Dinutuximab

SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--( BUSINESS WIRE )--United Therapeutics Corporation (Nasdaq: UTHR ), a public benefit corporation, today announced its financial results for the quarter ended June 30, 2024. Total revenues in the second quarter of 2024 grew 20 percent year-over-year to $714.9 million, compared to $596.5 million in the second quarter of 2023. “This quarter we drove record revenue from our foundational commercial business. Next year we expect data from our innovative clinical pipeline. All this while we march forward with our revolutionary organ manufacturing programs,” said Martine Rothblatt, Ph.D. , Chairperson and Chief Executive Officer of United Therapeutics. “We believe there is no other biotech with our combination of relentless focus, near-term commercial growth, and clinical potential.” Michael Benkowitz , President and Chief Operating Officer of United Therapeutics, added, “Our fifth straight quarter of record revenue was driven by continued traction for Tyvaso in pulmonary hypertension associated with interstitial lung disease, along with strong fundamentals for our other products in pulmonary arterial hypertension and neuroblastoma.” Second Quarter 2024 Financial Results Key financial highlights include (dollars in millions, except per share data): Three Months Ended June 30, Dollar Change Percentage Change 2024 2023 Total revenues $ 714.9 $ 596.5 $ 118.4 20 % Net income $ 278.1 $ 259.2 $ 18.9 7 % Net income, per basic share $ 6.26 $ 5.53 $ 0.73 13 % Net income, per diluted share $ 5.85 $ 5.24 $ 0.61 12 % Revenues The table below presents the components of total revenues (dollars in millions): Three Months Ended June 30, Dollar Change Percentage Change 2024 2023 Net product sales: Tyvaso DPI ®(1) $ 258.3 $ 193.6 $ 64.7 33 % Nebulized Tyvaso ®(1) 139.9 125.3 14.6 12 % Total Tyvaso 398.2 318.9 79.3 25 % Remodulin ®(2) 147.3 127.2 20.1 16 % Orenitram ® 107.1 95.1 12.0 13 % Unituxin ® 51.7 44.3 7.4 17 % Adcirca ® 5.7 7.5 (1.8 ) (24 )% Other 4.9 3.5 1.4 40 % Total revenues $ 714.9 $ 596.5 $ 118.4 20 % (1) Net product sales include both the drug product and the respective inhalation device. (2) Net product sales include sales of infusion devices, including the Remunity ® Pump. Total Tyvaso revenues grew by 25 percent to $398.2 million in the second quarter of 2024, compared to $318.9 million in the second quarter of 2023. This growth was primarily due to an increase in quantities sold, driven by the commercial launch of Tyvaso DPI in June 2022 and continued growth in commercial utilization by patients with pulmonary hypertension associated with interstitial lung disease and, to a lesser extent, price increases. The growth in Tyvaso DPI revenues resulted primarily from an increase in quantities sold and, to a lesser extent, price increases. The increase in Tyvaso DPI quantities sold was due to continued growth in the number of patients following the product’s launch and, to a lesser extent, increased commercial utilization following the implementation of the Part D redesign under the Inflation Reduction Act ( IRA ). The growth in Remodulin revenues resulted primarily from an increase in U.S. Remodulin revenues, driven by an increase in quantities sold and, to a lesser extent, lower Medicaid rebates. The growth in Orenitram revenues resulted primarily from an increase in quantities sold and, to a lesser extent, a price increase. The increase in quantities sold was driven, at least in part, by increased commercial utilization following the implementation of the Part D redesign under the IRA. The growth in Unituxin revenues resulted from a price increase and an increase in quantities sold. The table below presents the breakdown of total revenues between the United States and rest-of-world ( ROW ) (in millions): Three Months Ended June 30, 2024 2023 U.S. ROW Total U.S. ROW Total Net product sales: Tyvaso DPI (1) $ 258.3 $ — $ 258.3 $ 193.6 $ — $ 193.6 Nebulized Tyvaso (1) 130.2 9.7 139.9 119.6 5.7 125.3 Total Tyvaso 388.5 9.7 398.2 313.2 5.7 318.9 Remodulin (2) 122.5 24.8 147.3 103.5 23.7 127.2 Orenitram 107.1 — 107.1 95.1 — 95.1 Unituxin 46.8 4.9 51.7 39.5 4.8 44.3 Adcirca 5.7 — 5.7 7.5 — 7.5 Other 4.6 0.3 4.9 3.2 0.3 3.5 Total revenues $ 675.2 $ 39.7 $ 714.9 $ 562.0 $ 34.5 $ 596.5 (1) Net product sales include both the drug product and the respective inhalation device. (2) Net product sales include sales of infusion devices, including the Remunity Pump. Expenses Cost of sales. The table below summarizes cost of sales by major category (dollars in millions): Three Months Ended June 30, Dollar Change Percentage Change 2024 2023 Category: Cost of sales $ 75.9 $ 63.2 $ 12.7 20 % Share-based compensation expense (1) 1.9 0.9 1.0 111 % Total cost of sales $ 77.8 $ 64.1 $ 13.7 21 % (1) See Share-based compensation below. Cost of sales, excluding share-based compensation. Cost of sales for the three months ended June 30, 2024 increased as compared to the same period in 2023, primarily due to an increase in Tyvaso DPI royalty expense. Research and development. The table below summarizes the nature of research and development expense by major expense category (dollars in millions): Three Months Ended June 30, Dollar Change Percentage Change 2024 2023 Category: External research and development (1) $ 49.4 $ 49.3 $ 0.1 — % Internal research and development (2) 44.5 34.7 9.8 28 % Share-based compensation expense (3) 8.6 5.0 3.6 72 % Impairments (4) — — — — % Other (5) 37.1 — 37.1 NM (6) Total research and development expense $ 139.6 $ 89.0 $ 50.6 57 % (1) External research and development primarily includes fees paid to third parties (such as clinical trial sites, contract research organizations, and contract laboratories) for preclinical and clinical studies and payments to third-party contract manufacturers before FDA approval of the relevant product. (2) Internal research and development primarily includes salary-related expenses for research and development functions, internal costs to manufacture product candidates before FDA approval, and internal facilities-related expenses, including depreciation, related to research and development activities. (3) See Share-based compensation below. (4) Impairments primarily includes impairment charges to write down the carrying value of in-process research and development and of certain property, plant, and equipment as a result of research and development activities. There were no impairment charges during the three months ended June 30, 2024 and June 30, 2023. (5) Other primarily includes upfront fees and milestone payments to third parties under license agreements related to development-stage products and adjustments to the fair value of our contingent consideration obligations. (6) Calculation is not meaningful. Research and development, excluding share-based compensation. Research and development expense for the three months ended June 30, 2024 increased as compared to the same period in 2023, primarily due to increased expenditures related to upfront non-refundable licensing payments for drug delivery devices and increased expenditures related to organ manufacturing projects. Selling, general, and administrative. The table below summarizes selling, general, and administrative expense by major category (dollars in millions): Three Months Ended June 30, Dollar Change Percentage Change 2024 2023 Category: General and administrative $ 113.0 $ 102.0 $ 11.0 11 % Sales and marketing 25.4 20.1 5.3 26 % Share-based compensation expense (1) 39.2 7.9 31.3 396 % Total selling, general, and administrative expense $ 177.6 $ 130.0 $ 47.6 37 % (1) See Share-based compensation below. General and administrative, excluding share-based compensation . General and administrative expense for the three months ended June 30, 2024 increased as compared to the same period in 2023, primarily due to an increase in legal expenses related to litigation matters and an increase in personnel expense due to growth in headcount. Share-based compensation. The table below summarizes share-based compensation expense by major category (dollars in millions): Three Months Ended June 30, Dollar Change Percentage Change 2024 2023 Category: Stock options $ 8.1 $ 1.6 $ 6.5 406 % Restricted stock units 19.2 13.6 5.6 41 % Share tracking awards plan ( STAP ) 21.9 (1.9 ) 23.8 NM (1) Employee stock purchase plan 0.5 0.5 — — % Total share-based compensation expense $ 49.7 $ 13.8 $ 35.9 260 % (1) Calculation is not meaningful. The increase in share-based compensation expense for the three months ended June 30, 2024, as compared to the same period in 2023, was primarily due to an increase in STAP expense driven by a 39 percent increase in our stock price for the three months ended June 30, 2024, as compared to a one percent decrease in our stock price for the same period in 2023. Income tax expense. Income tax expense for the three months ended June 30, 2024 and 2023 was $77.2 million and $76.0 million, respectively. Our effective income tax rate ( ETR ) for the three months ended June 30, 2024 and 2023 was 22 percent and 23 percent, respectively. Our ETR for the three months ended June 30, 2024 decreased compared to our ETR for the three months ended June 30, 2023 primarily due to a lower amount of uncertain tax positions recorded. Share repurchase. In March 2024, we entered into an accelerated share repurchase agreement (the ASR agreement ) with Citibank, N.A. ( Citi ). Under the ASR agreement, we made an aggregate upfront payment of $1.0 billion to Citi and received an aggregate initial delivery of 3,275,199 shares of our common stock on March 27, 2024, representing approximately 80 percent of the total shares that would be repurchased under the ASR agreement measured based on the closing price of our common stock on March 25, 2024. The share purchase under the ASR agreement was divided into two tranches, resulting in upfront payments of $300 million and $700 million, respectively. The final settlement of the $300 million tranche occurred in June 2024, and we received an additional 181,772 shares of our common stock upon settlement. At the final settlement of the $700 million second tranche, which we expect to occur in the third quarter of 2024, we may be entitled to receive additional shares of common stock, or, under certain limited circumstances, be required to make a cash payment to Citi or, if we so elect, deliver shares of common stock to Citi. The final number of shares that we will ultimately repurchase pursuant to the ASR agreement will be based on the average of the daily volume-weighted average price per share of our common stock during the repurchase period, less a discount and subject to adjustments pursuant to the terms and conditions of the ASR agreement. Webcast We will host a webcast to discuss our second quarter 2024 financial results on Wednesday, July 31, 2024, at 9:00 a.m. Eastern Time. The webcast can be accessed live via our website at https://ir.unither.com/events-and-presentations . A replay of the webcast will also be available at the same location on our website. United Therapeutics: Enabling Inspiration At United Therapeutics, our vision and mission are one. We use our enthusiasm, creativity, and persistence to innovate for the unmet medical needs of our patients and to benefit our other stakeholders. We are bold and unconventional. We have fun; we do good. We are the first publicly-traded biotech or pharmaceutical company to take the form of a public benefit corporation ( PBC ). Our public benefit purpose is to provide a brighter future for patients through (a) the development of novel pharmaceutical therapies; and (b) technologies that expand the availability of transplantable organs . You can learn more about what it means to be a PBC here: unither.com/pbc . Forward-Looking Statements Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, statements related to our anticipated near-term commercial growth; the anticipated progress of our organ manufacturing programs; our clinical potential, including anticipated clinical trial data next year; our unique position in the biotech industry; and our goals of innovating for the unmet medical needs of our patients and to benefit our other stakeholders, furthering our public benefit purpose of developing novel pharmaceutical therapies and technologies that expand the availability of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of July 31, 2024, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events, or any other reason. ORENITRAM, REMODULIN, REMUNITY, TYVASO, TYVASO DPI, and UNITUXIN are registered trademarks of United Therapeutics Corporation and/or its subsidiaries. ADCIRCA is a registered trademark of Eli Lilly and Company. UNITED THERAPEUTICS CORPORATION CONSOLIDATED STATEMENTS OF OPERATIONS (In millions, except per share data) Three Months Ended June 30, 2024 2023 (Unaudited) Total revenues $ 714.9 $ 596.5 Operating expenses: Cost of sales 77.8 64.1 Research and development 139.6 89.0 Selling, general, and administrative 177.6 130.0 Total operating expenses 395.0 283.1 Operating income 319.9 313.4 Interest income 46.2 37.2 Interest expense (11.6 ) (14.8 ) Other income (expense), net 0.8 (0.6 ) Total other income, net 35.4 21.8 Income before income taxes 355.3 335.2 Income tax expense (77.2 ) (76.0 ) Net income $ 278.1 $ 259.2 Net income per common share: Basic $ 6.26 $ 5.53 Diluted $ 5.85 $ 5.24 Weighted average number of common shares outstanding: Basic 44.4 46.9 Diluted 47.5 49.5 SELECTED CONSOLIDATED BALANCE SHEET DATA (Unaudited, in millions) June 30, 2024 Cash, cash equivalents, and marketable investments $ 4,301.9 Total assets 6,723.2 Total liabilities 1,026.0 Total stockholders’ equity 5,697.2 Category: Earnings

AMSTERDAM, April 15, 2024 /PRNewswire/ -- Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology. Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand. On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for high-risk neuroblastoma (HRNB), indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.1 The approval decision was based on findings from a trial comparing outcomes from Study 3b (NCT02395666; investigational arm)2,4 and Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)3,4, where use of eflornithine resulted in improved event-free survival and overall survival when compared to outcomes for patients with high-risk neuroblastoma treated with the standard of care (SoC) without the drug.1 Dr David Gillen, Chief Medical Officer at Norgine, added, "These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine's passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need." Follow @norgine Notes to Editors: Project Orbis Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health). HRNB background Children diagnosed with HRNB undergo an intense SoC regimen that still leaves them vulnerable to relapse and death, a risk that is particularly acute during the first two years.5 Approximately 30% of patients who attain remission following upfront therapy will relapse, resulting in a poor prognosis and low likelihood of long-term survival (e.g. estimates as low as 15% of patients will live for five years after relapsing).6 Avoiding relapse is key to long-term survival, yet outside of the United States there are no approved therapies for sustaining remission following SoC treatment. The data with eflornithine demonstrate using it as maintenance therapy extends remission and reduces risk of relapse in patients with HRNB. About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma. FDA. News release. 13 December 2023. Available from: FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA Clinical Trial NCT02395666; Preventative trial of difluoromethylornithine (DFMO) in high risk patients with neuroblastoma that is in remission. Available from: Study Details | Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission | ClinicalTrials.gov. Clinical Trial NCT00026312; Isotretinoin with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating patients with neuroblastoma. Available from: Study Details | Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma | ClinicalTrials.gov Oesterheld J, Ferguson W, Kraveka JM, Bergendahl G, Clinch T, Lorenzi E et al. Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. 2024; 42 (1): 90-102. Basta NO, Halliday GC, Makin G, Birch J, Feltbower R, Bown N, et al. Factors associated with recurrence and survival length following relapse in patients with neuroblastoma. Br J Cancer. 2016;115:1048-57. London WB, Bagatell R, Weigel BJ, Fox E, Guo D, Van Ryn C, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early-phase trials. Cancer. 2017;123:4914-23. Logo -