Eli Lilly & Co.

Data from the QUASAR long-term extension study demonstrate more than 70% of patients were in clinical remission and more than 40% of patients were in endoscopic remission at Week 92 SAN DIEGO, Calif., May 5, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced new data from the TREMFYA® (guselkumab) Phase 3 QUASAR long-term extension (LTE) study in adults with moderately-to-severely active ulcerative colitis (UC). These data are among 24 abstracts highlighting the Company's research being presented at Digestive Disease Week (DDW) 2025. Data from the QUASAR LTE study demonstrate patients treated with TREMFYA® sustained clinical and endoscopic efficacy at Week 92: a, 1 72% of patients were in clinical remissionb with 99% of those patients remaining corticosteroid free for 8 or more weeks through Week 92. 43% of patients were in endoscopic remission.c Among patients achieving endoscopic improvementd at Week 44, 84% maintained endoscopic improvement through Week 92. Patients treated with TREMFYA® sustained clinical and endoscopic remission regardless of prior biologic and/or JAK inhibitor treatment history. "People living with ulcerative colitis seek treatments that both address the challenging symptoms of the disease and provide durable results," said Gary R. Lichtenstein, Vice Chief, Division of Gastroenterology and Hepatology, Development and Philanthropy at the University of Pennsylvania.e "These new data show TREMFYA delivers long-term, sustained clinical and endoscopic remission, marking important progress in UC care." Safety data were consistent with the well-established safety profile of TREMFYA® in inflammatory bowel disease (IBD) with no new safety concerns identified. "With these findings, TREMFYA shows the powerful impact it can have in achieving longer term remission in patients," said Esi Lamousé-Smith, MD, PhD, Vice President, Gastroenterology Disease Area Lead, Immunology, Johnson & Johnson Innovative Medicine. "This is a significant step forward in our mission to reshape the standard of care in inflammatory bowel disease." TREMFYA® is the first and only approved, dual-acting monoclonal antibody that blocks IL-23 while also binding to CD64, a receptor on cells that produce IL-23. IL-23 is a cytokine secreted by activated monocyte/macrophages and dendritic cells that is known to be a driver of immune-mediated diseases including UC.2,3,4,5,6 TREMFYA® received FDA approval in September 2024 for the treatment of adult patients with moderately to severely active UC and is currently administered via an IV induction regimen, followed by a SC maintenance regimen. In November 2024, a supplemental Biologics License Application (sBLA) was submitted to the FDA seeking approval of a SC induction regimen of TREMFYA® for the treatment of adults with moderately to severely active UC. TREMFYA® was also approved by the FDA in March 2025 for SC and IV induction options for the treatment of adults with moderately to severely active Crohn's disease (CD). For a full list of all data being presented at DDW visit:   Editor's Notes: a. Nonresponder imputation results. Data were analyzed using 2 methods: 'nonresponder imputation' (NRI) accounting for patients with treatment failure or missing data, and 'as observed'. NRI results were consistent with as observed. b. Clinical remission was defined as a Mayo stool frequency subscore of 0 or 1 and not increased from induction baseline, a Mayo rectal bleeding subscore of 0, and a Mayo endoscopic subscore (MES) of 0 or 1. c. Endoscopic remission (normalization) was defined as a MES of 0. d. Endoscopic improvement was defined as a MES of 0 or 1. e. Dr. Lichtenstein has received honorarium for his role as a consultant to Johnson & Johnson. He has not been compensated for any media work. ABOUT THE QUASAR PROGRAM (NCT04033445) QUASAR is a randomized, double-blind, placebo-controlled, parallel group, multicenter, Phase 2b/3 program designed to evaluate the efficacy and safety of TREMFYA® in adults with moderately to severely active ulcerative colitis who had an inadequate response or intolerance to conventional therapy (e.g., thiopurines or corticosteroids), prior biologics (TNF antagonists or vedolizumab) and/or JAK inhibitors (tofacitinib). QUASAR included a Phase 2b dose-ranging induction study, a confirmatory Phase 3 induction study, and a Phase 3 randomized withdrawal maintenance study. In the Phase 3 induction study, patients received either TREMFYA® 200 mg or placebo by IV infusion at Weeks 0, 4, and 8. In the Phase 3 maintenance study, patients received a SC maintenance regimen of either TREMFYA® 200 mg q4w, TREMFYA® 100 mg q8w, or placebo.7 The ongoing long-term extension study provides an additional 4 years of treatment. Efficacy, safety, pharmacokinetics, immunogenicity, and biomarkers are assessed at specified time points. ABOUT ULCERATIVE COLITIS Ulcerative colitis (UC) is a chronic disease of the large intestine, also known as the colon, in which the lining of the colon becomes inflamed and develops tiny open sores, or ulcers, that produce pus and mucus. It is the result of the immune system's overactive response. Symptoms vary but may typically include loose and more urgent bowel movements, rectal bleeding or bloody stool, persistent diarrhea, abdominal pain, loss of appetite, weight loss, and fatigue.8 ABOUT TREMFYA® (guselkumab) Developed by Johnson & Johnson, TREMFYA® is the first approved fully-human, dual-acting monoclonal antibody designed to neutralize inflammation at the cellular source by blocking IL-23 and binding to CD64 (a receptor on cells that produce IL-23). Findings for dual-acting are limited to in vitro studies that demonstrate guselkumab binds to CD64, which is expressed on the surface of IL-23 producing cells in an inflammatory monocyte model. The clinical significance of this finding is not known. TREMFYA® is a prescription medicine approved in the U.S. to treat: adults with moderate to severe plaque psoriasis who may benefit from taking injections or pills (systemic therapy) or phototherapy (treatment using ultraviolet or UV light). adults with active psoriatic arthritis. adults with moderately to severely active ulcerative colitis. adults with moderately to severely active Crohn's disease.4 TREMFYA® is approved in Europe, Canada, Japan, and a number of other countries for the treatment of adults with moderate-to-severe plaque psoriasis and for the treatment of adults with active psoriatic arthritis. In addition, TREMFYA ® is approved in Europe, Japan and Brazil for the treatment of adult patients with moderately to severely active UC and in Brazil and China for the treatment of adults with moderately to severely active CD. Johnson & Johnson maintains exclusive worldwide marketing rights to TREMFYA®. For more information, visit: . IMPORTANT SAFETY INFORMATION What is the most important information I should know about TREMFYA®? TREMFYA® is a prescription medicine that may cause serious side effects, including: Serious Allergic Reactions. Stop using TREMFYA® and get emergency medical help right away if you develop any of the following symptoms of a serious allergic reaction: Infections. TREMFYA® may lower the ability of your immune system to fight infections and may increase your risk of infections. Your healthcare provider should check you for infections and tuberculosis (TB) before starting treatment with TREMFYA® and may treat you for TB before you begin treatment with TREMFYA® if you have a history of TB or have active TB. Your healthcare provider should watch you closely for signs and symptoms of TB during and after treatment with TREMFYA®. Tell your healthcare provider right away if you have an infection or have symptoms of an infection, including: Liver problems. With the treatment of Crohn's disease or ulcerative colitis, your healthcare provider will do blood tests to check your liver before and during treatment with TREMFYA®. Your healthcare provider may stop treatment with TREMFYA® if you develop liver problems. Tell your healthcare provider right away if you notice any of the following symptoms: Do not use TREMFYA® if you have had a serious allergic reaction to guselkumab or any of the ingredients in TREMFYA®. Before using TREMFYA®, tell your healthcare provider about all of your medical conditions, including if you: have any of the conditions or symptoms listed in the section "What is the most important information I should know about TREMFYA®?" have an infection that does not go away or that keeps coming back. have TB or have been in close contact with someone with TB. have recently received or are scheduled to receive an immunization (vaccine). You should avoid receiving live vaccines during treatment with TREMFYA®. are pregnant or plan to become pregnant. It is not known if TREMFYA® can harm your unborn baby. Pregnancy Registry: If you become pregnant during treatment with TREMFYA®, talk to your healthcare provider about registering in the pregnancy exposure registry for TREMFYA®. You can enroll by visiting , by calling 1-877-311-8972, or emailing [email protected]. The purpose of this registry is to collect information about the safety of TREMFYA® during pregnancy. are breastfeeding or plan to breastfeed. It is not known if TREMFYA® passes into your breast milk. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What are the possible side effects of TREMFYA®? TREMFYA® may cause serious side effects. See "What is the most important information I should know about TREMFYA®?" The most common side effects of TREMFYA® include: respiratory tract infections, headache, injection site reactions, joint pain (arthralgia), diarrhea, stomach flu (gastroenteritis), fungal skin infections, herpes simplex infections, stomach pain, and bronchitis. These are not all the possible side effects of TREMFYA®. Call your doctor for medical advice about side effects. Use TREMFYA® exactly as your healthcare provider tells you to use it. Please read the full Prescribing Information, including Medication Guide, for TREMFYA® and discuss any questions that you have with your doctor. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Dosage Forms and Strengths: TREMFYA® is available as 100 mg/mL and 200 mg/2mL for subcutaneous injection and as a 200 mg/20 mL (10 mg/mL) single dose vial for intravenous infusion. ABOUT JOHNSON & JOHNSON At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.   Learn more at or at Follow us at @JNJInnovMed. Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies.  Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding TREMFYA®. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. References: 1 Lichtenstein GR, et al. Efficacy And Safety Of Guselkumab For Ulcerative Colitis Through Week 92 Of The Quasar Long-Term Extension Study. Poster presentation (#4241842) at Digestive Disease Week 2025. May 2025. 2 Atreya R, Abreu MT, Krueger JG, et al. Guselkumab, an IL-23p19 subunit-specific monoclonal antibody, binds CD64+ myeloid cells and potentially neutralizes IL-23 produced from the same cells. Poster presented at: 18th Congress of the European Crohn's and Colitis Organization (ECCO); March 1-4, 2023; Copenhagen, Denmark. Poster P504 3 Kreuger JG, Eyerich K, Kuchroo VK. Il-23 past, present, and future: a roadmap to advancing IL-23 science and therapy. Front Immunol. 2024; 15:1331217. doi:10.3389/fimmu.2024.1331217. 4 TREMFYA® [Prescribing Information]. Horsham, PA: Janssen Biotech, Inc. 5 Skyrizi® [Prescribing Information]. North Chicago, IL: AbbVie, Inc. 6 Omvoh™ [Prescribing Information]. Indianapolis, IN: Eli Lilly and Company. 7 National Institutes of Health: Clinicaltrials.gov. A Study of Guselkumab in Participants With Moderately to Severely Active Ulcerative Colitis (QUASAR). Identifier: NCT04033445. . Accessed March 2025. 8 Crohn's & Colitis Foundation. What is ulcerative colitis? Available at: . Accessed March 2025 SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

TREMFYA® subcutaneous induction demonstrates significant rates of clinical remission and endoscopic improvement at Week 24 in ulcerative colitis Findings build on recent FDA-approval of both routes of administration for induction therapy with TREMFYA® in Crohn's disease SAN DIEGO, May 5, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced new data from the Phase 3 ASTRO study evaluating TREMFYA® (guselkumab) subcutaneous (SC) induction therapy in adults with moderately to severely active ulcerative colitis (UC). The ASTRO Week 24 data build on the Week 12 SC induction data that showed statistically significant and clinically meaningful improvements compared to placebo across all clinical and endoscopic measures consistent with the U.S. Food and Drug Administration (FDA)-approved intravenous (IV) induction regimen evaluated in this population, in the Phase 3 QUASAR study.1,2 TREMFYA® is the first and only IL-23 inhibitor to demonstrate robust results with a fully SC regimen. These findings are among 24 abstracts highlighting the Company's research being presented at Digestive Disease Week (DDW) 2025. Data at Week 24 show patients treated with TREMFYA® 400 mg SC induction followed by SC maintenance dose regimens of either 100 mg every eight weeks (q8w) or 200 mg every four weeks (q4w) demonstrated statistically significant and clinically meaningful improvements across all clinical and endoscopic measures compared with patients receiving placebo.1 "Data from the ASTRO study demonstrate that subcutaneous induction treatment with TREMFYA provides clinically meaningful remission in patients with ulcerative colitis, similar to the effects seen with intravenous induction," said Millie Long, M.D., MPH, Professor of Gastroenterology and Hepatology at the University of North Carolina at Chapel Hill and study investigator.e "The availability of both subcutaneous and intravenous induction options would offer physicians and patients greater flexibility in their treatment approach." Furthermore, at Week 24, in prespecified analyses of subpopulations defined by prior advanced therapy treatment status, TREMFYA® demonstrated statistically significant results across endpoints in both biologic and JAK inhibitor-naïve and biologic and JAK inhibitor-refractory patients. Safety data from the ASTRO study were consistent with the well-established safety profile of TREMFYA®.1 "These results highlight the potential of TREMFYA to redefine ulcerative colitis care with a fully subcutaneous induction and maintenance regimen that offers a convenient option with meaningful clinical and endoscopic improvements," said Esi Lamousé-Smith, MD, PhD, Vice President, Gastroenterology Disease Area Lead, Immunology, Johnson & Johnson Innovative Medicine. "Our goal is to reshape UC care and empower prescribers with a differentiated and effective treatment that offers the option of patient self-administration from day one." TREMFYA® is the first and only approved, dual-acting monoclonal antibody that blocks IL-23 while also binding to CD64, a receptor on cells that produce IL-23. IL-23 is a cytokine secreted by activated monocyte/macrophages and dendritic cells that is known to be a driver of immune-mediated diseases including UC.3,4,5,6,7 TREMFYA® received FDA approval in September 2024 for the treatment of adult patients with moderately to severely active UC and is currently administered via an IV induction regimen, followed by a SC maintenance regimen. In November 2024, a supplemental Biologics License Application (sBLA) was submitted to the FDA seeking approval of a SC induction regimen of TREMFYA® for the treatment of adults with moderately to severely active UC. TREMFYA® was also approved by the FDA in March 2025 for SC and IV induction options for the treatment of adults with moderately to severely active Crohn's disease (CD). For a full list of all data being presented at DDW visit: Editor's Notes: a. Clinical remission was defined as a Mayo stool frequency subscore of 0 or 1 and not increased from baseline, a Mayo rectal bleeding subscore of 0, and a Mayo endoscopic subscore of 0, or 1 with no friability present on the endoscopy. b. Symptomatic remission per Mayo score is defined as a stool frequency subscore of 0 (normal number of stools) or 1 (1 to 2 stools more than normal) and a rectal bleeding subscore of 0 (no blood seen). c. Endoscopic improvement was defined as an endoscopy subscore of 0 or 1 with no friability present on the endoscopy. d. Clinical response is defined as decrease from baseline in the modified Mayo score by greater than or equal to (>=) 30 percent (%) and >=2 points, with either a >=1-point decrease from baseline in the rectal bleeding subscore or a rectal bleeding subscore of 0 or 1. e. Dr. Long is a paid consultant for Johnson & Johnson. She has not been compensated for any media work. ABOUT THE ASTRO STUDY (NCT05528510) ASTRO is a randomized, double-blind, placebo-controlled, parallel-group, multicenter, treat-through Phase 3 study designed to evaluate the efficacy and safety of TREMFYA® SC induction therapy (400 mg at Weeks 0, 4, and 8) in adults with moderately to severely active ulcerative colitis who had an inadequate response or intolerance to conventional therapy (e.g., thiopurines or corticosteroids), prior biologics (TNF antagonists or vedolizumab) and/or ozanimod or approved JAK inhibitors. Patients (n = 418) were randomized 1:1:1 to receive TREMFYA® 400 mg SC induction at Weeks 0, 4 and 8 followed by TREMFYA® 200 mg SC every 4 weeks (q4w); or TREMFYA® 400 mg SC induction at Weeks 0, 4 and 8, followed by TREMFYA® 100 mg SC every 8 weeks (q8w); or placebo. The maintenance dose regimens in ASTRO (200 mg SC q4w and 100 mg SC q8w) are the same as those evaluated in the Phase 3 QUASAR program which established the efficacy and safety profile of IV induction followed by SC maintenance therapy in patients with moderate to severely active UC.8 ABOUT ULCERATIVE COLITIS Ulcerative colitis (UC) is a chronic disease of the large intestine, also known as the colon, in which the lining of the colon becomes inflamed and develops tiny open sores, or ulcers, that produce pus and mucus. It is the result of the immune system's overactive response. Symptoms vary but may typically include loose and more urgent bowel movements, rectal bleeding or bloody stool, persistent diarrhea, abdominal pain, loss of appetite, weight loss, and fatigue.9 ABOUT TREMFYA® (guselkumab) Developed by Johnson & Johnson, TREMFYA® is the first approved fully-human, dual-acting monoclonal antibody designed to neutralize inflammation at the cellular source by blocking IL-23 and binding to CD64 (a receptor on cells that produce IL-23). Findings for dual-acting are limited to in vitro studies that demonstrate guselkumab binds to CD64, which is expressed on the surface of IL-23 producing cells in an inflammatory monocyte model. The clinical significance of this finding is not known. TREMFYA® is a prescription medicine approved in the U.S. to treat: adults with moderate to severe plaque psoriasis who may benefit from taking injections or pills (systemic therapy) or phototherapy (treatment using ultraviolet or UV light). adults with active psoriatic arthritis. adults with moderately to severely active ulcerative colitis. adults with moderately to severely active Crohn's disease.5 TREMFYA® is approved in Europe, Canada, Japan, and a number of other countries for the treatment of adults with moderate-to-severe plaque psoriasis and for the treatment of adults with active psoriatic arthritis. In addition, TREMFYA ® is approved in Europe, Japan and Brazil for the treatment of adult patients with moderately to severely active UC and in Brazil and China for the treatment of adults with moderately to severely active CD. Johnson & Johnson maintains exclusive worldwide marketing rights to TREMFYA®. For more information, visit: . IMPORTANT SAFETY INFORMATION What is the most important information I should know about TREMFYA®? TREMFYA® is a prescription medicine that may cause serious side effects, including: Serious Allergic Reactions. Stop using TREMFYA® and get emergency medical help right away if you develop any of the following symptoms of a serious allergic reaction: Infections. TREMFYA® may lower the ability of your immune system to fight infections and may increase your risk of infections. Your healthcare provider should check you for infections and tuberculosis (TB) before starting treatment with TREMFYA® and may treat you for TB before you begin treatment with TREMFYA® if you have a history of TB or have active TB. Your healthcare provider should watch you closely for signs and symptoms of TB during and after treatment with TREMFYA®. Tell your healthcare provider right away if you have an infection or have symptoms of an infection, including: Liver problems. With the treatment of Crohn's disease or ulcerative colitis, your healthcare provider will do blood tests to check your liver before and during treatment with TREMFYA®. Your healthcare provider may stop treatment with TREMFYA® if you develop liver problems. Tell your healthcare provider right away if you notice any of the following symptoms: Do not use TREMFYA® if you have had a serious allergic reaction to guselkumab or any of the ingredients in TREMFYA®. Before using TREMFYA®, tell your healthcare provider about all of your medical conditions, including if you: have any of the conditions or symptoms listed in the section "What is the most important information I should know about TREMFYA®?" have an infection that does not go away or that keeps coming back. have TB or have been in close contact with someone with TB. have recently received or are scheduled to receive an immunization (vaccine). You should avoid receiving live vaccines during treatment with TREMFYA®. are pregnant or plan to become pregnant. It is not known if TREMFYA® can harm your unborn baby. Pregnancy Registry: If you become pregnant during treatment with TREMFYA®, talk to your healthcare provider about registering in the pregnancy exposure registry for TREMFYA®. You can enroll by visiting , by calling 1-877-311-8972, or emailing [email protected]. The purpose of this registry is to collect information about the safety of TREMFYA® during pregnancy. are breastfeeding or plan to breastfeed. It is not known if TREMFYA® passes into your breast milk. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What are the possible side effects of TREMFYA®? TREMFYA® may cause serious side effects. See "What is the most important information I should know about TREMFYA®?" The most common side effects of TREMFYA® include: respiratory tract infections, headache, injection site reactions, joint pain (arthralgia), diarrhea, stomach flu (gastroenteritis), fungal skin infections, herpes simplex infections, stomach pain, and bronchitis. These are not all the possible side effects of TREMFYA®. Call your doctor for medical advice about side effects. Use TREMFYA® exactly as your healthcare provider tells you to use it. Please read the full Prescribing Information, including Medication Guide, for TREMFYA® and discuss any questions that you have with your doctor. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Dosage Forms and Strengths: TREMFYA® is available as 100 mg/mL and 200 mg/2mL for subcutaneous injection and as a 200 mg/20 mL (10 mg/mL) single dose vial for intravenous infusion. ABOUT JOHNSON & JOHNSON At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.   Learn more at or at Follow us at @JNJInnovMed. Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies.  Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding TREMFYA®. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. References: 1 Peyrin-Biroulet, et al. Efficacy and safety of subcutaneous guselkumab induction therapy in patients with Ulcerative Colitis: Results through week 12 from the phase 3 ASTRO study. Results from the Phase 3 ASTRO study. Oral presentation (#OP10) at the 20th Congress of the European Crohn's and Colitis Organization (ECCO). February 2025. 2 Long M, et al. Efficacy And Safety Of Subcutaneous Guselkumab Induction Therapy In Patients With Ulcerative Colitis: Results Through Week 24 From The Phase 3 Astro Study. Oral presentation (#4241895) at Digestive Disease Week 2025. May 2025. 3 Atreya R, Abreu MT, Krueger JG, et al. Guselkumab, an IL-23p19 subunit-specific monoclonal antibody, binds CD64+ myeloid cells and potentially neutralizes IL-23 produced from the same cells. Poster presented at: 18th Congress of the European Crohn's and Colitis Organization (ECCO); March 1-4, 2023; Copenhagen, Denmark. Poster P504. 4 Kreuger JG, Eyerich K, Kuchroo VK. Il-23 past, present, and future: a roadmap to advancing IL-23 science and therapy. Front Immunol. 2024; 15:1331217. doi:10.3389/fimmu.2024.1331217. 5 TREMFYA® [Prescribing Information]. Horsham, PA: Janssen Biotech, Inc. 6 Skyrizi® [Prescribing Information]. North Chicago, IL: AbbVie, Inc. 7 Omvoh™ [Prescribing Information]. Indianapolis, IN: Eli Lilly and Company. 8 National Institutes of Health: Clinicaltrials.gov. A Study of Guselkumab Therapy in Participants With Moderately to Severely Active Ulcerative Colitis (ASTRO). Identifier: NCT05528510. . Accessed March 2025. 9 Crohn's & Colitis Foundation. What is ulcerative colitis? Available at: . Accessed March 2025 SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

当大西洋两岸的德国与美国几乎同时为CGT疗法支付开创新模式，又将为中国的医保支付改革带来怎样的借鉴与启示？撰文| 杨景雄 孔凡懿 石洣珏在各国医疗费用持续攀升与技术迭代加速的双重挑战下，医疗支付体系的改革已成为全球性挑战。因此，针对全球公认的昂贵创新疗法CGT，各国都进行了不同的尝试。近期，CSL公司就和德国医保机构达成了一个创新付费协议：天价基因药Hemgenix不用提前全款买单，而是“治好了再付钱”，开创了一种全新的“风险分担”模式。具体来说，医保每年会根据实际治疗效果支付药费，如果治疗失败（比如患者又需要打原来的凝血针），药企就得自己承担损失，医保不用付钱。远在大西洋另一边的美国，即使作为世界最大的医疗市场，也未能避开昂贵CGT疗法给传统医保支付模式带来的严峻挑战。面对这一压力，美国医疗保险和医疗补助服务中心（Centers for Medicare & Medicaid Services, CMS）决定对既有支付体系框架进行改革，在2024年12月启动了CGT医保支付创新试点。简单来说就是启动了 “集中谈判+按疗效支付”的新模式，针对罕见病基因疗法，联邦政府直接和药企谈全国统一价，接受治疗的患者越多，药企返利越多，还派技术团队盯疗效，治不好药企还得退钱。无论是德国的“治好了再付钱”，还是美国的“治不好退钱”的CGT疗法支付新模式，其实都暗示出一个不约而同的全球性变革趋势：面对医疗技术迭代加速带来的成本压力，其定价机制逐步从市场导向转向政府主导按价值付费。当然，这也为中国探索贵价创新疗法支付机制提供了参考，在基本医保支付能力有限的情况下，如何平衡创新激励与医保可持续性？如何通过支付机制设计提升医疗公平？美国CGT医保支付试点：“治不好就退钱”美国公共医疗保障体系由联邦医疗保险计划（Medicare）与医疗补助计划（Medicaid）两大支柱构成。二者分别聚焦老年与低收入群体，共同构成基础保障网络。Medicare由联邦政府统一管理，覆盖65岁以上老年人及特定残障群体。Medicaid由联邦与州政府联合运作，侧重低收入人群（包括儿童、孕妇等弱势群体），各州可在联邦框架下自主制定具体覆盖范围与服务内容，资金由联邦和州政府按比例分担。长期以来，美国Medicaid以州为单位对部分药品开展价格谈判，以此控制支出。尽管2022年拜登政府出台的《通货膨胀削减法案》（Inflation Reduction Act, IRA）强制药企返利（如超出通胀的涨价）等措施对传统药品价格提供了一定约束，但公共医保项目医疗费用仍呈持续攀升势态，尤其在细胞与基因疗法（Cell & Gene Therapy, CGT）等昂贵创新疗法领域，支付挑战严峻。为了应对挑战，美国医疗保险和医疗补助服务中心（Centers for Medicare & Medicaid Services, CMS）于2024年12月4日正式启动了CGT医保支付创新试点。该试点打破传统支付模式，以联邦层面的集中谈判为基础，结合按疗效支付协议（Outcome-Based Agreement）与条件返利（rebate）机制，旨在为可从根本上治愈罕见病和严重遗传疾病的昂贵创新疗法建立可持续的支付解决方案。根据CMS规划，该试点初期将聚焦镰状细胞病（Sickle Cell Disease, SCD）的两种基因疗法——Vertex Pharmaceuticals开发的Casgevy与Bluebird Bio研发的Lyfgenia。此类疗法费用高达220万至310万美元，是史上最昂贵的精准治疗药物之一。然而根据美国疾病控制与预防中心（CDC）的数据显示，SCD患者人均寿命较美国平均水平短20余年，且其中约50%为Medicaid参保者。对于这一低收入及弱势群体而言，若无公共医保支持，患者将无法获得挽救生命的治疗方案。SCD若未得到有效治疗，会产生一系列并发症并造成患者反复住院，进一步加剧医疗负担。CMS希望通过此次试点解决昂贵创新疗法的可及性问题，确保低收入群体及时获得此类天价救命药。根据CMS网站发布的项目介绍书，此次试点的核心目标在于，从联邦层面进行价格谈判、统一市场准入标准并纳入按疗效付费，消除因各州政策差异导致的覆盖不均的问题，保障所有参与州的Medicaid患者可以公平获得基因治疗，最终达到人群健康水平上升的目的。具体而言，这一新支付模式的实施方式主要为三步：联邦层面（CMS）主导进行通过统一谈判与定价、设置分层返利机制等方式控制付费，最后各州可自主选择是否参与。统一谈判与定价：因SCD患者群体规模小但治疗成本极高，CMS选择先从SCD基因疗法试点。具体而言，CMS代表各州与药企进行集中谈判，达成全国统一价格和准入标准。为确保协议落地，CMS将提供技术支持，进行监督和疗效评估，核查各州执行时的合规性，并建立疗效追踪系统（电子健康档案）以评估返利触发条件。图1：CMS CGT准入模式实施时间表(来源：cms.gov)分层返利机制：为了进一步降低药品净成本，CMS在价格谈判中设计了具有借鉴意义的三重返利结构，包括量价挂钩、按疗效结果分期返利、固定价格让步等。药企需提供与采购量挂钩的基础折扣，并根据疗效达标率追加附加返利（如患者五年内复发或五年内人群疾病复发率≥n%，再返利x%）。因此只有患者达到特定疗效标准后，医保才需支付部分或全部药费；若疗效不达标，药企需退还部分费用或承担额外责任。此外，CMS的谈判要求药企提供与绩效无关的固定价格让步（如一次性降价x%），使得谈判结果对医保资金有即时保护效果。值得注意的是，此番价格谈判的结果并不推翻已有的折扣协议，而是将以附加协议的形式，在原有《通货膨胀削减法案》下的医疗补助计划药品返利方案（Medicaid Drug Rebate Program, MDRP）的降价协议上进一步降低价格，使得新药在原先折扣的基础上进一步享受新谈判带来的降价。图2：CMS CGT准入模式返利结构(来源：cms.gov)图3：CMS CGT准入模式返利时间点(来源：cms.gov)试点的实施方式上，州政府层面则体现为各州自愿参与机制，保留一定自主选择权。各州可以自主选择是否参与试点计划，若加入，州政府将直接采用CMS与药企谈判达成的统一价格及支付模式，不得另行议价或修改核心返利规则。CMS CGT创新支付模式试点是美国医保支付体系的一次重大变革。通过集中谈判，美国正在尝试降低昂贵创新药品的支付成本，提高Medicaid参保者的医疗可及性。CMS谈判中纳入按疗效支付的协议也真正将药企的收入与临床结果直接挂钩，既强化了企业对治疗有效性的责任，又减轻了医保资金负担。因此，美国业界认为，尽管本次试点仅涉及两款CGT，但从已公布的CMS文件中可以预计此次谈判对相关产品定价潜力的影响将超越《通货膨胀削减法案》带来的降价。SCD的患者病程较长、病情反复，所用医保资源较为可观。虽然纳入两款药物的前期费用很高，美国CMS认为CGT有望从根源治愈疾病，因此可以减少长期的医疗开支。如果试点成功，该模式可能会扩展至其他高成本的CGT，甚至影响全球医保支付模式的发展。对中国的启示：“最优性价比”或许是医保谈判的下一阶段目标美国CMS试点提出的“集中谈判+按疗效支付”模式为全球医保改革提供了新范例，也为中国医保支付模式的深化提供了可操作性的启示。中国国家医保谈判虽然已经展现出规模效应，但在按疗效支付等机制上仍有探索空间。具体而言，中国可借鉴CMS经验，推进支付模式向精细化方向改革，包括构建以“疗效”为核心的支付机制，在集中谈判基础上建立多层次的返利体系、建设独立疗效评估机制，以实现医保资金的精准支出和可持续运行。首先，最直接的借鉴就是如何保持量价挂钩与激励创新之间的平衡，“直接降价”模式未必是最佳选择。美国CMS CGT试点实践经验表明，分散化谈判模式存在显著弊端，各州政策割裂导致药品价格差异扩大、医保支出持续承压。相较之下，集中谈判通过规模化议价机制，在抑制价格攀升与促进医疗技术可及性方面拥有显著优势。CMS试点选择构建全国统一谈判框架，正是为了利用规模化采购优势实现成本结构优化，同时规避因分散决策而导致的管理低效与资源浪费问题。中国近年来的国家药品医保谈判同样在全球范围内引起关注。中美两国均通过国家层面主导的集中谈判提升买方议价能力，但具体实施路径存在差异。我国医保局使用以价格换取医保资格的策略使药企接受大幅降价来推动创新疗法的医保覆盖。无论实施路径如何，国家集中谈判在快速提升药品可及性的同时，也需要兼顾医保可负担性与激励创新之间的平衡。如果价格被过度压缩的同时，市场销量增幅有限，使企业销售收入难以覆盖前期的研发与生产成本，或使其利润率长期显著低于国际基准，可能抑制企业的研发动力，甚至出现制药企业参与药价谈判积极性降低的情况。中国医保谈判可在现有谈判框架上进一步优化现有支付模式。医保管理可以从单一的价格控制逐步转向综合的费用管理。与此同时，企业从不承担医保风险的角色，转向与医保部门共同承担风险。为此，未来可以探索引入多种风险共担机制（如按疗效支付、分期返利等），避免“一刀切”的支付政策。当今医疗技术快速发展，新药研发模式日益多样，因此新技术本身的复杂性也要求医保支付模式具备更高的适应性。美国CMS试点中的分层返利机制即为此提供了有益借鉴，通过将药企收益与患者临床疗效动态衔接，既确保医保资金的有效使用，又激励企业追求高质量创新，使具备真实临床价值的创新疗法获得可持续的发展空间。美国CMS试点的另一个可借鉴之处在于，如何向按疗效支付的改革迈步。我国现行基本医保支付模式目前主要采用单一价格谈判，按疗效支付模式尚未大规模应用。此次CMS试点的核心之一就是按疗效支付，精准使用医保资金，不为无效治疗买单。在按疗效支付协议的框架下，治疗方案需要达到双方约定患者获益的疗效指标，药企才能获得支付。因此按疗效支付可激起企业医疗质量方向上的创新动力，使企业更注重药品的临床效果和长期收益，而非仅关注上市后的销售。按疗效支付也将推动更多可提供高额临床获益的治疗方式被纳入医保体系，使得更多患者可以得到重焕新生的治疗。未来，中国可以考虑在罕见病、基因疗法等高价值技术领域引入按疗效支付的协议 ，形成精细化的医保支付体系。美国CMS试点的疗效挂钩协议为中国提供了可操作性极强的参考框架。从具体落地的角度上来看，按疗效支付模式的建立需要聚焦以下几个关键环节：一方面，具有分阶段支付与长期疗效观察能力的基础建设是执行按疗效支付模式重中之重的前提条件，也是其运行核心。中国可优先在罕见病、肿瘤免疫治疗（如CAR-T）等高值药品领域试行按疗效支付。例如，对某一CAR-T疗法可要求“完全缓解率”指标作为全额支付门槛，未达标则触发返利机制。另一方面，按疗效支付模式长期健康的运作需要一个完善的疗效评估体系。为保证评估结果的真实性，评估机构必须独立。第三方评估机构或相关部门应负责疗效数据追踪与验证，确保制药企业与医疗机构数据客观真实。此外，任何疗效评估体系都需要针对不同疾病制定量化、可追溯的疗效标准。以基因疗法为例，可设定“基因表达稳定性”“5年无进展生存率”等核心结果指标。需注意避免模糊定义，导致支付争议。同时，法律法规也有待完善，明确医保、药企、医院的责任边界，并建立纠纷仲裁机制。结论：昂贵药品，国家集中谈判+按疗效支付，是全球医保支付的趋势美国CMS试点模式的推出，标志着全球医保改革正在向高效、风险分担、可持续的方向迈进。这一创新支付试点揭示了两个核心理念：第一， 在高价创新疗法支付领域，集中谈判机制能产生显著的规模效应，是提升买方议价能力、控制药品价格的有效手段，中国在创新药谈判中已证明其可行性，美国的CMS试点也在朝这一方向靠拢。第二， 按疗效支付是未来医保改革的关键路径，它不仅能够确保资金的精准使用，更能加速推动优质药物进入市场。在全球医疗成本不断攀升、基因疗法和创新药物层出不穷的背景下，医保支付模式的创新已成为各国政府必须面对的挑战。美国CMS的新试点虽然刚刚开始，但其背后的逻辑正为全球医保体系验证一条可行的道路。对中国而言，需在现有医保谈判基础上，加快构建包含风险共担、数据驱动、全周期管理的创新支付体系，并开展实施方案试点，形成可复制的中国方案。参考资料：CMS. Cell and Gene Therapy (CGT) Access Model [DB/OL].[2025-03-28], https://www.cms.gov/priorities/innovation/innovation-models/cgt.关于作者：杨景雄，清华大学医院管理研究院23级研究生孔凡懿、石洣珏，清华大学医院管理研究院23级研究生清华大学医院管理研究院陈怡教授对本篇文章亦有指导一审| 黄佳二审| 李芳晨三审| 李静芝精彩推荐大事件 | IPO | 融资&交易 | 财报季 | 新产品 | 研发日 | 里程碑 | 行业观察 | 政策解读 | 深度案例 | 大咖履新 | 集采&国谈 | 出海 | 高端访谈 | 技术&赛道 | E企谈 | 新药生命周期 | 市值 | 新药上市 | 商业价值 | 医疗器械 | IND | 周年庆大药企 | 竞争力20强 | 恒瑞 | 石药 | 中生制药 | 齐鲁 | 复星 | 科伦 | 翰森 | 华润 | 国药 | 云南白药 | 天士力 | 华东 | 上药创新药企 | 创新100强 | 百济 | 信达 | 君实 | 复宏汉霖 | 康方 | 和黄 | 荣昌 | 亚盛｜康宁杰瑞｜贝达｜微芯｜再鼎｜亚虹跨国药企｜ＭＮＣ卓越｜辉瑞｜AZ｜诺华｜罗氏｜BMS｜默克｜赛诺菲｜GSK｜武田｜礼来｜诺和诺德｜拜耳供应链｜赛默飞｜药明｜凯莱英｜泰格｜思拓凡｜康龙化成｜博腾｜晶泰｜龙沙｜三星启思会 | 声音·责任 | 创百汇 | E药经理人理事会 | 微解药直播 | 大国新药 | 营销硬观点 | 投资人去哪儿 | 分析师看赛道 | 药事每周谈 | 中国医药手册