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Also known as Bitopertin, RO4917838
DISC-1459 · 3 trials · 3 indications
The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.
Percent change from baseline in PPIX concentration was analyzed using a mixed model repeated measures analysis in the ITT population.
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | - |
| DISC-1459 oral dose | EXPERIMENTAL | - |
| DISC-1459 Oral Dose Level 1 | EXPERIMENTAL | Oral dose, once a day |
| DISC-1459 Oral Dose Level 2 | EXPERIMENTAL | Oral dose, once a day |
| Name | Type | Description |
|---|---|---|
| Placebo | DRUG | Oral dose, once a day for 24 weeks |
| DISC-1459 | DRUG | Oral dose, once a day for 24 weeks |
Inclusion Criteria: 1. Aged 12 years or older at the time of study consent. 2. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis. 3. Minimum daily Sun Exposure Diary compliance ≥85% on...
DISC-1459 is being developed for the treatment of erythropoietic protoporphyria (EPP), a rare inherited disorder that causes severe skin pain on exposure to sunlight. Clinical trials have also enrolled patients with X-linked protoporphyria (XLP), a related condition. It is an investigational small molecule and has not been approved by the FDA.
DISC-1459 targets GlyT1, a glycine transporter. By inhibiting GlyT1, the drug is designed to modulate glycine availability and reduce the accumulation of protoporphyrin IX (PPIX), the toxic heme intermediate that builds up in erythropoietic protoporphyria and drives photosensitivity and skin pain.
DISC-1459 is being developed by Disc Medicine, Inc., which trades on the Nasdaq under the ticker IRON. The company is advancing the small molecule through late-stage clinical development for erythropoietic protoporphyria and related protoporphyrias.
DISC-1459 is in Phase 3 development. The pivotal Phase 3 APOLLO trial in erythropoietic protoporphyria and X-linked protoporphyria has completed, and a long-term extension study continues to enroll participants by invitation. The drug remains investigational and has not received FDA approval.
Three clinical trials of DISC-1459 are registered. The Phase 3 APOLLO study (NCT06910358) enrolled 183 participants with EPP or XLP and is completed. The Phase 2 HELIOS long-term extension (NCT05883748) is enrolling by invitation, and an earlier Phase 2 study (NCT05308472) enrolled 75 participants and is completed.
Yes, DISC-1459 is the development code for bitopertin, the same small molecule studied by Disc Medicine in erythropoietic protoporphyria and X-linked protoporphyria. The two names refer to the identical investigational drug, so trial records and publications using either name describe the same asset.