Veru, Inc.

Since its launch in 2015, it has been prescribed to more than 200,000 patients globally. The drug is currently approved for use in both men and women, expanding its market potential. Despite facing competition from other CDK4/6 inhibitors like Novartis' KISQALI and Eli Lilly's VERZENIO, IBRANCE maintains a strong position in the metastatic setting. LAS VEGAS, April 24, 2025 /PRNewswire/ -- DelveInsight's " IBRANCE Market Size, Forecast, and Market Insight Report" highlights the details around IBRANCE, a CDK4/6 inhibitor. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of IBRANCE. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Pfizer's IBRANCE (palbociclib) Overview IBRANCE is an oral medication that inhibits CDKs 4 and 6, which are crucial regulators of the cell cycle that drive cellular progression. In the U.S., IBRANCE is approved for treating adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer. It is used in combination with an aromatase inhibitor as a first-line endocrine therapy for postmenopausal women or men or with fulvestrant for patients whose disease has progressed after endocrine treatment. IBRANCE is currently authorized in over 90 countries and has been prescribed to more than 200,000 patients worldwide. Learn more about IBRANCE projected market size for metastatic HR+/HER2− breast cancer @ IBRANCE Market Potential The HR+ HER2- subtype is the most commonly found type of breast cancer, characterized by cancer cells that have estrogen and progesterone receptors but do not overexpress HER2. A patient's journey typically begins with the onset of concerning symptoms, leading to a comprehensive clinical evaluation and various imaging tests. DelveInsight estimates that approximately 211K new cases of HR+/HER2– breast cancer occurred in the US in 2024. CDK4/6 inhibitors currently dominate the first-line treatment market and represent a significant advancement in the management of HR+/HER2-ve breast cancer. The data from large, randomized clinical trials are both strong and consistent. While single-agent endocrine therapies have shown limited benefits in the first and subsequent treatment lines for women with ER-positive metastatic breast cancer (mBC), combination therapies have emerged as a more effective option for many patients. In recent years, the treatment landscape for HR+/HER2−ve breast cancer has been transformed with the introduction of highly active targeted therapies, including CDK4/6 inhibitors, mTOR inhibitors, PARP inhibitors, new oral SERDs, and PI3K inhibitors. These advances have expanded the range of treatment options and improved survival outcomes for these patients. The market for metastatic HR+/HER2− breast cancer in the 7MM is projected to grow during the forecast period (2025–2034) due to the introduction of several novel therapies in the market. Discover more about the metastatic HR+/HER2− breast cancer market in detail @ Metastatic HR+/HER2− Breast Cancer Market Report Emerging Competitors of IBRANCE Some of the drugs in the pipeline include Gedatolisib (Celcuity), ARV-471 (Arvinas and Pfizer), OP-1250 (Olema Pharmaceuticals), KEYTRUDA (Merck), and Rupitasertib (Evexta Bio), among others. In December 2024, Arvinas and Pfizer presented preliminary data from the ongoing Phase 1b portion of the TACTIVE-U sub-study of vepdegestrant in combination with abemaciclib among patients with locally advanced or metastatic ER+/HER2- breast cancer at the San Antonio Breast Cancer Symposium (SABCS) 2024. In December 2024, Olema Pharmaceuticals presented clinical results from the ongoing Phase Ib/II study of palazestrant in combination with ribociclib in patients with ER+/HER2- advanced or metastatic breast cancer at SABCS 2024. To know more about the number of competing drugs in development, visit @ IBRANCE Market Positioning Compared to Other Drugs Key Milestones of IBRANCE In February 2021, Pfizer Inc. announced that the U.S. Patent and Trademark Office (USPTO) has granted a U.S. Patent Term Extension (PTE) certificate for IBRANCE. This extension prolongs the validity of U.S. Patent No. RE47,739 ('739) by over four years, now lasting until March 5, 2027. In April 2019, Pfizer announced that the FDA has approved a supplemental New Drug Application (sNDA) to broaden the uses of IBRANCE in combination with an aromatase inhibitor or fulvestrant, now including men with hormone receptor-positive (HR+), HER2-negative (HER2-) advanced or metastatic breast cancer. In March 2017, Pfizer Inc. revealed that the FDA has approved a supplemental New Drug Application (sNDA) for IBRANCE, its pioneering cyclin-dependent kinase 4/6 (CDK 4/6) inhibitor, following the positive outcomes from the confirmatory Phase 3 trial PALOMA-2. In September 2016, Pfizer Inc. announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) had issued a positive recommendation for the marketing authorization of IBRANCE® (palbociclib) in the European Union (EU). This recommendation is for its use in treating women with hormone receptor-positive, HER2-negative (HR+/HER2-) locally advanced or metastatic breast cancer. In February 2016, Pfizer announced that the FDA has approved a new indication for IBRANCE 125mg capsules, expanding its use. The approval allows IBRANCE to be used in combination with fulvestrant for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer in women whose disease has progressed after endocrine therapy. In February 2015, Pfizer announced that the FDA granted accelerated approval for IBRANCE in combination with letrozole to treat postmenopausal women with estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+/HER2-) advanced breast cancer as the initial endocrine-based therapy for their metastatic disease. Discover how IBRANCE is shaping the metastatic HR+/HER2− breast cancer treatment landscape @ IBRANCE Drug IBRANCE Market Dynamics IBRANCE is a targeted therapy that has revolutionized the treatment of hormone receptor-positive, HER2-negative breast cancer, particularly in combination with aromatase inhibitors or letrozole. Developed by Pfizer, IBRANCE was one of the first CDK4/6 inhibitors approved by the FDA for metastatic breast cancer, and its approval has significantly changed the treatment landscape. The drug works by inhibiting cyclin-dependent kinases 4 and 6, which are key regulators of the cell cycle, thus preventing cancer cells from proliferating. Its market dynamics have been shaped by the growing demand for targeted therapies, as well as the increasing focus on precision medicine and improved patient outcomes. The global market for IBRANCE has been positively impacted by its proven efficacy in clinical trials and its ability to extend progression-free survival in breast cancer patients. The drug's market dominance is underpinned by its widespread acceptance and adoption by oncologists worldwide. However, competition is intensifying, with other CDK4/6 inhibitors such as VERZENIO (abemaciclib) and KISQALI (ribociclib) entering the market, offering similar benefits but with some distinct clinical profiles. This competitive landscape has led to strategic pricing and marketing strategies by Pfizer, aiming to maintain IBRANCE's market leadership while managing pressures from generic alternatives. Additionally, the evolving reimbursement landscape and regulatory policies around oncology drugs are crucial factors influencing the market dynamics. Health insurers and government healthcare programs often scrutinize the cost-effectiveness of cancer treatments, which can impact the pricing and accessibility of IBRANCE in different markets. Another factor is the rising focus on combination therapies, as IBRANCE is often used in conjunction with other drugs like letrozole, which boosts its efficacy and market potential. The shift toward personalized cancer treatment regimens is expected to continue fueling demand for CDK4/6 inhibitors like IBRANCE, especially in more advanced stages of breast cancer. In terms of geographical markets, IBRANCE has seen significant uptake in North America and Europe, where the healthcare infrastructure supports innovative cancer therapies. However, emerging markets, where the economic barriers to accessing expensive cancer treatments can be higher, present a challenge to the widespread use of IBRANCE. Pfizer's ongoing efforts to expand in these regions, through partnerships and pricing adjustments, will be key to ensuring continued growth in global market share. Overall, the IBRANCE market is poised for steady growth, driven by clinical advancements, increasing breast cancer diagnoses, and the continual push toward more targeted, effective therapies. Dive deeper to get more insight into IBRANCE's strengths & weaknesses relative to competitors @ IBRANCE Market Drug Report Table of Contents Related Reports Metastatic HR+/HER2− Breast Cancer Market Metastatic HR+/HER2− Breast Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key metastatic HR+/HER2− breast cancer companies, including Merck, Arvinas, Olema Pharmaceuticals, Celcuity, Roche, AstraZeneca, Daiichi Sankyo, Eli Lilly, Sermonix Pharmaceuticals, Genentech, Veru Pharma, DualityBio, BioNtech, Evgen Pharma, Carrick Therapeutics, EQRx, G1 Therapeutics, Immutep, among others. HR+/HER2− Breast Cancer Pipeline HR+/HER2− Breast Cancer Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key HR+/HER2− breast cancer companies, including Regor Therapeuics, Seagen Inc., CytomX Therapeutics, Taizhou EOC Pharma, Chia Tai Tianqing Pharmaceutical Group, AstraZeneca, Daiichi Sankyo, Inc., Tyme, Inc., Seagen Inc., Context Therapeutics, Eisai Inc., Shanghai Hengrui Pharmaceutical Co., Ltd., Jiangsu Simcere Pharmaceutical Co., Kind Pharmaceuticals, Merus N.V., Atossa Therapeutics, Roche, among others. Breast Cancer Market Breast Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key breast cancer companies including Veru, Sanofi, Roche, AstraZeneca, Eli Lilly, EQRx, Gilead, Sermonix Pharmaceuticals, Evgen Pharma, Tyme, Genentech, Daiichi Sankyo, among others. Metastatic Breast Cancer Pipeline Metastatic Breast Cancer Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key metastatic breast cancer companies, including Roche, RemeGen, SynCore Biotechnology, Allarity Therapeutics, Daiichi Sankyo Company, Jiangsu Alphamab Biopharmaceuticals Co., Ltd, Byondis B.V., Jiangsu Hansoh Pharmaceutical Co., Ltd., Shanghai Miracogen Inc., Ambrx, Inc., Daehwa Pharmaceutical Co., Ltd., Phoenix Molecular Designs, GlycoMimetics Incorporated, Rhizen Pharmaceuticals SA, Menarini Group, Samus Therapeutics, Inc., Hanmi Pharmaceutical Company Limited, Jiangxi Qingfeng Pharmaceutical Co. Ltd., Immutep Limited, Arvinas Inc., G1 Therapeutics, Mirati Therapeutics Inc., Chia Tai Tianqing Pharmaceutical, Shanghai Pharmaceuticals Holding Co., Ltd, Pfizer, OncoTherapy Science, Inc., Eisai Inc., Dizal Pharmaceuticals, Jiangsu Hengrui Medicine Co., Tyme, Inc, Orion Pharma, HiberCell, Inc., Rhizen Pharmaceuticals SA, Hutchison Medipharma Limited, OncoPep Inc., Taizhou Hanzhong biomedical co. LTD, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

As VERZENIO gains traction in early-stage treatments and broadens its approved uses, its revenue is expected to keep growing. While the global CDK4/6 inhibitor market remains highly competitive, VERZENIO's distinct clinical advantages and ongoing studies could contribute to further market expansion. LAS VEGAS, March 13, 2025 /PRNewswire/ -- DelveInsight's " VERZENIO Market Size, Forecast, and Market Insight Report" highlights the details around VERZENIO, which is a targeted treatment known as a CDK4/6 inhibitor. The drug is a non-chemotherapy oral tablet. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of VERZENIO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Eli Lilly and Company's VERZENIO (abemaciclib) Overview VERZENIO (abemaciclib) is a prescription medication used to treat adults with HR-positive, HER2-negative breast cancer that has metastasized (spread to other parts of the body). It is administered orally and is prescribed in combination with an aromatase inhibitor as an initial endocrine-based therapy. Additionally, the drug is being developed for the treatment of prostate cancer. VERZENIO is approved for the following indications: In combination with endocrine therapy for the adjuvant treatment of adults with HR+/HER2-, node-positive, early-stage breast cancer at high risk of recurrence. In combination with an aromatase inhibitor as the initial endocrine-based therapy for adults with HR+/HER2- advanced or metastatic breast cancer. In combination with fulvestrant for adults with HR+/HER2- advanced or metastatic breast cancer that has progressed after prior endocrine therapy. As monotherapy for adults with HR+/HER2- advanced or metastatic breast cancer that has progressed following endocrine therapy and prior chemotherapy in the metastatic setting. Learn more about VERZENIO projected market size for breast cancer @ VERZENIO Market Potential HR+/HER2- breast cancer is the most common subtype of breast cancer, defined by the presence of estrogen and progesterone hormone receptors but lacking HER2 overexpression. This form of breast cancer generally has a favorable prognosis and is primarily managed with hormone therapy to suppress hormone-driven tumor growth. According to DelveInsight's estimates, around 211,000 new cases of HR+/HER2− breast cancer were reported in the US in 2024. Over the past decade, endocrine therapy has remained the cornerstone of treatment, including for cases with visceral involvement. The current preferred approach involves combining endocrine therapy—using aromatase inhibitors or fulvestrant—with CDK4/6 inhibitors. The approval of CDK4/6 inhibitors has significantly transformed the treatment landscape for HR+/HER2− breast cancer. Currently, five selective CDK4/6 inhibitors— ENHERTU, DATROWAY, IBRANCE, KISQALI, and VERZENIO—are used in combination with endocrine therapy. In November 2023, the FDA approved TRUQAP (capivasertib) in combination with FASLODEX for patients with advanced HR+/HER2− breast cancer harboring specific biomarker alterations (PIK3CA, AKT1, or PTEN). The NCCN Guidelines have designated KISQALI as the only Category 1 Preferred CDK4/6 inhibitor for first-line treatment in combination with an aromatase inhibitor, strengthening its position in the U.S. market. This led to KISQALI recovering its lost market share, with its U.S. revenue contribution rebounding to 50%, following declines to 46% in 2020, 36% in 2021, and 38% in 2022. In January 2025, the FDA approved DATROWAY for patients with unresectable or metastatic HR+/HER2- breast cancer (IHC 0, IHC 1+, or IHC 2+/ISH-) who had previously received endocrine-based therapy and chemotherapy. Additionally, ENHERTU was approved for patients with unresectable or metastatic HR+, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer that had progressed after at least one prior endocrine therapy in the metastatic setting. Also, in January 2025, Roche reported positive topline results from its Phase III INAVO120 trial, assessing ITOVEBI in combination with IBRANCE and fulvestrant for patients with PIK3CA-mutated HR+/HER2−, endocrine-resistant, locally advanced, or metastatic breast cancer. Among the seven major markets, the US accounted for the largest market share in HR+/HER2− breast cancer, generating USD 7.5 billion in revenue in 2023. The market is expected to experience substantial growth from 2025 to 2034, driven by the introduction of novel therapeutic options. Discover more about the breast cancer market in detail @ Metastatic HR+/HER2- Breast Cancer Market Report Emerging Competitors of VERZENIO Some of the competing emerging key players in HR+/HER2- breast cancer market are, Merck (KEYTRUDA), Arvinas (ARV-471 (vepdegestrant)), Olema Pharmaceuticals (OP1250 (palazestrant)), Celcuity (Gedatolisib), Roche (Giredestrant (RG6171, GDC-9545); Inavolisib), AstraZeneca and Daiichi Sankyo (Datopotamab Deruxtecan (Dato-DXd)), AstraZeneca (Camizestrant (AZD9833); Capivasertib), Eli Lilly (LY3484356/Imlunestrant), Sermonix Pharmaceuticals (Lasofoxifene), Veru Pharma (Enobosarm), DualityBio/BioNtech (DB-1303), Evgen Pharma (SFX-01), Carrick Therapeutics (Samuraciclib (CT-7001)), EQRx/G1 Therapeutics (Lerociclib), Immutep (Eftilagimod Alpha (LAG-3lg/IMP321)), and others. To know more about the number of competing drugs in development, visit @ VERZENIO Market Positioning Compared to Other Drugs Key Milestones of VERZENIO In 2024, Phase III trials did not meet primary endpoints or were terminated for futility. Negative Phase III CYCLONE-2 results, in which VERZENIO added to abiraterone did not meet the primary endpoint of improved radiographic progression-free survival (rPFS) in men with metastatic castration-resistant prostate cancer (mCRPC). In December 2023, Eli Lilly and Company presented results from the MONARCH 3 clinical trial at the 2023 San Antonio Breast Cancer Symposium (SABCS). Results from MONARCH 3 show a numerical improvement in overall survival (OS) of 13.1 months for women with HR+, HER2- metastatic breast cancer treated with VERZENIO plus an aromatase inhibitor in the intent-to-treat population and 14.9 months for women with visceral disease. In March 2023, the US FDA approved an expanded indication for VERZENIO in combination with endocrine therapy for the adjuvant treatment of adult patients with HR+/HER2−, node-positive, early breast cancer (EBC) at a high risk of recurrence. This expanded adjuvant indication removes the Ki-67 score requirement for patient selection. In February 2022, the CHMP adopted a positive opinion recommending a change to the terms of the marketing authorization for the medicinal product VERZENIO. VERZENIO, in combination with endocrine therapy, is indicated for the adjuvant treatment of adult patients with HR+/HER2− negative, node-positive early breast cancer at high risk of recurrence. In October 2021, the US FDA approved VERZENIO, in combination with endocrine therapy (tamoxifen or an aromatase inhibitor), for the adjuvant treatment of adult patients with HR+/HER2−, node-positive, early breast cancer at high risk of recurrence and a Ki-67 score of ≥20% as determined by an FDA-approved test. Ki-67 is a marker of cellular proliferation. In September 2018, VERZENIO was approved in Europe for treating advanced HR+ breast cancer in women if the cancer has already spread to other parts of the body or is locally advanced.  In September 2018, VERZENIO was approved for the treatment of patients with HR+/HER2− unresectable or recurrent breast cancer in combination with fulvestrant or an aromatase inhibitor (AI) in Japan. In February 2018, the US FDA approved VERZENIO in combination with an aromatase inhibitor (AI) as initial endocrine-based therapy for the treatment of postmenopausal women with HR+/HER2− advanced or metastatic breast cancer In September 2017, the US FDA approved VERZENIO in combination with an endocrine therapy to treat adult patients who have HR+/HER2− advanced or metastatic breast cancer that has progressed after taking therapy that alters a patient's hormones (endocrine therapy). In October 2015, the US FDA granted Breakthrough Therapy Designation to abemaciclib for patients with refractory hormone-receptor-positive (HR+) advanced or metastatic breast cancer Discover how VERZENIO is shaping the breast cancer treatment landscape @ VERZENIO Breast Cancer VERZENIO Market Dynamics The oral administration of VERZENIO enhances patient convenience, while its expanded approval for the adjuvant treatment of adult patients with HR+/HER2- early breast cancer highlights its role as a differentiated CDK4/6 inhibitor in reducing the risk of recurrence. The rising prevalence of HR+/HER2- breast cancer is driving market demand for effective treatments like VERZENIO. Additionally, its demonstrated efficacy in combination with endocrine therapy and its ability to penetrate the central nervous system further strengthen its market position. Favorable reimbursement policies and increasing awareness among healthcare providers also contribute to its adoption. However, its adoption may be hindered by regulatory hurdles and potential side effects, including severe or life-threatening lung inflammation and serious liver problems. High treatment costs and access disparities in certain regions could also limit market penetration. Competition from other CDK4/6 inhibitors such as IBRANCE and KISQALI poses a challenge, along with concerns over long-term safety data. Additionally, the Phase III CYCLONE-2 trial failed to meet primary endpoints in mCRPC, which may impact its broader clinical prospects and investor confidence. Dive deeper to get more insight into VERZENIO's strengths & weaknesses relative to competitors @ VERZENIO Market Drug Report Table of Contents Related Reports CDK4/6 Inhibitor Market CDK4/6 Inhibitor Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key CDK4/6 inhibitor companies including Pfizer, Prelude Therapeutics, G1 Therapeutics, Pepper Bio, among others. Metastatic HR+/HER2− Breast Cancer Market Metastatic HR+/HER2− Breast Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key metastatic HR+/HER2− breast cancer companies including Pfizer, Novartis, Stemline Therapeutics, Olema Pharmaceuticals, Arvinas, Immutep, AstraZeneca, Roche, Genentech, Evexta Bio, EQRx, Sermonix Pharmaceuticals, Celcuity, Veru Pharma, Evgen Pharma, Eli Lilly, Biotheryx, among others. HR+/HER2− Breast Cancer Pipeline HR+/HER2− Breast Cancer Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key HR+/HER2− breast cancer companies, including Regor Therapeuics, Seagen Inc., CytomX Therapeutics, Taizhou EOC Pharma, Chia Tai Tianqing Pharmaceutical Group, AstraZeneca, Daiichi Sankyo, Inc., Tyme, Inc., Seagen Inc., Context Therapeutics, Eisai Inc., Shanghai Hengrui Pharmaceutical Co., Ltd., Jiangsu Simcere Pharmaceutical Co., Kind Pharmaceuticals, Merus N.V., Atossa Therapeutics, Roche, among others. Breast Cancer Market Breast Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key breast cancer companies, including Veru, Sanofi, Roche, AstraZeneca, Eli Lilly, EQRx, Gilead, Sermonix Pharmaceuticals, Evgen Pharma, Tyme, Genentech, Daiichi Sankyo, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . 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本文梳理了近一个月（2025年1月16日～2025年2月15日）在研药物的临床试验取得积极（研发成功率升高）进展的情况，同时，对部分药物的研究情况进行简述和分析，供研发人员参考。 据统计，在检索时间范围内，共有9个药物的临床试验数据取得积极进展，其中，处于Ⅲ期临床、Ⅱ期临床研究阶段的药物分别有3和5个（表1）。这些药物涉及的适应症包括蛛网膜下腔出血、血友病A型、急性髓性白血病、肥胖、类风湿性关节炎、缺血性中风和结直肠癌等。以下对部分药物进行简介。 表1：近一个月（2025年1月16日～2025年2月15日）临床试验数据积极的药物 Mim8 诺和诺德（Novo Nordisk）近日宣布，在研长效血友病A疗法Mim8，在针对70名患有血友病A（体内有和无凝血因子抑制物）的1至11岁儿童所进行的Ⅲ期临床试验FRONTIER3中获得积极中期结果。该公司预计在2025年递交Mim8的监管申请。 数据显示，Mim8在血友病A儿童中耐受性良好且疗效显著。在FRONTIER3试验的第一部分中，血友病A患者接受了为期26周的每周一次皮下注射Mim8治疗；在第二部分中，参与者可以选择继续每周一次给药，或在剩余26周中转为每月一次给药。本次中期分析报告涵盖了试验第一部分完成时的结果，同时还分享了部分第二部分的初步数据。 在试验第一部分接受每周一次预防性治疗的儿童中，需要治疗出血的平均年化出血率（ABR）为0.53；其中，中位数ABR为零，74.3%的参与者未出现需要治疗的出血。所有携带凝血因子抑制物的血友病A儿童（n＝14）均未报告发生需要治疗的出血。完成最初26周的试验后，45%的参与者选择转为每月一次使用Mim8，而其余55%则继续维持每周一次的给药方案。与此同时，试验第一部分中患者及其护理者报告的结果数据也显示，Mim8可能降低治疗负担，并改善血友病A儿童的身体功能和生活质量。在第26周时，98%的护理者表示他们更倾向于使用Mim8而非之前的治疗，其中73%的护理者表示“非常强烈”地倾向使用Mim8。此外，与基线相比，第26周时平均身体功能得分（衡量儿童进行日常体育活动，如跑步的能力）和平均生活质量总分均呈现出积极的改善趋势。 Mim8是一种模拟凝血因子Ⅷa（FⅧa）的在研新一代人源IgG4双特异性抗体，可以连接凝血因子Ⅸa与Ⅹ。Mim8旨在为血友病A患者提供持续止血效果，可用于每周一次、每两周一次或每月一次的预防性治疗。Mim8通过皮下注射给药，其作用是连接凝血因子Ⅸa与Ⅹ，从而替代因子凝血因子Ⅷ，恢复机体的凝血酶生成能力，帮助血液凝固。 Mim8将为血友病A患者提供一种新的治疗选择，特别是在预防出血事件和给药便利性方面。然而，罗氏的Hemlibra已经在市场上建立了强大的品牌影响力，诺和诺德需要在市场营销和患者教育方面投入更多资源，以确保Mim8的成功推广。 Enobosarm Veru公司近日宣布了Ⅱb期QUALITY临床研究的积极顶线结果。研究达到了主要终点，其在研药物Enobosarm显著减少了接受GLP-1类药物治疗的受试者的瘦体质量（Lean mass）流失。根据新闻稿，QUALITY是首个在人体中评估肌肉保护剂对接受GLP-1类药物治疗的肥胖或超重老年患者身体成分影响的试验。基于这些结果，Veru公司计划向FDA申请召开Ⅱ期临床结束会议，并着手启动Ⅲ期临床试验。 QUALITY是一项多中心、双盲、安慰剂对照的随机、剂量探索2b期研究，旨在评估Enobosarm（3mg和6mg）与安慰剂在168名超重或肥胖的60岁以上老年患者中的安全性和疗效，这些患者正在接受GLP-1受体激动剂Wegovy（Semaglutide，司美格鲁肽）的治疗。主要终点为基线至16周瘦体质量总量的变化，关键次要终点包括基线至16周总脂肪量、体重及通过爬楼梯测试评估的身体功能的变化。顶线疗效分析显示，该试验达到主要终点，接受Enobosarm联合GLP-1药物治疗的所有患者在16周时显示出瘦体质量总量的显著保护作用，相较于安慰剂组具有统计学意义上的显著优势。次要终点结果显示，Enobosarm联合GLP-1药物治疗在16周时，在总脂肪量减少方面优于安慰剂组。相较于安慰剂组，Enobosarm组患者中失去≥10%爬楼梯能力的受试者比例显著减少，结果具有临床意义。 Enobosarm（又名Ostarine、MK-2866、GTx-024和VERU-024，图1）是一种新型口服、每日一次的选择性雄激素受体调节剂（Selective androgen receptor modulator，SARM），此前已在5项临床研究当中接受评估，其中包括968名老年正常男性和绝经后女性，以及因晚期癌症导致肌肉减少的老年患者。 图1：Enobosarm的化学结构式 到2030年，全球肥胖和超重药物市场预计将达到1000亿美元。用于减肥的药物，如Ozempic、Wegovy、Zepbound、Mounjaro和其他GLP-1受体激动剂会导致脂肪和肌肉的显著减少。在美国，42%的老年人（>60岁）患有肥胖症，其中34%的患者还患有肌肉减少症或肌肉储备不足。因此，Enobosarm针对的是可能已经肌肉质量低（也称为肌肉减少性肥胖）的老年肥胖或超重患者，所有重要肌肉的肌肉质量进一步下降会增加肌肉无力、功能受限、行动障碍、跌倒、住院率增加和死亡率增加的风险。应该强调的是，Enobosarm可能与许多GLP-1受体激动剂减肥药物中的任何一种联合使用，不仅适用于老年或超重的高危患者，也适用于所有希望在服用GLP-1受体激动剂减肥时避免肌肉流失的超重或肥胖患者。Enobosarm与GLP-1受体激动剂的组合可能代表着数十亿美元的全球机会。 Rosnilimab 2025年2月12日，AnaptysBio公司宣布，其开发的PD-1+T细胞耗竭剂和激动剂Rosnilimab在中重度类风湿关节炎的全球Ⅱb期RENOIR临床试验中取得了积极结果，且6个月CDAI LDA响应率为有史以来最高。Rosnilimab为AnaptysBio的首发管线，也是礼来放弃之后全球范围内进展最快的PD-1激动剂抗体。与礼来Peresolimab相比，Rosnilimab活性更强。 此次试验在424名患者中进行，这些患者均为背景使用传统疾病修饰抗风湿药物（cDMARDs）的患者，其中59%为生物制剂或靶向合成DMARD（b/tsDMARD）初治患者，41%为经治患者。 主要终点：Rosnilimab在所有三个剂量（100mg每四周一次、400mg每四周一次、600mg每两周一次）中均达到了主要终点，即在第12周时与安慰剂相比，DAS-28 CRP评分从基线的平均变化具有统计学意义。 关键次要终点：在第12周时，Rosnilimab在至少一个剂量上达到了ACR20、ACR50和CDAI低疾病活动度（LDA）的关键次要终点，并且在所有剂量下均显示出数值上的优势。在第14周时，Rosnilimab在这些关键次要终点上的响应率创历史新高，69%的Rosnilimab治疗患者在第14周达到了CDAI LDA，并且在第28周时显示出持续的CDAI LDA和ACR50响应，以及28周潜在的加深的ACR70响应。 生物标志物数据：Rosnilimab在所有剂量下均显示出强大的药理学活性，包括减少PD-1high T细胞约90%、PD-1+T细胞约50%，并增加总调节性T细胞（Tregs），同时对总T细胞计数影响最小，显示出健康的免疫稳态。此外，Rosnilimab治疗的患者在整个试验期间C反应蛋白（CRP）从基线平均降低了约50%，而安慰剂组未观察到这一变化。 安全性：Rosnilimab在试验中表现出良好的安全性和耐受性，与安慰剂相比，不良事件发生率相似。 Anaptys计划在2025年第二季度发布完整的28周数据以及更多关于类风湿关节炎的转化医学数据。此外，公司还预计在2025年第四季度发布Rosnilimab在溃疡性结肠炎（UC）Ⅱ期临床试验的12周顶线数据。 去年礼来终止旗下PD-1激动剂的时候，引起市场震动，连带AnaptysBio股价大跌。如今AnaptysBio的中期数据证了自己，从目前看AnaptysBio的PD-1激动剂还是展现出了比JAK抑制剂还要优秀的数据。也让我们期待二季度的28周的数据。 【参考资料】 1. Informa数据库, 检索日期: 2025年2月21日. 2. 药渡数据库, 检索日期: 2025年2月21日. 3. 药明康德、医药魔方、学术经纬、丁香园、医药观澜、医麦客等网络公开资源. 本文其它内容请见《全球药物创新快讯》2025年第1期（总第148期）。