Zai Lab and Novocure Announce Results From the Phase 3 PANOVA-3 Trial of Tumor Treating Fields (TTFields) Therapy for Pancreatic Cancer to be Presented at 2025 ASCO Annual Meeting
'The data presented today from the PANOVA-3 trial of Tumor Treating Fields show a clinically meaningful and statistically significant improvement in overall survival for people with locally advanced pancreatic cancer,' said Vincent Picozzi, MD, MMM, medical oncologist and investigator in the PANOVA-3 trial. 'Importantly, we also saw an extension in the duration of time before pain progressed. Pain is a hallmark of this disease, and as a clinician, the potential of this therapy to address this aspect of pancreatic cancer is very encouraging. These results illustrate the potential of Tumor Treating Fields therapy concomitant with gemcitabine and nab-paclitaxel to become a standard of care for unresectable, locally advanced pancreatic cancer.'
The Phase 3 PANOVA-3 trial evaluated the use of TTFields therapy concomitantly with gemcitabine and nab-paclitaxel as a first-line treatment for unresectable, locally advanced pancreatic adenocarcinoma compared to gemcitabine and nab-paclitaxel alone. The trial met its primary endpoint, demonstrating a statistically significant improvement in median overall survival (mOS) for patients treated with TTFields.
'The encouraging data from the Phase 3 PANOVA-3 study demonstrate a meaningful improvement in outcomes for patients with unresectable, locally advanced pancreatic cancer—including pain reduction and a statistically significant improvement in overall survival,' said Rafael Amado, M.D., President, Head of Global Research and Development at Zai Lab. 'Pancreatic cancer remains one of the most challenging cancers to treat globally, with approximately 134,000 new cases diagnosed annually in China alone. Zai Lab participated in this trial and looks forward to continuing our collaboration with Novocure to bring this innovative therapy to patients in China as quickly as possible.'
'Most people with pancreatic cancer are diagnosed with advanced disease, which is very difficult to treat and only about 1 in 10 people are alive five years after diagnosis,' said Nicolas Leupin, MD, PhD, Chief Medical Officer, Novocure. 'The results shared today at ASCO and in the Journal of Clinical Oncology demonstrate that Tumor Treating Fields therapy improved overall survival and pain-free survival in unresectable, locally advanced pancreatic cancer. We plan to submit these data to the FDA in the second half of 2025 to support a premarket approval for Tumor Treating Fields therapy.'
Results from PANOVA-3
In the intent-to-treat population, patients treated with TTFields therapy concomitantly with gemcitabine and nab-paclitaxel had an mOS of 16.2 months compared to 14.2 months for patients treated with gemcitabine and nab-paclitaxel alone, a statistically significant 2.0-month improvement [hazard ratio (HR) 0.82; p=0.039 (N=571)].
TTFields therapy concomitant with gemcitabine and nab-paclitaxel demonstrated improvement in several secondary endpoints including the one-year survival rate and pain-free survival. Pancreatic cancer can cause significant pain as the disease progresses and managing pain is a key clinical challenge.
The one-year survival rate showed a statistically significant improvement in the TTFields concomitant with gemcitabine and nab-paclitaxel treated group with 68.1% [95% CI: 62.0–73.5] compared to those who received gemcitabine and nab-paclitaxel alone, 60.2% [95% CI: 54.2–65.7], p=0.029.
Patients treated with TTFields concomitant with gemcitabine and nab-paclitaxel had a median pain-free survival of 15.2 months [95% CI: 10.3–22.8] compared to a median 9.1 months in the group treated with gemcitabine and nab-paclitaxel alone [95% CI: 7.4–12.7]; HR 0.74 [95% CI: 0.56–0.97], p=0.027. This is a statistically significant 6.1-month extension in pain-free survival. Pain-free survival was defined as the time from baseline until an increase of 20 or more points was reported by patients on a visual scale for pain or until death.
Quality of life was also measured as a secondary endpoint. Analyses were performed for all patients using the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) with the pancreatic cancer specific PAN26 addendum. Deterioration-free survival in global health status, pain and digestive problems were significantly improved in patients receiving TTFields therapy concomitant with gemcitabine and nab-paclitaxel compared to the gemcitabine and nab-paclitaxel alone group. Full analysis of the quality of life results in PANOVA-3 will be shared at a future scientific conference.
There was no statistically significant difference in additional secondary outcome measures of progression-free survival, local progression-free survival, objective response rate, puncture-free survival or tumor resectability rate between the TTFields with gemcitabine and nab-paclitaxel and the gemcitabine and nab-paclitaxel arms.
TTFields therapy was well-tolerated, no new safety signals were observed, and safety was consistent with prior clinical studies. Mild to moderate skin adverse events (AEs) were the most common device-related AEs.
Data Presentation & Publication Details
The PANOVA-3 data, (LBA 3500) Phase 3 study of Tumor Treating Fields (TTFields) with gemcitabine and nab-paclitaxel for locally advanced pancreatic ductal adenocarcinoma (LA-PAC), will be presented today by Dr. Picozzi in Hall D1 during the 3:00 – 6:00 p.m. Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary oral session.
The Phase 3 PANOVA-3 publication in the Journal of Clinical Oncology, Tumor Treating Fields with gemcitabine and nab-paclitaxel for locally advanced pancreatic adenocarcinoma: randomized, open-label, pivotal phase 3 PANOVA-3 study, will be available online at https://ascopubs.org/doi/10.1200/JCO-25-00746.
Novocure Investor Event
Novocure will host an investor event featuring Dr. Picozzi and Novocure leadership after the oral presentation. Event details and a link to a live webcast of the event are available on the investor relations page of www.novocure.com. For more information or to request in-person attendance, please contact Novocure investor relations at investorinfo@novocure.com.
Regulatory & Ongoing Clinical Study of TTFields for Pancreatic Cancer
Novocure plans to file for regulatory approval for use of TTFields therapy in unresectable, locally advanced pancreatic adenocarcinoma based on PANOVA-3 in the U.S. in the second half of 2025. The company also plans to file for regulatory approval in EU, Japan and other key markets.
Novocure continues to follow patients in its Phase 2 PANOVA-4 trial exploring the use of TTFields therapy together with atezolizumab, gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. PANOVA-4 has completed enrollment with data anticipated in the first half of 2026.
About PANOVA-3
PANOVA-3 is an international prospective, randomized, open-label, controlled Phase 3 clinical trial designed to test the efficacy and safety of Tumor Treating Fields (TTFields) therapy used concomitantly with gemcitabine and nab-paclitaxel, as a first-line treatment for locally advanced pancreatic adenocarcinoma. Patients were randomized to receive either TTFields therapy concomitant with gemcitabine and nab-paclitaxel or gemcitabine and nab-paclitaxel alone.
The primary endpoint is overall survival. Secondary endpoints include progression-free survival, local progression-free survival, objective response rate, one-year survival rate, quality of life, pain-free survival, puncture-free survival, resectability rate, and toxicity.
The PANOVA-3 trial enrolled 571 patients who were randomized 1:1 and followed for a minimum of 18 months.
About PANOVA-4
PANOVA-4 is an international, multi-center, Phase 2 clinical trial designed to test the safety and efficacy of Tumor Treating Fields (TTFields) therapy together with atezolizumab, gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. The primary endpoint is disease control rate. Secondary endpoints include overall survival, progression-free survival, one-year survival rate, objective response rate, progression-free survival at six months, duration of response, and toxicity. The study is designed to enroll 76 patients and enrollment is complete.
About Pancreatic Cancer in China
Pancreatic cancer is one of the most common and deadliest cancers globally. In China, there were an estimated 134,374 new cases in 2022, and it is now the eighth most common cancer type 1. The current median survival of patients with locally advanced, unresectable pancreatic cancer is nine to twelve months, and the five-year survival rate was 7.2% 2, making it the malignancy with the lowest survival rate in China.
1 Xia C, Dong X, Li H et al. Cancer statistics in China and United States, 2022: profiles, trends, and determinants. Chin Med J (Engl) 2022; 135: 584-590.
2 Hu JX, Zhao CF, Chen WB et al. Pancreatic cancer: A review of epidemiology, trend, and risk factors. World J Gastroenterol 2021; 27: 4298-4321.
About Tumor Treating Fields
Tumor Treating Fields (TTFields) are electric fields that exert physical forces to kill cancer cells via a variety of mechanisms. TTFields do not significantly affect healthy cells because they have different properties (including division rate, morphology, and electrical properties) than cancer cells. These multiple, distinct mechanisms work together to target and kill cancer cells. Due to these multimechanistic actions, TTFields therapy can be added to cancer treatment modalities in approved indications and demonstrates enhanced effects across solid tumor types when used with chemotherapy, radiotherapy, immune checkpoint inhibition, or targeted therapies in preclinical models. TTFields therapy provides clinical versatility that has the potential to help address treatment challenges across a range of solid tumors.
To learn more about TTFields therapy and its multifaceted effect on cancer cells, visit tumortreatingfields.com.
About Zai Lab
Zai Lab 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 www.X.com/ZaiLab_Global, www.twitter.com/ZaiLab_Global.
About Novocure
Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer through the development and commercialization of its innovative therapy, Tumor Treating Fields. Novocure's commercialized products are approved in certain countries for the treatment of adult patients with glioblastoma, non-small cell lung cancer, malignant pleural mesothelioma and pleural mesothelioma. Novocure has several additional ongoing or completed clinical trials exploring the use of Tumor Treating Fields therapy in the treatment of glioblastoma, non-small cell lung cancer and pancreatic cancer.
Novocure's global headquarters is located in Baar, Switzerland, with U.S. headquarters located in Portsmouth, New Hampshire and research and development facilities located in Haifa, Israel. For additional information about the company, please visit Novocure.com and follow @Novocure on LinkedIn and Twitter.
Zai Lab Forward-Looking Statements
This press release contains forward-looking statements about future expectations, plans, and prospects for Zai Lab, including, without limitation, statements regarding the prospects of and plans for developing and commercializing TTFields therapy, the potential benefits of TTFields therapy, and the potential treatment of pancreatic cancer. These forward-looking statements may contain words such as 'aim,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'intend,' 'may,' 'plan,' '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 statements of historical fact or 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. 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 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 the SEC's website at www.sec.gov.
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Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; trial designs, clinical development plans and timing for NGN-401, including elements of the registrational clinical study trial design subject to final approval of the FDA, such as the proposed number of participants in the Embolden trial, and the timing of the conversion of the NGN-401 Phase 1/2 clinical trial to a registrational clinical trial, anticipated timing of additional updates for the Embolden registrational trial of NGN-401 for Rett syndrome; expected timing for additional interim data from the Company's NGN-401 Phase 1/2 trial for Rett Syndrome, expected future interactions with or positions of the FDA; and the time period over which existing cash resources may be sufficient to fund the Company's operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene's control. Such risks, uncertainties and assumptions include, among other things: the expected timing of additional results from the NGN-401 clinical trial; the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company's ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of initiating the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed with the Securities and Exchange Commission ("SEC") on August 11, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - Neurogene Inc. Condensed Consolidated Balance Sheet Data (In thousands of U.S. dollars) June 30,2025 December 31,2024 Assets Cash and cash equivalents $ 58,813 $ 136,586 Short-term investments 215,706 175,819 Other current assets 4,467 3,518 Non-current assets 18,330 19,807 Total assets $ 297,316 $ 335,730 Liabilities Current liabilities 15,440 15,157 Non-current liabilities 8,621 10,198 Total liabilities 24,061 25,355 Stockholders' equity 273,255 310,375 Total liabilities and stockholders' equity $ 297,316 $ 335,730 Neurogene Inc. Condensed Consolidated Statements of Operations (In thousands of U.S. dollars, except share information) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024 Revenue under licensing agreements — 925 — 925 Operating expenses: Research and development expenses 19,366 15,744 37,131 29,285 General and administrative expenses 6,715 5,315 14,869 10,553 Total operating expenses 26,081 21,059 52,000 39,838 Loss from operations (26,081 ) (20,134 ) (52,000 ) (38,913 ) Other income, net 4,065 1,642 7,337 3,500 Net loss $ (22,016 ) $ (18,492 ) $ (44,663 ) $ (35,413 ) Per share information: Net loss per share, basic and diluted $ (1.05 ) $ (1.09 ) $ (2.12 ) $ (2.09 ) Weighted-average shares of common stock outstanding, basic and diluted 21,055,378 16,941,524 21,025,996 16,922,630 View source version on Contacts Company Contact: Cara MayfieldVice President, Corporate Investor Contact: Melissa ForstArgot PartnersNeurogene@ Sign in to access your portfolio
