更生霉素 | 50-76-0

• 物质功能分类: 原料药 - 抗肿瘤药 - 抗生素类抗肿瘤药
• 分子结构分类: 有机化合物 - 有机酸及其衍生物 - 肽模拟物
• 中文名称: 更生霉素
• 中文别名: 更生霉素D；放线菌素D；放线菌素C1；硫化剂双-25；放线菌素 D；放线菌素
• 英文名称: actinomycin D
• 英文别名: dactinomycin；ActD；actinomycin C₁；actinomycin IV；Cosmegen；2-amino-4,6-dimethyl-3-oxo-1-N,9-N-bis[(3R,6S,7R,10S,16S)-7,11,14-trimethyl-2,5,9,12,15-pentaoxo-3,10-di(propan-2-yl)-8-oxa-1,4,11,14-tetrazabicyclo[14.3.0]nonadecan-6-yl]phenoxazine-1,9-dicarboxamide
• CAS: 50-76-0
• 化学式: C₆₂H₈₆N₁₂O₁₆
• 分子量: 1255.44
• InChiKey: RJURFGZVJUQBHK-IIXSONLDSA-N

物化性质

• 熔点：
  251-253 °C
• 沸点：
  848°C (rough estimate)
• 密度：
  1.0757 (rough estimate)
• 闪点：
  87℃
• 溶解度：
  乙醇、DMSO：在 2-8 °C 的水溶液中稳定。可溶
• 物理描述：
  Actinomycin d appears as bright red rhomboid prisms or red powder. (NTP, 1992)
• 颜色/状态：
  Bright red crystalline powder
• 蒸汽压力：
  0 mm Hg at 25 °C (est)
• 稳定性/保质期：
  Dilute soln are very sensitive to light /Trihydrate/
• 分解：
  When heated to decomposition it emits toxic fumes of /nitrogen oxides/.
• 解离常数：
  pKa = 11.90
• 碰撞截面：
  341.2 Ų [M+H]+ [CCS Type: TW, Method: calibrated with polyalanine and drug standards]

计算性质

• 辛醇/水分配系数(LogP)：
  3.8
• 重原子数：
  90
• 可旋转键数：
  8
• 环数：
  7.0
• sp3杂化的碳原子比例：
  0.61
• 拓扑面积：
  356
• 氢给体数：
  5
• 氢受体数：
  18

ADMET

代谢

肝脏的

hepatic

来源:DrugBank

代谢

达克替康似乎只有轻微代谢；在尿液中检测到了少量药物的单内酯。

Dactinomycin appears to be only slightly metabolized; small amounts of monolactones of the drug have been detected in the urine.

来源:Hazardous Substances Data Bank (HSDB)

毒理性

• 肝毒性

多柔比星与其他药物联合化疗与血清酶水平升高有关，升高的比例因剂量、联合使用的其他药物、监测频率以及用于定义升高的标准而异。ALT升高通常是无需调整剂量的无症状和短暂的，可能自行缓解。在许多情况下，由于暴露于其他潜在肝毒性药物，很难将肝功能测试异常归因于多柔比星。 多柔比星还可能引起一种被称为肝病变-血小板减少症综合征（HTS）的独特形式的临床明显肝损伤，这种综合征可能是严重甚至致命的。这种综合征似乎是由于窦状隙阻塞，但也可能存在直接肝和骨髓损伤的成分。在大规模研究中，接受包括多柔比星方案治疗的癌症儿童中，有1%到5%出现了急性肝损伤和提示HTS的血小板减少。这种综合征在年龄较小的儿童和高剂量多柔比星治疗中更为常见。发病时间通常在首次给药后3到6周内，常在第二个或第三个周期性化疗疗程后的5到10天内出现。症状的出现是突然的，儿童典型表现为右上象限疼痛或压痛、肝肿大、肝功能测试异常和过度出血的迹象，如鼻出血或瘀伤。血清转氨酶水平在病程早期显著升高（高于正常上限的10到100倍），但迅速下降，7到14天内可能恢复正常。血小板计数通常低于25,000/μL，也会迅速恢复。血清碱性磷酸酶通常正常，胆红素水平仅略有升高，除非病程进展并导致死亡。血清氨和INR也可能升高，急性综合征期间常发生腹水。损伤的整体模式类似于急性肝坏死，肝脏组织学显示中央小叶坏死和窦状隙阻塞的证据。恢复是迅速且通常是完全的。 可能性评分：C（临床明显肝损伤的可能原因）。

Chemotherapy with dactinomycin in combination with other agents is associated with serum enzyme elevations in a high, but variable proportion of patients depending upon the dose, other agents used as well as the frequency of monitoring and criteria used to define elevations. The ALT elevations are usually asymptomatic and transient and may resolve without dose modification. In many instances, it is difficult to attribute the liver test abnormalities to dactinomycin, because of the exposure to other potentially hepatotoxic agents. Dactinomycin can also cause a distinctive form of clinically apparent liver injury referred to as hepatopathy-thrombocytopenia syndrome (HTS) that can be severe and even fatal. This syndrome appears to be due to sinusoidal obstruction, but there may also be an element of direct hepatic and bone marrow injury. In large studies, between 1% and 5% of children with cancer treated with regimens including dactinomycin developed acute hepatic injury and thrombocytopenia suggestive of HTS. The syndrome is more common in younger children and with higher doses of dactinomycin. The time to onset is typically within 3 to 6 weeks after the initial dose, often arising 5 to 10 days after the 2nd or 3rd course of cyclic chemotherapy with dactinomycin. The onset of symptoms is sudden, and children characteristically present with right upper quadrant pain or tenderness, hepatomegaly, liver test abnormalities and signs of excessive bleeding such as epistaxis or bruising. Serum aminotransferase levels are markedly elevated (10 to 100 times ULN) early in the course, but fall rapidly and can be normal within 7 to 14 days. The platelet count is generally less than 25,000/μL and also resolves rapidly. Serum alkaline phosphatase is typically normal and bilirubin levels are minimally elevated, except if the course is progressive and fatal. Serum ammonia and INR may also be raised, and ascites often develops during the acute syndrome. The overall pattern of injury resembles acute hepatic necrosis, and liver histology shows centrolobular necrosis and evidence of sinusoidal obstruction. Recovery is rapid and usually complete. Likelihood score: C (Probable cause of clinically apparent liver injury).

来源:LiverTox

毒理性

• 药物性肝损伤

化合物：放线菌素

Compound:dactinomycin

来源:Drug Induced Liver Injury Rank (DILIrank) Dataset

毒理性

• 药物性肝损伤

药物性肝损伤标注：最令人关注的药物性肝损伤

DILI Annotation:Most-DILI-Concern

来源:Drug Induced Liver Injury Rank (DILIrank) Dataset

毒理性

• 药物性肝损伤

严重程度等级：8

Severity Grade:8

来源:Drug Induced Liver Injury Rank (DILIrank) Dataset

毒理性

• 药物性肝损伤

标签部分：警告和预防措施

Label Section:Warnings and precautions

来源:Drug Induced Liver Injury Rank (DILIrank) Dataset

吸收、分配和排泄

• 吸收

消化道吸收不良

poorly absorbed from gastrointestinal tract

来源:DrugBank

吸收、分配和排泄

链霉素从胃肠道吸收不良。该药物对组织极为刺激，因此必须静脉给药。

Dactinomycin is poorly absorbed from the GI tract. The drug is extremely irritating to tissues and, therefore, must be administered iv.

来源:Hazardous Substances Data Bank (HSDB)

吸收、分配和排泄

放线菌素D迅速分布到组织中，在骨髓和带核细胞（包括粒细胞和淋巴细胞）中浓度较高。该药物似乎很难穿越血脑屏障，或者根本不能穿越。

Dactinomycin is rapidly distributed into tissues, with high concentrations in bone marrow and nucleated cells, including granulocytes and lymphocytes. The drug appears to cross the blood-brain barrier poorly, if at all.

来源:Hazardous Substances Data Bank (HSDB)

吸收、分配和排泄

“血浆蛋白结合/达克替尼霉素/为5%。”

Plasma protein binding /of dactinomycin/ is 5%.

来源:Hazardous Substances Data Bank (HSDB)

吸收、分配和排泄

显然，放线菌素D会穿过胎盘。目前尚不清楚放线菌素D是否会分布到乳汁中。

Dactinomycin apparently crosses the placenta. It is not known if dactinomycin is distributed into milk.

来源:Hazardous Substances Data Bank (HSDB)

安全信息

• 危险等级：
  6.1(a)
• 危险品标志：
  T+
• 安全说明：
  S1,S22,S28,S36/37,S36/37/39,S45,S53
• 危险类别码：
  R40,R28,R61
• WGK Germany：
  3
• 海关编码：
  29419000
• RTECS号：
  AU1575000
• 包装等级：
  II
• 危险类别：
  6.1(a)
• 危险品运输编号：
  UN 3462 6.1/PG 2
• 储存条件：
  密封于4℃干燥避光条件下保存。

SDS

Name:	Actinomycin D Material Safety Data Sheet
Synonym:	Dactinomycin
CAS:	50-76-0

Section 1 - Chemical Product MSDS Name:Actinomycin D Material Safety Data Sheet
Synonym:Dactinomycin

---

Section 2 - COMPOSITION, INFORMATION ON INGREDIENTS

CAS#	Chemical Name	content	EINECS#
50-76-0	Actinomycin D	100.0	200-063-6

Hazard Symbols: T+
Risk Phrases: 28

---

Section 3 - HAZARDS IDENTIFICATION
EMERGENCY OVERVIEW
Very toxic if swallowed.Light sensitive.
Potential Health Effects
Eye:
May cause severe eye irritation. May cause eye injury.
Skin:
May cause skin sensitization, an allergic reaction, which becomes evident upon re-exposure to this material.
Ingestion:
May be fatal if swallowed. May cause gastrointestinal irritation with nausea, vomiting and diarrhea.
Inhalation:
May cause respiratory tract irritation. May cause effects similar to those described for ingestion. Inhalation may produce coughing, nausea, and pulmonary edema.
Chronic:
Repeated exposure may cause sensitization dermatitis.

---

Section 4 - FIRST AID MEASURES
Eyes: Immediately flush eyes with plenty of water for at least 15 minutes, occasionally lifting the upper and lower eyelids. Get medical aid immediately.
Skin:
Get medical aid if irritation develops or persists. Wash clothing before reuse. Flush skin with plenty of soap and water. Destroy contaminated shoes.
Ingestion:
Call a poison control center. If swallowed, do not induce vomiting unless directed to do so by medical personnel. Never give anything by mouth to an unconscious person. Get medical aid.
Inhalation:
Remove from exposure and move to fresh air immediately. If not breathing, give artificial respiration. If breathing is difficult, give oxygen. Get medical aid if cough or other symptoms appear. Do NOT use mouth-to-mouth resuscitation.
Notes to Physician:
Treat symptomatically and supportively.

---

Section 5 - FIRE FIGHTING MEASURES
General Information:
As in any fire, wear a self-contained breathing apparatus in pressure-demand, MSHA/NIOSH (approved or equivalent), and full protective gear. During a fire, irritating and highly toxic gases may be generated by thermal decomposition or combustion.
Extinguishing Media:
Use water spray, dry chemical, carbon dioxide, or chemical foam.

---

Section 6 - ACCIDENTAL RELEASE MEASURES
General Information: Use proper personal protective equipment as indicated in Section 8.
Spills/Leaks:
Wash area with soap and water. Clean up spills immediately, observing precautions in the Protective Equipment section. Sweep up, then place into a suitable container for disposal. Avoid generating dusty conditions. Provide ventilation.

---

Section 7 - HANDLING and STORAGE
Handling:
Wash thoroughly after handling. Use with adequate ventilation.
Minimize dust generation and accumulation. Avoid contact with eyes, skin, and clothing. Keep container tightly closed. Avoid ingestion and inhalation.
Storage:
Do not store in direct sunlight. Store in a tightly closed container. Store in a cool, dry, well-ventilated area away from incompatible substances. Keep refrigerated. (Store below 4C/39F.)

---

Section 8 - EXPOSURE CONTROLS, PERSONAL PROTECTION
Engineering Controls:
Facilities storing or utilizing this material should be equipped with an eyewash facility and a safety shower. Use adequate ventilation to keep airborne concentrations low. Use only under a chemical fume hood.
Exposure Limits CAS# 50-76-0: Personal Protective Equipment Eyes: Wear appropriate protective eyeglasses or chemical safety goggles as described by OSHA's eye and face protection regulations in 29 CFR 1910.133 or European Standard EN166.
Skin:
Wear appropriate protective gloves to prevent skin exposure.
Clothing:
Wear appropriate protective clothing to prevent skin exposure.
Respirators:
Follow the OSHA respirator regulations found in 29 CFR 1910.134 or European Standard EN 149. Use a NIOSH/MSHA or European Standard EN 149 approved respirator if exposure limits are exceeded or if irritation or other symptoms are experienced.

---

Section 9 - PHYSICAL AND CHEMICAL PROPERTIES

Physical State: Powder
Color: red
Odor: none reported
pH: Not available.
Vapor Pressure: Not available.
Viscosity: Not available.
Boiling Point: Not available.
Freezing/Melting Point: 245-252C
Autoignition Temperature: Not applicable.
Flash Point: 241 deg C ( 465.80 deg F)
Explosion Limits, lower: Not available.
Explosion Limits, upper: Not available.
Decomposition Temperature: 250 deg C
Solubility in water: In methanol
Specific Gravity/Density:
Molecular Formula: C62H86N12O16
Molecular Weight: 1255.5

---

Section 10 - STABILITY AND REACTIVITY
Chemical Stability:
Stable under normal temperatures and pressures.
Conditions to Avoid:
Incompatible materials, light, dust generation, excess heat.
Incompatibilities with Other Materials:
Strong oxidizing agents, strong bases, strong acids Hazardous Decomposition Products:
Nitrogen oxides, carbon monoxide, irritating and toxic fumes and gases, carbon dioxide.
Hazardous Polymerization: Has not been reported

---

Section 11 - TOXICOLOGICAL INFORMATION
RTECS#:
CAS# 50-76-0: AU1575000 LD50/LC50:
CAS# 50-76-0: Oral, mouse: LD50 = 13 mg/kg; Oral, mouse: LD50 = 20 mg/kg; Oral, rat: LD50 = 7200 ug/kg.
Carcinogenicity:
Actinomycin D - California: carcinogen, initial date 10/1/89 Other:
See actual entry in RTECS for complete information.

---

Section 12 - ECOLOGICAL INFORMATION

---

Section 13 - DISPOSAL CONSIDERATIONS
Products which are considered hazardous for supply are classified as Special Waste and the disposal of such chemicals is covered by regulations which may vary according to location. Contact a specialist disposal company or the local waste regulator for advice. Empty containers must be decontaminated before returning for recycling.

---

Section 14 - TRANSPORT INFORMATION

IATA
No information available.
IMO
No information available.
RID/ADR
No information available.

---

Section 15 - REGULATORY INFORMATION

European/International Regulations
European Labeling in Accordance with EC Directives
Hazard Symbols: T+
Risk Phrases:
R 28 Very toxic if swallowed.
Safety Phrases:
S 1 Keep locked up.
S 45 In case of accident or if you feel unwell, seek
medical advice immediately (show the label where
possible).
WGK (Water Danger/Protection)
CAS# 50-76-0: 3
Canada
None of the chemicals in this product are listed on the DSL/NDSL list.
CAS# 50-76-0 is not listed on Canada's Ingredient Disclosure List.
US FEDERAL
TSCA
CAS# 50-76-0 is not listed on the TSCA inventory.
It is for research and development use only.

---

SECTION 16 - ADDITIONAL INFORMATION
N/A

制备方法与用途

化学性质
鲜红色结晶性粉末，具有吸湿性，在光照下逐渐失去活性。其比旋光度为[α]18D -315°（0.02%，甲醇）。该物质不溶于石油醚，微溶于水，能溶解于甲醇、乙醇及乙酸乙酯，并且易溶于苯、氯仿和丙酮。剧毒，大鼠经口LD50为7200 μg/kg。

用途
干扰素诱导剂，临床用于抗肿瘤药物。该品对恶性葡萄胎疗效显著，同时对肾母细胞瘤、何杰金氏病、睾丸肿瘤、横纹肌肉瘤、神经母细胞瘤和网状细胞肉瘤也有效。此外，它对宫颈癌、卵巢癌、乳腺癌和消化道癌也有一定的治疗效果。与放疗联合应用可以提高放疗的敏感性。

用途
硫化剂，用于硅橡胶、聚氨酯橡胶、乙丙胶和其他橡胶；交联剂，可用于聚乙烯；不饱和聚酯固化剂。作为乙烯基硅橡胶有效的高温硫化剂，它是聚乙烯橡胶、乙丙橡胶的交联剂，能提高制品的拉伸强度和硬度，同时保持较低的伸长率及压缩变形，在烷基过氧化物中硫化后的气味是最轻微的。

生产方法
该产品是由放线菌产生的多肽抗生素。我国从桂林土壤中分离出Str. melanochromogehes菌株的发酵液中提取的产品称更生霉素，与国外报道的放线菌素D相同。

生产方法
使用产黑色素链霉菌培养液为原料，经过提取、酸性氧化铝柱层析、减压浓缩和石油醚重结晶制得。

类别
有毒物品

毒性分级
剧毒

急性毒性
口服-大鼠 LD50:7.2 毫克/公斤；口服-小鼠 LD50: 13 毫克/公斤

刺激数据
皮肤-兔 5 毫克/24小时 阳性

可燃性危险特性
可燃；燃烧时释放有毒氮氧化物烟雾；药物副作用：皮炎、骨髓伤害、消化系统受影响

储运特性
库房应通风低温干燥，并与食品原料分开存放

灭火剂
干粉、泡沫、砂土、二氧化碳，以及雾状水

反应信息

• 作为反应物：
  描述：

  更生霉素 在 platinum(IV) oxide 氢气 作用下， 以 甲醇 为溶剂， 生成
  参考文献：
  名称：

  放线菌素D的四环发色类似物：合成，结构阐明和从一种形式到另一种形式的相互转化，抗肿瘤活性和构效关系。

  摘要：

  放线菌素D的两个不同的四环发色类似物通过将放线菌素D中的两个发色DNA结合功能（即2-氨基和3-氧代）结合到1,4-恶嗪-2-酮或恶唑环系统中而合成。第三类似物在恶唑类似物的C-8处具有额外的醌功能。在所有类似物中，母体抗生素的肽内酯的化学完整性均保持不变，但其立体化学改变。该类似物被设计为放线菌素D的三环活性类似物或放线菌素D本身的运输修饰的前药形式。所有类似物都具有比AMD弱几倍的细胞毒性。它们对细胞外DNA也没有结合亲和力。尽管如此，第一和第三系列的类似物显示出改善的抗肿瘤活性（P388白血病，CDF1小鼠）。实际上，在恶嗪酮环的C-3位或8-氧代-8H-恶唑环的C-2位具有苯基取代基的这些类似物中的两个具有最高的抗肿瘤作用。大多数类似物在比放线菌素D更大的剂量范围内具有活性，并且在体外对人淋巴细胞白血病（CCFR-CEM）细胞的细胞毒性低6至16倍。具有最显着的抗肿瘤活性的类似物是

  DOI：

  10.1021/jm00365a015
• 作为产物：
  描述：

  (3-hydroxy-4-methyl-2-nitrobenzoyl)-L-threonyl-D-valyl-L-prolyl-sarcosyl-N-methyl-L-valine lactone 在 palladium on activated charcoal 氢气 、 potassium hexacyanoferrate(III) 作用下， 生成 更生霉素
  参考文献：
  名称：

  溶液和固态放线菌素相关肽内酯的构象和二聚化

  摘要：

  制备内酯 peptidiques 明显 a l'actinomycine et des actinomycinesrespondantes。Etude par RMN de 1 H des proprietes en solution de ces composes。结构结晶

  DOI：

  10.1021/ja00310a065

文献信息

• [EN] ACC INHIBITORS AND USES THEREOF<br/>[FR] INHIBITEURS DE L'ACC ET UTILISATIONS ASSOCIÉES
  申请人：GILEAD APOLLO LLC

  公开号：WO2017075056A1

  公开（公告）日：2017-05-04

  The present invention provides compounds I and II useful as inhibitors of Acetyl CoA Carboxylase (ACC), compositions thereof, and methods of using the same.

  本发明提供了化合物I和II，这些化合物可用作乙酰辅酶A羧化酶（ACC）的抑制剂，以及它们的组合物和使用方法。
• [EN] COMPOUNDS AND COMPOSITIONS COMPRISING CDK INHIBITORS AND METHODS FOR THE TREATMENT OF CANCER<br/>[FR] COMPOSÉS ET COMPOSITIONS COMPRENANT DES INHIBITEURS DES CDK ET MÉTHODES DE TRAITEMENT DU CANCER
  申请人：UNIV GEORGIA STATE RES FOUND

  公开号：WO2010129858A1

  公开（公告）日：2010-11-11

  Disclosed herein are compounds suitable for use as antitumor agents, methods for treating cancer wherein the disclosed compounds are used in making a medicament for the treatment of cancer, methods for treating a tumor comprising, administering to a subject a composition comprising one or more of the disclosed cytotoxic agents, and methods for preparing the disclosed antitumor agents.

  本文披露了适用作抗肿瘤药剂的化合物，用于治疗癌症的方法，其中所披露的化合物用于制备治疗癌症的药物，治疗肿瘤的方法包括向受试者施用包含一种或多种所披露的细胞毒性药剂的组合物，以及制备所披露的抗肿瘤药剂的方法。
• Cobalamin conjugates for anti-tumor therapy
  申请人：Weinshenker M. Ned

  公开号：US20050054607A1

  公开（公告）日：2005-03-10

  The present invention provides a cobalamin-drug conjugate suitable for the treatment of tumor related diseases. Cobalamin is indirectly covalently bound to an anti-tumor drug via a cleavable linker and one or more optional spacers. Cobalamin is covalently bound to a first spacer or the cleavable linker via the 5′-OH of the cobalamin ribose ring. The drug is bound to a second spacer of the cleavable linker via an existing or added functional group on the drug. After administration, the conjugate forms a complex with transcobalamin (any of its isoforms). The complex then binds to a receptor on a cell membrane and is taken up into the cell. Once in the cell, an intracellular enzyme cleaves the conjugate thereby releasing the drug. Depending upon the structure of the conjugate, a particular class or type of intracellular enzyme affects the cleavage. Due to the high demand for cobalamin in growing cells, tumor cells typically take up a higher percentage of the conjugate than do normal non-growing cells. The conjugate of the invention advantageously provides a reduced systemic toxicity and enhanced efficacy as compared to a corresponding free drug.

  本发明提供了一种适用于治疗肿瘤相关疾病的钴胺素-药物结合物。钴胺素通过可切割的连接剂间接共价结合到抗肿瘤药物上，还可以通过一个或多个可选的间隔物。钴胺素通过其核糖环的5'-OH与第一间隔物或可切割连接剂共价结合。药物通过其现有或添加的功能基团与可切割连接剂的第二间隔物结合。在给药后，结合物与转钴胺素（其任何同工异构体）形成复合物。然后，该复合物结合到细胞膜上的受体并被细胞摄取。一旦进入细胞，细胞内酶将切割结合物，从而释放药物。根据结合物的结构，特定类别或类型的细胞内酶影响切割。由于生长细胞对钴胺素的需求量较高，肿瘤细胞通常摄取结合物的比例高于正常非生长细胞。本发明的结合物与相应的游离药物相比，具有较低的全身毒性和增强的疗效。
• [EN] 2-QUINOLONE DERIVED INHIBITORS OF BCL6<br/>[FR] INHIBITEURS DE BCL6 DÉRIVÉS DE 2-QUINOLONE
  申请人：CANCER RESEARCH TECH LTD

  公开号：WO2018215798A1

  公开（公告）日：2018-11-29

  The present invention relates to compounds of formula I that function as inhibitors of BCL6(B- cell lymphoma 6) activity: Formula I wherein X1, X2, X3, R1, R2, R3, R4 and R5 are each as defined herein. The present invention also relates to processes for the preparation of these compounds, to pharmaceutical compositions comprising them, and to their use in the treatment of proliferative disorders, such as cancer,as well as other diseases or conditions in which BCL6 activity is implicated.

  本发明涉及作为BCL6（B细胞淋巴瘤6）活性抑制剂的I式化合物：式中X1、X2、X3、R1、R2、R3、R4和R5分别如本文所定义。本发明还涉及制备这些化合物的方法，包括含有它们的药物组合物，以及它们在治疗增生性疾病（如癌症）以及其他BCL6活性所涉及的疾病或病况中的用途。
• [EN] SUBSTITUTED N-HETEROCYCLIC CARBOXAMIDES AS ACID CERAMIDASE INHIBITORS AND THEIR USE AS MEDICAMENTS<br/>[FR] CARBOXAMIDES N-HÉTÉROCYCLIQUES SUBSTITUÉS UTILISÉS EN TANT QU'INHIBITEURS DE LA CÉRAMIDASE ACIDE ET LEUR UTILISATION EN TANT QUE MÉDICAMENTS
  申请人：BIAL BIOTECH INVEST INC

  公开号：WO2021055627A1

  公开（公告）日：2021-03-25

  The invention provides substituted N-heterocyclic carboxamides and related compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat a medical disorder, e.g., cancer, lysosomal storage disorder, neurodegenerative disorder, inflammatory disorder, in a patient.

  这项发明提供了替代的N-杂环羧酰胺和相关化合物，含有这些化合物的组合物，医疗工具包，以及使用这些化合物和组合物治疗患者的医疗疾病（例如癌症、溶酶体贮积症、神经退行性疾病、炎症性疾病）的方法。