骨髓纤维化市场：KOL洞察

基于一些世界领先的KOL的专业知识，本报告对骨髓纤维化治疗领域提供了公正和平衡的见解，重点关注影响市场动态的关键问题。它使 KOL 能够批判性地评估已上市和研发的疗法的潜力，提供可操作的资讯来指导有关产品定位和竞争的策略决策，帮助更佳理解当前和未来的治疗模式。

报告内容

• 2023年，FDA 授予 Empexiq（pacritinib；CTI Biopharma）孤儿药地位，用于治疗血小板数低于 50 x 109/L 的骨髓纤维化（MF）患者。这对治疗有何影响？
• 过去 12个月中，Jakafi（ruxolitinib；Incyte Corporation/Novartis）的使用情况有何变化？
• 第三阶段 FREEDOM2 试验于2024年 10月发布，报告指出 MF 患者（以ruxolitinib预先治疗）的疗效优于最佳可用疗法。这次试验将如何影响 fedranitinib 的使用？
• Ojjaara是否有潜力取代ruxolitinib或其他治疗贫血的药物？
• 端粒酶抑制剂在治疗骨髓纤维化方面的潜力以及它们是否是对 JAK 抑制剂治疗无反应的患者的一个良好选择？
• 骨髓纤维化治疗中尚未满足的需求有哪些？人工智慧（AI）工具和数位技术能否解决这些问题？
• 第二阶段研究 SENTRY-2 评估了 Xpovio 对未接受 JAK 抑制剂治疗的骨髓纤维化患者的疗效和安全性，并将脾臟缩小作为主要结果与已获批准的JAK 抑制剂进行了比较，对本研究有何期待？
• Syntara 公布了第2 阶段 SNT-5505 研究的积极资料，显示 46%的患者在 12 週时整体身体评分下降了 50%，而在 38 週时这一比例上升至 80%。这些资料令人信服，但与其他药物相比如何？
• KER050 与 luspatercept 等类似药物相比如何？
• 联合治疗在未来的骨髓纤维化治疗中将扮演什么角色？

Drawing on the expertise of the world's foremost KOLs, this report provides impartial and balanced insights into the myelofibrosis treatment landscape, highlighting the critical battlegrounds shaping market dynamics. Gain a deeper understanding of current and future treatment paradigms as KOLs critically evaluate the potential of launched and pipeline therapies, offering actionable intelligence to guide strategic decision-making on product positioning and competitiveness.

Key Questions Answered:

• In 2023, the FDA granted orphan drug status Enpaxiq (Pacritinib; CTI Biopharma for use in Myelofibrosis (MF) in patients with platelet counts of below 50 x 109 /L. How has this impacted utilisation?
• How has the use of Jakafi (Ruxolitinib; Incyte Coorporation/Novartis changed over the last 12 months?
• The Phase III FREEDOM2 trial was published in October 2024and reported efficacy gains in MF patients (previously treated with ruxolitinib) vs the best available therapy. What do you see as the impact of this trial on fedrantinib use?
• To what extent do you see Ojjaara replacing ruxolitinib or other therapies for patients with anaemia?
• What do you think is the potential of telomerase inhibitors in the treatment of myelofibrosis? Do you think they offer a good alternative to those patients who have not responded to JAK-inhibitor treatment?
• What are the key unmet needs in the treatment of myelofibrosis? Can artificial intelligence (AI) tools or digital technology address any of these concerns?
• The Phase II SENTRY-2 is assessing efficacy and safety of Xpovio in JAK-inhibitor Naive MF patients, and comparing against approved JAK inhibitors with spleen reduction the primary outcomes. What are your expectations for this trial?
• Syntara released positive data from its Phase II SNT-5505 study, indicating 46% of patients received a 50% decrease in their total system score at 12 weeks, increasing to 80% at 38 weeks How compelling is this data, how would you say this compares to other agents?
• How would you say KER050 compares with similar agents such as luspatercept, which do you expect to be more efficacious?
• What do you think the role of combinations will be in myelofibrosis treatment in the future?

Methodology

Therapy Trend reports are developed through in-depth interviews with the world's foremost Key Opinion Leaders (KOLs) on the current and future treatment landscapes in major disease areas. KOLs are carefully selected based on strict screening criteria, including their global reputation, clinical expertise, and influence within their therapeutic area. Each interview is guided by a meticulously crafted discussion guide. These guides are developed in collaboration with KOLs and peer-reviewed by industry experts to ensure that the questions are comprehensive and pertinent to current market dynamics. Through continuous market monitoring over a 12-month period following the publication of each report, we provide timely updates from KOLs on key news events, market shifts, and clinical developments.

What makes our reports different?

FirstWord Reports, a trusted industry leader with an exclusive focus on the pharmaceutical sector, delivers in-depth, actionable insights for biopharma professionals and decision-makers. Our deep industry knowledge enables us to provide relevant and valuable insights that help you identify emerging trends and navigate complex challenges effectively. Backed by extensive research and independent, impartial insights from leading experts and KOLs, our reports deliver the accuracy and reliability you need. With exclusive access to interviews and data unavailable elsewhere, along with ongoing market monitoring, we give you a comprehensive view of market dynamics. Covering over 40 dynamic disease areas and providing physician intelligence, including KOL insights and quantitative physician surveys, as well as industry expert views on issues in medical affairs, digital health, sales & marketing, market access, and other areas, our reports empower you to make smarter, data-driven decisions and maintain a competitive edge in a fast-moving industry.