骨髓纖維化市場：KOL洞察

基於一些世界領先的KOL的專業知識，本報告對骨髓纖維化治療領域提供了公正和平衡的見解，重點關注影響市場動態的關鍵問題。它使 KOL 能夠批判性地評估已上市和研發的療法的潛力，提供可操作的資訊來指導有關產品定位和競爭的策略決策，幫助更佳理解當前和未來的治療模式。

報告內容

• 2023年，FDA 授予 Empexiq（pacritinib；CTI Biopharma）孤兒藥地位，用於治療血小板數低於 50 x 109/L 的骨髓纖維化（MF）患者。這對治療有何影響？
• 過去 12個月中，Jakafi（ruxolitinib；Incyte Corporation/Novartis）的使用情況有何變化？
• 第三階段 FREEDOM2 試驗於2024年 10月發布，報告指出 MF 患者（以ruxolitinib預先治療）的療效優於最佳可用療法。這次試驗將如何影響 fedranitinib 的使用？
• Ojjaara是否有潛力取代ruxolitinib或其他治療貧血的藥物？
• 端粒酶抑制劑在治療骨髓纖維化方面的潛力以及它們是否是對 JAK 抑制劑治療無反應的患者的一個良好選擇？
• 骨髓纖維化治療中尚未滿足的需求有哪些？人工智慧（AI）工具和數位技術能否解決這些問題？
• 第二階段研究 SENTRY-2 評估了 Xpovio 對未接受 JAK 抑制劑治療的骨髓纖維化患者的療效和安全性，並將脾臟縮小作為主要結果與已獲批准的JAK 抑制劑進行了比較，對本研究有何期待？
• Syntara 公佈了第2 階段 SNT-5505 研究的積極資料，顯示 46%的患者在 12 週時整體身體評分下降了 50%，而在 38 週時這一比例上升至 80%。這些資料令人信服，但與其他藥物相比如何？
• KER050 與 luspatercept 等類似藥物相比如何？
• 聯合治療在未來的骨髓纖維化治療中將扮演什麼角色？

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.