Drugs / bitopertin
bitopertin
also known as
RO4917838 · DISC-1459 · Bitopertina · Bitopertine · Paliflutine · R-1678 · RG1678 · RO-4917939
Small molecule targets SLC6A9 via inhibition
Trials 29
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 114 trials · 1 met primary | ||||||
| Phase 1 | NCT07123363 | Jan 2026 → Dec 2035 | erythropoietic protoporphyria | Wake Forest University Health Sciences | Withdrawn | No outcome recorded |
| Phase 1 | NCT02019290 | Feb → Mar 2014 | — | Hoffmann-La Roche | Withdrawn | No outcome recorded |
| Phase 1 | NCT01665976 | Aug → Nov 2012 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01543529 | Feb → Jun 2012 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01495104 | Oct 2011 → Jan 2012 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01438749 | Oct 2011 → May 2012 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01510899 | Oct 2011 → Mar 2012 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01433575 | Sep → Nov 2011 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01356550 | Jun → Oct 2011 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01365403 | Apr → Jul 2011 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01116830 | Nov 2010 → Feb 2014 | schizophrenia | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01183585 | Aug → Sep 2010 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1 | NCT01613040 | Jan → Jul 2009 | — | Hoffmann-La Roche | Completed | Met primary |
| Phase 1 | NCT01636492 | Nov 2005 → Aug 2006 | — | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05828108 | Jul 2023 → Aug 2025 | Diamond-Blackfan anemia | National Heart, Lung, and Blood Institute (NHLBI) | Completed | No outcome recorded |
| Phase 25 trials · 1 met primary | ||||||
| Phase 2 | NCT05308472 | Oct 2022 → Feb 2024 | erythropoietic protoporphyria | Disc Medicine, Inc | Completed | No outcome recorded |
| Phase 2 | NCT03271541 | Oct 2017 → Jun 2018 | beta thalassemia | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 2 | NCT01674361 | Dec 2012 → Apr 2015 | obsessive-compulsive disorder | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 2 | NCT01234779 | Feb 2011 → Sep 2012 | schizophrenia | Hoffmann-La Roche | Completed | No outcome recorded |
| Phase 2 | NCT00616798 | Mar 2008 → Feb 2010 | schizophrenia | Hoffmann-La Roche | Completed | Met primary |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT05883748 | Aug 2023 → Jun 2028 expected | erythropoietic protoporphyria | Disc Medicine, Inc | Enrolling by invitation | No outcome recorded |
| Phase 37 trials · 1 met primary · 5 missed | ||||||
| Phase 3 | NCT06910358 | Apr 2025 → Sep 2026 | X-linked erythropoietic protoporphyria | Disc Medicine, Inc | Completed | No outcome recorded |
| Phase 3 | NCT01235520 | Jan 2011 → Jul 2014 | schizophrenia | Hoffmann-La Roche | Completed | Missed primary |
| Phase 3 | NCT01192867 | Dec 2010 → May 2014 | schizophrenia | Hoffmann-La Roche | Completed | Missed primary |
| Phase 3 | NCT01235585 | Dec 2010 → Sep 2014 | schizophrenia | Hoffmann-La Roche | Completed | Missed primary |
| Phase 3 | NCT01192906 | Dec 2010 → Jul 2014 | schizophrenia | Hoffmann-La Roche | Completed | Missed primary |
| Phase 3 | NCT01235559 | Dec 2010 → Mar 2015 | schizophrenia | Hoffmann-La Roche | Completed | Met primary |
| Phase 3 | NCT01192880 | Nov 2010 → Jul 2014 | schizophrenia | Hoffmann-La Roche | Completed | Missed primary |
| Phase not stated1 trial | ||||||
| — | NCT07603401 | — | X-linked erythropoietic protoporphyria | Disc Medicine, Inc | Available | No outcome recorded |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2026-06-12 | Disc Medicine, Inc | Results Disc Medicine Presents Positive Clinical Updates at the 2026 European Hematology Association (EHA) Annual Meeting discmedicine.com ↗
Updated data from the HELIOS open-label extension trial of bitopertin in EPP, to be presented in a poster session tomorrow, June 13, show sustained reductions in PPIX, significant improvement in light tolerance measures, and favorable longer-term safety in patients treated with bitopertin. |
| 2026-02-13 | Disc Medicine, Inc | Regulatory Disc Medicine Receives Complete Response Letter from FDA for Bitopertin for the Treatment of EPP discmedicine.com ↗ |
| 2025-10-16 | Disc Medicine, Inc | Regulatory Disc Medicine Announces Receipt of FDA Commissioner’s National Priority Voucher (CNPV) for Bitopertin in Erythropoietic Protoporphyria (EPP) discmedicine.com ↗
today announced that it received a Commissioner’s National Priority Voucher (CNPV) from the U.S. Food and Drug Administration (FDA) for bitopertin in erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). |
| 2025-09-30 | Disc Medicine, Inc | Regulatory Disc Medicine Announces Submission of New Drug Application (NDA) to US FDA for Accelerated Approval of Bitopertin for Patients with Erythropoietic Protoporphyria (EPP) discmedicine.com ↗
Disc submitted the NDA under the FDA’s accelerated approval pathway using reduction of protoporphyrin IX (PPIX) as a surrogate endpoint and requested a Priority Review based on bitopertin’s potential to address the significant unmet need for EPP patients. |
| 2025-06-12 | Disc Medicine, Inc | Results Disc Medicine Presents Positive Clinical Data Updates Across Portfolio at the European Hematology Association (EHA) 2025 Annual Congress discmedicine.com ↗
This year’s presentations included data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers. |
| 2024-11-04 | Disc Medicine, Inc | Regulatory Disc Medicine Announces Successful End of Phase 2 Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria (EPP), Including Potential for Accelerated Approval discmedicine.com ↗
Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced positive feedback from its end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), supporting the regulatory path forward for bitopertin in EPP. |
| 2024-06-14 | Disc Medicine, Inc | Results Disc Medicine Presents Positive Clinical Data Across Portfolio at the European Hematology Association (EHA) 2024 Congress discmedicine.com ↗
Updated analyses of data from AURORA and BEACON studies further demonstrate clinical activity of bitopertin across multiple measures of erythropoietic protoporphyria (EPP) and support development path forward |
| 2024-04-01 | Disc Medicine, Inc | Results Disc Reports Topline Results from Phase 2 AURORA Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP) discmedicine.com ↗
Treatment with bitopertin resulted in statistically significant reductions in PPIX, the primary endpoint, and significant improvements in the rate of phototoxic reactions with pain and the Patient Global Impression of Change (PGIC). |
| 2023-12-11 | Disc Medicine, Inc | Results Disc Presents Positive Updated Results from Phase 2 BEACON Study of Bitopertin and Other Programs at the 65th American Society of Hematology (ASH) Annual Meeting discmedicine.com ↗
The updated data are consistent with and confirm the initial positive results presented in June, demonstrating significant decreases in PPIX, robust and consistent improvements in sunlight tolerance across all study measures, including the precedented pivotal endpoint, and improvements in patient quality of life. |
| 2023-06-09 | Disc Medicine, Inc | Results Disc Presents Positive Initial Data from Phase 2 BEACON Trial of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP) at European Hematology Association (EHA) 2023 Congress discmedicine.com ↗
The initial trial data demonstrated consistent decreases in PPIX, significant increases in reported sunlight tolerance and improvements in measures of patient quality of life. |
| 2022-12-27 | Disc Medicine, Inc | Regulatory Disc Medicine Receives FDA Orphan Drug Designation for Bitopertin for the Treatment of Erythropoietic Protoporphyria discmedicine.com ↗
Disc Medicine, Inc. (“Disc”), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced today that the U.S. Food and Drug Administration (“FDA”) granted Orphan Drug Designation to bitopertin for the treatment of erythropoietic protoporphyria (“EPP”). |
| 2022-12-13 | Disc Medicine, Inc | Results Disc Medicine Announces Several Presentations Across Hematology Portfolio at the 64th American Society of Hematology Annual Meeting discmedicine.com ↗
We believe these data suggest that bitopertin may be disease-modifying and may be effective in treating the photosensitivity and modifying the hepatoxicity in EPP patients |
| 2022-11-03 | Disc Medicine, Inc | Results Disc Medicine Announces Multiple Presentations Across Hematology Portfolio at the 64th American Society of Hematology Annual Meeting discmedicine.com ↗
Bitopertin, a Selective Glycine Transporter 1 Inhibitor, Reduced PPIX Level and Improved Liver Fibrosis in a Mouse Model of Erythropoietic Protoporphyria (EPP) |
All press releases naming this drug 27 releases
Evidence & citations 13 cited values
Every value below carries the sentence it was read from. 32 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | bitopertin | “OBJECTIVE: To determine the efficacy and safety of bitopertin (RG1678), a glycine reuptake inhibitor, in patients with schizophrenia and predominant negative symptoms who were...” PMID 24696094 ↗ Jun 2014 |
| Known as | Bitopertina | ChEMBL registry synonym — accepted as the source's own label CHEMBL1171829 ↗ |
| Known as | Bitopertine | ChEMBL registry synonym — accepted as the source's own label CHEMBL1171829 ↗ |
| Known as | DISC-1459 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05883748 ↗ |
| Known as | Paliflutine | ChEMBL registry synonym — accepted as the source's own label CHEMBL1171829 ↗ |
| Known as | R-1678 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1171829 ↗ |
| Known as | RG1678 | “OBJECTIVE: To determine the efficacy and safety of bitopertin (RG1678), a glycine reuptake inhibitor, in patients with schizophrenia and predominant negative symptoms who were...” PMID 24696094 ↗ Jun 2014 |
| Known as | RO-4917939 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1171829 ↗ |
| Known as | RO4917838 | “This randomized, double-blind, placebo- and active-controlled, parallel group study will evaluate the safety and efficacy of RO4917838 (bitopertin) in patients with acute...” NCT01234779 ↗17“A Study of RO4917838 (Bitopertin) in Patients With Sub-optimally Controlled Symptoms of Schizophrenia (WN25306)” NCT01235585 ↗ “A Study of RO4917838 (Bitopertin) in Patients With Persistent, Predominant Negative Symptoms of Schizophrenia” NCT01192906 ↗ “bitopertin [RO4917838] level 1” NCT01235559 ↗ |
| Action | Inhibit | “Glycine transporter 1 inhibitor” CHEMBL1171829 ↗1“a glycine transport inhibitor” PMID 28318835 ↗ Mar 2017 |
| Modality | Small molecule | — CHEMBL1171829 ↗ |
| Route | Oral | “placebo 10mg, 30mg, or 60mg of RO4917838 once daily, p.o., as add-on therapy” NCT00616798 ↗ |
| Target | SLC6A9 | “Glycine transporter 1 inhibitor” CHEMBL1171829 ↗ |