Treatment with JNT-517 demonstrated a statistically significant mean blood phenylalanine reduction of 51% versus baseline JNT-517 was safe and well tolerated with no serious adverse events Jnana has adapted the ongoing trial to accelerate the program and aims to advance JNT-517 directly into a pivotal study in early 2025 BOSTON, Jan.

JNT-517治疗显示，与基线相比，平均血液苯丙氨酸降低了51%，具有统计学意义。JNT-517安全且耐受性良好，无严重不良事件。Jnana调整了正在进行的试验，以加速该计划，旨在将JNT-517直接推进2025年初的一项关键研究波士顿，1月。

30, 2024 (GLOBE NEWSWIRE) -- Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU).

2024年3月30日（环球通讯社）--Jnana Therapeutics是一家临床阶段的生物技术公司，利用其下一代化学蛋白质组学平台发现挑战药物靶标的药物，今天宣布了其正在进行的JNT-517临床研究的积极的，统计学上显着的中期结果。苯丙酮尿症（PKU）患者。

JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517.

JNT-517是苯丙氨酸（Phe）转运蛋白SLC6A19的小分子抑制剂，正在被评估为所有年龄和基因型的PKU潜在的一流口服治疗药物。在这些积极的中期结果的基础上，Jnana调整了1b期试验设计，以支持JNT-517加速进展的潜力。

“There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy. Across the spectrum of mild to severe disease, our results demonstrate a robust, sustained reduction in blood Phe levels, the registrational endpoint for PKU, giving us high confidence in the path forward for JNT-517,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics.

医学博士乔治·弗拉桑诺斯（GeorgeVratsanos）说：“对于目前尚未接受治疗的60%以上的北大患者，迫切需要一种口服，安全和有效的治疗方法。在轻度至重度疾病的范围内，我们的研究结果表明，血苯丙氨酸水平（PKU的注册终点）持续下降，这使我们对JNT-517的发展道路充满信心。，Jnana Therapeutics首席医疗官兼研发主管。

“We are also encouraged by this validation of the power of our RAPID platform to discover small molecules with compelling clinical benefit against challenging-to-drug targets.” JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe PKU. Following a 28-day.

“我们也对我们的RAPID平台发现小分子的能力感到鼓舞，这些小分子具有令人信服的临床益处，可以对抗药物靶标的挑战。”JNT-517正在一项针对轻度至重度PKU患者的随机，双盲，安慰剂对照试验中进行研究。28天后。