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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, also known as bitopertin, is an investigational small molecule being developed for erythropoietic protoporphyria (EPP), a rare genetic disorder characterized by severe photosensitivity. It is also being studied in X-linked protoporphyria (XLP). The drug is currently in Phase 2 and Phase 3 clinical trials.
DISC-1459 is a small molecule that targets the glycine transporter GlyT1. By inhibiting this transporter, it aims to reduce the production of protoporphyrin IX (PPIX), the photosensitizing molecule that accumulates in patients with erythropoietic protoporphyria and causes painful skin reactions to light.
DISC-1459 is being developed by Disc Medicine, Inc., a biopharmaceutical company focused on hematologic diseases. The company's stock trades under the ticker symbol IRON on the NASDAQ exchange.
DISC-1459 is in Phase 2 and Phase 3 clinical development. It has completed a Phase 2 trial (NCT05308472) and is currently in an open-label extension study (NCT05883748). A Phase 3 trial called APOLLO (NCT06910358) is active but not recruiting participants.
DISC-1459 has been studied in three clinical trials. A completed Phase 2 trial (NCT05308472) evaluated safety and PPIX concentrations in 75 adults with EPP. The HELIOS extension study (NCT05883748) is enrolling 230 participants with EPP or XLP. The Phase 3 APOLLO trial (NCT06910358) is active with 183 participants.
Yes, DISC-1459 is the same as bitopertin. The drug is referred to by both names in clinical trial records. Bitopertin was originally developed for other indications but is now being investigated by Disc Medicine for erythropoietic protoporphyria and X-linked protoporphyria.