Farabursen
| Clinical data | |
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| Synonyms | RGLS8429 |
| Routes of administration | Subcutaneous injection |
| Legal status | |
| Legal status |
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| E number | {{#property:P628}} |
| ECHA InfoCard | {{#property:P2566}} |
Farabursen (development code RGLS8429) is an investigational oligonucleotide drug developed by Regulus Therapeutics for the treatment of autosomal dominant polycystic kidney disease (ADPKD).[1][2] The drug is designed to inhibit microRNA-17 (miR-17) and preferentially targets the kidney.[3]
In April 2025, Novartis announced plans to acquire Regulus Therapeutics to advance farabursen's development,[4] with the acquisition completed in June 2025.[5]
Mechanism of action
Farabursen is a next-generation antisense oligonucleotide that works by inhibiting microRNA-17 (miR-17).[3] The drug is designed with preferential kidney distribution, allowing it to target the site of disease more effectively.[1]
MicroRNA-17 normally suppresses the expression of the PKD1 and PKD2 genes, which produce polycystin-1 (PC1) and polycystin-2 (PC2) proteins respectively.[6] These polycystin proteins play a crucial role in regulating kidney cell growth and preventing cyst formation. In ADPKD, mutations in these genes result in reduced protein levels, leading to uncontrolled cyst growth. By inhibiting miR-17, farabursen aims to increase the production of PC1 and PC2 proteins, potentially slowing or halting disease progression.[6]
Clinical trials
Phase 1 studies
A Phase 1 single ascending dose (SAD) study was completed in September 2022.[7] The trial demonstrated that farabursen had a favorable safety and pharmacokinetic profile, with the drug being well-tolerated and no serious adverse events reported. Plasma exposure was approximately linear across the four tested doses.[8][9]
Phase 1b multiple ascending dose study
The Phase 1b multiple ascending dose (MAD) study (NCT05521191) was a double-blind, placebo-controlled trial that evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of farabursen in adult patients with ADPKD.[10]The primary endpoints measured changes in urinary polycystin-1 (PC1) and polycystin-2 (PC2) levels, while exploratory endpoints examined changes in height-adjusted total kidney volume (htTKV) and overall kidney function.[6]
See also
- Autosomal dominant polycystic kidney disease
- Tolvaptan – The current standard of care for ADPKD
- MicroRNA
- Antisense therapy
References
- ↑ 1.0 1.1 "Novartis to acquire Regulus Therapeutics and farabursen, an investigational microRNA inhibitor to treat ADPKD, the most common genetic cause of renal failure" (Press release). Novartis. May 1, 2025.
- ↑ Fang C, Norouzi S, Garimella PS (September 2025). "Therapies in autosomal dominant polycystic kidney disease: beyond tolvaptan". Current Opinion in Nephrology and Hypertension. 34 (5): 368–374. doi:10.1097/MNH.0000000000001101. PMID 40726372 Check
|pmid=value (help). - ↑ 3.0 3.1 "Regulus Therapeutics Announces Successful Completion of its Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of Farabursen (RGLS8429) for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)" (Press release). Regulus Therapeutics. March 27, 2025.
- ↑ "Regulus Therapeutics Enters into Agreement to be Acquired by Novartis AG" (Press release). Regulus Therapeutics. April 30, 2025.
- ↑ "Novartis Completes Acquisition of Regulus Therapeutics" (Press release). Novartis. June 25, 2025.
- ↑ 6.0 6.1 6.2 "RGLS8429 Increases Urinary PC1 and PC2 and May Reduce... : Journal of the American Society of Nephrology". LWW. doi:10.1681/ASN.2024w1p (inactive 6 November 2025). Archived from the original on 29 April 2025. Retrieved 6 November 2025. Unknown parameter
|url-status=ignored (help) - ↑ Regulus Therapeutics Inc. (2025-08-25). A Phase 1, Double-Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Volunteers to Evaluate the Safety, Tolerability, and Pharmacokinetics of RGLS8429 (Report). clinicaltrials.gov.
- ↑ "Regulus Therapeutics Announces Positive Topline Data from the Second Cohort of Patients in its Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)". BioSpace. 2024-03-12. Retrieved 2025-11-06.
- ↑ "Regulus Therapeutics Announces First Patient Dosed in Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)" (Press release). Regulus Therapeutics. November 2, 2022.
- ↑ Regulus Therapeutics Inc. (2025-05-28). A Phase 1b, Double-Blind, Placebo-Controlled, Multiple Ascending Dose and an Open-Label Fixed-Dose Study in Patients With Autosomal Dominant Polycystic Kidney Disease to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of RGLS8429 (Report). clinicaltrials.gov.
External links
- Phase 1b Clinical Trial Information at ClinicalTrials.gov
- Regulus Therapeutics – Official website
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