Vertex Announces European Commission Approval of ALYFTREK ®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis
'Thousands of people with CF across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus KAFTRIO,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with CF.'
In two head-to-head pivotal clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior to KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor on ppFEV 1 and superior at reducing sweat chloride, demonstrating greater improvement in CFTR function.
'CF care has been transformed by the advent of highly effective CFTR modulators, and I am very pleased that we now have a new treatment option to even better address this multi-systemic disease,' said Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité Universitätsmedizin Berlin. 'Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care. By bringing more people closer to normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients.'
As a result of reimbursement agreements in Ireland and Denmark and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement bodies across the European Union member states to ensure access for all eligible patients as quickly as possible.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.
Today Vertex CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
Sweat chloride is used to diagnose CF, which measures CFTR function. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease.
About ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor)
In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane.
ALYFTREK is approved in the EU for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
For complete product information, please see the Summary of Product Characteristics (SmPC) that can be found at www.ema.europa.eu.
ALYFTREK is currently licensed in the U.S., the UK and the European Union and is under regulatory review in Canada, Switzerland, Australia and New Zealand.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com/en-global.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Reshma Kewalramani, M.D., and Professor Marcus A. Mall, M.D., in this press release and statements regarding the expectations for the potential benefits of ALYFTREK, expectations for the eligible patient population in the EU, expectations for patient access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the European Commission, and Vertex's continued work with reimbursement bodies across the EU member states to ensure access for all eligible patients as quickly as possible. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading 'Risk Factors' in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
20 minutes ago
- Yahoo
Heartflow raises $316.7 million in US IPO as medtech listings attempt comeback
By Ateev Bhandari and Pritam Biswas (Reuters) -Medical technology firm Heartflow said on Thursday it had raised $316.7 million in its U.S. initial public offering, setting the stage for its Nasdaq debut in another test of investor appetite for medical tech companies. The Mountain View, California-based company sold 16.67 million shares at $19 each, compared with a targeted range of $17 to $18 apiece. It upsized its offering and raised the proposed range earlier this week reflecting strong demand. The IPO valued the company at $1.54 billion. U.S. IPOs have picked up pace in a much-awaited recovery, as solid tech earnings and progress on trade deals have helped restore investor confidence. Strong recent debuts mark a reversal from early April, when uncertainty around U.S. President Donald Trump's tariffs paused dealmaking. Heartflow's stock will begin trading on the Nasdaq on Friday under the "HTFL" ticker symbol. The closing of the offering is expected on August 11. J.P. Morgan, Morgan Stanley and Piper Sandler are the lead underwriters for the offering. Following software firm Figma's blowout debut last week, analysts expect companies with a compelling growth story to be welcomed by the public markets. "We expect Heartflow to be well received at launch, especially following Figma's breakout success, which has helped reset sentiment toward high-growth names," IPOX CEO Josef Schuster said. Bain Capital-backed Heartflow leverages AI to create 3D models of the heart via a single specialized scan, improving detection and treatment of coronary artery disease (CAD). As AI gains widespread acceptance, companies embedding it into workflows for meaningful use cases are catching investor attention, with the technology powering gains across various Big Tech businesses. Heartflow's revenue grew 39% for the quarter ended March 31 from a year earlier. Its platform to detect CAD was used in 132,000 patients in 2024. Sign in to access your portfolio
Business Wire
an hour ago
- Business Wire
TruBridge Announces Second Quarter 2025 Results
MOBILE, Ala.--(BUSINESS WIRE)--TruBridge, Inc. (NASDAQ: TBRG), a healthcare solutions company, today announced financial results for the second quarter and six months ended June 30, 2025. Second Quarter 2025 Highlights* All comparisons are to the quarter ended June 30, 2024, unless otherwise noted Total bookings of $25.6 million compared to $23.3 million Total revenue of $85.7 million compared to $85.6 million Recurring revenue represented 95% of total revenue Financial Health revenue of $54.3 million compared to $54.5 million Financial Health revenue represented 63% of TruBridge's total revenue GAAP net income of $2.6 million compared to a net loss of $4.4 million Non-GAAP net income of $7.9 million compared to $3.0 million Adjusted EBITDA of $13.7 million compared to $13.4 million *As of the third quarter of 2024, TruBridge is now reporting two segments in its financial statements representing the two business units. Financial Health represents the previous Revenue Cycle Management (RCM) segment, and Patient Care represents the previous Electronic Health Record (EHR) segment, including the patient engagement business. Commenting on the results, Chris Fowler, chief executive officer of TruBridge, Inc., stated, 'During the second quarter, we continued to make improvements on many fronts, advancing steadily towards achievement of our long-term objectives. Strong bookings, as well as improved profitability and cash flow, give us confidence in the value we provide to our clients, our position in the market, and the benefits of the work we did to improve the financial health of our business. 'Our north star is client delight, and we are implementing a strategic plan designed to bring our client satisfaction levels back to historical levels and beyond. While we have brought down the top end of our revenue outlook for the full year as a result of client attrition and the reality of signing larger, more complex deals, we are also raising our Adjusted EBITDA range to incorporate the efficiencies realized by our offshoring initiative, our refinement of resource management, and cost optimization. We remain confident that the steps we are taking today to refine and optimize our approach will set us up for success in the quarters and years ahead as we work to deliver exceptional experiences to the communities we serve,' added Fowler. Financial Guidance For the third quarter of 2025, TruBridge expects to generate: Total revenue of $85 million to $87 million Adjusted EBITDA of $14 million to $16 million For the full year 2025, TruBridge expects to generate: Total revenue of $345 million to $350 million; revised from $345 million to $360 million Adjusted EBITDA of $62 million to $67 million; revised from $60 million to $66 million Conference Call TruBridge will hold a conference call and live webcast to discuss second quarter 2025 results on Friday, August 8, 2025, at 7:30 a.m. Central time/8:30 a.m. Eastern time. To access this interactive teleconference, dial (877) 407-0890 and request connection to the TruBridge earnings conference call. A 30-day online replay will be available approximately one hour following the conclusion of the live webcast. To listen to the live webcast or access the replay, visit the Company's investor relations website, About TruBridge TruBridge proudly supports rural and community hospitals and providers in their efforts to stay strong, independent, and deeply rooted in the communities they serve. Backed by more than 45 years of healthcare experience and trusted by over 1,500 clients nationwide, we offer a mix of technology, services, and strategic expertise — including revenue cycle management, electronic health records (EHR) and analytics — all designed singularly for the realities of rural and community healthcare. With a steadfast commitment to keeping care local, TruBridge helps hospitals flourish as the economic heart of their communities, delivering high-quality, personal care close to home. For more information, visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified generally by the use of forward-looking terminology and words such as 'expects,' 'anticipates,' 'estimates,' 'believes,' 'predicts,' 'intends,' 'plans,' 'potential,' 'may,' 'continue,' 'should,' 'will' and words of comparable meaning. Without limiting the generality of the preceding statement, all statements in this press release relating to the Company's future financial and operational results are forward-looking statements. We caution investors that any such forward-looking statements are only predictions and are not guarantees of future performance. Certain risks, uncertainties and other factors may cause actual results to differ materially from those projected in the forward-looking statements. Such factors may include: saturation of our target market and hospital consolidations; unfavorable economic or market conditions that may cause a decline in spending for information technology and services; significant legislative and regulatory uncertainty in the healthcare industry; exposure to liability for failure to comply with regulatory requirements; transition to a subscription based recurring revenue model and modernization of our technology; competition with companies that have greater financial, technical and marketing resources than we have; potential future acquisitions that may be expensive, time consuming, and subject to other inherent risks; our ability to attract and retain qualified personnel in a global workforce; disruption from periodic restructuring of our sales force; slower than anticipated development of the market for Financial Health services; potential inability to properly manage growth in new markets we may enter; potential failure to effectively implement a new enterprise resource planning software solution; exposure to numerous and often conflicting laws, regulations, policies, standards or other requirements through our domestic and international business activities; potential litigation against us and investigations; our use of offshore third-party resources; competitive and litigation risk related to the use of artificial intelligence; potential failure to develop new products or enhance current products that keep pace with market demands; failure of our products to provide accurate and timely information for clinical decision-making; breaches of security and viruses in our systems resulting in customer claims against us and harm to our reputation; failure to maintain customer satisfaction through new product releases free of undetected errors or problems; failure to convince customers to migrate to current or future releases of our products; failure to maintain our margins and service rates; increase in the percentage of total revenues represented by service revenues, which have lower gross margins; exposure to liability in the event we provide inaccurate claims data to payors; exposure to liability claims arising out of the licensing of our software and provision of services; dependence on licenses of rights, products and services from third parties; failure to protect our intellectual property rights; exposure to significant license fees or damages for intellectual property infringement; interruptions in our power supply and/or telecommunications capabilities; potential inability to secure additional financing on favorable terms to meet our future capital needs; our substantial indebtedness, and our ability to incur additional indebtedness in the future; pressures on cash flow to service our outstanding debt; restrictive terms of our credit agreement on our current and future operations; changes in and interpretations of financial accounting matters that govern the measurement of our performance; significant charges to earnings if our goodwill or intangible assets become impaired; fluctuations in quarterly financial performance due to various factors; volatility in our stock price; failure to maintain effective internal control over financial reporting; inherent limitations in our internal control over financial reporting; vulnerability to significant damage from natural disasters; market risks related to interest rate changes; potential material adverse effects due to macroeconomic conditions; we do not anticipate paying dividends on our common stock; actions of activist stockholders against us; and other risk factors described from time to time in our public releases and reports filed with the Securities and Exchange Commission. We also caution investors that the forward-looking information described herein represents our outlook only as of this date, and we undertake no obligation to update or revise any forward-looking statements to reflect events or developments after the date of this press release. TruBridge, Inc. Condensed Consolidated Statements of Operations (Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 * 2025 2024 * Revenues Financial Health $ 54,284 $ 54,509 $ 110,417 $ 107,948 Patient Care 31,445 31,091 62,520 61,769 Total revenues 85,729 85,600 172,937 169,717 Expenses Costs of revenue (exclusive of amortization and depreciation) Financial Health 29,308 30,269 56,499 59,866 Patient Care 11,962 13,073 24,284 25,237 Total costs of revenue (exclusive of amortization and depreciation) 41,270 43,342 80,783 85,103 Product development 8,113 8,207 16,360 18,894 Sales and marketing 8,041 7,815 13,450 14,408 General and administrative 18,076 18,878 37,540 38,274 Amortization 6,290 9,107 12,414 14,975 Depreciation 312 400 603 800 Total expenses 82,102 87,749 161,150 172,454 Operating income (loss) 3,627 (2,149 ) 11,787 (2,737 ) Other income (expense): Interest expense (3,065 ) (4,242 ) (6,447 ) (8,315 ) Other income 136 91 280 1,514 Total other expense (2,929 ) (4,151 ) (6,167 ) (6,801 ) Income (loss) before taxes 698 (6,300 ) 5,620 (9,538 ) Provision for (benefit from) income taxes (1,882 ) (1,912 ) 2,581 (3,296 ) Net income (loss) $ 2,580 $ (4,388 ) $ 3,039 $ (6,242 ) Net income (loss) per common share—basic $ 0.17 $ (0.29 ) $ 0.20 $ 0.42 Net income (loss) per common share—diluted $ 0.17 $ (0.29 ) $ 0.20 $ 0.42 Weighted average shares outstanding used in per common share computations: Basic 14,522 14,313 14,446 14,273 Diluted 14,522 14,313 14,446 14,273 *As described in the 2024 Annual Report, certain line items have been revised to correct an error related to the reversal of revenue from customers that was recognized improperly during 2023. These revisions increased revenue for the three and six months ended June 30, 2024 by $0.9 million and $1.7 million, respectively. These revisions had no cash flow consequences. Expand TruBridge, Inc. Condensed Consolidated Balance Sheets (In '000s, except per share data) June 30, 2025 (Unaudited) December 31, 2024 Assets Current assets Cash and cash equivalents $ 12,279 $ 12,324 Accounts receivable, net of allowance for expected credit losses of $5,208 and $5,861 56,432 53,753 Current portion of financing receivables, net of allowance for expected credit losses of $560 and $417 2,727 4,663 Inventories 444 767 Prepaid income taxes 3,459 2,886 Prepaid expenses and other current assets 14,473 15,275 Assets held for sale 445 606 Total current assets 90,259 90,274 Property & equipment, net 2,559 2,294 Software development costs, net 43,317 41,474 Operating lease right-of-use assets 2,617 3,092 Financing receivables, less current portion, less allowance for expected credit losses of $258 and $21 22 232 Other assets, less current portion 8,196 7,786 Intangible assets, net 70,608 76,707 Goodwill 172,573 172,573 Total assets $ 390,151 $ 394,432 Liabilities & Stockholders' Equity Current liabilities Accounts payable $ 19,672 $ 15,040 Current portion of long-term debt 2,980 2,980 Deferred revenue 9,368 10,653 Accrued vacation 5,235 4,770 Income taxes payable 623 3,538 Other accrued liabilities 12,302 15,994 Total current liabilities 50,180 52,975 Long-term debt, less current portion 163,108 168,598 Operating lease liabilities, less current portion 1,827 2,293 Deferred tax liabilities 1,863 1,871 Total liabilities 216,978 225,737 Stockholders' Equity Common stock, $0.001 par value; 30,000 shares authorized; 15,700 and 15,522 shares issued 15 15 Additional paid-in capital 204,376 201,066 Retained deficit (11,913 ) (14,952 ) Accumulated other comprehensive income 27 45 Treasury stock, 685 and 619 shares (19,332 ) (17,479 ) Total stockholders' equity 173,173 168,695 Total liabilities and stockholders' equity $ 390,151 $ 394,432 Expand TruBridge, Inc. Condensed Consolidated Statements of Cash Flows (In '000s) (Unaudited) Six Months Ended June 30, 2025 2024 * Operating activities: Net income (loss) $ 3,039 $ (6,242 ) Adjustments to net income (loss): Provision for credit losses 1,609 358 Deferred taxes (7 ) (5,224 ) Stock-based compensation 3,310 2,300 Depreciation 603 800 Gain on sale of business (53 ) (1,250 ) Amortization of acquisition-related intangibles 6,098 6,253 Amortization of software development costs 6,316 8,722 Amortization of deferred finance costs 259 213 Non-cash operating lease costs 537 897 Gain on disposal of property and equipment (120 ) - Changes in operating assets and liabilities: Accounts receivable (3,967 ) (1,085 ) Financing receivables 1,825 506 Inventories 323 (318 ) Prepaid expenses and other assets (1,827 ) 1,502 Accounts payable 5,082 5,750 Deferred revenue (1,284 ) 1,769 Operating lease liabilities (548 ) (583 ) Other liabilities (3,191 ) (2,375 ) Income taxes, net (3,487 ) (263 ) Net cash provided by operating activities 14,517 11,730 Investing activities: Purchase of business, net of cash acquired - (664 ) Sale of business, net of cash and cash equivalent sold 2,102 21,410 Investment in software development (8,159 ) (9,324 ) Purchases of property and equipment (902 ) (306 ) Net cash (used in) provided by investing activities (6,959 ) 11,116 Financing activities: Payments of long-term debt principal (1,750 ) (5,750 ) Proceeds from revolving line of credit 15,368 21,072 Payments of revolving line of credit (19,368 ) (33,379 ) Debt issuance cost - (529 ) Treasury stock purchases (1,853 ) (358 ) Net cash used in financing activities (7,603 ) (18,944 ) (Decrease) Increase in cash and cash equivalents (45 ) 3,902 Change in cash and cash equivalents included in assets sold - (41 ) Cash and cash equivalents, beginning of period 12,324 3,848 Cash and cash equivalents, end of period $ 12,279 $ 7,709 *As described in the 2024 Annual Report, certain line items have been revised to correct an error related to the reversal of revenue from customers that was recognized improperly during 2023. These revisions increased revenue for the six months ended June 30, 2024 by $1.7 million. These revisions had no cash flow consequences. Expand TruBridge, Inc. Consolidated Bookings (In '000s) (Unaudited) (Non-GAAP) Three Months Ended June 30, Six Months Ended June 30, In '000s 2025 2024 2025 2024 Financial Health (1) $ 13,705 $ 13,458 $ 26,485 $ 27,849 Patient Care (2) 11,908 9,832 21,109 19,010 Total Bookings $ 25,613 $ 23,290 $ 47,594 $ 46,859 (1) Generally calculated as the annual contract value (2) Generally calculated as the total contract value for system sales and SaaS, and annual contract value for maintenance and support Annual Contract Value Effective January 2025, the Company will be providing bookings on an Annual Contract Value ('ACV') basis in addition to the reported bookings amounts, which has historically represented a mix of ACV and Total Contract Value ('TCV') for Patient Care. This new methodology of reporting total bookings at ACV represents the newly contracted revenue that is expected to be recognized over a twelve-month period. Over the course of 2025, the Company will be providing total bookings under both methodologies for year over year comparability before fully transitioning to ACV in 2026. The below table represents bookings at the ACV methodology for the three and six months ended June 30, 2025: Six Months Ended June 30, In '000s 2025 2025 Financial Health $ 13,705 $ 26,485 Patient Care 5,921 10,480 Total Bookings (ACV) $ 19,626 $ 36,965 Expand TruBridge, Inc. Bookings Composition (In '000s, except per share data) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, In '000s 2025 2024 2025 2024 Financial Health Net new (1) $ 5,067 $ 6,453 $ 11,529 $ 15,446 Cross-sell (1) 8,638 7,004 14,956 12,402 Patient Care Non-subscription sales (2) 2,730 4,084 5,332 7,534 Subscription revenue (3) 9,178 5,749 15,777 11,477 Total Bookings $ 25,613 $ 23,290 $ 47,594 $ 46,859 (1) 'Net new' represents bookings from outside the Company's core client base, and 'Cross-sell' represents bookings from existing customers. In each case, such bookings are generally comprised of recurring revenues to be recognized ratably over a one-year period and an average timeframe for bookings-to-revenue conversion of four to six months following contract execution. (2) Represents nonrecurring revenues that generally exhibit a timeframe for bookings-to-revenue conversion of five to six months following contract execution. (3) Represents recurring revenues to be recognized on a monthly basis over a weighted-average contract period of five years, with a start date in the next 12 months and an average timeframe for commencement of bookings-to-revenue conversion of five to six months following contract execution. Annual Contract Value Effective January 2025, the Company will be providing bookings on an Annual Contract Value ('ACV') basis in addition to the reported bookings amounts, which has historically represented a mix of ACV and Total Contract Value ('TCV') for Patient Care. This new methodology of reporting total bookings at ACV represents the newly contracted revenue that is expected to be recognized over a twelve-month period. Over the course of 2025, the Company will be providing total bookings under both methodologies for year over year comparability before fully transitioning to ACV in 2026. The below table represents bookings at the ACV methodology for the three and six months ended June 30, 2025: Three Months Ended June 30, Six Months Ended June 30, In '000s 2025 2025 Financial Health Net new (1) $ 5,067 $ 11,529 Cross-sell (1) 8,638 14,956 Patient Care Non-subscription sales (2) 2,730 5,332 Subscription revenue (3) 3,191 5,148 Total Bookings (ACV) $ 19,626 $ 36,965 Expand TruBridge, Inc. Adjusted EBITDA - by Segment (In '000s) (Unaudited) (Non-GAAP) Three Months Ended June 30, Six Months Ended June 30, In '000s 2025 2024 * 2025 2024 * Financial Health $ 7,092 $ 8,209 $ 18,373 $ 15,006 Patient Care 6,651 $ 5,235 13,601 8,762 Total Adjusted EBITDA $ 13,743 $ 13,444 $ 31,974 $ 23,768 *As described in the 2024 Annual Report, certain line items have been revised to correct an error related to the reversal of revenue from customers that was recognized improperly during 2023. These revisions increased revenue for the three and six months ended June 30, 2024 by $0.9 million and $1.7 million, respectively. These revisions had no cash flow consequences. Expand TruBridge, Inc. Reconciliation of Non-GAAP Financial Measures (In '000s) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, Adjusted EBITDA: 2025 2024 * 2025 2024 * Total Adjusted EBITDA $ 13,743 $ 13,444 $ 31,974 $ 23,768 Adjusted EBITDA Margin 16.0 % 15.7 % 18.5 % 14.0 % Depreciation expense 312 400 603 800 Amortization of software development costs 3,245 5,980 6,316 8,722 Amortization of acquisition-related intangibles 3,046 3,126 6,098 6,253 Stock-based compensation 2,097 1,501 3,310 2,300 Severance and other nonrecurring charges 1,416 4,586 3,860 8,430 Interest expense and other income 2,929 4,151 6,340 8,051 Gain on disposal of property and equipment - - (120 ) - Gain on sale of AHT - - (53 ) (1,250 ) Income (loss) before taxes, as reported 698 (6,300 ) 5,620 (9,538 ) Provision for (benefit from) income taxes (1,882 ) (1,912 ) 2,581 (3,296 ) Net income (loss), as reported $ 2,580 $ (4,388 ) $ 3,039 $ (6,242 ) Net income (loss) margin 3.0 % (5.1 %) 1.8 % (3.7 %) *As described in the 2024 Annual Report, certain line items have been revised to correct an error related to the reversal of revenue from customers that was recognized improperly during 2023. These revisions increased revenue for the three and six months ended June 30, 2024 by $0.9 million and $1.7 million, respectively. These revisions had no cash flow consequences. Expand TruBridge, Inc. Reconciliation of Non-GAAP Financial Measures (In '000s, except per share data) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, Non-GAAP Net Income and Non-GAAP EPS: 2025 2024 * 2025 2024 * Net income (loss), as reported $ 2,580 $ (4,388 ) $ 3,039 $ (6,242 ) Pre-tax adjustments for Non-GAAP EPS: Amortization of acquisition-related intangible assets 3,046 3,126 6,098 6,253 Stock-based compensation 2,097 1,501 3,310 2,300 Severance and other nonrecurring charges 1,416 4,586 3,860 8,430 Non-cash interest expense 130 107 260 213 Gain on sale of AHT - - (53 ) (1,250 ) After-tax adjustments for Non-GAAP EPS: Tax-effect of pre-tax adjustments, at 21% (1,405 ) (1,957 ) (2,830 ) (3,349 ) Tax shortfall (windfall) from stock-based compensation - 4 (670 ) 113 Non-GAAP net income $ 7,864 $ 2,979 $ 13,014 $ 6,468 Weighted average shares outstanding, diluted 14,522 14,313 14,446 14,273 Non-GAAP EPS $ 0.54 $ 0.21 $ 0.90 $ 0.45 *As described in the 2024 Annual Report, certain line items have been revised to correct an error related to the reversal of revenue from customers that was recognized improperly during 2023. These revisions increased revenue for the three and six months ended June 30, 2024 by $0.9 million and $1.7 million, respectively. These revisions had no cash flow consequences. Expand TruBridge, Inc. Revenue Composition (In '000s) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 * 2025 2024 * Recurring revenues Financial Health $ 53,322 $ 52,798 $ 108,586 $ 104,914 Patient Care 28,115 27,135 55,562 55,678 Total recurring revenues 81,437 79,933 164,148 160,592 Non-recurring revenues Financial Health 962 1,711 1,831 3,034 Patient Care 3,330 3,956 6,958 6,091 Total non-recurring revenues 4,292 5,667 8,789 9,125 Total revenues $ 85,729 $ 85,600 $ 172,937 $ 169,717 *As described in the 2024 Annual Report, certain line items have been revised to correct an error related to the reversal of revenue from customers that was recognized improperly during 2023. These revisions increased revenue for the three and six months ended June 30, 2024 by $0.9 million and $1.7 million, respectively. These revisions had no cash flow consequences. Expand Explanation of Non-GAAP Financial Measures We report our financial results in accordance with accounting principles generally accepted in the United States of America, or 'GAAP.' However, management believes that, in order to properly understand our short-term and long-term financial and operational trends, investors may wish to consider the impact of certain non-cash or non-recurring items, when used as a supplement to financial performance measures that are prepared in accordance with GAAP. These items result from facts and circumstances that vary in frequency and impact on continuing operations. Management uses these non-GAAP financial measures in order to evaluate the operating performance of the Company and compare it against past periods, make operating decisions, and serve as a basis for strategic planning. These non-GAAP financial measures provide management with additional means to understand and evaluate the operating results and trends in our ongoing business by eliminating certain non-cash expenses and other items that management believes might otherwise make comparisons of our ongoing business with prior periods more difficult, obscure trends in ongoing operations, or reduce management's ability to make useful forecasts. In addition, management understands that some investors and financial analysts find these non-GAAP financial measures helpful in analyzing our financial and operational performance and comparing this performance to our peers and competitors. We do not provide a reconciliation of the non-GAAP guidance measure Adjusted EBITDA for the second quarter of 2025 or the fiscal year 2025 to net income for such periods, the most comparable GAAP financial measure, due to the inherent difficulty of forecasting certain types of expenses and gains, without unreasonable effort, which affect net income but not Adjusted EBITDA. As such, to supplement the GAAP information provided, we present in this press release and during the live webcast discussing our financial results the following non-GAAP financial measures: Adjusted EBITDA, Adjusted EBITDA Margin, Non-GAAP net income, and Non-GAAP earnings per share ('EPS'). We calculate each of these non-GAAP financial measures as follows: Adjusted EBITDA – Adjusted EBITDA consists of GAAP net income as reported and adjusts for (i) depreciation expense; (ii) amortization of software development costs; (iii) amortization of acquisition-related intangibles; (iv) stock-based compensation; (v) severance and other nonrecurring charges; (vi) interest expense and other income; (vii) gain on disposal of property and equipment; (viii) gain on sale of AHT; and (ix) the provision for (benefit from) income taxes. Adjusted EBITDA Margin – Adjusted EBITDA Margin is calculated as Adjusted EBITDA, as defined above, divided by total revenue. Non-GAAP net income – Non-GAAP net income consists of GAAP net income as reported and adjusts for (i) amortization of acquisition-related intangible assets; (ii) stock-based compensation; (iii) severance and other nonrecurring charges; (iv) non-cash interest expense; (v) gain on sale of AHT; and (vi) the total tax effect of items (i) through (v). Non-GAAP EPS – Non-GAAP EPS consists of Non-GAAP net income, as defined above, divided by weighted average shares outstanding (diluted) in the applicable period. Certain of the items excluded or adjusted to arrive at these non-GAAP financial measures are described below: Amortization of acquisition-related intangibles – Acquisition-related amortization expense is a non-cash expense arising primarily from the acquisition of intangible assets in connection with acquisitions or investments. We exclude acquisition-related amortization expense from non-GAAP financial measures because we believe (i) the amount of such expenses in any specific period may not directly correlate to the underlying performance of our business operations and (ii) such expenses can vary significantly between periods as a result of new acquisitions and full amortization of previously acquired intangible assets. Investors should note that the use of these intangible assets contributed to revenue in the periods presented and will contribute to future revenue generation, and the related amortization expense will recur in future periods. Stock-based compensation – Stock-based compensation expense is a non-cash expense arising from the grant of stock-based awards. We exclude stock-based compensation expense from non-GAAP financial measures because we believe (i) the amount of such expenses in any specific period may not directly correlate to the underlying performance of our business operations and (ii) such expenses can vary significantly between periods as a result of the timing and valuation of grants of new stock-based awards, including grants in connection with acquisitions. Investors should note that stock-based compensation is a key incentive offered to employees whose efforts contributed to the operating results in the periods presented and are expected to contribute to operating results in future periods, and such expense will recur in future periods. Severance and other nonrecurring charges – Non-recurring charges relate to certain severance and other charges incurred in connection with activities that are considered non-recurring. We exclude non-recurring expenses (primarily related to costs associated with our recent business transformation initiative and transaction-related costs) from non-GAAP financial measures because we believe (i) the amount of such expenses in any specific period may not directly correlate to the underlying performance of our business operations and (ii) such expenses can vary significantly between periods. Non-cash Interest expense – Non-cash interest expense includes amortization of deferred debt issuance costs. We exclude non-cash interest expense from non-GAAP financial measures because we believe these non-cash amounts relate to specific transactions and, as such, may not directly correlate to the underlying performance of our business operations. Interest expense and other income – Interest expense and other income represents (i) interest incurred on our term loan and revolving credit facility and (ii) non-cash interest expense. We exclude interest expense from non-GAAP financial measures because we believe these amounts relate to specific transactions and, as such, may not directly correlate to the underlying performance of our business operations. Gain on disposal of property and equipment – Gain on disposal of property and equipment represents the excess of proceeds received over the book value of assets disposed of during the period. We exclude gain on disposal of property and equipment from non-GAAP financial measures because we believe (i) the amount of such gain or loss in any specific period may not directly correlate to the underlying performance of our business operations and (ii) such gain or loss can vary significantly between periods. Gain on sale of AHT – Gain on sale of AHT represents the excess of proceeds received over the net assets sold from our sale of AHT, our previously wholly-owned post-acute business, in January 2024. We exclude gain on sale of AHT from non-GAAP financial measures because we believe the amount relates to a specific transaction and, as such, may not directly correlate to the underlying performance of our business operations. Tax shortfall (windfall) from stock-based compensation – ASU 2016-09, Improvements to Employee Share-Based Payment Accounting, became effective for the Company during the third quarter of 2017 and changes the treatment of tax shortfall and excess tax benefits arising from stock based compensation arrangements. Prior to ASU 2016-09, these amounts were recorded as an increase (for excess benefits) or decrease (for shortfalls) to additional paid-in capital. With the adoption of ASU 2016-09, these amounts are now captured in the period's income tax expense. We exclude this component of income tax expense from non-GAAP financial measures because we believe (i) the amount of such expenses or benefits in any specific period may not directly correlate to the underlying performance of our business operations; and (ii) such expenses or benefits can vary significantly between periods as a result of the valuation of grants of new stock-based awards, the timing of vesting of awards, and periodic movements in the fair value of our common stock. Management considers these non-GAAP financial measures to be important indicators of our operational strength and performance of our business and a good measure of our historical operating trends, in particular the extent to which ongoing operations impact our overall financial performance. In addition, management may use Adjusted EBITDA, Non-GAAP net income and/or Non-GAAP EPS to measure the achievement of performance objectives under the Company's stock and cash incentive programs. Note, however, that these non-GAAP financial measures are performance measures only, and they do not provide any measure of cash flow or liquidity. Non-GAAP financial measures are not alternatives for measures of financial performance prepared in accordance with GAAP and may be different from similarly titled non-GAAP measures presented by other companies, limiting their usefulness as comparative measures. Non-GAAP financial measures have limitations in that they do not reflect all of the amounts associated with our results of operations as determined in accordance with GAAP. Additionally, there is no certainty that we will not incur expenses in the future that are similar to those excluded in the calculations of the non-GAAP financial measures presented in this press release. Investors and potential investors are encouraged to review the 'Unaudited Reconciliation of Non-GAAP Financial Measures' above.
Business Wire
4 hours ago
- Business Wire
Rocket Pharmaceuticals Reports Second Quarter 2025 Financial Results and Highlights Recent Progress
CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the second quarter ending June 30, 2025. 'The second quarter of 2025 marked an important inflection point for Rocket as we refined our strategic focus around our AAV cardiovascular gene therapy platform and took multiple decisive steps to strengthen our financial foundation and thoughtfully adjust to market dynamics. We are fortifying our path to sustained value creation by leaning into programs with the highest value, conserving cash, and driving an efficient, agile organization,' said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. 'With regulatory alignment to resolve the clinical hold for RP-A501 in Danon disease in progress, momentum building for RP-A601 in PKP2-ACM as it advances toward a pivotal Phase 2 trial, and RP-A701 preparing to enter the clinic for the treatment of BAG3-DCM, Rocket is positioned as the leader in the development of gene therapies for inherited cardiomyopathies. While we are pausing additional investments in the FA and PKD programs, we are deeply grateful and committed to the patient communities and are exploring strategic alternatives to advance these programs externally. Finally, our recent reorganization ensures we are appropriately resourced to execute on our near-term milestones with nearly two years of capital.' Recent Pipeline and Operational Updates Investigation into the Serious Adverse Event (SAE) from the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing. Rocket previously disclosed an SAE, related to clinical complications from capillary leak syndrome and unfortunately leading to the patient's death. In response, Rocket paused dosing and initiated a root cause analysis, focusing on the recent addition of a C3 inhibitor to the pre-treatment regimen. On May 23, 2025, FDA placed the trial on clinical hold for further evaluation. Rocket is actively working with FDA, independent safety monitors, and clinical experts to ensure patient safety and resume the trial. While the clinical hold remains in place, the company cannot provide guidance on trial completion timing. Actively engaging with FDA on the advancement of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) in potential pivotal trial following initial positive Phase 1 data at the 28 th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). In July, Rocket received FDA Regeneration Medicine Advanced Therapy (RMAT) designation for RP-A601 for PKP2-ACM. Initial Phase 1 data demonstrating encouraging safety and preliminary efficacy outcomes of RP-A601 for PKP2-ACM were presented at ASGCT in May 2025. Rocket is engaging with FDA on potential pivotal trial design to evaluate the efficacy and safety of RP-A601. Phase 1 trial start-up activities are underway for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM). In June, an Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-DCM, received clearance from FDA. RP-A701 was recently granted FDA Fast Track designation, designed to facilitate the development and expedite the review of therapies for serious or life-threatening conditions that fill an unmet medical need. This enables increased communication with FDA, the potential for accelerated approval, and permits a rolling Biologics License Application (BLA) review. The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. BAG3-DCM is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality. Rocket estimates that the prevalence of BAG3-DCM in the U.S. is as many as 30,000 individuals. FDA review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADI TM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). Rocket previously disclosed that FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I. The Company continues to work with senior leaders and reviewers from FDA's Center for Biologics Evaluation and Research and submission of complete BLA to resolve Complete Response Letter is anticipated before the end of 2025. Ongoing strategic corporate restructuring and pipeline prioritization. As part of its broader strategic reorganization, Rocket implemented a workforce reduction of approximately 30%, across all functions, to align with a pipeline prioritization plan focused exclusively on its AAV cardiovascular gene therapy platform. This restructuring, together with other cost-saving measures, is expected to reduce operating expenses by nearly 25% over the next 12 months. The reorganization enables the company to focus on its late‑stage AAV gene therapy programs in Danon disease, PKP2‑ACM, and BAG3‑DCM, while advancing regulatory activities for KRESLADI™ in severe LAD‑I. As part of this realignment, Rocket is pausing additional investments in its Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs. The company continues to evaluate options to advance the FA program with health authorities in alignment with its refined strategic focus and broader corporate priorities. In July, Rocket appointed Chris Stevens as Chief Operating Officer. Chris Stevens is a highly seasoned executive with 25 years of experience across technical operations, product strategy, and general management, bringing immense commercial technical operations experience to Rocket. Stevens most recently served as the Executive Vice President and Chief Patient Supply Officer at Spark Therapeutics (subsidiary of Roche), where he successfully led teams across manufacturing, supply chain, quality, compliance, engineering, EHS, and facilities management, playing a key role in delivering gene therapies to patients. Second Quarter 2025 Financial Results Cash position. Cash, cash equivalents and investments as of June 30, 2025, were $271.5 million. Rocket expects such resources, excluding any potential proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADI TM, will be sufficient to fund its operations into the second quarter of 2027. R&D expenses. Research and development expenses were $42.7 million for the three months ended June 30, 2025, compared to $46.3 million for the three months ended June 30, 2024. The decrease of $3.7 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $2.3 million, professional fees of $2.0 million and building supplies and consumables of $0.8 million, offset by an increase in clinical trial expenses of $1.2 million. G&A expenses. General and administrative expenses were $25.0 million for the three months ended June 30, 2025, compared to $27.4 million for the three months ended June 30, 2024. The decrease in G&A expenses was primarily driven by decreases in commercial preparation related expenses of $1.4 million and compensation and benefits expense of $0.9 million. Net loss. Net loss was $68.9 million or $0.62 per share (basic and diluted) for the three months ended June 30, 2025, compared to $69.6 million or $0.74 (basic and diluted) for the three months ended June 30, 2024. Shares outstanding. 107,884,420 shares of common stock were outstanding as of June 30, 2025. Restructuring Expenses and Financial Guidance Restructuring expenses. Approximately $3.5 million in restructuring and restructuring-related charges were incurred in the first half of 2025. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as 'could,' 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's ability to realize the intended benefits of the restructuring plan and reduction in workforce, expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to fund its operations into the second quarter of 2027. There can be no assurance that the restructuring plan or the planned reduction in workforce will have the intended effect on the Company's operational results and strategic decisions, that any anticipated charges and any anticipated cost savings associated with the restructuring plan or the reduction in workforce will achieve their intended benefits. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
