Kempharm(KMPH) - 2025 Q1 - Quarterly Results

Q1 2025 Executive Summary Zevra reported Q1 2025 net revenue of $20.4 million, bolstered by a $150 million PRV sale, and progressed its rare disease pipeline Q1 2025 Key Financial Overview | Metric | Q1 2025 | | :----- | :------ | | Net Revenue | $20.4 million | | Product Net Revenue | $17.2 million | - Completed sale of Pediatric Rare Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, significantly bolstering the balance sheet for commercial launches and development programs[1][2][5] - Company is transforming into a patient-centric, commercial-stage rare disease therapeutics company, focusing on establishing MIPLYFFA® globally, maximizing OLPRUVA®'s commercial opportunity, and supporting growth through its development pipeline[2] Commercial and Pipeline Progress Zevra advanced commercial products MIPLYFFA® and OLPRUVA® with new enrollments and expanded market access, and progressed its celiprolol pipeline Commercial Highlights - All active patients in the U.S. Expanded Access Program (EAP) for arimoclomol have been enrolled to receive MIPLYFFA, and the EAP has since closed[6] MIPLYFFA® Commercial Metrics (Q1 2025) | Metric | Q1 2025 | Total (End of Q1 2025) | Market Access | | :----- | :------ | :--------------------- | :------------ | | New Prescription Enrollment Forms | 13 | 122 | 38% of covered lives | OLPRUVA® Commercial Metrics (Q1 2025) | Metric | Q1 2025 | Total (Since July 2023) | Market Access | | :----- | :------ | :---------------------- | :------------ | | New Patient Enrollment Forms | 5 | 28 | 78% of covered lives | Pipeline and Innovation Highlights - Filing of the MIPLYFFA MAA with the European Medicines Agency remains on track for the second half of 2025[6] - Enrolled 5 additional patients in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome, bringing the total to 32 patients[6] - Out-licensed IP related to a pre-clinical prodrug, entitling Zevra to a nominal upfront cash payment, potential future regulatory milestones, and mid-single-digit royalties on net sales[6] Corporate Milestones and Upcoming Events - On April 7, 2025, Zevra announced closing of the sale of its Pediatric Rare Disease Priority Review Voucher ('PRV') for gross proceeds of $150 million[5] - Zevra will participate in a fireside chat at the H.C. Wainwright 3rd Annual Bioconnect Investor Conference on Tuesday, May 20, 2025[8] Detailed Financial Results Zevra reported Q1 2025 net revenue of $20.4 million, a reduced net loss of ($3.1) million, and a cash position of $68.7 million, bolstered by a PRV sale Q1 2025 Financial Highlights (Summary) Q1 2025 Key Financial Highlights | Metric | Q1 2025 | Q1 2024 | Change (YoY) | | :-------------------------------- | :-------- | :-------- | :----------- | | Revenue, Net | $20.4 million | $3.4 million | +$17.0 million | | - MIPLYFFA net revenue | $17.1 million | N/A | N/A | | - OLPRUVA net revenue | $0.1 million | N/A | N/A | | - French EAP reimbursements | $2.3 million | N/A | N/A | | - Royalties (AZSTARYS®) | $0.9 million | N/A | N/A | | Cost of Goods Sold | $3.0 million | N/A | N/A | | Operating Expenses | $22.8 million | $22.2 million | +$0.6 million | | R&D expense | $3.3 million | $12.3 million | -$9.0 million | | SG&A expense | $19.5 million | $9.9 million | +$9.6 million | | Net Loss | ($3.1) million | ($16.6) million | -$13.5 million | | Net Loss per Share (Basic & Diluted) | ($0.06) | ($0.40) | -$0.34 | - R&D expense decreased by $9.0 million compared to Q1 2024, primarily due to decreased third-party costs upon completion of the KP1077 Phase 2 trial and reduced personnel-related costs[10] - SG&A expense increased by $9.6 million compared to Q1 2024, mainly due to increased personnel-related costs, professional fees, and other expenses associated with commercial, medical, and launch activities[10] Cash Position and Shares Outstanding | Metric | As of Mar. 31, 2025 | Pro Forma (incl. PRV sale) | As of Dec. 31, 2024 | | :-------------------------------- | :------------------ | :------------------------- | :------------------ | | Cash, cash equivalents and investments | $68.7 million | $217.0 million | $69.5 million (Cash & Securities) | | Common Stock Outstanding | 54,679,363 | N/A | 53,670,709 | | Fully Diluted Common Shares | 67,897,631 | N/A | N/A | Unaudited Condensed Consolidated Statements of Operations Unaudited Condensed Consolidated Statements of Operations (Q1 2025 vs Q1 2024) | Metric (in thousands) | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Revenue, net | $20,401 | $3,425 | | Cost of product revenue (excl. amortization) | $1,345 | $175 | | Intangible asset amortization | $1,615 | $1,528 | | Research and development | $3,258 | $12,277 | | Selling, general and administrative | $19,545 | $9,931 | | Total operating expenses | $22,803 | $22,208 | | Loss from operations | ($5,362) | ($20,486) | | Total other income | $3,445 | $3,794 | | Income (loss) before income taxes | ($1,917) | ($16,692) | | Income tax (expense) benefit | ($1,182) | $70 | | Net loss | ($3,099) | ($16,622) | | Basic and diluted net loss per share | ($0.06) | ($0.40) | | Weighted average shares outstanding | 54,095,543 | 41,778,774 | Unaudited Condensed Consolidated Balance Sheets Unaudited Condensed Consolidated Balance Sheets (Mar 31, 2025 vs Dec 31, 2024) | Metric (in thousands) | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :------------- | :---------------- | | Assets | | | | Cash and cash equivalents | $37,340 | $33,785 | | Securities at fair value, current | $25,291 | $35,711 | | Total current assets | $81,573 | $86,027 | | Total assets | $172,708 | $178,127 | | Liabilities | | | | Accounts payable and accrued expenses | $17,819 | $25,456 | | Total current liabilities | $26,976 | $34,065 | | Long-term debt | $60,090 | $59,504 | | Warrant liability | $13,030 | $17,804 | | Total liabilities | $131,685 | $138,461 | | Stockholders' Equity | | | | Total stockholders' equity | $41,023 | $39,666 | Product Information This section details Zevra's key products: MIPLYFFA® for NPC, OLPRUVA® for UCDs, and celiprolol for VEDS, including mechanisms, indications, and safety About MIPLYFFA® (arimoclomol) - MIPLYFFA® (arimoclomol) is Zevra's FDA-approved therapy for Niemann-Pick disease type C (NPC), approved on September 20, 2024[12](index=12&