Revvity Announces Financial Results for the Third Quarter of 2025




Revvity Announces Financial Results for the Third Quarter of 2025

• Revenue of $699 million; 2% reported growth; 1% organic growth
• GAAP EPS of $0.40; Adjusted EPS from continuing operations of $1.18
• Reaffirms full year 2025 organic growth and raises adjusted EPS guidance
• Authorizes new $1 billion share repurchase program

WALTHAM, Mass.–(BUSINESS WIRE)–Revvity, Inc. (NYSE: RVTY), today reported financial results for the third quarter ended September 28, 2025.

The Company reported GAAP earnings per share of $0.40, as compared to $0.77 in the same period a year ago. Revenue for the quarter was $699 million, as compared to $684 million in the same period a year ago. GAAP operating income from continuing operations for the quarter was $82 million, as compared to $98 million for the same period a year ago. GAAP operating profit margin from continuing operations was 11.7% as a percentage of revenue, as compared to 14.3% in the same period a year ago.

Adjusted earnings per share from continuing operations for the quarter was $1.18, as compared to $1.28 in the same period a year ago. Adjusted operating income was $182 million, as compared to $193 million for the same period a year ago. Adjusted operating profit margin was 26.1% as a percentage of revenue, as compared to 28.3% in the same period a year ago.

Adjustments for the Company’s non-GAAP financial measures have been noted in the attached reconciliations.

“We performed well during the third quarter as a number of key innovations and strategic partnerships have begun to come to fruition,” said Prahlad Singh, president and chief executive officer of Revvity. “Our strong level of execution is positioning the Company for even greater success in 2026 and beyond.”

Share Repurchase Authorization

The Company’s Board of Directors has authorized a new two-year $1 billion share repurchase program which replaces the remainder of the prior repurchase program which was announced in October 2024.

Financial Overview by Reporting Segment

Life Sciences

• Third quarter 2025 revenue was $343 million, as compared to $339 million in the same period a year ago. Revenue increased 1% and organic revenue was flat as compared to the same period a year ago.

• Third quarter 2025 adjusted operating income was $101 million, as compared to $111 million in the same period a year ago. Adjusted operating profit margin was 29.5% as a percentage of revenue, as compared to 32.6% in the same period a year ago.

Diagnostics

• Third quarter 2025 revenue was $356 million, as compared to $345 million in the same period a year ago. Revenue increased 3% and organic revenue increased 2% as compared to the same period a year ago.

• Third quarter 2025 adjusted operating income was $89 million, as compared to $94 million in the same period a year ago. Adjusted operating profit margin was 25.1% as a percentage of revenue, as compared to 27.2% in the same period a year ago.

Full Year 2025 Guidance

For the full year 2025, the Company is updating its full year revenue guidance to $2.83-$2.88 billion to reflect recent changes in foreign currency exchange rates and is reaffirming its organic growth guidance of 2% to 4%. The Company is also raising its adjusted EPS guidance to a range of $4.90 to $5.00.

Guidance for the full year 2025 for adjusted EPS and organic growth is provided on a non-GAAP basis and cannot be reconciled to the closest GAAP measures without unreasonable effort due to the unpredictability of the amounts and timing of events affecting the items the Company excludes from these non-GAAP measures. The timing and amounts of such events and items could be material to the Company’s results prepared in accordance with GAAP.

Webcast Information

The Company will discuss its third quarter 2025 results and its outlook for business trends during a webcast on October 27, 2025, at 8:00 a.m. Eastern Time. A live audio webcast and presentation will be available on the Investors section of the Company’s website, ir.revvity.com.

Use of Non-GAAP Financial Measures

In addition to financial measures prepared in accordance with generally accepted accounting principles (GAAP), this earnings announcement also contains non-GAAP financial measures. The reasons that we use these measures, a reconciliation of these measures to the most directly comparable GAAP measures, and other information relating to these measures are included below following our GAAP financial statements.

Factors Affecting Future Performance

This press release contains “forward-looking” statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements relating to estimates and projections of future earnings per share, cash flow and revenue growth and other financial results, developments relating to our customers and end-markets, and plans concerning business development opportunities, acquisitions and divestitures. Words such as “believes”, “intends”, “anticipates”, “plans”, “expects”, “estimates”, “projects”, “forecasts”, “will” and similar expressions, and references to guidance, are intended to identify forward-looking statements. Such statements are based on management’s current assumptions and expectations and no assurances can be given that our assumptions or expectations will prove to be correct. A number of important risk factors could cause actual results to differ materially from the results described, implied or projected in any forward-looking statements. These factors include, without limitation: (1) markets into which we sell our products declining or not growing as anticipated; (2) fluctuations in the global economic and political environments, including as the result of recently implemented and recently threatened tariff increases; (3) our failure to introduce new products in a timely manner; (4) our ability to execute acquisitions and divestitures, license technologies, or to successfully integrate acquired businesses or licensed technologies into our existing businesses or to make them profitable; (5) our ability to compete effectively; (6) fluctuation in our quarterly operating results and our ability to adjust our operations to address unexpected changes; (7) significant disruption in third-party package delivery and import/export services or significant increases in prices for those services; (8) disruptions in the supply of raw materials and supplies; (9) our ability to retain key personnel; (10) significant disruption in our information technology systems, or cybercrime; (11) our ability to realize the full value of our intangible assets; (12) our failure to adequately protect our intellectual property; (13) the loss of any of our licenses or licensed rights; (14) the manufacture and sale of products exposing us to product liability claims; (15) our failure to maintain compliance with applicable government regulations; (16) our failure to comply with data privacy and information security laws and regulations; (17) regulatory changes; (18) our failure to comply with healthcare industry regulations; (19) economic, political and other risks associated with foreign operations; (20) our ability to obtain future financing; (21) restrictions in our credit agreements; (22) significant fluctuations in our stock price; (23) reduction or elimination of dividends on our common stock; and (24) other factors which we describe under the caption “Risk Factors” in our most recent quarterly report on Form 10-Q and in our other filings with the Securities and Exchange Commission. We disclaim any intention or obligation to update any forward-looking statements as a result of developments occurring after the date of this press release.

About Revvity

At Revvity, “impossible” is inspiration, and “can’t be done” is a call to action. Revvity provides health science solutions, technologies, expertise and services that deliver complete workflows from discovery to development, and diagnosis to cure. Revvity is revolutionizing what’s possible in healthcare, with specialized focus areas in translational multi-omics technologies, biomarker identification, imaging, prediction, screening, detection and diagnosis, informatics and more.

With 2024 revenue of more than $2.7 billion and approximately 11,000 employees, Revvity serves customers across pharmaceutical and biotech, diagnostic labs, academia and governments. It is part of the S&P 500 index and has customers in more than 160 countries.

Stay updated by following our Newsroom, LinkedIn, X, YouTube, Facebook and Instagram.

Explanation of Non-GAAP Financial Measures

We report our financial results in accordance with GAAP. However, management believes that, in order to more fully understand our short-term and long-term financial and operational trends, investors may wish to consider the impact of certain non-cash, non-recurring or other items, which result from facts and circumstances that vary in frequency and impact on continuing operations. Accordingly, we present non-GAAP financial measures as a supplement to the financial measures we present in accordance with GAAP. These non-GAAP financial measures provide management with additional means to understand and evaluate the operating results and trends in our ongoing business by adjusting for 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. Management believes these non-GAAP financial measures provide additional means of evaluating period-over-period operating performance.

Contacts

Investor Relations:
Steve Willoughby

steve.willoughby@revvity.com

Media Relations:
Chet Murray

(781) 462-5126

chet.murray@revvity.com

Read full story here

New Poll Reveals Canadians Lack Understanding of Obesity as a Chronic Disease, Stigma Likely Bleeds Into Treatment




New Poll Reveals Canadians Lack Understanding of Obesity as a Chronic Disease, Stigma Likely Bleeds Into Treatment

– Ahead of generic alternatives to weight loss medication expected to hit the Canadian market in 2026, Phoenix’s poll also found two in five Canadians would be interested in trying them –

TORONTO–(BUSINESS WIRE)–Today, Phoenix, Canada’s leading digital health clinic for men, released new poll data with insights from over 1,500 Canadians on sentiments around weight loss and the use of GLP-1s. The poll reveals that, despite the growing prevalence of obesity in Canada, many Canadians do not understand it as a chronic disease, which may drive stigma around the condition and the use of GLP-1 drugs for weight loss. Despite this stigma, there is notable interest in using GLP-1s to manage obesity, including generic alternatives.

WEIGHT LOSS IN CANADA

A recent study found one-third of Canadians are obese, and Phoenix’s poll found weight loss is on the mind of many Canadians. The findings reveal 59 per cent of Canadians are currently trying or planning to lose weight, with notable demographic divides. Desire for weight loss is most prominent among Gen X, with 68 per cent indicating they are currently trying or planning to lose weight – a significantly higher percentage than Gen Z (44%), Millennials (58%) and the Baby Boomer generation (57%).

Among Canadians currently trying to or planning on losing weight, the primary motivations are to improve fitness and stamina (62%) and increase lifespan/longevity (50%), followed by enhancing appearance (44%), boosting self-esteem and confidence (40%), and disease prevention (34%).

Notably, the strongest motivators differed from generation to generation. Gen Z’s leading motivator was to boost self-esteem and confidence (71%); Gen X was most motivated by increasing lifespan/longevity (58%); and improving fitness and stamina was the primary motivator for Millennials (63%) and the Baby Boomer generation (70%). 23 per cent of the Baby Boomer generation was also motivated to lose weight to reduce their need for medications.

“There is a misconception that weight loss is only an issue of vanity. That is simply not true. For folks struggling with obesity, losing weight can have a significant impact on overall quality of life, from increasing mobility to improving sleep quality,” says Gavin Thompson, co-founder and co-CEO of Phoenix. “The benefits can go beyond the physical too. Weight loss can also significantly support an individual’s mental health, providing a boost to their self-esteem and confidence.”

STIGMA AROUND WEIGHT LOSS TRICKLES DOWN TO TREATMENT

While the prevalence of obesity among adults in Canada has increased since the pandemic according to Statistics Canada, many Canadians still do not understand obesity as a chronic disease. Less than one in three Canadians (32%) correctly identified that Health Canada classifies obesity as a progressive chronic disease.

The lack of understanding of obesity as a chronic disease drives the stigma around the condition and its treatment. One in three Canadians (34%) say that they would or did feel embarrassed sharing with their friends and family about using drugs like Ozempic or Wegovy for weight loss. This stigma is further perpetuated by celebrity culture, where taking GLP-1 medications for weight management is often viewed differently than using medication for other chronic illnesses. 62 per cent of Canadians agree that celebrities who share their weight loss should also disclose their use of weight loss drugs to the public. Gen Z felt especially strong about this, with 71 per cent agreeing celebrities should disclose their use of weight loss drugs.

“The common perception of obesity is that it’s solely the result of personal choices, but in reality, it’s a chronic disease influenced by many factors. Many people who struggle with obesity cannot manage it with exercise and healthy eating alone, but the stigma around obesity trickles down to its treatment too,” says Kevin Bache, co-founder and co-CEO of Phoenix. “People feel entitled to know that a celebrity has used medication to lose weight, in the same way they expect a celebrity to be transparent about using social media filters to hide their acne. But as with any chronic disease, someone’s choice to use medication as recommended by their doctor is deeply personal, and there should be no shame or expectation of transparency attached.”

NOTABLE INTEREST IN GLP-1S, ESPECIALLY GENERIC

Despite Statistics Canada reporting 68 per cent of Canadians are classified as overweight or obese, only five per cent of Canadians are currently taking GLP-1 medication for weight loss, and a further three per cent have done so in the past. However, 21 per cent of Canadians who indicated they have never taken a GLP-1 medication revealed they would consider taking one in the future.

A potential barrier to Canadians trying GLP-1 weight loss medication could be the cost, as interest in generic weight loss medication is high. 40 per cent of Canadians indicated interest in trying a generic GLP-1 medication for weight loss if they currently struggle with obesity or were to struggle in the future.

WHAT CANADIANS NEED TO KNOW ABOUT GLP-1s

• Do GLP-1 drugs mitigate the need to eat healthy and exercise? While eating less is a goal for many people who struggle with their weight, food intake is only one small piece of a healthy, sustainable weight loss plan. Lifestyle modifications, such as eating a nutritious diet or exercising regularly, are key to sustaining your results.
• Do I need a prescription to take a GLP-1 medication for weight loss? Yes, GLP-1 medication must be prescribed by a licensed physician for weight loss.
• Can anyone take a GLP-1 medication for weight loss? No. In Canada, GLP-1 drugs can only be prescribed by a licensed physician to patients who have a body mass index (BMI) of 30+ or BMI of 27+ with obesity-related health conditions.

Ontario, BC, Alberta, Saskatchewan, Manitoba, New Brunswick, Newfoundland, Nova Scotia and PEI. To learn more about Phoenix’s weight loss support, visit https://www.phoenix.ca/treatments/weight-loss.

ABOUT PHOENIX

Phoenix is Canada’s leading digital health clinic for men, specializing in three areas of treatment – erectile dysfunction, weight loss, and hair loss. The telehealth platform facilitates access to licensed Canadian physicians, treatment options, and free, discreet shipping of prescription medication from coast to coast. Visit Phoenix.ca to learn more.

SURVEY METHODOLOGY

These findings are from a survey conducted by Phoenix from September 24th to September 26th, 2025, among a representative sample of 1505 online adult Canadians who are members of the Angus Reid Forum. The survey was conducted in English and French. For comparison purposes only, a probability sample of this size would carry a margin of error of +/-2.53 percentage points, 19 times out of 20.

Contacts

MEDIA
Anne-Marie Tremble

Senior Account Manager, Talk Shop Media

annemarie@talkshopmedia.com
613-914-3551

Colorectal, Liver, and Lung Cancer Patients at Vinmec Experience Extended Survival with AIET Cell-Based Immunotherapy – Technology Transferred from Japan




Colorectal, Liver, and Lung Cancer Patients at Vinmec Experience Extended Survival with AIET Cell-Based Immunotherapy – Technology Transferred from Japan

Vinmec Health System attracts international patients to Vietnam for treatment

TOKYO–(BUSINESS WIRE)–#AIET–Significantly extended survival and improved quality of life (QoL) for cancer patients achieved through autologous cell-based AIET immunotherapy, utilizing patients’ own natural killer (NK) cells and T cells, has positioned Vinmec Hospital, Vietnam, as an emerging medical tourism destination for cancer treatment across Asian countries. This accomplishment was presented by Professor Nguyen Thanh Liem at the NCRM NICHE 2025. He acknowledged the technology transfer support from GN Corporation, Japan, enabling the implementation of standardized AIET protocols in accordance with Japanese regulations, bringing treatment access to patients at Vinmec since 2018.

AIET leverages patients’ NK and T lymphocytes alongside conventional treatments, increases treatment efficacy by 20–30% and significantly improves survival. In two clinical trials at Vinmec Hospital during 2016–2021 in advanced-stage cancers:

• Colorectal cancer patients’ average survival time increased by 14.3 months;
• Lung, liver and other cancers, average survival time increased by 18.7 months.

Among over 100 patients, including those with late-stage breast, ovarian, thyroid, and head-neck cancers, AIET improved survival rates, enhanced QoL with measurable improvements in: appetite, insomnia, drowsiness, dry mouth, nausea, depression, fatigue, overall symptom burden and physical function capacity. AIET causes virtually no side effects, as it uses patients’ own cells without any foreign biological materials or feeder layers during cultivation.

AIET cell therapy has been officially approved for implementation at Vinmec Hospital under Vietnam’s regenerative medicine regulations, aligned with Japan’s Act on the Safety of Regenerative Medicine.

AIET treatment protocol (one cycle):

• Collection of 100 ml of patient’s peripheral blood,
• Laboratory processing and cultivation over 15–21 days,
• Intravenous infusion of two doses of NK and T cells;

Patients may require 2–6 cycles, depending on cancer progression and stage, said Prof. Liem.

Prof. Liem acknowledged GN Corporation’s outstanding interdisciplinary research, yielding cell therapy solutions, being transferred to hospitals and institutes worldwide.

• BEES-HAUS and BHES-HAUS for male urethral stricture using buccal mucosal epithelium, encapsulated in Festigel scaffold;
• Novel cell therapies for treating epithelial and endothelial corneal diseases;
• EELS-TALC addressing articular cartilage damage using autologous chondrocytes, with reversed cellular aging.

GN Corporation and NCRM are now collaborating with SoulSynergy Ltd., Mauritius, an approved cell processing lab, to offer cell-based therapies practiced in Japan, to patients in Mauritius and African continent.

Contacts

Samuel JK Abraham

info@gncorporation.com

Guardant Health to Share Data Supporting Critical Role of Blood-Based Testing in Improving Cancer Screening Adherence at ACG 2025




Guardant Health to Share Data Supporting Critical Role of Blood-Based Testing in Improving Cancer Screening Adherence at ACG 2025

• Expanded cohort reiterates strong adherence rate of over 90% for Shield blood-based colorectal cancer screening test
• Survey findings show eligible individuals prefer a blood test for colorectal cancer and lung cancer compared with traditional screening methods

PALO ALTO, Calif.–(BUSINESS WIRE)–$GH–Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, today announced the company and its research collaborators will present data showing the critical role of blood-based testing in increasing cancer screening adherence at the American College of Gastroenterology (ACG) 2025 Annual Meeting in Phoenix, Arizona taking place Friday, October 24 – Wednesday, October 29, 2025.

Building off findings from Guardant Health’s 2023 ACG abstract, a study of an expanded cohort of 20,000 patients confirmed findings from earlier reports on the effectiveness of the Shield blood-based screening test in improving adherence to colorectal cancer (CRC) screening. Shield is the first and only blood test to receive FDA approval as a primary screening option for colorectal cancer in average-risk adults aged 45 and older. The findings demonstrated an over 90% adherence rate for Shield, tracking well above average screening adherence for overall CRC testing which ranges from 28-71%. ^1-4

A separate study led by researchers at Cedars Sinai found that individuals prefer a blood test for colorectal cancer and lung cancer over other methods, showing the potential for an innovative blood test like Shield to increase the overall screening rate in lung cancer screening. The survey found that more screening-eligible individuals (43.9%) prefer to do a blood test for colorectal cancer and lung cancer compared to traditional screenings.

“Today’s research reinforces what Guardant has long believed: blood-based cancer screening is the future,” said Dr. Craig Eagle, Chief Medical Officer at Guardant Health. “These studies highlight the power of a simple blood test in addressing today’s gaps in colorectal cancer screening adherence and potential to expand to an even broader population looking for a more pleasant option for their regular lung cancer screening. We’re eager to present these findings along with our research collaborators at the ACG meeting and proud to offer Shield, an FDA-approved test, as an accessible and convenient alternative to traditional screening methods.”

Guardant Health and collaborator presentations at ACG 2025

The full abstracts for Guardant Health and a list of all abstracts being presented at the ACG Annual Meeting can be found here.

About Shield

Shield is a non-invasive, blood-based screening test that detects alterations associated with colorectal cancer in the blood. It is intended as a screening test for individuals at average risk for the disease, age 45 or older, and is not intended for individuals at high risk for colorectal cancer. The Shield test can be considered in a manner similar to guideline-recommended non-invasive CRC screening options and can be completed during any healthcare visit. A positive Shield result raises concern for the presence of colorectal cancer or advanced adenoma and the patient should be referred for colonoscopy evaluation.

About Guardant Health

Guardant Health is a leading precision oncology company focused on guarding wellness and giving every person more time free from cancer. Founded in 2012, Guardant is transforming patient care and accelerating new cancer therapies by providing critical insights into what drives disease through its advanced blood and tissue tests, real-world data and AI analytics. Guardant tests help improve outcomes across all stages of care, including screening to find cancer early, monitoring for recurrence in early-stage cancer, and treatment selection for patients with advanced cancer. For more information, visit guardanthealth.com and follow the company on LinkedIn, X (Twitter) and Facebook.

Guardant Health Forward-Looking Statements

This press release contains forward-looking statements within the meaning of federal securities laws, including statements regarding the potential utilities, values, benefits and advantages of Guardant Health’s liquid biopsy tests or assays, which involve risks and uncertainties that could cause the actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors. These and additional risks and uncertainties that could affect Guardant Health’s financial and operating results and cause actual results to differ materially from those indicated by the forward-looking statements made in this press release include those discussed under the captions “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operation” and elsewhere in its Annual Report on Form 10-K for the year ended December 31, 2024 and in its other reports filed with or furnished to the Securities and Exchange Commission. The forward-looking statements in this press release are based on information available to Guardant Health as of the date hereof, and Guardant Health disclaims any obligation to update any forward-looking statements provided to reflect any change in its expectations or any change in events, conditions, or circumstances on which any such statement is based, except as required by law. These forward-looking statements should not be relied upon as representing Guardant Health’s views as of any date subsequent to the date of this press release.

¹ Denberg TD, Melhado TV, Coombes JM, et al. Predictors of nonadherence to screening colonoscopy. J Gen Intern Med. 2005;20(11):989-995.

² Gellad ZF, Stechuchak KM, Fisher DA, et al. Longitudinal adherence to fecal occult blood testing impacts colorectal cancer screening quality. Am J Gastroenterol. 2011;106(6):1125-1134.

³ Inadomi JM, Vijan S, Janz NK, et al. Adherence to colorectal cancer screening: a randomized clinical trial of competing strategies. Arch Intern Med. 2012;172(7):575-582.

⁴ Exact Sciences. Third quarter 2019 webcast and conference call. Updated October 29, 2019. https:/ investor.exactsciences.com/investor-relations/events-and-presentations/event-details/2019/Third-Quarter-2019-Webcast-Conference-Call/default.aspx

Contacts

Investor Contact:
Zarak Khurshid

investors@guardanthealth.com

Media Contact:
Meaghan Smith

press@guardanthealth.com

Incyte Announces New Data from Phase 3b TRuE-AD4 Trial of Opzelura® (Ruxolitinib Cream) in Adults with Moderate Atopic Dermatitis




Incyte Announces New Data from Phase 3b TRuE-AD4 Trial of Opzelura® (Ruxolitinib Cream) in Adults with Moderate Atopic Dermatitis

• Eight-week results from the TRuE-AD4 trial demonstrate treatment with Opzelura^® (ruxolitinib cream) significantly improved the clinical signs of atopic dermatitis (AD), including improved itch as early as Day 2, and was well tolerated in adults with moderate AD who had an inadequate response, intolerance or contraindication to topical corticosteroids (TCSs) and topical calcineurin inhibitors (TCIs)

• At Week 8, 70% of patients treated with Opzelura achieved a ≥75% improvement in the Eczema Area and Severity Index (EASI75) and 61.3% achieved Investigator’s Global Assessment Treatment Success (IGA-TS), co-primary endpoints of the study

• Based on these results, Incyte expects to file a Type-II variation application for ruxolitinib cream 1.5% for the treatment of adults with moderate AD in the European Union (EU) by end of year

WILMINGTON, Del.–(BUSINESS WIRE)–$INCY #ISAD2025–Incyte (Nasdaq:INCY) today announced new data from the Phase 3b TRuE-AD4 study evaluating the efficacy and safety of Opzelura^® (ruxolitinib cream) in adults with moderate atopic dermatitis (AD) who had an inadequate response, intolerance or contraindication to topical corticosteroids (TCSs) and topical calcineurin inhibitors (TCIs). These data will be presented during the Systemic and New Therapies for Atopic Dermatitis session on Sunday, October 26 at 10:55 a.m. AEDT (Saturday, October 25 at 7:55 p.m. ET) (Abstract #1177) at the 15th Georg Rajka International Symposium on Atopic Dermatitis (ISAD), held from October 24 – 26, 2025, in Melbourne.

Data from TRuE-AD4, which build on previously announced topline results, show the study met its co-primary endpoints at Week 8, with a statistically significant proportion of patients achieving both a ≥75% improvement in Eczema Area and Severity Index (EASI75) score from baseline (70.0% with Opzelura vs. 18.5% with vehicle, P<0.0001) and Investigator’s Global Assessment Treatment Success (IGA-TS; 61.3% vs. 13.6%; P<0.0001). Patients treated with Opzelura demonstrated improvement in EASI75 (43.8% vs. 3.7%, nominal P<0.0001) and IGA-TS (29.4% vs. 2.5%, nominal P<0.0001) at Week 2. More patients treated with Opzelura achieved both EASI75 and IGA-TS at Week 8 than vehicle (59.4% vs. 13.6%; nominal P<0.0001).

“The TRuE-AD4 data further reinforce the safety and efficacy profile of Opzelura and its ability to control key signs and symptoms of moderate AD, including improvement in bothersome symptoms like itch,” said Jim Lee, M.D., Group Vice President, Inflammation & Autoimmunity, Incyte. “These data will support the filing of a Type-II variation application for ruxolitinib cream 1.5% (Opzelura) in Europe, as we seek to meet the needs of more patients around the world who require nonsteroidal topical treatments for moderate AD.”

Additional key findings from the TRuE-AD4 study include:

• Improvement in itch as measured by a ≥4-point improvement in Itch Numeric Rating Scale (NRS4) score.
  • Nearly two-thirds (62.5%) of patients treated with Opzelura achieved Itch NRS4 by Week 8 (vs. 19.8% with vehicle, P<0.0001).
  • There was significant improvement in worst daily itch at Day 2 with 29.1% of patients treated with Opzelura achieving Itch NRS4 vs. 14.3% with vehicle (P=0.0072; by multiple imputation).
  • Current Itch NRS4 was also measured at 15 minutes (16.4% with Opzelura vs. 7.7% with vehicle).
• Improved Patient-Oriented Eczema Measure (POEM) and Dermatology Life Quality Index (DLQI) scores:
  • More patients treated with Opzelura vs. vehicle achieved a POEM score 0–2 (clear or almost clear) at Week 8 (39.7% vs. 8.6%).
  • Patients treated with Opzelura showed a mean DLQI score improvement at Week 8 (from 19.3 to 4.3 with Opzelura vs. 19.1 to 10.7 with vehicle).
• Opzelura was well tolerated with no serious infections, major adverse cardiovascular events (MACE), malignancies or thromboses reported during the 8-week vehicle-controlled period. The most common treatment-related adverse event observed in the Opzelura arm was application site acne (4.4% vs 0% in vehicle arm).

“TRuE-AD4 offers compelling support for the utility of Opzelura for patients with moderate AD who have limited treatment options due to inadequate responses or intolerances to TCS and TCI-based topical therapies, who may otherwise be recommended for systemic therapy,” said Dr. Andreas Wollenberg, Professor of Dermatology and Allergy, Augsburg University Hospital, Germany. “AD is a challenging, chronic condition and I believe that these data reinforce Opzelura as an important therapeutic option.”

Atopic dermatitis (AD) – the most common type of eczema –is a chronic, recurring, inflammatory and highly pruritic skin condition that affects up to 25% of children and up to 12% of adults worldwide, with an estimated prevalence among adults of 5.5% across 27 European countries.^{1,2,3,4,5,6,7} Signs and symptoms include irritated and itchy skin that can cause red lesions that may ooze and crust.

More information regarding the Georg RAJKA International Symposium on Atopic Dermatitis (ISAD) can be found at https://isad.org/rajka-symposium.

About TRuE-AD4

TRuE-AD4 (NCT06238817) is a randomized, double-blind, vehicle-controlled Phase 3b study designed to evaluate the efficacy and safety of Opzelura^® (ruxolitinib cream) in adults with moderate atopic dermatitis (AD). The study enrolled 241 patients (≥18 years old) who meet the specific inclusion criteria, including an Investigator’s Global Assessment (IGA) score of 3 and an Eczema Area and Severity Index (EASI) score greater than 7 at both screening and Day 1 and who have AD on 10% to 20% of their Body Surface Area (BSA; excluding scalp). Patients also had to have a documented history of inadequate response, intolerance or contraindication to topical corticosteroid (TCS)s and topical calcineurin inhibitor (TCI)s within the 12 months prior to the screening visit.

Patients were randomized 2:1 to receive Opzelura administered twice daily (BID) or vehicle (non-medicated cream) BID.

The co-primary endpoints of TRuE-AD4 are the proportion of patients achieving IGA Treatment Success (IGA-TS), defined as an IGA score of 0 (clear) or 1 (almost clear) with at least a two-point improvement from baseline, and EASI-75, defined as ≥75% improvement in EASI score, at Week 8. Key secondary endpoints include the proportion of patients with a ≥4-point improvement in Itch Numeric Rating Scale (NRS4) score at various time points. Other exploratory endpoints include the proportion of patients who achieved IGA-TS, NRS4, EASI-75, a decrease from baseline in the affected body surface area (%BSA), change from baseline in the skin pain NRS score, EASI90 and more, measured at various time points. The study also tracked the frequency, duration and severity of adverse events associated with the use of ruxolitinib cream.

For more information on the study, visit https://www.clinicaltrials.gov/study/NCT06238817.

About Opzelura^® (ruxolitinib) Cream

Opzelura (ruxolitinib) cream, a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib, approved by the U.S. Food & Drug Administration for the topical treatment of nonsegmental vitiligo in patients 2 years of age and older, is the first and only treatment for repigmentation approved for use in the United States. Opzelura is also approved in the U.S. for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 2 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. Use of Opzelura in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants, such as azathioprine or cyclosporine, is not recommended.

In Europe, Opzelura^® (ruxolitinib) cream 15mg/g is approved for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.

Incyte has worldwide rights for the development and commercialization of ruxolitinib cream, marketed in the United States as Opzelura.

Opzelura and the Opzelura logo are registered trademarks of Incyte.

IMPORTANT SAFETY INFORMATION

OPZELURA is for use on the skin only. Do not use OPZELURA in your eyes, mouth, or vagina.

OPZELURA may cause serious side effects, including:

Serious Infections: OPZELURA contains ruxolitinib. Ruxolitinib belongs to a class of medicines called Janus kinase (JAK) inhibitors. JAK inhibitors are medicines that affect your immune system. JAK inhibitors can lower the ability of your immune system to fight infections. Some people have had serious infections while taking JAK inhibitors by mouth, including tuberculosis (TB), and infections caused by bacteria, fungi, or viruses that can spread throughout the body. Some people have been hospitalized or died from these infections. Some people have had serious infections of their lungs while taking OPZELURA. Your healthcare provider should watch you closely for signs and symptoms of TB during treatment with OPZELURA.

OPZELURA should not be used in people with an active, serious infection, including localized infections. You should not start using OPZELURA if you have any kind of infection unless your healthcare provider tells you it is okay. You may be at a higher risk of developing shingles (herpes zoster) while using OPZELURA.

Increased risk of death due to any reason (all causes): Increased risk of death has happened in people 50 years of age and older who have at least 1 heart disease (cardiovascular) risk factor and are taking a medicine in the class of medicines called JAK inhibitors by mouth.

Cancer and immune system problems: OPZELURA may increase your risk of certain cancers by changing the way your immune system works. Lymphoma and other cancers have happened in people taking a medicine in the class of medicines called JAK inhibitors by mouth. People taking JAK inhibitors by mouth have a higher risk of certain cancers including lymphoma and lung cancer, especially if they are a current or past smoker. Some people have had skin cancers while using OPZELURA. Your healthcare provider will regularly check your skin during your treatment with OPZELURA. Limit the amount of time you spend in the sunlight. Wear protective clothing when you are in the sun and use a broad-spectrum sunscreen.

Increased risk of major cardiovascular events: Increased risk of major cardiovascular events such as heart attack, stroke, or death have happened in people 50 years of age and older who have at least 1 heart disease (cardiovascular) risk factor and taking a medicine in the class of medicines called JAK inhibitors by mouth, especially in current or past smokers.

Blood clots: Blood clots in the veins of your legs (deep vein thrombosis, DVT) or lungs (pulmonary embolism, PE) can happen in some people taking OPZELURA. This may be life-threatening. Blood clots in the vein of the legs (deep vein thrombosis, DVT) and lungs (pulmonary embolism, PE) have happened more often in people who are 50 years of age and older and with at least 1 heart disease (cardiovascular) risk factor taking a medicine in the class of medicines called JAK inhibitors by mouth.

Low blood cell counts: OPZELURA may cause low platelet counts (thrombocytopenia), low red blood cell counts (anemia), and low white blood cell counts (neutropenia). Your healthcare provider may do a blood test to check your blood cell counts during your treatment with OPZELURA and may stop your treatment if signs or symptoms of low blood cell counts happen.

Cholesterol increases: Cholesterol increase has happened in people when ruxolitinib is taken by mouth. Tell your healthcare provider if you have high cholesterol or triglycerides.

Before starting OPZELURA, tell your healthcare provider if you:

• have an infection, are being treated for one, or have had an infection that does not go away or keeps coming back
• have diabetes, chronic lung disease, HIV, or a weak immune system
• have TB or have been in close contact with someone with TB
• have had shingles (herpes zoster)
• have or have had hepatitis B or C
• live, have lived in, or have traveled to certain parts of the country (such as the Ohio and Mississippi River valleys and the Southwest) where there is an increased chance for getting certain kinds of fungal infections. These infections may happen or become more severe if you use OPZELURA. Ask your healthcare provider if you do not know if you have lived in an area where these infections are common.
• think you have an infection or have symptoms of an infection such as: fever, sweating, or chills, muscle aches, cough or shortness of breath, blood in your phlegm, weight loss, warm, red, or painful skin or sores on your body, diarrhea or stomach pain, burning when you urinate or urinating more often than usual, feeling very tired.
• have ever had any type of cancer, or are a current or past smoker
• have had a heart attack, other heart problems, or a stroke
• have had blood clots in the veins of your legs or lungs in the past
• have high cholesterol or triglycerides
• have or have had low white or red blood cell counts
• are pregnant or plan to become pregnant. It is not known if OPZELURA will harm your unborn baby. There is a pregnancy exposure registry for individuals who use OPZELURA during pregnancy. The purpose of this registry is to collect information about the health of you and your baby. If you become exposed to OPZELURA during pregnancy, you and your healthcare provider should report exposure to Incyte Corporation at 1-855-463-3463 or www.opzelura.pregnancy.incyte.com.
• are breastfeeding or plan to breastfeed. It is not known if OPZELURA passes into your breast milk. Do not breastfeed during treatment with OPZELURA and for about 4 weeks after the last dose.

After starting OPZELURA:

• Call your healthcare provider right away if you have any symptoms of an infection. OPZELURA can make you more likely to get infections or make worse any infections that you have.
• Get emergency help right away if you have any symptoms of a heart attack or stroke while using OPZELURA, including:
  • discomfort in the center of your chest that lasts for more than a few minutes, or that goes away and comes back
  • severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
  • pain or discomfort in your arms, back, neck, jaw, or stomach
  • shortness of breath with or without chest discomfort
  • breaking out in a cold sweat
  • nausea or vomiting
  • feeling lightheaded
  • weakness in one part or on one side of your body
  • slurred speech

• Tell your healthcare provider right away if you have any signs and symptoms of blood clots during treatment with OPZELURA, including: swelling, pain, or tenderness in one or both legs, sudden, unexplained chest or upper back pain, or shortness of breath or difficulty breathing.
• Tell your healthcare provider right away if you develop or have worsening of any symptoms of low blood cell counts, such as: unusual bleeding, bruising, tiredness, shortness of breath, or fever.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of OPZELURA in people 12 years of age and older treated for atopic dermatitis include: common cold (nasopharyngitis), bronchitis, ear infection, increase in a type of white blood cell (eosinophil) count, hives, diarrhea, inflamed hair pores (folliculitis), swelling of the tonsils (tonsillitis), and runny nose (rhinorrhea). For people 2-11 years: upper respiratory tract infection, COVID-19, application site reaction, fever, white blood cell count decreased.

These are not all of the possible side effects of OPZELURA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Incyte Corporation at 1-855-463-3463.

Please see the Full Prescribing Information, including Boxed Warning, and Medication Guide for OPZELURA at Opzelura.com.

INDICATION AND USAGE

OPZELURA is a prescription medicine used on the skin (topical) for the short-term and non-continuous chronic treatment of mild to moderate eczema (atopic dermatitis) in non-immunocompromised adults and children 2 years of age and older whose disease is not well controlled with topical prescription therapies or when those therapies are not recommended.

The use of OPZELURA along with therapeutic biologics, other JAK inhibitors, or strong immunosuppressants such as azathioprine or cyclosporine is not recommended.

It is not known if OPZELURA is safe and effective in children less than 2 years of age with atopic dermatitis.

About Incyte

A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia.

For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.

Incyte Forward-Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data from the TRuE-AD4 study, Incyte’s plans to file an application with regulators in Europe, and whether and when Opzelura might provide a successful treatment option to patients beyond its already-approved indications in specific regions, contain predictions, estimates, and other forward-looking statements.

These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA, EMA, and other regulatory authorities; the efficacy or safety of Incyte and its partners’ products; the acceptance of Incyte and its partners’ products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in our reports filed with the U.S. Securities and Exchange Commission, including our annual report on Form 10-K and our quarterly report on Form 10-K for the quarter ended June 30, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.

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SetPoint Medical to Present Data at ACR Convergence 2025 Demonstrating Sustained Efficacy and Safety for the SetPoint System to Treat Rheumatoid Arthritis




SetPoint Medical to Present Data at ACR Convergence 2025 Demonstrating Sustained Efficacy and Safety for the SetPoint System to Treat Rheumatoid Arthritis

Four presentations highlight 12-month clinical outcomes, as well as MRI-assessed joint protection and patient perspectives supporting the SetPoint System as first-of-its-kind treatment for rheumatoid arthritis (RA)

VALENCIA, Calif.–(BUSINESS WIRE)–SetPoint Medical, a company dedicated to developing therapies for people living with chronic autoimmune diseases, today announced it will share four presentations highlighting new data about the SetPoint System at the upcoming American College of Rheumatology (ACR) Convergence 2025, the largest global gathering of rheumatologists. Presentations will include clinical data demonstrating that the first-of-its-kind SetPoint System’s neuroimmune modulation therapy provides sustained clinical efficacy over 12 months, inhibits joint erosion progression, and is of strong interest to adults living with moderate-to-severe rheumatoid arthritis (RA), particularly among those not satisfied with current biologic or Janus kinase (JAK) inhibitor treatments.

“The RESET-RA data document sustained clinical efficacy through one year of therapy indicating that neuroimmune modulation is effective for RA, including for those who have inadequate response to one or more biologic or JAK inhibitors,” said David Chernoff, M.D., Chief Medical Officer of SetPoint Medical. “Importantly, magnetic resonance imaging (MRI) data provides objective evidence that the SetPoint System inhibits progression of joint erosions as early as three months.”

The data are based on outcomes from the RESET-RA study, a pivotal, randomized, sham-controlled, double-blind trial of 242 patients with moderately to severely active RA who were incomplete responders or intolerant to at least one biologic or targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARDs). The study met its primary efficacy endpoint and demonstrated a favorable safety profile leading to FDA approval for the SetPoint System in July 2025.

“We’re thrilled with the breadth of data being presented at ACR, further demonstrating the potential of the SetPoint System in treating people living with RA,” said Murthy V. Simhambhatla, Ph.D., CEO of SetPoint Medical. “The patient preference findings reveal that individuals with RA—particularly those with prior biologic exposure—view this treatment approach positively, underscoring the need for more therapeutic options that are safe and effective.”

ACR 2025 Abstract Details

Abstract 2278: Neuroimmune Modulation for the Treatment of Rheumatoid Arthritis: Results at 12 Months from a Randomized, Sham-Controlled, Double-Blind Study

Twelve-month data from the RESET-RA study showed that SetPoint System achieved durable efficacy, sustained disease improvement, and a favorable safety profile in adults with rheumatoid arthritis who had an inadequate response or intolerance to biologic or targeted synthetic DMARDs, such as JAK inhibitors.

Key Findings:

• The statistically significant ACR20 response at three months (p=0.0209) improved further in the treatment group during open label to: 52.1% at six months, 51.7% at nine months, and 55.8% at 12 months. Similar ACR20 responses were observed among patients in the control group after they crossed over to active treatment at three months.
• Disease activity scores showed a similar trend, with 49.3% achieving DAS28-CRP low disease activity or remission, 47.4% achieving CDAI low disease activity or remission, and 77.3% achieving a EULAR good/moderate response among patients who received SetPoint Therapy through 12 months without the addition of biologics or JAK inhibitors.
• 97.5% remained on stimulation therapy at 12 months, and therapy augmentation with a biologic or JAK inhibitor occurred in 24.8% of patients.
• The implant procedure was well tolerated, with a 1.7% serious adverse event rate related to the device, procedure or stimulation. No related serious adverse events were observed following the three-month perioperative period through 12 months of follow up.
• No deaths or unanticipated adverse device effects occurred.

Abstract 1675: Neuroimmune Modulation in Patients With Active Rheumatoid Arthritis With an Inadequate Response to TNF Inhibitors (TNFi)

A RESET-RA subgroup analysis demonstrated that vagus nerve-mediated neuroimmune modulation achieved statistically significant improvement in efficacy at three months and sustained clinical benefit through 12 months, supporting neuroimmune modulation as a non-pharmaceutical option after TNFi inadequate response.

Key Findings:

• ACR20 response at three months showed a statistically significant difference between treatment 42.4% vs. control 18.2% (p=0.0057).
• ACR responses, joint counts and disease activity measures improved further through 12 months of treatment. For treatment patients receiving active stimulation without the addition of biologics or JAK inhibitors (i.e., non-augmented), ACR20/50/70 response rates at 12 months were 60%, 38% and 16% respectively, with 52% and 49% achieving LDA or remission per DAS28-CRP and CDAI scores, respectively.
• Therapy augmentation with a biologic or JAK inhibitor occurred in 13% of patients through 12 months.

Abstract 2614: Impact of Vagus Nerve-Mediated Neuroimmune Modulation on Structural Joint Damage Using Gd-MRI RAMRIS Imaging in Biologic-Experienced Patients With Rheumatoid Arthritis

MRI imaging analysis from the RESET-RA study demonstrated that the SetPoint System inhibited joint erosion progression in RA patients.

Key Findings:

• In the pre-specified “erosive phenotype” subgroup of patients with active synovitis or osteitis at baseline, the proportion of patients with erosion progression at three months was significantly lower for treatment (18.9%) compared to control (37.8%, p=0.0156).
• The non-enriched, intention-to-treat (ITT) population showed that at three months, treatment had fewer patients with erosion progression compared to control, but the difference was not statistically significant.
• In the control group, after crossover to active treatment and stimulation for three months, the proportion of patients with erosion progression reduced to levels comparable to the treatment group.

Abstract 0370: Patient Preferences for Treatments of Rheumatoid Arthritis: A Discrete Choice Experiment Evaluating Preference for Advanced Drug Therapies and Neuroimmune Modulation Device

A national patient preference study showed that RA patients expressed strong interest in neuroimmune modulation therapy, particularly among those not satisfied with current biologic or JAK inhibitor treatments. The results highlight significant unmet needs in treatment satisfaction and demonstrate that patients value outcomes, such as symptom improvement, physical function, fatigue reduction, and joint protection, when considering future therapy options.

Key Findings:

• Only 32% of respondents were satisfied with their current RA therapy.
• Initial impression of SetPoint Therapy was regarded as “positive” or “extremely positive” by 45% of respondents.
• Neuroimmune modulation was preferred over switching to another biologic or JAK therapy among biologic-experienced patients.
• Key drivers of preference:
  • Out-of-pocket treatment cost
  • Improvement in symptoms
  • Level of physical function and fatigue
  • Effectiveness in protecting against irreversible joint damage

ACR 2025 Presentation Details

Abstract 0370: Patient Preferences for Treatments of Rheumatoid Arthritis: A Discrete Choice Experiment Evaluating Preference for Advanced Drug Therapies and Neuroimmune Modulation Device

• Session: (0357–0386) Patient Outcomes, Preferences, & Attitudes Poster I
• Location: Hall F1
• Date & Time: Sunday, October 26, 2025, 10:30 AM – 12:30 PM CT
• Presented by: Jeffrey Curtis, M.D., MS, MPH, University of Alabama at Birmingham in Hoover, ALA

Abstract 1675: Neuroimmune Modulation in Patients With Active Rheumatoid Arthritis With an Inadequate Response to TNF Inhibitors (TNFi)

• Session: Abstracts: Rheumatoid Arthritis – Treatment I: Preventative and Novel Treatments (1674–1679)
• Location: S105
• Date & Time: Monday, October 27, 2025, 1:15 – 1:30 PM CT
• Presented by: Guillermo Valenzuela, MD, FACR, Medical Director of Integral Rheumatology & Immunology Specialists and IRIS Research and Development in Plantation and Miami, FL

Abstract 2278: Neuroimmune Modulation for the Treatment of Rheumatoid Arthritis: Results at 12 Months from a Randomized, Sham-Controlled, Double-Blind Study

• Session: (2265–2289) Rheumatoid Arthritis – Treatment Poster III
• Location: Hall F1
• Date & Time: Tuesday, October 28, 2025, 10:30 AM – 12:30 PM CT
• Presented by: John Tesser, MD, FACP, FACR, MACR of Arizona Arthritis & Rheumatology Associates in Phoenix, AZ, and national rheumatology principal investigator of the RESET-RA study

Abstract 2614: Impact of Vagus Nerve-Mediated Neuroimmune Modulation on Structural Joint Damage Using Gd-MRI RAMRIS Imaging in Biologic-Experienced Patients With Rheumatoid Arthritis

• Session: Abstracts: Imaging of Rheumatic Diseases (2609–2614)
• Location: S105
• Date & Time: Tuesday, October 28, 2025, 4:15 – 4:30 PM CT
• Presented by: Charles Peterfy, MD, PhD, Spire Sciences, Inc. in Boca Raton, FL

About SetPoint System

The SetPoint System is indicated for use in the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response, loss of response, or intolerance to one or more biological or targeted synthetic disease modifying antirheumatic drugs. It should not be used if you have had a vagotomy or splenectomy, or if your healthcare provider determines that it is not safe for you to use the SetPoint System. Risks may include, but are not limited to, pain or infection after surgery, hoarseness, bruising, swelling, coughing and throat irritation.

For full safety information, please see Instructions for Use and our Important Safety Information at spm.care/ISI. Individual results may vary.

About SetPoint Medical

SetPoint Medical is a commercial-stage medical technology company dedicated to improving care for people living with chronic autoimmune diseases. The company’s FDA-approved SetPoint System is the first neuroimmune modulation therapy available for people living with rheumatoid arthritis (RA), offering a novel, device-based alternative for those who do not respond to or cannot tolerate biologic or targeted drug therapies. SetPoint’s proprietary integrated neurostimulation platform is designed to activate innate anti-inflammatory pathways in the vagus nerve to reduce inflammation and restore immunologic setpoint. With a vision to redefine the care of autoimmune conditions, SetPoint is also planning to evaluate its platform technology for other conditions including multiple sclerosis and Crohn’s disease. Learn more at setpointmedical.com.

Contacts

Emma Yang

Health+Commerce

media@setpointmedical.com

Bristol Myers Squibb Presents Encouraging Data from Phase 1 Breakfree-1 Study of CD19 NEX-T™ CAR T Cell Therapy in Three Chronic Autoimmune Diseases at ACR Convergence 2025




Bristol Myers Squibb Presents Encouraging Data from Phase 1 Breakfree-1 Study of CD19 NEX-T™ CAR T Cell Therapy in Three Chronic Autoimmune Diseases at ACR Convergence 2025

Positive, early results from 71 patients treated across three disease cohorts – systemic sclerosis, systemic lupus erythematosus and idiopathic inflammatory myopathies – are being disclosed

Across all cohorts, results support potential for immune reset, with 94% of evaluable patients remaining off chronic immunosuppressive therapy at the time of analysis

Results demonstrate BMS’ steady advancement toward its goal of bringing the science of cell therapy beyond blood cancer into autoimmune diseases

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #ACR25—Bristol Myers Squibb (NYSE: BMY) today announced updated data and first disclosure of results in chronic, refractory autoimmune diseases from the Phase 1 Breakfree-1 study (NCT05869955) of its investigational, autologous CD19-targeted NEX-T™ CAR T cell therapy BMS-986353 (CC-97540). The data, evaluating a total of 71 treated patients across the systemic sclerosis (SSc), systemic lupus erythematosus (SLE) and idiopathic inflammatory myopathies (IIM) cohorts, are being featured at the American College of Rheumatology (ACR) Convergence 2025, taking place from October 24-29 in Chicago, Illinois.

The preliminary Phase 1 safety and efficacy results presented are consistent with the potential for immune reset, as part of a treatment process that includes a one-time infusion of CD19 NEX-T, showing robust CAR T cell expansion, complete B cell depletion and re-emergence of a naive B cell phenotype across all three cohorts. At the time of analysis, 94% of evaluable patients remained off chronic immunosuppressive therapy.

All cohorts presented demonstrate an acceptable safety profile, with the majority of adverse events occurring shortly after infusion and resolving quickly with standard management protocols, as is expected with CAR T cell therapies.

“Over the last decade, BMS has been a leader in revolutionizing the treatment of certain blood cancers with cell therapy. Now, we are building on this expertise to bring the modality into the new frontier of autoimmune diseases,” said Lynelle B. Hoch, president, Cell Therapy Organization, Bristol Myers Squibb. “While early, these data demonstrate our focus and steady advancement toward introducing the potential of treatment-free remission, which just a few years ago was not thought to be possible for patients with severe autoimmune disorders.”

CD19 NEX-T pairs the CAR construct used in BMS’ FDA-approved Breyanzi (lisocabtagene maraleucel; liso-cel), approved in certain hematologic malignancies, with the next-generation NEX-T platform which aims to optimize the manufacturing process. CD19 NEX-T is being investigated within a robust, multi-cohort clinical trial program, evaluating the potential for cell therapy to address the underlying cause of multiple severe/refractory autoimmune diseases by inducing immune reset.

Updated Results from Phase 1 Study of Breakfree-1 in SSc (Abstract #0843)

Updated Phase 1 results from the SSc cohort of Breakfree-1 demonstrate unprecedented improvement in pulmonary function in patients with SSc-interstitial lung disease (ILD) and clinically meaningful improvement in skin thickness.

The full SSc cohort included 26 treated patients, with 19 efficacy evaluable at the time of analysis. A median relative predicted forced vital capacity (pFVC) increase from baseline of 10% was seen at six months (n=6) in subjects with ILD at baseline. This increase is notable as improvement in pFVC has not been seen with any other therapeutic modalities.

The treated patients experienced a safety profile consistent with the expected safety profile of cell therapy. All cytokine release syndrome (CRS) events were low grade and resolved in a median of two days. Two grade three immune effector cell-associated neurotoxicity syndrome (ICANS) events were transient and resolved completely within five days. The majority of all related treatment-emergent adverse events (TEAEs) occurred shortly after infusion, were low grade and resolved quickly with standard management.

“These updated results highlight the potential for patients with systemic sclerosis to achieve B cell depletion, with meaningful improvement in lung function and skin thickness, following a single infusion of CAR T cell therapy, even after discontinuing SSc-directed therapies prior to infusion,” said Dr. Dinesh Khanna, Professor of Medicine and Director, University of Michigan Scleroderma Program. “The potential for new treatment approaches to ‘reset’ a patient’s immune system, while early, is highly encouraging and provides hope for patients living with these chronic diseases.”

Updated Results from Phase 1 Study of Breakfree-1 in SLE (Abstract #1529)

Updated results from the SLE cohort of Breakfree-1 show rapid improvement in disease activity sustained at the time of follow-up (up to 18 months), indicating potential for long-term treatment-free remission.

Thirty-two SLE patients with active, severe organ involvement and highly refractory to prior treatment (median 7 prior SLE-specific therapies; range 3-13) were treated across two dose levels (DL). Based on the cumulative safety, efficacy and pharmacokinetics and pharmacodynamics, DL1 was chosen as the Phase 2 recommended dose (RP2D). The disclosure focuses on 26 patients treated at DL1. All efficacy-evaluable patients except one had resolution of clinical symptoms, as shown by substantial reductions in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) and Physician’s Global Assessment (PGA) scores by six months of follow-up. At the time of evaluation, 92% of patients remained off SLE-specific immuno-suppressive therapies (ISTs).

All inflammatory adverse events (AEs) were transient and fully reversible. Most CRS events were grade one or two (73.1% of patients at RP2D), with one patient experiencing a grade three event that resolved within one day. No high-grade ICANS was observed in the SLE cohort at the RP2D.

First Disclosure of Preliminary Results from Phase 1 Breakfree-1 Study in IIM (Abstract #LB14)

A late-breaking abstract at ACR, the first disclosure of data from the Breakfree-1 IIM cohort shows 91% of efficacy-evaluable patients (n=11) achieved a moderate-major composite assessment of the total improvement score.

Thirteen patients with IIM, characterized by severe muscle or skin involvement and highly-refractory disease (median 6 prior IIM-specific therapies; range 3-9), were treated at DL1. Promising initial results showed that of the efficacy-evaluable patients, 64% of patients achieved a major response, and 27% achieved a moderate response, based on Myositis Response Criteria-Total Improvement Score (MRC-TIS).

All efficacy-evaluable patients experienced a rapid and substantial improvement in muscle strength; median increase in MMT-8 score change from baseline was 17% at three months (n=6) and 22% at six months (n=3).

Most TEAEs occurred within 90 days; the highest proportion of patients experiencing TEAEs occurred within the first 29 days following infusion. All reported CRS was transient and low grade. One patient experienced grade three ICANS that resolved completely within three days of onset.

Updated Data from Broader Immunology Portfolio

BMS is also presenting findings at ACR that support its immunology portfolio, including pivotal Phase 3 POETYK PsA-1 trial of Sotyktu (deucravacitinib) in adults with active psoriatic arthritis (abstract #LB20), as well as new findings from the Phase 2 PAISLEY-SLE and PAISLEY long-term extension (LTE) studies for moderate-to-severe SLE (abstract #LB10).

ACTioN at ACR

In addition to advancing research, BMS is continuing to champion interdisciplinary collaboration to progress the promise of cell therapy across the spectrum of autoimmune diseases. At ACR Convergence 2025, BMS is convening a forum of ACTioN (Autoimmunity Cell Therapy Network), a group of leading academics and physicians across rheumatology, hematology, neurology and immunology – working to advance cell therapy in autoimmune diseases. This event brings together different experts of the Network, with the goals of gathering insights and discussing strategies for investment into research that can accelerate the development of treatments for patients. Learn more about ACTioN here.

About the CD19 NEX-T^™ Breakfree-1 Study

The Phase 1 Breakfree-1 study is an open-label, multicenter study to evaluate the tolerability, preliminary efficacy and pharmacokinetics of BMS-986353 (CC-97540), a CD19-directed CAR T cell therapy, for severe, refractory autoimmune diseases (systemic lupus erythematosus, systemic sclerosis, idiopathic inflammatory myopathies and rheumatoid arthritis). CD19 NEX-T is an investigational CD19-targeted CAR T cell therapy that expresses the CD19 CAR used in Breyanzi (lisocabtagene maraleucel; liso-cel), FDA-approved in certain hematologic malignancies, and is manufactured using a next-generation process (NEX-T) that aims to optimize manufacturing processes to help CAR T cells recognize and fight specific pathogenic cells.

Trial patients across cohorts follow a similar process: apheresis to collect T cells, after which the cells are manufactured into CD19 CAR T cells at a specialized facility utilizing the optimized, five-day NEX-T manufacturing process. After undergoing lymphodepletion, patients receive a single infusion of CD19 NEX-T cell therapy.

Primary outcomes are safety measures assessed for up to two years, and secondary outcomes include efficacy measures of disease remission, activity, and organ-specific parameters at 24 weeks, as well as pharmacokinetic outcomes. This study will also determine the recommended Phase 2 dose based on the dose-limiting toxicities observed in the dose-escalation phase to optimize the benefit-risk of this therapy in patients. As we continue to progress Breakfree-1, recruitment for the Phase 2 Breakfree-SLE study is currently ongoing.

Bristol Myers Squibb: Unlocking the Full Potential of Cell Therapy

A pioneer in harnessing the immune system to fight cancer and an established leader in cell therapy, Bristol Myers Squibb is uniquely positioned to explore the potential of this technology across blood cancers and into new frontiers, including autoimmune disease.

Bristol Myers Squibb is currently the only company with two approved CAR T cell therapies with two distinct targets, available in major markets around the world. Our bold vision for the future is one in which hundreds of thousands of appropriate patients can be treated with cell therapy’s transformational potential.

The building blocks to realize this ambition—a promising and differentiated pipeline, extensive translational and clinical data sets, a deep bench of talent, and robust manufacturing capabilities—are in our cells. We are laser-focused on advancing the field of cell therapy toward a true revolution for patients. Learn more about the science behind cell therapy and ongoing progress at Bristol Myers Squibb here.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, X, YouTube, Facebook and Instagram.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that future study results may not be consistent with the results to date, that BMS-986353 may not achieve its primary study endpoints or receive regulatory approval for the indications described in this release in the currently anticipated timeline or at all, any marketing approvals, if granted, may have significant limitations on their use, and, if approved, whether BMS-986353 for such indications will be commercially successful. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2024, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

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Deciphera Presents 2-Year Efficacy and Safety Results from MOTION Phase 3 Study of ROMVIMZA™ (vimseltinib) in Patients with Tenosynovial Giant Cell Tumor (TGCT) at the European Society for Medical Oncology Congress 2025




Deciphera Presents 2-Year Efficacy and Safety Results from MOTION Phase 3 Study of ROMVIMZA™ (vimseltinib) in Patients with Tenosynovial Giant Cell Tumor (TGCT) at the European Society for Medical Oncology Congress 2025

– Vimseltinib demonstrated statistically significant and clinically meaningful benefit vs placebo in antitumor response –

OSAKA, Japan & WALTHAM, Mass.–(BUSINESS WIRE)–Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), today announced the two-year efficacy and safety results from its MOTION Phase 3 study of vimseltinib in patients with TGCT in cases where surgical removal of the tumor is not an option will be presented as a poster during the 2025 European Society for Medical Oncology Congress (ESMO), taking place October 17-21 in Berlin, Germany.

“These long-term Phase 3 MOTION results add to the established body of evidence supporting vimseltinib as a best-in-class treatment for TGCT,” said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. “TGCT often causes debilitating pain, stiffness and impaired mobility and these results demonstrate the durable benefit that vimseltinib can offer patients.”

Summary of Data and Findings from the 2-year results of the MOTION Phase 3 Study

Methods

The global Phase 3 MOTION study (NCT05059262) aims to evaluate the efficacy and safety of vimseltinib for the treatment of TGCT in cases where surgical removal of the tumor is not an option.

The study consists of two parts. In Part 1, eligible study participants were assigned to receive either vimseltinib or matching placebo for 24 weeks. Participants assigned to placebo in Part 1 had the option to receive vimseltinib for Part 2. Part 2 was a long-term treatment phase in which all participants received open-label vimseltinib. Patients received vimseltinib 30 mg twice weekly in all periods. Objective response rate (ORR) based on best overall response was assessed by independent radiological review (IRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) and per Tumor Volume Score (TVS). Duration of response (DOR) and safety were also evaluated.

Efficacy

In these two-year results from the MOTION Phase 3 trial, vimseltinib continued to demonstrate robust and durable antitumor efficacy with a manageable safety profile that was consistent with prior reports. These long-term results support vimseltinib as a treatment option for patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability, where it is approved. These results are reported with two years of follow-up in patients randomized to vimseltinib in Part 1 and crossed over from placebo to vimseltinib in Part 2. The data cutoff for this analysis was February 22, 2025.

In total, 118 patients received vimseltinib. At data cutoff, 51% (60/118) remained on treatment. With at least 2 years of follow-up, results demonstrate robust and durable antitumor activity with vimseltinib per RECIST v1.1 and per TVS, including in patients who crossed over to vimseltinib in Part 2.

• Of 83 patients randomized to vimseltinib in Part 1, 73 continued open-label treatment in Part 2. Median (range) treatment duration was 23.6 months (2 to 36).
• Of the 40 patients randomized to placebo in Part 1, 35 crossed over to vimseltinib in Part 2. Median treatment duration for this group was 19.1 months (1 to 30).
• ORR on study per RECIST v1.1 was 48% (40/83) for patients randomized to vimseltinib and 54% (19/35) for those who crossed over to vimseltinib. ORR on study per TVS was 81% (67/83) for patients randomized to vimseltinib and 71% (25/35) for those who crossed over to vimseltinib.
• The corresponding median DOR per RECIST v1.1 and per TVS was still not reached.

Safety

Vimseltinib continued to have a manageable safety profile that was consistent with prior reports with no new safety signals.

• Most treatment-emergent adverse events (TEAEs) were grade 1/2, and grade 3/4 TEAEs were similar between randomized vimseltinib and crossover groups.
• There were no new TEAEs in ≥15% of patients receiving vimseltinib and no new serious adverse events in more than one patient.
• Serum enzyme elevations were consistent with the known mechanism of action of CSF1R inhibition, and there was no evidence of cholestatic hepatotoxicity or drug-induced liver injury.

About Vimseltinib

Vimseltinib is an oral, switch-control tyrosine kinase inhibitor specifically designed to selectively and potently inhibit CSF1R. Vimseltinib has been developed using Deciphera’s proprietary switch-control kinase inhibitor platform. It has been approved in the United States for adult patients with symptomatic TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity, and in the European Union for adult patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability.

About Tenosynovial Giant Cell Tumor (TGCT)

TGCT is caused by a dysregulation in colony-stimulating factor 1 (CSF1) gene leading to overproduction of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion.¹ TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS). TGCT is a rare, locally aggressive neoplasm that can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity. Systemic treatment options are limited and new therapeutic options are needed.^1,2

About Deciphera Pharmaceuticals Inc.

Deciphera, a member of Ono Pharmaceutical Co., Ltd., is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer. Deciphera is leveraging its proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from Deciphera’s platform in clinical studies, QINLOCK® (ripretinib) is Deciphera’s switch-control kinase inhibitor approved in many countries including the European Union and the United States for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. ROMVIMZA™ (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity, and in the European Union for adult patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability. For more information, visit www.deciphera.com and follow us on LinkedIn and X (@Deciphera).

About Ono Pharmaceutical Co., Ltd

Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of “Dedicated to the Fight against Disease and Pain,” Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company’s website at https://www.ono-pharma.com/en.

Cautionary Note Regarding Forward-Looking Statements

In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice.

1. Gelderblom H, Bhadri V, Stacchiotti S, et al. Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024;403(10445):2709-2719. doi:10.1016/S0140-6736(24)00885-7

2. Stacchiotti S, Dürr HR, Schaefer IM, et al. Best clinical management of tenosynovial giant cell tumour (TGCT): A consensus paper from the community of experts. Cancer Treat Rev. 2023;112:102491. doi:10.1016/j.ctrv.2022.102491

Contacts

Ono Pharmaceutical Co., Ltd.
Media:
media_inquiries@ono-pharma.com

Deciphera Pharmaceuticals, Inc.
Media:
David Rosen

Argot Partners

david.rosen@argotpartners.com
646-461-6387

KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Demonstrates Durable 5-Year Survival Benefit Versus Chemotherapy for Patients With Advanced Endometrial Carcinoma Following One Prior Platinum-Based Regimen




KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Demonstrates Durable 5-Year Survival Benefit Versus Chemotherapy for Patients With Advanced Endometrial Carcinoma Following One Prior Platinum-Based Regimen

At five years, KEYTRUDA plus LENVIMA showed a 16.7% overall survival (OS) rate for these patients with mismatch repair proficient (pMMR) advanced endometrial carcinoma versus 7.3% for chemotherapy alone in the pivotal Phase 3 KEYNOTE-775/Study 309 trial

Five-year OS results in the pMMR subgroup were consistent with the all-comers study population, which demonstrated an OS rate of 19.9% for KEYTRUDA plus LENVIMA versus 7.7% for chemotherapy

RAHWAY, N.J. & NUTLEY, N.J.–(BUSINESS WIRE)–$MRK #MRK–Merck (NYSE: MRK), known as MSD outside of the United States and Canada, and Eisai today announced long-term follow-up data continued to show durable benefit of KEYTRUDA^® (pembrolizumab), Merck’s anti-PD-1 therapy, plus LENVIMA^® (lenvatinib), the orally available multiple receptor tyrosine kinase inhibitor (TKI) discovered by Eisai, compared to chemotherapy for patients with advanced endometrial carcinoma following at least one prior platinum-based regimen in any setting. The findings, based on five years of follow-up from the Phase 3 KEYNOTE-775/Study 309 trial evaluating KEYTRUDA plus LENVIMA versus chemotherapy (treatment of physician’s choice of doxorubicin or paclitaxel), for these patients with advanced endometrial carcinoma are being presented during a poster session at the European Society for Medical Oncology (ESMO) Congress 2025 in Berlin, Germany (Presentation #1119P).

In the trial, a total of 827 patients (697 whose cancer was mismatch repair proficient [pMMR] and 130 whose cancer was mismatch repair deficient [dMMR]) were randomized to receive KEYTRUDA plus LENVIMA (n=411) or chemotherapy (n=416). The trial’s primary endpoints, overall survival (OS) and progression-free survival (PFS), were evaluated in patients with pMMR disease and in the intent-to-treat population (also known as the all-comers population), which included all randomized patients (both pMMR and dMMR).

In the pMMR subgroup, after a median follow-up of 68.8 months (range, 60.8–79.0 months for KEYTRUDA plus LENVIMA; range, 60.9–80.0 for chemotherapy), the five-year OS rate was 16.7% for KEYTRUDA plus LENVIMA versus 7.3% for chemotherapy alone. Median OS was 18.0 months (95% CI, 14.9-20.5) for KEYTRUDA plus LENVIMA versus 12.2 months (95% CI, 11.0-14.1) for chemotherapy alone (HR 0.70; 95% CI, 0.60-0.83). Five-year OS results in the pMMR subgroup were consistent with the all-comers population, which demonstrated an OS rate of 19.9% for KEYTRUDA plus LENVIMA versus 7.7% for chemotherapy; median OS was 18.7 months (95% CI, 15.6-21.3) for KEYTRUDA plus LENVIMA versus 11.9 months (95% CI, 10.6-13.3) for chemotherapy (HR 0.66; 95% CI, 0.57-0.77).

The long-term OS data were consistent with the primary analysis presented at the Society of Gynecologic Oncology (SGO) 2021 Annual Meeting and published in the New England Journal of Medicine. In the primary analysis, the median OS was 17.4 months (95% CI, 14.2-19.9) for KEYTRUDA plus LENVIMA versus 12.0 months (95% CI, 10.8-13.3) for chemotherapy in the pMMR subgroup, and 18.3 months (95% CI, 15.2-20.5) versus 11.4 months (95% CI, 10.5-12.9) in the all-comers population. In the five-year analysis, there were no new safety signals and the safety profile of KEYTRUDA plus LENVIMA was consistent with previously reported data on the combination.

“Endometrial carcinoma is difficult-to-treat in the recurrent or advanced stage, especially when tumors are mismatch repair proficient and therefore harder to target with immunotherapy alone,” said Dr. Vicky Makker, Principal Investigator and Gynecologic Medical Oncologist, Memorial Sloan Kettering Cancer Center. “Five-year follow-up data from the KEYNOTE-775/Study 309 trial show sustained survival benefit in patients treated with pembrolizumab plus lenvatinib and underscore the role of this combination as an effective treatment option for patients with advanced endometrial carcinoma who need further treatment after receiving prior platinum-based therapy.”

“Recent advances have led to steady improvement in outcomes for patients with advanced endometrial carcinoma,” said Dr. Gregory Lubiniecki, Vice President, Global Clinical Development, Merck Research Laboratories. “These five-year data highlight the durable survival benefit of KEYTRUDA plus LENVIMA in patients with advanced endometrial carcinoma who have received prior platinum-based therapy and are the result of our ongoing commitment to delivering impactful treatment options for people affected by women’s cancers.”

“The five-year results from KEYNOTE-775/Study 309 represent the longest reported follow-up for a trial evaluating an immunotherapy plus tyrosine kinase inhibitor combination in advanced endometrial carcinoma,” said Dr. Corina Dutcus, Senior Vice President, Oncology Global Clinical Development Lead at Eisai Inc. “These findings demonstrate the continued overall survival benefit observed with KEYTRUDA plus LENVIMA, further supporting the combination’s therapeutic value for patients facing this disease. We are deeply grateful to the patients, their families, and the dedicated investigators whose participation and commitment made this research possible.”

Treatment-related adverse events (TRAEs) occurred in 97.3% of patients receiving KEYTRUDA plus LENVIMA versus 93.8% of patients receiving chemotherapy and led to the discontinuation of KEYTRUDA and/or LENVIMA in 40.1% of patients (16.0% discontinued both drugs) versus the discontinuation of chemotherapy in 8.0% of patients. The most common adverse events (≥ 20%) in the KEYTRUDA plus LENVIMA group were hypertension (61.8%), hypothyroidism (55.7%), diarrhea (43.3%), nausea (40.1%), decreased appetite (37.9%), fatigue (28.8%), proteinuria (27.6%), vomiting (24.4%), arthralgia (23.9%), decreased weight (22.7%) and palmar-plantar erythrodysesthesia syndrome (20.7%).

Based on the primary analysis results in 2021 from the Phase 3 KEYNOTE-775/Study 309 trial, KEYTRUDA plus LENVIMA is approved in the U.S. for patients with advanced endometrial carcinoma that is pMMR or not microsatellite instability-high (MSI-H), as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation. KEYTRUDA plus LENVIMA is also approved in the European Union (EU) and Japan for certain patients with advanced or recurrent endometrial carcinoma regardless of mismatch repair status. Lenvatinib is approved as KISPLYX for advanced renal cell carcinoma in the EU.

Dr. Makker has provided consulting and advisory services for Merck and Eisai.

Study design and additional data from KEYNOTE-775/Study 309

KEYNOTE-775/Study 309 (ClinicalTrials.gov, NCT03517449) is a Phase 3, multicenter, open-label, randomized, active-controlled study evaluating KEYTRUDA plus LENVIMA for the treatment of patients with advanced endometrial carcinoma who had been previously treated with at least one prior platinum-based chemotherapy regimen in any setting, including in the neoadjuvant and adjuvant settings. Participants may have received up to two platinum-containing therapies in total, as long as one was given in the neoadjuvant or adjuvant treatment setting. The study excluded patients with endometrial sarcoma, carcinosarcoma, pre-existing Grade ≥3 fistula, uncontrolled blood pressure (>150/90 mmHg), significant cardiovascular impairment or event within the last 12 months or patients who had active autoimmune disease or a medical condition that required immunosuppression. The primary efficacy outcome measures were OS and PFS as assessed by blinded independent central review (BICR) according to Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1). Secondary efficacy outcome measures included objective response rate (ORR) as assessed by BICR and safety. The study randomized 827 patients 1:1 to receive:

• KEYTRUDA (200 mg intravenously every three weeks) plus LENVIMA (20 mg orally once daily); or
• Investigator’s choice, consisting of either doxorubicin (60 mg/m² every three weeks) or paclitaxel (80 mg/m² given weekly, three weeks on/one week off).

Treatment with KEYTRUDA plus LENVIMA continued until RECIST v1.1-defined progression of disease as verified by BICR, unacceptable toxicity or, for KEYTRUDA, a maximum of 35 cycles. Administration of KEYTRUDA plus LENVIMA was permitted beyond RECIST v1.1-defined disease progression if the treating investigator considered the patient to be deriving clinical benefit and the treatment was tolerated.

In the pMMR subgroup, KEYTRUDA plus LENVIMA demonstrated a five-year PFS rate of 6.3% versus 2.1% for chemotherapy alone. Median PFS was 6.7 months (95% CI, 5.6-7.4) for KEYTRUDA plus LENVIMA versus 3.8 months (95% CI, 3.6-5.0) for chemotherapy alone. At five years, ORR was 32.4% (95% CI, 27.5-37.6) for KEYTRUDA plus LENVIMA compared with 14.8% (95% CI, 11.3-19.0) for chemotherapy.

In the all-comers population, KEYTRUDA plus LENVIMA demonstrated a five-year PFS rate of 9.8% versus 3.2% for chemotherapy alone. Median PFS was 7.3 months (95% CI, 5.7-7.6) for KEYTRUDA plus LENVIMA versus 3.8 months (95% CI, 3.6-4.2) for chemotherapy alone. At five years, ORR was 33.8% (95% CI, 29.3-38.6) for KEYTRUDA plus LENVIMA compared with 14.4% (95% CI, 11.2-18.2) for chemotherapy.

The long-term PFS data were also consistent with the primary analysis, in which the median PFS was 6.6 months (95% CI, 5.6-7.4) for KEYTRUDA plus LENVIMA versus 3.8 months (95% CI, 3.6-5.0) for chemotherapy in the pMMR subgroup, and 7.2 months (95% CI, 5.7-7.6) versus 3.8 months (95% CI, 3.6-4.2) in the all-comers population.

In the all-comers population, 44.8% of patients treated with KEYTRUDA plus LENVIMA and 51.2% of patients treated with chemotherapy used subsequent systemic anticancer therapy (compared with 28.0% and 48.1%, respectively, at the primary analysis). In the chemotherapy group, 10.1% of patients crossed over to receive KEYTRUDA plus LENVIMA. At the data cut-off (Feb. 26, 2025), 86 patients were alive after treatment with KEYTRUDA plus LENVIMA, compared to 53 patients treated with chemotherapy.

While the trial was not powered to compare KEYTRUDA plus LENVIMA with chemotherapy in the dMMR subgroup, a clinically meaningful improvement was observed across efficacy endpoints. The five-year OS rate was 36.5% for KEYTRUDA plus LENVIMA versus 9.8% for chemotherapy alone. Median OS was 31.9 months (95% CI, 15.6-47.7) for KEYTRUDA plus LENVIMA versus 8.6 months (95% CI, 5.5-13.4) for chemotherapy alone. KEYTRUDA plus LENVIMA demonstrated a five-year PFS rate of 26.4% versus 10.8% for chemotherapy. Median PFS was 14.8 months (95% CI, 5.6-31.9) for KEYTRUDA plus LENVIMA versus 3.7 months (95% CI, 3.1-4.4) for chemotherapy alone. The ORR at five years was 41.5% (95% CI, 29.4-54.4) for KEYTRUDA plus LENVIMA compared with 12.3% (95% CI, 5.5-22.8) for chemotherapy.

About endometrial carcinoma

Endometrial carcinoma begins in the inner lining of the uterus, which is known as the endometrium, and is the most common type of cancer in the uterus. More than 90% of uterine body cancers occur in the endometrium. In the U.S., it is estimated there will be approximately 69,120 patients diagnosed with uterine body cancer and approximately 13,860 patient deaths from the disease in 2025. Globally, endometrial cancer is the sixth most common cancer in women and the 15th most common cancer overall. Following primary treatment, patients face a risk of their cancer returning, often as distant metastasis, which is associated with poorer outcomes.

About KEYTRUDA^® (pembrolizumab) injection, 100 mg

KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA^® (pembrolizumab) Indications in the U.S.

Endometrial Carcinoma

KEYTRUDA, in combination with carboplatin and paclitaxel, followed by KEYTRUDA as a single agent, is indicated for the treatment of adult patients with primary advanced or recurrent endometrial carcinoma.

KEYTRUDA, in combination with lenvatinib, is indicated for the treatment of adult patients with advanced endometrial carcinoma that is mismatch repair proficient (pMMR) or not MSI-H as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting are not candidates for curative surgery or radiation.

KEYTRUDA, as a single agent, is indicated for the treatment of adult patients with advanced endometrial carcinoma that is MSI-H or dMMR, as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.

See additional selected KEYTRUDA indications in the U.S. after the Selected Important Safety Information.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or the programmed death ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of anti–PD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. For patients with TNBC treated with KEYTRUDA in the neoadjuvant setting, monitor blood cortisol at baseline, prior to surgery, and as clinically indicated. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Pneumonitis occurred in 7% (41/580) of adult patients with resected NSCLC who received KEYTRUDA as a single agent for adjuvant treatment of NSCLC, including fatal (0.2%), Grade 4 (0.3%), and Grade 3 (1%) adverse reactions. Patients received high-dose corticosteroids for a median duration of 10 days (range: 1 day to 2.3 months). Pneumonitis led to discontinuation of KEYTRUDA in 26 (4.5%) of patients. Of the patients who developed pneumonitis, 54% interrupted KEYTRUDA, 63% discontinued KEYTRUDA, and 71% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT ≥3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT ≥3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT ≥3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.

Contacts

Media Contacts:

Merck:

Julie Cunningham

(617) 519-6264

John Infanti

(609) 500-4714

Eisai:

Michele Randazzo

(201) 248-2228

Investor Contacts:

Merck:

Peter Dannenbaum

(732) 594-1579

Steven Graziano

(732) 594-1583

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Genmab Announces New Data Demonstrating Investigational Rinatabart Sesutecan (Rina-S®) Achieved Anti-Tumor Activity in Heavily Pretreated Patients with Advanced Endometrial Cancer




Genmab Announces New Data Demonstrating Investigational Rinatabart Sesutecan (Rina-S®) Achieved Anti-Tumor Activity in Heavily Pretreated Patients with Advanced Endometrial Cancer

• Updated data from the Phase 1/2 RAINFOL™-01 trial showed rinatabart sesutecan (Rina-S^®) 100 mg/m² demonstrated 50% confirmed objective response rate (ORR), including two complete responses (CR), regardless of FRα expression
• A Phase 3 trial in endometrial cancer is underway
• U.S. FDA recently granted Breakthrough Therapy Designation to Rina-S for advanced endometrial cancer

COPENHAGEN, Denmark–(BUSINESS WIRE)–Genmab A/S (Nasdaq: GMAB) announced today updated data from cohort B2 of the Phase 1/2 RAINFOL™-01 trial evaluating rinatabart sesutecan (Rina-S^®), an investigational folate receptor alpha (FRα)-targeted, TOPO1-inhibitor antibody-drug conjugate (ADC). The study showed that at a median study follow-up of one year, treatment with Rina-S 100 mg/m² every 3 weeks (Q3W) resulted in a 50.0% confirmed objective response rate (ORR), including two complete responses (CR), in heavily pretreated patients with advanced endometrial cancer (EC) who had progressed following platinum-based chemotherapy and an immune checkpoint inhibitor. Additionally, at a median study follow-up of one year, 63.6% of responders (including CRs) in the 100 mg/m² cohort maintained their responses and remain on treatment. The responses were observed regardless of FRα expression levels. The updated results were presented at the European Society for Medical Oncology (ESMO) Congress in Berlin, Germany.

Continued evaluation of single-agent Rina-S 100 mg/m² in patients with advanced EC is ongoing in the Phase 2 RAINFOL-01 trial (NCT05579366) and the Phase 3 RAINFOL-03 trial (NCT07166094).

“Women with advanced endometrial cancer are often facing a difficult path, while doctors are confronted with not having enough treatment options,” said Noelle Cloven, M.D., Texas Oncology Fort Worth, Sarah Cannon Research Institute, and study investigator. “That’s why these data signals with Rina-S in the updated Phase 1/2 RAINFOL-01 data are encouraging – they point to the possibility of providing more choices for patients in the future.”

The B2 cohort of the Phase 1/2 RAINFOL-01 study (NCT05579366) is a dose expansion cohort evaluating the efficacy and safety of Rina-S in patients with advanced or recurrent endometrial cancer. In the study, 64 patients with heavily pretreated advanced or recurrent endometrial cancer whose disease had progressed on or after an anti-PD-(L)1 and platinum-based chemotherapy were enrolled and treated with Rina-S. Patients were administered either 100 mg/m² (n=22) (selected dose for Phase 3 clinical trial) or 120 mg/m² (n=42) of Rina-S. In the 100 mg/m² cohort, the confirmed ORR was 50.0%, including two CRs. Anti-tumor activity was also observed in patients treated with Rina-S 120 mg/m² Q3W, which resulted in 44.1% confirmed ORR and one CR. Study participants were previously treated with a median of three lines of therapy (range 1-8). Earlier results from this cohort were previously presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting.

Common treatment emergent adverse events (TEAEs; all grades) consisted primarily of cytopenias and low-grade gastrointestinal (GI) events. To date, there have been no signals of ocular toxicities, neuropathy, or interstitial lung disease (ILD) observed in Rina-S clinical trials consistent with prior reports. Serious TEAEs (Grade 3 or higher), occurred in 36.4% and 52.4% of patients treated with Rina-S 100 mg/m² and 120 mg/m², respectively. Hematologic adverse events did not require significant dose reduction and were associated with low rates of treatment discontinuation.

“With this updated data, we are seeing additional momentum behind the possibilities of Rina-S,” said Tahi Ahmadi, M.D., Executive Vice President and Chief Medical Officer, Head of Experimental Medicines at Genmab. “As a wholly owned, novel antibody-drug conjugate, Rina-S reflects Genmab’s vision to accelerate our innovative, late-stage pipeline that has the potential to redefine possibilities for patients with certain gynecologic cancers.”

Rina-S is advancing through late-stage development supported by a growing portfolio of clinical trials, including the ongoing Phase 1/2 RAINFOL-01 trial (NCT05579366), the Phase 3 RAINFOL-03 trial (NCT07166094) in patients with endometrial cancer now underway and the Phase 3 RAINFOL-02 trial (NCT06619236) in patients with platinum resistant ovarian cancer (PROC). The U.S. Food and Drug Administration (FDA) recently granted Breakthrough Therapy Designation (BTD) to Rina-S for the treatment of adult patients with recurrent or progressive EC who have disease progression on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy.

About the RAINFOL^TM -01 Trial

RAINFOL™-01 (NCT05579366) is an open-label, multicenter Phase 1/2 study, designed to evaluate the safety and efficacy of rinatabart sesutecan (Rina-S) Q3W at various doses in solid tumors that are known to express FRα. The study consists of multiple parts including Part A dose escalation; Part B tumor-specific monotherapy dose-expansion cohorts; Part C platinum-resistant ovarian cancer (PROC) cohort; Part D combination therapy cohorts; Part F a monotherapy endometrial cancer (EC) cohort.

About Endometrial Cancer

Endometrial cancer (EC) starts in the lining of the uterus, known as the endometriumⁱ and ranks as the second most prevalent gynecologic cancer globally, with increasing incidence and mortality rates^ii,iii. Patients with advanced or recurrent EC have a relatively poor prognosis and treatment options are limited for those patients who have progressed following treatment with chemotherapy and immune checkpoint inhibitor. FRα is overexpressed on multiple tumors, including EC, making it a promising therapeutic target. Anti-tumor activity with Rina-S was observed across a broad range of FRα expression, and there are currently no approved FRα-directed therapies approved for the treatment of endometrial cancer.

About Rinatabart Sesutecan (Rina-S; GEN1184)

Rinatabart sesutecan (Rina-S; GEN1184) is an investigational ADC. It is composed of a novel human monoclonal antibody directed at folate receptor α (FRα), a novel hydrophilic protease-cleavable linker, and exatecan, a topoisomerase I inhibitor payload. The clinical trial program for Rina-S continues to expand including ovarian, endometrial and other cancers of unmet need.

The safety and efficacy of rinatabart sesutecan has not been established. Please visit www.clinicaltrials.gov for more information.

About Genmab

Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For more than 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab’s vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO) antibody medicines^®.

Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit Genmab.com and follow us on LinkedIn and X.

This Media Release contains forward looking statements. The words “believe,” “expect,” “anticipate,” “intend” and “plan” and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with preclinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmab’s most recent financial reports, which are available on www.genmab.com and the risk factors included in Genmab’s most recent Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Media Release nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab^®; the Y-shaped Genmab logo^®; Genmab in combination with the Y-shaped Genmab logo^®; HuMax^®; DuoBody^®; HexaBody^®; DuoHexaBody^®, HexElect^®, KYSO^® and RAINFOL™; Rina-S is a trademark of ProfoundBio, US, Co. and Genmab (Suzhou) Co., Ltd.

ⁱ Mayo Clinic. Endometrial Cancer. https://www.mayoclinic.org/diseases-conditions/endometrial-cancer/symptoms-causes/syc-20352461.

ⁱⁱ Ferlay J, Ervik M, Lam F, et al. Global cancer observatory: Cancer today (version 1.1). International Agency for Research on Cancer. 05/28/2024 (https://gco.iarc.who.int/today).

ⁱⁱⁱ Concin N, Matias-Guiu X, Vergote I, et al. ESGO/ESTRO/ESP guidelines for the management of patients with endometrial carcinoma. International journal of gynecological cancer : official journal of the International Gynecological Cancer Society 2021;31(1):12-39. (In eng). DOI: 10.1136/ijgc-2020-002230.

Contacts

David Freundel, Senior Director, Global Communications & Corporate Affairs

T: +1 609 613 0504; E: dafr@genmab.com

Andrew Carlsen, Vice President, Head of Investor Relations

T: +45 3377 9558; E: acn@genmab.com