PTC299 is an orally active inhibitor of VEGFA mRNA translation that selectively inhibits VEGF protein synthesis at the post-transcriptional level. PTC299 is also a potent inhibitor of dihydroorotate dehydrogenase (DHODH). PTC299 shows good oral bioavailability and lack of off-target kinase inhibition and myelosuppression. PTC299 can be useful for the research of hematologic malignancies^[1][2].

VEGF^[2]

PTC299 inhibits hypoxia-induced VEGFA protein production in HeLa cells with an EC₅₀ of 1.64 ± 0.83 nM^[1].
PTC299 is the most potent inhibitor with an IC₅₀ of about 1 nM, over 10 to 1000-fold more potent than Brequinar, Vidofludimus or A 77-1726 in leukemia cells^[1].

上海金畔生物科技有限公司 has not independently confirmed the accuracy of these methods. They are for reference only.

467.34

C₂₅H₂₀Cl₂N₂O₃

1256565-36-2

Room temperature in continental US; may vary elsewhere.

DMSO : 50 mg/mL (106.99 mM; Need ultrasonic)

请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；一旦配成溶液，请分装保存，避免反复冻融造成的产品失效。
储备液的保存方式和期限：-80°C, 6 months; -20°C, 1 month。-80°C 储存时，请在 6 个月内使用，-20°C 储存时，请在 1 个月内使用。

请根据您的实验动物和给药方式选择适当的溶解方案。以下溶解方案都请先按照 In Vitro 方式配制澄清的储备液，再依次添加助溶剂：

——为保证实验结果的可靠性，澄清的储备液可以根据储存条件，适当保存；体内实验的工作液，建议您现用现配，当天使用； 以下溶剂前显示的百
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• 1.

  请依序添加每种溶剂： 10% DMSO    40% PEG300    5% Tween-80    45% saline

  Solubility: ≥ 0.83 mg/mL (1.78 mM); Clear solution

  此方案可获得 ≥ 0.83 mg/mL (1.78 mM，饱和度未知) 的澄清溶液。

  以 1 mL 工作液为例，取 100 μL 8.3 mg/mL 的澄清 DMSO 储备液加到 400 μL PEG300 中，混合均匀；向上述体系中加入50 μL Tween-80，混合均匀；然后继续加入 450 μL生理盐水定容至 1 mL。

  将 0.9 g 氯化钠，完全溶解于 100 mL ddH₂O 中，得到澄清透明的生理盐水溶液

• 2.

  请依序添加每种溶剂： 10% DMSO    90% corn oil

  Solubility: ≥ 0.83 mg/mL (1.78 mM); Clear solution

  此方案可获得 ≥ 0.83 mg/mL (1.78 mM，饱和度未知) 的澄清溶液，此方案不适用于实验周期在半个月以上的实验。

  以 1 mL 工作液为例，取 100 μL 8.3 mg/mL 的澄清 DMSO 储备液加到 900 μL玉米油中，混合均匀。

• [1]. Cao L, et al. Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of DihydroorotateDehydrogenase with Favorable Pharmaceutical Properties. Mol Cancer Ther. 2019 Jan;18(1):3-16.

  [2]. Bender Ignacio RA, et al. Brief Report: A Phase 1b/Pharmacokinetic Trial of PTC299, a Novel PostTranscriptional VEGF Inhibitor, for AIDS-Related Kaposi’s Sarcoma: AIDS Malignancy Consortium Trial 059. J Acquir Immune Defic Syndr. 2016 May 1;72(1):52-7.

  [3]. Packer RJ, et al. Phase I and pharmacokinetic trial of PTC299 in pediatric patients with refractory or recurrent central nervous system tumors: a PBTC study. J Neurooncol. 2015;121(1):217-224.

  [4]. Zeng Z, et al, Targeting dihydroorotate dehydrogenase in acute myeloid leukemia. Haematologica. 2018 Sep;103(9):1415-1417.