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12 个首次披露的小分子在新药研究领域的前景汇总

2025-04-16     来源:本站     点击次数:271

在落幕的 ACS Spring 2025 (美国化学会春季年会) 上,多个创新药物首次披露,涵盖了肿瘤、心血管、神经系统疾病等多个领域。

这些小分子药物代表了当下最前沿的药物化学设计趋势。这些首次亮相的“新星”是否有望成为下一代重磅新药?跟随小 M 一起来看看它们的背景信息。

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Section
小结

20250327公众号-熔解曲线与 Ct 值-05.png

这 12 个首次亮相的小分子药物,不仅展示了目前药物研发的多样化方向 (从传统抑制剂到蛋白降解剂、共价靶向剂、大环肽等),也反映出精准医疗背景下对突变蛋白、肿瘤微环境、脑部靶点等复杂机制的深入探索。部分分子已经进入临床阶段,值得持续关注。
 

产品推荐

FG-2101 (HY-172734)

LpxC 抑制剂

PRT3789 (HY-172735)

SMARCA2 选择性降解剂

BMS-986458 (HY-172736)

BCL6 降解剂

RP-1664 (HY-172737)

PLK4 抑制剂

BMS-986238 (HY-172320)

环肽 PD-L1 抑制剂

ALKS 2680 (HY-172412)

OX2R 激动剂

IAMA-6 (HY-172318)

NKCC1 抑制剂

ORIC-114 (HY-172429)

EGFR/HER2 exon 20 抑制剂

AZD2389 (HY-172317)

FAP 抑制剂

PF-07853578 (HY-163959)

PNPLA3 I148M 共价抑制剂

CK-4021586 (HY-159821)

心脏肌球蛋白抑制剂

BAY 3389934 (HY-172316)

双凝血因子 Factor IIa/Xa 抑制剂

Omecamtiv mecarbil (HY-14233)

心脏特异性肌球蛋白 (cardiac myosin) 激活剂

 

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[2] Koichi Ito, et al. Abstract B113: Discovery of PRT3789, a first-in-class potent and selective SMARCA2 degrader in clinical trials for the treatment of patients with SMARCA4 mutated cancers. Mol Cancer Ther 1 December 2023; 22 (12_Supplement): B113. 
[3] Lynda Groocock, et al. BMS-986458 a Potential First-in-Class, Highly Selective, Potent and Well Tolerated BCL6 Ligand Directed Degrader (LDD) Demonstrates Multi-Modal Anti-Tumor Efficacy for the Treatment of B-Cell Non-Hodgkin's Lymphoma. Blood. 2024 Volume 144, Supplement 1, Page 957.
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[7] Marco Borgogno, et al. Discovery of IAMA-6: A selective inhibitor of NKCC1 and clinical candidate to treat brain disorders. ACS. 2025 Mar 26.
[8] Hong, M.H, et al. A global phase 1b study of ORIC-114, a highly selective, brain penetrant EGFR and HER2 inhibitor, in patients with advanced solid tumors harboring EGFR Exon 20 or HER2 alterations. Annals of Oncology, Volume 34, S769.
[9] Anneli Nordqvist, et al. AZD2389, a first in class candidate drug for the treatment of metabolic dysfunction-associated steatohepatitis. ACS. 2025 Mar 26.
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[11] Sankar Sarkar, et al. Preclinical characterization of CK-4021586, a new class of cardiac myosin inhibitors for the treatment of hypertrophic cardiomyopathy. Biophysical Journal, Volume 122, Issue 3, 122a.
[12] Robert Silasi, et al. Treatment with a Novel Small Molecule Dual Factor IIa/Xa Inhibitor Protects Against Coagulopathy and Organ Dysfunction in a Baboon Model of Staphylococcus Aureus Sepsis. Blood. 2024 Volume 144, Supplement 1, Page 3988.

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