Pioneering Sustainable Growth and Innovation in Guatemala
A Legacy of Dedication and Growth
Magdalena's roots lie in a story of resilience and determination. Jorge Leal recalls the values instilled by his grandmother, Doña Elvira, whose work ethic and commitment to education shaped the family's vision. This legacy of dedication led to the founding of Magdalena, with the business initially driven by Leal's father and uncles.
Despite challenges, the company thrived under family leadership, achieving an impressive compound annual growth rate of 12% over four decades. Today, Magdalena's operations extend beyond sugar production to include alcohol and renewable energy, generating 10-12% of Guatemala's energy needs.
'Magdalena's growth is deeply rooted in our commitment to community, innovation, and resilience,' Leal reflects.
Embracing Circular Economy PracticesIn recent years, Magdalena has embraced a circular economy model, aiming to minimize waste and maximize resource efficiency. By repurposing by-products from sugarcane processing, the company has significantly reduced its environmental footprint.
'Our focus is to use every resource to its fullest potential,' Leal explains. For example, water used in operations is recirculated, and almost all by-products are utilized, with minimal exceptions like certain ashes. These efforts have cut costs, benefitted local communities, and garnered international recognition, including the AMCHAM Guatemala Environmental Award.
The company's approach to sustainability is holistic, encompassing water management, carbon footprint reduction, and biodiversity conservation. Magdalena's recent sustainability reports detail advancements in these areas, providing a model for other businesses in the region.
Innovating Through Biochemistry and TechnologyMagdalena's forward-thinking approach is perhaps most evident in its growing biochemistry division. Collaborating with the Catholic University of Portugal, the company is leveraging cutting-edge research to develop innovative solutions for health and nutrition.
Among its notable achievements is the development of an FDA-approved enzyme that doubles the efficiency of protein absorption in the human body. The company has also integrated artificial intelligence into its research processes, accelerating discoveries and enabling impactful innovations.
'This work transforms our vision, allowing us to address global challenges like nutrition and sustainability,' Leal shares.
Renewable Energy ProjectsMagdalena is also a major player in Guatemala's energy sector, with ongoing investments in renewable energy. The company is constructing a 98-megawatt solar park, set to begin operations in March 2025. With plans to expand capacity to 300 MW, this project underscores Magdalena's commitment to clean, sustainable energy.
Guatemala's energy system, inspired by the Chilean model, is considered one of the most advanced globally. However, recent underinvestment has created challenges. 'Recognizing the need, we decided to invest in renewable energy independently, demonstrating our trust in Guatemala's potential,' Leal explains.
Future plans include exploring geothermal energy and evaluating wind energy technologies, aligning with Magdalena's vision of sustainability and innovation.
Building a Global VisionMagdalena is undergoing a strategic transformation, creating two core divisions: one focused on sugar and another on biochemistry. The company aims to increase sugar production by 50% in the next three to four years while expanding operations internationally.
'In biochemistry, our focus is global. We're tackling critical issues like nutrition and exploring solutions that benefit not just Guatemala but also regions like Africa, where we are establishing a presence,' Leal states.
This global perspective has been fueled by partnerships with local and international universities, bringing cutting-edge knowledge and technology to Guatemala. The company has also successfully attracted and repatriated Guatemalan talent working abroad, enriching its operations with diverse expertise.
A Commitment to Community and OpportunityMagdalena's success is inseparable from its commitment to the Guatemalan people. The company has invested heavily in education, biodiversity, and community resilience, creating a positive ripple effect across the country.
'Guatemala is full of opportunities, driven by its people's warmth, dedication, and creativity,' Leal concludes. 'Our role is to harness these strengths and contribute to the nation's sustainable development.'
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Travere Therapeutics Reports Second Quarter 2025 Financial Results
SAN DIEGO--(BUSINESS WIRE)--Travere Therapeutics, Inc., (NASDAQ: TVTX) today reported its second quarter 2025 financial results and provided a corporate update. Travere Therapeutics reports its second quarter 2025 financial results and provides a corporate update. Share 'We continue to make strong progress against our strategic priorities, putting Travere on a trajectory for both near- and long-term growth. This quarter marked our strongest commercial performance to date, with increased momentum for FILSPARI resulting in significant growth in a dynamic IgAN market,' said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. 'Looking ahead, we are well positioned to continue advancing FILSPARI toward becoming a foundational treatment. In parallel, we are preparing for a potential approval in FSGS, where FILSPARI would become the first FDA-approved therapy —bringing a long-awaited option to patients and further extending our impact in rare kidney diseases. We also look forward to our upcoming August PDUFA date for REMS modification, which could result in important label updates that support broader access to FILSPARI. Additionally, we are making steady progress toward restarting enrollment in our pivotal study of pegtibatinase, moving us closer to potentially delivering the first disease-modifying therapy for people living with classical HCU.' Financial Results for the Quarter Ended June 30, 2025 Net product sales for the second quarter of 2025 were $94.8 million, compared to $52.2 million for the same period in 2024. The increase is attributable to growth in sales of FILSPARI. Total revenue for the second quarter of 2025 was $114.4 million, inclusive of a $17.5 million milestone payment from CSL Vifor received during the quarter. Research and development (R&D) expenses for the second quarter of 2025 were $49.4 million, compared to $54.3 million for the same period in 2024. For the six months ended June 30, 2025, R&D expenses were $96.3 million, compared to $103.8 million for the same period in 2024. The decrease is largely attributable to lower costs associated with the development of pegtibatinase and decreased expense related to the development of sparsentan as the PROTECT and DUPLEX trials advance to completion. On a non-GAAP adjusted basis, R&D expenses were $45.4 million for the second quarter of 2025, compared to $50.6 million for the same period in 2024. Selling, general, and administrative (SG&A) expenses for the second quarter of 2025 were $76.2 million, compared to $64.8 million for the same period in 2024. For the six months ended June 30, 2025, SG&A expenses were $149.1 million, compared to $129.0 million for the same period in 2024. The difference is largely attributable to increased investment in commercialization of FILSPARI in IgAN, increased amortization expense related to FILSPARI royalties, and investment in preparing for a potential FSGS launch in January 2026. On a non-GAAP adjusted basis, SG&A expenses were $55.5 million for the second quarter of 2025, compared to $48.3 million for the same period in 2024. Total other expense, net for the second quarter of 2025 was $0.1 million, compared to total other expense, net of $1.9 million for the same period in 2024. The difference is primarily attributable to a non-cash charge to other expense during the second quarter of 2024 related to the Renalys Pharma collaboration. Net loss for the second quarter of 2025 was $12.8 million, or $0.14 per basic share, compared to a net loss of $70.4 million, or $0.91 per basic share for the same period in 2024. For the six months ended June 30, 2025, net loss was $54.0 million, compared to $206.5 million for the same period in 2024. On a non-GAAP adjusted basis, net income for the second quarter of 2025 was $11.9 million, or $0.13 per basic share, compared to a net loss of $50.1 million, or $0.65 per basic share for the same period in 2024. As of June 30, 2025, the Company had cash, cash equivalents, and marketable securities of $319.5 million. Program Updates FILSPARI ® (sparsentan) – IgAN U.S. net product sales totaled $71.9 million in 2Q 2025, representing 165% growth year-over-year. 745 new patient start forms (PSFs) were received during the quarter, reflecting continued uptake among new and repeat prescribers. The Company expects a PDUFA action date of August 28, 2025 for its supplemental New Drug Application (sNDA) to remove the embryo-fetal toxicity REMS monitoring requirement and to modify the frequency of liver monitoring REMS to every three months. In April, the European Commission converted conditional marketing authorization of FILSPARI to standard marketing authorization for the treatment of adults with primary IgAN in the European Union. In the UK, the Medicines and Healthcare products Regulatory Agency converted conditional approval of FILSPARI to standard approval for the treatment of adults with primary IgAN. As a result, Travere received a $17.5 million milestone payment from CSL Vifor and remains eligible to receive additional milestone payments related to market access and sales-based achievements. At the 62 nd European Renal Association (ERA) Congress (June 4-7) and International Podocyte Conference (June 10-13), the Company presented new data on the use of FILSPARI in IgAN. Highlights included: In the Phase 2 SPARTACUS Study, patients with IgAN receiving sodium-glucose cotransporter-2 inhibitor (SGLT2i) therapy who replaced their maximally tolerated RASi with FILSPARI achieved rapid and sustained albuminuria (~56% from baseline) and proteinuria (~45% from baseline) reductions and stable eGFR. Over 50% of patients reached ≥50% reduction in UACR from baseline, and nearly one-third achieved UACR levels below 0.2 g/g. In a preclinical model of IgAN, FILSPARI protected from mesangial deposition of IgA, suggesting a potential role of endothelin-1 and angiotensin II as modulators of disease activity, offering new insights into IgAN as a tissue-specific autoimmune disease. Data evaluating biomarkers of disease progression in IgAN from the Phase 2 SPARTAN Study showed rapid and sustained reductions in urinary BAFF and sC5b9 as well as reductions in proinflammatory and profibrotic biomarkers after starting FILSPARI, suggesting disease-modifying activity. The ongoing SPARTAN Study is expanding to include post-kidney transplant patients with recurring IgAN and the Company is initiating a new open label study of FILSPARI in post-kidney transplant patients with recurrent IgAN or FSGS. In the second half of 2025, the Company anticipates the publication of the final version of the updated Kidney Disease Improving Global Outcomes (KDIGO) clinical guidelines for IgAN. The draft guidelines published in August 2024 recommended FILSPARI as a foundational kidney-targeted therapy and lowered the targeted proteinuria level for all IgAN patients to under 0.5 g/day or preferably complete remission (under 0.3 g/day). The Company's partner, Renalys Pharma, Inc., expects topline results from its registrational Phase 3 clinical trial of sparsentan for the treatment of IgAN in Japan in the second half of 2025. FILSPARI ® (sparsentan) – FSGS In May, the Company announced the FDA accepted its supplemental new drug application (sNDA) for FILSPARI in FSGS. The FDA has assigned a PDUFA target action date of January 13, 2026, and indicated it is planning to hold an advisory committee meeting. If approved, FILSPARI would be the first and only FDA-approved treatment for FSGS. At ERA, the Company presented analyses from the Phase 3 DUPLEX Study showing that partial and complete proteinuria remission were achieved earlier and more frequently with FILSPARI than irbesartan, and patients who achieved partial or complete remission in the study, irrespective of the treatment arm, had lower risk of kidney failure. These results validate the observational results from PARASOL and reinforce FILSPARI's potential as a nephroprotective therapy that may help delay progression to kidney failure. The Company also presented preclinical data in an animal model of immune-mediated FSGS showing optimized dual endothelin and angiotensin blockade with FILSPARI protecting the integrity of the glomerular filtration barrier, reducing glomerular permeability and lowering albuminuria. Pegtibatinase (TVT-058) – Classical HCU The Company remains on track to restart enrollment in the Phase 3 HARMONY Study in 2026. Conference Call Information Travere Therapeutics will host a conference call and webcast today, August 6, 2025, at 4:30 p.m. ET to discuss company updates as well as second quarter 2025 financial results. To participate in the conference call, dial +1 (800) 549-8228 (U.S.) or +1 (646) 564-2877 (International), conference ID 41856 shortly before 4:30 p.m. ET. The webcast can be accessed on the Investor page of Travere's website at Following the live webcast, an archived version of the call will be available for 30 days on the Company's website. Use of Non-GAAP Financial Measures To supplement Travere's financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the Company uses certain non-GAAP adjusted financial measures in this press release and the accompanying tables. The Company believes that these non-GAAP financial measures are helpful in understanding its past financial performance and potential future results. They are not meant to be considered in isolation or as a substitute for comparable GAAP measures and should be read in conjunction with the consolidated financial statements prepared in accordance with GAAP. Travere's management regularly uses these supplemental non-GAAP financial measures internally to understand, manage and evaluate its business and make operating decisions. In addition, Travere believes that the use of these non-GAAP measures enhances the ability of investors to compare its results from period to period and allows for greater transparency with respect to key financial metrics the Company uses in making operating decisions. Investors should note that these non-GAAP financial measures are not prepared under any comprehensive set of accounting rules or principles and do not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future the Company may exclude other items, or cease to exclude items that it has historically excluded, for purposes of its non-GAAP financial measures; because of the non-standardized definitions, the non-GAAP financial measures as used by the Company in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by the Company's competitors and other companies. As used in this press release, (i) the historical non-GAAP net loss measures exclude from GAAP net loss, as applicable, stock-based compensation expense, amortization and depreciation expense, and income tax; (ii) the historical non-GAAP SG&A expense measures exclude from GAAP SG&A expenses, as applicable, stock-based compensation expense, and amortization and depreciation expense; (iii) the historical non-GAAP R&D expense measures exclude from GAAP R&D expenses, as applicable, stock-based compensation expense, and amortization and depreciation expense. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families, and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop, and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit FILSPARI ® (sparsentan) U.S. Indication FILSPARI (sparsentan) is indicated to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. IMPORTANT SAFETY INFORMATION Because of the risks of hepatotoxicity and birth defects, FILSPARI is available only through a restricted program called the FILSPARI REMS. Under the FILSPARI REMS, prescribers, patients and pharmacies must enroll in the program. Hepatotoxicity Some Endothelin Receptor Antagonists (ERAs) have caused elevations of aminotransferases, hepatotoxicity, and liver failure. In clinical studies, elevations in aminotransferases (ALT or AST) of at least 3-times the Upper Limit of Normal (ULN) have been observed in up to 3.5% of FILSPARI-treated patients, including cases confirmed with rechallenge. Measure transaminases and bilirubin before initiating treatment and monthly for the first 12 months, and then every 3 months during treatment. Interrupt treatment and closely monitor patients who develop aminotransferase elevations more than 3x ULN. FILSPARI should generally be avoided in patients with elevated aminotransferases (>3x ULN) at baseline because monitoring for hepatotoxicity may be more difficult and these patients may be at increased risk for serious hepatotoxicity. Embryo-Fetal Toxicity FILSPARI can cause major birth defects if used by pregnant patients based on animal data. Therefore, pregnancy testing is required before the initiation of treatment, during treatment and one month after discontinuation of treatment with FILSPARI. Patients who can become pregnant must use effective contraception before the initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI. Contraindications FILSPARI is contraindicated in patients who are pregnant. Do not coadminister FILSPARI with angiotensin receptor blockers (ARBs), ERAs, or aliskiren. Warnings and Precautions Hepatotoxicity: Elevations in ALT or AST of at least 3-fold ULN have been observed in up to 3.5% of FILSPARI-treated patients, including cases confirmed with rechallenge. While no concurrent elevations in bilirubin >2-times ULN or cases of liver failure were observed in FILSPARI-treated patients, some ERAs have caused elevations of aminotransferases, hepatotoxicity, and liver failure. To reduce the risk of potential serious hepatotoxicity, measure serum aminotransferase levels and total bilirubin prior to initiation of treatment and monthly for the first 12 months, then every 3 months during treatment. Advise patients with symptoms suggesting hepatotoxicity (nausea, vomiting, right upper quadrant pain, fatigue, anorexia, jaundice, dark urine, fever, or itching) to immediately stop treatment with FILSPARI and seek medical attention. If aminotransferase levels are abnormal at any time during treatment, interrupt FILSPARI and monitor as recommended. Consider re-initiation of FILSPARI only when hepatic enzyme levels and bilirubin return to pretreatment values and only in patients who have not experienced clinical symptoms of hepatotoxicity. Avoid initiation of FILSPARI in patients with elevated aminotransferases (>3x ULN) prior to drug initiation because monitoring hepatotoxicity in these patients may be more difficult and these patients may be at increased risk for serious hepatotoxicity. Embryo-Fetal Toxicity: FILSPARI can cause fetal harm when administered to a pregnant patient and is contraindicated during pregnancy. Advise patients who can become pregnant of the potential risk to a fetus. Obtain a pregnancy test prior to initiation of treatment with FILSPARI, monthly during treatment, and one month after discontinuation of treatment. Advise patients who can become pregnant to use effective contraception prior to initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI. FILSPARI REMS: Due to the risk of hepatotoxicity and embryo-fetal toxicity, FILSPARI is available only through a restricted program called the FILSPARI REMS. Prescribers, patients, and pharmacies must be enrolled in the REMS program and comply with all requirements ( Hypotension: Hypotension has been observed in patients treated with ARBs and ERAs. There was a greater incidence of hypotension-associated adverse events, some serious, including dizziness, in patients treated with FILSPARI compared to irbesartan. In patients at risk for hypotension, consider eliminating or adjusting other antihypertensive medications and maintaining appropriate volume status. If hypotension develops, despite elimination or reduction of other antihypertensive medications, consider a dose reduction or dose interruption of FILSPARI. A transient hypotensive response is not a contraindication to further dosing of FILSPARI, which can be given once blood pressure has stabilized. Acute Kidney Injury: Monitor kidney function periodically. Drugs that inhibit the renin-angiotensin system (RAS) can cause kidney injury. Patients whose kidney function may depend in part on the activity of the RAS (e.g., patients with renal artery stenosis, chronic kidney disease, severe congestive heart failure, or volume depletion) may be at particular risk of developing acute kidney injury on FILSPARI. Consider withholding or discontinuing therapy in patients who develop a clinically significant decrease in kidney function while on FILSPARI. Hyperkalemia: Monitor serum potassium periodically and treat appropriately. Patients with advanced kidney disease, taking concomitant potassium-increasing drugs (e.g., potassium supplements, potassium-sparing diuretics), or using potassium-containing salt substitutes are at increased risk for developing hyperkalemia. Dosage reduction or discontinuation of FILSPARI may be required. Fluid Retention: Fluid retention may occur with ERAs, and has been observed in clinical studies with FILSPARI. FILSPARI has not been evaluated in patients with heart failure. If clinically significant fluid retention develops, evaluate the patient to determine the cause and the potential need to initiate or modify the dose of diuretic treatment then consider modifying the dose of FILSPARI. Most common adverse reactions The most common adverse reactions (≥5%) are hyperkalemia, hypotension (including orthostatic hypotension), peripheral edema, dizziness, anemia, and acute kidney injury. Drug interactions Renin-Angiotensin System (RAS) Inhibitors and ERAs: Do not coadminister FILSPARI with ARBs, ERAs, or aliskiren due to increased risks of hypotension, syncope, hyperkalemia, and changes in renal function (including acute renal failure). Strong and Moderate CYP3A Inhibitors: Avoid concomitant use of FILSPARI with strong CYP3A inhibitors. If a strong CYP3A inhibitor cannot be avoided, interrupt FILSPARI treatment. When resuming treatment with FILSPARI, consider dose titration. Monitor blood pressure, serum potassium, edema, and kidney function regularly when used concomitantly with moderate CYP3A inhibitors. Concomitant use with a strong CYP3A inhibitor increases sparsentan exposure which may increase the risk of FILSPARI adverse reactions. Strong CYP3A Inducers: Avoid concomitant use with a strong CYP3A inducer. Concomitant use with a strong CYP3A inducer decreases sparsentan exposure which may reduce FILSPARI efficacy. Antacids and Acid Reducing Agents: Administer FILSPARI 2 hours before or after administration of antacids. Avoid concomitant use of acid reducing agents (histamine H2 receptor antagonist and PPI proton pump inhibitor) with FILSPARI. Sparsentan exhibits pH-dependent solubility. Antacids or acid reducing agents may decrease sparsentan exposure which may reduce FILSPARI efficacy. Non-Steroidal Anti-Inflammatory Agents (NSAIDs), Including Selective Cyclooxygenase-2 (COX-2) Inhibitors: Monitor for signs of worsening renal function with concomitant use with NSAIDs (including selective COX-2 inhibitors). In patients with volume depletion (including those on diuretic therapy) or with impaired kidney function, concomitant use of NSAIDs (including selective COX-2 inhibitors) with drugs that antagonize the angiotensin II receptor may result in deterioration of kidney function, including possible kidney failure. CYP2B6, 2C9, and 2C19 Substrates: Monitor for efficacy of concurrently administered CYP2B6, 2C9, and 2C19 substrates and consider dosage adjustment in accordance with the Prescribing Information. Sparsentan decreases exposure of these substrates, which may reduce efficacy related to these substrates. P-gp and BCRP Substrates: Avoid concomitant use of sensitive substrates of P-gp and BCRP with FILSPARI. Sparsentan may increase exposure of these transporter substrates, which may increase the risk of adverse reactions related to these substrates. Agents Increasing Serum Potassium: Monitor serum potassium frequently in patients treated with FILSPARI and other agents that increase serum potassium. Concomitant use of FILSPARI with potassium-sparing diuretics, potassium supplements, potassium-containing salt substitutes, or other drugs that raise serum potassium levels may result in hyperkalemia. Please see the full Prescribing Information, including BOXED WARNING, for additional Important Safety Information. Forward Looking Statements This press release contains 'forward-looking statements' as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words 'on-track,' 'positioned,' 'look forward to,' 'will,' 'would,' 'may,' 'might,' 'believes,' 'anticipates,' 'plans,' 'expects,' 'intends,' 'potential,' or similar expressions. In addition, expressions of strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: continued progress with the FILSPARI launch in IgAN; statements and expectations regarding near- and long-term growth trajectories; statements regarding advancing FILSPARI towards becoming a foundational treatment; statements regarding the potential for FILSPARI to be approved for the treatment of FSGS, and the expected timing thereof; statements regarding potential REMS modifications, the expected timing thereof, and potential label updates that support broader access; statements and expectations regarding the restarting of enrollment in the Company's pivotal study of pegtibatinase, and the potential for pegtibatinase to be the first disease-modifying therapy for people living with classical HCU; statements relating to the clinical trials and other studies and models described herein, including but not limited to Renalys's registrational Phase 3 clinical trial, and expectations regarding the timing and outcome thereof; expectations regarding the KDIGO guidelines; statements and expectations regarding future milestone payments; and statements regarding financial metrics and expectations related thereto. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties related to the Company's sNDA for FILSPARI in FSGS, including the timing and outcome thereof. There is no guarantee that the FDA will grant approval of FILSPARI for FSGS on the anticipated timeline, or at all. The Company also faces risks and uncertainties related to its business and finances in general, the success of its commercial products, risks and uncertainties associated with its preclinical and clinical stage pipeline, risks and uncertainties associated with the regulatory review and approval process, risks and uncertainties associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. Specifically, the Company faces risks associated with the ongoing commercial launch of FILSPARI in IgAN, the timing and potential outcome of its and its partners' clinical studies, market acceptance of its commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, risks related to the challenges of manufacturing scale-up, risks associated with the successful development and execution of commercial strategies for such products, including FILSPARI, and risks and uncertainties related to the new administration, including but not limited to risks and uncertainties related to tariffs and the funding, staffing and prioritization of resources at government agencies including the FDA. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading 'Risk Factors', as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission. TRAVERE THERAPEUTICS, INC. (in thousands, except share and per share data) (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 GAAP operating loss $ (12,650 ) $ (67,704 ) $ (55,327 ) $ (206,941 ) R&D operating expense (49,362 ) (54,330 ) (96,251 ) (103,750 ) Stock compensation 3,990 3,774 8,716 7,431 Non-GAAP R&D expense (45,372 ) (50,556 ) (87,535 ) (96,319 ) SG&A operating expense (76,216 ) (64,776 ) (149,055 ) (128,999 ) Stock compensation 6,659 6,146 13,425 12,246 Amortization & depreciation 14,016 10,345 26,818 20,225 Subtotal non-GAAP items 20,675 16,491 40,243 32,471 Non-GAAP SG&A expense (55,541 ) (48,285 ) (108,812 ) (96,528 ) Subtotal non-GAAP items 24,665 20,265 48,959 39,902 Non-GAAP operating income (loss) $ 12,015 $ (47,439 ) $ (6,368 ) $ (167,039 ) GAAP net loss $ (12,755 ) $ (70,409 ) $ (53,981 ) $ (206,470 ) Non-GAAP operating loss adjustments 24,665 20,265 48,959 39,902 Income tax provision 20 85 59 276 Non-GAAP net income (loss) $ 11,930 $ (50,059 ) $ (4,963 ) $ (166,292 ) Per share data: Net income (loss) per common share $ 0.13 $ (0.65 ) $ (0.06 ) $ (2.15 ) Weighted average common shares outstanding 88,945,624 77,500,245 88,652,428 77,318,369 Expand
Business Wire
19 minutes ago
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Sarepta Therapeutics Announces Second Quarter 2025 Financial Results and Recent Corporate Developments
BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the second quarter of 2025. 'We are very pleased that following a rapid review of the safety data, the FDA swiftly recommended that we take the ambulatory patient population off shipment pause and, following that, we have already resumed deliveries. Infusions are taking place for the ambulatory community. We will continue to work with the FDA to define the path and develop the risk mitigation necessary to bring ELEVIDYS back to the non-ambulatory community as well,' said Doug Ingram, chief executive officer, Sarepta. 'With the temporary pause lifted, the execution of our restructuring, cost savings and plans designed to meet our financial obligations, and with important siRNA readouts expected later this year, we are well positioned to achieve our strategic objective to remain a patient-centric, financially disciplined organization into the next decade and beyond.' Corporate Highlights ELEVIDYS Updates On July 28, 2025, the FDA recommended that Sarepta remove its voluntary pause and resume shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory individuals living with Duchenne. The notification came after the FDA completed its review of ELEVIDYS safety data. Shipments have resumed so that patient infusions can continue. Discussions between FDA and Sarepta are underway on next steps in the safety labeling process for acute liver injury/acute liver failure (ALI/ALF) and risk-mitigation approach for non-ambulatory individuals living with Duchenne. Financial Updates In the second quarter of 2025, the Company delivered strong financial results, reporting both GAAP and non-GAAP operating profit. The quarter was cash flow positive and total cash, cash equivalents and short- and long-term investments increased by $202.8 million from the previous quarter. The Company is taking steps to further strengthen its financial foundation and is focused on proactive liability management. Among many things, it is evaluating opportunities to enhance operational efficiency and adjust manufacturing commitments based on latest demand to further strengthen our liquidity position. The strategic restructuring announced on July 16, 2025, will sharpen the Company's focus on high-impact programs, particularly its siRNA platform, while reducing expenses by approximately $400 million annually starting in 2026. These strategic cost reductions and pipeline prioritization are designed to position Sarepta to repay its 2027 convertible notes and continue delivering transformative therapies to patients with rare genetic diseases. Expected Near-Term Milestones (2025/2026) Facioscapulohumeral muscular dystrophy (FSHD) Study Status: Phase 1/2 study of SRP-1001 FSHD1 patients underway; Cohorts 1, 2, and 3 in part 1 are fully enrolled in the single ascending dose (SAD) study Next Milestone: Preliminary data in second half of 2025 focused on safety, DUX4 mRNA knockdown, DUX4 regulated gene expression, and functional assessments Myotonic dystrophy type 1 (DM1) Study Status: Phase 1/2 study of SRP-1003 in patients with DM1 underway with recent authorization to dose escalate; Cohorts 1 and 2 in the SAD arm of the study fully enrolled Recently Achieved Collaboration Milestone: In July 2025, the program reached the first of two pre-specified enrollment targets, and the safety review warranted dose escalation in the Phase 1/2 study, triggering $100.0 million milestone payment to Arrowhead Pharmaceuticals, Inc. (Arrowhead) Next Milestone: Preliminary Phase 1 data in second half of 2025; primary endpoint is safety, with key secondary endpoints including DMPK knockdown, DMPK-mediated splice indices, and functional assessments such as Video Hand Opening Time (VHOT) Spinocerebellar ataxia type 2 (SCA2) Study Status: Phase 1 study of SRP-1004 in patients with SCA2 underway as a randomized, placebo-controlled, SAD study, and Cohort 1 in this arm of the study has been fully enrolled Next Milestone: On track for First Participant In (FPI), Cohort 2 in the third quarter of 2025 Huntington's Disease (HD) Sarepta intends to file its Clinical Trial Application (CTA) for SRP-1005 in HD by the end of 2025 and initiate its clinical trial in the first half of 2026 2025 ASGCT Annual Conference: Sarepta showcased data spanning different age groups including statistically significant functional outcomes for 8- and 9-year-old patients, and new protein expression and safety results in participants 2 years old at the time of treatment. Part 2 analysis of EMBARK study of 8- and 9-year-olds (n=14) demonstrated statistically significant differences across all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort Results from ENDEAVOR study show treatment in 2-year-old participants with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) All the posters and presentations are available on the Company's website here. As previously announced, there will be no conference call for this quarterly earnings release. Q2 2025 Financial Highlights 1 Revenues Total revenues were $611.1 million for the three months ended June 30, 2025, as compared to $362.9 million for the same period of 2024, an increase of $248.2 million. The increase primarily reflects an increase of $160.1 million in net product revenue of ELEVIDYS as a result of its expanded label approval in June 2024. In addition, collaboration and other revenues increased $95.6 million primarily due to $63.5 million of collaboration revenue recognized related to a milestone payment received from F. Hoffman-La Roche Ltd.'s ('Roche') for the regulatory approval of ELEVIDYS in Japan (the 'Japan Approval Milestone'). Furthermore, contract manufacturing revenues and royalty revenues increased $27.0 million and $5.1 million, respectively, associated with an increase in commercial ELEVIDYS supply delivered to Roche as well as royalty revenue from sales of ELEVIDYS by Roche, respectively. Total revenues were $1,355.9 million for the six months ended June 30, 2025, as compared to $776.4 million for the same period of 2024, an increase of $579.5 million. The increase primarily reflects an increase of $401.2 million in net product revenue of ELEVIDYS as a result of its expanded label approval in June 2024. In addition, collaboration and other revenues increased $174.9 million primarily related to the $63.5 million of collaboration revenue recognized from the Japan Approval Milestone and the $112.0 million of collaboration revenue recognized related to Roche's expiration of an option for a program during the six months ended June 30, 2025, as compared to $48.0 million of collaboration revenue recognized in the same period of 2024 related to Roche's declined option to acquire certain ex-US rights to an external, early-stage Duchenne development program. Furthermore, contract manufacturing revenues and royalty revenues increased $38.6 million and $8.8 million, respectively, associated with an increase in commercial ELEVIDYS supply delivered to Roche as well as royalty revenue from sales of ELEVIDYS by Roche, respectively. Cost of sales (excluding amortization of in-licensed rights) Cost of sales (excluding amortization of in-license rights) were $152.6 million for the three months ended June 30, 2025, as compared to $44.5 million for the same period of 2024, an increase of approximately $108.1 million. Cost of sales (excluding amortization of in-license rights) were $290.1 million for the six months ended June 30, 2025, as compared to $95.1 million for the same period of 2024, an increase of $195.0 million. The increases in both periods primarily reflect the depletion of previously expensed ELEVIDYS inventory, an increased demand for ELEVIDYS following the expanded label approval in June 2024, an increase in products sold to Roche under the Roche collaboration agreement, as well as an increase in the write-offs of certain batches of products not meeting quality specifications for the periods ended June 30, 2025, as compared to the same periods of 2024. Operating expenses and others Research and development expenses were $204.4 million for the three months ended June 30, 2025, as compared to $179.7 million for the same period of 2024, an increase of $24.7 million. The increase is primarily due to an increase in ELEVIDYS clinical material expense as well as $13.0 million of costs associated with our settlement agreement with Brammer Bio MA, LLC ('Brammer'), an affiliate of Thermo Fisher Scientific, Inc., to resolve outstanding claims related to the termination of the development, commercial manufacturing, and supply agreement with Brammer (the 'Brammer Settlement'). For the three months ended June 30, 2025, non-GAAP research and development expenses were $181.7 million, as compared to $153.9 million for the same period of 2024, an increase of $27.8 million. Research and development expenses were $977.8 million for the six months ended June 30, 2025, as compared to $380.1 million for the same period of 2024, an increase of approximately $597.7 million. The increase primarily reflects a $583.6 million increase in up-front and milestone expense associated with the licensing and collaboration agreement and stock purchase agreement with Arrowhead, as well as $13.0 million of costs associated with the Brammer Settlement. For the six months ended June 30, 2025, non-GAAP research and development expenses were $930.9 million, as compared to $332.0 million for the same period of 2024, an increase of $598.9 million. Selling, general and administrative expenses were $137.9 million for the three months ended June 30, 2025, as compared to $138.8 million for the same period of 2024, a decrease of $0.9 million. The decrease is primarily driven by a net decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the three months ended June 30, 2024, partially offset by an increase in professional services used for the commercialization of ELEVIDYS. For the three months ended June 30, 2025, non-GAAP selling, general and administrative expenses were $113.4 million, as compared to $106.0 million for the same period of 2024, an increase of $7.4 million. Selling, general and administrative expenses were $271.5 million for the six months ended June 30, 2025, as compared to $265.8 million for the same period of 2024, an increase of $5.7 million. The increase is primarily driven by an increase in compensation and other personnel expenses as well as an increase in professional services used for the commercialization of ELEVIDYS, partially offset by a net decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the six months ended June 30, 2024. For the six months ended June 30, 2025, non-GAAP selling, general and administrative expenses were $220.5 million, as compared to $206.5 million for the same period of 2024, an increase of $14.0 million. Other income (expense), net for the three months ended June 30, 2025 and 2024, was $38.1 million and $14.3 million, respectively. Other (expense) income, net for the six months ended June 30, 2025 and 2024, was $(45.1) million and $20.8 million, respectively. The changes in each period primarily reflect the fair value adjustments of our investments in publicly-traded companies, including Arrowhead, during the periods ended June 30, 2025. Income tax (benefit) expense for the three months ended June 30, 2025 and 2024, was $(43.3) million and $7.1 million, respectively. Income tax expense for the six months ended June 30, 2025 and 2024, was $20.7 million and $12.4 million, respectively. Income tax (benefit) expense for all periods presented primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, we have included the following non-GAAP measurements: Non-GAAP net income (loss) is defined by us as GAAP net income (loss) excluding interest income/expense, net, depreciation and amortization expense, stock-based compensation expense, gain/loss on strategic investments, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items. Non-GAAP earnings per share is defined by us as non-GAAP net income, as defined previously, divided by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding, adjusted for the inclusion of additional shares under the 'if-converted' method, if applicable and not anti-dilutive. Non-GAAP net loss per share is defined by us as non-GAAP net loss, as defined above, divided by the weighted-average number of shares of common stock outstanding as the inclusion of dilutive common stock equivalents outstanding is anti-dilutive. Non-GAAP operating income (loss) is defined by us as GAAP operating income (loss) excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items. The following components are used to adjust our GAAP financial measures into the previously defined non-GAAP measurements: Interest, depreciation and amortization - Interest income (expense), net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of our operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to our operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal. Stock-based compensation expenses - Stock-based compensation expenses represent non-cash charges related to equity awards we have granted. Although these are recurring charges to operations, we believe the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within our control. Therefore, we believe that excluding these charges facilitates comparisons of our operational performance in different periods. Other items - We evaluate other items of expense and income on an individual basis. We take into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to our ongoing business operations, and (c) whether we expect the items to continue or occur on a regular basis. These other items include the gain/loss on strategic investments and changes in the fair value of derivatives and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results: a) The (gain) loss on strategic investments as it is a non-cash item and the results of such gains and losses are not representative of our normal business operations, which accordingly, would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures. We are making this change beginning in 2025 because, as our strategic investments have increased, we recognized that the resulting variability can impede comparability between periods of our financial performance for our ongoing business operations. b) The change in fair value of derivatives related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus Therapeutics, Inc. selling shareholders as well as to an academic institution under a separate license agreement as these are non-cash items and are not considered to be normal operating expenses due to the variability of amounts and lack of predictability as to occurrence and/or timing. Expand We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP operating (loss) income, non-GAAP net (loss) income, and non-GAAP diluted (loss) earnings per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table 'Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.' About EXONDYS 51 EXONDYS 51 uses Sarepta's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or 'skipping', of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information About EXONDYS 51 Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended. The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting. Other adverse events may occur. To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or For further information, please see the full US Prescribing Information for EXONDYS 51 (eteplirsen). About VYONDYS 53 VYONDYS 53 (golodirsen) uses Sarepta's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or 'skipping,' of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for VYONDYS 53 CONTRAINDICATIONS: VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Kidney Toxicity: Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. ADVERSE REACTIONS: Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%). Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture. Other adverse events may occur. To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or For further information, please see the full US Prescribing Information for VYONDYS 53 (golodirsen). About AMONDYS 45 AMONDYS 45 (casimersen) uses Sarepta's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or 'skipping,' of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for AMONDYS 45 CONTRAINDICATION: AMONDYS 45 is contraindicated in patients with a known serious hypersensitivity to casimersen or any of the inactive ingredients in AMONDYS 45. Instances of hypersensitivity including angioedema and anaphylaxis have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment, and consider slowing the infusion, interrupting, or discontinuing the AMONDYS 45 infusion and monitor until the condition resolves. AMONDYS 45 is contraindicated in patients with known serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45. Kidney Toxicity: Kidney toxicity was observed in animals who received casimersen. Although kidney toxicity was not observed in the clinical studies with AMONDYS 45, kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking AMONDYS 45. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse Reactions: Adverse reactions occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were (AMONDYS 45, placebo): upper respiratory infections (65%, 55%), cough (33%, 26%), pyrexia (33%, 23%), headache (32%, 19%), arthralgia (21%, 10%), and oropharyngeal pain (21%, 7%). Other adverse reactions that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were: ear pain, nausea, ear infection, post-traumatic pain, and dizziness and light-headedness. Other adverse events may occur. To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or For further information, please see the full US Prescribing Information for AMONDYS 45 (casimersen). About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as 'micro-dystrophin') in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Important Safety Information for ELEVIDYS CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration of ELEVIDYS may result in elevations of liver enzymes (e.g., GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, a consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness including dysphagia, dyspnea and hypophonia were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene between exons 1 to 17 and exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea or hypophonia as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Pre-existing Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all subjects developed anti-AAVrh74 antibodies. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥ 5%) reported in clinical studies were vomiting, nausea, liver function test increased, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information for ELEVIDYS (delandistrogene moxeparvovec-rokl). About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Forward-Looking Statements In order to provide Sarepta's investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as 'believes,' 'anticipates,' 'plans,' 'expects,' 'will,' 'may,' 'intends,' 'prepares,' 'looks,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our future operations, financial performance and projections, business plans, market opportunities, priorities and research and development programs and technologies; our clinical trials; the potential benefits of our technologies and scientific approaches; ongoing discussions with FDA related to ELEVIDYS, including the safety labeling process and risk-mitigation approach for non-ambulatory individuals; and expected plans and milestones, including multiple clinical data readouts and milestones expected in 2025 and into early 2026 for our siRNA franchise and working with FDA to resume shipments of ELEVIDYS to non-ambulatory individuals. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, the COVID-19 pandemic and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading 'Risk Factors' in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 and our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands, except per share amounts) For the Three Months Ended June 30, For the Six Months Ended June 30, 2025 2024 2025 2024 GAAP net income (loss) $ 196,892 $ 6,460 $ (250,616 ) $ 42,579 Interest income, net (1,921 ) (14,010 ) (9,846 ) (29,741 ) Depreciation and amortization expense 10,173 8,118 19,550 16,261 Stock-based compensation expense 37,025 50,482 78,453 91,174 Change in fair value of derivatives — — — 10,100 (Gain) loss on strategic investments* (36,721 ) (148 ) 54,007 (1,079 ) Income tax effect of adjustments 9,728 (4,389 ) (8,870 ) (5,472 ) Non-GAAP net income (loss) $ 215,176 $ 46,513 $ (117,322 ) $ 123,822 GAAP net earnings (loss) per share - diluted: $ 1.89 $ 0.07 $ (2.57 ) $ 0.44 Add: impact of GAAP to Non-GAAP adjustments $ 0.13 $ 0.36 $ 1.37 $ 0.71 Non-GAAP net earnings (loss) per share - diluted** $ 2.02 $ 0.43 $ (1.20 ) $ 1.15 Weighted average number of shares of common stock used in computing diluted earnings (loss) per share:*** GAAP 106,623 99,144 97,685 99,129 *Beginning in the first quarter of 2025, (gain) loss on strategic investments was included as a non-GAAP measurement to adjust our GAAP financial measures. Non-GAAP financial results for the three and six months ended June 30, 2024, have been updated to reflect this change for comparability. Please refer to the 'Use of Non-GAAP Measures' section above for additional detail. **Non-GAAP earnings per share is calculated using diluted shares whereas non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. ***The difference between the weighted average number of shares of common stock used in computing diluted GAAP and non-GAAP earnings per share for the three and six months June 30, 2024, is a result of the exclusion of the potential share settlement of the 2027 Convertible Notes from the GAAP earnings per share as the inclusion of such shares was anti-dilutive during those periods. Expand Expand Expand For the Three Months Ended June 30, For the Six Months Ended June 30, 2025 2024 2025 2024 GAAP selling, general and administrative expenses $ 137,897 $ 138,796 $ 271,526 $ 265,799 Stock-based compensation expense (21,748 ) (30,676 ) (45,859 ) (55,095 ) Depreciation expense (2,776 ) (2,136 ) (5,176 ) (4,233 ) Non-GAAP selling, general and administrative expenses $ 113,373 $ 105,984 $ 220,491 $ 206,471 Expand Expand Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of June 30, 2025 As of December 31, 2024 Assets Current assets: Cash and cash equivalents $ 510,598 $ 1,103,010 Short-term investments 289,541 251,782 Accounts receivable, net 527,295 601,988 Inventory 994,036 749,960 Manufacturing-related deposits and prepaids 207,921 276,262 Other current assets 127,594 90,461 Total current assets 2,656,985 3,073,463 Property and equipment, net 371,857 340,336 Right of use assets 143,041 148,310 Non-current inventory 194,668 187,986 Strategic investments 195,522 3,710 Non-current investments 34,604 133,163 Other non-current assets 83,137 76,205 Total assets $ 3,679,814 $ 3,963,173 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 136,702 $ 214,442 Accrued expenses 377,399 373,513 Deferred revenue, current portion 395,431 130,256 Other current liabilities 10,416 13,473 Total current liabilities 919,948 731,684 Long-term debt 1,139,458 1,137,124 Lease liabilities, net of current portion 214,419 192,473 Deferred revenue, net of current portion — 325,000 Contingent consideration 47,400 47,400 Other non-current liabilities 1,204 1,750 Total liabilities 2,322,429 2,435,431 Stockholders' equity: Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding — — Common stock, $0.0001 par value, 198,000,000 shares authorized; 98,356,950 and 97,706,074 issued and outstanding, respectively, at June 30, 2025 and 96,900,496 issued and outstanding at December 31, 2024 10 10 Treasury stock, at cost, 650,876 and 0 shares at June 30, 2025 and December 31, 2024, respectively (25,263 ) — Additional paid-in capital 5,844,279 5,738,924 Accumulated other comprehensive loss, net of tax (51 ) (218 ) Accumulated deficit (4,461,590 ) (4,210,974 ) Total stockholders' equity 1,357,385 1,527,742 Total liabilities and stockholders' equity $ 3,679,814 $ 3,963,173 Expand Source: Sarepta Therapeutics, Inc.
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US FDA approves Jazz Pharma's drug for rare brain tumor
By Padmanabhan Ananthan and Puyaan Singh (Reuters) -The U.S. Food and Drug Administration said on Wednesday that it has approved Jazz Pharmaceuticals' drug to treat diffuse midline glioma, a rare and aggressive tumor, in adults and children aged one year and older. The approval expands Jazz's cancer drug portfolio beyond its existing treatments for certain blood and lung cancers. This is the first FDA-approved systemic therapy for diffuse midline glioma with a specific mutation that has progressed despite prior treatments, the agency said. The drug, Modeyso, is expected to become available in the U.S. in the coming weeks and is administered as a once-weekly oral capsule. Diffuse midline glioma (DMG) is a rare and aggressive brain tumor that primarily affects children and young adults. It develops in the brain's and spinal cord's midline structures, such as the brainstem, thalamus, and spinal cord. An estimated 3,940 people are living with this tumor in the United States, according to NIH data. The FDA decision was based on data from 50 patients in five clinical studies, showing that the drug helped shrink tumors in about 22% of cases. Among those who responded, the benefit lasted a median of just over 10 months. "We think it fits very well in terms of addressing a very high unmet need," said Rob Iannone, Chief Medical Officer at Jazz Pharmaceuticals, ahead of the decision. Jazz Pharmaceuticals acquired the drug in March through its $935 million purchase of Chimerix. The company says it plans to work with doctors and advocates to make the treatment available as quickly as possible. Solve the daily Crossword
