预约演示
更新于：2026-04-02
Mavodelpar
更新于：2026-04-02
概要
基本信息
非在研机构- |
最高研发阶段临床2期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评快速通道 (美国) |
登录后查看时间轴
结构/序列
分子式C31H30FNNaO5 |
InChIKeyWVHRCLAVADJBSR-MAOGHAJMSA-N |
CAS号1604815-32-8 |
关联
10
项与 Mavodelpar 相关的临床试验ISRCTN85791974
A Phase I, randomized, open-label, single-dose, three-period crossover bioequivalence and food effect study with orally administered mavodelpar tablet and capsule formulations in healthy subjects
CTIS2023-504007-91-00
A Phase 1, Open-Label, Single-Dose Study To Evaluate The Pharmacokinetics Of Mavodelpar In Subjects With Impaired Hepatic Function
ISRCTN12840543
A five-part, Phase I, open-label, drug-drug interaction study examining the effect of gemfibrozil, fluconazole and carbamazepine on the safety, tolerability and pharmacokinetics of REN001, and the effect of REN001 on the safety, tolerability and pharmacokinetics of fexofenadine and rosuvastatin in healthy subjects
100 项与 Mavodelpar 相关的临床结果
登录后查看更多信息
100 项与 Mavodelpar 相关的转化医学
登录后查看更多信息
100 项与 Mavodelpar 相关的专利（医药）
登录后查看更多信息
4
项与 Mavodelpar 相关的文献（医药）2023-03-01Kidney360
PPARδ Agonism Ameliorates Renal Fibrosis in an Alport Syndrome Mouse Model
Article
作者: Miner, Jeffrey H. ; Omachi, Kohei ; O'Carroll, Colin
Key Points:
A peroxisome proliferator-activated receptor δ agonist, REN001, ameliorates kidney dysfunction in a mouse model of Alport syndrome.REN001 suppresses glomerular injury and renal fibrosis.REN001 decreases the levels of inflammation- and fibrosis-related proteins.
Background:
Alport syndrome is a genetic kidney disease caused by mutation in any of the COL4A3, COL4A4, or COL4A5 genes encoding the type IV collagen α3, α4, and α5 chains. Defects of type IV collagen α3α4α5 cause glomerular basement membrane abnormalities and lead to defects in glomerular filtration and ESKD. Treatment with angiotensin-converting enzyme inhibitors (ACEis) dramatically slows disease progression but does not stop progression to renal failure. Therefore, novel therapeutic options with different modes of action from ACEis are needed. Peroxisome proliferator-activated receptor (PPAR) δ agonists have shown renoprotective effects in several acute kidney injury mouse models. In this study, we investigated the effects of a potent and selective PPARδ agonist, REN001 (formerly HPP593), in a mouse model of Alport syndrome.
Methods:
We administered REN001 from the early stages to the late stages of disease by once daily intraperitoneal injections.
Results:
REN001 treatment halved proteinuria at the late stages of disease in Col4a3
−/−
mice. BUN levels were also decreased, and histological and molecular analyses showed that REN001 ameliorated renal inflammation and fibrosis.
Conclusions:
These results indicate that REN001 slows kidney disease progression in Alport mice. REN001 has a different mechanism of action from ACEis, so we, therefore, hypothesize that combining the two treatments may show additive effects to attenuate renal injury and slow progression to renal failure.
2019-11-01Kidney international reports3区 · 医学
Sotatercept Safety and Effects on Hemoglobin, Bone, and Vascular Calcification
3区 · 医学
ArticleOA
作者: Smith, William T ; Giuseppi, Ana Carolina ; Hruska, Keith A ; Dellanna, Frank ; Malluche, Hartmut H ; Sherman, Matthew L ; Coyne, Daniel W ; Singh, Hem N ; Connarn, Jamie N
INTRODUCTION:
Patients with end-stage kidney disease (ESKD) exhibit anemia, chronic kidney disease‒mineral bone disorder (CKD-MBD), and cardiovascular disease. The REN-001 and REN-002 phase II, multicenter, randomized studies examined safety, tolerability, and effects of sotatercept, an ActRIIA-IgG1 fusion protein trap, on hemoglobin concentration; REN-001 also explored effects on bone mineral density (BMD) and abdominal aortic vascular calcification.
METHODS:
Forty-three patients were treated in REN-001 (dose range: sotatercept 0.3‒0.7 mg/kg or placebo subcutaneously [s.c.] for 200 days) and 50 in REN-002 (dose range: 0.1‒0.4 mg/kg i.v. and 0.13‒0.5 mg/kg s.c. for 99 days).
RESULTS:
In REN-001, frequency of achieving target hemoglobin response (>10 g/dl [6.21 mmol/l]) with sotatercept was dose-related and greater than placebo (0.3 mg/kg: 33.3%; 0.5 mg/kg: 62.5%; 0.7 mg/kg: 77.8%; 0.7 mg/kg [doses 1 and 2]/0.4 mg/kg [doses 3‒15]: 33.3%; placebo: 27.3%). REN-002 hemoglobin findings were similar (i.v.: 16.7%-57.1%; s.c.: 11.1%‒42.9%). Dose-related achievement of ≥2% increase in femoral neck cortical BMD was seen among only REN-001 patients receiving sotatercept (0.3‒0.7 mg/kg: 20.0%‒57.1%; placebo: 0.0%). Abdominal aortic vascular calcification was slowed in a dose-related manner, with a ≤15% increase in Agatston score achieved by more REN-001 sotatercept versus placebo patients (60%‒100% vs. 16.7%). The most common adverse events during treatment were hypertension, muscle spasm, headache, arteriovenous fistula site complication, and influenza observed in both treatment and placebo groups.
CONCLUSION:
In patients with ESKD, sotatercept exhibited a favorable safety profile and was associated with trends in dose-related slowing of vascular calcification. Less-consistent trends in improved hemoglobin concentration and BMD were observed.
Cells
Treatment of VLCAD-Deficient Patient Fibroblasts with Peroxisome Proliferator-Activated Receptor δ Agonist Improves Cellular Bioenergetics
Article
作者: Van’t Land, Clinton ; Vockley, Jerry ; Mohsen, Al-Walid ; Karunanidhi, Anuradha ; Dorenbaum, Alejandro ; D’Annibale, Olivia M. ; Phua, Yu Leng
Background: Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency is an autosomal recessive disease that prevents the body from utilizing long-chain fatty acids for energy, most needed during stress and fasting. Symptoms can appear from infancy through childhood and adolescence or early adulthood, and include hypoglycemia, recurrent rhabdomyolysis, myopathy, hepatopathy, and cardiomyopathy. REN001 is a peroxisome-proliferator-activated receptor delta (PPARδ) agonist that modulates the expression of the genes coding for fatty acid β-oxidation enzymes and proteins involved in oxidative phosphorylation. Here, we assessed the effect of REN001 on VLCAD-deficient patient fibroblasts. Methods: VLCAD-deficient patient and control fibroblasts were treated with REN001. Cells were harvested for gene expression analysis, protein content, VLCAD enzyme activity, cellular bioenergetics, and ATP production. Results: VLCAD-deficient cell lines responded differently to REN001 based on genotype. All cells had statistically significant increases in ACADVL gene expression. Small increases in VLCAD protein and enzyme activity were observed and were cell-line- and dose-dependent. Even with these small increases, cellular bioenergetics improved in all cell lines in the presence of REN001, as demonstrated by the oxygen consumption rate and ATP production. VLCAD-deficient cell lines containing missense mutations responded better to REN001 treatment than one containing a duplication mutation in ACADVL. Discussion: Treating VLCAD-deficient fibroblasts with the REN001 PPARδ agonist results in an increase in VLCAD protein and enzyme activity, and a decrease in cellular stress. These results establish REN001 as a potential therapy for VLCADD as enhanced expression may provide a therapeutic increase in total VLCAD activity, but suggest the need for mutation-specific treatment augmented by other treatment measures.
100 项与 Mavodelpar 相关的药物交易
登录后查看更多信息
研发状态
10 条进展最快的记录, 后查看更多信息
登录
| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 慢性进行性眼外肌麻痹 | 临床3期 | 澳大利亚 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 比利时 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 加拿大 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 丹麦 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 法国 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 德国 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 匈牙利 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 意大利 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 荷兰 | 2022-02-01 | |
| 慢性进行性眼外肌麻痹 | 临床3期 | 新西兰 | 2022-02-01 |
登录后查看更多信息
临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2/3期 | 155 | 衊網網鬱鑰範窪糧衊衊 = 鏇築壓糧齋艱遞淵繭齋 窪鹽醖鹽蓋觸範廠夢積 (積遞糧鏇衊顧鹹觸鹽顧, 獵窪鬱齋願憲顧壓淵製 ~ 廠衊糧鏇夢構選選鑰襯) 更多 | - | 2024-05-28 | |||
临床2期 | 213 | (Mavodelpar) | 繭鑰憲壓壓繭衊憲遞顧(壓選範獵範願淵獵衊夢) = 鏇簾遞願鹽觸積夢餘膚 範襯築簾廠蓋齋鏇壓顧 (艱壓蓋顧壓齋積醖糧壓, 積鑰觸鬱膚憲艱餘鬱鹽 ~ 窪憲糧廠膚壓蓋衊襯壓) 更多 | - | 2024-04-03 | ||
Placebo (Matched Placebo) | 繭鑰憲壓壓繭衊憲遞顧(壓選範獵範願淵獵衊夢) = 艱醖簾製顧齋鏇鏇獵艱 範襯築簾廠蓋齋鏇壓顧 (艱壓蓋顧壓齋積醖糧壓, 醖願繭鬱選壓範餘鏇範 ~ 觸遞鏇選糧衊夢憲鹹網) 更多 |
登录后查看更多信息
转化医学
使用我们的转化医学数据加速您的研究。
登录
或
药物交易
使用我们的药物交易数据加速您的研究。
登录
或
核心专利
使用我们的核心专利数据促进您的研究。
登录
或
临床分析
紧跟全球注册中心的最新临床试验。
登录
或
批准
利用最新的监管批准信息加速您的研究。
登录
或
特殊审评
只需点击几下即可了解关键药物信息。
登录
或
生物医药百科问答
全新生物医药AI Agent 覆盖科研全链路，让突破性发现快人一步
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。
生物序列数据库
生物药研发创新
免费使用
化学结构数据库
小分子化药研发创新
免费使用