FDA Events for Regulus Therapeutics (RGLS)
This section highlights FDA-related milestones and regulatory updates for drugs developed by Regulus Therapeutics (RGLS).
Over the past two years, Regulus Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
RGLS8429. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
RGLS8429 - FDA Regulatory Timeline and Events
RGLS8429 is a drug developed by Regulus Therapeutics for the following indication: Autosomal Dominant Polycystic Kidney Disease (ADPKD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RGLS8429
- Announced Date:
- March 27, 2025
- Indication:
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Announcement
Regulus Therapeutics Inc. announced positive topline results from all patients in the fourth cohort in its Phase 1b MAD study of farabursen for the treatment of ADPKD.
AI Summary
Regulus Therapeutics Inc. announced positive topline results from all patients in the fourth cohort of its Phase 1b MAD study for farabursen, a novel treatment for autosomal dominant polycystic kidney disease (ADPKD). In this cohort, 26 patients received a fixed 300 mg dose every other week for three months. The study data showed a significant increase in urinary polycystins PC1 and PC2 levels compared to placebo, confirming the drug’s mechanism of action. Importantly, patients experienced a near halt in the growth of height-adjusted total kidney volume (htTKV) over the four-month period, suggesting that farabursen may slow the progression of kidney enlargement and cyst development. These consistent results support the selected 300 mg dose for optimal kidney exposure and strengthen confidence as the company moves toward a pivotal Phase 3 trial set to begin in the third quarter of 2025.
Read Announcement- Drug:
- RGLS8429
- Announced Date:
- January 29, 2025
- Indication:
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Announcement
Regulus Therapeutics Inc. announced positive clinical and regulatory updates from its ADPKD program.
AI Summary
Regulus Therapeutics Inc. announced positive updates from its ADPKD program. In an interim analysis of the fourth cohort from its Phase 1b Multiple Ascending Dose study, the company observed a clear mechanistic dose response with farabursen. The results showed improvements in biomarker levels related to polycystin proteins and a marked reduction in the growth rate of height-adjusted total kidney volume. These findings indicate that farabursen may help slow down kidney volume expansion in patients with ADPKD. Additionally, the treatment was well tolerated among participants, reinforcing its potential as a safer option. Regulus is encouraged by these outcomes, which build a strong case for advancing the program into a pivotal Phase 3 study. The company’s efforts bring hope for a more effective treatment option for those affected by this challenging kidney disease.
Read Announcement- Drug:
- RGLS8429
- Announced Date:
- October 8, 2024
- Indication:
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Announcement
Regulus Therapeutics Inc. announced it has completed enrollment of 26 patients in the fourth cohort in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD.
AI Summary
Regulus Therapeutics announced that it has successfully enrolled 26 patients in the fourth cohort of its Phase 1b MAD study for RGLS8429, a potential treatment for autosomal dominant polycystic kidney disease (ADPKD). In this cohort, patients are receiving a fixed dose of 300 mg administered every other week over a three‐month period. This milestone comes as earlier cohorts demonstrated promising trends, including a mechanistic dose response evident through increased urinary polycystins (PC1 and PC2) and reductions in height adjusted total kidney volume (htTKV), an important indicator of disease progression. The company is optimistic about these early results and plans to share topline data from the fourth cohort in early 2025. Regulus also aims to discuss further development steps in an End of Phase 1 meeting with the FDA scheduled by the end of 2024.
Read Announcement- Drug:
- RGLS8429
- Announced Date:
- June 24, 2024
- Indication:
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Announcement
Regulus Therapeutics Inc. announced positive topline results from the third cohort of patients in its Phase 1b MAD study of RGLS8429 for the treatment of ADPKD.
AI Summary
Regulus Therapeutics Inc. announced positive topline results from the third group of patients in its Phase 1b MAD study of RGLS8429 for treating autosomal dominant polycystic kidney disease (ADPKD). In this cohort, 16 subjects received 3 mg/kg of RGLS8429 or placebo every other week for three months. The study showed a clear dose response with statistically significant increases in the urinary biomarkers PC1 and PC2 compared to placebo. Exploratory MRI imaging indicated that 70% of patients treated with the 3 mg/kg dose experienced a reduction in total kidney volume. RGLS8429 was well tolerated with no safety concerns noted. These findings support the ongoing development of a fixed 300 mg dose in an open-label fourth cohort and provide promising evidence that RGLS8429 may effectively target the underlying genetic cause of ADPKD.
Read Announcement
Regulus Therapeutics FDA Events - Frequently Asked Questions
As of now, Regulus Therapeutics (RGLS) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Regulus Therapeutics (RGLS) has reported FDA regulatory activity for RGLS8429.
The most recent FDA-related event for Regulus Therapeutics occurred on March 27, 2025, involving RGLS8429. The update was categorized as "Top-line results," with the company reporting: "Regulus Therapeutics Inc. announced positive topline results from all patients in the fourth cohort in its Phase 1b MAD study of farabursen for the treatment of ADPKD."
Currently, Regulus Therapeutics has one therapy (RGLS8429) targeting the following condition: Autosomal Dominant Polycystic Kidney Disease (ADPKD).
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:RGLS) was last updated on 7/11/2025 by MarketBeat.com Staff
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