK®** has Orphan Drug Exclusivity until **September 20, 2029**[188](index=188&type=chunk) - The litigation is ongoing, with Fennec having filed a Third Amended Complaint to focus the case on two key patents (**US '018** and **US '793**)[191](index=191&type=chunk)[194](index=194&type=chunk) [Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) No material changes have occurred to the risk factors previously disclosed in the company's **2024 Annual Report on Form 10-K** - The company states there are no material changes from the risk factors disclosed in its **2024 Annual Report on Form 10-K**[195](index=195&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=33&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities or use of proceeds during the period - The company reported no unregistered sales of equity securities during the period[196](index=196&type=chunk) [Defaults Upon Senior Securities](index=33&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities during the period - The company reported no defaults upon senior securities[197](index=197&type=chunk) [Mine Safety Disclosures](index=33&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section is not applicable to the company's operations - This item is not applicable to the company[198](index=198&type=chunk) [Other Information](index=33&type=section&id=Item%205.%20Other%20Information) No director or officer adopted or terminated a **Rule 10b5-1** trading arrangement, and the company issued a press release announcing Q1 2025 financial results - No director or officer adopted or terminated a **Rule 10b5-1** trading arrangement during the quarter ended March 31, 2025[199](index=199&type=chunk) - A press release announcing **Q1 2025** financial results was issued on **May 13, 2025**, and furnished as **Exhibit 99.1**[200](index=200&type=chunk) [Exhibits](index=33&type=section&id=Item%206.%20Exhibits) The report includes **CEO** and **CFO** certifications as required by **Sarbanes-Oxley Act** sections and the **Q1 2025** earnings press release - Exhibits filed with the report include **CEO** and **CFO** certifications (**Exhibits 31.1, 31.2, 32.1**) and the **Q1 2025** earnings press release (**Exhibit 99.1**)[201](index=201&type=chunk)

Financial Data and Key Metrics Changes - The company recorded net product sales of approximately $8.8 million in Q1 2025, reflecting an 18% growth compared to $7.4 million in Q1 2024 and an 11% increase over Q4 2024 [16][17] - Selling and marketing expenses decreased to $2.9 million in Q1 2025 from $5.2 million in Q1 2024, primarily due to the elimination of European pre-commercialization expenses [17] - General and administrative expenses were $6.1 million in Q1 2025, consistent year-over-year but increased from $4.1 million in Q4 2024 [18] - Cash and cash equivalents stood at $22.6 million as of March 31, 2025, with a cash burn of approximately $4 million in Q1 2025 [18] Business Line Data and Key Metrics Changes - The company focused on enhancing field execution and supporting sustainable growth across key PedMark segments, particularly expanding into the adolescent and young adult (AYA) patient population [5][6] - The targeting model implemented in Q1 2025 allowed for better engagement with high-volume cisplatin prescribers, leading to early traction in key accounts [7][8] - The AYA segment is estimated to have a market potential greater than the pediatric market, with approximately 20,000 cisplatin chemotherapy patients treated annually in the U.S. [9] Market Data and Key Metrics Changes - The product Pedmark has launched commercially in Germany and the UK, with recent acceptance for use in Scotland [12] - The investigator-initiated trial in Japan evaluating Pedmark was fully enrolled as of October 2024, with results expected in the second half of 2025 [13] Company Strategy and Development Direction - The company aims to deepen relationships with key accounts and expand adoption with existing customers while generating evidence-based data through investigator-sponsored trials [14] - A focus on enhancing the FENIC HEAR's patient support program is intended to improve access and streamline reimbursement processes [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving significant quarterly growth in 2025, particularly in Q2 and Q3, as foundational initiatives take effect [17][19] - The company does not anticipate proposed tariffs to materially impact gross margins or overall financial performance [19] Other Important Information - The company is focused on increasing patient adherence through the PhenicHERS program, which has been redesigned to support home administration of the product [24][27] - Management is exploring strategic opportunities in oncology supportive care, with potential synergies identified [69] Q&A Session Summary Question: Average number of vials per patient in the AYA segment - The average is about 30 vials per patient, with approximately 50% adherence to the full treatment course [22][24] Question: Capabilities driving adherence improvement in the PhenicHERS program - The program has been redesigned to focus on home administration and patient education to improve adherence [25][27] Question: Operating expenses and future revenue growth expectations - Operating expenses are front-end loaded, with expectations for a decrease in the second half of the year, aiming for revenue growth to reach breakeven [29][49] Question: Initial inroads into the AYA settings - The company is making progress in both NCI centers and community settings, with a focus on large academic centers and community practices [57] Question: Future opportunities for Pedmark in ex-U.S. territories - The company is targeting Spain, Italy, and France for future introductions of Pedmark, following recent launches in Germany and the UK [66] Question: Strategic opportunities in oncology supportive care - The company is open to exploring strategic opportunities as sales grow and the organization stabilizes [69]

Financial Data and Key Metrics Changes - The company recorded net product sales of approximately $8.8 million in Q1 2025, reflecting an 18% growth compared to $7.4 million in Q1 2024 and an 11% increase over Q4 2024 [15][16] - Selling and marketing expenses decreased to $2.9 million in Q1 2025 from $5.2 million in Q1 2024, primarily due to the elimination of European pre-commercialization expenses [17] - General and administrative expenses were $6.1 million in Q1 2025, consistent year-over-year but increased from $4.1 million in Q4 2024 [18] - Cash and cash equivalents stood at $22.6 million as of March 31, 2025, with a cash burn of approximately $4 million in Q1 2025 [18] Business Line Data and Key Metrics Changes - The company has made significant progress in targeting prescribers for Pedmark, particularly in the adolescent and young adult (AYA) patient population, which is expected to have a larger market potential than the pediatric market [9][10] - The targeting model implemented has shown early traction, with tier one targets prescribing Pedmark and integrating it into treatment plans [7][10] Market Data and Key Metrics Changes - The AYA segment is estimated to have approximately 20,000 cisplatin chemotherapy patients treated annually in the U.S., with a favorable reimbursement profile [9] - The product has launched commercially in Germany and the UK, with recent acceptance for use in Scotland [12] Company Strategy and Development Direction - The company is focused on enhancing field execution and supporting sustainable growth across key segments, particularly through a data-driven targeting model [5][6] - There is a strategic emphasis on deepening relationships with key accounts and expanding adoption among existing customers [14] - The company is exploring additional strategic opportunities in oncology supportive care, with ongoing discussions about potential synergies [66] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory, anticipating significant quarterly growth in net product sales as foundational initiatives take effect [17] - The company remains optimistic about the impact of its strategic focus and disciplined execution, expecting continued progress throughout the year [14][71] Other Important Information - The company does not anticipate that proposed tariffs will materially impact gross margins or overall financial performance, as Pedmark is manufactured in the U.S. [19] Q&A Session Summary Question: Average number of vials per patient and adherence rates - The average is about 30 vials per patient, with approximately 50% adherence to the full treatment course [21][23] Question: Improvements in the FENICHERS program - The program has been redesigned to enhance home administration capabilities, which is critical for improving adherence [26][27] Question: Operating expenses and future revenue expectations - Operating expenses are expected to be front-end loaded, with a potential decrease in the second half of the year [30][31] - The company aims for revenue growth to reach breakeven, targeting approximately $8.5 to $9 million in sales [47][48] Question: Expansion into ex-U.S. markets - The company is focusing on major European markets, with Spain, Italy, and France as potential future opportunities for Pedmark [62][63] Question: Strategic opportunities in oncology supportive care - The company is open to exploring strategic opportunities and partnerships that could enhance its offerings in supportive care [66]

Financial Performance - Adherex Technologies Inc. reported a quarterly loss of $0.04 per share, better than the Zacks Consensus Estimate of a loss of $0.12, representing an earnings surprise of 66.67% [1] - The company posted revenues of $8.75 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 9.32%, but down from $25.38 million year-over-year [2] - Over the last four quarters, the company has only surpassed consensus EPS estimates once [2] Market Performance - Adherex Technologies shares have declined approximately 0.8% since the beginning of the year, compared to a decline of 0.6% for the S&P 500 [3] - The company's current consensus EPS estimate for the upcoming quarter is -$0.10 on revenues of $9.44 million, and for the current fiscal year, it is $0.03 on revenues of $54.54 million [7] Industry Outlook - The Medical - Biomedical and Genetics industry, to which Adherex Technologies belongs, is currently ranked in the top 35% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, suggesting that the company's stock may outperform the market based on current estimates [5][6]

Core Viewpoint - Fennec Pharmaceuticals Inc. is set to release its first quarter 2025 financial results on May 13, 2025, and will host a conference call to discuss these results [1] Group 1: Financial Results Announcement - The company will announce its first quarter 2025 financial results before the U.S. financial markets open on May 13, 2025 [1] - A conference call and webcast will be held on the same day to discuss the financial and business results [1] Group 2: Conference Call & Webcast Details - The conference call is scheduled for 8:30 a.m. ET on May 13, 2025 [2] - Links for the webcast and participant registration are provided, with a recommendation to connect at least 15 minutes prior to the call [2] Group 3: Company Overview - Fennec Pharmaceuticals focuses on developing and commercializing PEDMARK, aimed at reducing platinum-induced ototoxicity in pediatric patients [3] - PEDMARK received FDA approval in September 2022, European Commission approval in June 2023, and U.K. approval in October 2023 under the brand name PEDMARQSI [3] - The product has Orphan Drug Exclusivity in the U.S. and Pediatric Use Marketing Authorization in Europe, providing significant market protection [3]

Group 1 - Adherex Technologies Inc. (FENC) closed at $6.19, with an 11.9% gain over the past four weeks, and a mean price target of $13.20 suggests an upside potential of 113.3% [1] - The mean estimate includes five short-term price targets with a standard deviation of $2.05, indicating variability among analysts; the lowest estimate of $10 suggests a 61.6% increase, while the highest estimate of $15 indicates a 142.3% increase [2] - Analysts show strong agreement in revising earnings estimates higher, which correlates with potential stock price movements, as the Zacks Consensus Estimate for the current year has increased by 134.6% over the past month [11][12] Group 2 - FENC holds a Zacks Rank 1 (Strong Buy), placing it in the top 5% of over 4,000 ranked stocks based on earnings estimates, indicating strong potential upside in the near term [13] - While price targets are often viewed as a key metric, they can mislead investors; analysts may set overly optimistic targets due to business incentives, which can inflate expectations [7][8] - A low standard deviation in price targets suggests a high degree of agreement among analysts regarding the stock's price movement direction, serving as a starting point for further research [9]

Group 1 - Adherex Technologies Inc. (FENC) has experienced a significant downtrend, with a stock decline of 22.2% over the past four weeks, indicating strong selling pressure [1] - The stock is currently in oversold territory, as indicated by an RSI reading of 29.08, suggesting that the heavy selling may be exhausting itself and a rebound could be imminent [5] - There is a strong consensus among Wall Street analysts regarding FENC's ability to report better earnings than previously predicted, with a 124.3% increase in the consensus EPS estimate over the last 30 days [7] Group 2 - FENC holds a Zacks Rank 1 (Strong Buy), placing it in the top 5% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, indicating a strong potential for a turnaround [8] - The upward trend in earnings estimate revisions typically correlates with price appreciation in the near term, further supporting the case for a potential rebound in FENC's stock price [7]

Financial Performance - As of December 31, 2024, the company had $24.6 million in money market investments, an increase from $11.9 million at December 31, 2023, indicating a significant growth in liquid assets [555]. - The company has not experienced any loss or write-down of its money market investments for the years ended December 31, 2024, and 2023, reflecting stable investment performance [556]. - The company has a history of significant losses and limited revenue generation from product sales since inception, highlighting financial challenges [15]. Investment Strategy - The investment policy prioritizes preservation of principal, liquidity, and return on investment, with minimal risk associated with fluctuating interest rates [557]. Regulatory and Market Challenges - The commercialization of PEDMARK® is critical, as the company is a single product entity with limited commercial experience, complicating future financial forecasts [19]. - Regulatory approvals for PEDMARK® have been obtained, but ongoing regulatory review and potential restrictions remain a concern for market acceptance [19]. - The company may require additional financing for further product development and regulatory approvals, which could impact operational capabilities [15]. - The company faces substantial competition in the biopharmaceutical market, which could adversely affect its market position and financial performance [19]. Operational Risks - The company is subject to foreign currency risks, holding approximately CAD$0.16 million and €0.24 million as of December 31, 2024, to manage obligations in Canada and the EU [558]. - The company relies on third parties for raw materials and manufacturing, and any failure in their performance could materially impact business operations [17].

Financial Performance - Full-Year PEDMARK® net product sales reached $29.6 million, a 40% increase year-over-year, with Q4 2024 net product sales of $7.9 million[1] - The company reported a Q4 2024 EBITDA loss of $0.6 million and had $26.6 million in cash, cash equivalents, and short-term investments[1] - Cash flow from operations decreased by $0.6 million in Q4 2024, but there was a $13.4 million increase in cash and cash equivalents from December 31, 2023, to December 31, 2024[7] - Selling and marketing expenses for FY 2024 were $18.4 million, up from $12.1 million in FY 2023, primarily due to increased payroll and marketing efforts[7] - General and administrative expenses for FY 2024 were $23.1 million, compared to $20.6 million in FY 2023, influenced by European pre-commercialization expenses and costs associated with the Norgine transaction[8] Debt Management - Early repayment of $13 million of the company's convertible debt facility was completed, optimizing the balance sheet and saving approximately $1.5 million in future annual interest payments[7] Product Development and Approval - PEDMARK® is the first FDA-approved therapy to reduce the risk of ototoxicity associated with cisplatin in pediatric patients with localized, non-metastatic solid tumors[18] - PEDMARK received FDA approval in September 2022, European Commission approval in June 2023, and U.K. approval in October 2023[31] - The Japan clinical trial (STS-J01) for PEDMARK® is fully enrolled, with results expected in the second half of 2025, potentially leading to registration in Japan[7] - PEDMARQSI® is now commercially available in the United Kingdom and Germany, expanding global access to the product[2] Clinical Efficacy and Safety - PEDMARK has proven efficacy and safety data established through two Phase 3 clinical studies, COG ACCL0431 and SIOPEL 6[21] - The COG ACCL0431 protocol enrolled patients with various childhood cancers treated with intensive cisplatin therapy, while SIOPEL 6 focused on hepatoblastoma patients[21] - Hypersensitivity reactions occurred in 8% to 13% of patients during clinical trials, necessitating monitoring for such reactions[25] - Hypernatremia occurred in 12% to 26% of patients, while hypokalemia occurred in 15% to 27% of patients during clinical trials[27] - The most common adverse reactions in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia, while hypokalemia was most common in COG ACCL0431[29] Market Potential and Strategy - Over 10,000 children in the U.S. and Europe may receive platinum-based chemotherapy annually, with many requiring lifelong hearing aids due to ototoxicity[20] - Fennec Pharmaceuticals emphasizes the potential market impact and commercialization plans for PEDMARK, alongside anticipated regulatory milestones and funding opportunities[32] - The company is focused on expanding access to PEDMARQSI® for cancer patients at risk of hearing loss, with significant milestones achieved in Europe[5]

Financial Data and Key Metrics Changes - In 2024, the company reported a revenue growth of approximately 40% year-over-year, with net product sales of $29.6 million compared to $21.3 million in 2023 [28][30] - For Q4 2024, net product sales were $7.9 million, reflecting a quarterly growth rate of approximately 13% [28] - The company managed to burn only approximately $0.6 million in cash for Q4 2024, demonstrating operational efficiency [34] Business Line Data and Key Metrics Changes - The pediatric segment continued to show growth, while the adolescent and young adult (AYA) segment emerged as a significant opportunity [38] - Selling and marketing expenses for Q4 2024 were $3.9 million, down from $4.6 million in Q3 2024, while full-year expenses increased to $18.4 million from $12.1 million in 2023 [29][30] Market Data and Key Metrics Changes - The U.S. AYA oncology landscape is estimated to have approximately 20,000 cisplatin chemotherapy patients treated annually, with significant market potential [11][12] - The product PEDMARK is gaining traction in major academic centers, which is critical for broader market acceptance [14] Company Strategy and Development Direction - The company is focused on expanding awareness of the unmet need for preventing cisplatin-induced ototoxicity and establishing PEDMARK as the standard of care [9][10] - The PEDMARK strategy will be utilized throughout 2025 to drive growth, with a focus on both pediatric and AYA populations [7][8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth potential for 2025, particularly in the second half of the year as foundational initiatives take effect [29][41] - The company anticipates additional compendia listings and is actively engaging with stakeholders to support positive outcomes for PEDMARK's inclusion in guidelines [17][18] Other Important Information - The company has launched PEDMARQSI in Germany and the U.K., with pricing set at £8277 in the U.K. and over €10,500 per vial in Germany [20][21] - The company reported a cash position of $26.6 million as of December 31, 2024, following a significant debt repayment [33] Q&A Session Summary Question: Did the pediatric business return to sequential growth in Q4, and what was the AYA contribution? - Management confirmed continued growth in the pediatric segment and highlighted the AYA segment as an incredible opportunity, though specific breakdowns were not provided [38] Question: What is the expected inflection in the AYA segment for 2025? - Management indicated significant opportunities in the AYA segment and expected accelerated growth in the second half of 2025 [41] Question: What progress has Norgine made in Europe? - Management noted that the product is commercially available in the U.K. and Germany, with ongoing updates expected as more countries get approved [48][49] Question: What is the competitive landscape for PEDMARK in the U.S.? - Management acknowledged the presence of compounded products but emphasized the adoption of PEDMARK in major institutions, indicating progress despite competition [72] Question: Will clinical trials be run for potential label expansions? - Management indicated openness to discussions regarding label expansions and will provide updates as developments occur [75] Question: How does the company prioritize commercial efforts between community centers and academic institutions? - Management emphasized the importance of both settings and noted that awareness levels in the AYA space present a significant opportunity for growth [90]