Repotrectinib

Total revenues grew 9% y-o-y to $110.0 million for the second quarter of 2025; reaffirming full-year 2025 revenue guidance of $560 million to $590 million VYVGART reached record patient utilization in the second quarter; updated national guidelines elevate VYVGART’s role as a treatment for both acute and maintenance gMG Operating loss improved by 28% year-over-year to $54.9 million for the second quarter of 2025, and by 37% to $34.2 million on an adjusted basis1; on track to achieve profitability1 in the fourth quarter of 2025 ZL-1310 (DLL3 ADC) data presented at ASCO 2025 showed a 67% ORR across all doses (n=33) and 79% at 1.6mg/kg (n=14) in 2L ES-SCLC, with a differentiated safety profile; registrational study to be initiated in the second half of 2025 The success of the global Phase 3 FORTITUDE-101 study of bemarituzumab highlighted its potential as a first-line treatment for gastric cancer patients with FGFR2b overexpression; China regulatory submission expected in the second half of 2025 EAACI Congress 2025 presentation of ZL-1503 (IL-13/IL-31R) underscores its promising potential as a novel treatment option for moderate-to-severe atopic dermatitis Conference call and webcast today, August 7, 2025 , at 8:00 a.m. ET ( 8:00 p.m. HKT) SHANGHAI & CAMBRIDGE, Mass. --(BUSINESS WIRE)--Aug. 7, 2025-- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced financial results for the second quarter of 2025, along with recent product highlights and corporate updates. “Zai Lab is entering a pivotal period – defined by innovation, scale and strong execution,” said Dr. Samantha Du , Founder, Chairperson, and CEO of Zai Lab . “We are making meaningful progress throughout our business – expanding patient impact, accelerating global innovation, and operating with financial discipline. Updated ASCO data for ZL-1310 (DLL3 ADC) reaffirm its best-in-class potential in second-line SCLC, and we are moving swiftly into pivotal development while exploring opportunities in first-line SCLC and other neuroendocrine carcinomas. We were also encouraged by positive data for bemarituzumab in first-line gastric cancer and by emerging preclinical results for our IL-13/IL-31 bispecific antibody in atopic dermatitis, reinforcing both our near-term commercial opportunities and the potential of our global pipeline, respectively. With multiple launches ahead, a robust pipeline, and profitability1 within reach, Zai Lab is executing on its vision to become a leading global biopharma company.” “VYVGART continues to lead our commercial momentum, with record patient utilization driven by longer treatment durations and growing adoption in the maintenance setting,” said Josh Smiley , President and COO of Zai Lab . “The July update to national MG guidelines further strengthens VYVGART’s role in both acute and chronic care, and we expect momentum to accelerate in the second half. We are also preparing for several high-impact launches – including KarXT and bemarituzumab – that, together with pipeline-in-a-product assets like VYVGART and povetacicept, will fuel our next wave of growth. With a 28% year-over-year reduction in operating loss and a 37% improvement on an adjusted basis, we are on track to achieve profitability1 in the fourth quarter. Backed by a strong cash position2, a growing commercial business, and an advancing global pipeline, Zai Lab is well equipped to deliver long-term shareholder value.” 1 Refers to adjusted income (loss) from operations (non-GAAP), calculated as GAAP income (loss) from operations adjusted to exclude certain non-cash expenses, including depreciation, amortization, and share-based compensation. For additional information on this adjusted profitability measure, refer to the “Non-GAAP Measures” section. 2 Cash position includes cash and cash equivalents, current restricted cash, and short-term investments. Second Quarter 2025 Financial Results Product revenue, net was $109.1 million in the second quarter of 2025, compared to $100.1 million for the same period in 2024, representing 9% y-o-y growth, 10% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for VYVGART, XACDURO, and NUZYRA, partially offset by softer sales for ZEJULA: VYVGART and VYVGART Hytrulo were $26.5 million in the second quarter of 2025, compared to $18.1 million in the first quarter of 2025. Sales grew 46% quarter over quarter driven by an extension of duration of therapy and increasing market penetration. ZEJULA was $41.0 million in the second quarter of 2025, compared to $45.0 million for the same period in 2024. Sales were softer due to evolving competitive dynamics within the PARPi class. XACDURO, which was launched since the fourth quarter of 2024, was $4.6 million in the second quarter of 2025. NUZYRA was $14.3 million in the second quarter of 2025, compared to $12.3 million for the same period in 2024. This growth was supported by increasing market coverage and penetration. Research and Development (R&D) expenses were $50.6 million in the second quarter of 2025, compared to $61.6 million for the same period in 2024. The decrease reflects reduced personnel and clinical trial costs, driven by ongoing resource prioritization and efficiency efforts. Selling, General and Administrative expenses were $71.0 million in the second quarter of 2025, compared to $79.7 million for the same period in 2024. The decrease was primarily driven by reduced personnel costs because of resource prioritization and efficiency efforts. Loss from operations was $54.9 million in the second quarter of 2025, $34.2 million when adjusted to exclude certain non-cash expenses including depreciation, amortization, and share-based compensation. A reconciliation of loss from operations (GAAP) to adjusted loss from operations (non-GAAP) is included at the end of this release. Net loss was $40.7 million in the second quarter of 2025, or a loss per ordinary share attributable to common stockholders of $0.04 (or loss per American Deposit Share (ADS) of $0.37 ), compared to a net loss of $80.3 million for the same period in 2024, or a loss per ordinary share of $0.08 (or loss per ADS of $0.82 ). These decreases in net loss were primarily due to product revenue growing faster than net operating expenses. Cash and cash equivalents, short-term investments, and current restricted cash totaled $832.3 million as of June 30, 2025 , compared to $857.3 million as of March 31, 2025 . VYVGART and VYVGART Hytrulo were $26.5 million in the second quarter of 2025, compared to $18.1 million in the first quarter of 2025. Sales grew 46% quarter over quarter driven by an extension of duration of therapy and increasing market penetration. ZEJULA was $41.0 million in the second quarter of 2025, compared to $45.0 million for the same period in 2024. Sales were softer due to evolving competitive dynamics within the PARPi class. XACDURO, which was launched since the fourth quarter of 2024, was $4.6 million in the second quarter of 2025. NUZYRA was $14.3 million in the second quarter of 2025, compared to $12.3 million for the same period in 2024. This growth was supported by increasing market coverage and penetration. Recent Pipeline Highlights Below are key product updates since our last earnings release: Oncology Pipeline ZL-1310 (DLL3 ADC): In June 2025 , Zai Lab presented positive data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. In second-line (2L) SCLC, objective response rate (ORR) was 67% across all dose levels (n=33) and 79% at 1.6 mg/kg dose (n=14). ZL-1310 demonstrated a well-tolerated safety profile at target doses of less than 2.0 mg/kg, with Grade ≥3 treatment-related adverse events (TRAEs) of 6%, no Grade ≥2 interstitial lung disease, and no drug discontinuations. In May 2025 , the U.S. Food and Drug Administration (FDA) granted Fast Track designation to ZL-1310 for treatment of ES-SCLC. Bemarituzumab (FGFR2b): In June 2025 , Zai Lab announced the Phase 3 FORTITUDE-101 clinical trial evaluating first-line bemarituzumab plus chemotherapy (mFOLFOX6) met its primary endpoint of overall survival (OS) at a pre-specified interim analysis.​ At the interim analysis, bemarituzumab plus chemotherapy significantly improved OS in patients with FGFR2b overexpression compared to chemotherapy alone. The most common treatment-emergent adverse events (>25%) in patients treated with bemarituzumab plus chemotherapy were reduced visual acuity, punctate keratitis, anemia, neutropenia, nausea, corneal epithelium defect and dry eye. While ocular events were consistent with the Phase 2 experience and observed in both arms, they occurred with greater frequency and severity in the Phase 3 bemarituzumab arm. Detailed results from FORTITUDE 101 will be shared at a future medical meeting. Zai Lab plans to move rapidly toward regulatory submission in China in the second half of 2025. Tumor Treating Fields (TTFields): In May 2025 , Zai Lab partner Novocure presented results from the Phase 3 PANOVA-3 trial for pancreatic cancer as a late-breaking oral presentation at the 2025 ASCO Annual Meeting. The data demonstrated that TTFields therapy, when added to standard of care, achieved an OS benefit supported by significantly improved quality of life and extended pain-free survival, a key outcome for patients with pancreatic cancer. Zai Lab participated in the study in Greater China (mainland China , Hong Kong , Macau and Taiwan , collectively) and plans to file for regulatory approval in China in the second half of 2025.​ In June 2025 , Zai Lab presented positive data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. In second-line (2L) SCLC, objective response rate (ORR) was 67% across all dose levels (n=33) and 79% at 1.6 mg/kg dose (n=14). ZL-1310 demonstrated a well-tolerated safety profile at target doses of less than 2.0 mg/kg, with Grade ≥3 treatment-related adverse events (TRAEs) of 6%, no Grade ≥2 interstitial lung disease, and no drug discontinuations. In May 2025 , the U.S. Food and Drug Administration (FDA) granted Fast Track designation to ZL-1310 for treatment of ES-SCLC. Immunology Pipeline Efgartigimod (FcRn): In July 2025 , the China Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (MG) (2025) was published, emphasizing the importance of Minimal Symptom Expression (MSE), as the primary treatment goal in MG. The guidelines have highlighted VYVGART’s ability to achieve MSE rapidly and provide durable benefit. VYVGART is now recommended for early use in mild-to-moderate and highly active patients, and for sustained long-term treatment to maximize potential benefit. ZL-1503 (IL-13/IL-31R): In June 2025 , Zai Lab announced new preclinical data highlighting the potential of ZL-1503 as a promising treatment for moderate-to-severe atopic dermatitis and other IL-13 and IL-31-driven diseases. Its favorable preclinical safety profile, prolonged half-life and durable suppression of both inflammatory and pruritogenic pathways support the continued advancement of ZL-1503 toward clinical development. Anticipated Major Milestones in 2025 and the First Half of 2026 Upcoming Potential NMPA Submissions Bemarituzumab (FGFR2b) in first-line gastric cancer in the second half of 2025 Tumor Treating Fields (TTFields) in first-line pancreatic cancer in the second half of 2025 Efgartigimod (FcRn) for prefilled syringe in generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) in the second half of 2025 Upcoming Potential NMPA Approvals Xanomeline-Trospium (or KarXT) (M1/M4-agonist) in schizophrenia Tisotumab Vedotin (Tissue Factor ADC) in recurrent or metastatic cervical cancer following progression on or after chemotherapy Repotrectinib (ROS1/TRK) in NTRK+ solid tumors Expected Clinical Developments and Data Readouts Global Pipeline ZL-1310 or Zocilurtatug pelitecan (DLL3 ADC) Second-Line ES-SCLC: Zai Lab to provide data update and to initiate a global registrational study of ZL-1310 monotherapy in the second half of 2025. First-Line ES-SCLC: Zai Lab to provide data readout for dose escalation of ZL-1310 doublet in combination with atezolizumab. Other neuroendocrine carcinomas: Zai Lab to provide data readout from the global Phase 1/2 study in patients with selected solid tumors. ZL-1503 (IL-13/IL-31R) Zai Lab to advance into a global Phase 1 study in moderate-to-severe atopic dermatitis in the second half of 2025. ZL-6201 (LRRC15 ADC) Zai Lab to advance into global Phase 1 development for patients with sarcoma and potentially other LRRC15-positive solid tumors, such as breast cancer and other malignancies. Regional Pipeline Bemarituzumab (FGFR2b) Zai Lab partner Amgen to provide detailed results from the Phase 3 FORTITUDE-101 study of bemarituzumab combined with chemotherapy versus chemotherapy alone in first-line gastric cancer at an upcoming medical meeting. Zai Lab participated in the study in Greater China . Zai Lab partner Amgen to provide potential data readout from the Phase 1b/3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab versus chemotherapy and nivolumab in first-line gastric cancer. Zai Lab participated in the study in Greater China . Xanomeline-Trospium (or KarXT) (M1/M4-agonist) Zai Lab partner Bristol Myers Squibb to provide data readout from the Phase 3 ADEPT-2 study of KarXT in Alzheimer’s Disease Psychosis in the second half of 2025. Zai Lab participated in the study in Greater China . Efgartigimod (FcRn) Lupus Nephritis (LN): Zai Lab and partner argenx to provide topline results from the Phase 2 study in LN in the fourth quarter of 2025. Seronegative gMG: Zai Lab partner argenx to provide topline results from the global Phase 3 ADAPT-SERON study in seronegative gMG in the second half of 2025. Zai Lab participated in the study in Greater China . Ocular myasthenia gravis: Zai Lab partner argenx to provide topline results from the global Phase 3 ADAPT-OCULUS study in the first half of 2026. Zai Lab participated in the study in Greater China . Sjogren's disease: Zai Lab to join the registrational UNITY study of efgartigimod subcutaneous given by prefilled syringe in Sjogren’s disease in Greater China in the third quarter of 2025. Povetacicept (APRIL/BAFF) Primary Membranous Nephropathy (pMN): Zai Lab plans to partner with Vertex to execute the global pivotal Phase 2/3 study of povetacicept in pMN in Greater China . The study is expected to start in the second half of 2025. IgA Nephropathy (IgAN): Zai Lab partner Vertex will conduct an interim analysis of the global Phase 3 RAINIER study following 36 weeks of treatment with the potential to file for U.S. accelerated approval in the first half of 2026. VRDN-003 (IGF-1R, subcutaneous) Zai Lab to initiate a registrational study in thyroid eye disease in Greater China in the second half of 2025. Zai Lab partner Viridian to provide topline results from the global registrational REVEAL-1 and REVEAL-2 studies in the first half of 2026. Conference Call and Webcast Information Zai Lab will host a live conference call and webcast today, August 7, 2025 , at 8:00 a.m. ET ( 8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company’s website at http://ir.zailaboratory.com. Participants must register in advance of the conference call. Details of registration links are as follows: For webcast: https://edge.media-server.com/mmc/p/5xsbgbn3 For dial-in: https://register-conf.media-server.com/register/BI52be4f1f4178480fa60b0d17b5c6ae3d All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering. A replay will be available shortly after the call and can be accessed by visiting the Company’s website. About Zai Lab Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) is an innovative, research-based, commercial-stage biopharmaceutical company based in China and the United States . We are focused on discovering, developing, and commercializing innovative products that address medical conditions with significant unmet needs in the areas of oncology, immunology, neuroscience, and infectious disease. Our goal is to leverage our competencies and resources to positively impact human health worldwide. For additional information about Zai Lab , please visit www.zailaboratory.com or follow us at https://x.com/ZaiLab_Global. Non-GAAP Measures In addition to results presented in accordance with GAAP, we disclose growth rates that have been adjusted to exclude the impact of changes due to the translation of foreign currencies into U.S. dollars. We have also presented a measure of adjusted loss from operations that adjusts GAAP loss from operations to exclude the impact of certain non-cash expenses including depreciation, amortization, and share-based compensation, which we refer to as “profitability.” These adjusted growth rates and adjusted loss from operations are non-GAAP financial measures. We believe that these non-GAAP financial measures are important for an understanding of the performance of our business operations and financial results and provide investors with an additional perspective on operational trends and greater transparency into our historical and projected operating performance. Although we believe the non-GAAP financial measures enhance investors’ understanding of our business and performance, these non-GAAP financial measures should not be considered an exclusive alternative to the corresponding GAAP financial measures. Zai Lab Forward-Looking Statements This press release contains certain forward-looking statements, including statements relating to our strategy and plans; potential of and expectations for our business, commercial products, and pipeline programs; our goals, objectives, and priorities and our expectations under our growth strategy (including our expectations regarding our commercial products and launches, clinical stage products, revenue growth, profitability, and cash flow); clinical development programs and related clinical trials; clinical trial data, data readouts, and presentations; risks and uncertainties associated with drug development and commercialization; regulatory discussions, submissions, filings, and approvals and the timing thereof; the potential benefits, safety, and efficacy of our products and product candidates and those of our collaboration partners; the anticipated benefits and potential of investments, collaborations, and business development activities; our profitability and timeline to profitability; our future financial and operating results; and financial guidance, including with respect to our capital allocation and investment strategy and our expected path to profitability. All statements, other than statements of historical fact, included in this press release are forward-looking statements, and can be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “poised,” “positioned,” “possible,” “potential,” “will,” “would,” and other similar expressions. Such statements constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees or assurances of future performance. Forward-looking statements are based on our expectations and assumptions as of the date of this press release and are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements. We may not actually achieve the plans, carry out the intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including but not limited to (1) our ability to successfully commercialize and generate revenue from our approved products; (2) our ability to obtain funding for our operations and business initiatives; (3) the results of our clinical and pre-clinical development of our product candidates; (4) the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approvals of our product candidates; (5) risks related to doing business in China ; and (6) other factors identified in our most recent annual and quarterly reports and in other reports we have filed with the U.S. Securities and Exchange Commission (SEC). We anticipate that subsequent events and developments will cause our expectations and assumptions to change, and we undertake no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as may be required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. Our SEC filings can be found on our website at www.zailaboratory.com and on the SEC’s website at www.SEC.gov. Zai Lab Limited Unaudited Condensed Consolidated Balance Sheets (in thousands of U.S. dollars ($), except for number of shares and per share data) June 30 , 2025 December 31 , 2024 Assets Current assets Cash and cash equivalents 732,159 449,667 Restricted cash, current 100,111 100,000 Short-term investments — 330,000 Accounts receivable (net of allowance for credit losses of $26 and $25 as of June 30, 2025 and December 31, 2024 , respectively) 88,499 85,178 Notes receivable 10,843 4,233 Inventories, net 61,700 39,875 Prepayments and other current assets 40,750 41,527 Total current assets 1,034,062 1,050,480 Restricted cash, non-current 1,114 1,114 Property and equipment, net 50,160 47,961 Operating lease right-of-use assets 16,787 21,496 Land use rights, net 2,860 2,907 Intangible assets, net 56,519 56,027 Other non-current assets 2,599 5,768 Total assets 1,164,101 1,185,753 Liabilities and shareholders’ equity Current liabilities Accounts payable 107,357 100,906 Current operating lease liabilities 5,584 8,048 Short-term debt 174,509 131,711 Other current liabilities 44,051 58,720 Total current liabilities 331,501 299,385 Deferred income 29,233 31,433 Non-current operating lease liabilities 11,307 13,712 Other non-current liabilities 325 325 Total liabilities 372,366 344,855 Commitments and contingencies Shareholders’ equity Ordinary shares (par value of $0.000006 per share; 5,000,000,000 shares authorized; 1,104,032,910 and 1,082,614,740 shares issued as of June 30, 2025 and December 31, 2024 , respectively; 1,099,112,890 and 1,077,702,540 shares outstanding as of June 30, 2025 and December 31, 2024 , respectively) 7 7 Additional paid-in capital 3,308,491 3,264,295 Accumulated deficit (2,542,248 ) (2,453,083 ) Accumulated other comprehensive income 46,348 50,515 Treasury Stock (at cost, 4,920,020 and 4,912,200 shares as of June 30, 2025 and December 31, 2024 , respectively) (20,863 ) (20,836 ) Total shareholders’ equity 791,735 840,898 Total liabilities and shareholders’ equity 1,164,101 1,185,753 Zai Lab Limited Unaudited Condensed Consolidated Statements of Operations (in thousands of $, except for number of shares and per share data) Three Months Ended June 30 , Six Months Ended June 30 , 2025 2024 2025 2024 Revenues Product revenue, net 109,085 100,106 214,735 187,255 Collaboration revenue 892 398 1,729 398 Total revenues 109,977 100,504 216,464 187,653 Expenses Cost of product revenue (43,003 ) (35,148 ) (81,455 ) (68,767 ) Cost of collaboration revenue (217 ) (85 ) (412 ) (85 ) Research and development (50,614 ) (61,625 ) (111,343 ) (116,270 ) Selling, general, and administrative (71,038 ) (79,710 ) (134,460 ) (148,904 ) Loss from operations (54,895 ) (76,064 ) (111,206 ) (146,373 ) Interest income 8,843 9,330 17,449 18,988 Interest expenses (1,262 ) (492 ) (2,449 ) (605 ) Foreign currency gains (losses) 2,837 (4,108 ) 3,488 (6,176 ) Other income (expense), net 3,750 (8,943 ) 3,553 418 Loss before income tax (40,727 ) (80,277 ) (89,165 ) (133,748 ) Income tax expense — — — — Net loss (40,727 ) (80,277 ) (89,165 ) (133,748 ) Loss per share - basic and diluted (0.04 ) (0.08 ) (0.08 ) (0.14 ) Weighted-average shares used in calculating net loss per ordinary share - basic and diluted 1,091,933,150 975,937,790 1,086,413,130 974,541,780 Zai Lab Limited Unaudited Condensed Consolidated Statements of Comprehensive Loss (in thousands of $) Three Months Ended June 30 , Six Months Ended June 30 , 2025 2024 2025 2024 Net loss (40,727 ) (80,277 ) (89,165 ) (133,748 ) Other comprehensive (loss) income, net of tax of nil: Foreign currency translation adjustments (2,955 ) 3,605 (4,167 ) 5,147 Comprehensive loss (43,682 ) (76,672 ) (93,332 ) (128,601 ) Zai Lab Limited Non-GAAP Measures (unaudited) ($ in thousands) Growth on a Constant Exchange Rate (CER) Basis Three Months Ended June 30 , Year over Year % Growth Six Months Ended June 30 , Year over Year % Growth 2025 2024 As reported At CER* 2025 2024 As reported At CER* Product revenue, net 109,085 100,106 9 % 10 % 214,735 187,255 15 % 16 % Loss from operations (54,895 ) (76,064 ) (28 )% (28 )% (111,206 ) (146,373 ) (24 )% (24 )% * The growth rates at CER were calculated assuming the same foreign currency exchange rates were in effect for the current and prior year periods. Reconciliation of Loss from Operations (GAAP) to Adjusted Loss from Operations (Non-GAAP) Three Months Ended June 30 , Six Months Ended June 30 , 2025 2024 2025 2024 GAAP loss from operations (54,895 ) (76,064 ) (111,206 ) (146,373 ) Plus: Depreciation and amortization expenses 3,735 2,941 7,193 5,953 Plus: Share-based compensation 16,973 18,638 32,773 36,618 Adjusted loss from operations (34,187 ) (54,485 ) (71,240 ) (103,802 ) View source version on businesswire.com: https://www.businesswire.com/news/home/20250807049974/en/ For more information, please contact: Investor Relations: Christine Chiou / Lina Zhang +1 (917) 886-6929 / +86 136 8257 6943 christine.chiou1@zailaboratory.com / lina.zhang@zailaboratory.com Media: Shaun Maccoun / Xiaoyu Chen +1 (415) 317-7255 / +86 185 0015 5011 shaun.maccoun@zailaboratory.com / xiaoyu.chen@zailaboratory.com Source: Zai Lab Limited

LOUISVILLE, Ky., July 07, 2025 (GLOBE NEWSWIRE) -- Onco360®, the nation’s leading independent specialty pharmacy, has added Augtyro™ (repotrectinib) manufactured by Bristol Myers Squibb to its comprehensive list of limited distribution therapies. Augtyro was initially approved on November 15, 2023, for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). In June 2024, Augtyro obtained an indication for the treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity and have progressed following treatment or have no satisfactory alternative therapy. The approval was based on results from the TRIDENT-1 clinical trial (NCT03093116), which evaluated Augtyro in adult patients with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive solid tumors.1 “We remain committed to support the specialized needs of patients battling cancer, as well as those facing rare and complex diseases. We are grateful for the opportunity to work alongside the team at Bristol Myers Squibb in support of patients prescribed Augtyro.” said Benito Fernandez, Chief Commercial Officer. Please see the full Prescribing Information for Augtyro. IMPORTANT SAFETY INFORMATION FOR AUGTYRO Warnings and Precautions Central Nervous System Adverse Reactions Among the 426 patients who received AUGTYRO in Study TRIDENT-1, a broad spectrum of central nervous system (CNS) adverse reactions including dizziness, ataxia, and cognitive disorders occurred in 77% of patients with Grade 3 or 4 events occurring in 4.5%.Dizziness, including vertigo, occurred in 65%; Grade 3 dizziness occurred in 2.8% of patients.Ataxia, including gait disturbance and balance disorder, occurred in 28% of patients; Grade 3 ataxia occurred in 0.5%.Cognitive impairment, including memory impairment and disturbance in attention, occurred in 25% of patients. Cognitive impairment included memory impairment (15%), disturbance in attention (12%), and confusional state (2%); Grade 3 cognitive impairment occurred in 0.9% of patients.Mood disorders occurred in 6% of patients. Mood disorders occurring in >1% of patients included anxiety (2.6%); Grade 4 mood disorders (mania) occurred in 0.2% of patients.Sleep disorders including insomnia and hypersomnia occurred in 18% of patients. Sleep disorders observed in >1% of patients were somnolence (9%), insomnia (6%) and hypersomnia (1.6%).The incidences of CNS adverse reactions reported were similar in patients with and without CNS metastases.Advise patients not to drive or use machines if they are experiencing CNS adverse reactions. Withhold and then resume at same or reduced dose upon improvement, or permanently discontinue AUGTYRO based on severity. Interstitial Lung Disease (ILD)/Pneumonitis ILD/pneumonitis (pneumonitis [2.8%] and ILD [0.2%]) occurred in 3.1%; Grade 3 ILD/pneumonitis occurred in 1.2%.Monitor patients for new or worsening pulmonary symptoms indicative of ILD/pneumonitis. Immediately withhold AUGTYRO in patients with suspected ILD/pneumonitis and permanently discontinue AUGTYRO if ILD/pneumonitis is confirmed. Hepatotoxicity Increased alanine transaminase (ALT) occurred in 38%, increased aspartate aminotransferase (AST) occurred in 41%, including Grade 3 or 4 increased ALT in 3.3% and increased AST in 2.9%.Monitor liver function tests, including ALT, AST and bilirubin, every 2 weeks during the first month of treatment, then monthly thereafter and then as clinically indicated. Withhold and then resume at same or reduced dose upon improvement or permanently discontinue AUGTYRO based on the severity. Myalgia with Creatine Phosphokinase (CPK) Elevation AUGTYRO can cause myalgia with or without creatine phosphokinase (CPK) elevation. Myalgia occurred in 13% of patients, with Grade 3 in 0.7%. Concurrent increased CPK within a 7-day window was observed in 3.7% of patients.Advise patients to report any unexplained muscle pain, tenderness, or weakness. Monitor serum CPK levels during AUGTYRO treatment and monitor CPK levels every 2 weeks during the first month of treatment and as needed in patients reporting unexplained muscle pain, tenderness, or weakness. Initiate supportive care as clinically indicated. Based on severity, withhold and then resume AUGTYRO at same or reduced dose upon improvement. Hyperuricemia 21 patients (5%) experienced hyperuricemia reported as an adverse reaction, 0.7% experienced Grade 3 or 4 hyperuricemia. One patient without pre-existing gout required urate-lowering medication.Monitor serum uric acid levels prior to initiating AUGTYRO and periodically during treatment. Initiate treatment with urate-lowering medications as clinically indicated. Withhold and then resume at same or reduced dose upon improvement, or permanently discontinue AUGTYRO based on severity. Skeletal Fractures Fractures occurred in 2.3% of patients and involved the ribs (0.5%), feet (0.5%), spine (0.2%), acetabulum (0.2%), sternum (0.2%), and ankles (0.2%). Some fractures occurred at sites of disease and prior radiation therapy.Of 26 evaluable patients in an ongoing open-label study in pediatric patients, fractures occurred in one 12- year-old patient (ankle/foot) and one 10-year-old patient (stress fracture). AUGTYRO is not approved for use in pediatric patients less than 12 years of age.Promptly evaluate patients with signs or symptoms (e.g., pain, changes in mobility, deformity) of fractures. There are no data on the effects of AUGTYRO on healing of known fractures and risk of future fractures. Embryo-Fetal Toxicity Based on literature reports in humans with congenital mutations leading to changes in tropomyosin receptor tyrosine kinase (TRK) signaling, findings from animal studies, and its mechanism of action, AUGTYRO can cause fetal harm when administered to a pregnant woman.Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with AUGTYRO and for 2 months following the last dose, since AUGTYRO can render some hormonal contraceptives ineffective.Advise male patients with female partners of reproductive potential to use effective contraception during treatment with AUGTYRO and for 4 months after the last dose. Adverse Reactions The safety of AUGTYRO was evaluated in 426 patients in TRIDENT-1. The most common adverse reactions (≥20%) were dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea. Drug Interactions Effects of Other Drugs on AUGTYRO Avoid concomitant use with P-gp inhibitors, strong or moderate CYP3A inducers, and strong or moderate CYP3A inhibitors. Discontinue CYP3A inhibitors for 3 to 5 elimination half-lives of the CYP3A inhibitor prior to initiating AUGTYRO. Effects of AUGTYRO on other DrugsCertain CYP3A4 Substrates Avoid concomitant use unless otherwise recommended in the Prescribing Information for CYP3A substrates, where minimal concentration changes can cause reduced efficacy. If concomitant use is unavoidable, increase the CYP3A4 substrate dosage in accordance with approved product labeling.Repotrectinib is a CYP3A4 inducer. Concomitant use of repotrectinib decreases the concentration of CYP3A4 substrates, which can reduce the efficacy of these substrates. Contraceptives Repotrectinib can decrease progestin or estrogen exposure to an extent that could reduce the effectiveness of hormonal contraceptives.Avoid concomitant use of AUGTYRO with hormonal contraceptives. Advise females of childbearing potential to use an effective nonhormonal contraceptive. About Onco360 Oncology Pharmacy:Onco360 is the nation’s largest independent Oncology Pharmacy and clinical support services company. Onco360 was founded in 2003 to bring together the stakeholders involved in the cancer treatment process and serve the specialized needs of oncologists, patients, hospitals, cancer centers of excellence, manufacturers, health plans, and payers. It dispenses nationally through its network of URAC-, and ACHC-accredited Oncology Pharmacies. Onco360 is headquartered in Louisville, Kentucky, and is a flagship specialty pharmacy brand of PharMerica Corporation, a leading institutional pharmacy, specialty infusion, and hospital services company servicing healthcare facilities in the United States. For more information about Onco360, please visit Onco360.com. Media Contact: Benito Fernandez, Chief Commercial OfficerBenito.Fernandez@Onco360.com516-640-1332 References: 1Augtyro (Repotrectinib) [Package Insert]. Princeton, NJ. Bristol Myers Squibb Company. 2024. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. 2Repotrectinib Expands Treatment Options for Lung Cancers with ROS1 Fusions. National Cancer Institute. Repotrectinib Shrinks ROS1-Positive NSCLC Tumors - NCI. Access June 19, 2025.