Ajax Therapeutics has received Orphan Drug Designation from the U.S. Food and Drug Administration for AJ1-11095, a next-generation JAK2 inhibitor being developed for myelofibrosis.

Ajax Therapeutics公司已获得美国食品药品监督管理局（FDA）授予的AJ1-11095孤儿药资格，该药物是一种正在开发用于治疗骨髓纤维化的下一代JAK2抑制剂。

AJ1-11095 was created through structure-based drug design and advanced computational methods to selectively bind the Type II conformation of JAK2. Preclinical studies have shown potential to reverse bone marrow fibrosis, reduce mutant allele burden, and maintain activity in cells that develop resistance to long-term Type I JAK2 inhibition..

AJ1-11095 是通过基于结构的药物设计和先进的计算方法开发的，旨在选择性结合 JAK2 的 II 型构象。 临床前研究表明，其具有逆转骨髓纤维化、减少突变等位基因负荷的潜力，并在对长期 I 型 JAK2 抑制产生耐药性的细胞中仍保持活性。

Myelofibrosis is a rare cancer characterised by enlarged spleen, bone marrow scarring, anaemia, and symptoms such as fatigue, night sweats, itching, and abdominal discomfort. Although Type I JAK2 inhibitors are widely used to relieve symptoms and reduce spleen size, they do not target the root cause of the disease. .

骨髓纤维化是一种罕见的癌症，其特征为脾脏肿大、骨髓瘢痕、贫血以及疲劳、夜间盗汗、瘙痒和腹部不适等症状。尽管I型JAK2抑制剂被广泛用于缓解症状和缩小脾脏大小，但它们并不能针对疾病的根源。

Many patients eventually discontinue treatment due to limited benefit, loss of response, side effects, or disease progression.

许多患者最终因疗效有限、反应丧失、副作用或疾病进展而停止治疗。

The rare blood cancer affects around 20,000 people in the United States.

这种罕见的血癌在美国影响约 20,000 人。

AJ1-11095 is the first JAK2 inhibitor in clinical development that targets the Type II conformation of the JAK2 kinase. Existing approved JAK2 inhibitors work through the Type I conformation. The therapy is currently being assessed in a Phase 1 study for patients who previously received a Type I JAK2 inhibitor but did not respond or later lost response (NCT06343805)..

AJ1-11095 是首个针对 JAK2 激酶 II 型构象、处于临床开发阶段的 JAK2 抑制剂。目前已获批的 JAK2 抑制剂均通过 I 型构象发挥作用。该疗法目前正在一项 1 期研究中进行评估，适用于先前接受过 I 型 JAK2 抑制剂但未产生应答或后来失去应答的患者 (NCT06343805)。

The designation marks a key step for the company as it progresses AJ1-11095 through clinical development.

这一指定标志着该公司在推进AJ1-11095的临床开发过程中迈出了关键一步。

The need for more effective treatment options remains significant, particularly for patients who no longer benefit from standard therapies.

对更有效的治疗选项的需求仍然很大，特别是对于那些不再从标准疗法中受益的患者。

Source: globenewswire.com

来源：globenewswire.com