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Part
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Kidney Drugs Research (A)
Key Takeaways
- Bardoxolone methyl aims to treat rare and genetic chronic kidney diseases (CKDs), including Autosomal dominant polycystic kidney disease (ADPKD) and Alport Syndrome.
- Tesevatinib works as a receptor tyrosine kinase inhibitor, mitigating the growth of cysts in ADKPD.
- RGLS4326 specifically targets the miR-17, the microRNA molecule responsible for PKD1 and PKD2 protein production, to reduce kidney cyst formation, cyst cell growth, preserve kidney function, and direct regulation of PKD1 and PKD2 protein production in animal trials involving ADKPD.
Introduction
Columns A to E and rows 14-16 of the attached spreadsheet provide data regarding novel pharmaceutical drugs in the clinical or preclinical trial stage for kidney diseases. The data provided did not include insights for preclinical drugs, as those available are repurposed drugs. Bardoxolone methyl, Tesevatinib, and RGLS4326 / RGLS8429 were the three drugs provided in different clinical trial stages. Further information regarding why some information was unavailable or not included has been provided in the Research Strategy section. Provided below are some selected findings.
Selected Findings
- Bardoxolone methyl aims to treat rare and genetic chronic kidney diseases (CKDs), including Autosomal dominant polycystic kidney disease (ADPKD) and Alport Syndrome.
- A pharmaceutical company called Reata Pharmaceuticals is responsible for developing bardoxolone methyl.
- Tesevatinib works as a receptor tyrosine kinase inhibitor, mitigating the growth of cysts in ADKPD. Kadmon Corporation is developing this drug to treat ADKPD and other indications where epidermal growth factor receptor (EGFR) inhibition could affect the disease progression.
- RGLS4326 specifically targets the miR-17, the microRNA molecule responsible for PKD1 and PKD2 protein production, to reduce kidney cyst formation, cyst cell growth, preserve kidney function, and direct regulation of PKD1 and PKD2 protein production in animal trials involving ADKPD.
- Regulus Therapeutics first studied its RGLS4326 compound but announced that it would stop investing in that compound. The company's statement read, "we have determined that advancing our next-generation compound RGLS8429 is more compelling than further development of RGLS4326."
Research Strategy
We analyzed the ClinicalTrials.gov website and database to identify the requested information. However, we did not find specific data highlighting novel drugs. Most data available referenced repurposed drugs and similar and could not be included in the report. We also analyzed medical journals such as the American Journal of Kidney Diseases (AJKD) and the US Food and Drug Administration (FDA) to obtain similar insights. We either found previously outlined info or insights that did not fulfill the criteria for selection. However, we provided the drugs in the report from the research pipeline provided by Polycystic Kidney Disease (PKD) Foundation. Although the pipeline included multiple drugs for CKD, those in the preclinical trial stage were repurposed drugs. However, we included them in the image provided. Additionally, some other drug developments were either discontinued or did not have studies in the US. Further research would unlikely reveal additional results based on the selection criteria.
Lastly, we included a mixture of biotech and pharmaceutical companies. However, we did not include the startup available because the company discontinued its drug development.